California Healthcare Institute

Event Spotlight: CHI Unveils CEO Survey Results at J.P. Morgan Healthcare Conference

2012-01-13T16:33:00.000-08:00

The J.P. Morgan Healthcare Conference marks the start of each year for the biomedical community.

This year’s 30th annual meeting drew 8,000 attendees to the San Francisco area where biomedical companies set up shop inside the Westin St. Francis Hotel to showcase their latest research and unveil strategies for the year ahead. In attendance were members of the investment community, global industry leaders, technology innovators and media.

CHI highlighted results of its CEO Survey during a press conference attended by news outlets including Nature, BioWorld Today, Chemical & Engineering News and San Francisco Business Times, among others. The press briefing featured a panel of biomedical industry executives from Omniox, NuVasive and Theravance, who each shared their unique experience with raising capital, advancing important biomedical research and working with the U.S. Food and Drug Administration. CHI President and CEO David Gollaher, Ph.D., moderated the panel alongside BayBio President and CEO Gail Maderis and PwC National Life Sciences Partner Tracy Lefteroff.

Findings of the CEO Survey reflect issues discussed throughout the biomedical industry and provide an early glimpse into the 2012 California Biomedical Industry Report, due in February. The report, published annually by CHI, BayBio and PwC, provides a snapshot of the biomedical industry in California, the largest biomedical cluster in the world and the source of the greatest number of products in clinical development.

Among the notable findings were:

• Nearly three quarters (74 percent) of biomedical industry CEOs surveyed said their companies have had to delay a research or development project in the past year.

• Lack of funding was the top reason for project delays cited by private company CEOs, and accounted for more than one-third (40 percent) of delays by all public and private companies in the survey.

• Eight in 10 CEOs surveyed agreed or strongly agreed that the current FDA regulatory approval process has slowed the growth of their organization.

Additionally, 80 percent of CEOs surveyed do not believe that U.S. FDA has the best regulatory approval process in the world, and three-quarters believe that within five years, another country could conceivably recreate the ecosystem that has made the U.S. the leading biomedical region in the world.

“Sound public policy and managerial and operational improvements at FDA, along with responsible congressional oversight, will encourage biomedical innovation and, ultimately, job growth here in California,” Gollaher said. “Working collaboratively with other stakeholders, Congress, FDA and the biomedical industry can maintain the high standards of safety and effectiveness that address patients’ need, while improving our ability to attract investment and grow in 2012 and beyond.”

The survey was conducted in November 2011 and targeted approximately 100 pharmaceutical, biotechnology, medical device, diagnostics or medical equipment companies that conduct business in California.

Thank you to everyone who was able to attend or call into our CEO Survey results briefing! We will unveil the full 2012 California Biomedical Industry Report at BioMed Innovation Night, happening Feb. 8 at the Sacramento Memorial Auditorium. Register here for the event and check back with us at http://www.chi.org/ on Feb. 8 for full report details.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Igor Gonda, Ph.D., President and CEO, Aradigm Corp.

2011-12-09T08:46:00.000-08:00

Gonda, Ph.D., joined Hayward, Calif.-based specialty pharmaceutical company Aradigm Corp. from Genentech, where he led a team focused on developing inhalation products for severe respiratory disease. Today, Gonda leads Aradigm scientists on a mission to revolutionize the quality of life of patients with severe pulmonary disease. The Aradigm team pioneered the AERx iDMS pulmonary delivery of insulin that was used in Phase 3 clinical trials conducted by Novo Nordisk. Now, Aradigm is uniquely positioned to develop a portfolio of its own products to treat patients with severe respiratory diseases. Current activities include development programs addressing the treatments of bronchiectasis, cystic fibrosis, inhaled bioterrorism infections and smoking cessation.

Q: Share with me a short history of Aradigm.

A: Aradigm was a venture-founded company and we went public in 1996. We were at one point a very large company with about 400 employees and we had very large manufacturing operations focused on the development of pain management therapies and inhaled insulin. Then, the inhaled insulin program was taken over by Novo Nordisk. The deal, however, was later terminated. The company completely changed its view of how we should operate.

So, now, we are mostly virtual. We kept the intellectual property and the know-how of the company and, now, have 12 people. The core of the company is basically the R&D group that I brought with me from Genentech. We also brought a medical director who was a former Roche employee. He was working on the same Genentech product that we were working on, which is the first modern drug that was approved for cystic fibrosis patients.

Q: What are some of the limitations in lung disease therapies that Aradigm is addressing today?

A: The common denominator, particularly for cystic fibrosis, bronchiectasis and chronic obstructive pulmonary disease, is that the severity of the disease rapidly grows when these people get an infection with a particular microorganism called Pseudomonas aeruginosa. There has been a strong association between severity of the disease and reduced life expectancy and a person’s colonization with this microorganism. About 80 percent of adult cystic fibrosis patients are colonized with Pseudomonas aeruginosa.

There are two products approved, two inhalation antibiotics, for the treatment of Pseudomonas aeruginosa patients in cystic fibrosis. The problem that these patients with cystic fibrosis have is that they are spending enormous amount of time just treating their disease, and, because they are born with the disease, typically, for many years, it is not just a job for the patient, but it is the job for the families who look after them and some of these families have more than one cystic fibrosis child. So, it is a huge burden that the disease imposes on the patient and on the family.

Our idea, initially, was very simple and that was to provide cystic fibrosis patients with a once-daily inhaled antibiotic. One of the antibiotics that is approved is twice-a-day and the other one is three times a day.

Q: Talk a little about how you became involved in developing treatments for bronchiectasis (brong-ke-EK-ta-sis), a condition in which damage to the airways causes them to widen and become flabby and scarred?

A. This is a program we feel particularly proud about because it is a totally unmet medical need and we seem to be paving the way to provide something for the patients who have not been able to get satisfactory treatment.

This is a disease where inhaled antibiotics have so far failed because the problem with treating the pulmonary Pseudomonas aeruginosa infections of these patients was that many of them did not tolerate the inhaled antibiotics well.

Q: What does the competitive landscape look like for inhaled antibiotics?

A: There is another company developing a once-daily inhaled antibiotic for cystic fibrosis. They were put on clinical hold so I’m not sure if and when they will be able to proceed. Ours is a different class of antibiotics. Our antibiotic is also a broad-spectrum antibiotic so we actually can go after some of the other bacteria, both gram negative and gram positive bacteria, as opposed to just going specifically after Pseudomonas aeruginosa.

Q: Talk about your tobacco smoking cessation product and how that fits into your product pipeline.

A: The most common cause of respiratory disease is smoking. Smoking causes more healthcare damage than malaria, tuberculosis and HIV put together. In the United States, roughly about 1,200 people a day die from smoking-related diseases.

We suspected that if we emulate the pharmacokinetics of cigarettes, that is, if we deliver nicotine deep in the lungs and it will then absorb quickly into the pulmonary artery which feeds the brain, that it will have a profound impact on the craving for cigarettes. So, that is exactly what we have done. We dissolved nicotine in a small amount of water and put it into a little inhaler that was developed for deep lung delivery. Most of the original investment in this inhaler technology was for pain management — to achieve quick relief of pain — which we developed about a decade ago.

And we found exactly what we suspected: In a single breath, single inhalation of pure nicotine dissolved in water, you can get very rapid entry of the nicotine into the bloodstream and an immediate and sustained impact on craving for cigarettes. So, we are very excited about looking for a partner for this program.

Q: We recently saw the FDA move to control electronic cigarettes. What do you anticipate will be some of your regulatory hurdles in terms of tobacco cessation?

A: We are not sure whether our product will be controlled as a tobacco product, and, therefore will need a very different path to acceptance by the regulatory authorities, or whether the FDA would prefer us to develop it as a prescription product, and, which, initially, a doctor would prescribe it because a lot of these nicotine replacement products eventually become over-the-counter products that do not require prescription.

Q: What are some of your other goals for the next year or two?

A: We’d like to bring Pulmaquin — the inhaled antibiotic — to the patients with bronchiectasis and cystic fibrosis as fast as possible. So, that is what our company is focusing on. And, in the background to that, we are looking for a partner that would be able to do global developing and marketing of the smoking cessation product.

Q: Are you looking for those partnerships now?

A: We are seeking partnerships for the inhaled antibiotic and for smoking cessation. Partnerships bring valuable expertise and resources to the company.

CHI-Advancing California biomedical research and innovation

Speaker Spotlight: Marc Boutin, Executive Vice President & COO, National Health Council

2011-10-25T12:13:00.000-07:00

Marc Boutin

The National Health Council brings together all segments of the healthcare community to provide a united voice for the more than 133 million people with chronic diseases and disabilities and their family caregivers.
Boutin has been actively involved in health advocacy, policy, and both federal and state legislation throughout his career. He has designed and directed advocacy strategies for legislative initiatives, which have included issues ranging from access to healthcare to cancer prevention. Before joining the Council, Boutin served as the vice president of government relations and advocacy at the American Cancer Society for New England. In addition, he was a faculty member at Tufts University Medical School, where he lectured on healthcare policy.

At the CHI 2011 Annual Meeting, to be held Nov. 3 in San Francisco, Boutin will discuss the Moddern Cures Solution, a proposal to update the U.S. regulatory system to remove barriers to innovation and provide greater predictability in the search for answers to the nation’s unmet medical needs. It is not too late to register.

Q: Let’s talk about the impetus for the National Health Council. What was the driving need behind its formation?
A: The National Health Council was formed in 1920, so we have a long history. We were formed by a group of about 20 patient organizations that was looking for a trade association.

Q: Were there any other large trade associations of its kind in existence?
A: No, and there currently is nothing out there. We still serve in that function, so half our work is really directed at member services. Our work includes anything from board management, governance, corporate structures, volunteers, income development and benchmarking studies. We still maintain what is called the standards of excellence. The standards are a set of nonprofit standards, essentially, that the patient groups have to meet in order to be in membership. They are actually the most strident nonprofit standards in the industry.

Q: How is your organization funded?
A: We are a dues-based organization so about half our income comes from dues and the other half comes from sponsorships.

Q: Can you give me an example of some of the leading patient advocacy groups that make up your membership?
A: Some of the original founders include American Cancer Society, American Heart, American Diabetes and we run the gamut from those very large groups such as Easter Seals to small groups that represent various disorders like Sjögren’s disease or Alpha-1.

Q: What unique function does your group serve?
A: We are able, through our process, to engage the community, create a common understanding and then a common platform from which to advocate on systemic issues. And when they come together, they tend to have a great deal of impact.

Q: What are some of those messages you are working to convey through this organized effort?
A: In terms of public policy, we really are focused on two different things. One would be access to care, so we’ve been heavily involved with the implementation of health reform in a variety of different initiatives right down to the level of helping craft part of the regulatory language, as well as the legislative language, that created the Affordable Care Act. So, for example, we worked on defining the essential health benefits the Secretary of Health and Human Services is working on. We did our own actuarial analysis of what it would mean to put certain benefits into the design. We did recommendations on regulatory language.

On the other hand, we are extremely interested in development of new treatments, and so there is the dual focus.

Q: Tell me a little about your latest effort, the Moddern Cures Solution, which stands for Modernizing Our Drug and Diagnostics Evaluation and Regulatory Network?
A: We are promoting legislative language that will do two things. It will, one, allow a company to bring a medicine to market without a patent, and that is significant. A lot of people don’t understand, but when somebody identifies a new potential medicine in the early pre-clinical phases, it typically will go to a company that could develop and commercialize that product and bring it to U.S. Food and Drug Administration approval. In 80 percent to 90 percent of those cases, new potential treatments are dismissed not because the science is not good, but because they have weak patents.

It just makes no sense from a patient perspective, and this legislation corrects that. It says if you’ve got good science, you can bring it to market, receive a return on your investment, and still allow a generic to enter at a predetermined time.

Q: Does this proposed change include medical devices?
A: That is the second half of the legislation. There are mechanisms that address specific issues such as creating the incentives to invest in the device/diagnostic arena.

Q: Is this modeled after any other country’s system?
A: No, and actually these initiatives were identified by the chief medical officers of the patient groups. In other words, they came to the council about five years ago and said, “We’ve doubled the investment in the development of new treatments, and we are not getting double the output of new treatments.” And they start to look at the lifecycles of the development of treatments and diagnostics and identify barriers.

And then asked us to look at possible solutions and we settled on these four major issues and developed solutions that we think could work, but are not actually modeled on existing solutions. We are, however, already seeing Europe and other countries look at this as a potential metric that they would copy.

Q: What has the reception been like so far from the policy side?
A: Extremely positive. It has been described by some as one of the few major health policies that has bipartisan support and the current Congress and administration could actually enact. It is exciting.

Q: What kind of dialogue do you hope to get out of the 2011 CHI Annual Meeting?
A: I am hoping, given the audience that will be at that meeting, that there will be a great deal of synergy on what we are doing on the development of new treatments and how it will positively impact the environment that your audience is working and living in.

I figured it is an incredible event, and I was really pleased to be invited to participate.

CHI-Advancing California biomedical research and innovation

Event Spotlight: CHI Co-Hosts Congressional Briefing on Infection Prevention

2011-10-18T07:02:00.000-07:00

On Oct. 12, CHI and MassBio co-hosted a congressional briefing in Washington D.C. titled, “Infection Control: Detection, Prevention, and Treatment.” Participants were joined by Reps. Jackie Speier (D-CA) and Edward Markey (D-MA). The partnership was hailed as a powerful combination of two of the most significant state powers in the biotech industry. As a father of a child suffering from cystic fibrosis, MassBio President and CEO Bob Coughlin said passionately, “I don’t care if [cystic fibrosis] is cured in California or Massachusetts. All boats rise.”

Coughlin and CHI President and CEO, David L. Gollaher, Ph.D, made opening remarks and introduced Reps. Speier and Markey, who each made their own comments. Next, a panel of experts provided educational presentations on challenges and advances in combating healthcare-associated infections (HAIs). The program, held in the Gold Room of the Rayburn House Office Building, attracted more than 50 congressional staffers, patient advocates, and industry representatives.

Speakers outlined the scope and effect of HAIs. Estimates show that HAIs take the lives of 100,000 patients a year – patients who enter healthcare facilities in good health, or without such infections yet die from exposure in healthcare settings. As Speier noted, “That’s the equivalent of 333 [Boeing] 747 airplanes crashing every year.” This makes HAIs the 10th leading cause of death, and the No. 1 cause of death in non-cardiac intensive care units. In the U.S. alone, antibiotic-resistant infections are responsible for $20 billion in excess healthcare costs, $35 billion in societal costs and $8 million in additional hospital days.

Despite these numbers, HAIs remain a dangerously under-recognized threat. Continuing her airplane metaphor, Speier said, “If that many planes were crashing every year, we’d expect the FAA to do something about it.” Dr. Arnold Huang, Ph.D., of Thermo Fisher Scientific, later polled the audience to demonstrate the high degree of recognition of October as breast cancer awareness month. He juxtaposed those numbers with the audience’s lack of knowledge about sepsis – a condition that causes four to five times as many deaths each year.

Despite the high death rate and the continued appearance of new strains of antibiotic-resistant bacterium (such as MRSA), the number of new antibiotics in the development pipeline has been shrinking during the past several decades. Rick Winningham, CEO of Theravance, described the trends that have led to diminished approvals of new antibiotics. Overall, “antibiotic drug development is dying,” he said. He attributed this to market dynamics which have led large pharmaceutical firms to exit the market entirely, leaving small firms to overcome tremendous obstacles. These dynamics include: decreases in expected returns on investment; rising costs of clinical trials and the number of required patients; and regulatory challenges and delays from the FDA. Winningham laid responsibility for these on both Congress – for having taken the FDA to task over past high-profile safety issues – and industry itself, for problems in effectively pricing previous products.

Speakers ended the day by noting the promising introduction of the Generating Antibiotic Incentives Now Act (GAIN), and giving a call to action. The GAIN Act would give developers of select antibiotic products five or more years of additional data exclusivity and provide for priority review of their products by the FDA among other provisions. Despite this positive development, participants like Kathy Warye of Becton Dickinson stressed a fundamental challenge to inspiring advocacy to address HAIs: Because they strike and kill patients relatively quickly and are not long-term chronic diseases, it’s impossible to put a face on the problem in the way that advocates for other diseases and conditions have succeeded in doing. Further, the public fails to relate deaths from these sorts of HAIs to our lack of effective antibiotics. As Winningham said, “Leadership has to drive this change.”

CHI-Advancing California biomedical research and innovation

Guest Blog: Fish & Richardson Attorneys Explore European Patent System

2011-10-10T10:39:00.000-07:00

Steven Carlson

Germany is a top country for patent enforcement. Knowing the German system is fundamental for technology companies that sell products in Europe, both for patentees and for companies pulled into German courts as defendants.

Alexander Harguth

In Germany, patent cases frequently take less than a year, at a fraction of the cost of U.S. cases.

A tremendous advantage to patentees is that infringement cases are tried separately from challenges to the validity of a patent. Likewise, many of the procedural “outs” that defendants enjoy in the United States (such as motions to dismiss, early claim construction rulings, and summary judgment proceedings) are unavailable in Germany — accordingly, the first time the parties appear in court is typically for the trial on the merits. Defendants, therefore, must be ready to act quickly to develop their case, to prepare their validity challenge, and to pursue other available defenses.

Customs actions provide another way to enforce patents. Customs authorities may seize goods upon the patentee’s request, after which the case may be referred to the trial courts. Therefore, potential infringers must be aware, for example, when presenting goods at a trade show, there could be a seizure action carried out on the floor of the exhibition.

German damages awards are typically lower than in the United States. Nonetheless, there may be nowhere better for winning an injunction if the patentee has strong patent rights. Although infringement relief will be limited to Germany’s borders, being enjoined from selling in Europe’s biggest market may be intolerable for many companies. A German enforcement strategy, or a parallel strategy of enforcement in Germany and the United States, may, therefore, result in a global settlement of disputes.

We have written a new book on the German IP system: “Patents in Germany and Europe: Procurement, Enforcement and Defense.” As native attorneys of Germany and the United States, we have tried to present Germany’s system to the U.S. audience in an accessible and authoritative handbook, including chapters on:

• Sources of law in Germany;

• The German court system;

• Procurement of IP rights in the German and European patent offices;

• Invalidity challenges through oppositions and nullity actions;

• Enforcement actions through infringement trials and customs proceedings; and

• Employee rights in inventions.

Moreover, key provisions of German and European law are provided in the appendices, as well as sample pleadings, ending with a list of key distinctions between U.S. and German/European law.

The book is available on Amazon.com. If your company has particular interest in the German system, we will be happy to provide a complimentary copy along with an in-person seminar where feasible. Please feel free to us contact directly, at Harguth@fr.com or Carlson@fr.com.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Mitchell Kronenberg, President and Chief Scientific Officer, La Jolla Institute for Allergy & Immunology

2011-09-27T11:35:00.000-07:00

Kronenberg is an internationally recognized scientist and one of the most highly cited immunologists in the world. He was appointed president of La Jolla Institute in September 2003. In addition to his executive duties, he serves as chief scientific officer and conducts an active research program. He received his doctorate from the California Institute of Technology in 1983, and stayed on to complete his postdoctoral work before joining the faculty of the UCLA School of Medicine in 1986. At UCLA, he rose through the ranks to full professor. In 1997, he came to La Jolla Institute to head the division of developmental immunology, a position he held for 14 years, before stepping down recently to devote more time to his duties as president and chief scientific officer.

Over the years, Kronenberg has received many major awards, most recently a prestigious Merit Award for scientific achievement from the National Institutes of Health. He has also been a Burroughs Wellcome Fund Visiting Professor at Harvard University and recently was asked to deliver the Joseph S. Ingraham immunology lecture at the Indiana University School of Medicine.

Q: Tell me a little about the institute and its most significant milestones. How did it first get started?
A: The La Jolla Institute for Allergy and Immunology was established in 1988 by a coalition of leaders from academia and industry who envisioned a unique and dynamic partnership that would lead to breakthroughs in the understanding of the immune system and improvements in human health. We began our laboratory operations in 1989 with the arrival of two pioneering immunologists: Kimishige and Teruko Ishizaka. They were M.D./Ph.D.s from Johns Hopkins University renowned for their seminal discovery about the molecular origins of allergies. They started us down a path of scientific excellence that has included major discoveries on a number of immune-mediated diseases over the years. Among these are autoimmune diseases, like type 1 diabetes and Crohn’s disease, and cancer and heart disease, where the role of the immune system and inflammation is becoming increasingly appreciated. Of course, the immune system is highly protective, and we also have important findings related to vaccine development and infectious diseases such as H1N1 influenza.

Our significant progress is evidenced by the fact that we were recently ranked by Thomson Reuters among the top five organizations worldwide for research impact in immunology. In addition, five potential therapies, based on our institute’s discoveries, are currently in the pharmaceutical pipeline.

Q: Why was it important to dedicate an institute to exploring the body’s defense system?
A: The immune system goes to every part in your body to defend your body and we know you can’t live without your immune system. If you’re highly immunocompromised, you die rapidly from infection. And we also know that vaccination is probably the most effective public health measure we have ever found. So, immunology is very important in doing good in our bodies, and, yet, it also can do bad by causing inflammation leading to autoimmune diseases. There are about 83 known autoimmune diseases. There are probably other inflammatory conditions not classified as autoimmune, in which the immune system plays a role, including atherosclerosis. So, the immune systems does great things to keep us alive every day, and is a great target in terms of vaccinations for keeping us healthy, in other words, giving us more immunity.

However, sometimes the immune system is irrationally exuberant, if I can quote Alan Greenspan. It could begin attacking the pancreas, causing type 1 diabetes or early onset diabetes. It could be attacking the nerves and you get multiple sclerosis. It could be contributing to arthritis. I already mentioned atherosclerosis and many, many more diseases.

We think the immune system, aside from its inherent intellectual fascination, has the greatest implications for health and disease of people. If you’re going to form an institute and dedicate it to one area of biomedical research, it seems to me that immunology would be a very good one because of its wide implications for human health and disease.

Q: We are in a unique position in San Diego as one of the clusters of biomedical research, including early-stage academic research. Talk about some of your industry/academic collaborations on important science.
A: We have a very interesting, long-term partnership with a Japanese pharmaceutical corporation called Kyowa Hakko Kirin. Our partnership actually started with Kirin many years ago, a name that may sound familiar to you because they also make Kirin beer. The current organization was formed by the merger of Kyowa Hakko and Kirin Pharma in 2008, and it is a moderately large pharmaceutical company in Japan. They also have 45 people who work in the building here. Our census is about 350 for our institute. It’s like having an endowment in the sense that every lab, every year gets money to buy equipment and engage in projects of their choosing. In return, and within certain limitations, the company gets first rights to negotiate for our intellectual property and they also enjoy the ease of collaboration by co-locating with us. There are two projects that originated from this institute that are in clinical development with Kyowa Hakko Kirin and others that are in preclinical research.

Q: How else is the institute funded?
A: We’re dependent on NIH for about 75 percent of our annual revenues and every year we’ve had increases in our NIH funding. No. 2 is our contract research agreement with Kyowa Hakko Kirin, which gives us about 13 percent of our annual budget. Other sources include philanthropy contributions and also grants from other agencies such as the Juvenile Diabetes Research Foundation, Crohn’s & Colitis Foundation of America, American Cancer Society and other private agencies that support biomedical research, as well as our licensing income.

Q: Talk about your new Center for RNAi Research, and what you hope to accomplish.
A: We received a large NIH grant in 2010, in a very competitive process, to set up a center for RNA interference (RNAi) screening. We opened the center in August. RNAi is a young and very powerful technology that we will use to try and understand what each gene in our genome does. The center will incorporate robotics and high-throughput screening methods and will have particular expertise in the use of real-life experimental models of disease.

The grant pays for four projects that relate to the immune system, but we don’t want the facility to be confined only to immune system work. We intend to explore genetic questions that will be important in disease processes of all kinds. In addition to our own research, we want it to be open to the academic groups on the Torrey Pines Mesa and to the universities. There will be a fee-for-service arrangement. We hope to have all kinds of projects. We even hope to expand the size and scope of this facility.

Q: When you aren’t spending your time at work, what are you doing?
A: I like to spend time with my family. My wife is also a busy scientist here and we have three sons, two of whom are grown and attending universities and one who lives with us still. I lift weights one day a week and run a couple days a week and I read compulsively. I was recently reading a book called “The Women” by T.C. Boyle and it’s actually a biography of Frank Lloyd Wright. I have been reading another book that’s called “Let the Great World Spin” by Colum McCann and it won the Man Booker Prize. It’s a wonderful novel about intersecting lives, Irish immigrants, people in the ghetto in New York City. It is a fantastic story.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Daniel Laser, CEO and President, Wave 80 Biosciences

2011-08-24T08:21:00.000-07:00

Daniel Laser

Wave 80 Biosciences Inc. develops next-generation molecular diagnostics for infectious disease, cancer, autoimmune disease and other human health conditions. The company began operations in 2003, with a small team of engineers, biologists, and chemists working out of warehouse space in San Francisco’s Dogpatch neighborhood to develop high-performance, cost-effective technologies for processing complex fluid samples.

In 2007, the company initiated a development program to address emerging needs for viral load monitoring to accompany expanding access to antiretroviral therapy among the estimated 30 million people living with HIV in low- and middle-income countries. The first versions of the EOSCAPE-HIV system are scheduled to enter beta testing in clinics in Kenya and South Africa in 2012.

President and CEO Daniel Laser spoke with the CHI Blog to explain how Wave 80, a name born of an anagram, has contributed to improving tests that treat for both infectious and noninfectious diseases, including hepatitis C, hepatitis B, cardiovascular disease, autoimmune disease and cancer.

Q: How did the company get a name like Wave 80 Biosciences?

A: My wife and I came up with it sitting at the Top of the Mark in 2003 while applying for the first grant application that got the company started. It is actually an anagram that includes the name of the chemist whose discovery of electro-osmosis led to one of our seminal patented technologies, an architect whose work inspired certain structural aspects of our product’s design, and the word “silicon,” which is the basic element of our key technology components. My wife is an English major so she probably deserves the credit.

Q: How did you decide to start this company, and what were you looking to accomplish at the time?

A: The company has had a couple of major phases of its development. The current phase started in 2007 when we began focusing on HIV.

In the earlier phase of the company, we were more of a research and development shop doing specialized work for the U.S. military. There were some challenges that the military felt were on the horizon in the wake of 9/11, relating to new chemical and biological weapons and battlefield medicine more generally. I had become aware of those military R&D interests through involvement with Professor William Perry at Stanford, who had previously served as the U.S. Secretary of Defense. Through my work with Perry’s group, the Center for International Security and Cooperation, I saw an overlap with some of the work I had been involved with as a graduate student. My graduate work was focused on the semiconductor industry, but I saw the opportunity to port the technology into healthcare and became intrigued.

It was not clear that it could solve the issues that the U.S. military was confronting, but it just seemed like it might, and it was pretty clear that the only way that work was going to get done would be if I made it happen. There was no one else who was likely to both be aware of the technology and also be aware of the military-specific needs.

Q: So what brought Wave 80 into HIV?

A: We were doing foundational work with the U.S. military regarding how to move proteins around inside miniaturized assay cartridges, initially unrelated to HIV. However, we established a link with the head of research and development at a group called the International AIDS Vaccine Initiative (IAVI). The head of R&D at IAVI, Wayne Koff, found out about Wave 80’s work for the military and was acutely aware of unmet needs in HIV in the developing world. Wayne had the foresight to see how the military technology could be paired with a specific assay method and that it might be fruitful, which is exactly what how it turned out.

There is now a fairly extensive and well-developed infrastructure for HIV patient care and clinical research in sub-Saharan Africa as well as Southeast Asia, the Caribbean and Latin America. I think maybe more remarkable, at least to me, is the extent to which this relatively new HIV patient care infrastructure is serving as the basis for improving treatment of both infectious and noninfectious diseases such as tuberculosis, hepatitis, cardiac disease, diabetes and many others.

A positive outcome of the great tragedy of the HIV epidemic is this significant improvement in the healthcare infrastructure of countries around the world. Wave 80 is building HIV and other products to increase these clinics’ capabilities in caring for the local populations.

Q: Talk about your partnership with the San Francisco Public Health Laboratory and San Francisco General Hospital to pilot the quicker, point-of-care test.

A: One of the challenges that comes with HIV is identifying individuals who are HIV-positive as early in the infection cycle as possible. Standard HIV diagnostics that one sees in an HIV clinic or in a primary care setting are based on what is called a serologic response. This is when HIV antibodies appear in the patients’ blood, but these antibody-based diagnostics only turn positive several weeks after infection.
Something that we are at the forefront of now, along with UCSF, SF General and the SF Public Health Lab, is identifying HIV-infected patients during what is called the acute phase before the antibodies appear. It turns out that transmission risk is highest in the acute phase, so, if you can diagnose HIV in its first few weeks, then the prospects for reducing transmission go up greatly.

I think we can all look forward to, if not the end of the epidemic, a significant decrease in the number of new infections as these universal “test and treat” measures take hold.

Q: What do you see as some of your biggest goals in the next few years?

A: We have one overarching goal which is to move the products fully into the clinical setting. We are entering the validation process right now. If it were a U.S. product, this would essentially mean preparing the FDA submission. We are bringing this product through the FDA, but for the global market it is not really desirable to hold up the product while waiting for U.S. regulatory approval.

Instead, what we do is we work through a very rigorous set of validation procedures. The U.S. Centers for Disease Control and Prevention (CDC) will oversee the bulk of early validation. The World Health Organization and the Ministries of Health of the countries into which the machines are going to be placed will also be involved. We gradually work through approvals and demonstrations of our diagnostic specs and capabilities in the settings where our products will be deployed.

Alongside that, we have the goal of using Wave 80’s HIV diagnostic platform in HIV vaccine clinical trials. This is actually a major driver of the funding package that we have from the U.S. government, in order to help differentiate between someone who is HIV positive versus a vaccine trial participant who is HIV negative, but whose blood may carry antibodies from exposure to a potential vaccine.

CHI-Advancing California biomedical research and innovation

Speaker Spotlight: Naser Partovi, Founder and CEO, Sanitas Inc.

2011-07-27T14:49:00.000-07:00

Naser Partovi

Partovi has over 25 years of management, corporate development and operating experience. Last year, he established Sanitas Inc., a mobile health technology company that helps patients with chronic conditions manage their treatment. This year, Sanitas is testing its Wellaho product in heart failure patients.

Prior to starting Sanitas, Partovi was president and CEO of SKY MobileMedia, a leading provider of multimedia applications framework for cellular handsets and new generation of connected multi-media devices. Before that, Partovi was a managing director at Enterprise Partners Venture Capital based in San Diego, where he managed a portfolio of companies in the communications industry, including Ascendant Systems, DragonWave, Inc., Quorum Systems and ReliOn, Inc. Before joining Enterprise, Naser served as vice president of strategy and business development for Nortel's optical networks business.
Partovi graduated from Canada’s McGill University with a master’s degree in electrical engineering.

Q: Tell me a little about your background.
A: I am an engineer by training. I graduated from McGill University. Then, I was working at a company called Nortel, a telecommunication company. I was the vice president of business development and strategy at Nortel when I left in 2000. I was recruited from there and joined Enterprise Partners, which was the largest venture capital firm in Southern California. I was a general partner there until 2006, when I left to run one of the companies I had invested in based in San Diego, SKY MobileMedia. We sold SKY in 2008, and, then last year, I started this new company, Sanitas.

Q: Describe, simply, the product that Sanitas is offering.
A: Wellaho is a product to help patients with chronic conditions to manage their treatment. The way we differentiate ourselves from what is out there on the market is what we call the three pillars of our treatment plan. One is education. One is monitoring of progress. And one is getting support from your friends and family and your doctors. There are other companies who are providing bits and pieces of these services. To the best of our knowledge, Wellaho is the only one that’s providing all the three services combined as a continuity of care system.

Q: Monitoring of progress … is that where mobile health technology comes in?
A: Yes. When you or your loved one is diagnosed with a chronic condition, you can do a Google search and you will hit 1 million sites and you need to figure out what is reliable information and what is not. We look up the patients’ own health record. We find out exactly what their diagnosis is, and what their treatment plan is, and we dynamically build a tailored education plan for the patient. Either your primary care doctor, or, in the case of heart failure, your cardiologist, will decide the signs and symptoms they want you to monitor. They set up the parameters.

For example, if I have heart failure and I’m pretty healthy otherwise, they might say, “Okay, your resting shortness of breath should be at this level, and, if you have walked a mile, it should be at this level.” Patients fill in information about themselves using a cell phone or they can do it on their iPad or iPhone or Android application. We bring up the information and say, “Okay, what is your level of shortness of breath?” or “How far did you walk?” “These are your medications. Which one did you take today?”

We tailor monitoring to each patient’s needs based on what their cardiologist wants to know. And the cardiologist can see this information immediately or they can see it the next time they log in, which could be a week. We’re not a real-time monitoring system.

Q: Besides heart health, does this application apply to other conditions, such as cancer?
A: The first module we introduced and the one that we are conducting the field trials on is heart failure. We will announce other modules as they become available. The reason we are introducing different condition modules in a sequence is because we go very deep inside each condition. For example, in heart failure we have over 350 modules. A lot of other applications targeting the conditions go an inch deep, miles wide. We are at the other end of the spectrum. We are going an inch wide and miles deep because we believe that for each condition you need very detailed information on how to treat, what diagnostics options are available and so on and so forth. We have modules scheduled for the next three years to come.

Q: You have a unique perspective as a mobile tech investor. What can you tell me is most influencing investments in the mobile healthcare space?
A: In mobile health, there are a lot of really good companies coming up with a lot of products. My biggest worry about this is who is going to pay for it. The reimbursement mechanism is not out there and insurance companies are not paying and the government is not paying for it. There are a lot of issues around liabilities because, if this is real-time monitoring and the doctor misses that information, what happens? So, I think there are a lot of elements of this mobile health area that need to be figured out.

These things take a lot of time in the healthcare. You need to have a lot of clinical trials, prove the efficacy of the system, go back to the insurance companies, and go back to CMS to convince them that this is improving outcomes. We have tried to take the low-tech route to helping patients because, for example, with heart failure, if we can even improve 1 percent of readmissions to the hospitals by a combination of education, monitoring and support, then that is lots of money saved by hospitals and insurance companies.

Q: What are you looking forward to hearing about at the CHI mobile health event “Take this Pill and Tweet Me in the Morning?”
A: What I would like to learn from other speakers as well as the audience is their experience in dealing with patients, patient adaption, how often patients actually stick with it. How do we convince the insurance companies and the government that this is as important as medication and surgery? So, this is a big cultural change that needs to happen.

CHI-Advancing California biomedical research and innovation

Guest Column: Patenting Genes and the Future of Personalized Medicine

2011-07-25T10:16:00.000-07:00

By Ashwin Mudaliar, Stanford University

Patents pertaining to the use and appropriation of biological information have been in the news prominently over the last year. The majority of attention has been paid to the case of Association for Molecular Pathology v. U.S. Patent and Trademark Office, which challenges the legality of Myriad Genetics' patent of the breast cancer genes BRCA1 and BRCA2, and more broadly, the patentability of human genes in the United States. However, Prometheus Laboratories Inc. v. Mayo Collaborative Services, which considers the patentability of medical methods, may have just as broad an impact over the greater landscape of biotechnology intellectual property.

The Patent Act of 1952 specifies four categories upon which an invention can be judged patentable: proper subject matter, novelty, non-obviousness and utility. It further specifies that a patentable invention be any “process, machine, manufacture, composition of matter, or improvement thereof.” If a prospective invention does not meet all four of these categories, it is not considered germane in view of the law and is thus not eligible for patent protections, as determined by the United States Patent and Trademark Office (USPTO). Laws of nature, physical phenomena, and abstract ideas are explicitly barred from patentability on the basis of not being proper subject matter.

Method patents, which are patents on processes of performing a set of steps (methods) to obtain certain results, have traditionally been awarded as long as a substantive transformation through the method was demonstrated and if the method in question was tied to a specific apparatus. However, the scope of method patents was brought into question in the case of Bilski v. Kappos (In re Bilski), which stems directly from USPTO’s rejection of Bernard Bilski and Rand Warsaw's patent application for a method of hedging commodities risk, whose claims fell under the unofficial category of business method claims. The examiner at the USPTO rejected the claims of the application because the method was not tied to a specific apparatus and that it "merely manipulate[d]" abstract concepts to solve a mathematic question without a substantive transformation. In further appeals through the legal system, the United States Court of Appeals for the Federal Circuit, which is tasked with handling all patent related matters, affirmed the initial rejections of the Bilski patent and went further by significantly modifying the standards for resolving whether any invention is statutory subject matter under the Patent Act through its promulgation of a new test, termed the machine-or-transformation test, to determine patent eligibility for any process claim. This test aims to determine if a claimed process or method is tied to a specific apparatus and/or if the same claim transforms a specific article into a different state. If either stipulation is met, then the claim is deemed statutory assuming it meets the other categories of patentability (novelty, usefulness, etc.). The plaintiffs appealed to the Supreme Court to reconsider this decision, which obliged by granting certiorari to hear the case.

In Prometheus v. Mayo, Prometheus Laboratories, a diagnostics and therapeutics company from San Diego, Calif., sued Mayo Medical Laboratories for patent infringement on two methods for determining the optimal drug therapy for a gastrointestinal autoimmune disease when Mayo Medical announced that it would sell its own diagnostic test based on similar methods. The original patent specified a method for measuring the metabolites 6-methyl-mercaptopurine (6-MMP) and 6-thioguanine (6-TG) following the administration of a thiopurine drug for autoimmune diseases. Based on the level of both, drug dosage would be altered for maximal efficacy and minimal toxicity. In March of 2008, a district court in California invalidated two patents held by Prometheus on the grounds that the methods were not patentable subject matter. However, in September of 2009, the United States Court of Appeals for the Federal Circuit overturned this ruling and claimed that the methods described in the patents satisfied the machine-or-transformation test, and more specifically that the administration and determination steps in the original claim brought about sufficient transformation. The lawyers for Mayo Medical argue that observed correlations between blood test results and health are simply “basic, natural biological relationships,” and thus non-patentable, but the Federal Court ruled that any kind of treatment that alters the body in a therapeutic manner could be patentable.

The court argued that the claims in the plaintiff’s patent did not simply cover natural correlations (metabolite levels) or data-gathering (measurement) because the, “asserted claims are in effect claims to methods of treatment, which are always transformative when a defined group of drugs is administered to the body to ameliorate the effects of an undesired condition (emphasis added).” The court also ruled that measurement that goes beyond “mere inspection” is transformative. Mayo Medical Laboratories quickly filed an appeal to the Supreme Court.

On June 28, 2010, the Supreme Court issued its ruling on the In re Bilski appeal, which affirmed the Federal Circuit’s ruling that Bilski process was not patentable subject matter. However, the Court also ruled that the machine-or-transformation test was not the exclusive test to evaluate method patents but rather just a “useful and important tool” amongst others. It did not offer further or more specific guidance. The Supreme Court then vacated the initial federal circuit court decision in Prometheus and ordered that this court reconsider its decision in light of the new ruling on Bilski. In its reconsideration of Prometheus on Dec. 17, 2010, the federal circuit court reconfirmed that method claimed by Prometheus constituted patentable subject matter and stated that the Bilski decision “did not undermine [its] preemption analysis of Prometheus’s claims.” As such, its ruling was substantively unchanged. Mayo Medical appealed again to the Supreme Court on May 17, and was granted a writ of certiorari on June 20 for consideration. A definitive ruling is expected next summer.

The issues at stake in Prometheus case are very important to the future of commercial diagnostic tests and, more broadly, the commercialization of personalized medicine. Very often, diagnostic methods similar to those described in the Prometheus patents are used to deliver safer and more effective treatments to patients who might otherwise react adversely to a therapeutic intervention. If the Supreme Court were to side with the defendants and invalidate the Prometheus patents in a broad decision, it could potentially call into the question the validity of a number of previously issued diagnostic patents that companies rely on to protect their investments. Considering that many of these sorts of diagnostic tests are created by smaller biotech companies, invalidation of the “method of medical treatment” system could hinder innovation and prevent early-stage biotechs from attracting investments. This is especially critical as we enter an age of increasingly personalized medicine, where therapeutic regimes will be closely tailored to the specific physiological states of individual patients. Without adequate protection for the research and development of such diagnostic methods, we could see a marked reduction in the number of private companies and research institutions that engage in the type of research that leads to these interventions as well as the quality of that research. Given these considerations, the Supreme Court’s ruling next year will have an enormous impact on the future of life sciences and medical technology.

CHI-Advancing California biomedical research and innovation

Event Spotlight: CHI Co-Hosts Congressional Briefing on Alzheimer’s Disease

2011-07-21T13:26:00.000-07:00

On July 14, CHI and the Healthcare Institute of New Jersey (HINJ) co-hosted a congressional briefing in Washington, D.C. focused on Alzheimer’s disease. Congresswoman Linda Sanchez of California delivered particularly poignant remarks. Sanchez’s father suffers from Alzheimer’s disease, giving her and her siblings, including fellow Congresswoman Loretta Sanchez, personal experience in caring for a parent suffering from this mind-robbing disease. Despite this challenge, Linda Sanchez considers herself fortunate to have the support of her six siblings, asking, “I wonder what it would have been like to be an only child?”

CHI President and CEO David L. Gollaher, Ph.D., and Dean Paranicas, who serves as president and CEO of HINJ, delivered opening remarks, followed by presentations on cutting-edge research and scientific advances achieved by CHI and HINJ members. The event wrapped up with closing remarks from Reps. Chris Smith (R-NJ) and Linda Sanchez (D-CA). The program, held in the Capitol Visitor Center, attracted more than 60 congressional staffers, patient advocates and industry representatives.

The speakers on this day made clear that the experience of the Sanchez family is sadly common, and growing at a rate that will be both emotionally and economically devastating. As Gollaher noted, “The Alzheimer’s crisis is of great import because of its personal and economic effects” which will have “great implications for this country and the rest of the world as populations get older.” To illustrate the scope of the crisis, he noted that 5.4 million people are living with Alzheimer’s disease, and someone develops the disease every 69 seconds. Further, in 2010, family members and friends provided 17 billion hours of unpaid care to those with Alzheimer’s and other dementias — care valued at approximately $202 billion. The effect of the disease in California alone is particularly acute with more than 500,000 Californians suffering from the disease today and estimates that the number will double to 1 million by 2040.

Yet, participants and presenters also made clear that there is hope. California leads the nation in National Institutes of Health funding, making Alzheimer’s disease one of the largest concentrations in the California R&D pipeline, Gollaher noted. A panel of top researchers then offered their unique approaches to treating and potentially curing Alzheimer’s disease. Dr. Stuart Lipton, director of the Del E. Webb Neuroscience, Aging and Stem Cell Research Center at Sanford-Burnham Medical Research Institute, described his ongoing work with NitroMemantine– a combination of an existing Alzheimer’s therapy called memantine and nitroglycerin. As both drugs are already approved for other indications, the compound’s potential speed to market is dramatically shorter than that of a new drug.

Joseph Hammang, Ph.D., the senior director of worldwide science policy for Pfizer, noted that improved diagnostic tools and personalized approaches to treating the disease that rely on a person’s unique genetic make-up are potentially critical advances in treating Alzheimer’s, which actually includes a number of distinct diseases. He said Pfizer has committed $100 million over five years on collaborations with public institutions, demonstrating its continued commitment to work with academic partners in the U.S. and around the world.

Kimberly Scearce-Levie, Ph.D., head of in vivo neurobiology for Genentech, detailed her team’s recent advances in methods to block a protein that composes the plaque thought to be responsible for cognitive impairment in AD sufferers. The Genentech team has developed a way to hijack the body’s own mechanism for passing iron from blood into the brain, essentially tricking the body into transporting a critical blocking agent through its own defenses..

Bob Nelson, Ph.D., research fellow at Lundbeck Research USA, continued the conversation by saying he believes that the story is likely to be more complicated. Lundbeck Research has begun to challenge conventional wisdom about the disease by asking slightly different questions. Are the identified plaque compounds associated with AD strictly bad substances? Might blocking them also have harmful effects? Might the abundance of plaque-causing proteins be caused by victim’s inability to remove or reabsorb the protein rather than due to a problem of overproduction?

Lastly, Wayne Poon, Ph.D., director of the UCI MIND Brain Bank and Tissue Repository, described his approach to solving a basic problem in Alzheimer’s disease diagnosis. The disease, he said, is only diagnosable when a patient has died and his or her brain can be examined. Because of this, up to 15 percent to 20 percent of patients thought to have AD are later found to have been misdiagnosed.

In closing, Rep. Smith of New Jersey, chairman of the Alzheimer’s Disease Caucus for 11 years, thanked the panel for its efforts. The U.S. and the world are facing a figurative pandemic, he said, which is on the verge of a breakout with the arrival of a “demographic winter.”

“We have a crisis and funding is flat-lined,” he said. “We simply aren’t going to have the people to take care of these [AD sufferers].”

Rep. Sanchez concluded, “I wish I was here to celebrate a cure…Yet, with the talent and work of many, we will be able to celebrate that day in the future.”

CHI-Advancing California biomedical research and innovation

Speaker Spotlight: Anthony Costello, COO Mytrus

2011-07-15T11:33:00.000-07:00

Anthony Costello

Mytrus is testing the boundaries of traditional clinical trial research, with the first all-electronic, home-based clinical trial to gain U.S. Food and Drug Administration approval. As co-founder and chief operating officer of San Francisco-based Mytrus, Costello is leading the charge. Costello is a leader in the areas of technology innovation and clinical operations. After beginning his research career at Genentech, Costello went on to co-found several technology start-up companies including Nextrials, a clinical research firm where he worked for 10 years as the vice president of product development. He is a frequent presenter on topics related to the efficient use of technology and clinical research.

Q: What brought you into the clinical research industry?

A: I got into the clinical research industry in the ’90s when I started working at Genentech. At the time, I was in data management and spent pretty much my whole career at Genentech in data management. I ended up overseeing the data management group for their oncology center and working mostly on the Herceptin trials and the early trials for what is now Avastin.

Back in those days, everybody was doing clinical research on paper. We did all of the international Phase 3 trials for Herceptin on three-part no-carbon-required paper, mailing it around the world and then data entering everything. Although I loved Genentech and it has certainly launched my career, I wanted to develop a technology that was faster and more effective. I left Genentech in 1999 to co-found a company that makes electronic data capture systems for clinical trials, called Nextrials.

Q: What happened next?

A: I left Nextrials in 2008, after almost 10 years there, and I met Dr. Steve Cummings (Mytrus CEO and chairman) and got excited by his idea of building more of a consumer-focused clinical trial that would allow patients to participate from home. I joined an advisory board that Steve was putting together to sort of vet out this idea, and we ended up deciding to form Mytrus around that. I joined as a co-founder and as the vice president of product development and commercialization and I now serve as chief operating officer.

Q: Mytrus is hoping to validate a new kind of study model. If this kind of remote results monitoring works, what might the future look like for pharmaceutical companies and their trials?

A: We hope that it’s going to make a big difference. Our hypothesis that is staring to play out is that there are some kinds of trials – we estimate about 15 percent to 20 percent – conducted in the United States annually that can be done using this method. They do not require specialized equipment at a site. They do not require specialized training, and, if a patient is willing and able to correspond via the web and provide their own updates about what is happening to them during the study, then those trials are suitable to use this method. It really changes once you free your mind, as Steve likes to say, from the idea that you have to have a bricks-and-mortar site for trials.

You do not have to have as much monitoring because the patients are the direct source for data and you do not have data being transcribed from the patient to a chart and then from a chart to an electronic data capture system and then from an EDC system to an alpha system. All those data transcription events are the reason monitoring was effective — to make sure nothing got missed or skipped or inadvertently entered. So, you could get rid of a lot of that. You don’t have to go through the contracting and start-up and hierarchy approval with hundreds of clinics around the country.

Q: What are the technology requirements for participation?

A: If you do not have an Internet connection, you cannot do it. In fact, depending on the trial, there are some studies where it is really going to be critical to have an Internet connection in the home. There are other studies where it would be perfectly suitable to have a smart phone or an iPad or even a shared Internet connection in some sort of public place if the patient is comfortable using that and it is private enough that someone can enter their own data. Patients are much more apt these days to use the Internet for their own medical research and they are much more comfortable communicating with doctors over email or over the Internet.

Q: What are you looking forward to hearing about at the CHI mobile health event “Take this Pill and Tweet Me in the Morning?”

A: There is just a lot more focus on health 2.0 technologies these days and so we’re excited about being involved. It seems like the lineup of speakers are people on that leading edge of moving trials or studies or health in a new direction so it is a perfect kind of conference for us.

Hear more from Anthony Costello at “Take This Pill and Tweet Me in the Morning,” happening Aug. 4 at the Salk Institute in San Diego.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: David Shuey, The Willis Group

2011-07-05T12:30:00.000-07:00

David Shuey

David Shuey, executive vice president and North America practice leader for The Willis Group’s life sciences practice, thinks “startups now realize they can’t be one-trick ponies – they have to have multiple product candidates in the pipeline.” With 19 years of hands-on experience serving the life sciences industry, he should know. His clients range from early-stage R&D companies to large pharmaceutical firms. Shuey has expertise in therapeutics, medical device, generics, nutraceuticals, consumer products, contract research organizations, laboratories and contract manufacturing. His focus is on casualty lines, particularly product liability and international human clinical trials. Read below to find out how Willis Group’s unique technology platform has helped companies of all sizes remain competitive in the global economy.

Q: Within Willis, how big is the life sciences practice?
A: We are a $3.5 billion revenue public company. Within Willis, life sciences is a large practice and a focus. I think management sees it is a growing area. We’re going to need new medical devices and therapeutics no matter what happens.

Q: Tell me a little about your focus at The Willis Group.
A: I have been with Willis for 22 years with 19 of those focused in life sciences. I started off in medical practice liability for doctor groups and integrated health systems. At the time, I was working in the Baltimore area, around D.C. and the northern Virginia area, and a lot of VC money was coming into the area. They were hiring scientists out of the NIH with a molecule and an idea for a company. So, I started working with a lot of those nascent companies as they were getting started and developed the expertise in life sciences and spent the rest of my career with life sciences companies.

Q: What do you notice are the major differences between biomedical startups today and back then?
A: From a risk perspective, things have definitely changed. I think it was easier to raise money back then. The concept of failing early with molecules and accelerating the discovery process using rational drug design techniques wasn’t available back then and is now – so that would be a change. Back then, the focus was on gene therapy, and, while still popular, I think regenerative medicine has taken over. A lot of med device companies that were developing things for labs are now moving into the clinical setting with personalized medicine a purpose.

Q: Can you give me an example of how The Willis Group came to the aid of a biomedical company or academic research institution in recent times?
A: One of my favorite stories involves a large client that was making an acquisition and it was an orthopedic implant, a Class 3 medical device. During due diligence, our client learned that the company they were acquiring had received an FDA non-approvable letter because their sales and medical reps were recommending an alteration to the implantable orthopedic device during surgery. Our client thought that might be a problem, so asked for a large contingency hold-back in the buy-and-sell agreement. So, we stepped in and wrote a three-year risk or loss mitigation program with a per person coverage limit and a three-year policy aggregate for them. The program had a provision that, if no claims came after three years, our client could commute the policy and get their money back. The insurance company would keep the risk charge and the unused premium at the end of the policy would be returned to the client. The fact that we could transfer that risk from the balance sheet to a risk policy allowed the deal to go through.

Q: With clinical trials management moving overseas for many in the industry, what implications does this have for business moving forward?
A: Even the smallest R&D therapeutics company will take their trials oversees, so small companies become international companies very quickly. That’s a change we’ve identified. We are particularly adept at managing global foreign trials clinical programs. We have something called CTTrack which is a dashboard that allows clients to see where they are in relation to a deadline to meet with insurance and certificate requirements around the world. We maintain a matrix of all the requirements around the world. It is kept real-time on our clients’ desktop so that when they conduct a trial in Israel they can flip on the matrix and go to Israel and find out what the requirements are and what kind of lead time they need to conduct the clinical trials.

Q: What other new offerings does Willis Group provide its clients?
A: We’ve put together a unique program called Diagnostics 2.0. We come in and conduct a mini enterprise risk-management analysis. We conduct an in-depth interview that is designed to uncover the organizational risk. At the end of the process, we’ve done a diagnostic of the risk, so now we can deliver a customized risk/review report. Then, we can go in and look at the insurance programs structure, the pricing, the coverage and match it up with the risk diagnostic. It really peals the onion on risk exposure and it is unique to Willis.

CHI-Advancing California biomedical research and innovation

The Evolving Business Model for Biomarkers

2011-07-01T10:26:00.000-07:00

Biomarkers will be one of the major drivers of biotech research, drug and diagnostics development in 21^st century medicine. Dr. Jogarao Gobburu (Division of Pharmacometrics, FDA), Stephen Little (Qiagen), Mark Erwin (Prometheus Laboratories), Dr. Bryan Dechairo, (Medco Research Institute) and Dr. Christoph Huls (Merck Serono), explored the evolving business model for biomarkers and personalized medicine during a panel discussion at the 2011 BIO International Convention in Washington D.C. Mark Erwin summarized the common theme throughout the discussion: “Personalized medicine holds the promise for not only greatly improving healthcare for patients all over the world, but to generate cost savings and increase efficiencies in the healthcare system. All of us as healthcare stakeholders need to do what we can to incentivize this innovation.” Watch the video.

CHI-Advancing California biomedical research and innovation

How Biotech Companies are Shaping the Future in Rare Diseases

2011-06-30T07:16:00.001-07:00

Biotech companies are navigating new trends in addressing rare diseases, conditions effecting 200,000 or fewer Americans. Jeremy Springhorn (Alexion Pharmaceuticals), Andrew Curtis (Pfizer), Marc Beer, (Aegerion Pharmaceuticals) and Susan Kahn (National Tay-Sachs & Allied Disease Association), shared their perspectives on the future of new treatments for rare diseases during a panel discussion at the International BIO Convention in Washington D.C. Alexion Pharmaceuticals invented Soliris, the first and only therapy for treating Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare, progressive and life-threatening disease defined by hemolysis, the destruction of red blood cells. Pfizer created a new research unit in 2010 with the goal of leveraging existing scientific experience in rare diseases, such as hemophilia, to discover novel, life-saving medicines for patients with large unmet medical need. Aegerion Pharmaceuticals is developing a new drug to treat Homozygous Familial Hypercholeterolemia (HoFH), a rare genetic lipid disorder resulting in an accumulation of low density lipoprotein (LDL-C) often referred to as bad cholesterol in the blood. The National Tay-Sachs & Allied Disease Association is directly funding research to treat and cure Tay-Sachs, Canavan and related genetic disease and supporting affected families and individuals. Tay-Sachs disease is a rare progressive neurological genetic disorder that is caused by the absence or insufficient level of a vital enzyme called Hexosaminidase (Hex-A). All children with classic Tay-Sachs disease die early in childhood, usually by the age of 5, and today there is no cure or effective treatment. Jeremy Springhorn summed up the sentiments of the panelists: “To commit to putting the resources it takes to developing treatments for rare diseases, our focus is and will always remain on the patient.”

MedTech Innovators Highlight Need for U.S.-Korea Free Trade Agreement

2011-06-28T06:24:00.001-07:00

CHI was joined by the U.S.-Korea Business Council, the Silicon Valley U.S. Export Assistance Center, Sunnyvale Mayor Melinda Hamilton and other key stakeholders at a medical technology industry roundtable held June 24 with Jong hyun Choi, Minister of Economic Affairs, Embassy of South Korea at Accuray in Sunnyvale, Calif. The meeting focused on the support of the California medical technology community for the U.S.-Korea Free Trade Agreement (FTA), the first FTA to include an entire chapter with commitments to openness and transparency in medical technology regulation and payment policies. CHI members Mark Deem, partner of the medical technology incubator, The Foundry, and Tom Loarie, executive chairman of Mercator MedSystems, shared their experiences bringing medical technology innovations to market and highlighted the need for open markets and fair regulatory and payment schemes worldwide to support development of technologies that can improve the lives of patients. Fred Kinder, a former CyberKnife patient, spoke of the need for access to medical technologies for all patients. Korea is one of the world’s largest and fastest growing markets for medical technology, with expected growth of 10-15 percent per year over the next several years. Following the meeting Accuray CEO Euan Thomson, Ph.D., gave a demonstration of the CyberKnife System to highlight how the technology is used to treat tumors in patients with cancer. Watch NBC News coverage of the event.

BIO Kicks Off With Run for Partnership for a Healthier America

2011-06-27T08:53:00.000-07:00

Attendees at the Annual International BIO Convention kicked off the meeting Monday morning with a healthy 5K run hosted by the Diabetes Advocacy Alliance at Bluemark Park just outside the nation’s Capitol. I was joined by CHI member Sarah Nordstrom from Novo Nordisk and other biotech runners who came out to support the Partnership for a Healthier America, a partner to First Lady Michelle Obama’s Let’s Move initiative to combat childhood obesity. I was still on California time (4:00 a.m.!), so it was early to get up and run, but well worth the effort to raise awareness for a great cause! Watch the video.

Community Spotlight: CHI Participates in Rolling Readers Program

2011-06-20T16:57:00.000-07:00

This month marked the end of an exciting journey I began this year into the world of volunteer reading and 1st grade learning.

As a volunteer for the Rolling Readers program, I began each Wednesday morning at Ocean Beach Elementary School, a community-oriented school that values education and learning through traditional and creative means. CHI allowed me the opportunity to bring my love of reading to the school each week, where I spent time on the colored floor mat of Ms. Tanner’s classroom – a lively bunch of students eager to read and quick with questions.

Rolling Readers connects volunteers to underprivileged classrooms, where we delve into classic tales (Curious George and Shel Silverstein books) and modern fantasies (John Lithgow’s I’m a Manatee).

The organization’s mission is a simple one: to inspire children to love reading. Children who love reading are more likely to become lifelong learners, so the saying goes.

I found inspiration through these staggering Rolling Readers statistics:

• In low income neighborhoods, the ratio of books to children is 1:300—which means that, on average, there is one book for every 300 children.

• By age four, children who live in poor families will have heard 32 million fewer words than children living in professional families.

Since every child deserves a book of their own, I was especially excited to participate in the annual Rolling Readers book drive. Each student in the classroom received a special edition of the popular Dr. Seuss book The Cat in the Hat (appropriately written in La Jolla).

The students erupted with joy when they saw the book. It is satisfying to know that they will be taking the book home to share, which may encourage reading among their friends and family.

Theodor Seuss Geisel, himself an advocate for elementary literacy, once said: "The more that you read, the more things you will know. The more that you learn, the more places you'll go.”

CHI-Advancing California biomedical research and innovation

Rep. Kevin McCarthy, Biomedical Industry Leaders Focus on FDA, NIH, at CHI Roundtable

2011-06-08T15:45:00.000-07:00

The House Majority Whip, California Congressman Kevin McCarthy met with California biomedical innovators at a CHI-California Healthcare Institute roundtable at Gilead Sciences in Foster City, Calif. on June 7. In addition to Gilead, among the prestigious research institutions and companies represented were The J. David Gladstone Institutes, Amgen, Thallo Bioscience, Proteus Biomedical, and Abbott Vascular. CHI President & CEO David Gollaher introduced Congressman McCarthy, who was honored with CHI’s Leadership Award earlier in the year, and thanked him for his support for California biomedical research, investment and innovation. Rep. McCarthy was eager to hear about the latest research, treatments and new medical technologies from participants. They shared news of breakthrough research demonstrating that the use of HIV medication reduced the risk of HIV infection in uninfected people, a discovery that topped Time Magazine’s list of medical breakthroughs of 2010, the development of embedded computers and sensors inside proven drugs and devices to monitor real-time patient compliance and response, and less invasive heart valve replacement surgeries.

Rep. McCarthy was interested in the group’s perspective on what policies are stifling innovation and U.S. competitiveness. The U.S. Food and Drug Administration (FDA) is not keeping pace with U.S. biomedical innovation. The Agency-industry partnership is strained by unexplained regulatory delays, by a lack of clear standards for what clinical data are necessary for product approval, and by a bureaucracy whose communications are neither consistent nor predictable. In this regulatory environment, companies are launching products first abroad forcing jobs and investment offshore. NIH funding must also be preserved to ensure a healthy pipeline of new research for product development. Rep. McCarthy is optimistic on American ingenuity and innovation and wants to make better government to advance it: “We have the framework to turn it around, but do we have the willpower?”

CHI-Advancing California biomedical research and innovation

Event Spotlight: Therapeutic Advancements, Innovations and Treatments in Musculoskeletal Health

2011-05-27T09:46:00.000-07:00

On May 24, CHI hosted an insightful briefing in Washington, D.C. highlighting the importance of therapeutic advancements and innovations in musculoskeletal disease and injuries. The program was held on Capitol Hill, and was attended by congressional staff, patient advocates, industry representatives and members of the press, among others. The assembled panel of experts addressed a variety of issues in the area of musculoskeletal conditions, providing an excellent awareness-raising overview and highlighting some of the successful research, technologies, and treatments being offered by CHI members.

Todd Gillenwater, CHI’s senior vice president of public policy, opened the program by detailing the impact of musculoskeletal disease in the United States. In particular, he noted that in the U.S. alone, these conditions are a leading cause of disability, accounting for more than 130 million patient visits to healthcare providers each year. Further, he said the annual cost of treatment associated with musculoskeletal disease is estimated at a staggering $254 billion.

Then, CHI’s invited panel of experts took the podium in turn to present their own unique perspectives on musculoskeletal health. First to the podium was Dr. Said Ibrahim, professor of medicine and vice chair of the Department of Medicine at the University of Pennsylvania School of Medicine. His presentation focused on health disparity research in the area of joint replacement procedures, which has shown that minorities, African Americans in particular, are less likely than Caucasians to opt for elective joint replacement procedures. His findings, adjusted for key factors such as insurance coverage, indicated that the disparity was related to the fact that African Americans were less familiar with the procedures and more concerned about things like post-operative pain and recovery time. Ibrahim said he believes that this disparity is now well enough understood to be effectively addressed by a targeted educational campaign.

Dr. Deborah Kado, associate professor with the Departments of Orthopedic Surgery and Medicine at the Geffen School of Medicine at UCLA, presented research on hyperkyphosis — a musculoskeletal aging condition that creates humped necks or upper backs in men and women. The condition is now recognized as a geriatric syndrome, yet surprisingly little is known about its causes or potential means of prevention. Preliminary studies, including Kado’s own National Institutes of Health-funded projects, have demonstrated that the condition decreases lung function and quality of life, while increasing rates of falls, injury and mortality. Kado’s team has received renewed NIH funding to continue its work on hyperkyphosis through a 1,800-member cohort study of men over the age of 65.

Next to the podium was Laetitia Cousin, vice president of regulatory and clinical affairs and quality assurance with San Diego-based NuVasive. Cousin discussed NuVasive’s explosive growth led by their innovative, minimally disruptive spinal surgery devices and techniques. NuVasive’s next-generation surgical methods and tools make their procedure’s quality-adjusted life year (QALY) outcomes equal to or better than that of hip surgery, helping to erode spinal surgery’s poor 1990s-era reputation. Cousin closed by laying out two main obstacles to NuVasive’s willingness and ability to reinvest its recent profits in additional R&D and growth: U.S. Food and Drug Administration clearance/approval delays and the medical device excise tax. NuVasive recommended improved FDA transparency through guidance documents; improved FDA reviewer retention and expertise through training; improved FDA review process efficiency through earlier and more frequent collaboration with industry; and repeal of the device tax.

Last to present was Scott Simonet, Ph.D, vice president of research for Amgen. Simonet described the 15-year process to discover and develop Amgen’s denosumab, a drug that regulates bone density and helps the human body limit bone reabsorbtion. With precursor science first discovered in 1995, the product received FDA and European regulatory approval to treat osteoporosis in 2010, as well as FDA approval for cancer-related indications in the same year. While Simonet was sparing in his willingness to express complaints associated with denosumab’s approval process, he did make a point to show the length and complexity of the product’s discovery and development chain.

The presentations spurred a lively and candid follow-on discussion between the panelists and the audience. As the event closed, many expressed their thanks to CHI for organizing the session, with more than one attendee saying that that they had found it “very educational.”

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Frank Ahmann, President and COO LoneStar Heart

2011-05-26T11:01:00.000-07:00

Frank Ahmann

LoneStar Heart got its start in Southern California as CardioPolymers, with the goal of changing the way we treat heart failure and other cardiac abnormalities. Frank Ahmann, a veteran of the biotech and medical device industries, brought his expertise to LoneStar Heart following a management career in Europe and North America at Baxter International’s biotech and cardiovascular group, now Edwards LifeSciences. Among many products, he launched the first comprehensive system for the collection, processing, storage, and administration of hematopoietic stem cells. A German citizen who grew up in Mexico, Ahmann has strong ties to the European medical market and speaks English, Spanish and German. He holds a bachelor’s degree from the University of Texas at Austin and a master’s in business administration from Columbia University Graduate School of Business.

Q: Tell me something that makes LoneStar Heart unique.

A: We are not a typical venture capital-funded company. We have been funded privately by a very small group of wealthy individuals based in Texas. In the future, we expect to fund our growth through corporate partnerships and investments as well as the continued participation of our private investors.

Q: Talk about the technologies that originated at University of California San Francisco and led to the foundation of LoneStar Heart.

A: The original idea before LoneStar Heart was even formed was a company by the name of CardioPolymers Inc., based here in Southern California with technology from the University of California San Francisco (UCSF) and a gentleman by the name of Dr. Randall Lee, who was an electrophysiologist and professor there.

He was looking at a way to use an implantable hydrogel in combination with stem cells or growth factors. One of the shortcomings of stem cell therapies is that they don’t linger in the heart muscle very long or at all, and the effect of stem cell therapy and growth factor therapies is very, very limited and lasts for a very short time. So, nobody has really been able to show in significant clinical studies that stem cells in the heart regenerate tissue and improve long-term and permanently the condition of the failing heart.

The issue became “Can we make this better? Can we find a way for these stem cells to actually stay in the heart?” The one way to do that is to mix in these stem cells with the hydrogel.

Q: Have hydrogels been used in any other stem cell applications before?

A: Not in the heart at all. It is completely novel for the heart. As we were testing it, we found out that the hydrogel, without stem cells, had a very significant and positive effect on improving the mechanics of the heart, which led to the product that it is today: Algisyl. This product is essentially a gel that is derived from brown algae. It is extremely purified to remove all endotoxins and make it inert. It is liquid when you inject it and then it solidifies to the right degree when it is in the heart.

Normally, what you would expect is that the material will degrade after a few weeks. In our case, the material stayed put in preclinical models. After two years, the hydrogels were still there, in the same quantity and in the same position as they were in the beginning.

Suddenly, we found ourselves with a long-term, implantable device. It is a device because it has no pharmacologic or immunologic action.

Q: So how might this be useful in congestive heart failure?

A: A large majority of patients that have heart failure have a dilated, stretched out heart muscle that has a decreasing pumping efficiency and is incapable of delivering enough blood to the entire body. This is a vicious circle that continues to get worse with time and the implantable hydrogel actually stops or even reverses this negative trend. It accomplishes this by reducing the tension and the stress to the heart muscle purely through mechanical means.

Beyond this first step, to be able to achieve true regeneration of tissue and provide even greater efficacy, you will most likely have to combine a couple approaches. So, our idea has been all along to have the implantable hydrogel, which acts to mechanically improve the heart, combined with bioreactive small molecules or biologics that we have licensed from the University of Texas to provide the ability for tissue regeneration.

Q: What are you noticing in your initial studies?

A: Up to this point, we have treated a total of 11 patients — a majority of them are longer than one year out from when they received the implant. The safety, feasibility, and initial signals of efficacy look very positive. Therefore, we will soon begin a controlled randomized study in Europe to show efficacy delivering the product through surgical techniques. Later, an interventional catheter-based delivery will be added to the product mix.

We expect that in two years we will have enough data to receive approval to market in the European Union with the first version of the product.

CHI-Advancing California biomedical research and innovation

Event Spotlight: VC in the OC

2011-05-25T11:52:00.000-07:00

Record-breaking attendance of more than 600 VCs and entrepreneurs convened for OCTANe’s Annual VC in the OC event, May 24, in Irvine, Calif. OCTANe CEO Matthew Jenusaitis kicked off with highlights from the OC Innovation Report, demonstrating that by quantitative and qualitative measures on the state of innovation, overall environment and investment, Orange County is an epicenter for medical technology and high tech in the U.S.

Next up, the “Alliance Strategies for Growth in the Life Sciences Industry” panel (sponsored by CHI member Deloitte) featured perspectives from Steve Pal (Allergan), Sharon Stevenson (Okapi Venture Capital), John Kehl (Edwards Lifesciences), Glenn Snyder and Jacques Mulder (Deloitte).

“Don’t overlook alliance opportunities with middle-size companies and consider the advantages of these compared to larger companies to open up the range of potential partners,” Stevenson advises.

“As a company, alliances are critical to Allergan to keep our growth rate of 12-15 percent per year," said Pal. "We are looking at technologies in all stages of development, whether early-stage or late-stage commercialization."

Kehl weighed in on Edwards Lifesciences investment in R&D: “Edwards will be flexible in structuring the deal, but the technology must be centrally relevant to our business. In the current environment, it is taking more and more to get product to market, so the R&D line is sacred and will grow through alliances.” Check out Deloitte’s study: "Critical Factors for Alliance Formation."

Mark Stevens, Partner at Sequoia Capital gave the lunch keynote. He described he and his wife Mary’s vision in creating the USC Stevens Institute for Innovation with a $200 million donation to develop a university-wide resource for USC students and faculty to transition beyond concepts into real-world implementation and address untapped opportunities. As far as new sources of start-up capital, Stevens recommends entrepreneurs access microfunds. Microfunds are typically run by high-wealth individuals with industry-specific experience who are very hands-on, investing smaller amounts and combining that with their bandwidth and connections to shepherd the company through early-stage development.

Don Milder, partner with Versant Ventures, provided his perspective on healthcare investing on the OC VC panel. He sees the U.S. Food and Drug Administration improving but still in need of reform.

"Never underestimate the power of one transforming a bureaucracy," he said. "That one individual who is making strides to improve FDA for all of us in med tech is Josh Makower."

CHI-Advancing California biomedical research and innovation

FDA Drug Lag: Challenges and Opportunities Working with FDA

2011-05-23T12:02:00.000-07:00

CHI and Hogan Lovells convened a half-day program on the U.S. Food and Drug Administration (FDA) drug lag and the challenges and opportunities in working with the agency on new drug applications. Our guests heard from speakers including CHI’s CEO David Gollaher, Ph.D.; Rick Winningham, chief executive officer of Theravance Inc.; Marcea Lloyd, senior vice president, government and corporate affairs and general counsel at Amylin Pharmaceuticals; Beth Seidenberg, M.D., partner with Kleiner Perkins Caufield & Byers; Joseph T. Kennedy, vice president, general counsel and secretary for Transcept Pharmaceuticals, Shelley Chu, M.D., Ph.D. principal of Frazier Healthdcare; and Hogan Lovells partners Robert Church, Kevin Clayton, David Fox and Lynn Mehler.

Gollaher opened the proceedings in his keynote address, by providing highlights from CHI’s recent publication, Competitiveness and Regulation: The FDA and the Future of America’s Biomedical Industry. According to the report, which uses FDA’s own data to establish the relationship between regulation and the competitiveness of the industry, today’s FDA is not keeping pace with the innovation being produced here. The relationship between FDA and industry is strained by unexplained regulatory delays, a lack of clear standards for clinical data and an inconsistent and unpredictable pattern of communication, which has led to an increase in risk and a crisis in biomedical R&D funding. The resulting flight of product launches to EU countries should be cause for concern for everyone from policymakers to the patients who are most in need.

Subsequent speakers, including Lloyd and Winningham provided insights as executives from biomedical companies with products either currently in review at FDA or on the market, into the struggles these innovators are facing, with the capital markets in crisis and an uncertain regulatory process. Key takeaways from these people on the front lines included:

This business is about patients. Period.
Success depends upon taking, recognizing and managing risk.
Surround yourself with smart advisors and even smarter employees.
Precise communication is key – among all your trusted advisors and employees involved in the clinical trials, data analysis and regulatory approval of products, and regulators.
It’s not just about strategy…perfect execution is key.
Only the smart and persistent will survive in this climate.
More companies will be forced to specialize and put all resources toward the most successful projects due to the higher barrier to entry.

Dr. Seidenberg, partner with Kleiner Perkins Caufield & Byers and Dr. Chu, principal with Frazier Healthcare, provided valuable insights from the venture capital perspective, explaining the consequences of increased risk, including money and people going overseas, not just to the EU, but to China. High tech and life sciences are both catalysts for growth overseas and Seidenberg noted that foreign executives are coming here to get educated and going back to their homelands to research, develop and commercialize medical technologies that have greater chance of being commercialized sooner overseas, rather than in the US. The regulatory and business environment in the US is “absolutely influencing our investing,” said Seidenberg, “we can’t sit back and let the US lose its competitive edge.”

When panelists, including Hogan Lovells experts from their global life sciences practice, Church, Clayton, Fox and Mehler, were asked what we can do to stem this tide of medical innovation leaving the U.S. in search of friendlier tax and regulatory environments, here were some of the insights shared:

We need more dialogue between industry and regulators.
We need clarification on the decision-making process, timelines and goals.
We need more expert reviewers at FDA.
We need to open the lens to assess risk and benefit in a systemic way.
Without real, substantive change, the pain is going to go beyond investors and innovators – this is a crisis that is going to hurt everybody – most notably, the patients who need innovative, world-class therapies.

Stay tuned, as CHI and Hogan Lovells plan to release an Executive Summary on the proceedings from the meeting. For more information, please call CHI at (858) 551-6677.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Scott Johnson, President and Founder, Myelin Repair Foundation

2011-05-05T09:21:00.000-07:00

A special executive spotlight from CHI and BayBiotech Review

Scott Johnson

Scott Johnson was only 20 years old when he was diagnosed with multiple sclerosis. In 2002, after waiting 26 years for a cure, Johnson took things into his own hands and leveraged his background as a business consultant and serial entrepreneur to create the Accelerated Research Collaboration model to reform the medical research and drug development system that has failed to produce desperately needed new patient treatments for millions living with chronic and debilitating diseases. In 2006, Johnson was named as one of Scientific American magazine's top 50 worldwide for business, science and policy leadership. In 2010, he received a Northern California Ernst & Young Entrepreneur of the Year Award. In 2011, Fast Company magazine named the Myelin Repair Foundation one of the top 10 biotech companies.

Q: What was the impetus of starting MRF?

A: In 1976, at the age of 20, I was diagnosed with multiple sclerosis. Unlike many who are diagnosed with MS, I was fortunate to be able to pursue an active business career, including leading three start-up companies. In 2001, I read a news brief in BusinessWeek that described work being done at Yale on myelin repair in MS. (Myelin is the insulating substance coating the nerves in the brain and spinal cord. It is also the substance that is damaged in MS. This damage is what causes the disease’s unpredictable symptoms). Up to that point, everything I had read about MS had focused on how to cure the disease or how to slow the immune reaction. So I picked up the phone, tracked the scientist down and started asking questions.

I learned two important lessons from my conversation with this scientist and others. First, scientists were starting to believe that regeneration or repair in the central nervous system was possible, and that it probably happens naturally in healthy people. Second, and perhaps the real lesson, was that it seemed that if you did things slightly differently throughout the entire continuum of medical research and drug discovery that the process of developing new medicines could be greatly accelerated. That was really the genesis of the Myelin Repair Foundation.

Q: What is the Accelerated Research Collaboration model and how is it different from traditional approaches to basic research and drug discovery?

A: The traditional model for medical research and drug discovery typically divides into several parts: basic biology, often conducted in an academic laboratory; pharmaceutical drug development; clinical trials; and FDA approval. For a variety of reasons, over the past several decades, these entities have grown farther and farther apart. The result has been a failure, essentially, to connect the vital dots that would lead to bringing a new medicine to market. That is why, in spite of a doubling of investment in research from 1995-2005, the number of new drug approvals has remained flat.

The Accelerated Research Collaboration model optimizes and integrates the contributions of all those who participate in the value chain of making new medicines. As a result, it provides a larger number of therapeutically relevant treatment targets in the pipeline, it translates discoveries made in animal models to the human system, it builds partnerships with pharmaceutical companies to develop and bring new treatments to market, and, ultimately, improves quality of life for millions living with diseases for which there are currently no effective treatment or cures.

Q: What are some of the exciting, new initiatives the MRF will be launching in the next 1-5 years?

A: The success of our discovery biology program has brought us many high-quality therapeutic targets and several new research tools that may be used more broadly in research for other neurological diseases.

At this time, we have two development paths before us: to validate the most promising novel myelin repair therapeutic targets through contract research organizations and to investigate the potential for repositioning of more than 40 targets that are in clinical development by various companies for other indications. We expect to pursue these paths in parallel and have developed a robust translational medicine platform to accomplish this.

We are also investing in the development of myelin repair biomarkers. Establishing one or more clearly measurable biomarkers will help us select the most promising of our targets for development.

Q: How are you measuring success in the near and long term?

A: Near-term success is already being measured by the number of targets identified in our funded laboratories as well as the number of new research tools that are enabling this research. In the longer term, of course, the only real measure of success will be one or more successful clinical trials for a myelin repair therapy. We are currently aiming for a myelin repair clinical trial for one of our targets in 2014.

Q: What is your fundraising strategy?

A: Though many think of us as a biotech company, we are in fact a non-profit organization supported by generous individuals, corporations and foundations with either an interest in finding a cure for MS or an interest in improving the outcomes of all medical research. In six and a half years, we have raised gifts and pledges totaling $43 million. We anticipate the next phase of our work will require as much as $80 million to complete.

Q: What is your partnership strategy?

A: There has been considerable interest from several companies in our both our portfolio of targets and our research tools. At this time, we are seeking partnerships with pharmaceutical companies who have an interest in myelin repair or who have a compound in clinical development for another disease indication that from our research appears to affect the same mechanisms for myelin repair. We are reaching gout to those companies now.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Tony Recupero, President and CEO Baxano

2011-04-26T16:52:00.000-07:00

Baxano, based in San Jose, Calif., was founded in 2005 by Dr. Jeffery Bleich, who had the vision to create flexible tools to provide precision lumbar decompression from the “inside out.” Baxano’s mission is to develop innovative tools that restore spine function, preserve healthy tissue, and enable a better quality of life for the patients it serves.

Recupero brings over 20 years of experience in the medical device industry to Baxano. Prior to Baxano, Recupero led one of the largest and most effective direct sales forces in the spine market as vice president of sales for Kyphon Inc. Prior to Kyphon, Recupero was the national sales manager for Sulzer Spine-Tech Inc. He began his medical device career in sales and sales management with United States Surgical Corporation. Recupero is a graduate of The General Management Program at Harvard Business School and holds a bachelor’s degree in communications from State University of New York at Albany.

Q: Share with me a short history. What first brought you to Baxano?

A: I have been in spine technologies for the last 12 years and have gravitated to the introductions of revolutionary technologies. First, with Sulzer Spine-Tech and then with Kyphon, a local company that was acquired by Medtronic in 2007. I have always looked for technologies that represent advancements in care as opposed to “me-too” products.

I ran into Dr. Jeff Bleich, who is the founder of Baxano, a few years ago, in 2006. I saw the potential that the technology had – not only to provide patients with degenerative spinal stenosis a less invasive treatment but to potentially address a large subset of patients undergoing fusion today.

I have always been attracted to technologies that represent a leap forward and doing less invasive surgery with patients that help them recover faster. Ultimately, that is what attracted me to the company.

Q: How would you describe the focus of Baxano?

A: We are very focused on developing less-invasive, cost-effective technologies that can provide surgeons, patients and payors with options that will hopefully provide better patient care, while being cost-conscious because there is obviously a lot of stress on the healthcare system right now.

Q: Your product is aimed at patients with lumbar spinal stenosis. Talk about the product and what it is addressing in the marketplace?

A: There are 225,000 lumbar decompressions done in the U.S. every year. The procedure is typically done for patients that have lumbar spinal stenosis. That is the removal of some bone and some soft tissue that is creating pressure on nerve roots in and as they exit the spine.

There are also many spinal fusions done each year, and many of those fusions are done for spinal stenosis as well. Probably, at least another 250,000 fusions are done for a variety of reasons – a large subset is spinal stenosis.

What our device enables a surgeon to do is, instead of having to go from the outside in, they can go from the inside out so that it preserves tissue. We thread a wire through that space where the nerve lives, and it comes back out through the skin. We can then pull through thin, flexible devices that enable the surgeon to remove the tissue that’s pressing on the nerve without having to do as much unnecessary tissue removal.

Q: What sort of data are you gathering around your devices?

A: We have clinical trials that are enrolling right now that are single-arm trials that evaluate the use of the iO-Flex system and compare the outcomes to historical controls.

Basically, we are looking at standard spinal patient outcome measurement tools. There are four different measurement devices that are used to tell everything from patient pain to mobility. We also are studying the economics of it: the potential to save hospitals money by reducing operative time and length of stay, while improving long term outcomes.

The reality is that with devices these days, you just can’t simply add cost to a procedure; you have to demonstrate that your device either provides better patient care or that it somehow removes cost from the system.

We believe that we have potential to reduce operating time, but we are backing that up with clinical studies.

Q: When does that data come out?

A: We think we will finish enrolling the first of two studies in late 2011 and we will submit for publication the following year, so we have one year of data. So we would hope that we would have published data by 2012.

Q: How has the company been financed to date?

A: Last year, we closed a $30 million round of financing from some really top-tier investors, led by CMEA out of San Francisco. What was also validating for us is that one of the new investors is Kaiser Permanente Ventures. Kaiser, as a healthcare system, is typically very cost-focused, so for them to invest, it really made a statement that this is the technology with potential.

Q: Tell me about some of the company’s goals for the near future.

A: Our goal is to begin enrollment of a clinical trial that will demonstrate the utility of the device in lumbar spinal stenosis, as compared to some of the other techniques that are being done to treat the condition right now.

We also have goals set around numbers of surgeons that we want to train to use the devices.

We plan to expand our commercialization and we want to be able to, at the appropriate time, provide information to patients so that that they know there are alternative technologies and procedures that can help them.

Q: How well is Baxano positioned to fit within the parameters of healthcare reform?

A: We are really well positioned to address where healthcare is going. Payors are becoming more critical of technologies and procedures that do not have evidence of efficacy. Comparative effectiveness is the new direction in healthcare.

So, we think that this technology fits well with where healthcare is headed – less invasive, less cost.

This interview has been edited for length from its original version.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Brian Kennedy, CEO The Buck Institute

2011-03-30T12:18:00.000-07:00

Brian Kennedy

Brian Kennedy joined the Buck Institute in July following a career in academia where he earned international recognition for his work in the basic biology of aging. He became only the second CEO in the institute’s history, coming from the University of Washington in Seattle where he served in the Department of Biochemistry. Kennedy earned his doctorate from the Massachusetts Institute of Technology, where he conducted research with famed biologist Leonard Guarente, Ph.D., whose pioneering studies led to the discovery that Sirtuins (SIR2) modulate aging.

Projections show that six out of 10 Baby Boomers will be living with one or more chronic conditions by 2030, and the Buck has dedicated its resources to extending healthspan, the healthy years of an individual’s life. The independent nonprofit organization, based in Novato, Calif., is the nation’s first freestanding U.S. institute devoted solely to basic research on aging and age-associated disease. Buck Institute scientists work in an innovative, interdisciplinary setting to understand the mechanisms of aging and to discover new ways of detecting, preventing and treating conditions such as Alzheimer’s and Parkinson’s disease, cancer and stroke.

Q: What do we know now about aging that we didn’t know, say, 10 or 20 years ago?

A: You can look at it from different ways. From a mechanistic perspective, we know a lot more potential things that are happening in our body that are driving the aging process. But we still don’t know the relevant contributions of different things like mitochondrial dysfunction — all those things happen with aging but we still don’t know how each of them contributes to the diseases of aging and mortality.

We have made a tremendous amount of progress in two areas. One is in the discovery of modulating agents. I think that it is clear now that aging is not intractable. You can actually slow aging — in all the model organisms now, from simple things like yeast and worms to mice and rats. The second area is dietary restriction. Last year it was shown that dietary restrictions extended the lifespan of primates.

Q: What are some of the biggest misunderstandings when it comes to aging and age-related research?

A: One of the biggest concerns is that the FDA doesn’t consider aging a disease. Now, why is that? I think the main reason is that 100 percent of the population gets it. So it is kind of a semantic argument, but it’s a problem because if you don’t consider something a disease, you cannot conduct a clinical trial.

There’s a very small amount of money that gets spent by the federal government on aging research. It is tiny in proportion to cancer. The NIH spends about $5 billion a year on cancer, about $3.5 billion on heart and lung disease, $2 billion on diabetes. They spend a little over $1 billion on aging but half of that is on Alzheimer’s, which is what I consider money well spent but is not what I would call a basic aging property. What we are saying is that we are spending a lot of money on the effects of aging, but aging is one of the causes. If you want to do something effective, you try to fix the primary problem, not the effects. But at the same time, the budget is shrinking on a federal level and we have to find ways to raise the money we need to do this research.

Q: The Buck mission is to increase the healthy years of life. So, in other words, you aren’t looking to necessarily extend life but to improve healthspan? Or, simply, take the edge off getting old?

A: It has long been known that cancer, neurodegeneration like Alzheimer’s disease, diabetes – these things increase dramatically in prevalence as we get older. And what we’re learning is that aging is actually a cause of these diseases – it is one of the causes. If you slow aging, you’re going to get benefits across a wide spectrum of disease.

What we want to emphasize is that extending healthspan is the most important thing, but extending lifespan might come along with that. But if you just tell people that you want to extend lifespan then they think about their 85-year-old grandmother who is sick and they think, “I don’t want to live longer than 85 when I am sick.” Now, the truth is that none of the models that we study do that. What they do is they extend healthspan — they make animals live longer until they start to get sick.

Q: Is it true that by studying aging, we might, for instance find the cure for cancer?

A: I think that what we’re going to find is effective, preventative measures to avoid the onset of cancer. And that’s probably true for cardiovascular disease, too. Things that slow aging are really preventing the onset of disease, and it is not yet clear in some of those cases that if you waited until after somebody got sick and then gave them the drug, whether that would be beneficial or not. That is something that is being studied but it is unknown at this point. The great study last year with rapamycin, a FDA-approved drug given to mice at 600 days of age (equivalent to a 65-year-old human), demonstrated how mice lived 15 percent longer, and lived healthier. So, I think that if you had a drug you could give to somebody at age 60, and have that kind of effect, there would be a lot of excitement around it.

Q: Talk about some of your most recent research. How does, for example, a compound intended for liver preservation become one looked at for Huntington’s disease?

A: People are starting to really understand that a lot of these different diseases have related components. Even though the mechanism of the disease is different, the events that make you prone to getting the disease in the first place may be more similar. Aging is one of those things. So the idea now is that if you have a drug, especially one that is clinically approved for one disease, thinking about it on a broader spectrum level is a useful thing to do.

From an academic perspective, we are looking at the most effective route to take the basic understanding that we have about aging and translate that to effective therapies for diseases. And using things that we already know are safe is one route to go down that path.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Bill Lis, CEO Portola Pharmaceuticals

2011-03-15T12:09:00.000-07:00

Bill Lis, CEO Portola Pharmaceuticals

Bill Lis, CEO of CHI-member company Portola Pharmaceuticals has almost 20 years of experience in the biomedical industry. In 2007, he joined his old colleagues there after being at Scios Inc., a Johnson & Johnson company, for four years. He came in as chief business officer, was promoted to chief operating officer and then CEO. For Lis, it was a unique opportunity to join Portola as a senior executive and work again with a talented group of scientists whom he had had success with several years ago at COR Therapeutics. “We’re very passionate about thrombosis. It remains one of the most important areas for drug discovery and development because of the significant unmet medical need and our scientists have contributed several significant advances to the area over the past two decades,” said Lis in a recent interview with CHI.

“We’ve also begun to use our expertise and understanding of biological pathways and cell signaling in cardiovascular disease to expand our efforts into inflammatory diseases. What continues to emerge is the role of inflammation in cardiovascular disease and autoimmune disorders. So, I’m excited by the combined opportunities ahead of us.”

Q: What does the company look like today?

A: We now have roughly 86 employees. About 30 percent to 35 percent of them are people that have worked together for more than 15 years at COR Therapeutics and Millennium Pharmaceuticals. That is very unique in our industry and it’s what provides the foundation for the success we’ve had thus far. We also have a talented group of people who’ve joined Portola from other biotech companies and academia and they have enhanced our capabilities. What’s also unique about Portola is that we have fully integrated discovery research and development capabilities. We believe this gives us a competitive advantage and increases our R&D productivity.

Q: Who financed its early days?

A: Initially, we got funding from a very distinguished group of venture capitalists. We have, since then, added additional institutional investors or what you would call cross-over investors. These investors will, hopefully, help us expand and access a larger market for financing opportunities in the future.

Q: How did you get to the point where you are today, with roughly $1 billion in licensing deals with Merck and Novartis?

A: The value of Portola is really built around its people. These are people truly committed to the science of developing drugs that positively impact the lives of patients with very serious diseases and disorders.

As a team, we’ve executed on financing the company with private equity financing and on partnerships. But, as I said, we really got to where we are today based on the world-class R&D team that we have put into place. This is a unique team and we’ve built what we think is the appropriate amount of infrastructure and drug discovery and development capabilities. We really focus on the serious illnesses, the conditions that we think have the greatest medical needs, and then we’ve built compounds that we believe have pharmacologic properties that can significantly advance patient care over current standard-of-care agents.

Q: Talk about Portola’s lead development programs, elinogrel and betrixaban. What are these products addressing, from both a patient and a market standpoint?

A: As far as our lead products and their ability to address unmet needs — these are pretty straight forward. Elinogrel, a P2Y12 platelet receptor antagonist, and betrixaban, a direct acting oral Factor Xa inhibitor targeting the coagulation cascade, target a combined antiplatelet and anticoagulant market, for patients at risk for life-threatening blood clots. This market is projected to surpass $20 billion in the next several years. The products follow our R&D philosophy to discover compounds based on validated targets. Importantly, their pharmacologic properties are distinct from those of our competitors. Our goal now is to demonstrate in Phase 3 trials that these properties provide clear clinical advantages for patients. We hope to distinguish them broadly and for specific high-risk subpopulations.

Q: If you weren’t doing this, what would you be doing?

A: I have always had an interest in politics and world events. But I can’t think of any other industry that does more to improve human health and the quality of people’s lives worldwide.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Leland Wilson, CEO of Vivus

2011-01-11T10:40:00.000-08:00

Leland Wilson

Wilson has been a director of Vivus since the company was formed in April 1991 and chief executive officer since November 1991. Prior to joining Vivus, Wilson was vice president of marketing and corporate development of Genelabs Technologies Inc. from 1989 to 1991. Wilson was group product director, later promoted to director of marketing, at LifeScan, a Johnson & Johnson company, from 1986 to 1989. From 1973 to 1986, Wilson served in several research, marketing and sales positions for Syntex Research and Syntex Laboratories Inc. He received his bachelor’s and master’s degrees from Pennsylvania State University and was a lieutenant in the United States Army.

Q: Vivus is among a small group of companies seeking FDA approval of the first prescription weight loss therapy in over a decade. Talk about what this means to your company and to those who await such a treatment.

A: We are fortunate in that we have two late-stage products in development. Clearly, though, the excitement in the stock market is around Qnexa because of the Phase 3 data that we have and because of the lack of effective products in this therapeutic category, which, I think, by everyone’s recognition is underserved.

Q: Can you share with us some highlights of the top-line results for Qnexa?

A: It is never-before-seen weight loss and that would include average weight loss in the neighborhood of up to 15 percent, in some cases held over a two-year period. We also noted the weight loss caused massive changes in risk of major cardiovascular events, which could help prevent early morbidity and mortality due to cardiovascular disease.

Q: Qnexa is also being tested for sleep apnea. Who, among those nighttime sufferers, might stand to benefit from this therapy?

A: It’s a devastating condition that affects breathing – you stop breathing during the night or your breathing is slowed or interfered with so that you don’t get enough oxygen. It’s an area of unmet medical need for patients.

We’ll be doing additional clinical trials in this area once we get approval for Qnexa.

Q: You, like others in the space, received a complete response letter (CRL) recently from the FDA. Any advice for companies working directly with the FDA under similar circumstances, waiting to advance important new therapies?

A: We got the CRL “lite” and fully expect, with minimal work on our part, to have an acceptable new drug application (NDA) submission. We have resubmitted our response to them, and we’ll be holding a meeting with them this month. We’ll resubmit formally, hopefully, soon thereafter. They wanted us to analyze our data in such a way as to give them assurance that this slight increase in heart rate (about 1.6 beats per minute) does not have any long-term sequela as far as long-term morbidity and concerns.

Also, topiramate carries with it a concern for teratogenic potential (birth defects). The issue there was two-fold. One, was to inform by a collection of information around the teratogenic potential of topiramate, and also Qnexa. It is largely a literature search on topiramate.

Q: Its sounds as if you are optimistic about your submission.

A: I am very optimistic.

Q: What else is on the docket for 2011?

A: We’re going to put in a NDA for our second product, avanafil, and we have also filed a European submission for Qnexa.

Avanafil is a PDE5 much in the lineage of Viagra, Cialis, etc. But it has some superior characteristics – more specific for the enzyme we’re tying to target (phosphodiesterase type 5).

We have excellent results in after 15 minutes of taking the oral pill, which is as much as 45 minutes to an hour and 45 minutes faster than the leading compounds in the market.

Q: Is the strategy still to seek a marketing partnership for Qnexa following FDA approval?

A: Yes, clearly Europe is an area where we need a partner. I think we’re going to be in a very good negotiating position to drive a good deal.

Q: Any remaining hurdles as Vivus prepares to launch its weight loss drug pending FDA approval?

A: The biggest area we have issues with is reimbursement. CMS does not cover medical treatments for obesity. The paradox in that is bariatric surgery is covered, but as a medical treatment it is not. On the private side, I would say obesity is largely not covered, however, some employers who are progressively minded do cover it. They pay the extra money to prove that overall it is a better outcome for their employees.

Certainly, in the last year, there’s been a huge rise in the awareness around obesity, specifically the contribution to the growth of diabetes as well as healthcare costs.

We’ve worked diligently on a pharmaeconomics model submitted for publication around cost of obesity and the savings that can be incurred on the public health side with the simple reduction of 5 percent or 10 percent weight loss.

We have brought our clinical data to private payers and to a number of folks in the public health area and they are very impressed with our data. While we may not have an entirely welcoming reimbursement environment the day of our launch, we’re quite confident that, say, a year after our launch it will be a very different picture. I think people will understand what obesity does and how much of a root cause it is of some of these other disease areas.

Q: What keeps you awake at night?

A: Our job here is to get a successful resubmission. The data all looks very, very good. We have to get an A+ on this report we’re filing and it’s a big one – a massive amount of work and data analysis. It is about pulling together and getting this done in a timely fashion.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Dr. Ed Holmes, President and CEO of the Sanford Consortium for Regenerative Medicine

2010-12-07T10:32:00.000-08:00

Dr. Ed Holmes

The Sanford Consortium for Regenerative Medicine combines the intellectual resources of four of the world's leading biomedical research institutions (UC San Diego, the Salk Institute for Biological Studies, the Sanford-Burnham Institute for Medical Research and The Scripps Research Institute) to pursue collaborative research projects that advance the search for breakthrough cures. The Sanford Consortium unites San Diego's brightest researchers to work side by side to harness the regenerative power of stem cells to diagnose, treat and cure degenerative diseases and injuries. The Sanford Consortium integrates the collective knowledge of these foremost biomedical research institutions, with the support of surrounding industry and local community, to create a global resource for stem cell research.

Dr. Holmes is a distinguished professor of the University of California and vice chancellor/dean emeritus of Health Sciences at UC San Diego. He is also executive deputy chairman of the Biomedical Research Council and chairman of the National Medical Research Council, Singapore. Holmes has served on numerous advisory boards including the National Diabetes and Digestive and Kidney Diseases Advisory Council of the National Institutes of Health, the board of directors of Tularik and the scientific advisory board of GlaxoSmithKline, which he chairs. He is a member of the American Society for Clinical Investigation and the Association of American Physicians. He is also a fellow of the American Association for the Advancement of Science and a member of the Institute of Medicine of the National Academy of Sciences. He holds a medical degree from the University of Pennsylvania.

Q: Tell me about the origins of the Sanford Consortium.

A: We came into being after the bond issue (Prop. 71) was passed and the California Institute for Regenerative Medicine (CIRM) was formed. The San Diego community, including UCSD, Scripps, Salk and Burnham, decided to approach this by working together.

Q: The Sanford Consortium seems unique in that way. How did scientists in San Diego overcome the boundaries that can typically block them from coming together?

A: I think what’s interesting about this is it is really something that sort of happened on three levels. One, that the scientists actually wanted to work together. Also, the four institutional leaders (UCSD, Salk, Scripps and Burnham) saw an advantage to working together. And, then, we had community leaders in the form of Malin Burnham and John Moores and Irwin Jacobs in the beginning. And, now, Denny Sanford.

I think the four institutions and the scientists within them all working together is really what got us started. And that’s continued to be the foundation of what we do.

Q: Tell me about the building itself. What kind of progress has been made to date?

A: To get started, we need a place to house our folks. But it is important to recognize that, certainly, CIRM providing a $43 million major facilities grant was essential in this, but we would not have gotten there without the $30 million gift from Denny Sanford.

This is a beautiful location in every sense of the word—both views and the prime space. Chancellor Marye Anne Fox and UCSD helped us in that regard. This is one example of her vision—without her support we wouldn’t have gotten the loan we needed (public debt guaranteed by the UC Regents).

Denny gave us the $30 million and we’ll be using less than $20 million of that for construction of the building; the rest will be used to fund research. As for the rest, $43 million came from the CIRM grant and we sold $62 million worth of bonds, but it yielded $65 million worth of proceeds.

We are now on track for Sept. 22, 2011. We will be able to get in there July 1 and start installing equipment so that, by and large, we have a certificate of occupancy and, by the end of September, we’re ready to go.

Q: We’re at a dynamic time for stem cell research—especially with the first human trial on spinal cord injury. What excites you most about the research about to happen here?

A: We’ve begun to engage a wide group of scientists from all four institutions and you can see the genuine excitement. We’ve also begun to identify three theme areas to work in. Neurosciences, because that’s an important area for stem cell research. Also, in the area of cancer biology we’ve got a tremendous amount of strength, and in cardiovascular disease.

But what we’ve discovered is there’s so much more and we don’t want to exclude people, so we’ll probably concentrate on these three areas but embrace, though special projects, other things that would be important to bring into this.

We’re in the position to choose among the very best people, but, at the same time, we envision the consortium to be more than this building. It’s the science but it’s also all four institutions. Just because you’re not located in the building does not mean that you’re not a part of the Sanford Consortium. So, they’ve come up with some clever ways to engage people who might not be in the building long-term with special projects.

Q: Are there plans for engaging the local business community?

A: What we see as a tremendous opportunity as we build this out is, “How can we leverage what we have with partnerships in the private sector?”

One example is the basement in this building has a phenomenal capability for doing preclinical imaging studies that we think would be valuable to our investigators, but might also represent an opportunity to partner with the private sector. Some biotechs, particularly small biotechs, who cannot afford to buy some of the equipment that we will have. We would like to leverage both our science and our resources to work with this private sector and we’re just beginning our conversations.

We eventually want to do something with people. And it’s very hard to come up with things you do for people that don’t involve collaborations with the private sector. So, we have begun some very preliminary conversations right now with some of the biotech people in town. We’re also interested in talking to pharmaceutical and medical device leaders.

Q: A lot of this depends on the leveraging of federal funds and right now this is still up in the air, with stem cell research held up in the courts. What concerns do you have there?

A: Nobody can predict what’s going to happen in Washington and obviously it’s an up and down situation. But I would say the disquiet in Washington has been a boon for California. You find people who want to come anyway, but they want to come even more now. It’d be really nice if the federal government gets it sorted out—and I, personally, think they will. I’m optimistic the federal government will straighten itself out and begin to make investments.

Q: What kind of design plans does the building call for to get scientists working collaboratively?

A: For collaboration to work, first you need opportunity. And you need to run into people at the water cooler. We have rooms intended to reduce certain types of behavior and encourage collaboration. We have team rooms so six people can hook up computers at a time. It’s a smartboard, so they can write on the screen so it captures the image plus what they annotate on their screen.

We also learned that scientists go 10x horizontal before they go 1x vertical. Or, as we say, “The ability to collaborate is inversely proportional to the square of the distances.”

The building calls for wide open staircases and large open spaces that connect. It should allow people to more vertically circulate.

Q: Did you model this building after any other facilities in the country?

A: The exterior was designed to acknowledge that it has to fit contextually with the iconic structure that is the Salk. It started with the scientists back in 2007. They said, “This is what we think we need.” We had a lab planner work with them and come up with a program. And the architects then worked with the program to develop it.

This is a LEED gold building. We’re using chilled beams throughout.

We’re also taking advantage of the fact that we live in a great part of the world. We face the ocean here on top of the Torrey Pines Mesa, which allows for some fantastic views. We plan to build an open-air reception space and fully take advantage of the Southern California climate.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Cheryl Hankin, Founder and CSO of BioMedEcon

2010-11-22T15:28:00.000-08:00

Cheryl Hankin

Cheryl Hankin, Ph.D., is the founder of BioMedEcon and serves as the company's president and chief scientific officer.

Hankin’s experience in health economics and outcomes research includes roles as director of outcomes research Johnson & Johnson companies, assistant professor at Boston University School of Public Health and research health scientist at the Department of Veterans Affairs, Health Services Research and Development Center of Excellence.

BioMedEcon provides health economics and outcomes research for pharmaceutical, biotechnology, drug delivery and medical device companies. Hankin offers extensive experience in comparative effectiveness research, competitive product analysis, and pricing, coverage and reimbursement.

Q: It looks like your career has spanned the industry, academia and the public sector. What led you to start BioMedEcon?

A: Several years ago, I was in the public sector working with post-traumatic stress disordered veterans in the VA system and noticed that they weren’t getting better. It occurred to me that we had large scale claims databases that provided all sorts of information about the types of care, medications, surgeries, procedures these patients received. This was maybe 15 or 20 years ago and I said, “Why not mine this information to find out what works best for these patients?”

Q: At the time were you involved in patient care?

A: Yes, I was a clinical psychologist working primarily with extremely traumatized patients. I was so interested in identifying best practices for these patients that I applied for and received grant funding from the VA to complete dual post-doctoral fellowships in health services research and mental health. I became a research scientist at the VA Health Services Research and Development Centers of Excellence, where my interests broadened to include other illnesses such as cardiovascular disease, diabetes, cancer, etc. I had very early and excellent training in this field before it was in vogue.

Q: How did your career progress from there?

A: ALZA Corp., a drug delivery technology company, had a need for a health economics and outcomes researcher. This was my first foray into private industry. ALZA was acquired by J&J and I joined J&J to head up outcomes research for one of their companies on the East Coast.

Prior to receiving my doctorate, I was a corporate accountant working with Silicon Valley hi-tech start-ups. I really enjoy that entrepreneurial energy and longed to return to it. So, in 2004, I started my own company and I have never looked back. Health economics and outcomes research is the perfect combination of my skills and interests in patient care, research, corporate finance and marketing.

Q: What is the primary focus at BioMedEcon today?

A: We work with new and emerging companies as well as well-established pharmaceutical manufacturers to identify and demonstrate the value of their products. The therapeutic “space” we work within includes respiratory disease, acute and chronic pain, dermatology, diabetes, obesity, endocrinology and psychiatry. Our clients find us solely by word-of-mouth referrals based on the recommendations of those who know our work, and our client relationships are long-standing.

Q: What kinds of questions are you answering?

A: In this highly competitive and cost-conscious environment, we serve as the bridge between clinical research and commercialization with answers to questions such as: What is the unique value proposition of our client’s product and what is the most meaningful manner in which to demonstrate this value to health plans, providers and patients? Our mission is to optimize product access, coverage, pricing and reimbursement. We collaborate closely with public and private payers, clinical opinion leaders, high prescribers and patient advocacy groups to ensure that these value propositions are meaningful and compelling.

Q: How, then, do you go about demonstrating that value?

A: We use a variety of techniques to demonstrate value. We may re-examine clinical trial data, create study protocols, identify additional endpoints for inclusion in pivotal trials, conduct market research with payers and providers, prepare systematic literature reviews and meta-analyses, develop pharmacoeconomic models, convene expert panels and advisory boards, examine large-scale retrospective databases, develop and validate measures, conduct chart reviews—the possibilities for creative approaches are endless. We also develop and implement publication and presentation plans to ensure that these compelling and meaningful results are appropriately disseminated.

Q: With comparative effectiveness an important piece of the health reform legislation, what’s happening in this area for BioMedEcon?

A: It’s certainly a burgeoning field, and we are seeing a growing interest in our capabilities from new types of "consumers." For example, physician groups and professional associations have asked us to help them demonstrate their ability to provide "accountable care." In another case, a professional medical society has asked us to identify patient and provider characteristics associated with variation in patient adherence to treatment. In yet another example, a healthcare institution has just asked us to examine risk factors associated with the occurrence of specific “never events” (adverse events that should never happen and that are not reimbursed by CMS, such as hospital-acquired infections). So, our client-base now extends beyond the traditional pharmaceutical industry. I believe this is due to both the potentially broad impact of health reform legislation on all sectors of healthcare delivery and to our proven track record in demonstrating value.

Q: What keeps you awake at night?

A: I love the work that we do. I stay awake at night not because I’m worried or unhappy, but because I get carried away with the intricate and delightful puzzles we are presented with. I may be thinking about an economic or financial forecasting model, or a presentation we’re preparing, and before I know it, the hours have slipped away. I’m very lucky. I can’t think of a more enjoyable occupation.

CHI-Advancing California biomedical research and innovation

Legislator Spotlight: Assemblyman Jerry Hill (D-San Mateo)

2010-10-18T14:06:00.000-07:00

Assemblyman Jerry Hill

Jerry Hill was elected to the California State Assembly in November 2008. In Sacramento, Assemblyman Hill has demonstrated an ability to reach across party lines. This year, 19 out of his 20 bills approved by the Legislature and sent to the governor received bi-partisan support. Hill chairs the Majority Caucus in the Assembly and is a member of Speaker Perez' leadership team, where he is responsible for negotiating key issues and guiding legislative priorities. As chair of the Select Committee on Biotechnology and as a member of the Committee on Improving State Government, Hill has tirelessly tackled some of the most pressing issues facing San Mateo County and California, including government efficiency and economic development. He is working closely with transportation officials and residents on issues related to the construction of a high-speed rail through San Mateo County. He has also assumed a leadership role in the investigation of the San Bruno natural gas pipeline explosion and in the shaping of a legislative response.

Hill’s district includes the cities of Belmont, Brisbane, Burlingame, Daly City, Foster City, Half Moon Bay, Hillsborough, Millbrae, Pacifica, San Bruno, San Mateo, South San Francisco and parts of unincorporated San Mateo County.

Q: As a legislator from San Mateo County, your interest in the biomedical industry seems quite natural. How do you promote the value of the industry to your colleagues in other parts of the state that may not have as strong a biomedical presence?

A: It’s important to start, initially, with the tremendous benefit that’s derived from the industry. I’ve been to BioMarin Pharmaceutical in Marin County, Exelixis in South San Francisco, UCSF, Edwards Lifesciences in Irvine, Life Technologies in San Diego, etc. With each one comes the realization of the life-saving products, devices and therapies, that are being developed through this industry.

The first part is making sure my colleagues and members of the Legislature are aware of what is actually happening in California. It’s important that they know these companies exist and how crucial the therapies and devices that they manufacture are to modern medicine. Each one of them should be able to identify someone in their family who has benefitted from this, whether it’s a cancer therapy from Genentech or a heart valve from Edwards Lifesciences.

Q: Do you see the potential for ramping up California's biomedical manufacturing capacity in communities hard hit by the recession in the interior parts of the state?

A: I see that as a real potential, however, I also see our tax structure as a deterrent to that. One of the stories that I tell repeatedly is the fact that Genentech, obviously a homegrown San Mateo County company, built a facility in the last year in Hillsboro, Oregon. What we’ve lost is the benefit, to some extent. When these companies begin they are local. They have local roots, they generally have families in the Bay Area and people don’t want to leave.

In this economic downturn, the biomedical sector is one of the only areas that actually increased in employment. Once legislators see the lifesaving benefit, then they can also see the economic benefit in terms of good, high paying jobs.

Q: Let's discuss Prop. 24. As you know, California has had difficulties in attracting and retaining existing companies, let alone developing missed opportunities such as biomedical manufacturing. How do you think Prop. 24 plays into that conversation?

A: I certainly don’t support it. I think its bad public policy. When I explain to anyone how the current tax structure works, in practicality, people say, “You’re kidding.” They ask, “Build a bigger plant, hire more employees, pay more money and I’m going to pay more income tax?” And, I say, “Yes, that’s true.” They don’t believe it; they think it’s crazy. But, sadly, that’s not how it’s playing out in the press or in the campaign. The biomedical business is unique. And, that’s the other part we have to convince my colleagues about, the uniqueness of biomedical industry. That it takes 10 to 15 years for a therapeutic to be developed, a billion and a half dollars and, at the end of the day, the odds of producing a successful therapy are slim.

Q: What can the biomedical industry do to better educate legislators and others about the value of our work?

A: To me, it’s a matter of communication, and I think the industry has done a good job of being there and involving legislators on issues that are important to them.

I was in Sacramento the other day and Johnson & Johnson had an exhibit at the California Museum on their biomedical devices. It was open to staff members and I went because of my involvement and interest. That’s what’s important and what I hope to work with the industry on early next session, to bring the industries either here or to organize road trips to their locations.

To sit at Edwards and watch 400 employees making heart valves, that take eight hours to make, is phenomenal. I think you have to see that and really understand the magnitude of the industry and the benefit that it brings to this state.

We have to do this early, instead of six months or a year later when this legislation affects them. By then, it’s too late.

Q: Talk a little about the importance of STEM education. I know you pushed to restore cuts to UC and CSU as part of the budget negotiations. How well are we funding science education?

A: We have done a poor job, lately, in supporting and funding science education in California, especially at the K-12 level. We don’t compensate teachers enough to bring highly trained people into the field to teach science at a level we need to develop an educated workforce. Fortunately, we have a higher education system that has been able to attract from all over the world. That is truly our lifeblood for sustaining our leadership in the sciences. We want it to be homegrown, though, and we have to put the resources to develop better science education in our K-12 and sustain it at the college level.

Q: What do you consider some of your biggest accomplishments as the Chair of the Select Committee on Biotechnology?

A: I think the biggest accomplishments have been that we have really done what we have set out to do, which is highlight the industry, to really look at it from a number of areas.

We were able to educate, inform and enlighten colleagues on the value of the industry, which is, I think the most important part. Because of term limits and how fast time flies, people don’t realize that every two years a third of that Legislature changes.

CHI-Advancing California biomedical research and innovation

Event Spotlight: Larry Bock, Executive Director, USA Science & Engineering Festival

2010-10-07T13:29:00.000-07:00

Larry Bock

Each year, the San Diego Science Festival plays host to hundreds of thousands of people who participate in engaging, hands-on science activities across town, concluding with a free, two-day Expo held downtown.
Planned as the West Coast’s largest science event, the festival has attracted corporate and individual donors dedicated to encouraging the nation’s youth to pursue careers focused in science, research and mathematics.
This year, following a successful San Diego event in the spring, USA Science & Engineering Festival Executive Director Larry Bock was inspired to bring the fun to our nation’s capital during October. Bock, who was the inspiration behind the popular San Diego festival, is a successful serial entrepreneur who has founded, co-founded or financed the early stage growth of 40 companies in the life and physical sciences from inception to achieving an aggregate market capitalization in excess of $30 billion.

Q: I understand this event is geared toward reinvigorating the interest of Americans in the sciences. Explain the root of the issue and how it might be reversed?

A: There is a perfect storm of events going on. Americans are not pursuing advanced science positions, and, because of H1B visa issues, we are not retaining foreigners who are trained in the area. Right now, something like 80 percent of people being trained with advanced degrees in the sciences in the United States are from abroad. Also, the opportunities are now greater abroad. So, even if we did try to retain them, they are more motivated to go back. We have to reinvigorate the interest of Americans in science and math. And why aren’t Americans interested in it? Our premise is our society gets what it celebrates. We celebrate Lindsay Lohan and Britney Spears and we generate a lot of them. But we don’t celebrate science and engineering. We need to celebrate the rock stars of science, such as computer scientists Larry Page and Sergey Brin who founded Google.

Q: Do you envision a day where we’re celebrating scientists as celebrities?

A: That’s our goal. We try to do that by having a festival where it’s a pinnacle moment for society to come together to celebrate science and engineering, art, music, film and comedy. What the festival’s organizers are trying to do is bring scientists into schools to show them that scientists really are rock stars. This year, we’ll bring 25 Nobel Laureates into schools to have brown bag lunches with students. We also have 100 scientists to go in and really get kids excited about what scientists do.

Q: How does this event tie into classroom-based teaching?

A: Basically, we try to get these scientists to come into the schools for a presentation but then form a long-term relationship with the schools. We’re bringing in scientists like National Institutes of Health Director Francis Collins, AIDS researcher Tony Fauci and Paul Anastas, the “father of green chemistry” at the Environmental Protection Agency. Science and engineering professionals will fan out across the D.C. area in October to speak about their careers at various middle and high schools.

Q: How did the San Diego Science Festival help you prepare for this year’s event in D.C.?

A: I wasn’t planning on doing it in D.C., but one of my sponsors in San Diego, Lockheed Martin, encouraged me to do it on a national scale and they gave us a huge sponsorship to do that. They said, “What would it take to make it a national event?” With the generous help of our corporate sponsors, we have raised over $2 million, along with $2.5 million in media partnerships in under a year.
In Washington, we have organizations from all over participating. We have probably over 750 organizations participating. We have approximately 150 universities. We have well over 100 professional science and engineering societies. We have about 100 government agencies and federal labs, all of which are based in Washington.

Other community-based organizations, such as Girl Scouts and Boy Scouts and Project Lead the Way, are also participating on the premise of giving back to the community with the added bonus of learning something about science.

Q: Give me a few of the event’s highlights.

A: We have exciting guests like the MythBusters (Discovery Channel), Bill Nye the Science Guy, Storm Chasers (Discovery Channel), the inventor of the Rubik’s Cube. Then, we have the sciences of pretty much anything you can imagine, including the physics of NASCAR, the chemistry of Thanksgiving dinner, the science of Harry Potter. Through the generous contribution of time, energy and resources of our participating organizations, we are also able to offer sophisticated exhibits like virtual reality environments, surgical robots and fighter jet simulators.

Q: Give me an idea of what’s involved in the science of Harry Potter.

A: It’s actually looking at the real science behind the magic of Harry Potter. There is real science now to do cloaking, which is what Harry does when he puts on the invisible cape.

Q: How did you first get involved in this event?

A: I was taking a year abroad in Europe for fun when I saw the science festivals there. In Europe they’ve been going on for years, taking place all over England, the United Kingdom, Italy, Germany. The one in India draws 1 million people to it. That was my impetus to do the inaugural San Diego Science Festival. I loved the month of activity, but, for me, the really “aha” moment was when Balboa Park was completely filled. In the Spring of 2009, the inaugural San Diego Science Festival drew 200,000 people to participate in more than 500 free activities.

Q: What will be unique about this year’s event?

A: We’re on the National Mall, from the U.S. Capitol Building to 7th street. And then we’ve closed down Pennsylvania Avenue between 12th and 14th Streets. I think we have one of the largest footprints of any expo in Washington separate from the inauguration.

Q: How many people do you anticipate will attend the event?

A: Well, of course it’s weather dependent. My goal would be for a quarter of a million people each day during the Expo Days, held Oct. 23 and 24. Our goal is also to have a million people nationwide involved in this celebration, between what we’re doing and all the satellite events going on nationwide.

Q: At the end of the day, what do you hope to achieve?

A: My dream for this event is that it will have been considered the “Woodstock of science.” Hopefully, with less rain.

Click here to find out all the details of this year’s event already underway in Washington, D.C.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Terry Gregg, President and CEO DexCom

2010-10-01T11:46:00.000-07:00

Terry Gregg, DexCom President & CEO

Terrance H. Gregg is president and chief executive officer of DexCom Inc. He is also a special venture partner with Galen Partners, a private equity firm specializing in the healthcare industry. In 2002, he retired as president of Medtronic MiniMed, a world leader in diabetes management systems. He became president and chief operating officer of MiniMed Inc. in 1996 and was instrumental in Medtronic's $3.4 billion acquisition of MiniMed in 2001. He also served in executive positions with Smith and Nephew and Allergan Inc. Gregg served as the 2003-2004 Chair of the Research Foundation Board of the American Diabetes Association. He received his bachelor’s of science from Colorado State University in 1971. In 2003, Gregg and his wife were recognized by the American Diabetes Association with an award for Outstanding Service in Diabetes Research Funding.

Q: Share with me a brief history of DexCom and some of the important milestones of the company.

A: Dexcom designs, develops, and commercializes continuous glucose monitoring systems for managing diabetes. The company was really founded in 1999. The premise of that original founding was to develop a fully implanted subcutaneous sensor that would reside in place, in the abdomen, for upwards of a year, maybe 18 months. At the time, reliable, home-based continuous glucose monitors had yet to hit the market.

But by the mid 2000s, it was clear that the opportunity for a home based continuous glucose monitor that provided real-time values to the patient was going to be big.

Around 2003, it became apparent to the company that the amount of resources, capital, etc., in order to commercialize that product, would be more than the company could raise. But they took the lessons learned and the knowledge and then converted the product into an ambulatory one, for use at home.

A three-day sensor was approved by the FDA in March 2006. About a year later, June in 2007, the product was then extended to seven days. That became what we call the Seven System. In March 2009, the next generation, the Seven Plus System was approved by the FDA. That’s a product we have on the market today. The fourth generation sensor is currently pending PMA review by the FDA.

Q: You spent most of your career with Medtronic before retiring. What brought you out of retirement to join DexCom?

I have, personally, been pursuing a hospital glucose sensor since around 1997. One of the reasons I came out of retirement to join DexCom was that I believed that they had a sensor that would be adequate for that intensive care environment. Studies show that when a patient's blood glucose level is high for prolonged periods, recovery from surgery takes longer and the risk of infection increases. Currently, tight glycemic control is achieved by obtaining samples every 30 to 60 minutes, however this testing practice may be too intermittent and cumbersome. So, we developed a blood-dwelling sensor that goes into a peripheral vein for continuous glucose monitoring and we licensed that technology in co-development to Edwards Lifesciences for the ICU. It is pending before the FDA although it has received CE marks in Europe.

Q: What other work has been going on at DexCom?

A: We’ve engaged in two relationships with pump companies, one being J&J Animus (that product will go into the FDA at the end of this year) and the second relationship is with Insulet Corp., which has a disposable insulin infusion system called the Omnipod. That product is actually pending review before the FDA now.

Q: What would you like to call attention to when it comes to diabetes?

A: I think that the most compelling thing that one can do now is to encourage patients to take on their own responsibility for the treatment of their disease. If you look at what the future holds, we’re not going to be able to adequately afford the care that patients will need. Unfortunately, diabetes is growing at a rate that we can’t stop it. We have to educate patients and patients have to be more demanding.

Q: Given what we know and how much work has left to be done, how far are we from an artificial pancreas? One that can monitor blood glucose levels in real-time and respond to them by releasing insulin just as a normal pancreas would?

A: I would say we’re in the five- to 10-year horizon to get to a semi-closed loop system. We’re also unfortunately dealing with a regulatory arena that is more restricted than we’ve seen in a long time. I think two things are going to happen. There will be delays in development in the U.S. And, at the same time, we’re all looking outside the U.S. as fertile ground to move our newer technology. But reimbursement is a concern. It’s an interesting dynamic that we are going to struggle with at least for the next four years, in my opinion.

Q: DexCom recently worked with the FDA on changes sought by the agency in the way DexCom labels certain glucose monitoring devices. Any advice for companies going through similar issues?

A: Read all the guidance documents you can and interpret them in the most conservative way you can. I think the second thing is be very proactive but cooperative with the agency. Working to a joint conclusion that is in the best interest of the patient is really what the FDA wants. I think companies want that as well. Certainly we continue to have what I consider to be a fabulous relationship with the agency. Even in spite of the warning letter, there was no interruption of the review of our files.

Q: What are you doing when you’re not in the office?

A: This isn’t a job; this is a passion. I get to do this. Certainly, I have fun. We have adult children and two grandkids that live in San Diego County. We have a beautiful sailboat that I get down to once in awhile. Certainly, I am blessed in that I have been remunerated more than I ever should be. My wife and I are strong philanthropists. To date, I think we’ve given over $2 million to diabetes research and we will continue to do things of that nature. It’s something we believe very strongly in. When you are blessed like that you should give back.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Stanley Crooke, Founder, Chairman & CEO, Isis Pharmaceuticals

2010-09-22T15:15:00.000-07:00

Stanley Crooke, CEO of Isis Pharmaceuticals

Dr. Stan Crooke is founder, chairman and chief executive officer of Carlsbad, Calif.-based Isis Pharmaceuticals. In 2006, he was named in Nature Biotechnology as one of biotechnology’s influential individuals. Crooke is currently on the board of Regulus Therapeutics Inc., a company jointly owned by Isis. Prior to founding Isis, Crooke was president of research and development for SmithKline Beckman Corp. (SKB). He also coordinated the research and development activities of SKB including its instruments, diagnostics, animal health and clinical laboratory businesses. Prior to joining SKB, Crooke helped establish the anticancer drug discovery and development program at Bristol-Myers, which succeeded in bringing to market a significant number of drugs. During his career, Crooke has supervised the development of 19 drugs on the market and others in development. He received his master's and doctorate degrees from Baylor College of Medicine and his bachelor's in pharmacy from Butler University.

Q: Share with me a brief history of Isis. How did the company first get started?

A: I founded it 21 years ago to pursue what was a novel idea at the time that had no data, which was the notion of antisense technology. We’ve persevered in that process in what I think has proven to be an important enterprise. When I started the company I said the probability of our success was near zero and that it would be 20 years and at least $2 billion before we really knew, and I think that’s been proven the case.

Q: Tell me what antisense technology means.

A: It’s the only direct route from genomics to drugs that I know of. It’s the use of chemically modified oglionucleotides (short chains of nucleotides made synthetically to replicate the human DNA sequence) to interact with specific messenger or other RNAs to cause their function to be disrupted.
Think of it as the third platform for drug discovery. Small molecules are 120 years old now. Protein therapeutics are really quite old, but monoclonal antibodies are about 35 or 40 years old. We think antisense will take place alongside those as a third platform.

Q: How did you get to the point to where you were discovering and developing drugs that bind to RNA instead of proteins?

A: I’ve always been interested in RNA as a place in drug action. I feel the proposition is simpler. It seemed to me that it was time to evaluate whether one could create oglionucelotides that had good drug properties. And, if we could, then the hope was antisense drugs would be more specific because you can build them to be gene-specific. And the technology would be much more efficient than small molecule or protein-based drug discovery because the basic rules of how the oglionucleotide interacts with the target RNA are understood.

Armed with that information, then it becomes a much more rational process for drug discovery. All of those things, I think, have been borne out as we’ve made progress over the last 20 years.

Q: Talk a little about Isis’ strategy to license drugs in the earlier stages of development. I hear you’ve generated more than $1.6 billion from the successful execution of this partnership strategy?

A: It’s really quite simple. With small molecule and protein therapeutics you never know when you’re going to get another drug.

So, the idea was to invest in innovation tied to a technology that was sufficient enough that we could generate large numbers of drug opportunities per unit of time with a small group of people. And not to build an infrastructure that gets in the way of innovation. Our goal has been to keep the company small. It’s a mantra here at Isis that we intend to stay small, around 300 to 400 people, which is where we are now. And, armed with that technology, we can create five new drugs a year. Today, we have 23 drugs in development. Next year, we’ll have 28. And we will do the discovery, the early development to proof of concept and then let organizations that are geared to the brute force of Phase 3 and marketing and sales do that.

Q: What’s on the immediate horizon for Isis?

A: Mipomersen, our Phase 3 cholesterol-lowering drug being developed with Genzyme, gets filed in the first half of next year, in the U.S. and Europe, for the first group of patients. These are patients who have severe high cholesterol and extreme cardiovascular risk. Then, there will be subsequent filings for an ever-growing volume of patients and long-term safety. It’s going to treat people who have high cholesterol, who can’t get their cholesterol down with existing drugs and who can’t take statins. It’s an exciting drug that’s going to make a big difference in patients’ lives.

Q: When you’re not at work, where else do you spend your time?

A: My wife and I have a home in Sedona, Ariz. so we like hiking in the red rocks. Other than that, I like to read, play chess and generally goof around.

CHI-Advancing California biomedical research and innovation

Childhood Obesity Summit Calls Attention to Public Policy

2010-09-15T11:30:00.000-07:00

Michael Goran, founding director of USC's Childhood Obesity Research Center (left), and Dr. Kenneth Moritsugu of the Johnson & Johnson Diabetes Institute tackle the topic of childhood obesity. Photo courtesy USC.

Given the knowledge that childhood obesity rates have more than tripled in the past 30 years, lawmakers and healthcare leaders convened Sept. 10 on the campus of the University of Southern California to discuss ways of tackling the problem at home, in the schools and through public policy decisions made at the local and national levels.

To find out more about this issue and its related healthcare disparities among racial and ethnic groups, I traveled to Los Angeles to attend Childhood Obesity: A Call to Action.

The event, organized by CHI-member USC in cooperation with members of the Congressional Hispanic Caucus, Congressional Black Caucus and Congressional Asian Pacific American Caucus, was designed to help lay the groundwork for federal policy as Congress considers legislation regarding elementary and secondary education, child nutrition and transportation programs.

Rep. Lucille Roybal-Allard (D-East Los Angeles), who represents USC's Health Sciences Campus, led the congressional delegation attending the event, which included Reps. Michael Honda (D-Silicon Valley/San Jose), Judy Chu (D-East Los Angeles/El Monte), Grace Napolitano (D-Suburban Los Angeles), Diane Watson (D-Central Los Angeles) and Donna Christensen (D-Virgin Islands).

Roybal-Allard called childhood obesity a “national crisis” and noted how research demonstrated that obese youth were more likely to become obese adults. The summit highlighted some of USC’s own research and policy expertise in childhood obesity, particularly among racial and ethnic minorities. One program, initiated by USC Pharmacy School Dean Dr. Mel Baron, used comic book-like “fotonovelas” to increase awareness of diabetes and related health issues among Latino communities.

Los Angeles City Councilwoman Jan Perry spoke of the efforts to eliminate so-called “food deserts,” or regions where fast-food restaurants dominate but access to grocery stores selling fresh fruits and vegetables is limited to none.

And an effort to improve the accuracy of health messages on television and in movies is gaining traction, according to Sandra de Castro Buffington, who serves as director of USC’s Hollywood, Health & Society program.

Corporations, too, are doing their part, as exemplified by Dr. Kenneth Moritsugu of the Johnson & Johnson Diabetes Institute.

He mentions, though, that we have a complicated route ahead. “Twenty-six percent of Americans are obese and we have no good data on type 2 diabetes,” he said.

Overall, the program served as an excellent example of how such a large problem has many solutions, and good public policy decisions and corporate leadership can lead the way.

For more information on the Childhood Obesity: A Call to Action event held at USC, click here.

For more information about childhood obesity, visit the website for the Centers for Disease Control and Prevention.

CHI-Advancing California biomedical research and innovation