Speaker Spotlight: Roy D. Baynes, M.D., Ph.D., Senior Vice President of Oncology Therapeutics, Gilead Sciences

Dr. Roy Baynes

Dr. Baynes serves as senior vice president of oncology therapeutics at Gilead Sciences Inc. of Foster City, Calif. There, he oversees a pipeline of products focused on treating chronic lymphocytic leukemia, indolent non-Hodgkin’s lymphoma, colorectal cancer, myelofibrosis and pancreatic cancer. “We stand at an incredibly exciting time in cancer research and drug discovery,” said Baynes, who came to Gilead from Amgen, where he served as vice president of global development and as therapeutic area head of hematology oncology. Baynes received his medical degree and doctorate from the University of the Witwatersrand, South Africa.


Q: Cancer is an incredibly challenging field, full of discovery and of setbacks. What first drew you to this kind of research?


A: Firstly, it was about the science. The molecular and biological understanding of disease has always been an integral part of cancer research. In part, this relates to accessibility to tissue from biopsies or, in the case of hematological malignancies, from blood.


The second driver was really an intensely personal one. Most people are touched directly or indirectly by this dreadful disease, cancer. I have had too many close friends and family members who have either done battle with cancer or are doing battle with cancer. I’ve always taken the view that cancer patients just simply cannot wait, which has given a great sense of urgency to cancer research.


And, thirdly, there was the opportunity. Most people who come through academic programs are reasonably multi-talented and can go in any direction. So, oftentimes, it’s a combination of scientific interest, personal motivation and the opportunity. At the time I was ready to start my clinical and research career, the biggest opportunity was in the oncology and hematology arena. I fell in love immediately with the discipline and have been there ever since.


Q: Knowledge about cancer genotypes has led to greater, more targeted treatment for women with breast cancer, especially, and other cancers. What is on the horizon for molecularly targeted therapies?


A: We stand at an incredibly exciting time in cancer research and drug discovery. The biological underpinnings and basis of disease are becoming more clearly understood. Our ability to segment and select patients who will ultimately benefit from specific approaches is being enhanced on a daily basis.


The unmet medical need remains huge. You pull together the rapidly advancing science and biology and the unmet needs related to this grievous condition, cancer, and it’s a very compelling area to be working in.


Gilead has a tremendous history of medicinal chemistry and antiviral drug development. About two years ago, Gilead made a decision to get back into the cancer arena, driven largely by the notion that the molecular understanding had reached a level of sophistication where indeed it lent itself to meaningful drug development.


The company is working quickly to advance the cancer arena and, more specifically, the targeted therapeutics arena. And a lot of this has been built on licensing and acquiring assets. It’s been a thoughtful, deliberate process.


Q: Are there policies at the local, state or federal level that have a significant impact on your work?


A: When it comes to drug discovery, it’s important to begin with the end in mind. I think our goal is to come forward with innovative medicines that can address serious unmet medical needs and transform the lives of patients. So, that’s our guiding principle and, I think, if one stays true to that mission, the policy considerations have to fall in line.


Q: What are you most looking forward to in terms of our July program, “California: Uniting Science & Policy to Advance Cancer Care?”


A: CHI has developed an energizing program with a diverse range of speakers and very topical discussion points relating to oncology. I’m particularly looking forward to the session I’ll be moderating, “The State of Cancer Research in the Golden State,” because I think the Golden State has a tremendous amount to be proud of. As the birthplace of biotechnology, and as home to several prominent academic research centers and a strong biotech industry presence, California has played a key role in the personalized medicine innovation we are seeing in oncology today.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Bernd J. Larsen, President & CEO, CODAN USA

Bernd J. Larsen

CODAN in the U.S. is a part of the privately-held, European-based CODAN group of companies, which for more than 50 years has been a market leader in the area of I.V. drug delivery systems. CODAN’s core business is the manufacturing and sales of I.V. sets and accessories for both infusion and transfusion therapy. Larsen has been president of CODAN in the U.S. for 11 years and previously held senior-level executive positions with Maersk Medical in Denmark, Germany and the U.S.

Q: Share with us a brief history of the company and describe your products.

A: CODAN was founded in 1959 in Denmark. Our first U.S. facility opened in 1971 in Burbank, Calif., primarily focused on product assembly and sales. In 1997, the company was moved to new more spacious facilities in Santa Ana, Calif. where we are today. I should perhaps mention that, as of a few years ago, we also operate an assembly facility across the border in Tijuana, Mexico. We manufacture both private-label OEM products built to customer specifications as well as CODAN branded products, including CODAN for Kids for the neonatal/pediatric arena, specialty anesthesia sets and blood sets sold under the name Walrus by CODAN, CODAN Rx for the hospital pharmacy, CODAN CYTO for the delivery of cytotoxic drugs, and a catalogue of other miscellaneous I.V. products.

Q: What are your biggest goals for the year ahead and beyond?

A: Our management team recently completed our “Road to 2017” strategy plan and this plan outlines how we will transition the company emphasis to CODAN labeled products while continuing to offer private label products. The CODAN labeled products will be spearheaded by our anesthesia product line where we have established an excellent reputation with anesthesiologists and where we can leverage our leadership position in raw material, manufacturing, inspection and sterilization processes, and in packaging and distribution networks.

Q: If you could change a public policy at the federal or state level, what would it be?

A:  There is some uncertainty at FDA around the 510(k) regulatory process and an opportunity to improve the predictability of these processes to speed the delivery of I.V. drug delivery systems to patients.  

Q: What is the most rewarding part of your job?

A: Working with good people inside and outside of the company is very rewarding to me. It has also been rewarding to see our company’s impressive growth in new products and revenue over the past four years, despite a tough economy.

Q. Describe your leadership style.

A. I try to follow an old management philosophy: “He who informs inspires! He who ignores injures!” I strive to give our employees as much information as possible about the company and where we are going vis-à-vis our targets so they feel trusted and an important part of what we are working to achieve as a company. I also believe in letting people do their jobs and not micromanaging them. I believe that most people will rise to the challenge if they feel empowered to do so.  

Q. What are you doing when you are not at work?

A. My main hobbies are reading for pleasure and writing, and I hope to publish a book about my experiences in America in the near future. I used to be an avid hiker, but while I still enjoy it, I do not get out there as much as I used to. I also enjoy spending time with my family, my wife Marianne, my five children and 11 grandchildren.


CHI-Advancing California biomedical research and innovation

CODAN USA headquarters in Santa Ana, Calif.

Community Spotlight: CHI Supports Volunteer Reading Program



CHI Communications Specialist Heather Chambers with OB Elementary students.

























While the end of the school year is typically a time for celebration, for me, it was bittersweet to say goodbye to a group of students in Ocean Beach Elementary School’s classroom 7.

For the second year in a row, I had the opportunity to share my love of reading with a classroom full of first and second graders. I am grateful that CHI values its employees and their personal pursuits and has allowed me the time to read to the children each Wednesday morning.

The Rolling Readers program is designed to reach underprivileged children and encourage lifelong learning that starts with reading at the grade-school level. It pairs adult volunteers with schools that offer the free lunch program.

Research shows that children who aren't reading proficiently by the end of third grade are four times less likely to finish high school on time. If they are poor and not reading proficiently, they are 13 times less likely to finish high school. And for children who live in areas of concentrated poverty, the prospects are even grimmer.

In order to encourage leaning in this fun, “free time,” we explored the world of fantasy, adventure, mystery and even foreign language.

The highlight of the Rolling Readers program is its annual book drive. This year, each student took home a copy of “City Dog, Country Frog” by author Mo Willems. It is satisfying to know that they will have a book to call their own, to share with friends and encourage reading among family members.

As these students move forward with their education, hopefully they will find comfort and joy in reading. I certainly found joy in reading to them.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Ralf Otto, Ph.D., VP Operations and Fremont Site Head, Boehringer Ingelheim

Ralf Otto

Otto joined Boehringer Ingelheim (BI) in 2001. At BI, he has served as executive director of a large-scale cell culture manufacturing facility and also played a key role in a technology and capacity upgrade project of the facility. Beginning in 2010, Otto worked in BI’s Pharma production network as head of network competitiveness, holding global responsibility for improving sites’ competitiveness with a focus on Brazil and China. In 2011, he joined BI Fremont as site head.

Otto previously worked for Henkel in Duesseldorf, Germany, where he spent three years in various biotechnology-related positions. Since 2002, he has routinely taught commercial biotechnology courses at the University of St. Gallen (Switzerland), University of Stuttgart, and the University of Karlsruhe (both in Germany). He graduated with a degree in biotechnology and carried out his doctorate at the German Cancer Research Center/University of Stuttgart. His scientific writing has appeared in numerous international journals and books, and he holds more than 20 patents.

Q: Share a brief history of BI and its Fremont facility, acquired from Amgen.

A: Boehringer Ingelheim (BI) was founded in 1885 by Albert Boehringer in Ingelheim am Rhein, Germany. More than 125 years later, the company is still family-owned and is one of the world’s 20 leading pharmaceutical companies. The Boehringer Ingelheim Corporation currently has more than 42,000 employees and 145 affiliated companies spread across the globe. BI has six facilities in the United States: our U.S. headquarters and R&D including Biologics Skill Center in Ridgefield, Conn.; our generic company, Roxane, in Columbus, Ohio; a manufacturing facility in Bedford, Ohio; our animal medical facilities in St. Joseph, Mo. and Fort Dodge, Iowa; our chemical facility in Petersburg, Va.; and lastly, our biotechnology facility in Fremont, Calif.

BI acquired the Fremont facility in March 2011 from Amgen, making it BI’s first biotechnology facility in the U.S. This facility encompasses more than 300,000 square feet of state-of-the-art development and manufacturing space. BI’s vision was to transform the site into a development, clinical manufacturing, and innovation site that can develop and produce multiple drugs for different contract manufacturing clients and our own biologics.

Q: How many employees does the site have today? What kind of manufacturing capabilities?

A: BI Fremont has approximately 300 employees. The site is capable of developing and manufacturing non-clinical, clinical, and commercial biopharmaceuticals and includes a non-GMP pilot plant and process development labs. The facility boasts two trains that enable us to manufacture multiple products simultaneously, up to the 12,000-liter level from cell harvesting, cultivation, and purification, ending with fill and finish. Additionally, we are currently extending our services into disposable technology that would complement the usual bioreactors.

Q: Tell me some of your biggest goals for the year.

A: I want us to make BI Fremont the first choice for clients on the West Coast, especially in the Bay Area. While Fremont is BI's first biotech facility in the U.S., BI has been a successful contract manufacturer in other countries for decades. We are excited to expand our innovation to California. I want us to continue building a high-performing team in Fremont, ensuring that every employee can deliver his or her best. As for productivity, we have set the goal to beat our benchmark and to cultivate a culture of innovation that drives superior solutions for BI patients and clients. Our culture is based on values that make this a great place to work, and together we are focused on ways to build a sustainable future for the site.

Q: What are the biggest challenges to achieving these goals?

A: With the acquisition we inherited systems that need to be significantly transformed to support our multi-faceted business direction. We also need to ensure that employees have the right skills through training and by hiring new talent so that we can fulfill our objectives. Change is always a challenge, but we already have accomplished great achievements at high speed—for example, a four-fold increase in productivity, multi-product operations, and new product introduction—so we are sure that we can deliver even more on our ambitious targets.

Q: If you could change a public policy at the state or federal level, what would it be?

A: California is one of only three states where manufacturers are required to pay both a sales and property tax for equipment. We support current state legislation that would credit manufacturers for sales tax purchases.

Q: Some may be surprised to hear that BI is a family-owned company. Describe your corporate culture and how it may differ from others.

A: We provide value to the customer by being innovative and entrepreneurial in everything we do. We call this approach “value through innovation.” Value through innovation means coming up with new and better ways of improving health, which reflect and anticipate the needs of our customers and patients. We also emphasize both personal and professional development and encourage employees to never stop learning. Based on our values, we can guarantee sustainability as BI’s 125 years record shows.

Q: What is the most rewarding part of your job?

A: In a short period of time, I have seen great results from our staff in Fremont. Fremont has a diverse workforce that is extremely flexible, responsive, and experienced, which has achieved great success in just our first year. I am excited about our prospects for this year and beyond.

Q: What are you doing when you are not at work?

A: I spend time over the weekends traveling around California with my family. I am very much impressed by the country, people, and culture.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Dr. Alain Baron, President and CEO, Elcelyx Therapeutics

Dr. Alain Baron

Elcelyx Therapeutics is a privately held biotechnology company focused on creating a wide range of products based on the science of nutrient sensing. The company’s novel product candidates are directed at the prevention and treatment of obesity and diabetes. Since joining Elcelyx Therapeutics in 2010, Baron has been focused on advancing the company’s new products to treat metabolic diseases based on a completely novel platform, called “gut sensory modulation.” He brought his expertise as an entrepreneur-in-residence and a member of the life science team at Morgenthaler Ventures, a position he has held since 2008, along with prior experience as senior vice president of research at Amylin Pharmaceuticals. He has held faculty positions at Indiana University School of Medicine in Indianapolis and UCSD, along with the Veterans Administration Medical Center in San Diego. Baron graduated from McGill University in Canada with a bachelor’s degree in biology and a medical degree from the Medical College of Georgia Augusta. He completed postdoctoral studies at UCSD.



Q: Share with us a brief history of the company. How did your leaders come together? How was the company originally funded?


A: Elcelyx was founded by two former Amylin Pharmaceuticals employees, myself and Martin Brown. Elcelyx was first funded by Morgenthaler Ventures in a Series A round in March of 2010. Currently, the company is funded by Morgenthaler Ventures, Kleiner Perkins Caufield & Byers, and Technology Partners.


Q: Tell me some of your biggest goals for the year.


A: Elcelyx is developing new products to treat metabolic diseases based on a completely novel platform, named “gut sensory modulation,” that shows promise to produce very safe and effective oral therapies.


Q: What are the biggest challenges to achieving these goals?


A: Recognizing the importance of safety of therapeutics for metabolic diseases in the current regulatory environment, Elcelyx has focused its efforts at maximizing safety in its development of novel therapeutics.


Q: If you could change a public policy at the state or federal level, what would it be?


A: Given the time and risks involved with drug development, there is a great need to improve dialogue with the U.S. Food and Drug Administration to make their decisions more predictable.


Q: What is the most rewarding part of your job?


A: The collaborative environment created and nurtured at Elcelyx makes for a very productive and enjoyable workplace. Knowing we are working to address large, unmet medical needs is all the more rewarding.


Q: What are you doing when you’re not at work?


A: I love to spend time with my family and friends, read, play tennis and travel.

CHI-Advancing California biomedical research and innovation

Event Spotlight: CHI Hosts Annual Reception and Hill Meetings in Washington, D.C.



House Energy & Commerce Committee Chairman Fred Upton accepts CHI Chairman's Award from CHI President and CEO David Gollaher, Ph.D., and CHI Chairman and Gen-Probe CEO Carl Hull.

CHI honored House Energy and Commerce Committee Chairman Fred Upton (R-MI) with the CHI Chairman’s Award during a reception May 9 in Washington, D.C., at the Folger Shakespeare Library. Guests at the reception and private dinner enjoyed an evening with the California delegation and leaders driving biomedical innovation at the event, called “Invest. Innovate. Compete.” During the dinner, Rep. Brian Bilbray (R-San Diego) expressed his support for the biomedical community and stressed the importance of bringing life-saving cures to patients in need.

Prior to the event, CHI board members participated in meetings on Capitol Hill to discuss the impact of the U.S. Food and Drug Administration and user fee reauthorizations, the importance of National Institutes of Health funding, and the significance of the California life sciences community to continued innovation and job creation. Meetings were held with Energy and Commerce Committee members including: Rep. Brian Bilbray (R-Carlsbad), Rep. Lois Capps (D-Santa Barbara), Rep. Anna Eshoo (D-Atherton), Rep. Phil Gingrey (R-GA), and Rep. Mike Rogers (R-MI). Other member meetings included Sen. Barbara Boxer (D-CA), Rep. David Dreier (R-San Dimas), Rep. Jackie Speier (D-Hillsborough), House Majority Whip Kevin McCarthy (R-Bakersfield) and key legislative staff at the Senate Health, Labor, Education and Pensions (HELP) Committee. Over the course of the day, one message was repeatedly conveyed: the important and successful role of CHI in communicating and contextualizing the importance of consistent, predictable and efficient FDA regulatory processes to continued biomedical research, investment and innovation in our state.

Also that day, CHI released a report on FDA with respect to the agency’s approval of drugs and biologics, building on our earlier study, Competitiveness and Regulation: The FDA and the Future of America’s Biomedical Industry (2011). The report, Managing Priorities: Therapeutic Area Variation in FDA Drug Regulation, presents new data that illustrate important differences in FDA performance from one therapeutic area to another and examines misalignment between regulation and public health needs. It comes as Congress considers critical legislation that would renew and expand fees paid by drug and medical-device companies to help fund the FDA. Current legislation allowing the FDA to collect fees from drug and medical-device companies expires Sept. 30.

Genetic Engineering and Biotechnology News highlighted the report’s findings in a recent article and The Burrill Report featured a one-on-one interview between CHI CEO David L. Gollaher, Ph.D., and Burrill’s Daniel Levine.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Kleanthis Xanthopoulos, Ph.D., CEO of Regulus Therapeutics Inc.



Kleanthis Xanthopoulos

Xanthopoulos understands the biotech business from the inside out. Xanthopoulos is president and CEO of Regulus Therapeutics Inc., which was formed in late 2007 by Alnylam Pharmaceuticals of Cambridge, Mass. and Carlsbad, Calif.-based Isis Pharmaceuticals. It is testing drugs that use microRNA technology, a gene “silencing” technique used for treating various diseases. Regulus has created leading strategic alliances with GlaxoSmithKline and Sanofi-Aventis to develop microRNA therapeutics. Regulus moved to La Jolla in mid-2010 in the proximity of leading academic institutions including The Scripps Research Institute, Salk Institute, Sanford-Burnham Medical Research Institute and UC San Diego.

Before joining Regulus, Xanthopoulos spent time as managing director of Enterprise Partners Venture Capital, where he analyzed the best deals in the biomedical space. Prior to that, he co-founded and served as president and CEO of Anadys Pharmaceuticals, Inc., and served as a member of the board until its acquisition by Roche in late 2011. And before Aurora Biosciences became Vertex Pharmaceuticals, he was vice president from 1997 to 2000. Xanthopoulos participated in The Human Genome Project as a section head of the National Human Genome Research Institute from 1995 to 1997. Previously, he was an associate professor at the Karolinska Institute in Stockholm, Sweden after completing a postdoctoral research fellowship at The Rockefeller University, New York.

An Onassis Foundation scholar, Xanthopoulos received his bachelor’s degree in biology with honors from Aristotle University of Thessaloniki, Greece, and received both his master’s and doctorate degrees in microbiology from the University of Stockholm, Sweden.


Q: You went from being head of a biotech to the investor world, and, now, as head of this company spun out of Alnylam and Isis. What has the transition back been like?


A: Entering biotech life again was a perfectly natural transition, like coming home. I fell in love with the groundbreaking biology and was enamored with the opportunity to translate this big idea into innovative medicines. I like to say that my time spent at the VC firm was as a biotech sabbatical — taking time off from my true passion of building companies.


Q: What led Regulus to locate on the Torrey Pines Mesa as opposed to, say, Carlsbad where Isis is located?


A: It made terrific sense to be incubated within Isis. There is a lot of educational knowledge that we extracted from Isis, and the best way to do this is by osmosis. So, being there was very important for the first years of Regulus’ life. But, ultimately, we wanted our own culture and identity, and we felt that, based on some local collaborations and activities that we have, it would be much better served if we were in the Torrey Mesa as opposed to North County.


Q: Regulus has made some major industry collaborations, big names such as GlaxoSmithKline. What can we expect from the Regulus partnerships in the years to come?


A: Regulus has a dominant locked-in position in the world of microRNAs, which are remarkable and elaborate. They manage control of transcription and post-transcription mechanisms by dictating the half-life of messenger RNA. More than 700 microRNAs have so far been identified in humans, so that means 700 targets. And that, for a small company like us, means collaboration. We have 40 active academic institutions that work with Regulus and many of them are in very close proximity, including one with UCSD. It is part of the strategy and one that we pursued and have benefitted from exceptionally well.


Within six months of formally announcing Regulus, we had the partnership with Glaxo. It is encompassed around four microRNAs. Only one had been identified before the collaboration. So what you should expect is steady stream of news over the period of the collaboration.


Q: Talk about the environment for startups in California. What would be your advice to start-ups looking to grow within the state, such as Regulus has?


A: The good news is that any good, solid idea that is addressing significant needs in the pharmaceutical drug development stage is going to get funded. The bad news is the other ideas, or maybe product ideas alone, have a much more difficult time. Re-licensed or repurposed drug stories, I think, are going to have a harder time moving forward. In the early part of this year, we have seen a resurgence of platform technology stories emerge successful on the biotech scene. These platform stories are getting attention from not only VC firms, but institutional investors looking to take additional risks in their portfolio. This is a departure from the past several years in which companies with later-stage assets were the only investment opportunities for institutional investors. The tide seems to have turned and the early platform companies are now vying for the same Wall Street monies as the later-stage companies. A truly innovative breakthrough idea, such as the one Regulus is pursuing, will get funded.

Q: When Regulus first began, the CEO of Alnylam told investors that he expected Regulus to "dominate the microRNA space.” How do you think Regulus has stood up to that test?


A: Unfortunately, we did better than expected. And by unfortunately, I mean, we have scared away a lot of competition. Many people think that is good. I, personally, think that a little bit of competition is very healthy. So, unfortunately, we don’t have that, so the case of microRNA almost exclusively relies on how good Regulus is going to do.


Q: That has also brought you some healthy collaborations and financial backing. Talk about your growth as a company and where you see yourselves going in the next five to 10 years.


A: The biggest risk for Regulus five years ago was, “Can you target microRNAs and have a sort of peak effect without having a lot of side effects?” That was the big biological risk. Today, we have been able to show in 25 different models of human studies and animal studies that you can do that with a good therapeutic model. So, now you can say that the proof of concept has been established.


So, how do we look five years from now? We are very likely going to be a public company. We are still going to be less than 100 people. We are religiously defending our turf when we can, maintaining that the best brain trust one can possibly have in-house, but also utilizing a lot of extra hands outside. We going to have at least three programs in the clinic and maintain four to six preclinical programs at any given time.

Q: What are some of your proudest accomplishments since Regulus was formed in September 2007?


A: For me, it is all about people, strategy and finance. I think we have been, so far, good on all three fronts, and that’s something I am proud about. We have brilliant people, who are all tremendously seasoned drug hunters or hugely experienced people in finance and operations. We are now at about 55 employees. We are one of the few companies that has been growing at significant rates over the past four years. We have raised over $110 million. The vast majority of this is from the alliances, so it is non-dilutive financing. And I think we have a very good strategy of how to methodically move the pipeline from a big idea to a meaningful set of preclinical and clinical progress.




CHI-Advancing California biomedical research and innovation

Executive Profile: Brian O'Callaghan, President and CEO, Sangart



Brian O'Callaghan

O'Callaghan joined Sangart as president and CEO in June 2008. He brings a breadth of experience to Sangart, having held senior positions with a number of pharmaceutical and biotechnology companies in both Europe and the U.S. These include senior positions with Pfizer in the U.K. and Merck Serono in Germany, before becoming president and CEO of BioPartners, a Swiss-based biotechnology company. Since relocating to the U.S., O'Callaghan has held senior management positions at Novartis, Covance, and, most recently, NPS Pharmaceuticals, where he served as chief commercial officer. O'Callaghan brings his extensive experience to lead Sangart through regulatory submission and commercial launch of the MP4 platform.

Q: In looking at the history of Sangart in San Diego, it was interesting to me that you have both these civilian and military applications. Tell me more about Sangart's connection with the Letterman Army Institute of Research.

A: The company was founded by a guy named Dr. Bob Winslow. Bob started this journey when he was with the Letterman Army Institute of Research about 20-30 years ago. His experience there culminated in him founding Sangart about 14 years ago.

What is great is that some of the people still with Sangart today were with Bob at the Letterman Army Institute of Research. Literally decades of research has gone into developing what became MP4, which is Sangart’s platform product.


What Bob discovered was that you could replicate a low-volume setting for some of the properties of blood. As a result, Bob developed the MP4 molecule as an agent that could very effectively transmit gases into the body.


There are the two gases that Sangart is currently focusing on with MP4: oxygen and carbon monoxide.


The oxygenated compound (MP4OX) is designed to deliver oxygen in the capillaries, where red blood cells may not naturally reach, when the body is suffering from hemorrhagic shock. The carbon monoxide formulation (MP4CO) is designed to deliver therapeutic levels of carbon monoxide to patients with sickle cell anemia.


Q: How does this compare to standard care available today?


A: There is no real treatment right now for trauma-related ischemia. When patients suffer blood loss due to a traumatic event, they eventually get to a critical care setting. They get all sorts of fluids, including blood, as well as surgery, of course. If the lactic acid levels, which are routinely measured, show lactic acidosis, an indicator of a lack of oxygen, then they receive MP4OX, in a blind setting, as an addition to standard of care. We measure the resolution of lactic acidosis, which indicates the delivery of oxygen to the ischemic tissue. We also look at other endpoints, such as the ICU setting to see if they are spending less time in the hospital on ventilators, etc.


Our Phase IIb study is evaluating MP4OX plus standard of care in trauma patients, with lactic acidosis due to hemorrhagic shock. It’s running in about 50 sites in about 15 countries throughout the world.


Q: How do you balance the need to raise capital with everyday activities?


A: We have been insulated more than most from all that is going on in the last two to three years because our investors are very patient.


We are funded through to our next major milestone events. Those being the generation of the next rounds of clinical data from the PhIIb study for MP4OX and the PhIb study for MP4CO.


With positive data from our two clinical trials, we are confident of raising additional funding from existing or alternative investors.


Q: Where do the biggest opportunities lie?

A: The biggest opportunity, by far, is in trauma. You can take oncology and cardiology and the other major killers, and you can combine all the deaths that come from those and they don't compare to trauma. Trauma is, by far, the biggest killer in our society.

The good news is that if you do get into a critical care setting, your chances of survival are actually very high. This product will be serving a critical unmet medical need where there are not any products like it out there. The market is estimated to be about $6 billion in the developed worlds at least.

When you look at the pharmacoeconomic aspects of keeping people out of ICU and off ventilators, while also getting them out of the hospital faster, you are potentially saving thousands of dollars per day by using MP4OX. We are estimating savings of anywhere between $5,000 to $10,000 a day, while also contributing to patients being discharged, on average, a week earlier.

Q: What does the future of traumatic brain injury and other types of severe blood loss treatment look like 20 years from now?

A: Traumatic brain injury is one of the things the Army wants us to look at. Traumatic brain injury might have more of a relevance to the military in the battlefield than the civilian setting. What they are looking for are newer formulations that are more robust to be used in the battlefields. So they are looking for formulations that can basically be used in the soldiers' backpacks or in forward positioned surgical units.

Q: What differentiates Sangart from others in the field?

A: Studies show that our MP4 products can potentially be safer and more effective as therapies for trauma and sickle cell disease. We have investors who are very patient and willing to continue investing in us given our strong clinical data. Finally, and very importantly, we are willing to do our development overseas rather than the U.S. should it serve MP4’s needs.



CHI-Advancing California biomedical research and innovation

Executive Profile: Rob Perez, Executive Vice President and Chief Operating Officer, Cubist Pharmaceuticals

Rob Perez

Perez has served as executive vice president and chief operating officer of Cubist since August 2007.

He grew up in the Los Angeles area and received his undergraduate degree from California State University at Los Angeles and his MBA from the Anderson School at UCLA. He began his career as a sales representative in South Central Los Angeles working for Zeneca Pharmaceuticals. Ultimately, he became a regional business director, responsible for sales, marketing and national accounts for the western regional business unit.


After Zeneca, he joined Biogen, in 1995, where he worked with the team that developed a commercial model for Biogen’s first product, Avonex for multiple sclerosis. He eventually led the U.S. neurology business before leaving to join Cubist. At Cubist, he says, they saw a potential in Cubicin that no one else saw.


“I have always worked at companies that have had to compete against larger firms with more resources, so the hallmark of all the businesses that we’ve built has been our ability to learn quickly,” Perez said. “I try to find people and develop processes that allow our team to gain a competitive advantage by listening to those closest to the customer, and by then making incremental changes to the business based on this feedback.”


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


A: The company was founded in 1992, and, in the fall of 1996, Cubist completed its initial public offering. To date, Cubicin (daptomycin for injection) has been used to treat more than a million patients and Cubist is well on its way to surpassing $1 billion in U.S. annual net revenues.


The history of Cubist is a great story that is really about the people who have collectively advanced the science and the business to where we are today, a successful pharmaceutical company. At Cubist we don’t fear failure and that is a large part of what has made us successful. Our culture is one that rewards success, but, at the same time, we view failure as an opportunity to learn. The best example of this at Cubist was the “rescuing” of daptomycin after the Phase 3 results from community-acquired pneumonia were known. Daptomycin failed to meet statistical noninferiority criteria in a clinical trial for severe community-acquired pneumonia. We needed to understand why it did not work in the lungs and we launched an effort to isolate the effect. We discovered the reason and published the results. We ended up with a drug approved in the U.S. for complicated skin and skin structure infections in 2003, and, three years later, received an expanded U.S. label for Cubicin for the treatment of S. aureus bloodstream infections (bacteremia), including right-sided infective endocarditis caused by MRSA and MSSA.


More recently, 2011 was a transformational year for Cubist with positive Phase 2 results for two of our antibiotic candidates, propelling them into Phase 3 — one program already enrolling patients and the other expected to start this year. Also in 2011, we partnered with Optimer Pharmaceuticals to co-promote their therapy, Dificid, for the treatment of clostridium-dificile associated diarrhea (CDAD). We capped off the year with a business development deal to acquire Adolor Corporation, adding a new U.S. commercial product in Entereg and a promising pipeline candidate in CB-5945, also on a path to begin Phase 3 trials in 2012.


Q: What are some of your biggest goals for the upcoming year?


A: The transformational year I just mentioned has put a lot on our plate for 2012. We are going to be focused on executing our business plan aimed at continued momentum in Cubicin sales and putting more marketing muscle behind our newly acquired drug Entereg toward our goal of achieving peak annual sales of $100 million. Also, our sales force will continue to co-promote Dificid as part of our strategic partnership with Optimer.


In the clinic, we expect to have three Phase 3 programs up and running this year. There is a great and growing unmet medical need for antibiotics to treat serious infections, and two of the three programs are focused on antibiotics. The third program we acquired through the Adolor acquisition focuses on a therapy to treat a gastrointestinal side effect of opioid pain management.


Q: What are the biggest challenges facing developers of novel antibiotics today?


A: The challenges we face today started appearing on the horizon several years ago. Despite the remarkable public health success of anti-infective agents, there is a trend toward increasing numbers of bacterial infections that exhibit resistance to new and standard antimicrobials, with 70 percent of hospital-acquired infections caused by bacteria that are resistant to at least one antimicrobial. At the same time, many pharmaceutical companies have backed away from research and development of new antimicrobials because of increased incentives to develop drugs in other therapeutic areas and because of contradicting market forces within the antimicrobial marketplace. As a consequence, anti-infective research has virtually dried up, leaving very few new anti-infective agents in the near term. Cubist is one of the few companies with a promising pipeline in this area. Some public health leaders have identified the challenge and are prompting government action. Now is the time to identify and implement appropriate incentives that will drive antimicrobial research and development.


Recommended incentives have recently been manifested in the Generating Antibiotic Incentives Now (GAIN) Act, which has now been introduced in both the Senate and the House. Dr. Barry Eisenstein, senior vice president of scientific affairs for Cubist, was invited to testify at a congressional subcommittee hearing on the topic. The bi-partisan legislation calls for the enactment of specific economic and market incentives to entice pharmaceutical companies to develop new antibiotics to treat the growing number of serious infections caused by multi drug-resistant (MDR) bacteria. For example, the legislation’s provision for extending the exclusivity granted to some badly needed antibiotics is designed to have the same impact on antibiotic R&D as the Orphan Drug Act had on drugs to treat rare diseases, which now enjoy a healthy pipeline. CHI recently released a report on the subject, available here.


Another challenge that we are working through with others in industry is clear guidance from FDA in defining new regulatory approaches to facilitate antimicrobial development and approval. Recent meetings on this front have been promising, and we look forward to working closely with FDA to ensure more therapies are approved for patients battling sometimes life-threatening infectious diseases. More on this topic can be found at a website sponsored by Cubist: http://www.battlingsuperbugs.com/


Q: What is the most rewarding part of developing products that address unmet medical needs in the acute care environment?


A: We know that Cubicin has been used to treat more than a million patients and we know that if our pipeline of much needed therapies is someday approved, millions more could benefit from much needed therapies. We use the term patients, but these are people who are moms and dads, children, friends, colleagues, and neighbors. Everyone at Cubist comes to work each and every day with the goal of contributing to the advancement of therapies and leaves at the end of the day knowing that somewhere that day Cubist made a difference in someone’s life.


Q: When you’re not working, where do you like to spend your time?


A: I have three great passions outside of work. First and foremost is my family. My wife and I take such great pleasure in watching our kids’ many interests/activities. Second, I enjoy spending time trying to make a difference in the lives of kids who don’t have the educational opportunities that my kids have been blessed with. Finally, my one vice is basketball. I am admittedly a “hoop junkie.” I play with friends whenever I can (although at my age it is not really basketball, but something closely resembling the sport that I love) and also avidly follow the Lakers, and UCLA basketball. There are a lot of demands on my time, but setting priorities helps me maintain a healthy balance in life. For our corporate intranet I publish a blog sharing musings about the business/industry and observations from a personal perspective — I repurpose part of my blog posts on my twitter account @robperez32.




CHI-Advancing California biomedical research and innovation

Executive Spotlight: Jeff Dunn, President and CEO, SI-Bone

Jeff Dunn

SI-BONE Inc. of San Jose, Calif. is the leading sacroiliac (SI) joint medical device company dedicated to the development of tools and products for diagnosing and treating patients with low back issues related to SI joint pathology. The company has developed, and is manufacturing and marketing, less invasive approaches using implants for the treatment of SI joint disorders.

Dunn has more than 30 years of executive experience, including serving as the CEO of six other companies. He has taken one company public, sold all the others to larger public companies, served on numerous boards of directors and is an advisor to other CEOs. Prior to SI-BONE Inc., Dunn was CEO of INBONE Technologies, which he built into a leading ankle replacement and small bone fusion medical device company. Dunn led the sale of INBONE to Wright Medical in April 2008. He holds a bachelor’s degree from Colgate University and a master’s of business administration from Babson College.

Q: How did SI-BONE form?

A: It was Dr. Mark Reiley, who founded Kyphon and invented kyphoplasty (a procedure that attempts to stop the pain caused by spinal bone fractures) with whom I got together about six years ago when I became the CEO of two of Mark’s companies. One was an ankle replacement company and one was a small bone fusion company. One company was in Colorado; one was in Berkeley, Calif., and I was the CEO of both of those at the same time. We merged those companies into a company called INBONE Technologies, which was acquired by Wright Medical Group in April 2008. Wright Medical is not in the spine business, so, as part of the deal, we took all the assets related to SI-BONE — the patents, the 510(k)s, etc. — and we spun that company out into an independent company.

For the next seven or eight months, we had no employees; it was just Mark and I. On Nov. 26, 2008, we received 510(k) clearance from the FDA. We then raised private money, about $4.5 million from friends and family. We hired our first employee in April 2009 and basically spent that year setting up manufacturing, quality systems, setting up the training programs for the surgeons, doing work around the diagnosis and finding the diagnostic algorithms to teach surgeons how to diagnose SI joint pain.

We have trained more than 700 surgeons to date. We did our very first surgery in June of 2009. We have grown as a company approximately 50 percent every single quarter, quarter over quarter, since June 2009.

Q: I also saw that you recently raised some venture funding.

A: We raised $16 million from Montreux Equity Partners and also Skyline Ventures. Our very first venture money came in last summer from Skyline, and then we had a bunch of private investors also participate who were friends of Mark’s and mine that have made money with us before.

Q: Talk a little bit about this type of surgery and the market SI-Bone is addressing.

A: There is $50 billion a year spent on lower back pain in the United States. Clinical studies show that 22 percent of that is related to the SI joint. The only surgery to treat the SI joint over the last number of decades is an open surgery where the patient endures a 12-inch incision. It is very bad for the patient, obviously. Ours is a minimally invasive surgery. It takes about an hour. So, we are treating something that almost has never been treated, but accounts for 22 percent of all lower back pain. As you probably know, the No. 1 reason that people visit their physician is for the flu. The second most important reason is back pain. So, it is a huge problem, and we are addressing an underserved, unmet need that afflicts millions of patients.


Q: How does the company stand out in the competitive landscape for minimally invasive surgery and surgery like yours?

A: Well, in the United States, there is only one very small company and one medium sized private company which have introduced products to compete with us. Let’s just say we have 95-plus percent market share. In Europe, there is one other competitor. We believe in our product. It is significantly better, easier to use for surgeons, better for the patient.
The key, though, is to educate the surgeons on how to diagnose this thing. We believe that everything is about patient selection because many people with lower back pain have an under-diagnoses. We spend a tremendous amount of money on educating surgeons, physician assistants, physical therapists, etc. about how to effectively diagnose these patients.


Q: Are you seeing patients that have gone through fusions or other surgeries come to you afterward? Are these new patients?

A: We have plenty of patients that are both de novo, who have been
diagnosed with sacroiliac joint issues or dysfunction, and previous spine fusion patients.

In the United States, there are hundreds of thousands of lumbar fusions each year. Literature shows that about 75 percent of all patients that have lumbar fusions develop adjacent segment disorder within five years. So, if you fuse a part of the lumbar spine, typically it affects other pieces of the orthopedic anatomy. The majority of people who have those kinds of surgeries within five years will have some kind of SI joint issues.

Q: You touched on this a little bit, but what does the reimbursement landscape look like for your device?

A: Our reimbursement rates are typically running about 94 percent. So, the reimbursement is actually quite reasonable. But, of course, as we get bigger as a company, the payers, i.e. the insurance companies and Medicare and the key medical societies including NASS, AAOS, CNS, AANS, ISAAS, ISIS and the AMA, are going to want to see efficacy, economic justification and safety data, so that is why we are investing so much in clinical studies. We are launching multiple studies.

Q: What else is good to know about SI-BONE?

A: What I am most proud of is we have hired 100 people in the last two years and we just have a tremendously talented group of people that are working together to help these patients. They are thriving as I am on the patients. What’s more fun than helping patients? Additionally, we have an advisory board made up of brilliant medical professionals like Dr. Steve Garfin, Dr. Frank Phillips, Dr. Paul Anderson and others. I would go out on a limb and say that our advisory board is among the most talented and respected spine advisory board in the United States.

So, we have a very good group of people that are trying to solve this problem and help educate people here and in Europe about how to help these patients.

CHI-Advancing California biomedical research and innovation

CHI Spotlight: Opening a New Door for Unfunded Research Projects

Marc Boutin

The National Health Council (NHC) is the only organization of its kind that brings together all segments of the health community to provide a united voice for the more than 133 million people with chronic diseases and disabilities and their family caregivers. Made up of more than 100 national health-related organizations and businesses, its core membership includes approximately 50 of the nation’s leading patient advocacy groups, which control its governance.


Marc Boutin serves as the organization’s executive vice president and chief operating officer. Throughout his career, he has been actively involved in health advocacy, policy, and both federal and state legislation. 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.

Q: I understand the National Health Council has started a new program that connects promising biomedical research with funding sources. Talk a little about the impetus for your newest endeavor, HealthResearchFunding.org?

A: HealthResearchFunding.Org was conceived several years ago by a group of chief medical officers and research directors from NHC member patient advocacy organizations. They recognized the need to make the best use of the significant expertise and funds spent on the rigorous scoring of research proposals. With the help of the National Institutes of Health (NIH), the NHC set out to develop an innovative solution to make the most out of the nation's investment in biomedical research.

Q: How might someone who is interested participate?

A: Researchers whose proposals have been peer reviewed, deemed meritorious, but unfunded by either the NIH or by an NHC member organization, are invited to register with HealthResearchFunding.org. Once registered, researchers can upload requests for proposals and search for funding sources. There is no charge for use of the site by investigators or their institutions.

By using this online resource, potential research supporters can avoid duplication of effort and more efficiently identify and evaluate peer-reviewed research proposals. On the other hand, researchers gain another opportunity to showcase their worthy proposals and possibly find a funding source.

The database will be expanded later this year to include NHC business and industry members and funding sources outside NHC membership.

Q: Who may search the database?

A: Registered investigators have the ability to search for funding sources through the database, but cannot view or search for proposals from other researchers. Funding sources are able to search for research proposals, individual researchers and information from other funding organizations.

Q: How does HealthResearchFunding.org help researchers?

A: HealthResearchFunding.org helps researchers obtain a broad audience of potential funding sources from the nonprofit sector for their proposed research.

By offering investigators the opportunity to promote their peer-reviewed research proposals through the database, we hope to increase the likelihood of potential funding for worthy health research. By utilizing the database, researchers gain an organized and unvarying environment in which to exhibit their proposals alongside their peers. As the system grows, their respective research institutions could spend less time, effort, and resources looking for financial support and more time conducting research to aid in the development of new treatments for patients.

Q: How does this database help funding sources?

A: HealthResearchFunding.org helps participating funding organizations avoid duplication of effort as they seek to fund research by making use of the significant public investment of intellectual capital, time, and funds in the NIH and NHC member peer review process. When on the database, funding organizations are able to upload requests for proposals and have the ability to post ideas for collaboration with other funding sources.

Q: What is the ultimate goal for this program?

Our goal is to help researchers and funding sources in their development of new treatments and cures for people with chronic conditions.

To do so, we hope to expedite the process by which participating non-government funding organizations underwrite biomedical research by linking them with researchers whose proposals to NIH and NHC member organizations have been deemed meritorious and worthwhile.

To learn more, visit http://www.healthresearchfunding.org/ or write to healthresearchfunding@nhcouncil.org.


CHI-Advancing California biomedical research and innovation

Speaker Spotlight: Judith Kjelstrom, Ph.D., Director of the UC Davis Biotechnology Program

Judith Kjelstrom

Judith Kjelstrom, Ph.D., also known as Dr. Judy, has a rich background in health science and biotechnology education and training, clinical laboratory science, microbiology and immunology and program administration. She directs the UC Davis biotechnology program and the advanced degree program for corporate employees. Besides co-directing the UC Davis-Howard Hughes Medical Institute's Integrating Medicine into Basic Science graduate training program, she is also a lecturer in the departments of microbiology and molecular and cellular biology at UC Davis. Kjelstrom received her bachelor's degree in biology from Sacramento State University and her doctorate in microbiology from UC Davis.

If you have not already secured your ticket to hear Dr. Judy speak next week, contact CHI!

Q: As the director for UC Davis’ Biotechnology Program, how would you describe the environment for biotech education?

A: The environment at UC Davis continues to be strong in all aspects of biotechnology education from undergraduate to doctorate level. UC Davis continued to increase its prominence in research, especially in the sciences. The campus received more than $684 million in research funds in the last year. While federal research dollars fell slightly as stimulus funds tapered off, awards from non-governmental organizations quadrupled and state research funding grew by half.

Due to the breath of expertise and the collaborative nature of the various schools and colleges, we are able to offer research opportunities in biomedical application, agricultural and environmental biotech as well as the newer industrial areas such as biofuels. Enrollments in biotech-related programs continue to grow. For example, the Designated Emphasis in Biotechnology (DEB) graduate program, which is housed within the biotechnology program, has grown from 20 students in 2000 to 220 Ph.D. students in 2012. Having fellowship support for graduate training is critical for recruiting the most talented students to campus.

We recently received another five-year renewal for the National Institutes of Health training grant in biomolecular technology, in which the DEB is the formal graduate education program. Other related training grants, in which I am involved, are the National Science Foundation-funded Integrative Graduate Education and Research Traineeship (IGERT) program and the UC Davis-Howard Hughes Medical Institute Integrating Medicine into Basic Science training program, which provide additional scholarship support for DEB students. Our industry partners provide additional training through paid internships and co-ops, which are required for all DEB graduate students.

Q: What do you see as some of the biggest challenges, from an educational standpoint, going forward?

A: In a recent statement by Chancellor Linda Katehi, reduced funding from the state of California has challenged all of the UC campuses. Increased dependence on student tuition and private funding to meet our education and research missions puts in question the very nature of the university and its relationship with the state and public. In the 1960s, the California Master Plan led to the creation of what is recognized widely as the best public research university in the world. But, in the past 20 years, the commitment to the master plan has eroded and continues to do so. The state's contribution to students on the UC campuses has been reduced from $17,000 per student in 1990-1991 to less than $7,000 per student in 2010-2011. In the past four years, UC Davis alone has lost 40 percent of its state budget, while tuition has increased by 84 percent. The future competitiveness of both California and America depend on a healthy and widely accessible UC system.

Moreover, in today's academic environment, research is a vital part of this learning. For a nation that wants to be a global leader through innovation, this must be a skill accessible to all citizens. The ability to think creatively, act entrepreneurially and drive change when needed must be part of our public identity.

The biotechnology program has been hit hard by budget reductions and is unable to expand our staff and operational budgets to meet the growing demand for our programs in graduate education and K-14 outreach. Paid industrial internships for the DEB students are in constant demand, outreach programs such as BiotechSYSTEM, Teen Biotech Challenge, Biotechnology in the Classroom are underfunded, but are critical pieces of biotech education in the regional high schools. Without corporate support, we cannot meet the needs of students involved in biotechnology.

Q: What are some of the challenges you have already overcome?

A: We continually seek new corporate partners for DEB industrial internships and sponsorships for our “Train the Trainer” workshops and biotech enrichment programs for middle school and high school students. High school students who enter the Teen Biotech Challenge are hungry for summer research experiences, but funding and research mentors are limited. To address this need, the biotechnology program has partnered with the Institute of Regenerative Cures at the School of Medicine to apply for a creativity award through the California Institute of Regenerative Medicine to provide summer research experiences for winners of the TBC.

Q: What are you most looking forward to hearing about at BioMed Innovation Night?

A: I am eager to meet Percival Barretto-Ko in person and learn more about ScienceWoRx. He and I share a passion for medicine as well as teaching. I would like to explore strategies as to how we might develop a similar partnership in the Sacramento Valley. Academic-industry-government partnerships will enable us to build a strong STEM workforce.






CHI-Advancing California biomedical research and innovation