BioPharm America™ 2010 Program

Today there is no question that "personalized medicine" is happening, and happening fast. Last year, a single American Society of Clinical Oncology abstract led to a doubling in KRAS testing nearly overnight. Only a year later, KRAS testing rates for colorectal and lung cancer patients stand at nearly 100% at major cancer centers in the USA and Europe.

Now, the question within the healthcare business community must shift from "if" and "when" will you need to incorporate personalized medicine into your business model, to "what role should my company and I play in personalized medicine?". This is a difficult question given the variables at play. Biomarker IP, diagnostics regulation paradigms, next generation technical capabilities, and pricing and reimbursement models are all in a state of flux that has never been seen before. What is clear is that there is a role for every capability; for discovering new compounds, for biomarker discovery led by clinical development, for detection and analysis of biomarkers and, finally, for providing people with high value health plans and pharmacy benefits. The key is that depending on the therapeutic area and stage of the care cycle, these capabilities are either "good enough" or "not good enough." This panel will focus on finding the areas where your capability is not yet "good enough" and therefore where medical value can be added, and how you can partner with others to deliver the value proposition without losing the opportunity to extract that value in the form of dollars and cents.

Moderator: Harry Glorikian – Managing Partner, Scientia Advisors

Panelists:

• Bryan Dechairo – Senior Director, Development Head, Personalized Medicine, Medco Health Solutions
• Susan Graf – Global Head, Strategic Evaluation, Roche Partnering
• Dana Hendricks – Director, Worldwide Business Development, Pfizer
• Chris Varma – Entrepreneur-in-residence, Third Rock Ventures

So, you founded a company, raised capital, had some ups and downs during clinical trials, dealt with the FDA, had investors breathing down your neck, managed a financial crisis, and worked 80-hour weeks. Then you sold the company, made some money—if you’re fortunate, spent a few weeks in the Bahamas—and now you do it all over again?

This session will take a close look at experienced entrepreneurs. What motivates them and why do they select their projects? What were some of their best/worst, easiest/hardest decisions as CEO? Why did certain deals happen, while others fell through? Find out what makes biotech entrepreneurs “tick” and get their take on some pressing issues around financing, partnering and company building.

Moderator: Vaughn Kailian – Managing Director, MPM Capital

Panelists:

• Kevin Johnson – Venture Advisor, Index Ventures
• John Mendlein – Chairman, aTyr Pharma, Fate Therapeutics and Alevium Pharmaceuticals
• Mark Skaletsky – President and CEO, Fenway Pharmaceuticals

Pharma companies are increasingly reliant on external research to fill their pipelines. This session will look at the strategic approach of pharma and large biotech companies to access innovation in the future including an increase in early stage deal activity. The market for post-proof-of-concept assets drew significant competition over the past two years, with most assets getting a good hard look from potential partners. As unpartnered late stage assets begin to appear picked-over, larger companies have more recently turned their heads toward early stage deals, where deal terms have gone from modest to eye-popping, especially in popular therapeutic areas. In addition, larger companies have started to express interest in investing in specific mechanisms of action rather than just disease states as a whole, leading to a secondary level of competition in some classes.

Compounding this new licensing environment is the trade-off that many large companies are making to invest in arguably higher risk early stage assets rather than the potential for quick return of commercial-ready assets that could seamlessly fill “the bag” of their existing sales force. Between in-licensing, acquisitions, collaborative approaches and corporate venture programs, which models will allow the greatest access to innovation in the future?

Moderator: Gautam Aggarwal – Senior Practice Executive, Campbell Alliance

Panelists:

• Daniel Curran – VP, Corporate Development, Millennium: The Takeda Oncology Company
• George S. Golumbeski – Senior VP, Business Development, Celgene Corporation
• Claudia Karnbach – Senior Director, Business Development and Licensing, Oncology, Bayer Healthcare Pharmaceuticals
• James Sabry – VP, Genentech Partnering, Genentech

This session will explore the options for building a biotech company in today’s capital environment. Do you stay “lean and mean” to improve focus and capital efficiency, but also concentrate your risk profile? Or do you make the larger investment in a broader pipeline and infrastructure, to mitigate the risk of a single product failure? This panel of biotech executives and VCs will explore the different models for drug development in a high risk, capital intensive industry.

Moderator: John Carroll – Editor-in-chief, FierceBiotech

Panelists:

• Michael French – President and CEO, Marina Biotech
• Michael Gilman – CEO, Stromedix
• Mark Levin – Partner, Third Rock Ventures
• Eric Risser – VP, Business Development, MacroGenics, Inc.
• David U'Prichard – Partner, Druid BioVentures

In the past the way to commercialization for new technologies and drug candidates was led by traditional venture capital; nowadays the path also includes corporate venture capital (CVCs). Parented by pharmaceutical companies CVCs invest into innovative companies to track technologies and create options to in-license or acquire at a more advanced stage. During the credit crisis, small innovative companies were increasingly capitalized by CVCs first, followed by VCs. Is this merely a trend or is it the new model? What are the pros and cons?

Moderator: Michael Lytton – Executive VP, Biogen Idec

Panelists:

• David Donabedian – VP, Strategic Alliances, US CEEDD, GlaxoSmithKline
• Gwen Melincoff – Senior VP, Business Development, Shire Pharmaceuticals
• Ed Torres – Managing Director, Lilly Ventures
• Alan Walts – Managing Director, Genzyme Ventures

With restless investors, dormant credit and equity markets, and limited exit opportunities, many early stage biotech companies are facing unprecedented financial challenges that make it increasingly difficult to bridge the gap between basic science and commercial viability. Can partnerships between industry, academia, and philanthropic organizations accelerate proof-of-concept? This panel will explore innovative business and organizational models that align with scientific innovation.

Moderator: Anna Protopopas – Senior VP, Corporate Development, Millennium: The Takeda Oncology Company

Panelists:

• Sohini Chowdhury – Acting VP, Research Programs, Michael J. Fox Foundation
• Alan Crane – General Partner, Polaris Venture Partners
• Tilman Gerngross – CEO and Founder, Adimab
• Hambleton Lord – Managing Director, Launchpad Venture Group
• Uwe Schoenbeck – CSO, Worldwide R&D, External R&D Innovation (ERDI), Pfizer

The dual pressure of increasing drug development costs and the decreased availability of capital is forcing biotechnology companies to be more resourceful in attracting capital. Financing specific projects has therefore developed as a new trend with disparate parties stepping up to further the development of drug candidates. Disease foundations jumped into project financing through venture philanthropy, and VCs are now partnering with CROs to source, develop, finance and sell pharma’s latent assests. In a different vein, corporate strategics increasingly lead deals around financing of biotechnology companies, providing validation for venture capitalists. This panel will feature disease foundations, venture capitalists, corporate strategics, and project financing specialist to discuss these novel financing mechanisms.

Moderator: Christopher J. Denn – Partner, Life Sciences, Goodwin Procter

Panelists:

• Louis DeGennaro – Executive VP and CSO, The Leukemia & Lymphoma Society
• Maggie Flanagan LeFlore – Managing Director, MedImmune Ventures, Inc.
• Derek Lee – CFO, Talaris Advisors
• Lauren Silverman – Managing Director, Novartis Option Fund

As we begin to emerge from the capital crisis VCs are increasingly looking at ways to support their portfolios. To do so they are seeking greater capital efficiency and looking for management with the ability to advance projects into development at lower cost. Global drug development resources and R&D collaboration offer a way to reduce expenditure and extend cash. This raises the question—where along the drug development value chain should capital efficiencies be targeted?

Moderator: Morrie Ruffin – Managing Partner, Adjuvant Global Advisors

Panelists:

• Kees Been – President and CEO, EnVivo Pharmaceuticals
• Ivan Gergel – Executive VP, R&D, Endo Pharmaceuticals
• Jeff Jonker – CBO, Satori Pharmaceuticals
• Alain Stricker-Krongrad – CSO, Charles River Laboratories

In a brief and historically aberrant flirtation with early stage licensing beginning in 2005 and ending in 2007, pharma transacted relatively large deals for pre-proof-of-concept programs. In a return to normalcy, pharma’s licensing interests have retreated firmly back to their traditional late stage comfort zone: projects well past proof-of-concept. As a result, today’s headline grabbing deals involve biotechs that have achieved robust Ph IIb data typically in one or more trials with large numbers of patients. Examples include Rigel’s recent Ph IIb deal with AstraZeneca for their Syk inhibitor for RA (USD 100 million upfront and USD 1.24 billion potential value) and Targacept’s Ph IIb deal for their nicotinic channel blocker for refractory depression (USD 200 million upfront and USD 1.2 billion potential value), also with AstraZeneca.

The large valuations for today’s late stage deals (including very substantial upfront payments) look like good news for the “sell side”. Beyond the headlines, though, lies the fact that these successful sellers achieved value inflection after pursuing clinical development for years, while consuming hundreds of millions of dollars of financing. The vast majority of today’s biotechs seeking partners will never see that type of “capital runway”. Today’s companies need to exit on much earlier, often, pre-proof-of-concept data. And the enormous increase in the inventory of companies with pre-proof-of-concept programs seeking pharma partners (in addition to having their own large pre-proof-of-concept inventories) means the “buy side“ can be extremely choosy.

Earlier stage biotechs need to redirect focus from achievement of proof-of-concept to demonstration of proof-of-relevance: that is, the ability to recognize and communicate the indisputable clinical and commercial value of compounds at early stages of development. This talk will focus on the importance of screening early stage programs for proof-of-relevance potential and strategies for achieving value inflection from demonstration of proof-of-relevance in early stage development.

Presenter: Ed Saltzman – President, Defined Health

There has been a renewed interest in antibiotics after a period of merger/change of ownership-induced torpor over the last five years. Most of these agents are focused on Gram positive pathogens like MRSA and are advances on established classes as opposed to novel approaches. Gram negative pathogens look to be the major danger in the coming years, and currently there is a limited choice of marketed and developmental agents able to deal with the ESBL and carbapenemase producing pathogens, with clinicians resorting to decades old approaches like polymixin B. Extreme and multi-drug resistant TB, and the continuing increase in problems caused by Clostridium difficile add to the maelstrom of unmet need in this space. Publicly available information on the current development pipeline and presentations/posters at major conferences like ICAAC do not show a wealth of novel and imminent answers to these problems.

Given the level of unmet need, is this market an attractive prospect for new entrants? If so, where might a new company want to place its bets?

This panel will discuss the future evolution of the battle against pathogens and give some insight into opportunities for all sizes of companies in this longstanding market that remains potentially lucrative into the future.

Moderator: Timothy Joslin – Managing Director, Europe, Defined Health

Panelists:

• Georg Buchner – VP of Corporate and Business Development, Novacta Bioscience
• Stefan Fischer – CEO, FAB Pharma
• Peter Hammann – Head External Opportunities, TSU Infectious Diseases, sanofi-aventis Deutschland GmbH

Decades of advances in nutrition, medicine, diagnostics and preventative healthcare are manifesting in increasing longevity and a growing elderly well population. As demographics shift, progressive degenerative conditions such as Alzheimer's disease, osteoporosis and other musculoskeletal disorders are becoming increasingly prevalent.

Osteoporosis is the poster child for fragility in the aging. The osteoporosis market currently is nearly USD 10 billion worldwide, but faces imminent threat from generic substitution of the largest drug classes used in this setting, bisphosphonates and hormonal agents. It is as yet unclear whether newer formulations of current drug classes or novel MOAs will be shown to provide sufficient clinical benefit to justify their use versus inexpensive generic bisphosphonates. To add further uncertainty to the future of this space, treatment focus to date has been on post-menopausal women, while there exists a large underserved elderly male osteopenic segment.

In our minds, an untapped market potential resides in new markets in the related but relatively underrecognized conditions that can be grouped under the generic heading of diseases of fragility or frailty in the aging, such as sarcopenia and cachexia. The epidemiology and unmet needs in these settings are significant, but the underlying pathophysiological processes are complex, the science supporting developmental agents is immature and clinical endpoints for which to evaluate such drugs are as yet undefined.

In this Therapeutic Insight session, Defined Health and distinguished panelists will characterize the frailty market and discuss pioneering development projects as well as opportunities and obstacles in the creation of value from today’s development pipelines.

Moderator: Michael C. Rice – Senior Consultant, Defined Health

Panelists:

• Helen K. Edelberg, MD, MPH – Senior Director, Therapeutic Strategy Unit on Aging, sanofi-aventis
• Alan Ezekowitz – Senior VP and Franchise Head, Respiratory and Immunology, Merck & Co., Inc.
• Laurie Glimcher – Irene Heinz Given Professor of Immunology, Immunology and Infectious Diseases, Harvard School of Public Health

While glycemic control remains an important objective (and the recognized regulatory marker) for getting to market for type II diabetes agents, there is a growing concern that diabetes as an underlying cause of cardiovascular disease (CVD) is the dominant cost-driver in this ballooning population, and one that isn’t addressed by the new drug classes. Paradoxically, in recent years the cardiac safety concerns associated with Avandia have led to the FDA rewriting guidelines for diabetes drug approval to include a thorough assessment of CV safety prior to approval. Thus, far from needing to show CV benefit, drugs seeking approval for diabetes now need to run large trials to establish that they do not increase the risk of CVD.

In this session of Defined Health’s Therapeutic Insight 2010, the panel will focus on questions such as:

• Are new classes of diabetes drugs that target improved glycemic control, weight loss reduction and/or better tolerability the path forward for patients, physicians, payers and regulators?
• How important or critical are cardiovascular outcomes studies in addition to cardiac safety studies to physicians, payers and regulators? How different are these studies in terms of cost and time investments needed?
• To what extent should/could, and how can diabetes and CVD drug development strategies overlap? Which novel therapeutic targets might address common risk factors between diabetes and CVD? What would be candidate indications or patient populations to pursue drugs for such targets?
• Are there other markers that could add value to a diabetes drug in development such as—improved renal function, weight loss, beta cell preservation, renal protection or macrovascular benefit?

Moderator: Vasantha Malladi, PhD – Consultant, Defined Health

Panelists:

• Charlie Tobler – VP, Global CV/Metabolic Franchise Leader, Takeda Pharmaceuticals International
• Hans-Jürgen Wörle – Therapeutic Area Head Metabolism, Boehringer Ingelheim Pharma