Life Science Leader Magazine

MAR 2014

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EXCLUSIVE LIFE SCIENCE FEATURE leaders LIFESCIENCELEADER.COM MARCH 2014 44 I t hardly made for a comfortable feeling. The companies that attracted big dollars last year were overeager to justify the trust put in them. Other small companies occu- pied another notch up on the anxiety scale, lest they miss out on the high funding tide — one still rising, but whose ultimate crest remains unknown. The large companies, for their part, ranged from defensive but determined, to self- congratulatory. On balance, however, general optimism overcame uncertainty and generated great energy at the event, buoying up the mood of the crowd to match the sunny vista of Union Square outside the St. Francis. Energy is good. Energy plus data is bet- ter. Although I agree companies should not project value or growth on what might happen in clinical trials, the acid test for any company pitching its con- cept is solid, preferably human, data. That axiom applies especially to the cur- rent race for accelerated review at the FDA. Another principle that boiled up again and again at the event: Business models matter. There is no stage too early to define one. When a company creates a new platform or product, it should ask itself, "How will this work in the patient/practice setting? Who will be willing to pay for it, and how much? How will we make the product, distribute it, and ensure its safe supply?" Thus, when Novartis said it was feel- ing good about the potential break- through drugs in its pipeline, giving honorable mention to its CART (chi- meric antigen receptor therapy) proj- ect with the University of Pennsylvania, my tweeted comment was, "I still have doubts on CART biz model — cell manipulation unwieldy." My doubts do not concern CART's basic viability but the issue of practical limitation — how widely can such a complicated proce- dure be applied? For example, even after decades of use and improvement, blood stem-cell transplants have never become commonplace. Any successful immunotherapy helps prove we can employ the immune system to fight cancer and perhaps other dis- eases, but the challenge is to do it with the most practical, cost-effective treat- ment forms and delivery means possible. If not oral drugs, then injectables or even IV formulations would seem, at least at this point, superior to cell transfer. But who am I to argue with Novartis? The company reached the traditional Big Pharma double-digit growth levels in 2013, reporting a third-quarter rate of 17 percent. Nevertheless, Novartis also declared a new level of introspection: Get ready for some business-unit dives- titures, central-procurement and manu- facturing-quality initiatives, and increas- ing emphasis on productivity in 2014 to overtake patent and revenue losses. BIG PHARMA EDGES BACK IN During the conference, Chief Editor Rob Wright and I met with, or simply met, My first tweet at this year's JP Morgan Healthcare Conference was, "Interesting Celgene bases forecasts on 'operational momentum' not pending clinical trial results." It was far from the only place the word momentum appeared at the conference; for the key question on everyone's mind was how much the stunning surge in IPOs and other funding for small life sciences companies in 2013 would carry over into the new year. If I had to hazard a description of the consensus answer, it would be, "No more boom, but no bust either." MAINTAINING MOMENTUM: 2014 PHARMA & FUNDING FORECASTS AT JPMHCC By W. Koberstein 0 3 1 4 _ F e a t u r e _ J P M _ F . i n d d 2 0314_Feature_JPM_F.indd 2 2 / 2 0 / 2 0 1 4 4 : 4 6 : 1 3 P M 2/20/2014 4:46:13 PM

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