I expounded more on the reasons that I felt the Phase III would fail at seekingalpha (see link above). Most importantly, the decision to proceed to expensive Phase III testing (1100 patients in 2 studies) was done prematurely based entirely on a very small study of 32 patients, further split into 20 patients with HLA-DR2 or DR4. These 20 patients were further split into 14 SPMS and 6 PPMS patients, a ridiculously small number of patients to try to analyze. No attempt was made to determine if every 6 month dosing was best by doing a larger and more controlled phase II looking at other doses. Maybe the medicine would have worked with monthly dosing. Second, the mechanism of action — tolerance induction — is unlikely as the MINDSET RRMS study failed to show an effect on inflammation, much more suscetible to tolerance induction than the degeneration of SPMS. I may have felt different about the prospects if a plausible MOA was advanced and supported by evidence. An additional reason I may not have mentioned is that 2 years may have been too short for the study.
As long as I’m rambling, I’ll add my two cents about small companies in Biotech and the decisions to proceed to phase III. BioMS could not have proceeded as far as they did without either a partnership, private equity or a second public offering. They rightly calculated that a product just starting Phase III would have more value than one that just has a 20 patient phase II. Initiating the MAESTRO studies allowed them to gain a reasonably good deal with Lilly, a company that could easily spare 97 million (87 + 10 milestone at 200 patient review) to have a shot at a 1 - 2 billion dollar a year drug. Thus, BioMS had nothing to lose by rushing the drug along. A larger company would have done a larger phase II forst and then decide whether to advance the drug to expensive phase III studies or just let the agent die. BioMS, with one drug, could not afford to let the drug die in Phase II. I’m going to keep this in mind when I look at other small companies that may have rushed a product along.
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