Biotech Values

[DewDiligence]

The Squalamine timeline in AMD:

[Reposted from #47685 on Yahoo.]

The launch date for Squalamine in AMD depends on several factors:

1. Starting the phase-3 trials in early 2005 as currently planned.

2. The speed with which patients are enrolled in the phase-3 trials.

3. Whether the FDA agrees to accept an NDA for review based on one year patient data, allowing GENR to submit two-year data in a supplemental filing that doesn’t hold up the NDA itself.

4. How much time is spent by GENR preparing to submit the NDA after the phase-3 trials have produced the requisite data. This in turn depends on completing substantial portions of the non-clinical NDA components, especially the “CMC” (Chemistry, Manufacturing, and Controls) section, while the phase-3 trials are still in progress.

5. The FDA’s willingness to grant a six-month priority review for the NDA (rather than a standard 10-month review).

6. Readiness of the company to launch following an FDA approval.

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I see no reason to be unduly concerned about #1, #4, and #6 (assuming a suitable partner is directly involved by that time).

#3 (NDA submission based on one-year data) is hugely important, but I think Squalamine’s benign safety profile makes this highly probable.

#5 (a six-month FDA review) seems very likely because the FDA has been willing to grant a six-month review for other AMD drug candidates.

The major wild card, in my opinion, is #2 (the speed of patient enrollment). Wet AMD is a very large disease indication. It’s reasonable to expect that, with a sufficient number of participating trial sites and a sufficient amount of information dissemination about the trials, patients with wet AMD ought to be queued up to enroll in a trial for an efficacious drug that does not require a laser or a needle in the eye. Still, if the launch timetable is going to slip, this is probably where it will happen.

In summary, I think a product launch in 2008 is a somewhat conservative projection. If patient enrollment in the phase-3 trials proceeds expeditiously and there are no major problems with the other issues described above, 2007 is very doable. If everything goes perfectly, even late 2006 is possible, although I am certainly not counting on such an outcome.

We will undoubtedly see more erroneous reports on this subject, such as the recent one in the WSJ. When that happens, do not necessarily assume that the author knows something that you don’t!