Replies to post #50 on Biotech Values

[DewDiligence]

The Special Protocol Assessment:

katie, drbio, et al: It is my expectation that GENR and its partner-to-be will deal with the vagaries of the FDA by submitting a Special Protocol Assessment (SPA) for the phase-3 Squalamine trial(s) in AMD.

An SPA, once endorsed by the FDA, becomes a binding contract which assures FDA approval if the safety and efficacy endpoints of the trial(s) are met. As such, the SPA process is akin to a miniature, compressed version of the FDA’s NDA/BLA approval process.

To fully understand SPA’s, please read this:

http://www.fda.gov/cder/guidance/3764fnl.htm

To steal an old cliché about the internet, when it comes to working with the FDA, the SPA changes everything!”

By way of background, I loosely follow a great many drug and biotech companies –not with sufficient DD to own them but rather to stay abreast of trends affecting the industry. SPA’s have been gathering momentum in recent months and are on the verge of becoming de rigueur for all high-risk pivotal trials. By exploiting the SPA process, several small biotechs are embarking on phase-3 trials earlier in the clinical development program than they might have been willing to do without the “cover” of an SPA. I see no reason why GENR and partner will not adopt these same tactics.

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Regarding the alternate delivery mechanisms drbio referred to, I think those studies would be highly desirable, provided that they are done in parallel with the pivotal phase-3 trials using the existing delivery mechanism.