Can GENR proceed directly to phase 3?
>> It is difficult for me to envision the FDA approving a large scale PIII, or a partner willing to invest the substantial resources in it, based solely on a single-center, non-controlled, ex-US trial, regardless of how good the results appear. IMHO you need a larger confirmatory trial, with a placebo group. <<
Hi Bob. Your view seems to be in the majority here, and is reinforced by Dr. Levitt’s mention of a planned phase-2 “scheduling” trial at the R&R webcast.
However, I think that by exploiting the advantages of a Special Protocol Assessment (see message #51), GENR and partner might still be able to hop directly to phase-3 pivotal trials without incurring undue risk of failure.
In phase 3, it is likely that each of the main subtypes of wet AMD (predominantly classic, minimally classic, and occult) will require its own trial because the FDA tends to view these subtypes as distinct diseases. In a pinch, the minimally-classic and occult subtypes might be combined into one trial (as has been done with Lucentis), but the predominantly-classic subtype will almost certainly be studied separately from the others because Visudyne is the approved standard of care in this setting.
Conducting two, or even three, phase-3 trials whose designs are bolstered by an SPA may obviate the need for additional preparatory phase-2 studies. Running additional phase-2 trials in parallel with the phase-3 studies is clearly preferable from a time-to-market standpoint, and this may be an issue GENR’s partner deems to be of considerable importance. Regards, Dew
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