Replies to post #189954 on Biotech Values

[mcbio]

AAVL’s phase-1 data were not as good as some people think. Of the 6 non-control patients (3 in the high-dose arm), only one of them had a VA gain of >=3 lines (15 letters), which is the standard regulatory endpoint in wet AMD.

Moreover, REGN opted not to fork over money for this program on the come, settling instead for a mere right of first negotiation.

Interesting, thanks for the additional color. I'm feeling a bit better about my decision to wait for the pending data before giving any consideration. ; )

From a scientific standpoint, wet AMD is a multifaceted disease that seems ill suited to gene therapy, IMO.

Are you reasonably optimistic that a dual VEGF+PDGF inhibition approach will be effective in wet AMD?

[biocqr]

AAVL> ...neither the P1 or P2A for AVA-101 are targeting naive patients so not sure VA gains >15 letters is as important. The 8 subjects in the P1 had an avg 18 prior anti-VEGF injections.

P2A endpoints...

The primary endpoint is safety and secondary endpoints include retinal thickness, visual acuity and the need for rescue injections with anti-VEGF therapy (Lucentis).

Also the mean change in VA was +7.5 letters in the combined high/low dose arms vs. -3.5 in the Lucentis arm after 52 wks. The 7.5 letter gain compares favorably to the Lucentis P1 where the mean gain was only 5.5 letters in 24 wks in naive patients.

Also, rescue injections were reduced 89% in the AVA-101 arms.

[biocqr]

AAVL > Dew... endpoints for regulatory approval include # of pats with VA gains >15 letters and mean VA gain...correct? How are those endpoints weighted? Is one more valid than the other? I would think mean VA gain is the more valid measurement.

rescue injections..posted the wrong slide...