Replies to post #186852 on Biotech Values

[DewDiligence]

AGTC abandoned the Leber Congenital Amaurosis program for business reasons, according to the S-1/A filing prior to the Mar 2014 IPO:

http://www.sec.gov/Archives/edgar/data/1273636/000119312514094588/d615962ds1a.htm (p.96)

…we do not plan to conduct additional clinical trials with this product candidate, as we believe the small number of persons affected by the RPE65 form of LCA2, which we estimate at approximately 600 in the United States and Europe combined, are being adequately served by ongoing and planned clinical trials conducted by multiple academic research centers in the United States and several European countries.

I recommend the Dec 2014 Piper Jaffray webcast, which is accessible at http://www.media-server.com/m/p/84gjky9f .

[mcbio]

AGTC

Perhaps because they've got some failures? E.g. They have (had?) an experimental treatment for Leber's Congenital Amaurosis listed in Clinicaltrials that appears to use the same AAV vector they plan to use going forward. Started in 2007 and radio silent. (Note that there were competing treatments that used similar tech and some had some (limited) success - seems to depend upon where you inject it?).

And the non-eye trial they list on their presentation is furthest along (in an on-going ph2b per their presentation), but the data on clinicaltrials says it was essentially a washout. So not sure why it is in ph2b. BTW - I realize that they are now pursuing eyes only, but suggest that dragging along a washout isn't encouraging.

Another key question is why Genzyme walked away from AGTC and their tech before. Does that raise questions in general about their tech and pipeline or was it just something specific to one drug and a move related in part to Sanofi/REGN's close ties?