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Re: DewDiligence post# 192557

Monday, 06/15/2015 6:36:04 PM

Monday, June 15, 2015 6:36:04 PM

Post# of 252903
AAVL CC notes:

All told, the CC was painful to listen to. In response to nearly every question, the AAVL executives gave non-answers that merely repeated the figures from the press release (http://finance.yahoo.com/news/avalanche-biotechnologies-inc-announces-positive-200200882.html ). They also repeated at every opportunity that this was a “positive” phase-2 study, as though they were trying to convince themselves.

Management had no credible explanation for the inverted outcome on the OCT-thickness metric. (The worst-case scenario here, which nobody discussed, is that the increase in OCT thickness for patients in the AVA-101 arm was due to the AVA-101 procedure itself.) Management also danced around the question of whether a mean reduction of one injection per year (for the AVA-101 arm vs the control arm) was a meaningful practical benefit.

The statsig gain in VA from baseline (for the AVA-101 arm vs the control arm) seemed impressive at first glance, but the control arm performed much worse on VA than Lucentis PRN-dosed patients have performed in any other trial I’m aware of.

Bottom line: I remain unconvinced that gene therapy is a fruitful approach to treating a multifaceted disease such as wet AMD (#msg-112775480). I would not go so far as to say the AVL-101 program is dead, by I think it faces an uphill battle.

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