Biotech Values

[DewDiligence]

KYTH attributes the delayed unblinding of the ATX-101 phase-3 North American trials to changes in the PDUFA-V regulations, which strikes me as an odd explanation:

http://finance.yahoo.com/news/kythera-biopharmaceuticals-announces-first-quarter-201000822.html

In August 2012, the Company completed enrollment in its pivotal U.S. and Canadian Phase III clinical trials. The Company is refining its guidance and expects to report topline results late in the third quarter or early in the fourth quarter of 2013 [i.e. about three months later than the prior guidance].

In accordance with the trial protocol, the primary efficacy endpoint is taken 12 weeks after last treatment. There is also a protocol-specified follow-up safety visit, which is an additional 12 weeks after the primary efficacy endpoint. The Prescription Drug User Fee Act, known as PDUFA V, became effective October 2012, and the FDA has subsequently provided guidance on what constitutes a complete New Drug Application (NDA) under PDUFA V. Based on the FDA guidance, the Company will now unblind the trial results after the final follow-up safety visit instead of after the primary efficacy endpoint, as previously planned. As a result of this change, the Company will submit complete study reports that include the efficacy data and all follow-up safety data as part of a planned NDA filing for ATX-101.

Only one analyst on the CC asked about this, and the answer was vague and unhelpful, IMO. The question for investors is whether the protocol change dictating that the 12-week safety extension of the trial be conducted in a blinded manner suggests that ATX-101 has some undisclosed safety issue.

I haven’t heard any other biotech company cite PDUFA-V as a reason for modifying a trial protocol, so my kneejerk response to KYTH’s plan is to be skeptical.