Biotech Values

[DewDiligence]

ICPT starts phase-3 NASH trial called REGENERATE:

http://finance.yahoo.com/news/intercept-pharmaceuticals-announces-initiation-regenerate-110500145.html

The REGENERATE study population will be primarily comprised of NASH patients with stage 2 or stage 3 liver fibrosis…. In addition, the trial will include an exploratory cohort of NASH patients with early stage 1 liver fibrosis and concomitant diabetes, obesity or elevated alanine aminotransferase (ALT) who are at increased risk of progression to cirrhosis.

REGENERATE has been designed as a double-blind, placebo-controlled Phase 3 clinical trial expected to enroll approximately 2,000 NASH patients at up to 300 qualified centers worldwide and assess the potential benefits of OCA treatment on liver-related and other clinical outcomes.

The trial will include a pre-planned interim histology analysis after 72 weeks of treatment in 1,400 patients, which is intended to serve as the basis for seeking initial U.S. and international marketing approvals of OCA for the treatment of NASH patients with fibrosis. Two co-primary endpoints will be assessed in the interim analysis: (i) fibrosis improvement with no worsening of NASH and (ii) NASH resolution with no worsening of fibrosis.

The trial design wrt to the interim analysis has been previously discussed on this board. The co-primary endpoints in the interim analysis—both of which must be met in the ITT population in order for the trial to be usable for accelerated approval—are a high hurdle, IMO. (Note, however, that the interim analysis does not have a composite primary endpoint—i.e. it is not a requirement that any specific patient achieve both conditions (i) and (ii) above.)

Please see #msg-116991033 and #msg-113831454 for related info.