Re: ICPT's phase-3 NASH design
The trial, called REGENERATE, will have an interim look after 72 weeks for potential FDA accelerated approval and EMA approval, which will include about 1,400 of the 2,500 total patients in the trial.
The co-primary endpoints at the interim look are: i) the proportion of OCA-treated patients relative to placebo achieving at least one stage of liver fibrosis improvement with no worsening of NASH; and ii) the proportion of OCA-treated patients relative to placebo achieving NASH resolution with no worsening of liver fibrosis.
Note that this is not a composite primary endpoint—i.e. it is not a requirement that any patient achieve both conditions i) and ii) above.
The trial will test two doses of OCA: 10mg qD and 25mg qD. The phase-2 FLINT study tested only the 25mg dose. The phase-3 trial is presumably testing a lower dose to see if this can mitigate the increase in LDL seen in FLINT.
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