Intermune longs breathing easy, Immunocellular gets orphan designation in Europe, Questcor impuned by Citron. Let's go over the stories we've been watching, commenting upon and (in some cases) trading this week.
Intermune reported highly positive results from the phase 3 ASCEND Confirmatory trial of perfenidone in patients with idiopathic pulmonary fibrosis (IPF). The drug significantly reduced IPF disease progression as measured by change in percent predicted forced vital capacity (FVC) from baseline to week 52 (p < 0.000001). Additionally, Secondary endpoints of 6 minute walk distance and progression free survival were both statistically significant (p = 0.0360) and (p<0.0001) respectively.
Recall that ITMN received a positive AD COMM recommendation in 2010 and the FDA decided to overrule the AD COMM and request another clinical trial because only one of the two previous trials achieved statistical significance. In response, ITMN ran the ASCEND trial and used an enriched trial population to ensure that only patients most likely to benefit from perfenidone were enrolled. Meanwhile, the drug has been approved, available and saving lives in the EU for the past 3 years. In this case, the highly statistically significant results in the ASCEND trial show that the FDA made the wrong call in overruling the AD COMM back in 2010.
We predicted that the ASCEND trial would be successful over a year ago in an article published on Seeking Alpha. These trial data suggest that perfendione will be easily approved upon NDA resubmission later this year.