PTC Therapeutics, Inc. (NASDAQ:PTCT) shares tumbled nearly 16% this morning after investors learned that the company’s Ataluren Confirmatory Trial (ACT CF) in nonsense mutation cystic fibrosis (nmCF) did not achieve its primary or secondary endpoints.
PTC plans to discontinue current clinical development of ataluren in cystic fibrosis, close ongoing extension studies and withdraw its application for marketing authorization in cystic fibrosis in Europe.
“We are disappointed with the outcome of this trial as there are no treatments that target the underlying cause of nonsense mutation cystic fibrosis, one of the most difficult forms to treat,” said Stuart W. Peltz, Ph.D., chief executive officer of PTC Therapeutics. “We are particularly grateful to patients and investigators who participated in our trials. We remain committed to patients receiving ataluren in other indications.”
ACT CF was a double-blind, placebo-controlled, 48-week clinical trial comparing ataluren to placebo in nmCF patients six years of age or older not receiving chronic inhaled aminoglycosides. The Phase 3 study, conducted in 16 countries, enrolled 279 patients who were randomized to receive either ataluren or placebo. In the intent-to-treat population, the primary endpoint of lung function as measured by absolute change in percent-predicted FEV1 (forced expiratory volume in one second), over 48 weeks from baseline, there was a 0.6% difference in favor of ataluren versus placebo (-1.4% change on ataluren versus -2.0% change on placebo; p=0.534). For the secondary endpoint of rate of pulmonary exacerbations, there was a trend in favor of ataluren, with the rate in the ataluren group being 14% lower than the placebo group (p=0.401). The results were not statistically significant.
On the ratings front, PTC Therapeutics has been the subject of a number of recent research reports. In a report issued on January 9, Cowen analyst Ritu Baral reiterated a Hold rating on PTCT. Separately, on the same day, Jefferies’ Gena Wang reiterated a Hold rating on the stock and has a price target of $12.
According to TipRanks, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Ritu Baral and Gena Wang have a yearly average return of 9.2% and 11.6% respectively. Baral has a success rate of 45% and is ranked #431 out of 4512 analysts, while Wang has a success rate of 52% and is ranked #578.
PTC Therapeutics, Inc. engages in the discovery, development, and commercialization of orally administered, small molecule therapeutics targeting an area of RNA biology. Its preclinical and discovery programs are focused on the development of new treatments for multiple therapeutic areas, including neuromuscular disease, oncology and infectious disease. It product, candidate ataluren, is used for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation.