Horizon Pharma PLC (NASDAQ: HZNP) saw its shares take a big step back on Thursday after the company reported topline results from one of its late-stage trials. Specifically, the company announced that the Phase 3 trial, STEADFAST, evaluating Actimmune (interferon gamma-1b) for the treatment of Friedreich’s ataxia (FA) did not meet its primary endpoint compared with treatment with placebo.
As for the primary endpoint, Actimmune did not show a statistically significant change from baseline in the modified Friedreich’s Ataxia Rating Scale (FARS‐mNeuro) at 26 weeks. For those that don’t know what the FARS-mNeuro is, it is an exam-based rating scale that measures disease progression based on functional parameters such as speech, ability to swallow, upper and lower limb coordination, gait and posture.
Additionally, the secondary endpoints did not meet statistical significance. No new safety findings were identified on initial review of data other than those already noted in the Actimmune prescribing information for approved indications.
As a result, Horizon Pharma, in conjunction with the independent Data Safety Monitoring Board, the principal investigator and the Friedreich’s Ataxia Research Alliance Collaborative Clinical Research Network in FA, has determined that, based on the trial results, the FA development program will be discontinued, including the 26-week extension study and the long-term safety study.
Timothy P. Walbert, board chair, president and chief executive of Horizon Pharma, commented:
A well-designed, rigorous study like STEADFAST would not have been possible without the extraordinary drive of the FA community, particularly the people who enrolled in the study, the clinical trial investigators and the Friedreich’s Ataxia Research Alliance. While the results were not what we hoped for, this is the very reason why research and development is important – to find answers that may help inform future research.
The company noted that this will not impact full-year 2016 adjusted net sales or adjusted EBITDA guidance.
Shares of Horizon Pharma traded on Thursday 19% lower to $15.70, with a consensus analyst price target of $30.33 and a 52-week trading range of $13.05 to $23.44.