Horizon Pharma: Phase 3 Trial Did Not Meet Primary, Secondary Endpoints
Horizon Pharma announced that the Phase 3 trial, STEADFAST, evaluating ACTIMMUNE for the treatment of Friedreich's ataxia, did not meet its primary endpoint of a statistically significant change from baseline in the modified Friedreich's Ataxia Rating Scale at 26 weeks versus treatment with placebo. In addition, 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.
The Company, 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. The company will continue to work with FARA and the principal investigator to further analyze the data to help inform future research efforts as well as future data presentation or publication.
"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," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "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 announcement today does not impact Horizon Pharma's full-year 2016 adjusted net sales or adjusted EBITDA guidance, and the Company believes it is well-positioned for growth in 2017 and beyond based on its existing portfolio of medicines.
Disclosure: None