Horizon Pharma, a biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, has announced that the phase III trial, STEADFAST (Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich's Ataxia study), evaluating ACTIMMUNE (interferon gamma-1b) for the treatment of Friedreich's ataxia (FA) did not meet its primary endpoint of a statistically significant change from baseline in the modified Friedreich's Ataxia Rating Scale (FARS‐mNeuro) at 26 weeks versus treatment with placebo. FARS‐mNeuro 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.
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 (FARA) Collaborative Clinical Research Network (CCRN) 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.
Patients participating in the ongoing extension studies should contact their study site coordinator for further information and next steps.
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. "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."
"FARA's mission is to drive research to develop therapies that will treat and cure Friedreich's ataxia and we remain passionately committed to that mission so that one day soon patients and caregivers impacted by this devastating disease will have effective treatment options," said Ronald J. Bartek, co-founder and founding president, FARA. "We want to extend our sincere appreciation to all of the patients, patient families and investigators who were a part of this study as well as Horizon for collaborating with us so impressively on this important research."
The announcement 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.