Horizon Pharma plc Initiates Phase 3 Trial of ACTIMMUNE(R) (interferon gamma-1b) for the Treatment of People With Friedreich's Ataxia

Horizon Pharma plc

, a biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, today announced it has initiated the Phase 3 Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia study ("STEADFAST") of ACTIMMUNE (interferon gamma-1b) for the treatment of people with Friedreich's Ataxia (FA), a degenerative neuro-muscular disorder. "As a rare disease with no currently approved treatments, Friedreich's Ataxia represents an area of significant unmet medical need," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "The Phase 3 study represents a significant next step in evaluating ACTIMMUNE in this population, which is approximately four thousand people in the United States. We look forward to continuing to work with the FDA with the goal of providing a potential treatment option to the FA community." This Phase 3 trial (NCT02415127) is a randomized, multi-center, double-blind, placebo-controlled study with patients randomized 1:1 to receive subcutaneous doses of either ACTIMMUNE or placebo three times a week for a total of 26 weeks. Approximately 90 patients will be enrolled at four sites in the United States. The primary endpoint will measure the change in neurological outcome and evaluate the effect of ACTIMMUNE versus placebo as measured by the modified Friedreich's Ataxia Rating Scale (mFARS), focused on objective neurologic measures such as upper and lower extremity coordination improvement from baseline. FARS is used to measure neurological signs associated with FA, with higher scores reflecting a greater level of disability. In addition to safety and efficacy, the STEADFAST trial will evaluate the pharmacokinetic characteristics of ACTIMMUNE in people with FA. "This clinical trial is an example of the progress the scientific community has made in understanding FA," said Ronald J. Bartek, president, director and co-founder, Friedreich's Ataxia Research Alliance (FARA). "Less than 20 years ago, we had not identified the cause of FA, and today we have a late-stage clinical study that brings us one step closer to potentially having the first treatment for people with FA." In April 2015, ACTIMMUNE was granted Fast Track status for FA by the U.S. Food and Drug Administration (FDA). This designation provides greater access to and more frequent communication with the FDA throughout the entire drug development and review process, with the goal of possibly expediting approval. Fast Track designation also gives Horizon Pharma the opportunity to potentially submit sections of the ACTIMMUNE registration dossier for FA on a rolling basis, and allows ACTIMMUNE to be considered for priority review at the time of submission based on forthcoming clinical data.