Trial with interferon gamma-1b missed primary and secondary endpoints


Horizon Pharma is axing further development of its interferon gamma-1b protein therapy Actimmume® for the Friedreich’s ataxia (FA) indication, after a Phase III study missed both its primary and secondary endpoints. The placebo-controlled trial, Steadfast (Safety, Tolerability, and Efficacy of Actimmune Dose Escalation in Friedreich's Ataxia study), failed to show that Actimmune therapy resulted in statistically significant changes from baseline in the modified Friedreich’s ataxia rating scale (FARS-mNeuro), at 26 weeks.

“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,” stated Timothy P. Walbert, the firm’s chairman, president, and CEO.

In May 2016 Horizon had reported licensing to an undisclosed third party the U.S., European, and Canadian IP rights to interferon gamma-1b for the FA indication. Also in May 2016 Horizon and Boehringer Ingelheim agreed to a €25 million deal for Horizon to acquire rights to interferon gamma-1b outside of North America and Japan. The deal effectively gave Horizon global rights to the drug.

Actimmune is already approved in the U.S. for reducing the frequency and severity of serious infections associated with chronic granulomatous disease and for delaying time to disease progression in patients with severe malignant osteopetrosis.

In September Horizon announced an agreement to buy Raptor Pharmaceutical for $800 million. The firm maintains that the acquisition will strengthen its U.S. orphan drug business and provide a springboard for expanding its orphan drug business in Europe and other international markets. Acquired Raptor products include the cysteine-depleting therapy, Procysbi®, for the treatment of nephropathic cystinosis, and the inhaled levofloxacin formulation Quinsair™, for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adult cystic fibrosis patients. Both drugs have been approved in specific countries.








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