Drug Development

Horizon Pharma folds its cards on Friedreich’s Ataxia after Actimmune flunks PhIII

Timothy P. Walbert, Horizon

Timothy P. Walbert, Horizon

A year-and-a-half after launching their Phase III study of their lead drug Actimmune for Friedreich’s Ataxia, Horizon Pharma has had to call it quits after racking up a full slate of endpoint failures.

Not only did the drug (interferon gamma-1b) flunk the primary on the modified Friedreich’s Ataxia Rating Scale at 26 weeks compared to a placebo, it also failed the secondary guide posts as well. The Dublin-based company is now shuttering the program and walking away.

Trading of Horizon’s shares $HZNP was halted ahead of the announcement early Thursday, then plunged 22% when trading was resumed.

Horizon Pharma has turned to buyouts to expand its portfolio. The company bagged Raptor Pharmaceuticals $RPTP and a pair of rare disease drugs on the market — along with a multi-drug pipeline — in an $800 million cash deal two months ago. Horizon paid $9 a share for Raptor, which also has been pursuing a troubled development path for a new drug for Huntington’s drug, now in a pivotal trial.

“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.”


The best place to read Endpoints News? In your inbox.

Full-text daily reports for those who discover, develop, and market drugs. Join 21,000+ biopharma pros who read Endpoints News by email every day.

Free Subscription

BioNJ CEO Summit 2017

Immuno Oncology 360