Horizon Pharma Craters as Rare Neuromuscular Drug Flunks Late-Stage Study
December 8, 2016
By Alex Keown, BioSpace.com Breaking News Staff
DUBLIN – Shares of Ireland-based Horizon Pharma were down more than 18 percent this morning after the company terminated its Phase III trial to expand use of Actimmune after the drug failed to meet endpoints in treating Friedreich's ataxia, a rare neuromuscular disorder.
The company said Actimmune did not meet the 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.
Additionally, the trial’s secondary endpoint failed to meet statistical significance, the company said this morning. As a result of the failures, Horizon said the Friedreich's Ataxia program will be discontinued, which includes a 26-week extension study and the long-term safety study.
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.
Friedreich's ataxia is a rare genetic disease that causes nervous system damage and movement problems. It typically manifests in childhood and leads to impaired muscle coordination (ataxia) that worsens over time, according to the National Institute of Neurological Disorders. The spinal cord and peripheral nerves of FA patients degenerates over time, as does the cerebellum. The disorder also causes problems in the heart and spine, and some people with the condition develop diabetes. The disorder does not affect thinking and reasoning abilities, the NIND said on its website. FA afflicts one in about every 50,000 people. The disease can shorten life expectancy, with heart disease being the most common cause of death. However, the NIND noted that some people with less severe features of Friedreich's ataxia live into their sixties, seventies or older.
Actimmune (interferon gamma-1b) is a biologically manufactured protein similar to one the body makes naturally to help prevent infection. It is currently approved by the U.S. Food and Drug Administration for use in two rare diseases. It is indicated for reducing the frequency and severity of serious infections associated with Chronic Granulomatous Disease, a genetic disorder that affects the functioning of some cells of the immune system. In addition, Actimmune is approved for delaying time to disease progression for patients with severe, malignant osteopetrosis, a genetic disorder that affects normal bone formation.
Actimmune generated about $80 million in the first three quarters of 2016.
Although the FA trial failed, Horizon has a number of other drugs in its pipeline to treat orphan diseases, particularly following its nearly $800 million acquisition of Raptor Pharmaceutical Corp. That deal brought 11 medicines across three business units: orphan, rheumatology and primary care, under the Horizon banner.
Horizon said the termination of the FA trial will not impact the company’s full-year 2016 adjusted net sales. Despite the closure of the trial, Horizon said it is still well-positioned for growth in 2017 and beyond based on its pipeline.