Biogen Idec has stopped development of dexpramipexole after it failed to show efficacy in a phase III trial involving patients with amyotrophic lateral sclerosis (ALS).
Dexpramipexole was unable to show a benefit on just about every measure of efficacy employed in the EMPOWER trial, which enrolled almost 950 patients with ALS – also known as motor neurone disease.
The trial did not meet its primary endpoint, a joint rank analysis of muscle function and survival, and no efficacy was seen in the individual components of function or survival, said Biogen. It also failed to show efficacy in secondary endpoints and additional analyses of multiple subpopulations failed to demonstrate any efficacy among these groups, said the pharma company.
The news is a devastating blow to sufferers of ALS around the world, as dexpramipexole had been the first compound to make it through to phase III testing for many years. Other ALS drugs are coming through development but remain at a very early stage.
Jane Gilbert, chief executive of the ALS Association in the US, said she was “deeply saddened” by the news, adding it would be a major disappointment “for the thousands of people living with ALS and their families”.
There is only one approved treatment for ALS – Sanofi’s Rilutek (riluzole) – which was launched in the 1990s and provides only a modest benefit on survival in the disease, which is characterised by progressive muscle weakness and atrophy around the body.
“We share the disappointment of members of the ALS community, who had hoped that dexpramipexole would offer a meaningful new treatment option,” said Biogen’s head of R&D Douglas Williams.
The company said it was committed to finding an effective treatment for ALS and has a number of other research programmes underway, including a research collaboration with Duke University and the Hudson Alpha Institute to sequence the genomes of up to 1,000 people with ALS over the next five years to gain “a deeper understanding about the fundamental genetic causes of the disease”.
The failure also weighed on shares in Biogen, with the stock down nearly 1.5 per cent on the Nasdaq as investors reacted to the demise of one of the company’s main near-term pipeline prospects.
Biogen’s oral multiple sclerosis (MS) drug BG-12 (dimethyl fumarate), which was filed for approval in February 2012, has now taken on even more importance for the company’s growth prospects.