Therapeutic focus – ALS field entering pivotal stage

The past few months have seen a sudden flurry of activity within the niche but major unmet medical field that is amyotrophic lateral sclerosis (ALS) – last month Actelion paid €10m for an option to acquire the private French group, Trophos, should olesoxime prove successful in a phase III ALS trial which will read out next year; while in May Teva’s ALS candidate, talampanel, failed a phase II study.

The only FDA-approved drug for ALS, riluzole, offers a modest increase in average survival time. Similar to many neurodegenerative disorders, a genuine disease-modifying therapeutic agent is desperately needed for patients with ALS, estimated to affect 50,000 people worldwide. Actelion’s move on Trophos and Teva’s setback throws the spotlight on a rather thin-looking late stage pipeline of ALS candidates, although the next 18 months could yet bring some much needed success stories (see tables below).