Overview

Seeking to Improve the Lives of Patients with ALS

Knopp’s most advanced R&D program is the development of dexpramipexole, a novel, oral investigational treatment for amyotrophic lateral sclerosis (ALS). ALS is a devastating disease of progressive paralysis with very limited treatment options. Following rapid development in Phase 1 and Phase 2 clinical trials, we licensed dexpramipexole for late-stage development and commercialization to Biogen Idec, whose large, Phase 3 trial in ALS failed to meet prespecified endpoints. Knopp plans to continue development of dexpramipexole in specific ALS populations that may benefit from treatment with the drug.

In our Phase 2 study, dexpramipexole showed trends toward improvement in both survival and functional decline. A statistically significant result was observed in a prespecified exploratory endpoint known as the Combined Assessment of Function and Survival (CAFS), a joint rank analysis of functional outcomes adjusted for mortality. Adverse events noted in the trial included falls, muscle weakness, post-lumbar puncture syndrome, and headache, with no trend in differences in frequency among dose groups. Infrequent, reversible neutropenia was also observed.