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.
Knopp's pipeline consists of investigational drug products that have not been approved by the U.S. Food and Drug Administration. These investigational drug products are still undergoing clinical study to verify their safety and effectiveness.