Catalyst Pharma adds to rally on Firdapse outlook, up 15%

Catalyst Pharmaceuticals (CPRX +14.7%) is up on 50% higher volume. Shares have rallied over 50% since last week when it delivered a corporate overview at Oppenheimer’s healthcare event.

Investors appear more confident with the prospects of Firdapse (amifampridine), approved in the U.S. in late November 2018 for a rare autoimmune disorder called Lambert-Eaton myasthenic syndrome (LEMS). Commercial launch began in mid-January. There has been a bit of controversy over its $375K list price since patients had previous access to it at no cost from a New Jersey drug maker, creating concerns about its market potential.

Per its slide presentation, there are two potential catalysts that should happen later this year: the release of topline data from a Phase 3 clinical trial in congenital myasthenic syndromes (CMS) and topline data from another Phase 3 in muscle-specific tyrosine kinase myasthenia gravis (MuSK-MG).

Topline data from a proof-of-concept Phase 2 study in spinal muscular atrophy type 3 should be available in H1 2020.

https://seekingalpha.com/news/3445407-catalyst-pharma-adds-rally-firdapse-outlook-15-percent