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Catalyst shares jazzed by a new round of positive results for a rare neuromuscular disease

Catalyst CEO Patrick J. McEnany

Catalyst Pharmaceuticals says it scored promising data from a tiny mid-stage study of its lead drug Firdapse in an ultra-rare neuromuscular disease, setting up a shot at a pivotal study that can aim at an FDA approval. But this time the biotech — which has some credibility problems after an embarrassing setback last year — wants some feedback from the agency before setting up a timeline.

A year ago, the FDA sent Catalyst back to the drawing board with Firdapse, handing the biotech a rare refuse-to-file letter for the drug as a treatment for Lambert-Eaton myasthenic syndrome (LEMS), a rare neuromuscular condition, later saying it would need to run a new study and gather more data ahead of an approval. The FDA has handed the company a breakthrough therapy designation for that indication.

Today, investigators for the company say the drug delivered a statistically significant result for primary and secondary endpoints for another rare neuromuscular condition: cases of myasthenia gravis with anti-MuSK antibodies. The study recruited 7 patients for this study and the company says the patient population in the US probably hovers around 4,500.

Gary Ingenito, Catalyst CMO

Catalyst’s shares $CPRX jumped 22% on the news.

“These data announced today should allow us to accelerate our MuSK-MG program over the coming months, as we expect to consult with our external experts and regulatory agencies on a pivotal clinical development plan,” said Catalyst CEO Patrick J. McEnany.

“While several effective treatment options exist for the anti-acetylcholine receptor form of myasthenia gravis (AcHR-MG), MuSK-MG has been particularly refractory to current MG treatment options and represents an unmet medical need in the MG community of patients,” stated Gary Ingenito MD, PhD, Catalyst’s Chief Medical Officer.  Dr. Ingenito continued: “If the significant clinical effect observed in this trial is reproduced in a multicenter trial, amifampridine phosphate would, upon approval, likely become the first line standard of care for MuSK-MG.”


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