Catalyst Pharma’s Firdapse shows treatment effect in certain patients with myasthenia gravis; shares jump 14%

Catalyst Pharmaceuticals (CPRX +13.7%) perks up on almost a 5x surge in volume in response to its announcement of positive top-line results from an investigator-sponsored study in Italy assessing Firdapse (amifampridine phosphate) for the treatment of patients with myasthenia gravis (MG) with anti-MuSK antibodies, an ultra-rare subpopulation of MG patients.

The company says both co-primary efficacy endpoints in the seven-subject study were met: the change from baseline in QMG score and MG-ADL score. Several secondary endpoints were also met.

CEO Patrick J. McEnany says, “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. I would like to thank Dr. Mantegazza, his associates at Carlo Besta Neurological Institute, and the patients that participated in this important clinical trial.”

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