Catalyst Pharmaceuticals Phase IIb clinical trial for Firdapse tablets, published in SAGE Open Medicine, for the treatment of MuSK antibody positive Myasthenia Gravis (MuSK-MG), has met endpoints, as it provided evidence that amifampridine phosphate was safe and effective in treating MuSK-MG patients.
The study was conducted under the supervision of Silvia Bonanno, M.D. and her team at the Istituto Neurologico Carlo Besta in Milan, Italy. As showing safety and efficacy, the co-primary endpoints were statistically met as well as the secondary endpoints.
Gary Ingenito, Chief Medical Officer of Catalyst Pharmaceuticals said that after getting the encouraging results of the pilot study, the company’s multi-center pivotal Phase 3 trial to test Firdapse for the treatment of MuSK-MG is underway. He said that Catalyst Pharmaceutical’s pivotal trial is being conducted under an FDA Special Protocol Assessment (SPA).
“If our Phase 3 trial is successful, we hope that Catalyst will be able to offer physicians and patients alternatives in the treatment of MuSK-MG,” said Ingenito.
About 15% of MG patients test negative for the acetylcholine receptor antibody. These patients have seronegative (SN) MG. Approximately 40-50% of these patients with SNMG, approximately 4,500 patients in the United States, test positive for the anti-MuSK antibody.
The disease is characterized by a predominance in females, a prevalent involvement of cranial and bulbar muscles, high incidence of respiratory crises and a resistance to treatment.
Catalyst Pharmaceuticals, describing the disease, notes that, although many patients with MuSK-MG are presently treated with anticholinesterase inhibitors or immunosuppressants, such patients do not generally respond adequately to these treatments.