Two patients out of 10 have dropped out of Catalyst Pharmaceuticals’ (NASDAQ:CPRX)-financed, investigator-sponsored Phase II/III trial of Firdapse (amifampridine phosphate; 3,4-DAP) in the myasthenia gravis MuSK protein-seropositive subgroup (MuSK-MG), a source familiar said.
The source said the 10th patient was enrolled and dosed in early December, and final read-outs from this cohort are expected this month. After unblinding, data analysis will take one month, the source said. Catalyst stated in a company filing top-line results will be published in 1H17.
The company had not commented publically on other trial aspects or results, except Catalyst CEO Patrick McEnany said if the pilot study produces a positive response, the company will initiate a larger Phase III, multicentre study in the US later in 2017 or in early 2018.