Catalyst Pharma Closes Patient Enrollment In Pivotal Phase 3 Trial Of Firdapse

(RTTNews) - Catalyst Pharmaceutical Partners Inc. (CPRX) announced that it has completed the patient enrollment for its pivotal Phase 3 trial evaluating the safety and efficacy of Firdapse for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome or LEMS.

The company believed that it has enrolled a sufficient number of LEMS patients to ensure that 36 patients will be randomized into the double-blind, placebo-controlled, discontinuation portion of the trial. The company continues to screen additional, previously identified LEMS patients who have expressed interest in participating in this study. As allowed in the protocol for this study, all LEMS patients who are not randomized can continue to receive Firdapse as participants in the two-year follow-up period.

The Firdapse Phase 3 trial utilizes a randomized, double-blind, placebo-controlled, discontinuation design. The trial is being conducted at 22 sites in the United States, Canada, South America and Europe. After enrolled patients have been treated with Firdapse for at least 91 days, they are randomly assigned to either continue on Firdapse or be discontinued to placebo over a 2-week period.

The primary endpoint of the Phase 3 trial is a comparison of changes in patients randomized to continue Firdapse versus those who transition to placebo that occur in both the QMG score, which measures muscle strength, and subject global impression score, on which the subject rates their global impression of the effects of a study treatment during a 14-day double-blind efficacy evaluation period.

The secondary endpoints are change in the investigator's assessment of worsening of disease symptoms and changes in walking speed (Timed 25-foot walking test) during the two-week, double-blind testing period.