US-based Catalyst Pharmaceutical Partners has completed patient enrolment in a Phase III trial assessing the safety and efficacy of Firdapse for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome (LEMS).
The company said that based on the enrolment and randomisation success metrics achieved so far, it believes that it has enrolled enough number of LEMS patients to ensure that 36 patients will be randomised into the trial.
Currently, the company is screening additional, previously identified LEMS patients who have expressed interest in participating in the double-blind, placebo-controlled, discontinuation portion of the Phase III trial.
As allowed in the protocol for the trial, all LEMS patients who are not randomised can continue to receive Firdapse as participants in the two-year follow-up period.
Catalyst chairman and CEO Patrick McEnany said completing enrolment in the LEMS trial marks a key milestone for the company.
"We are on track to report top-line data from the double-blind portion of the trial in the third quarter," McEnany said.
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"If the trial is successful, we expect to begin a rolling NDA filing with the FDA in early 2015, bringing us closer to providing broad access to an FDA-approved treatment for LEMS patients."
The Firdapse Phase III trial is being carried out at 22 sites in the US, Canada, South America and Europe.
Following treatment with Firdapse for at least 91 days, patients are randomly assigned to either continue on Firdapse or be discontinued to placebo over a two-week period.
In order to get additional long-term safety and efficacy data, patients will be given open label Firdapse treatment for a two-year follow-up period after the randomisation phase of the trial.
Primary endpoint of the trial is a comparison of changes in patients randomised to continue Firdapse versus those who transition to placebo that occur in both the QMG score and subject global impression score.
The trial’s secondary endpoints are change in the investigator’s assessment of worsening of disease symptoms and changes in walking speed during the two-week, double-blind testing period.
Catalyst is mainly focused on developing safe and effective, approved medicines to treat orphan neuromuscular and neurological diseases.