The independent Data Monitoring Committee (:DMC) has approved the continuation of Catalyst Pharmaceutical Partners Inc.’s (CPRX) phase III study (n=36) on Firdapse in the U.S. and EU. Firdapse is being evaluated for the Lambert-Eaton myasthenic syndrome (:LEMS) indication.
The company expects to complete enrolling patients for the randomized, double-blind, placebo-controlled, discontinuation study by year end. Top-line results are expected in the second quarter of 2014.
We remind investors that Firdapse enjoys orphan drug status in the U.S., EU and Switzerland. Catalyst Pharma acquired the marketing rights to Firdapse from BioMarin Pharmaceutical Inc. (BMRN) in Oct 2012. Only the North American rights were licensed. Firdapse is currently in a phase III study for LEMS in the U.S.
Furthermore, in Aug 2013, Catalyst Pharma had announced that Firdapse was given Breakthrough Therapy designation by the U.S. Food and Drug Administration (:FDA). The FDA gave the above-mentioned designation for LEMS indication. The designation from the FDA was based on encouraging data from clinical trials of Firdapse in patients with LEMS.
Meanwhile, Catalyst Pharma also plans to evaluate Firdapse for the treatment of myasthenia gravis and congenital myasthenic syndrome, among others.
BioMarin Pharma currently markets Firdapse in the EU for the treatment of LEMS where it was approved in Dec 2009. Firdapse generated sales of $14.2 million in 2012, up 7.6% year over year.
We note that Catalyst Pharma has been putting substantial efforts to develop its pipeline. For this, the company completed a direct offering of 8.8 million shares of its common in Sep 2013. The company raised $15.1 million by offering the shares at $1.72 per share.
The company intends to use the funds to develop Firdapse and start new studies on another candidate, CPP-115. Catalyst Pharma is looking to evaluate CPP-115 to treat epilepsy and other central nervous disease indications.
Catalyst Pharma carries a Zacks Rank #3 (Hold). Currently, companies which look attractive include Actelion Ltd. (ALIOF) and AMAG Pharmaceuticals, Inc. (AMAG) with a Zacks Rank #1 (Strong Buy) and a Zacks Rank #2 (Buy), respectively.