The independent Data Monitoring Committee (DMC) has approved
the continuation of
Catalyst Pharmaceutical Partners Inc.
) phase III study (n=36) on Firdapse in the U.S. and EU. Firdapse
is being evaluated for the Lambert-Eaton myasthenic syndrome
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.
) in Oct 2012. Only the North American rights were licensed.
Firdapse is currently in a phase III study for LEMS in the
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
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
AMAG Pharmaceuticals, Inc.
) with a Zacks Rank #1 (Strong Buy) and a Zacks Rank #2 (Buy),
ACTELION LTD (ALIOF): Get Free Report
AMAG PHARMA INC (AMAG): Free Stock Analysis
BIOMARIN PHARMA (BMRN): Free Stock Analysis
CATALYST PHARMA (CPRX): Free Stock Analysis
To read this article on Zacks.com click here.