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$CPRX 2.30 Catalyst Pharmaceutical Partners Reports Successful Completion of Type B Breakthrough Therapy Meeting With FDA on Firdapse Tablets Development Program
First Meeting With FDA Since Receiving Breakthrough Therapy Designation for Firdapse

Anticipated Timeline for NDA Submission Remains on Track

CORAL GABLES, Fla., Jan. 6, 2014 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Catalyst) (CPRX), a specialty pharmaceutical company focused on developing safe and effective, approved medicines targeting orphan neuromuscular and neurological diseases, today announced the successful completion of a Type B meeting with the U.S. Food and Drug Administration (FDA) about Firdapse(TM) tablets (Amifampridine), its lead product being evaluated for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS).

Catalyst provided FDA with an update on the development program for Firdapse, which received Breakthrough Therapy designation from the FDA in August 2013. The Company also confirmed with FDA the clinical, nonclinical, and chemistry and manufacturing controls requirements that FDA will require to approve a New Drug Application (NDA) for Firdapse.

"Catalyst remains committed to completing our late-stage clinical trial of Firdapse as expeditiously as possible and working with the FDA to provide a complete NDA filing," said Patrick McEnany, President and CEO of Catalyst. "We believe that data obtained to date demonstrate the clinical value and benefit of Firdapse for LEMS patients, who today have no safe and effective, approved drug to treat their disease."

This Type B meeting with the FDA was Catalyst's first meeting as the sponsor of the IND for Firdapse. Catalyst will file rolling submissions of the NDA modules as completed in anticipation of receiving a priority review of its NDA for Firdapse.

The Company provided a briefing package to the FDA that described all completed, in-progress, and planned preclinical studies, clinical studies, and drug manufacturing activities. This package included summaries of 54 preclinical studies, six clinical studies, and information related to drug manufacturing (the clinical supplies and the to-be marketed commercial product). The FDA concurred that the Company's completed, in-progress, and planned development activities represented a nearly complete package of information that would be needed for a complete NDA.

Catalyst also will submit data from additional in-vitro preclinical studies. Based on the discussions at this meeting and based on past communications and meetings with the FDA about Firdapse, all of these studies and remaining development activities constitute information needed to file a complete NDA and seek approval for Firdapse. The Company does not anticipate that these additional studies will impact the NDA filing timeline or materially add to its forecast of the aggregate development costs for Firdapse.

The Company and FDA also discussed the acceptability of the primary and secondary endpoints specified in the protocol for the ongoing Phase 3 trial. FDA requested a slight modification in the analyses to be conducted for the endpoints, which the Company believes will not require any changes in the data being collected or the number of patients needed to complete enrollment.

Update on Phase 3 Clinical Trial Status

The Company anticipates meeting previously disclosed timelines for submission and approval of an NDA for Firdapse for the treatment of LEMS.

In order to accelerate the enrollment in this clinical trial, over the last few months the Company has expanded the on-going clinical trial that was initiated by BioMarin Pharmaceutical, Inc. (BMRN), to include many more sites internationally. The initiation of these clinical trial sites in numerous different countries has presented challenging logistical, contractual and regulatory issues that are nearing closure. These issues included site identification followed by numerous IRB/ethics committee filings, contracts, clinical trial applications, translations, IRB approvals, multilingual drug labeling, and other multinational issues.

There are currently 22 active sites with a sufficient number of already identified LEMS patients to complete enrollment of the trial, based on the screening, enrollment, and randomization success metrics achieved to date. As a result, the Company expects to complete screening and enrollment of the clinical trial during this quarter and to report top line data from the trial in the third quarter of this year.
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