Catalyst Pharmaceutical Partners Provides Update on Research and Development Pipeline

Mon Feb 11, 2013 8:03am EST

* Reuters is not responsible for the content in this press release.

CORAL GABLES, Fla., Feb. 11, 2013 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc.
(Nasdaq:CPRX), a specialty pharmaceutical company focused on the development and commercialization
of novel prescription drugs targeting rare (orphan) neurological diseases and disorders, today
provided an update on its research and development pipeline.

"We are providing this information today to update our shareholders, patients, physicians, key
opinion leaders and the financial community on our drug development activities. We are primarily
focused on rapidly advancing the development of FirdapseTM for the treatment of Lambert-Eaton
Myasthenic Syndrome (LEMS), which is our lead product candidate," said Patrick J. McEnany, Chief
Executive Officer of Catalyst.

Portfolio update


In October 2012, Catalyst acquired the North American rights to Firdapse, a proprietary form of
amifampridine phosphate (3-4 diaminopyridine or 3-4 DAP), from BioMarin Pharmaceutical Inc.
("BioMarin"). Firdapse was approved in December 2009 by the European Medicines Agency for the
treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), a rare and sometimes fatal autoimmune
disease characterized by muscle weakness. Firdapse has been granted orphan drug designation by the
U.S. Food & Drug Administration, (FDA) for the treatment of LEMS, making the product eligible to
obtain seven-year marketing exclusivity, if Catalyst is the first pharmaceutical company to obtain
approval of an NDA for its formulation of amifampridine.

As part of its license agreement with BioMarin, Catalyst is taking over the sponsorship of their
ongoing Phase III clinical trial evaluating amifampridine phosphate for the treatment of LEMS. The

* is designed as a randomized double-blind, placebo-controlled discontinuation trial as
recommended by FDA to BioMarin;
* has a goal to enroll approximately 30 LEMS patients (approximately one third enrolled
* currently has 7 active sites (expected to be increased to approximately 25 in the near future);
* has defined as a primary endpoint-change in muscle strength during the 2-week, double-blind
discontinuation period as determined using a validated questionnaire (Quantitative Myasthenia
Gravis score); and
* has defined as a secondary endpoint-change in walking speed (timed 25-foot walk test) during the
discontinuation period.

For further details on this trial, please go to:
; Search "amifampridine phosphate".

With respect to the trial, Catalyst expects:

* to complete enrollment by the end of 2013; and
* to report top-line results from the double-blind portion of this clinical trial during the
second quarter of 2014.

Assuming positive results are obtained from the trial, Catalyst hopes:

* to file an NDA for Firdapse in the first quarter of 2015;
* to obtain approval from the FDA of such NDA by the end of 2015; and
* to commercially launch this product sometime in the first half of 2016.

Firdapse may also be an effective treatment for other neuromuscular orphan indications:

* Congenital Myasthenic Syndrome; and
* Myasthenia Gravis.

Catalyst believes Firdapse can achieve peak annual revenues from sales in the United States of
approximately $100 million.


On August 27, 2009, Catalyst entered into a license agreement with Northwestern University
(Northwestern), under which it acquired worldwide rights to commercialize new GABA
aminotransferase inhibitors and derivatives of vigabatrin which were discovered and patented by
Northwestern. Catalyst has designated the lead compound to be developed under this license as
CPP-115. CPP-115 has been granted orphan drug designation by the FDA for the treatment of
infantile spasms and orphan medicinal product designation in the European Union (EU) for West's
syndrome (a form of infantile spasms). This means this product will be eligible to obtain the
seven-year and ten-year marketing exclusivities available from the FDA and the EU, respectively,
if Catalyst is the first pharmaceutical company to obtain approval of an NDA/MAA for CPP-115.

Based on the results of pre-clinical studies to date, Catalyst believes CPP-115 is:

* more potent; and
* may have fewer side effects (e.g., visual field defects, or VFDs) than vigabatrin.

In October 2011, a pre-IND meeting was conducted with the FDA, during which preclinical and
clinical requirements were defined that would allow Catalyst to complete a development program
through Phase II of CPP-115 for the treatment of infantile spasms.

During the fourth quarter of 2011, Catalyst completed its IND-enabling studies, filed an IND, and
began a Phase I(a) human trial of CPP-115 to evaluate its safety. On May 22, 2012, Catalyst
reported positive results from this double-blind, placebo-controlled, clinical trial evaluating
the safety, tolerability and pharmacokinetic profile of CPP-115. The key findings were:

* CPP-115 was well tolerated at all six doses administered in the study; there were no significant
adverse events, and no cardiovascular or respiratory events were reported in the study; and
* CPP-115 was rapidly absorbed (time to peak blood concentration was about 30 minutes).

Subject to the availability of funding, Catalyst hopes to begin further human clinical trials
evaluating CPP-115 later in 2013. To fund such trials and studies, Catalyst intends to pursue
grants from NIH and foundations. In addition, Catalyst hopes to identify a strategic partner to
work with it in the development and future commercialization of CPP-115.


Catalyst, as a co-inventor, with scientists at New York University and the Feinstein Institute for
Medical Research, recently filed a provisional patent application with the U.S. Patent and
Trademark Office for the use of GABA aminotransferase inhibitors, including CPP-109 and CPP-115,
in the treatment of Tourette's disorder. Catalyst also recently entered into a license agreement
with NYU and the Feinstein Institute granting it worldwide rights with respect to such patent.

Catalyst is currently providing CPP-109 and financial support for a small Phase I/II trial being
undertaken at Mt. Sinai School of Medicine in New York to evaluate the use of CPP-109 in treating
Tourette's disorder. This is a 6-10 patient, open-label trial, from which Catalyst anticipates top
line results during the fourth quarter of 2013. If the results of the study show evidence of
reduced numbers of tics, Catalyst hopes to develop CPP-109 (and/or CPP-115) for this indication,
subject to the availability of additional funds. The Company believes that this indication should
qualify for orphan drug designation from the FDA.

Key development milestones

* Q1 2013

- Report Firdapse Data Monitoring Committee meeting results

* Q4 2013

- Complete enrollment of Firdapse phase III clinical trial

- Report results from Tourette's Disorder study

* Q2 2014

- Report top-line results from Firdapse phase III clinical trial

* Q1 2015

- File NDA for Firdapse

Project discontinuation

CPP-109 for addiction

For several years, Catalyst has been conducting its own clinical trials and studies, as well as
supporting investigator-sponsored trials and studies, evaluating CPP-109 for the treatment of
cocaine and methamphetamine addiction. However, based on the previously announced top-line results
obtained from its most recent Phase II(b) trial of CPP-109 in cocaine dependent subjects,
Catalyst's management and Board of Directors have determined not to focus its future product
development efforts on evaluating CPP-109 for the treatment of drug addictions. Catalyst is
disappointed for all its stakeholders, including patients, investigators, families and advocacy
groups, but believes this is the correct decision for the company under the circumstances.

About Catalyst Pharmaceutical Partners

Catalyst Pharmaceutical Partners, Inc., is a specialty pharmaceutical company focused on the
development and commercialization of prescription drugs targeting rare (orphan) neurological
diseases and disorders, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and
Tourette's disorder. Catalyst's lead candidate, FirdapseTM for the treatment of LEMS, is currently
undergoing testing in a global, multi-center, pivotal phase III trial. Catalyst is also developing
a potentially safer and more potent vigabatrin analog (designated CPP-115 by Catalyst) to treat
infantile spasms, and epilepsy, as well as other neurological conditions associated with reduced
GABAergic signaling, like Tourette's disorder, post-traumatic stress disorder, and movement
disorders associated with the treatment of Parkinson's Disease.

Forward-Looking Statements 

This press release contains forward-looking statements. Forward-looking statements involve known
and unknown risks and uncertainties, which may cause the Company's actual results in future
periods to differ materially from forecasted results. A number of factors, including whether the
Phase III trial of Firdapse will be completed on the timeline described above and will be
successful, whether the Company will, even if the Phase III trial is successful, be permitted to
file an NDA for Firdapse, whether Catalyst will obtain funding to support future development
efforts of CPP-115 and/or CPP-109, whether any of the Company's product candidates will ever be
approved for commercialization and the timing of any such approvals, the level of potential sales
that can be achieved by any of the Company's product candidates that are approved for
commercialization, and those factors described in the Company's filings with the U.S. Securities
and Exchange Commission (SEC), could adversely affect the Company. Copies of the Company's filings
with the SEC are available from the SEC, may be found on the Company's website or may be obtained
upon request from the Company. The Company does not undertake any obligation to update the
information contained herein, which speaks only as of this date.

CONTACT: For Further Information Contact:
         Patrick J. McEnany
         Catalyst Pharmaceutical Partners
         Chief Executive Officer
         (305) 529-2522
         Melody Carey
         Rx Communications Group
         (917) 322-2571

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