Catalyst Pharmaceutical Partners Provides Update on Research and Development Pipeline

Catalyst Pharmaceutical Partners Provides Update on Research and Development

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 Firdapse^TM 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 trial:

    *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);
    *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

    *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

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, Firdapse^TM 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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