Catalyst Pharmaceutical Partners Reports Positive Cardiac Safety Results of Its Phase 3 Product, Firdapse Tablets

Catalyst Pharmaceutical Partners Reports Positive Cardiac Safety Results of
Its Phase 3 Product, Firdapse Tablets

CORAL GABLES, Fla., Jan. 8, 2014 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical
Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on
developing safe and effective, approved medicines targeting orphan
neuromuscular and neurological diseases, today announced positive results from
a study jointly funded with, and conducted by BioMarin Pharmaceuticals
(Nasdaq:BMRN) to assess the cardiac safety of Firdapse™ tablets (amifampridine
also known as 3,4-DAP). Firdapse is approved in the E.U., where it is marketed
by BioMarin. Firdapse is also currently in Phase 3 development in the U.S. for
Lambert-Eaton Myasthenic Syndrome (LEMS) by Catalyst.

The study met the pre-specified primary endpoint, demonstrating that at and
above therapeutic levels, there was no effect of Firdapse (administered as
phosphate salt) on heart rate or cardiac depolarization. None of the study
subjects developed new, clinically relevant electrocardiographic/morphological
changes following administration of Firdapse. Additionally, there was no
significant effect of Firdapse on cardiac repolarization as assessed using the
QT interval.

The QT interval represents the amount of time the heart's electrical system
takes to repolarize, or recharge, after each beat. As prolongation of the QT
interval may increase the risk for cardiac arrhythmias and sudden death, the
U.S. Food and Drug Administration (FDA) requires a thorough QT (TQT) study for
most new drugs in development. A TQT study is a specialized clinical trial
designed to assess whether an investigational medication has the potential to
prolong the QT interval.

"The findings of this cardiac safety study are clear. Firdapse did not lead to
QT prolongation, even at high concentrations in the blood," said Charles W.
Gorodetzky, MD, PhD, Chief Medical Officer for Catalyst. Dr. Gorodetzky
continued, "This study was designed in accordance with existing FDA
guidelines. We are confident in these results and will continue to work toward
making Firdapse available to patients in the U.S. if we are able to obtain
marketing authorization from FDA."

"This is the first formal human evaluation of cardiac safety of Firdapse and
we are pleased with the outcome of the study," said Steven Miller, Ph.D.,
Chief Scientific and Chief Operating Officer for Catalyst. Dr. Miller
continued, "It is also important to caution the patient community that these
findings are only relevant to Firdapse and do not necessarily indicate that
other unapproved forms of 3,4-DAP would be safe, because the drug absorption
and blood levels that result from other forms of 3,4-DAP may be different than

At a pre-IND meeting in 2010, FDA requested TQT study results for Firdapse at
exposures higher than typical therapeutic levels be included as part of the
clinical safety package in any New Drug Application filed for 3,4-DAP.

Study Details

This randomized double-blind study, designed in accordance with the FDA's
published guidance on clinical evaluation of QT/QTc interval (ICH E14),
compared the effects of Firdapse at or above therapeutic concentrations to
placebo on the QT interval in 59 healthy human volunteers. The primary
endpoint was to determine whether Firdapse administered at therapeutic and
supratherapeutic concentrations differed from placebo in the mean change in
the QTc interval.

All heart rate correction methodologies that satisfied the pre-specified
selection criteria, including QTcF and QTcB (QTc calculated by Fridericia's
and Bazett's formulas respectively), met the primary endpoint. Moxifloxacin,
an antibiotic known to prolong the QT interval, was administered as a positive

Catalyst expects that BioMarin will present the full data set from this study
at a major medical meeting at some time in the future and will submit the data
for publication thereafter.

About Catalyst Pharmaceutical Partners

Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company
focused on the development and commercialization of novel prescription drugs
targeting rare (orphan) neuromuscular and neurological diseases, including
Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette
Syndrome. Catalyst's lead candidate, Firdapse™ for the treatment of LEMS, is
currently undergoing testing in a global, multi-center, pivotal Phase 3 trial
and recently received Breakthrough Therapy Designation from the U.S. Food and
Drug Administration (FDA). In 2012, Catalyst licensed Firdapse from BioMarin
and Catalyst assumed management of the Phase 3 pivotal trial, initiated by
BioMarin. Firdapse is the first and only European approved drug for
symptomatic treatment in adults with LEMS.

Catalyst is also developing a potentially safer and more potent vigabatrin
analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well
as other neurological conditions associated with reduced GABAergic signaling,
like post-traumatic stress disorder and Tourette Syndrome. CPP-115 has been
granted U.S. orphan drug designation for the treatment of infantile spasms by
the FDA and has been granted E.U. orphan medicinal product designation for the
treatment of West Syndrome by the European Commission.

Forward-Looking Statements

This press release contains forward-looking statements. Forward-looking
statements involve known and unknown risks and uncertainties, which may cause
Catalyst's actual results in future periods to differ materially from
forecasted results. A number of factors, including the timing of completion of
Catalyst's currently ongoing Phase 3 trial of Firdapse™, whether the Phase 3
trial will be successful, whether the receipt of breakthrough therapy
designation for Firdapse will expedite the development and review of Firdapse
by the FDA, whether FDA will give any NDA for 3,4-DAP priority review, or
accept rolling submissions of the NDA modules as completed, whether the
product will be found to be safe and effective, whether an NDA for Firdapse
will ever be accepted for filing by the FDA, the timing of any such NDA filing
or acceptance, whether Catalyst will be the first company to receive an
approval for 3,4-DAP, giving it 7-year marketing exclusivity for its product,
whether any of Catalyst's product candidates will ever be approved for
commercialization or successfully commercialized, and those other factors
described in Catalyst's Annual Report on Form 10-K for the fiscal year 2012
and other filings with the U.S. Securities and Exchange Commission (SEC),
could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are
available from the SEC, may be found on Catalyst's website or may be obtained
upon request from Catalyst. Catalyst does not undertake any obligation to
update the information contained herein, which speaks only as of this date.

CONTACT: Media/Investor Contacts
         Donna LaVoie or David Connolly
         LaVoie Group
         (617) 374-8800
         Company Contact
         Patrick J. McEnany
         Catalyst Pharmaceutical Partners, Inc.
         Chief Executive Officer
         (305) 529-2522
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