CORAL GABLES, FL – February 11, 2013 -- 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.
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: www.clinicaltrials.gov; Search “amifampridine
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. Page 3
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
- 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
n the study; and
- CPP-115 was rapidly absorbed (time to peak blood concentration was about 30
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
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 Page 4
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
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
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.
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.