Catalyst Pharmaceuticals Provides Update on its Clinical Trial for Patients with Congenital Myasthenic Syndromes
December 08 2016 - 8:03AM
Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq:CPRX), a
biopharmaceutical company focused on developing and commercializing
innovative therapies for people with rare debilitating diseases,
today provided an update on its clinical trial with amifampridine
phosphate in patients with congenital myasthenic syndromes (CMS).
After discussions with the U.S. Food and Drug Administration
(FDA) the study has been expanded beyond pediatric patients to
include adult CMS patients and the enrollment size has been
increased to approximately 20 patients. Further, there are now a
total of five sites participating in the study:
- Children’s Healthcare of Atlanta
- Johns Hopkins Pediatric Neurology
- Boston Children’s Hospital
- Nationwide Children’s Hospital
- University of California, Los Angeles, Department of
Neurology
Amifampridine phosphate, Firdapse®, has received
Breakthrough Therapy Designation from the FDA for the treatment of
Lambert-Eaton Myasthenic Syndrome (LEMS), as well as orphan drug
designations for LEMS, CMS, and Myasthenia Gravis.
Patrick J. McEnany, Catalyst's Chief Executive
Officer said, "We are pleased to enhance and expand our clinical
study to include adults with CMS, as we evaluate the efficacy and
safety of amifampridine phosphate in patients of all ages diagnosed
with certain genetic mutations of CMS. Assuming the data from our
study is positive, we will work towards including data and
information on the benefits of amifampridine phosphate for CMS in
our new drug application that we plan to submit for Firdapse.
Additionally, we also expect to include in a new submission the
positive results seen to date in children with CMS who are
currently being treated with amifampridine phosphate under an
investigator treatment IND. We continue to believe that we
will report top-line results from this study in the second half of
2017.”
Dr. Gary Ingenito, Ph.D., Catalyst's Chief
Medical Officer said, "Though CMS is primarily diagnosed in infancy
and childhood, adult patients who have been diagnosed with other
neuromuscular diseases have been found to have CMS; thus, we are
pleased to add this population to our clinical study.
Genetically-confirmed CMS patients older than two years of age
diagnosed with acetylcholine receptor defect, Rapsyn deficiency,
MuSK deficiency, Dok-7 deficiency, SYT2 mutations, SNAP25B
deficiency, and fast channel syndrome may be eligible for this
study. Genetic testing will be provided to potential participants
whose CMS has not been genetically-confirmed within an eligible
diagnosis."
Additional information about this trial
(NCT02562066) can be found on www.clinicaltrials.gov.
About Congenital Myasthenic Syndromes
Congenital myasthenic syndromes, or CMS, are
rare neuromuscular disorders comprising a spectrum of genetic
defects and is characterized by fatigable weakness of skeletal
muscles with usual onset at or shortly after birth or early
childhood; in rare cases symptoms may not manifest themselves until
later in childhood or adulthood. The severity and course of the
disease are variable, ranging from minor symptoms to progressive
disabling weakness; symptoms may be mild, but sudden severe
exacerbations of weakness or even sudden episodes of respiratory
insufficiency also occur.
Congenital myasthenic syndromes are rare,
estimated at one-tenth that of myasthenia gravis, which in itself
is rare. Based on currently available information, Catalyst
estimates that there are between 1,000 and 1,500 CMS patients in
the United States.
About Catalyst Pharmaceuticals
Catalyst Pharmaceuticals is a biopharmaceutical
company focused on developing and commercializing innovative
therapies for people with rare debilitating diseases, including
Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic
syndromes (CMS), infantile spasms, and Tourette's Disorder.
Firdapse for the treatment of LEMS has received Breakthrough
Therapy Designation from the U.S. Food and Drug Administration
(FDA) and orphan drug designation for LEMS, CMS and Myasthenia
Gravis. Firdapse is the first and only approved drug in Europe for
symptomatic treatment in adults with LEMS.
Catalyst is also developing CPP-115 to treat
infantile spasms, epilepsy and other neurological conditions
associated with reduced GABAergic signaling, like post-traumatic
stress disorder and Tourette's Disorder. 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. In addition, Catalyst is developing a generic version
of Sabril® (vigabatrin).
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 whether the receipt of breakthrough
therapy designation for Firdapse will expedite the development and
review of Firdapse by the FDA or the likelihood that the product
will be found to be safe and effective, the timing of Catalyst's
second clinical trial evaluating Firdapse for the treatment of LEMS
and whether the trial will be successful, whether Catalyst's
assumptions in its updated business plan will be accurate and the
impact of unanticipated events or delays in projected activities on
Catalyst's cash requirements and on Catalyst's ability to get to an
accepted NDA submission for Firdapse without the need for
additional funding, what clinical trials and studies will be
required before Catalyst can resubmit an NDA for Firdapse for the
treatment of CMS and whether any such required clinical trials and
studies will be successful, whether the investigator-sponsored
study evaluating Firdapse for the treatment of MuSK-MG will be
successful, whether any NDA for Firdapse resubmitted to the FDA
will ever be accepted for filing, the timing of any such NDA filing
or acceptance, whether, if an NDA for Firdapse is accepted for
filing, such NDA will be given a priority review by the FDA,
whether Firdapse will ever be approved for commercialization,
whether Catalyst will be the first company to receive approval for
amifampridine (3,4-DAP), giving it 7-year marketing exclusivity for
its product, whether CPP-115 will be determined to be safe for
humans, what additional testing will be required before CPP-115 is
"Phase 2 ready", whether CPP-115 will be determined to be effective
for the treatment of infantile spasms, post-traumatic stress
disorder, Tourette's Disorder or any other indications, whether
Catalyst can successfully design and complete a bioequivalence
study of its version of vigabatrin compared to Sabril that is
acceptable to the FDA, whether any such bioequivalence study the
design of which is acceptable to the FDA will be successful,
whether any ANDA that Catalyst submits for a generic version of
Sabril will be accepted for filing, whether any ANDA for Sabril
accepted for filing by the FDA will be approved (and the timing of
any such approval), 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 2015 and its 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.
Investor Contact
Brian Korb
The Trout Group LLC
(646) 378-2923
bkorb@troutgroup.com
Company Contact
Patrick J. McEnany
Catalyst Pharmaceuticals
Chief Executive Officer
(305) 420-3200
pmcenany@catalystpharma.com
Media Contacts
David Schull
Matt Middleman, M.D.
Russo Partners
(212) 845-4271
(212) 845-4272
david.schull@russopartnersllc.com
matt.middleman@russopartnersllc.com
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