Catalyst Pharmaceuticals Announces Publication of CPP-115 Clinical Efficacy Data for Infantile Spasms in Epilepsy & Behavior ...
September 19 2016 - 08:03AM
Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a
biopharmaceutical company focused on developing and commercializing
innovative therapies for people with rare debilitating diseases,
today announced that the journal of
Epilepsy & Behavior
Case Reports has accepted for publication a case report on
the efficacy of CPP-115 in a child with refractory infantile
spasms.
The case report presents a child treated with
CPP-115 through an investigational new drug protocol who
experienced a significant reduction of seizures with no evidence of
retinal dysfunction. The research paper made the following
conclusions:
- The case study suggests sustained efficacy and tolerability of
CPP-115 in treating epileptic spasms
- It reported reduction in seizure frequency and documented
improvements in the EEG interictal and ictal record temporally
associated with CPP-115 initiation for this patient
- It reported that in the context of refractory infantile spasms
and associated morbidity, mortality, and poor neurodevelopmental
outcomes, CPP-115 is potentially a promising alternative to
vigabatrin therapy
Prior to treatment with CPP-115, the patient had
failed ten drugs and the ketogenic diet, and had approximately 100
seizures per day. One year after starting CPP-115 and coming off of
clobazam and vigabatrin, the patient’s reported seizures have seen
a marked reduction in frequency and his cognition and behavior have
improved.
“There is a significant unmet medical need in
the area of refractory infantile spasms, as parents of children who
have infantile spasms have a very difficult choice when it comes to
treatment options, weighing both drug-related risks and adequate
treatment,” said Patrick J. McEnany, Chairman and CEO of
Catalyst. “Our pediatric epilepsy experts advise us that
approximately half of the children diagnosed with infantile spasms
are refractory to the medications approved for the treatment of
infantile spasms.”
About the Published Paper
The research paper was authored by Kyra
Doumlele, Erin Conway, Julie Hedlund, Patricia Tolete, Dr. Orrin
Devinsky, New York University School of Medicine, Comprehensive
Epilepsy Center, New York, New York. The manuscript can be accessed
at
http://www.sciencedirect.com/science/article/pii/S2213323216300391.
About West Syndrome / Infantile
Spasms
An infantile spasm is a type of seizure seen in
an epilepsy syndrome of infancy and childhood known as West
Syndrome. The onset of infantile spasms is usually in the first
year of life, typically between 4-8 months. Spasms often occur in
clusters of up to 100 at a time, and infants may have dozens of
clusters and several hundred spasms per day. Infantile spasms
usually stop by age five, but may be replaced by other seizure
types. Many underlying disorders, such as birth injury, metabolic
disorders and genetic disorders can give rise to spasms, making it
important to identify them (symptomatic IS). In some children, no
cause can be found (cryptogenic IS). Mental retardation occurs in
70-90% of persons with infantile spasms, usually involving severe
to profound retardation. Early control of seizures is critical for
reducing developmental delays and levels of mental retardation, but
~5% of infants with this condition eventually die from
complications caused by the seizures.
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
Mysthenia 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, what study design
for a second trial evaluation of Firdapse for the treatment of LEMS
will be acceptable to the FDA, the timing of such trial, and
whether it 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
Media Contacts
David Schull
Matt Middleman, M.D.
Russo Partners
(212) 845-4271
(212) 845-4272
david.schull@russopartnersllc.com
matt.middleman@russopartnersllc.com
Company Contact
Patrick J. McEnany
Catalyst Pharmaceuticals
Chief Executive Officer
(305) 420-3200
pmcenany@catalystpharma.com
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