Catalyst Pharmaceuticals Announces Publication of Phase 3 Clinical Trial Data for Firdapse® for the Treatment of Lambert-Eat...
February 26 2019 - 8:03AM
Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq: CPRX), a
biopharmaceutical company focused on developing and commercializing
innovative therapies for people with rare debilitating, chronic
neuromuscular and neurological diseases, today announced the online
publication in the Journal of Clinical Neuromuscular Disease of the
results of its second Phase 3 clinical trial (LMS-003) evaluating
Firdapse® (amifampridine phosphate) for the treatment of
Lambert-Eaton Myasthenic Syndrome (LEMS).
The positive topline results from this trial
were previously announced, but the full results of safety,
efficacy, and other clinical data are now available online. The
LMS-003 trial was a randomized, double-blind, placebo-controlled
design. Both co-primary endpoints were met with statistical
significance (QMG score: p=0.0004 and Subject Global Impression
Score: p=0.0003). This was further supported by similar success
with other endpoints (Triple Timed Up and Go walk test and the
Clinical Global Impression of Improvement). The study provided
statistically and clinically significant evidence that Firdapse was
safe and effective in treating LEMS patients.
These data are more fully described in the
manuscript entitled, “Amifampridine Phosphate (Firdapse) Is
Effective in a Confirmatory Phase 3 Clinical Trial in LEMS”. The
article can be accessed at the Journal of Clinical Neuromuscular
Disease 2019;20:111-119.
https://journals.lww.com/jcnmd/Fulltext/2019/03000/Amifampridine_Phosphate__Firdapse__Is_Effective_in.2.aspx
“The results of this second pivotal Phase 3
trial again demonstrate that Firdapse is effective for the
treatment of LEMS,” said Gary Ingenito, M.D., Ph.D., Chief Medical
Officer of Catalyst Pharmaceuticals. “We are pleased to offer
access, for all adults LEMS patients, to the first FDA approved,
evidence-based treatment available 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, chronic neuromuscular
and neurological diseases, including Lambert-Eaton myasthenic
syndrome (LEMS), congenital myasthenic syndromes (CMS), MuSK
antibody positive myasthenia gravis (MuSK-MG), and spinal muscular
atrophy (SMA) Type 3. Catalyst's new drug application for Firdapse®
(amifampridine) 10 mg tablets for the treatment of adults with LEMS
was recently approved by the U.S. Food & Drug Administration
("FDA"), and Firdapse is now commercially available in the United
States. Prior to its approval, Firdapse for LEMS had received
breakthrough therapy designation and orphan drug designation from
the FDA.
Firdapse is currently being evaluated in
clinical trials for the treatment of CMS, MuSK-MG and SMA Type 3
and has received Orphan Drug Designation from the FDA for CMS and
myasthenia gravis. Firdapse (amifampridine) 10 mg tablets is the
first and only approved drug in Europe for the symptomatic
treatment in adults with LEMS.
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 (i) whether Catalyst will be
successful in commercializing Firdapse, (ii) whether, even if
Catalyst is successful in commercializing Firdapse, Catalyst will
become profitable, (iii) whether Firdapse will ever be approved for
the treatment of CMS, MuSK-MG, SMA Type 3, or any other disease,
and (iv) those other factors described in Catalyst's Annual Report
on Form 10-K for the fiscal year 2017 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
Solebury Trout
(646) 378-2923
bkorb@troutgroup.com
Company Contact
Patrick J. McEnany
Catalyst Pharmaceuticals
Chief Executive Officer
(305) 420-3200
pmcenany@catalystpharma.com
Media Contact
David Schull
Russo Partners
(212) 845-4271
david.schull@russopartnersllc.com
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