Catalyst Pharmaceuticals Announces Enrollment of First Patient in Phase 3 Trial of Firdapse® in MuSK Antibody Positive Myast...
April 19 2018 - 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 that the
first patient has been enrolled into its Phase 3 clinical trial
(designated as MSK-002) to evaluate the efficacy and safety of
Firdapse® (amifampridine phosphate) in patients with MuSK antibody
positive Myasthenia Gravis (MuSK-MG).
“By conducting this Phase 3 study in patients
with MuSK-MG, we hope to provide a potential treatment option for
people suffering from this rare condition,” said Patrick J.
McEnany, Chairman and CEO of Catalyst. “Catalyst continues to build
a leadership position in developing therapies to treat rare
neuromuscular diseases with this next step for an important
investigational product to potentially treat the symptoms of
MuSK-MG.”
In August 2017, Catalyst announced that it had
reached an agreement with the U.S. Food and Drug Administration
(FDA) under a Special Protocol Assessment (SPA) for the protocol
design, clinical endpoints, and statistical analysis approach in
the Phase 3 trial. Catalyst has also received Orphan Drug
designation for Firdapse for the treatment of Myasthenia
Gravis.
“There is a significant unmet medical need to
treat the symptoms of MuSK-MG, and these patients are eagerly
awaiting a new treatment option,” said Gary Ingenito, M.D., Ph.D.,
Chief Medical Officer of Catalyst. “The previous Catalyst
supported, proof-of-concept investigator-sponsored study in MuSK-MG
patients showed impressive clinical improvement in multiple
measures. We’re pleased to have begun enrolling patients in this
study and look forward to working closely with the MG community to
advance Firdapse through this Phase 3 trial.”
About the MSK-002 Clinical TrialCatalyst
previously announced that it had reached agreement with the FDA on
a special protocol assessment for its Phase 3 trial (designated as
MSK-002) of Firdapse in patients with MuSK-MG. This is a
particularly severe form of myasthenia gravis that affects about
3,000 to 4,800 patients in the U.S., for which there are no
approved effective therapies and is therefore an unmet medical
need. The double-blind, placebo-controlled withdrawal trial will be
conducted at clinical trial sites in the U.S. and Italy and is
targeted to enroll about 60 subjects diagnosed with MuSK-MG.
The trial will employ a primary endpoint of Myasthenia Gravis
Activities of Daily Living (MG-ADL) and a secondary endpoint of
Quantitative Myasthenia Gravis Score (QMG). At the FDA’s
request, the trial will also enroll up to 10 generalized myasthenia
gravis patients who will be assessed with the same clinical
endpoints, but achieving statistical significance in this subgroup
of patients is not required and only summary statistics will be
provided. We anticipate that it will take about 12 months to
complete the enrollment for the trial.
Additional information about MSK-002 can be
found on www.clinicaltrials.gov (NCT03304054).
About myasthenia gravis and MuSK-MGMyasthenia
gravis (MG) is a rare, debilitating, autoimmune disease that
affects the neuromuscular junction—the place where nerve cells and
muscle cells communicate. In MG, the body attacks special receptors
on nerve cells that are sensitive to an important chemical called
acetylcholine (ACh). ACh is an important chemical because it helps
send messages from nerves across the neuromuscular junction to
muscles, where these special receptors interpret the message. These
special receptors are called acetylcholine receptors (AChR). There
is a second chemical that is needed for proper nerve-muscle
communication and it is called muscle-specific kinase (MuSK). MuSK
also helps nerve-muscle communication across the neuromuscular
junction. Unlike AChR-MG, in which the body attacks the receptor,
in patients with MuSK-MG, their bodies attack cells that produce
the MuSK chemical. It’s estimated that up to 8% of all people with
MG (or about 3,000-4,800 total patients in the U.S.) have the
MuSK-MG type.
About Catalyst PharmaceuticalsCatalyst
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, spinal muscular atrophy (SMA) type 3 and infantile spasms.
Firdapse® (amifampridine phosphate) has received Breakthrough
Therapy Designation from the U.S. Food and Drug Administration
(FDA) for the treatment of LEMS 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
refractory infantile spasms. 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 StatementsThis 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
the results of the MSK-002 study will be successful, (ii) the
timing of the enrollment of the MSK-002 study, (iii) whether the
NDA recently submitted for Firdapse for LEMS will be accepted by
the FDA, and the timing of any such acceptance, (iv) 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,
(v) whether, if an NDA for Firdapse is accepted for filing, such
NDA will be given a priority review by the FDA, (vi) whether
Firdapse will ever be approved for commercialization, (vii) whether
Catalyst will be the first company to receive an approval for
amifampridine (3,4-DAP), giving it 5-year marketing exclusivity for
its product, and (viii) 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 ContactBrian KorbSolebury Trout (646)
378-2923bkorb@troutgroup.com
Company ContactPatrick J. McEnanyCatalyst
PharmaceuticalsChief Executive Officer(305)
420-3200pmcenany@catalystpharma.com
Media ContactDavid SchullRusso Partners(212)
845-4271david.schull@russopartnersllc.com
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