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 SAGE Open Medicine of the results of an
investigator-sponsored Phase IIb clinical trial (MSK-001)
evaluating Firdapse® (amifampridine phosphate) for the treatment of
MuSK antibody positive Myasthenia Gravis (MuSK-MG).
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 MSK-001 trial
was a randomized, double-blind, placebo-controlled, double
crossover design. The co-primary endpoints were statistically met
(QMG score: p=0.0003 and MG-Activities of Daily Living Profile
Score: p=0.0006) as well as the secondary endpoints. The study
provided evidence that amifampridine phosphate was safe and
effective in treating MuSK-MG patients. The study was conducted
under the supervision of Silvia Bonanno, M.D. and her team at the
Istituto Neurologico Carlo Besta in Milan, Italy.
These data are more fully described in the
manuscript entitled, “Amifampridine phosphate in the treatment of
muscle-specific kinase myasthenia gravis: a phase IIb, randomized,
double-blind, placebo-controlled, double crossover study”, which
was recently published online in SAGE Open Medicine. The article
can be accessed at:
https://journals.sagepub.com/doi/pdf/10.1177/2050312118819013 .
“The results of the pilot study are very
encouraging, such that our multi-center pivotal Phase 3 trial
evaluating Firdapse for the treatment of MuSK-MG is currently
underway. Our pivotal trial is being conducted under an FDA Special
Protocol Assessment (SPA),” said Gary Ingenito, M.D., Ph.D., Chief
Medical Officer of Catalyst Pharmaceuticals. “If our Phase 3 trial
is successful, we hope that Catalyst will be able to offer
physicians and patients alternatives in the treatment of
MuSK-MG.”
About MuSK antibody positive Myasthenia
Gravis (MG)
About 15% of MG patients test negative for the
acetylcholine receptor antibody. These patients have seronegative
(SN) MG. Approximately 40-50% of these patients with SNMG (equating
to an estimate of approximately 4,500 patients in the United
States) test positive for the anti-MuSK antibody. MuSK is a protein
that is required for the maintenance of the neuromuscular junction
and patients with the anti-MuSK antibody are identified as having
MuSK-MG. MuSK-MG is a clinically distinguishable, more severe form
of MG. The disease is characterized by a predominance in females, a
prevalent involvement of cranial and bulbar muscles, high incidence
of respiratory crises and a resistance to treatment. Although many
patients with MuSK-MG are presently treated with anticholinesterase
inhibitors or immunosuppressants, such patients do not generally
respond adequately to these treatments.
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 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 Lambert-Eaton Myasthenic Syndrome
(LEMS) was recently approved by the U.S. Food & Drug
Administration ("FDA"), and Firdapse is expected to be commercially
available in the United States early in the first quarter of 2019.
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.
About Firdapse®
(amifampridine)
Firdapse® (amifampridine) 10 mg tablets is an
oral, nonspecific, voltage-dependent, potassium (K+) channel
blocker that causes depolarization of the presynaptic membrane and
slows or inhibits repolarization. This action results in the
opening of slow voltage-dependent calcium (Ca2+) channels, allowing
for a subsequent influx of Ca2+. In turn, it induces the exocytosis
of synaptic vesicles containing Acetylcholine (ACh) to release more
ACh into the synaptic cleft, enhancing neuromuscular transmission,
and providing for improved muscle function. Firdapse is approved in
the U.S. and the European Union for use by patients with LEMS.
Important Safety
Information
CONTRAINDICATIONS
FIRDAPSE is contraindicated in patients
with:
- A history of seizures
- Hypersensitivity to amifampridine phosphate or another
aminopyridine
WARNINGS AND PRECAUTIONS
Seizures:
FIRDAPSE can cause seizures. Consider discontinuation or dose
reduction of FIRDAPSE in patients who have a seizure while on
treatment. FIRDAPSE is contraindicated in patients with a history
of seizures.
Hypersensitivity: If a
hypersensitivity reaction such as anaphylaxis occurs, FIRDAPSE
should be discontinued and appropriate therapy initiated.
ADVERSE REACTIONS
The most common (> 10%) adverse reactions
are: paresthesia, upper respiratory tract infection, abdominal
pain, nausea, diarrhea, headache, elevated liver enzymes, back
pain, hypertension, and muscle spasms.
To report SUSPECTED ADVERSE REACTIONS,
contact Catalyst Pharmaceuticals at 1-844-347-3277
(1‑844-FIRDAPSE) or FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.
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 Catalyst’s ongoing clinical trials
evaluating Firdapse for the treatment of CMS, MuSK-MG and SMA type
3 will be successful; (iv) whether Firdapse will ever be approved
for the treatment of CMS, MuSK-MG, SMA type 3, or any other
disease, and (v) 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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