-- FDA grants Special Protocol
Assessment agreement for Phase 3 MuSK-MG trial --
Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq:CPRX), a
biopharmaceutical company focused on developing and commercializing
innovative therapies for people with rare debilitating
neuromuscular and neurological diseases, today provided an update
on the status of its Firdapse® (amifampridine phosphate)
development activities.
Patrick J. McEnany, Chief Executive Officer of
Catalyst, stated: “We are pleased to report that we have reached an
agreement with the FDA under a Special Protocol Assessment (SPA)
for the trial design, clinical endpoints and statistical analysis
approach for our Phase 3 clinical trial evaluating Firdapse for the
treatment of patients with MuSK antibody positive Myasthenia Gravis
(MuSK-MG). We appreciate the FDA’s engagement and guidance during
the SPA process, and we look forward to dosing the first patients
in this trial in early 2018.”
Mr. McEnany continued, "Over the last few weeks,
our management team has carefully examined the timelines for our
development plans for Firdapse, including all necessary steps that
are required for us to submit a complete new drug application (NDA)
for Firdapse for the treatment of Lambert-Eaton Myasthenic Syndrome
(LEMS). Those efforts have led us to slightly refine our
timeline for the planned submission to the FDA of our NDA, which we
now expect will be submitted in the first quarter of 2018.
Previously we had reported that we expected to submit our NDA by
the end of this year.”
Dr. Steven Miller, Catalyst’s Chief Operating
Officer and Chief Scientific Officer added, “The additional short
term preclinical studies that the FDA required us to complete
before we could submit another NDA for amifampridine phosphate are
abuse liability studies that the FDA and Controlled Substance Staff
require for the evaluation of the potential effect of any new
chemical entities on behaviors relating to abuse liability and
dependence/withdrawal effects. Our discussions with the FDA
regarding the protocols for these studies took longer than
initially expected and, as a result, we now expect to have the
final report in December.”
Second Phase 3 clinical trial (LMS-003)
evaluating Firdapse for the treatment of LEMS
Catalyst expects to complete enrollment shortly
and to announce top-line results from its confirmatory Phase 3
study (LMS-003) in the fourth quarter of this year.
Preclinical Abuse Liability
Studies
Catalyst is conducting three preclinical abuse
liability studies required by the FDA’s guidance for “Assessment of
Abuse Potential of Drugs” that was finalized in January 2017.
Catalyst has completed the studies for Self-Administration and
Physical Dependence, and has obtained top-line results indicating
that amifampridine phosphate does not exhibit abuse liability in
these two assessment models. The third study for Drug
Discrimination is currently ongoing and is expected to be completed
in the fourth quarter of this year. Catalyst remains
confident that all three studies will indicate that amifampridine
phosphate does not have any abuse liability and that no further
preclinical or clinical studies for abuse liability will be
required, although there can be no assurance.
Submission of NDA for Firdapse for
LEMS
Catalyst intends to request a confirmatory
pre-NDA meeting with the FDA to discuss the proposed NDA filing
package. Since the briefing package to the FDA, to be submitted
prior to this meeting, needs to include results from the LMS-003
trial and findings from the three abuse liability studies, Catalyst
believes that any pre-NDA meeting that may be granted will not take
place until December 2017 or January 2018. As a result, Catalyst
has concluded that the NDA submission for Firdapse for LEMS is
likely to be submitted (assuming the results of its studies are
successful) in the first quarter of 2018 rather than before the end
of 2017 as previously reported.
Registration trial evaluating Firdapse
for the treatment of MuSK-MG
Catalyst has just recently received agreement
from the FDA on a special protocol assessment (SPA) for a study
evaluating the safety and efficacy of amifampridine phosphate
treatment in patients with MuSK antibody positive myasthenia gravis
(MuSK-MG). This is a particularly severe form of myasthenia
gravis that affects about 3,000 to 4,800 patients in the US, for
which there are no approved effective therapies and is therefore an
unmet medical need. The protocol that the FDA has reviewed is
for a multi-site, international (US and Italy), double-blind,
placebo-controlled, clinical trial that 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.
Catalyst anticipates that enrollment in this trial will commence in
the first quarter of 2018, and that it will take about 12 months to
complete the enrollment for the trial.
Phase 3 clinical trial (CMS-001)
evaluating Firdapse for the treatment of Congenital Myasthenic
Syndromes (CMS)
Catalyst recently reported that it expects to
report top-line data from its ongoing clinical trial evaluating
Firdapse for the treatment of CMS in the first half of 2018, and
Catalyst continues to believe that this timeline remains accurate.
Catalyst also hopes to include in any NDA that it submits for
Firdapse for LEMS those limited types of CMS that are generally
considered mechanistically similar to LEMS. Catalyst intends
to confirm with the FDA at any pre-NDA meeting that it may be
granted, that any such inclusion will not slow down the FDA's
review of a submitted NDA for Firdapse for LEMS.
Development of commercial plan for
Firdapse
As previously reported, Catalyst intends to
recommence its efforts to refine and begin implementation of its
commercialization plan for Firdapse before the end of this
year.
* * * * * * * * * * * * * * * * *
Detailed information about each of Catalyst's
ongoing studies and trials can be found in the Company's filings
with the Securities and Exchange Commission, including its Annual
Report on Form 10-K for 2016 and its Quarterly Report on Form 10-Q
for the period ended June 30, 2017.
About Special Protocol
Assessment
A Special Protocol Assessment (SPA) is a process
that allows the sponsor of a clinical trial to submit their
proposed trial design (clinical trial protocol and also the
statistical analysis plan, if desired) to the FDA for review and
concurrence prior to initiating the clinical trial. The FDA
responds to these submissions within 45 days either with agreement,
or with disagreement, and may provide written comments in either
case. Should the FDA disagree, a new submission with revised
documents would be necessary in order to reach agreement, and
meetings with the FDA prior to any SPA submission may be necessary
in some cases. As stated in the FDA's "Guidance for Industry:
Special Protocol Assessment, ...a special protocol assessment
documents our agreement that the design and planned analysis of a
study can adequately address objectives in support of a regulatory
submission. However, final determinations for marketing application
approval are made after a complete review of a marketing
application and are based on the entire data in the
application." Therefore, the possibility exists that the
Agency may determine that a clinical trial is inadequate at a later
date in spite of having agreed to it in the past.
About Catalyst
Pharmaceuticals
Catalyst Pharmaceuticals is a biopharmaceutical
company focused on developing and commercializing innovative
therapies for people with rare debilitating neuromuscular and
neurological diseases, including Lambert-Eaton myasthenic syndrome
(LEMS), congenital myasthenic syndromes (CMS), MuSK antibody
positive myasthenia gravis, and infantile spasms. Firdapse 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, and possibly refractory 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 trial evaluating Firdapse for the treatment of LEMS and
whether that trial will be successful, what clinical trials and
studies will be required before Catalyst can submit an NDA for
Firdapse for the treatment of CMS and whether any such required
clinical trials and studies 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 Catalyst can successfully
complete a registration trial evaluating Firdapse for the treatment
of MuSK-MG that is acceptable to the FDA, whether any such future
trial evaluating Firdapse for the treatment of MuSK-MG will be
successful, whether Catalyst has sufficient funding to conduct such
a trial, whether Firdapse will ever be approved for
commercialization and successfully commercialized, whether Catalyst
will be the first company to receive approval for amifampridine
(3,4-DAP), giving it 5-year marketing exclusivity for its product,
and those other factors described in Catalyst's Annual Report on
Form 10-K for the fiscal year 2016 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 Contact
David Schull
Russo Partners
(212) 845-4271
david.schull@russopartnersllc.com
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