Catalyst Pharmaceuticals, Inc. (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
initiation of a company-sponsored, adequate and well-controlled,
proof-of-concept clinical trial evaluating safety, tolerability and
efficacy of Firdapse® (amifampridine phosphate) as a symptomatic
treatment for patients with Spinal Muscular Atrophy (SMA) Type 3.
The study will be conducted by a team of
researchers led by Lorenzo Maggi, MD, and Giovanni Baranello, MD,
of the Fondazione Istituto Neurologico Carlo Besta in Milan, Italy,
a major referral center for SMA patients. The study is designed as
a randomized (1:1), double-blind, 2-period, 2-treatment, crossover,
outpatient proof-of-concept study to evaluate the safety,
tolerability and potential efficacy of amifampridine in ambulatory
patients diagnosed with SMA Type 3. The study is planned to include
approximately 12 patients, and Catalyst anticipates reporting
top-line results from the study in the first half of 2019.
Patrick J. McEnany, Chief Executive Officer of
Catalyst, said, "We are very pleased that Drs. Maggi and Baranello
and their team at the Fondazione Istituto Neurologico Carlo Besta
have agreed to conduct this study for Catalyst of Firdapse in
patients diagnosed with Spinal Muscular Atrophy Type 3. If
the results from this trial support the safety and efficacy of
Firdapse as a treatment for ambulatory patients with SMA Type 3, we
intend to submit an application for orphan drug designation and to
pursue a clinical program required to obtain approval of Firdapse
for this indication."
Studies in SMA animal models and SMA patients
have shown that the neuromuscular junction, or NMJ, displays
significant structural and functional defects that precede overt
disease symptoms, suggesting that impaired NMJ function may
contribute to SMA pathogenesis and symptoms. Defects in the
NMJ appear to precede degeneration of motor neurons suggesting that
abnormal formation and/or lack of maintenance of this structure may
be a key contributor to the disease pathogenesis. These
defects have been observed at both the pre- and postsynaptic
components of the NMJ, which likely contribute to the failure to
maintain the NMJ and muscle innervation in mouse models of
SMA. Finally, NMJ dysfunction has been observed in SMA
patients, mainly in the Type 3 subgroup, which contributes to the
symptoms associated with the disease, and also is the primary
reason for Catalyst's choice to limit this new trial to SMA Type 3
patients.
Amifampridine enhances neuromuscular junction
transmission and therefore may help to preserve the NMJ and motor
neurons in these patients. Catalyst hopes that Amifampridine
treatment will provide symptomatic relief to SMA Type 3 patients,
and the assessment of this symptomatic relief, should it occur, is
the primary outcome variable of this new trial. In addition,
as amifampridine enhances NMJ transmission, it is also hoped that
the enhanced NMJ function will slow the progression of the nerve
degeneration pathology of SMA. However, due to the duration
of the treatment periods in this new trial, it will not be possible
in this trial to assess the long-term progression of SMA in these
patients.
About Spinal Muscular Atrophy (SMA) Type
3
Spinal Muscular Atrophy (SMA) is a spectrum of
genetic disorders of the Survival Motor Neuron (SMN) protein that
affects the function of the neuromuscular junction. The
pathogenesis may, in part, progress due to the lack of retrograde
signaling from dysfunctional neuromuscular junctions leading to
nerve damage and ultimately nerve cell death. As a spectrum
of genetic disorders of the SMN protein, the condition varies in
severity and the disease has been classified into Types (SMA Types
1 through 4) based primarily on clinical symptoms of the
disease. The overall incidence of SMA is believed to be 1 in
6,000 to 10,000 live births, with over half of the cases diagnosed
as SMA Type 1. Due to the poor prognosis of SMA Type 1
patients, the actual prevalence is lower, since well over half of
the SMA patients are Type 1 and have a very short life span.
Due to the heterogeneity of the disease and the variations in life
expectancy, prevalence is difficult to determine and not well
defined for the different types of SMA. Current estimates
place the prevalence of SMA Types 2 and 3 at about 1.5 per 100,000
people, with the majority of these being SMA Type 3 due to the
longer life span of SMA Type 3 patients. Based on currently
available data, Catalyst estimates the prevalence of SMA Type 3 in
the United States to be about 2,500 to 3,500 patients.
SMA Type 3 (sometimes called Kugelberg-Welander
disease) includes clinically heterogeneous patients. They
typically reach all major motor milestones in childhood and
independent walking by adulthood. However, during infancy
they typically have proximal muscular weakness. Some might
need wheelchair assistance in childhood, whereas others might
continue to walk and live productive adult lives with minor
muscular weakness. Patients who lose ambulation often develop
scoliosis and other medical problems related to poor mobility and
muscle tone, such as obesity and osteoporosis. Two subgroups
of severity have been suggested based on the probability of being
able to walk by age 10 and on the subsequent probability of losing
the ability to walk by age 40. Significant differences in
losing the ability to walk have been observed in relation to those
with an onset of weakness before (SMA 3a) and after (SMA 3b) age
3.
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, 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 (i) whether Firdapse will provide
symptomatic relief to SMA Type 3 patients or slow the progression
of the nerve degeneration pathology of SMA, (ii) whether Catalyst’s
estimate of patients in the U.S. with SMA Type 3 will be determined
to be accurate, (iii) whether Catalyst can obtain orphan drug
designation for this indication, (iv) whether this proof-of-concept
study evaluating Firdapse for the treatment of SMA Type 3 will be
successful, (v) even if this trial is successful, what other future
trials and studies will be required for Catalyst to seek approval
to commercialize Firdapse for the treatment of SMA Type 3, (vi)
whether Firdapse will ever be approved for commercialization for
the treatment of any disease, and (vii) 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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