Catalyst Pharmaceuticals to Present at MDA Clinical & Scientific Conference Details of Registry for Study of Long-Term Safety and Quality of Life in Duchenne Muscular Dystrophy Patients Treated with AGAMREE
February 27 2024 - 8:03AM
Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a
commercial-stage, patient-centric biopharmaceutical company focused
on in-licensing, developing, and commercializing novel high-quality
medicines for patients living with rare and difficult to treat
diseases, today announced an upcoming poster about the
establishment of a registry to study long-term safety and quality
of life (QoL) in patients with Duchenne muscular dystrophy (DMD)
who are treated with AGAMREE® (vamorolone) at the upcoming 2024 MDA
Clinical & Scientific Conference taking place March 3-6 in
Orlando.
The poster will detail the establishment of a
multicenter, observational, prospective, longitudinal registry at
approximately 25 U.S. sites. Investigators will assess the progress
of 250 male patients who are at least 2 years old at week 12 and
then every 12 months for up to five years. The first interim
analysis is expected in May 2025.
Poster Information:
Title: An Imminent Registry to
Study Long-term Safety and Quality of Life in Patients with
Duchenne Muscular Dystrophy treated with Vamorolone
(AGAMREE®)Poster Number: M174
"This registry will provide real-world data
regarding long-term use of the novel corticosteroid, generating
additional data to the current clinical results and underscoring
the potential of AGAMREE® to reshape the DMD treatment paradigm
with a focus on improving the quality of life for patients," said
Gary Ingenito, MD, Ph.D., Chief Medical and Regulatory Officer of
Catalyst. "We encourage physicians treating patients with DMD to
consider enrolling eligible patients in this registry."
Learn more about #MDAConference here:
https://www.mdaconference.org/
About
AGAMREE® (vamorolone)AGAMREE's
unique mode of action is based on differential effects on
glucocorticoid and mineralocorticoid receptors and modifying
further downstream activity. As such, it is considered a novel
corticosteroid with dissociative properties in maintaining efficacy
that we hope has the potential to demonstrate comparable efficacy
to steroids, with the potential for a better-tolerated side effect
profile. This mechanism of action may allow vamorolone to emerge as
an effective alternative to the current standard of care
corticosteroids in children, adolescents, and adult patients with
DMD. In the pivotal VISION-DMD study, vamorolone met the primary
endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002)
at 24 weeks of treatment and showed a good safety and tolerability
profile. The most commonly reported adverse events versus placebo
from the VISION-DMD study were cushingoid features, vomiting, and
vitamin D deficiency. Adverse events were generally of mild to
moderate severity.
AGAMREE was granted U.S. FDA approval
on October 26, 2023, and was granted Orphan Drug and Rare
Pediatric Disease designation status for DMD in the U.S.,
making it eligible for seven years of orphan drug exclusivity upon
approval. AGAMREE also has issued and pending patents that could
provide protection until 2040. In Europe, it has received
Promising Innovative Medicine (PIM) status from
the UK MHRA for DMD.
References:[1] Dang UJ et al. (2024) Neurology
024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.[2]
Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014.
doi:10.1001/jamaneurol.2022.2480. Link.[3] Liu X et al.
(2020). Proc Natl Acad Sci USA 117:24285-24293[4] Heier
CR et al (2019). Life Science Alliance DOI: 10.26508[5] Ward et
al., WMS 2022, FP.27 - Poster 71. Link.[6] Hasham et al., MDA 2022
Poster presentation. Link.[7] Applicable drug labeling: Summary of
Product Characteristics (SmPC). English.
About Catalyst Pharmaceuticals
With exceptional patient focus, Catalyst is
committed to developing and commercializing innovative
first-in-class medicines that address rare and difficult-to-treat
diseases. Catalyst's flagship U.S. commercial product is FIRDAPSE®
(amifampridine) Tablets 10 mg, approved for the treatment of
Lambert Eaton myasthenic syndrome ("LEMS") for adults and for
children ages six to seventeen. In January 2023, Catalyst acquired
the U.S. commercial rights to FYCOMPA® (perampanel) CIII, a
prescription medicine approved in people with epilepsy aged four
and older alone or with other medicines to treat partial-onset
seizures with or without secondarily generalized seizures and with
other medicines to treat primary generalized tonic-clonic seizures
for people with epilepsy aged 12 and older. Further, Canada's
national healthcare regulatory agency, Health Canada, has approved
the use of FIRDAPSE for the treatment of adult patients in Canada
with LEMS. Finally, on July 18, 2023, Catalyst acquired an
exclusive license for North America for AGAMREE® (vamorolone) oral
suspension 40 mg/mL, a novel corticosteroid treatment for Duchenne
Muscular Dystrophy. AGAMREE previously received FDA Orphan Drug and
Fast Track designations and was approved by the FDA for
commercialization in the U.S. on October 26, 2023.
For more information about Catalyst
Pharmaceuticals, Inc., please visit the Company's website
at www.catalystpharma.com. For Full Prescribing and Safety
Information for FIRDAPSE®, please visit www.firdapse.com. For
Full Prescribing Information, including Boxed WARNING for FYCOMPA®,
please visit www.fycompa.com. For Full Prescribing Information
for AGAMREE®, please visit www.agamree.com.
Forward-Looking Statements
This press release contains forward-looking
statements, as that term is defined in the Private Securities
Litigation Reform Act of 1995. These include statements regarding
Catalyst's expectations, beliefs, plans or objectives regarding the
intended use of net proceeds therefrom. 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
DMD patient will agree to enroll in the registry, and (ii) those
factors described in Catalyst's Annual Report on Form 10-K for the
fiscal year 2022 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.
Source: Catalyst Pharmaceuticals, Inc.
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