Catalyst Pharmaceuticals, Inc. (“Catalyst” or “Company”)
(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 that the
peer-reviewed journal Neurology has published Santhera
Pharmaceutical's ("Santhera") study titled “Efficacy and Safety of
Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy”
presenting the findings from Santhera's VISION-DMD study conducted
by Santhera [1]. Catalyst holds the exclusive rights to
commercialize AGAMREE® (vamorolone) in North America.
Santhera reports that the VISION-DMD study was a
randomized, double-blind, placebo-controlled, and
prednisone-controlled clinical trial of 2 doses of vamorolone in
participants with Duchenne muscular dystrophy (“DMD”) in the ages
four years to younger than seven years at baseline that was
conducted at 33 sites in 11 countries from 2018 to 2021. Based on
the published study, Santhera has reported that the results of the
VISION-DMD study support the long-term efficacy and safety profile
of vamorolone and conclude that vamorolone was generally well
tolerated, consistent with the 24-week study findings, as published
previously in JAMA Neurology [2]. As cited in Santhera's recent
press release, the Neurology publication of their study states:
“Vamorolone is a
dissociative corticosteroid that selectively binds to the
glucocorticoid receptor and has shown similar efficacy and reduced
safety concerns in comparison with prednisone in Duchenne muscular
dystrophy (DMD)[2]. This study [VISION-DMD] was conducted to
determine the efficacy and safety of vamorolone over 48 weeks and
to study crossover participants (prednisone to vamorolone; placebo
to vamorolone).
A total of 121
participants with DMD were randomized. Vamorolone at a dose of 6
mg/kg/d showed maintenance of improvement for all motor outcomes to
week 48 (e.g., for primary outcome, time to stand from supine
[TTSTAND] velocity, week 24 least squares mean [LSM] [SE] 0.052
[0.0130] rises/s vs week 48 LSM [SE] 0.0446 [0.0138]). After 48
weeks, vamorolone at a dose of 2 mg/kg/d showed similar
improvements as 6 mg/kg/d for North Star Ambulatory Assessment
(NSAA) (vamorolone 6 mg/kg/d– vamorolone 2 mg/kg/d LSM [SE] 0.49
[1.14]; 95% CI −1.80 to 2.78, p = 0.67), but less improvement for
other motor outcomes. The placebo to vamorolone 6 mg/kg/d group
showed rapid improvements after 20 weeks of treatment approaching
benefit seen with 48-week 6 mg/kg/d of vamorolone treatment for
TTSTAND, time to run/walk 10 m, and NSAA. There was significant
improvement in linear growth after crossover in the prednisone to
vamorolone 6 mg/kg/d group, and rapid reversal of
prednisone-induced decline in bone turnover biomarkers in both
crossover groups. There was an increase in BMI after 24 weeks of
treatment that then stabilized for both vamorolone groups.
Improvements of motor
outcomes seen with 6 mg/kg/d of vamorolone at 24 weeks of treatment
were maintained to 48 weeks of treatment. Vamorolone at a dose of 6
mg/kg/d showed better maintenance of effect compared with
vamorolone at a dose of 2 mg/kg/d for most (3/5) motor outcomes.
Bone morbidities of prednisone (stunting of growth and declines in
serum bone biomarkers) were reversed when treatment transitioned to
vamorolone.”
AGAMREE® (vamorolone) oral suspension 40 mg/ml
has been approved for commercialization in the United States for
the treatment of DMD.
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 steroid, 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
2024;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. German.
About Duchenne muscular dystrophy
(DMD)DMD, the most common form of muscular dystrophy, is a
rare and life-threatening neuromuscular disorder characterized by
progressive muscle dysfunction, ultimately leading to loss of
ambulation, respiratory failure, and fatality. Current standard
treatment for DMD involves corticosteroids, which often come with
significant side effects. It is estimated that between 11,000 and
13,000 patients in the U.S. are affected by DMD, with
approximately 70% of patients currently receiving concomitant
corticosteroid treatment.
About Catalyst
PharmaceuticalsWith 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 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 StatementsThis
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 AGAMREE will be
found to have a better safety profile than other corticosteroids,
(ii) whether Catalyst's commercial launch of AGAMREE in the United
States will be successful, and (iii) 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.
Investor Contact
Mary Coleman, Catalyst Pharmaceuticals, Inc.
(305) 420-3200
mcoleman@catalystpharma.com
Media Contact
David Schull, Russo Partners
(858) 717-2310
david.schull@russopartnersllc.com
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