Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq:
CPRX), today announced the completion of its acquisition from
Santhera Pharmaceuticals Holdings ("Santhera") of an exclusive
license for North America for vamorolone, a potential treatment for
patients suffering with Duchenne Muscular Dystrophy. The license is
for exclusive commercial rights in the U.S., Canada, and Mexico, as
well as the right of first negotiation in Europe and
Japan should Santhera pursue partnership opportunities.
Additionally, Catalyst will hold North American rights for any
future approved indications of vamorolone.
Vamorolone is a promising best-in-class
dissociative anti-inflammatory steroid treatment for Duchenne
Muscular Dystrophy ("DMD"). In clinical studies, vamorolone
demonstrated efficacy with a significant reduction of
steroid-associated side effects and benefits for bone health,
growth, and behavior, offering the potential to address an
important unmet medical need in DMD patients. Vamorolone has
received FDA Orphan Drug and Fast Track designations and has been
granted a PDUFA action date of October 26, 2023.
"With the addition of vamorolone, we have
accomplished another important milestone in the execution of our
portfolio expansion strategy," stated Patrick J. McEnany, Chairman
and CEO of Catalyst. "The license for vamorolone reflects our
strategic focus on opportunities where we can not only apply novel
technology to address critical unmet patient needs, but where we
can leverage Catalyst's existing integrated capabilities and
infrastructure to commercialize the drug efficiently. We believe
that vamorolone, if approved, has the potential to deliver
significant near and long-term value and to be a very meaningful
advancement to the current standard of care paradigm for DMD
patients."
Mr. McEnany continued, "Vamorolone fortifies our
neuromuscular portfolio with an innovative therapy that, in
clinical studies, demonstrated an enhanced safety and tolerability
profile as compared to prednisone. We plan to include vamorolone
for DMD patients in our Catalyst Pathways® specialty pharmacy
program to help ensure that all patients have access to the full
patient benefits of the program. Our planned strategy to facilitate
access to vamorolone underscores our steadfast commitment to
improving the lives of patients suffering from rare neurological
conditions."
Duchenne Muscular Dystrophy, or DMD, the most
common form of muscular dystrophy, is a rare, fatal neuromuscular
disorder characterized by progressive muscular dysfunction, leading
to loss of ambulation, respiratory failure, and death.
Corticosteroids are the current standard of care for treating DMD;
however, this treatment is associated with significant side effect
burdens. The U.S. prevalence for DMD is estimated to be between
11,000 and 13,000 patients. Of patients currently being treated for
DMD, approximately 75% receive concomitant steroid treatment.
Transaction Details
As previously disclosed, Catalyst has made an
all-cash purchase payment of $75 million to acquire the
license. Simultaneously, Catalyst has made a strategic equity
investment into Santhera by acquiring 1,414,688 of Santhera's post
reverse-split ordinary shares (representing approximately 11.26% of
Santhera's outstanding ordinary shares following the transaction)
at an investment price of CHF 9.477 (corresponding to a
mutually agreed volume-weighted average price prior to signing),
with the approximately $15 million USD in equity investment
proceeds to be used by Santhera for Phase IV studies in DMD and
further development of additional indications for
vamorolone. Catalyst will also be obligated under certain
circumstances to make milestone payments and to pay royalties to
Santhera.
Vamorolone Commercial Operational
Plan
- Catalyst expects to launch
vamorolone early in the first quarter of 2024, assuming regulatory
approval on the PDUFA date of October 26, 2023.
- Catalyst anticipates minimal sales
and marketing personnel expansion with fewer than 10 additional
team members required, resulting from the exceptional synergy
within its existing neuromuscular franchise.
- Catalyst plans to incorporate
vamorolone for DMD into its Catalyst Pathways® specialty pharmacy
program to ensure that patients have access to full patient
benefits.
Further details on the vamorolone commercial
operational plan will be discussed during the Company's
second-quarter 2023 financial results conference call, which is
expected to be held in the second week of August. Catalyst also
expects to discuss on a preliminary basis the anticipated
accounting treatment for the transaction on the conference
call.
About Vamorolone
Vamorolone is an investigational drug candidate
with a mode of action based on binding to the same receptor as
glucocorticoids but modifying its downstream activity and as such,
is considered a dissociative anti-inflammatory steroid drug [2-5].
This mechanism has shown the potential to 'dissociate' efficacy
from steroid safety concerns, and therefore vamorolone could emerge
as an alternative to existing corticosteroids, the current standard
of care in children, adolescent, 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 [1]. 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.
Vamorolone has been granted Orphan Drug status
for DMD in the U.S. and Europe and has received
Fast Track and Rare Pediatric Disease designations by
the U.S. FDA and Promising Innovative Medicine (PIM)
status from the UK MHRA for DMD. Vamorolone is an
investigational medicine and is currently not approved for use by
any health authority.
Vamorolone will be eligible for 7 years of orphan drug
exclusivity upon approval and also has issued and pending patents
that could provide protection to 2040.
_______________________________References:
[1] Guglieri M et al (2022). JAMA Neurol.
2022;79(10):1005-1014.doi:10.1001/jamaneurol.2022.2480. Link.[2]
Mah JK et al (2022). JAMA Netw Open. 2022;5(1):e2144178.
doi:10.1001/jamanetworkopen.2021.44178. Link.[3] Guglieri M et
al (2022) JAMA. doi:10.1001/jama.2022.4315[4] Heier CR et al
(2019). Life Science Alliance DOI: 10.26508[5] Liu X et al
(2020). Proc Natl Acad Sci USA 117:24285-24293
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy ("DMD") is a rare
inherited X-chromosome-linked disease, which almost exclusively
affects males. DMD is characterized by muscle inflammation and
damage which are present at birth or shortly thereafter.
Inflammation leads to fibrosis of muscle and is clinically
manifested by progressive muscle degeneration and weakness. Major
milestones in the disease are the loss of ambulation, the loss of
self-feeding, the start of assisted ventilation, and the
development of cardiomyopathy. DMD reduces life expectancy to
before the fourth decade due to respiratory and/or cardiac failure.
Corticosteroids are the current standard of care for the treatment
of DMD.
About Catalyst
PharmaceuticalsWith exceptional patient focus, Catalyst is
committed to developing and commercializing innovative
first-in-class medicines that address rare neurological and
epileptic diseases. Catalyst's U.S. commercial product portfolio
consists of FIRDAPSE® (amifampridine) Tablets 10 mg, approved
for the treatment of Lambert-Eaton myasthenic syndrome ("LEMS") for
adults and children ages six to seventeen. In January 2023,
Catalyst acquired the U.S. commercial rights of 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.
For more information, 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.
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 the NDA for vamorolone
will be approved by the PDUFA date, or at all, (ii) whether, if the
NDA for vamorolone is approved by the FDA, the product can be
successfully commercialized by Catalyst in the licensed territory,
(iii) whether if vamorolone is commercialized by Catalyst, the
results of operations will prove to be accretive to Catalyst, (iv)
whether Catalyst and Santhera will successfully develop additional
indications for vamorolone and obtain the approvals required to
commercialize the product in the licensed territory for those
additional indications, (v) whether, if vamorolone is
commercialized by Catalyst, the drug will be successfully
integrated into Catalyst's business activities, and (vi) those
factors described in Catalyst's Annual Report on Form 10-K for the
2022 fiscal year, Catalyst's Quarterly Report on Form 10-Q for the
first quarter of 2023, and Catalyst's other filings with
the 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.
Contact Information
Media Contact
David Schull
Russo Partners
(858) 717-2310
david.schull@russopartnersllc.com
Investor Contact
Mary Coleman
Catalyst Pharmaceuticals, Inc.
(305) 420-3200
mcoleman@catalystpharma.com
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