Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a
commercial-stage biopharmaceutical company focused on in-licensing,
developing, and commercializing novel medicines for patients living
with rare diseases, today announced that Catalyst has entered into
a definitive agreement with Santhera Pharmaceuticals Holding
(“Santhera”) (SIX: SANN) under which Catalyst will enter into an
exclusive North America license, manufacturing and supply agreement
for Santhera’s investigational product candidate, vamorolone, a
dissociative steroid currently under FDA review for the treatment
of Duchenne Muscular Dystrophy (“DMD”). The licensing agreement
delivers a promising complementary product to Catalyst's growing
rare neuroscience product portfolio and enables further expansion
into other rare neurological diseases that address significant
unmet medical needs. In addition to the North American rights,
which consist of the United States, Canada, and Mexico territories,
the transaction provides Catalyst the right of first negotiation
for vamorolone in Europe and Japan should Santhera pursue
partnership opportunities.
Vamorolone was studied for the indication of
DMD, a rare neuromuscular disorder. Santhera filed a New Drug
Application (“NDA”) for vamorolone, supported by clinical data from
the positive pivotal Phase 2b VISION-DMD study, which met the
primary endpoint with statistical significance over placebo. The
vamorolone NDA has been accepted and is currently under review by
the U.S. Food and Drug Administration ("FDA") for the treatment of
DMD. FDA assigned a Prescription Drug User Fee Act ("PDUFA") target
action date of October 26, 2023.
“We are extremely pleased to be entering into a
partnership with Santhera on the vamorolone program and the
prospect of delivering a potential therapy with a desirable profile
for DMD patients," said Patrick J. McEnany, Chairman and CEO of
Catalyst. “We believe this transaction provides us with a highly
complementary drug candidate with outstanding clinical data that
will accelerate our offerings of effective and innovative treatment
advances to people living with rare neurological disorders. If
approved, vamorolone would represent an important inflection point
in our long-term growth strategy, further leveraging the company as
a growing leader in rare neurological diseases. Upon closing the
transaction, we will be well-positioned to capitalize on the
synergies of our expanded and exceptional capabilities in
preparation for the launch of vamorolone, which, assuming approval
of the NDA by the PDUFA date, is anticipated to occur early in
2024. We look forward to collaborating with our partners who share
our commitment to providing innovative new treatment advances to
patients living with rare neurological disorders."
Mr. McEnany continued, “Vamorolone has the
potential to be a differentiated treatment for DMD with a desirable
profile in comparison to the current standard of care options,
addressing an important unmet medical need for Duchenne patients
starting at an early age. The FDA has granted vamorolone Orphan
Drug, Fast Track, and Rare Pediatric Disease designations. If
approved, vamorolone would further broaden our commercial portfolio
with a novel asset enabling us to further build upon our growth
momentum with differentiated medicines that treat rare neurological
and neuromuscular disorders.”
Transaction Terms
Under the terms of the agreement, Catalyst will make a $75 million
upfront payment to Santhera and a concurrent strategic equity
investment of $15 million into Santhera at an investment price of
CHF 0.9477, corresponding to a mutually agreed volume-weighted
average price prior to signing, with the equity investment proceeds
to be used by Santhera for Phase IV studies in DMD and further
development of additional indications. In addition, Santhera may
receive future regulatory and commercial milestone payments tied to
FDA approval and calendar year sales of vamorolone, as well as
commercial royalties. Furthermore, Catalyst and Santhera will enter
into a Joint Steering Committee to oversee the development of
vamorolone for additional indications beyond DMD.
The agreement is structured as an all-cash
transaction with no financing contingencies. The transaction is
expected to be completed in the third quarter of 2023, subject to
customary closing conditions and regulatory clearances in the
United States and does not impact Catalyst’s 2023 revenue guidance.
The proposed transaction is subject to agreed terms and other
closing conditions, including third-party approvals. Catalyst
anticipates that upon closing of this transaction, it will be in a
position to provide greater details regarding its plans and
prospects for vamorolone, as well as the accounting treatment for
the transaction.
Moelis & Company LLC is acting as the
exclusive financial advisor to Catalyst, and Akerman LLP and Cooley
LLP are acting as legal advisors to Catalyst.
About VamoroloneVamorolone 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 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 and
adolescent subjects 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 in 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.
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
DystrophyDMD is a rare inherited X-chromosome-linked
disease, which almost exclusively affects males. DMD is
characterized by inflammation which is 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 SantheraSanthera
Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical
company focused on the development and commercialization of
innovative medicines for rare neuromuscular and pulmonary diseases
with high unmet medical need. The company has an exclusive license
for all indications worldwide to vamorolone, a dissociative steroid
with novel mode of action, which was investigated in a pivotal
study in patients with DMD as an alternative to standard
corticosteroids. For vamorolone in the treatment of DMD, Santhera
has a new drug application (“NDA”) under review by the U.S. FDA, a
marketing authorization application (“MAA”) under review by the
European Medicines Agency (“EMA”) and an MAA submitted to the UK
Medicines and Healthcare products Regulatory Agency (“MHRA”). The
clinical stage pipeline also includes lonodelestat to treat cystic
fibrosis (“CF”) and other neutrophilic pulmonary diseases. Santhera
out-licensed rights to its first approved product, Raxone®
(idebenone), outside North America and France for the treatment of
Leber's hereditary optic neuropathy ("LHON”) to Chiesi Group.
For further information, please visit
www.santhera.com.
About Catalyst
Pharmaceuticals With exceptional patient focus,
Catalyst is committed to developing and commercializing innovative
first-in-class medicines that address rare neurological and
epileptic 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 and up. 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. For additional information about the Company, please
visit 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 more information
about Catalyst Pharmaceuticals, Inc., visit the Company's
website at www.catalystpharma.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 proposed agreement to license vamorolone and
make the equity investment will be completed, (ii) whether the NDA
for vamorolone will be approved by the PDUFA date, or at all, (iii)
whether the licensing agreement, if it is completed and the NDA for
vamorolone is approved by the FDA, can be successfully
commercialized by Catalyst in the territory, (iv) whether if the
vamorolone is commercialized by Catalyst, the results will prove to
be accretive to Catalyst, (v) whether Catalyst and Santhera will
successfully develop additional indications for vamorolone and
obtain the ability to commercialize the product for those
additional indications, (vi) whether Catalyst will, if vamorolone
is commercialized by Catalyst, be successfully integrated into
Catalyst's business activities, and (vii) 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:
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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