Six-month priority review granted for CUTX-101
copper histidinate with PDUFA target action date set for
June 30, 2025
CUTX-101 has potential to be the first
FDA-approved treatment for Menkes disease, a rare and fatal
pediatric disease
SOLANA
BEACH, Calif. and AHMEDABAD, India and MIAMI, Jan. 6, 2025
/PRNewswire/ -- Sentynl Therapeutics, Inc. ("Sentynl"), a
U.S.-based biopharmaceutical company wholly-owned by Zydus
Lifesciences, Ltd. ("Zydus Group"), and Fortress Biotech,
Inc. (Nasdaq: FBIO) ("Fortress") announced that the U.S. Food
and Drug Administration (FDA) has accepted for filing and Priority
review Sentynl's New Drug Application (NDA) for CUTX-101, the
product candidate for the treatment of Menkes disease. Menkes
disease is a rare X-linked recessive pediatric disease caused by
gene mutations of the copper transporter ATP7A. Recent
estimates suggest a prevalence of 1 in 34,810 to as high as 1
in 8,664 live male births.
Sentynl's NDA is supported by positive topline clinical efficacy
results for CUTX-101, demonstrating statistically significant
improvement in overall survival for Menkes disease subjects who
received early treatment with CUTX-101, with a nearly 80% reduction
in the risk of death compared to an untreated historical control
cohort. Median overall survival was 177.1 months for CUTX-101 early
treatment cohort compared to 16.1 months for the untreated
historical control cohort.
"The NDA acceptance of CUTX-101 marks an important step towards
our vision to transform lives and meaningfully impact patients,
caregivers, and the rare disease community at large," said
Punit Patel, President and CEO,
Zydus Americas. "We remain committed to providing access to
path-breaking discoveries that can address unmet needs globally, as
evident in the potential impact of CUTX-101 on Menkes disease
patients and their families."
"Menkes disease presents a difficult journey for patients
and their caregivers, as ATP7A mutations impact the
transport of copper to a range of organs and systems, such as the
lungs, brain and heart. With no known cure or current FDA-approved
treatments, death typically occurs between 2 to 3 years of age,"
said Matt Heck, President &
Chief Executive Officer of Sentynl. "We are eager for the FDA to
review our application for CUTX-101, which has the potential to be
the first FDA-approved therapy for this devastating condition."
CUTX-101 was granted Breakthrough Therapy, Fast Track, Rare
Pediatric Disease and Orphan Drug Designations by the FDA and
Orphan Designation by the European Medicines Agency (EMA). Under
the Prescription Drug User Fee Act (PDUFA), the FDA set a six-month
period with a target action date of June 30,
2025.
In December 2023, Sentynl assumed
full responsibility for the development and commercialization of
CUTX-101 from Cyprium Therapeutics ("Cyprium"), a Fortress
subsidiary company focused on the development of novel therapies
for the treatment of Menkes disease and related copper metabolism
disorders.
"We congratulate Sentynl on the NDA acceptance for filing and
review of CUTX-101, a program that Cyprium advanced and proudly
supports," said Lindsay A.
Rosenwald, M.D., Chairman, President and Chief Executive
Officer of Fortress and Chairman of Cyprium. "The drug has a
demonstrated safety and efficacy profile for the treatment of
Menkes disease and, if approved, CUTX-101 will fill a significant
unmet need for children suffering from this rare, fatal pediatric
disease."
About CUTX-101 (Copper Histidinate)
CUTX-101 is an
investigational drug currently under NDA review with the U.S. FDA
to treat patients with Menkes disease. CUTX-101 is a subcutaneous
injectable formulation of copper histidinate manufactured under
current good manufacturing practice ("cGMP") that is intended to
improve tolerability due to its physiological pH. In a Phase 1/2
clinical trial conducted by Stephen G.
Kaler, M.D., M.P.H., at the National Institutes of Health
("NIH"), early treatment of patients with Menkes disease with
CUTX-101 led to an improvement in neurodevelopmental outcomes and
survival. Cyprium previously reported positive topline clinical
efficacy results for CUTX-101, demonstrating statistically
significant improvement in overall survival for Menkes disease
subjects who received early treatment (ET) with CUTX-101, compared
to an untreated historical control cohort, with a nearly 80%
reduction in the risk of death. Median overall survival (OS) was
177.1 months for CUTX-101 ET cohort
compared to 16.1 months for the untreated historical control
cohort. CUTX-101 has been granted FDA Breakthrough Therapy, Fast
Track, Rare Pediatric Disease and FDA Orphan Drug Designations.
Additionally, the European Medicines Agency granted Orphan
Designation for CUTX-101. An expanded access protocol for
patients with Menkes disease is ongoing at multiple U.S. medical
centers.
About Menkes Disease
Menkes disease is a rare
X-linked recessive pediatric disease caused by gene mutations of
the copper transporter ATP7A. The minimum birth prevalence for
Menkes disease is believed to be 1 in 34,810 live male births, and
potentially as high as 1 in 8,664 live male births, based on recent
genome-based ascertainment (Kaler SG, Ferreira CR, Yam LS.
Estimated birth prevalence of Menkes disease and ATP7A-related
disorders based on the Genome Aggregation Database (gnomAD).
Molecular Genetics and Metabolism Reports 2020 June 5;24:100602). The condition is characterized
by distinctive clinical features, including sparse and depigmented
hair ("kinky hair"), connective tissue problems, and severe
neurological symptoms such as seizures, hypotonia, failure to
thrive, and neurodevelopmental delays. Mortality is high in
untreated Menkes disease, with many patients dying between 2-3
years of age. Milder versions of ATP7A mutations are
associated with other conditions, including Occipital Horn Syndrome
and ATP7A-related Distal Motor Neuropathy. Currently, there
is no FDA-approved treatment for Menkes disease and its
variants.
About Sentynl Therapeutics
Sentynl Therapeutics, Inc.
("Sentynl") is a U.S.-based biopharmaceutical company focused on
bringing innovative therapies to patients suffering from rare
diseases. The company was acquired by the Zydus Group in 2017.
Sentynl's experienced management team has previously built multiple
successful pharmaceutical companies. With a focus on
commercialization, Sentynl looks to source effective and
well-differentiated products across a broad spectrum of therapeutic
areas to address unmet needs. Sentynl is committed to the highest
ethical standards and compliance with all applicable laws,
regulations and industry guidelines. For more information, visit
https://sentynl.com.
About Zydus Group
Zydus Lifesciences Ltd. with an overarching purpose of empowering
people with freedom to live healthier and more fulfilled lives, is
an innovative, global lifesciences company that discovers,
develops, manufactures, and markets a broad range of healthcare
therapies. The group employs over 27,000 people worldwide,
including 1,400 scientists engaged in R & D, and is driven by
its mission to unlock new possibilities in lifesciences through
quality healthcare solutions that impact lives. The group aspires
to transform lives through path-breaking discoveries. For more
information, visit https://www.zyduslife.com/zyduslife.
About Fortress Biotech
Fortress Biotech, Inc.
("Fortress") is an innovative biopharmaceutical company focused on
acquiring and advancing assets to enhance long-term value for
shareholders through product revenue, equity holdings and dividend
and royalty revenue. The company has seven marketed prescription
pharmaceutical products and over 20 programs in development at
Fortress, at its majority-owned and majority-controlled partners
and subsidiaries and at partners and subsidiaries it founded and in
which it holds significant minority ownership positions. Such
product candidates span six large-market areas, including oncology,
rare diseases and gene therapy, which allow it to create value for
shareholders. Fortress advances its diversified pipeline through a
streamlined operating structure that fosters efficient drug
development. The Fortress model is focused on leveraging its
significant biopharmaceutical industry expertise and network to
further expand the company's portfolio of product opportunities.
Fortress has established partnerships with some of the world's
leading academic research institutions and biopharmaceutical
companies to maximize each opportunity to its full potential,
including AstraZeneca, City of Hope, Fred Hutchinson Cancer Center,
Nationwide Children's Hospital and Sentynl. For more information,
visit www.fortressbiotech.com.
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