Sentynl receives worldwide proprietary rights
and U.S. FDA documents
pertaining to CUTX-101 copper
histidinate
CUTX-101 has potential to be the first
FDA-approved treatment for Menkes disease;
rolling
submission of New Drug Application to FDA is ongoing and expected
to be completed in 2024
SOLANA
BEACH, Calif. and AHMEDABAD, India, Dec. 6, 2023
/PRNewswire/ -- Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based
biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd.
(Zydus Group), today announced the execution of an Assignment and
Assumption Agreement with Cyprium Therapeutics, Inc. (Cyprium), a
Fortress Biotech, Inc. (Nasdaq: FBIO) (Fortress) subsidiary
company. Under the agreement, Cyprium completed the transfer of its
worldwide proprietary rights and U.S. FDA documents pertaining to
CUTX-101, the copper histidinate product candidate for the
treatment of Menkes disease, to Sentynl.
Sentynl now assumes full responsibility for the development and
commercialization of CUTX-101. In 2021, Sentynl and Cyprium
reported positive results from a safety and efficacy analysis of
data integrated from two completed pivotal studies in patients with
Menkes disease treated with CUTX-101. A rolling submission of the
CUTX-101 New Drug Application (NDA) to the FDA is ongoing, with
expected completion in 2024.
Speaking on the development, Dr. Sharvil Patel, Managing
Director, Zydus Lifesciences said, "We have been committed to
providing access to path-breaking discoveries that can bridge unmet
healthcare needs, globally. The rights for CUTX-101 is a
significant milestone towards our vision to transform lives and
meaningfully impact patients, healthcare providers and the rare
disease community at large. This novel, breakthrough therapy could
unlock possibilities for the treatment of the life threatening
Menkes disease."
"Menkes disease has a devastating impact on patients and their
caregivers. With no current approved treatments, death usually
occurs between 6 months and 3 years old," said Matt Heck, President & Chief Executive
Officer of Sentynl. "We are committed to advancing CUTX-101, which
has the potential to not only become the first FDA-approved
treatment for Menkes disease, but also positively impact the lives
of the patients and their caregivers affected by this rare, fatal
condition."
"We know first-hand the difficult journey that patients and
families of Menkes disease face," commented Drew and Jamie Eckman, Founders of the Menkes
Foundation and parents of Wesley, who was diagnosed with Menkes
disease at 8.5 months old and passed away three months later. "The
development of CUTX-101 represents incredible progress in the fight
against Menkes disease. We hold a strong amount of hope for a time
when there is an option available to treat this condition,
providing a path forward for caregivers and their children."
About CUTX-101 (Copper Histidinate)
CUTX-101 is an
investigational drug currently under a rolling NDA submission with
FDA 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 and
will be managed by Sentynl after the transfer.
About Menkes Disease
Menkes disease is a rare
X-linked recessive pediatric disease caused by gene mutations of
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 before the age
of two years old. 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 is a
U.S.-based biopharmaceutical company focused on bringing innovative
therapies to patients living with 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
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 has a significant presence in cancer related
therapies and offers a wide range of solutions with cytotoxic,
supportive & targeted drugs. The group employs nearly 25,000
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/
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