Fortress Biotech, Inc. (NASDAQ:FBIO) (“Fortress”), a
biopharmaceutical company dedicated to acquiring, developing and
commercializing novel pharmaceutical and biotechnology products,
today announced that the U.S. Food and Drug Administration (FDA)
has granted Fast Track Designation to Cyprium Therapeutics’
(“Cyprium”) Copper Histidinate, also referred to as CUTX-101, for
patients diagnosed with classic Menkes disease who have not
demonstrated significant clinical progression.
As a subsidiary of Fortress, Cyprium is developing CUTX-101 for
Menkes disease under a Cooperative Research and Development
Agreement (CRADA) with the Eunice Kennedy Shriver National
Institute of Child Health and Human Development (NICHD), part of
the National Institutes of Health (NIH). The CRADA was established
in March 2017. Stephen G. Kaler, M.D., Senior Investigator and
Head, Section on Translational Neuroscience and former NICHD
Clinical Director, leads the Menkes disease research program at
NICHD and serves as principal investigator of the CUTX-101 Phase 3
clinical trial.
“We are pleased that the FDA granted Fast Track Designation for
Copper Histidinate based on the promising clinical data observed to
date,” stated Lung S. Yam, M.D., Ph.D., President and Chief
Executive Officer of Cyprium. “Menkes disease is a rare and fatal
pediatric disease, and patients with classic Menkes disease
currently have no approved therapeutic options. This designation
signifies the FDA’s recognition of the significant unmet medical
need that Copper Histidinate has potential to address. We
appreciate our ongoing discussions with the FDA and look forward to
working closely with the agency and Dr. Kaler as we advance the
development of Copper Histidinate, which is currently being
evaluated in a Phase 3 study.”
The FDA's Fast Track program facilitates the development of
drugs intended to treat serious conditions and that have the
potential to address unmet medical needs. A drug program with Fast
Track status is afforded greater access to the FDA for the purpose
of expediting the drug's development, review and potential
approval. In addition, the Fast Track program allows for
eligibility for Accelerated Approval and Priority Review, if
relevant criteria are met, as well as for Rolling Review, which
means that a drug company can submit completed sections of its New
Drug Application (NDA) for review by the FDA, rather than waiting
until every section of the NDA is completed before the application
is submitted for review.
About CUTX-101 (Copper Histidinate)
CUTX-101 is in development to supplement blood and brain copper
levels in patients with Menkes disease and related disorders.
CUTX-101 is a subcutaneous injectable formulation of Copper
Histidinate manufactured under cGMP that is intended to improve
tolerability due to physiological pH and bypass the oral absorption
of copper, which is impaired in patients with Menkes disease. In a
Phase 1/2 clinical study conducted by Dr. Kaler at NIH, early
treatment of Menkes patients with CUTX-101 led to an improvement in
neurodevelopmental outcomes and survival. A Phase 3 study of
CUTX-101 in Menkes disease is ongoing.
Cyprium and NICHD are also collecting natural history data on
classic Menkes disease patients who were not treated with copper
supplements. Data from this natural history study will serve as a
historical control cohort to further demonstrate the efficacy and
safety of CUTX-101.
CUTX-101 previously has been granted orphan drug designation by
the U.S. Food and Drug Administration.
About Menkes Disease and Related Copper Metabolism
Disorders
Menkes disease is a rare X-linked pediatric disease caused by
gene mutations of copper transporter ATP7A, which affects
approximately one in 100,000 newborns per year. Biochemically,
Menkes patients may have low levels of copper in their blood and
brain, as well as abnormal levels of catecholamines, but definitive
diagnosis is typically made by sequencing the ATP7A gene. The
condition is characterized by distinctive clinical features,
including sparse and depigmented hair (“kinky hair”), failure to
thrive, connective tissue problems and severe neurological symptoms
such as seizures. Mortality is high, with many patients dying
before the age of three. 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 Cyprium Therapeutics
Cyprium Therapeutics, Inc. (Cyprium), a Fortress Biotech
company, is focused on the development of novel therapies for the
treatment of Menkes disease and related copper metabolism
disorders. In March 2017, Cyprium entered into a Cooperative
Research and Development Agreement (CRADA) with the Eunice Kennedy
Shriver National Institute of Child Health and Human Development
(NICHD), part of the National Institutes of Health (NIH), to
advance the clinical development of Phase 3 candidate CUTX-101
(Copper Histidinate injection) for the treatment of Menkes
disease. In addition, Cyprium and NICHD entered into a
worldwide, exclusive license agreement to develop and commercialize
adeno-associated virus (AAV)-based gene therapy, called AAV-ATP7A,
to deliver working copies of the copper transporter that is
defective in Menkes patients, and to be used in combination with
CUTX-101. AAV-ATP7A gene therapy was also previously granted orphan
drug designation by the FDA. Cyprium is a majority‐owned
subsidiary of Fortress Biotech (NASDAQ:FBIO) and is based in New
York City.
About Fortress Biotech
Fortress Biotech, Inc. (“Fortress”) is a biopharmaceutical
company dedicated to acquiring, developing and commercializing
novel pharmaceutical and biotechnology products. Fortress develops
and commercializes products both within Fortress and through
certain subsidiary companies, also known as Fortress Companies. In
addition to its internal development programs, Fortress leverages
its biopharmaceutical business expertise and drug development
capabilities and provides funding and management services to help
the Fortress Companies achieve their goals. Fortress and the
Fortress Companies may seek licensings, acquisitions, partnerships,
joint ventures and/or public and private financings to accelerate
and provide additional funding to support their research and
development programs. For more information, visit
www.fortressbiotech.com.
Forward-Looking Statements
This press release may contain “forward-looking statements”
within the meaning of Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934, as amended.
Such statements include, but are not limited to, any statements
relating to our growth strategy and product development programs
and any other statements that are not historical facts.
Forward-looking statements are based on management’s current
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to differ materially from those currently anticipated include:
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perform under and maintain financing and strategic agreements and
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completing clinical trials; our dependence on third-party
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personnel; the early stage of products under development; our need
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described in our SEC filings. We expressly disclaim any obligation
or undertaking to release publicly any updates or revisions to any
forward-looking statements contained herein to reflect any change
in our expectations or any changes in events, conditions or
circumstances on which any such statement is based, except as
required by law.
Contacts:
Cyprium Therapeutics, Inc. Lung Yam, M.D., Ph.D., President and
CEOir@cypriumtx.com
Fortress Biotech, Inc.Jaclyn Jaffe, Investor Relations(781)
652-4500ir@fortressbiotech.com
Fortress Biotech Media RelationsTony Plohoros6 Degrees(908)
940-0135tplohoros@6degreespr.com
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