Fortress Biotech Announces Publication of Preclinical Data on AAV-ATP7A Gene Therapy Combined with CUTX-101 (Copper Histidina...
September 12 2018 - 8:30AM
Fortress Biotech, Inc. (NASDAQ: FBIO) (“Fortress”), a
biopharmaceutical company dedicated to acquiring, developing and
commercializing novel pharmaceutical and biotechnology products,
today announced the publication of preclinical data on
adeno-associated virus (AAV)-based gene therapy combined with
subcutaneous CUTX-101 (Copper Histidinate) in a mouse model of
Menkes disease, a fatal infantile disorder of human copper
metabolism. In March 2017, Fortress’ subsidiary Cyprium
Therapeutics, Inc. (“Cyprium”) licensed preclinical AAV-ATP7A gene
therapy from the Eunice Kennedy Shriver National Institute of Child
Health and Human Development (NICHD) to develop in combination with
CUTX-101. The data were published online and will be included in
the September 21, 2018 edition of the journal Molecular Therapy:
Methods & Clinical Development.
The preclinical study was conducted by the NICHD laboratory of
Stephen G. Kaler, M.D., and evaluated low-, intermediate- and
high-dose recombinant AAV serotype 9 (rAAV9)-ATP7A delivered to the
cerebrospinal fluid (CSF) in combination with subcutaneous CUTX-101
in a mottled-brindled (mo-br) mouse model that closely mimics the
biochemical and clinical phenotypes of Menkes disease. The rAAV9
construct carried the genetic instructions for a compact,
reduced-size (rs) version of the Menkes copper transporter, ATP7A.
Mutant mice that received high-dose CSF-directed rAAV9-rsATP7A in
combination with CUTX-101 demonstrated improved long-term survival
(53%) compared to mice that did not receive treatment (0%) or were
administered either treatment by itself (0%, 0%). This synergistic
treatment effect represents the most successful rescue to date of
the mo-br mouse model. In addition, mutant mice treated with the
high-dose CSF-directed rAAV9-rsATP7A in combination with CUTX-101
showed higher brain copper levels, normalized brain neurochemicals,
improvement of brain mitochondrial abnormalities, and normal growth
and neurobehavioral outcomes.
“We are highly encouraged by these preclinical data, which
provide new insights on a potentially effective therapeutic
approach for this difficult pediatric disease that currently has no
FDA-approved treatment options,” said Lung S. Yam, M.D., Ph.D.,
President and Chief Executive Officer of Cyprium. “We have made
excellent progress toward new drug approval of subcutaneous
CUTX-101 for Menkes disease based on Dr. Kaler’s clinical trials
and were granted FDA Fast Track Designation earlier this summer. We
look forward to continuing to work with Dr. Kaler and NICHD to also
advance the AAV-ATP7A gene therapy program toward the clinic and
nominating a viral gene therapy candidate for drug
development.”
The study “Cerebrospinal fluid-directed rAAV9-rsATP7A plus
subcutaneous Copper Histidinate advance survival and outcomes in a
Menkes disease mouse model” can be accessed online at:
https://doi.org/10.1016/j.omtm.2018.07.002.
About Menkes Disease and Related Copper Metabolism
Disorders
Menkes disease is a rare X-linked pediatric disease caused by
gene mutations of the copper transporter ATP7A, which affects
approximately one in 100,000 newborns per year. Biochemically,
Menkes patients have low levels of copper in their blood and brain,
as well as abnormal levels of certain neurochemicals. 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 CUTX-101 (Copper Histidinate)
CUTX-101 is in clinical development to supplement blood and
brain copper levels in patients diagnosed with classic Menkes
disease who have not demonstrated significant clinical progression.
CUTX-101 is a subcutaneous injectable formulation of Copper
Histidinate manufactured under cGMP that is intended to improve
tolerability due to physiological pH and to bypass the oral
absorption of copper, which is impaired in patients with Menkes
disease. In a Phase 1/2 clinical trial conducted at the National
Institutes of Health (NIH), early treatment of Menkes patients with
CUTX-101 led to an improvement in neurodevelopmental outcomes and
survival. A Phase 3 trial of CUTX-101 in Menkes disease is ongoing
at the Eunice Kennedy Shriver National Institute of Child Health
and Human Development (NICHD).
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 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. CUTX-101 was
granted U.S. Food and Drug Administration (FDA) Fast Track
Designation, and both CUTX-101 and AAV-ATP7A have received FDA
orphan drug designation previously. 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
expectations and are subject to risks and uncertainties that could
negatively affect our business, operating results, financial
condition and stock price. Factors that could cause actual results
to differ materially from those currently anticipated include:
risks relating to our growth strategy; our ability to obtain,
perform under and maintain financing and strategic agreements and
relationships; risks relating to the results of research and
development activities; uncertainties relating to preclinical and
clinical testing; risks relating to the timing of starting and
completing clinical trials; our dependence on third-party
suppliers; our ability to attract, integrate and retain key
personnel; the early stage of products under development; our need
for substantial additional funds; government regulation; patent and
intellectual property matters; competition; as well as other risks
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 RelationsLaura Bagby 6 Degrees(312)
448-8098lbagby@6degreespr.com
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