Cyprium, NICHD establish CRADA to advance
clinical development of Phase 3 candidate CUTX-101 (Copper
Histidinate injection) in Menkes disease, a rare and fatal
pediatric disease
Fortress Biotech, Inc. (Nasdaq:FBIO) today announced the launch of
a new subsidiary, Cyprium Therapeutics, Inc. (Cyprium), to develop
novel therapies for the treatment of Menkes disease and related
copper metabolism disorders. As part of its formation, Cyprium has
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 have
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.
Under the terms of the CRADA, Cyprium is authorized to reference
the Investigational New Drug application currently held by NICHD
and any data generated at NICHD to advance the development of
CUTX-101, including the filing of a New Drug Application and
commercialization in the U.S. and other territories. In addition,
Cyprium will be responsible for the manufacturing of CUTX-101 under
Current Good Manufacturing Practice (“cGMP”) regulations to support
ongoing clinical studies at NICHD. Cyprium will also provide
financial and logistical support for clinical and basic science
research on Menkes disease and other related copper metabolism
disorders at NICHD.
Stephen G. Kaler, M.D., Senior Investigator and Head, Section on
Translational Neuroscience, Molecular Medicine Branch, and former
NICHD Clinical Director, leads the Menkes disease research program
at NICHD and developed the AAV-ATP7A gene therapy technology. Dr.
Kaler will serve as principal investigator of the CUTX-101 clinical
trials and studies of AAV-ATP7A gene therapy.
Lindsay A. Rosenwald, M.D., Fortress Biotech’s Chairman,
President and Chief Executive Officer, said, “Fortress Biotech’s
mission is to develop products that improve the lives of patients
across multiple diseases. We are pleased to launch another
subsidiary company, Cyprium Therapeutics, which we believe will
further broaden our rare disease platform through the development
of novel therapies for the treatment of Menkes disease and other
related disorders.”
CUTX-101 Development Pathway
CUTX-101 is in development to supplement 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 to bypass the oral absorption of
copper, which is impaired in patients with Menkes disease. In Phase
1/2 clinical studies conducted at NICHD, early treatment of Menkes
patients with CUTX-101 led to an improvement in neurodevelopmental
outcomes and survival.
Cyprium and NICHD plan to conduct a retrospective natural
history study to collect data on Menkes disease patients who have
not been treated with copper supplements. Data from this natural
history study may serve as a historical control to demonstrate the
efficacy and safety of CUTX-101. Cyprium expects to request a
meeting with the U.S. Food and Drug Administration (FDA) in 2017 to
determine a regulatory pathway for CUTX-101. CUTX-101 has been
granted orphan drug designation by the FDA.
AAV-ATP7A Gene Therapy Development Pathway
AAV-ATP7A gene therapy, which is currently in preclinical
development at NICHD, has demonstrated the ability to rescue
neurological phenotypes and improve survival when co-administered
with copper injections in a mouse model of Menkes disease. Cyprium
will work with NICHD to determine optimal vector design and expects
to nominate a candidate for clinical development in 2018. AAV-ATP7A
gene therapy has been granted orphan drug designation by the
FDA.
Lung S. Yam, M.D., Ph.D., Named Cyprium Chief Executive
Officer
Fortress also announced the appointment of Lung S. Yam, M.D.,
Ph.D., as Chief Executive Officer of Cyprium. Dr. Yam co-founded
Cyprium as part of his Business Development Consultant role at
Fortress, where he identified multiple opportunities across diverse
therapeutic areas leading to the in-licensing of multiple assets,
including CUTX-101 and the AAV-ATP7A gene therapy program. In
addition to his roles at Cyprium and Fortress, Dr. Yam serves as
Senior Analyst at Opus Point Partners, LLC, an affiliated
life-science focused asset management firm. Prior to joining Opus
Point Partners, Dr. Yam was an equity research associate at Rodman
& Renshaw, LLC. Dr. Yam earned M.D. and Ph.D. degrees from New
York University School of Medicine, and a B.A. in biology from
Johns Hopkins University where he graduated Phi Beta Kappa.
Dr. Yam stated, “We are thrilled to collaborate with Dr. Kaler
and his team at NICHD to develop novel therapies for patients with
Menkes disease and related disorders. Dr. Kaler has dedicated much
of his career to improving treatment options for Menkes patients,
including leading clinical studies of Copper Histidinate injections
and developing an AAV-based gene therapy that has demonstrated
encouraging preclinical results. The Cyprium team looks forward to
continuing this important work with the goal of generating the
first approved therapies for the treatment of Menkes disease.”
Cyprium was founded by Dr. Rosenwald, Dr. Yam and Michael S.
Weiss, Executive Vice Chairman, Strategic Development at Fortress.
Dr. Rosenwald will serve as the Executive Chairman of Cyprium’s
Board of Directors, and Dr. Yam and Mr. Weiss will be additional
members of the Board.
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 of the ATP7A gene. The
condition is characterized by distinctive clinical features,
including sparse and depigmented hair (“kinky hair”), failure to
thrive, connective tissue disorders 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 diseases, including Occipital Horn
Syndrome and ATP7A-related Distal Motor Neuropathy. Currently,
there is no FDA-approved treatment for Menkes disease and its
variants.
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 of its subsidiary companies, also known as Fortress
Companies. Additionally, Fortress recently acquired a controlling
interest in National Holdings Corporation (NASDAQ:NHLD), a
diversified independent brokerage company (together with its
subsidiaries, “NHLD”). 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.
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within the meaning of Section 27A of the Securities Act of 1933 and
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Contacts:
Cyprium Therapeutics, Inc.
Jaclyn Jaffe, Investor Relations
ir@cypriumtx.com
Fortress Biotech, Inc.
Lucy Lu, MD, Executive Vice President & Chief Financial Officer
(781) 652-4500
ir@fortressbiotech.com
Fortress Biotech Media Relations
Laura Bagby
6 Degrees
(312) 448-8098
lbagby@6degreespr.com
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