Full Press Release Details
Cyprium Therapeutics, a Fortress Biotech
Partner Company, and Sentynl Therapeutics, a Wholly-owned Subsidiary of the Zydus Group, Sign Development and Asset Purchase Agreement
for Cyprium Therapeutics' Copper Histidinate Product Candidate for Treatment of Menkes Disease
Sentynl acquires CUTX-101, Copper
Histidinate, for $20 million in upfront and regulatory milestone payments through NDA approval, as well as potential sales milestones
Cyprium will retain 100% ownership
over any FDA priority review voucher that may be issued at NDA approval for CUTX-101
Rolling submission of New Drug Application
to the FDA for CUTX-101 on track to begin in the third quarter of 2021 and to be completed during the fourth quarter of 2021
New York, NY & Solana Beach, CA,
February 24, 2021 - Cyprium Therapeutics, Inc. ("Cyprium"), a Fortress Biotech, Inc. (Nasdaq: FBIO) ("Fortress")
partner company, and Sentynl Therapeutics, Inc. ("Sentynl"), a U.S.-based specialty pharmaceutical company owned by
the Zydus Group, today announced the execution of an asset purchase agreement to commit development funding for and acquire Cyprium's
proprietary rights to CUTX-101, its Copper Histidinate product candidate for the treatment of Menkes disease. Under the terms of
the agreement, Sentynl will make an upfront cash payment to Cyprium, provide additional cash payments upon the achievement of certain
regulatory milestones, and pay royalties and commercial milestone payments based on the net sales of CUTX-101. Cyprium will retain
development responsibility of CUTX-101 through approval of the New Drug Application ("NDA") by the U.S. Food and Drug
Administration ("FDA"), and Sentynl will be responsible for commercialization of CUTX-101 as well as progressing newborn
screening activities. Continued development of CUTX-101 will be overseen by a Joint Steering Committee consisting of representatives
from Cyprium and Sentynl.
"We are very pleased to partner with
Sentynl to potentially expedite the development and commercialization of CUTX-101. The drug has demonstrated a compelling safety
and efficacy profile in topline analysis for the treatment of Menkes disease. We look forward to working with Sentynl to begin
the rolling submission of the NDA to the FDA this year. If approved, CUTX-101 will provide a major breakthrough and fill a significant
unmet need for children suffering from this rare, fatal pediatric disease," said Lung S. Yam, M.D., Ph.D., President and
Chief Executive Officer of Cyprium.
Matt Heck, Co-founder, President &
Chief Executive Officer of Sentynl, commented, "Our mission, at the Zydus Group, has been to provide healthcare professionals
with access to innovative treatment solutions that enable them to meet the needs of individual patients. We are excited to collaborate
with Cyprium in order to broaden our pediatric rare disease portfolio and commercialize CUTX-101, a potentially transformative
product, for children with Menkes disease, an unmet healthcare need."
Fortress' Chairman, President and
Chief Executive Officer, Lindsay A. Rosenwald, M.D., added, "This transaction with Sentynl further validates the Fortress
business model of identifying and developing promising treatments for patients, while pursuing partnership opportunities that potentially
maximize shareholder value. We are thrilled with the progress Cyprium has made developing this treatment for Menkes disease and
look forward to working with Sentynl to make CUTX-101 available to patients."
Under the agreement, Cyprium is eligible
to receive up to $20 million in upfront development and regulatory cash milestones through NDA approval, as well as potential sales
milestones. Royalties on CUTX-101 net sales ranging from the mid-single digits up to the mid-twenties are also payable. Cyprium
will retain 100% ownership over any FDA priority review voucher that may be issued at NDA approval for CUTX-101. Torreya Partners
advised Cyprium on this transaction.
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 three 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 CUTX-101 (Copper Histidinate)
CUTX-101 is in clinical development to
treat patients with Menkes disease. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under
current good manufacturing practice ("cGMP") and 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. In August 2020, Cyprium 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 (HC) cohort, with a nearly
80% reduction in the risk of death. A Cyprium-sponsored expanded access protocol for patients with Menkes disease is ongoing at
multiple U.S. medical centers.
About Sentynl Therapeutics
Sentynl Therapeutics is a San Diego-based
commercial-stage specialty pharma company that specializes in acquiring, developing and launching unique products. The
company was acquired by the Zydus Group in 2017. Sentynl's highly experienced and knowledgeable 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. Sentynl is committed to the highest ethical standards and
compliance with all applicable laws, regulations, and industry guidelines.
Zydus Cadila is an innovative, global
pharmaceutical company that discovers, develops, manufactures and markets a broad range of healthcare therapies, including small
molecule drugs, biologic therapeutics and vaccines. The group employs nearly 25,000 people worldwide, including 1,400 scientists
engaged in R & D, and is dedicated to creating healthier communities globally. www.zyduscadila.com.
About Cyprium Therapeutics
Cyprium Therapeutics, Inc. ("Cyprium")
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 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 patients with Menkes disease, and to be used
in combination with CUTX-101. CUTX-101 was granted FDA Breakthrough Therapy, Fast Track and Rare Pediatric Disease Designations,
and both CUTX-101 and AAV-ATP7A have received FDA Orphan Drug Designation previously. Additionally, the European Medicines Agency
previously granted Orphan Drug Designation to CUTX-101. Cyprium was founded by Fortress Biotech, Inc. (Nasdaq: FBIO) and is based
in New York City. For more information, visit www.cypriumtx.com.
About Fortress Biotech
Fortress Biotech, Inc. ("Fortress")
is an innovative biopharmaceutical company that was ranked in Deloitte's 2019 and 2020 Technology Fast 500 , annual
rankings of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy
tech sectors, based on percentages of fiscal year revenue growth over three-year periods. Fortress is focused on acquiring, developing
and commercializing high-potential marketed and development-stage pharmaceutical products and product candidates. The company
has five marketed prescription pharmaceutical products and over 25 programs in development at Fortress, at its majority-owned
and majority-controlled partners and at partners 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 driven by a world-class business development team that is focused on leveraging its significant
biopharmaceutical industry expertise 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 Alexion Pharmaceuticals, Inc., AstraZeneca, City of Hope, Fred Hutchinson Cancer
Research Center, St. Jude Children's Research Hospital and Nationwide Children's Hospital. For more information, visit