Cyprium Therapeutics, a Fortress Biotech Subsidiary Company, Completes Asset Transfer of CUTX-101 Copper Histidinate Product Candidate for Treatment of Menkes Disease, to Sentynl Therapeutics, a Wholly-owned Subsidiary of Zydus Lifesciences Ltd.
December 06 2023 - 8:30AM
Cyprium Therapeutics, Inc. (“Cyprium”), a Fortress Biotech, Inc.
(Nasdaq: FBIO) (“Fortress”) subsidiary company, today announced
that it executed an Assignment and Assumption Agreement (the
“Agreement”) with Sentynl Therapeutics, Inc. (“Sentynl”), a
U.S.-based specialty pharmaceutical company wholly owned by the
Zydus Lifesciences Ltd., in which, Cyprium completed the transfer
of its proprietary rights and assigned its FDA documents pertaining
to CUTX-101, Copper Histidinate product candidate for the treatment
of Menkes disease, to Sentynl.
Cyprium received $4.5 million at closing and
remains eligible to receive up to $129 million in aggregate
development and sales milestones under the Agreement, in addition
to royalties on net sales of CUTX-101 as follows: (i) 3% of annual
net sales up to $75 million; (ii) 8.75% of annual net sales between
$75 million and $100 million; and (iii) 12.5% of annual net sales
in excess of $100 million. Cyprium will retain 100% ownership over
any FDA priority review voucher that may be issued at NDA approval
for CUTX-101.
“We are pleased to complete the CUTX-101 asset
transfer with Sentynl. Cyprium has made progress with the CUTX-101
rolling NDA submission, and we expect that Sentynl will complete
the filing in 2024. The transfer of the CUTX-101 program to Sentynl
will result in a reduction in Cyprium’s development-related spend,
while potentially expediting commercialization upon FDA approval.
The drug has demonstrated a compelling safety and efficacy profile
for the treatment of Menkes disease and, if approved, CUTX-101 will
fill a significant unmet need for children suffering from this
rare, fatal pediatric disease,” said Fortress’ Chairman, President
and Chief Executive Officer and Cyprium’s Chairman, Lindsay A.
Rosenwald, M.D.
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. 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 Drug
Designation for CUTX-101. A Cyprium-sponsored expanded access
protocol for patients with Menkes disease is ongoing at multiple
U.S. medical centers and will be administered by Sentynl subsequent
to 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 Cyprium
TherapeuticsCyprium 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 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 2023, Cyprium
completed the transfer of its proprietary rights and assigned its
FDA documents pertaining to CUTX-101 Copper Histidinate product
candidate for the treatment of Menkes disease, to Sentynl
Therapeutics, Inc. Cyprium and NICHD also previously 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; AAV-ATP7A gene therapy is currently in
pre-clinical development. 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
Miami. For more information, visit www.cypriumtx.com.
About Fortress BiotechFortress
Biotech, Inc. (“Fortress”) is an innovative biopharmaceutical
company focused on efficiently acquiring, developing and
commercializing or monetizing promising therapeutic products and
product candidates. The company has eight marketed prescription
pharmaceutical products and over 25 programs in development at
Fortress, at its majority-owned and majority-controlled partners
and subsidiaries and at partners and subsidiaries 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 focused on leveraging its
significant biopharmaceutical industry expertise and network 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 AstraZeneca, City of Hope, Fred Hutchinson Cancer Center,
St. Jude Children’s Research Hospital, Nationwide Children’s
Hospital and Sentynl. For more information, visit
www.fortressbiotech.com.
Forward-Looking StatementsThis
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. As used
below and throughout this press release, the words “we”, “us” and
“our” may refer to Fortress individually or together with one or
more partner companies, as dictated by context. Such statements
include, but are not limited to, any statements relating to our
growth strategy and product development programs, ability to
generate shareholder value, ability of our products to receive
necessary approvals, including FDA approval, ability of our
products and therapies to help patients 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;
financing and strategic agreements and relationships; Sentynl’s
ability to successfully develop and obtain regulatory approval for
CUTX-101; our need for substantial additional funds and uncertainty
relating to financings; our ability to identify, acquire, close and
integrate product candidates successfully and on a timely basis;
our ability to attract, integrate and retain key personnel; the
early stage of products under development; 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; the ability to secure and maintain
third-party manufacturing, marketing and distribution of our and
our partner companies’ products and product candidates; 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 may be required by law, and we claim the
protection of the safe harbor for forward-looking statements
contained in the Private Securities Litigation Reform Act of 1995.
The information contained herein is intended to be reviewed in its
totality, and any stipulations, conditions or provisos that apply
to a given piece of information in one part of this press release
should be read as applying mutatis mutandis to every other instance
of such information appearing herein.
Company Contacts:Jaclyn Jaffe and Nicole
McCloskey
Cyprium Therapeutics, Inc. ir@cypriumtx.com
Fortress Biotech, Inc.(781) 652-4500ir@fortressbiotech.com
Media Relations Contact:Tony Plohoros6
Degrees(908) 591-2839tplohoros@6degreespr.com
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