Fortress Biotech and Cyprium Therapeutics Announce U.S. FDA Acceptance and Priority Review of NDA for CUTX-101 for Treatment of Menkes Disease
Rhea-AI Summary
Fortress Biotech and its subsidiary Cyprium Therapeutics announced that the U.S. FDA has accepted the New Drug Application (NDA) for CUTX-101 (Copper Histidinate) for the treatment of Menkes disease, a rare pediatric condition. The NDA has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2025.
In December 2023, Sentynl Therapeutics assumed responsibility for the development and commercialization of CUTX-101. Cyprium will receive royalties and up to $129 million in aggregate development and sales milestones, and retains ownership of any Priority Review Voucher issued upon NDA approval.
The NDA submission is supported by positive clinical efficacy results, showing a nearly 80% reduction in the risk of death for early-treated Menkes disease patients compared to untreated controls. Median overall survival was 177.1 months for the treated cohort versus 16.1 months for the control group. CUTX-101 has previously received multiple FDA designations, including Breakthrough Therapy and Orphan Drug.
If approved, CUTX-101 could become the first FDA-approved treatment for Menkes disease.
Positive
- FDA acceptance and Priority Review of CUTX-101 NDA.
- PDUFA target action date set for June 30, 2025.
- Potential for up to $129 million in development and sales milestones.
- Nearly 80% reduction in risk of death for early-treated patients.
- Median overall survival of 177.1 months for treated cohort.
Negative
- None.
Insights
The FDA's acceptance of CUTX-101's NDA with Priority Review status represents a major catalyst for Fortress Biotech. The
The clinical data showing a
CUTX-101's potential approval would mark a paradigm shift in Menkes disease treatment, addressing a critical unmet need in this rare X-linked disorder. The survival benefit demonstrated in clinical trials is unprecedented - extending median survival from 16 months to nearly 15 years represents a transformative therapeutic advance.
The partnership with Sentynl/Zydus provides established commercial infrastructure for launch, critical for rare disease medications. The multiple regulatory designations and Priority Review status reflect the treatment's potential impact on this currently untreatable condition. With no FDA-approved therapies available, CUTX-101 could become the standard of care, potentially capturing the entire addressable market.
This development carries strategic significance beyond the immediate therapeutic impact. The partnership structure with Sentynl optimizes commercialization while allowing Fortress/Cyprium to retain significant economic interest through milestones, royalties and crucially, the potential Priority Review Voucher. This follows Fortress's recent success with two FDA approvals in Q4 2024, demonstrating execution capability in bringing products to market.
The orphan drug status in both US and EU markets provides extended market exclusivity, enhancing long-term value proposition. For investors, this represents a near-term catalyst with the June 30, 2025 PDUFA date, backed by compelling clinical data and multiple shots on goal through milestone payments, royalties and PRV monetization potential.
Priority review granted for CUTX-101 with PDUFA target action date set for June 30, 2025
Cyprium is eligible to receive royalties and up to
Cyprium also retains ownership over any Priority Review Voucher that may be issued at NDA approval
MIAMI, Jan. 06, 2025 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) and its majority-owned subsidiary, Cyprium Therapeutics, Inc. (“Cyprium”), today announced the acceptance for review of the New Drug Application (“NDA”) by the U.S. Food and Drug Administration (“FDA”) for CUTX-101 (Copper Histidinate) for the treatment of Menkes disease, a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. The NDA has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2025.
In December 2023, Sentynl Therapeutics, Inc. (“Sentynl”), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (“Zydus Group”), assumed full responsibility for the development and commercialization of CUTX-101 from Cyprium. The NDA submission was completed by Sentynl who will be responsible for commercialization upon approval. As described below, Cyprium is eligible to receive royalties and retains ownership of any Priority Review Voucher that may be issued.
The CUTX-101 NDA submission is supported by positive topline clinical efficacy results for CUTX-101, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment with CUTX-101, with a nearly
“We are thrilled that the NDA for CUTX-101 for the treatment of Menkes disease was accepted for review by the FDA and look forward to working with our partner, Sentynl, and the FDA during its review period. CUTX-101 could be the first FDA-approved treatment for Menkes disease, making this submission an important milestone for our company and for the patients suffering from this rare, often fatal, pediatric disease,” said Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer and Cyprium’s Chairman. “Our late-stage portfolio continues to advance with two recent FDA approvals received in Q4 of 2024 for Emrosi™ and Unloxcyt™ and this acceptance and Priority Review for the CUTX-101 NDA. We look forward to the potential achievement of additional upcoming milestones across our extensive portfolio of commercial and clinical-stage assets.”
“Menkes disease presents a difficult journey for patients and their caregivers, as ATP7A mutations impact the transport of copper to a range of organs and systems, such as the lungs, brain and heart. With no known cure or current FDA-approved treatments, death typically occurs between 2 to 3 years of age,” said Matt Heck, President & Chief Executive Officer of Sentynl. “We are eager for the FDA to review our application for CUTX-101, which has the potential to be the first FDA-approved therapy for this devastating condition.”
If the CUTX-101 NDA is approved, the product may be eligible for a Rare Pediatric Disease Priority Review Voucher (PRV), for which Cyprium would retain ownership, and which can be redeemed for a subsequent marketing application or sold or transferred to a third party. Cyprium is also eligible to receive royalties and up to
About CUTX-101 (Copper Histidinate)
CUTX-101 is an investigational drug currently under NDA review by the FDA to treat patients with Menkes disease. CUTX-101 is a subcutaneous injectable formulation of copper histidinate manufactured under current good manufacturing practice (“cGMP”) that is intended to improve tolerability due to its 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. 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 Designation for CUTX-101. An expanded access protocol for patients with Menkes disease is ongoing at multiple U.S. medical centers.
About Menkes Disease
Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the 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 between 2-3 years of age. 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”) 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. CUTX-101 is an investigational drug currently under NDA review by the FDA to treat patients with Menkes disease. In 2023, Cyprium completed the transfer of its proprietary rights and assigned its FDA documents pertaining to CUTX-101 to Sentynl Therapeutics, Inc. Cyprium and NICHD also have an ongoing 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 and has received FDA Orphan Drug Designation. Cyprium was founded by, and is a majority-owned subsidiary of, Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit www.cypriumtx.com.
About Fortress Biotech
Fortress Biotech, Inc. (“Fortress”) is an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue. The company has seven marketed prescription pharmaceutical products and over 20 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, Nationwide Children’s Hospital and Sentynl. For more information, visit www.fortressbiotech.com.
About Sentynl Therapeutics
Sentynl Therapeutics, Inc. (“Sentynl”) is a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients suffering from rare diseases. The company was acquired by the Zydus Group in 2017. Sentynl’s experienced 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 to address unmet needs. Sentynl is committed to the highest ethical standards and compliance with all applicable laws, regulations and industry guidelines. For more information, visit https://sentynl.com.
About Zydus Group
Zydus Lifesciences Ltd. with an overarching purpose of empowering people with freedom to live healthier and more fulfilled lives, is an innovative, global lifesciences company that discovers, develops, manufactures, and markets a broad range of healthcare therapies. The group employs over 27,000 people worldwide, including 1,400 scientists engaged in R & D, and is driven by its mission to unlock new possibilities in lifesciences through quality healthcare solutions that impact lives. The group aspires to transform lives through path-breaking discoveries. For more information, visit https://www.zyduslife.com/zyduslife.
Forward-Looking Statements
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Company Contact:
Jaclyn Jaffe
Fortress Biotech, Inc.
(781) 652-4500
ir@fortressbiotech.com
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FAQ
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