FBIOP Fortress Biotech Inc

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 80% reduction in the risk of death compared to an untreated historical control cohort. Median overall survival was 177.1 months for CUTX-101 early treatment cohort compared to 16.1 months for the untreated historical control cohort. CUTX-101 was previously granted FDA Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug Designations. Additionally, the European Medicines Agency previously granted Orphan Drug Designation for CUTX-101.

“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 $129 million in aggregate development and sales milestones from Sentynl.

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 DiseaseMenkes 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 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. 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 TherapeuticsSentynl 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 GroupZydus 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.

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Company Contact:Jaclyn Jaffe Fortress Biotech, Inc.(781) 652-4500ir@fortressbiotech.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com