ATP7A

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 $129 million in aggregate development and sales milestones Cyprium also retains ownership over any Priority Review Voucher that may be issued at NDA approval Jan. 06, 2025 -- 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. 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. 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. 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. 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. 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. 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. The content above comes from the network. if any infringement, please contact us to modify.

January 6, 2025 Six-month priority review granted for CUTX-101 copper histidinate with PDUFA target action date set for June 30, 2025 CUTX-101 has potential to be the first FDA-approved treatment for Menkes disease, a rare and fatal pediatric disease Solana Beach, CA, Ahmedabad, India, and Miami, FL – January 6, 2025 — Sentynl Therapeutics, Inc. (“Sentynl”), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (“Zydus Group”), and Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) announced that the U.S. Food and Drug Administration (FDA) has accepted for filing and Priority review Sentynl’s New Drug Application (NDA) for CUTX-101, the product candidate for the treatment of Menkes disease. Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. Recent estimates suggest a prevalence of 1 in 34,810 to as high as 1 in 8,664 live male births. Sentynl’s NDA 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. “The NDA acceptance of CUTX-101 marks an important step towards our vision to transform lives and meaningfully impact patients, caregivers, and the rare disease community at large,” said Punit Patel, President and CEO, Zydus Americas. “We remain committed to providing access to path-breaking discoveries that can address unmet needs globally, as evident in the potential impact of CUTX-101 on Menkes disease patients and their families.” “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.” CUTX-101 was granted Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug Designations by the FDA and Orphan Designation by the European Medicines Agency (EMA). Under the Prescription Drug User Fee Act (PDUFA), the FDA set a six-month period with a target action date of June 30, 2025. In December 2023, Sentynl assumed full responsibility for the development and commercialization of CUTX-101 from Cyprium Therapeutics (“Cyprium”), a Fortress subsidiary company focused on the development of novel therapies for the treatment of Menkes disease and related copper metabolism disorders. “We congratulate Sentynl on the NDA acceptance for filing and review of CUTX-101, a program that Cyprium advanced and proudly supports,” said Lindsay A. Rosenwald, M.D., Chairman, President and Chief Executive Officer of Fortress and Chairman of Cyprium. “The drug has a demonstrated 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.” ### About CUTX-101 (Copper Histidinate) CUTX-101 is an investigational drug currently under NDA review with the U.S. 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. 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 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 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. 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. Contacts: Media: Andrea Moody, andrea.moody@fleishman.com Sentynl: Michael Hercz, ir@sentynl.com < Back This website uses cookies for data analytics. This website may also use cookies to enhance your experience with the website. We do not use cookies to sell or share your personal information. By selecting “Accept all,” you agree to our use of cookies. 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Encouraging preclinical studies demonstrate potential to combine AAV-ATP7A gene therapy with CUTX-101, which could be the first FDA-approved treatment for Menkes disease Cyprium Therapeutics, a majority-owned subsidiary of Fortress Biotech, is developing AAV-ATP7A gene therapy to be used in conjunction with CUTX-101 for the treatment of Menkes disease MIAMI, March 04, 2024 (GLOBE NEWSWIRE) -- Cyprium Therapeutics, Inc. (“Cyprium”), a majority-owned subsidiary of Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), today announced that the National Institute of Neurological Disorders and Stroke (“NINDS”) of the National Institutes of Health (“NIH”) has awarded a three-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital and Principal Investigator, Stephen G. Kaler, M.D., M.P.H., to fund completion of preclinical studies, manufacturing and preparation of an Investigational New Drug Application for a first-in-human clinical trial to advance adeno-associated virus (“AAV”)-ATP7A gene therapy, also known as AAV-ATP7A, for the treatment of Menkes disease. Often lethal if untreated, Menkes disease is an X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase. 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 genome-driven ascertainment. In 2017, Cyprium entered into a worldwide, exclusive license agreement with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (“NICHD”) to develop and commercialize AAV-ATP7A gene therapy to deliver working copies of the copper transporter defective in patients with Menkes disease, and to be used in combination with CUTX-101 (Copper Histidinate), which is being developed by Sentynl Therapeutics, Inc. (“Sentynl”). AAV-ATP7A was previously granted Orphan Drug Designation by the U.S. Food and Drug Administration (“FDA”). “By combining CUTX-101 with working copies of ATP7A delivered by AAV, we hope to enhance clinical outcomes in Menkes disease, a fatal rare pediatric disease. This funding allows us to further evaluate the preclinical safety, tolerability and dosing of AAV9-codon-optimized, reduced-size ATP7A, which we propose to administer in a first-in-human clinical trial for Menkes disease. Advances in viral gene therapy and newborn screening make it feasible to envision changing the natural history of this difficult illness, in combination with CUTX-101, potentially the first FDA-approved treatment for Menkes disease,” said Dr. Kaler, a physician-scientist in the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children’s Hospital, Principal Investigator of the preclinical and clinical studies, and professor of Pediatrics and Genetics at The Ohio State University College of Medicine. Preclinical studies have demonstrated a synergistic effect of AAV-ATP7A and CUTX-101 in a reliable mouse model of Menkes disease. In early studies, cerebrospinal fluid (“CSF”)-directed AAV gene therapy rescued 22-53% of mice with a mutation in the human Menkes disease homolog (mottled-brindled) when combined with CSF or subcutaneous copper. In addition, mutant mice treated with CSF-directed AAV9- reduced-size ATP7A in combination with CUTX-101 showed higher brain copper levels, normalized brain neurochemicals, improvement of brain mitochondrial abnormalities, and near-normal growth and neurobehavioral outcomes. More recently, based on successful intravenous AAV9 gene therapy in mice and humans with spinal muscular atrophy at the Center for Gene Therapy, Nationwide Children’s Hospital, the Kaler Laboratory evaluated intravenous administration of AAV9-codon-optimized, reduced-size ATP7A combined with subcutaneous administration of CUTX-101 in Menkes disease mouse models. This regimen led to 95% long-term rescue of mottled-brindled mutant mice (n=19/20), using an AAV9 dose of 2.6 x 1013 vg/kg body weight, the most successful long-term rescue of this mouse model to date. Lindsay A. Rosenwald, M.D., Executive Chairman, President and Chief Executive Officer of Fortress, stated, “We are honored to have the NIH’s support, and Dr. Kaler’s and Nationwide Children’s leadership, to progress the development of AAV-ATP7A gene therapy, to help provide a more effective therapeutic option to patients with Menkes disease. As we continue the research and development of AAV-ATP7A in combination with CUTX-101, there is great potential for this next generation approach to enhance the treatment of Menkes disease, which currently has no FDA-approved treatment. We look forward to continuing to advance AAV-ATP7A with Dr. Kaler and supporting Sentynl as they finalize and complete the NDA submission for CUTX-101.” The content and research reported in this release are solely the responsibility of the authors and do not necessarily represent the official views of the NIH. About AAV-ATP7A Gene Therapy AAV-ATP7A is an adeno-associated virus (AAV)-based gene therapy in preclinical development for the treatment of Menkes Disease. AAV-ATP7A is being developed to deliver working copies of the copper transporter that is defective in Menkes patients and to be used in combination with CUTX-101 (Copper Histidinate). AAV-ATP7A was developed in part under an Exclusive License Agreement with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and in conjunction with the Research Institute at Nationwide Children’s Hospital and Principal Investigator, Dr. Stephen G. Kaler. About CUTX-101 (Copper Histidinate) CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate in clinical development by Sentynl Therapeutics to treat patients with Menkes Disease. 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. An expanded access protocol, administered by Sentynl Therapeutics, for patients with Menkes disease is ongoing at multiple U.S. medical centers. Sentynl acquired the CUTX-101 program via asset purchase in December 2023 and will owe Cyprium milestone and royalty payments in connection with the development and commercialization of the product. 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-driven 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 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. 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 is currently in pre-clinical development and has been granted FDA Orphan Drug Designation. Cyprium was founded by Fortress Biotech, Inc. (Nasdaq: FBIO) and is based in Miami. For more information, visit . About Fortress Biotech Fortress 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 . Forward-Looking Statements This 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. 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