Date: 2017-03-14
Type of information: Establishment of a new subsidiary in the US
Compound: Cyprium Therapeutics (US - NY)
Company: Fortress Biotech (USA - NY) Cyprium Therapeutics (US - NY)
Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases
Type agreement: establishment of a new subsidiary in the US
Action mechanism:
Disease: Menkes disease and related copper metabolism disorders
Details:
- • On March 14, 2017, Fortress Biotech announced the launch of a new subsidiary, Cyprium Therapeutics, to develop novel therapies for the treatment of Menkes disease and related copper metabolism disorders. As part of its formation, Cyprium has 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 National Institutes of Health (NIH), to advance the clinical development of Phase 3 candidate CUTX-101 (Copper Histidinate injection) for the treatment of Menkes disease. In addition, Cyprium and NICHD have 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 Menkes patients, and to be used in combination with CUTX-101.
- Under the terms of the CRADA, Cyprium is authorized to reference the Investigational New Drug application currently held by NICHD and any data generated at NICHD to advance the development of CUTX-101, including the filing of a New Drug Application and commercialization in the U.S. and other territories. In addition, Cyprium will be responsible for the manufacturing of CUTX-101 under Current Good Manufacturing Practice regulations to support ongoing clinical studies at NICHD. Cyprium will also provide financial and logistical support for clinical and basic science
research on Menkes disease and other related copper metabolism disorders at NICHD.
- Stephen G. Kaler, M.D., Senior Investigator and Head, Section on Translational Neuroscience, Molecular Medicine Branch, and former NICHD Clinical Director, leads the Menkes disease research program at NICHD and developed the AAV-ATP7A gene therapy technology. Dr. Kaler will serve as principal investigator of the CUTX-101 clinical trials and studies of AAV-ATP7A gene therapy.
- CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under cGMP that is intended to improve tolerability due to physiological pH, and to bypass the oral absorption of copper, which is impaired in patients with Menkes disease. In Phase 1/2 clinical studies conducted at NICHD, early treatment of Menkes patients with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival.
Cyprium and NICHD plan to conduct a retrospective natural history study to collect data on Menkes disease patients who have not been treated with copper supplements. Data from this natural history study may serve as a historical control to demonstrate the efficacy and safety of CUTX-101.
Cyprium expects to request a meeting with the FDA in 2017 to determine a regulatory pathway for CUTX-101.
CUTX-101 has been granted orphan drug designation by the FDA.
AAV-ATP7A gene therapy, which is currently in preclinical development at NICHD, has demonstrated the ability to rescue neurological phenotypes and improve survival when co-administered with copper injections in a mouse model of Menkes disease. Cyprium will work with NICHD to determine
optimal vector design and expects to nominate a candidate for clinical development in 2018. AAV-ATP7A gene therapy has been granted orphan drug designation by the FDA.
Lung S. Yam, M.D., Ph.D., Named Cyprium Chief Executive Officer
Fortress also announced the appointment of Lung S. Yam, M.D., Ph.D., as Chief Executive Officer of Cyprium. Dr. Yam co-founded Cyprium as part of his Business Development Consultant role at Fortress, where he identified multiple opportunities across diverse therapeutic areas leading to the in-licensing of multiple assets, including CUTX-101 and the AAV-ATP7A gene therapy program. In addition to his roles at Cyprium and Fortress, Dr. Yam serves as Senior Analyst at Opus Point Partners, an affiliated life-science focused asset management firm. Prior to joining Opus Point
Partners, Dr. Yam was an equity research associate at Rodman & Renshaw, Dr. Yam earned M.D. and Ph.D. degrees from New York
University School of Medicine, and a B.A. in biology from Johns Hopkins University where he graduated Phi Beta Kappa.
Cyprium was founded by Dr. Rosenwald, Dr. Yam and Michael S. Weiss, Executive Vice Chairman, Strategic Development at Fortress. Dr. Rosenwald will serve as the Executive Chairman of Cyprium’s Board of Directors, and Dr. Yam and Mr. Weiss will be additional members of the Board.
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