FBIO Climbs In Pre-Market- Will CUTX-101 Become The First FDA-Approved Menkes Therapy?

Core Viewpoint - Fortress Biotech Inc. (FBIO) shares have increased over 3% to $4.40 in premarket trading due to its subsidiary Cyprium Therapeutics nearing a significant FDA decision regarding CUTX-101, a treatment for Menkes disease, with a decision expected by January 14, 2025 [1][4] Group 1: FDA Decision and Product Development - CUTX-101 is a subcutaneous injectable formulation of copper histidinate, developed by Cyprium for Menkes disease, and is awaiting its second review by the FDA [1] - The FDA previously declined to approve CUTX-101 in October 2023 due to concerns about the manufacturing site's cGMP compliance [2] - The development and commercialization responsibilities for CUTX-101 were fully transferred to Sentynl Therapeutics in December 2023, with Cyprium eligible to receive up to $129 million in development and sales milestones, along with royalties on net sales [2] Group 2: Menkes Disease Overview - Menkes disease is caused by mutations in the ATP7A gene, which regulates copper metabolism, leading to severe clinical symptoms including sparse hair, connective tissue issues, neurological symptoms, and high mortality rates in untreated cases [3] - If approved, CUTX-101 will be the first and only FDA-approved treatment for Menkes disease [4]