Fortress Biotech Announces FDA Approves Its ZYCUBO To Treat Menkes Disease In Pediatric Patients

Fortress Biotech, Inc. (Nasdaq: FBIO) ("Fortress") and its majority-owned subsidiary, Cyprium Therapeutics, Inc. ("Cyprium"), today announced that the U.S. Food and Drug Administration ("FDA") has approved ZYCUBO® (copper histidinate, formerly known as CUTX-101) for the treatment of Menkes disease in pediatric patients.

In December 2023, Sentynl Therapeutics, Inc. ("Sentynl"), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences Limited ("Zydus Group"), assumed full responsibility for the development and commercialization of CUTX-101 from Cyprium. A Rare Pediatric Disease Priority Review Voucher (PRV) was issued in connection with FDA approval and, pursuant to the transaction with Sentynl, will be transferred to Cyprium. Cyprium is also eligible to receive tiered royalties on net sales of ZYCUBO and up to $129 million in aggregate development and sales milestones from Sentynl.

Menkes disease is a rare X-linked recessive pediatric disease caused by mutations of the copper transporter ATP7A encoded by the ATP7A gene. Patients with Menkes disease are born with the inability to absorb dietary copper and subsequently have impaired copper transport across the blood-brain barrier, and, until now, there has been no approved treatment in the United States. ZYCUBO® is a subcutaneous injectable formulation of copper histidinate that restores copper homeostasis and maintains copper levels in patients with Menkes disease.