Fortress Biotech (FBIO) investor relations material

Strategic overview and asset transfer

• Focus on developing therapies for Menkes disease and related copper metabolism disorders.

• Completed asset transfer to Sentynl Therapeutics in December 2023; Sentynl now commercializes ZYCUBO.

• Eligible for tiered royalties and up to $129M in milestones from Sentynl.

• Entered agreement to sell FDA Priority Review Voucher for $205M.

Product pipeline and regulatory milestones

• ZYCUBO (Copper Histidinate Injection) NDA approved January 2026.

• ZYCUBO received FDA Breakthrough Therapy, Orphan Drug, Fast Track, and Rare Pediatric Disease designations.

• European Medicines Agency granted Orphan designation for ZYCUBO.

• AAV-ATP7A gene therapy in preclinical stage, with clinical candidate nomination expected within 12 months.

Disease background and unmet need

• Menkes disease is a rare, X-linked recessive pediatric disorder caused by ATP7A mutations.

• Estimated birth prevalence may be as high as 1 in 8,664 live male births, higher than previously recognized.

• Disease leads to severe neurological symptoms, failure to thrive, and premature death if untreated.

• Under-diagnosis is common; newborn screening could increase early identification.