BMN 401 (INZ-701) - (ENERGY-3)

Aug. 4, 2025: BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported Q2 2025 results and provided multiple pipeline/business catalysts. The company completed its acquisition of Inozyme on July 1, 2025, adding BMN 401 (formerly INZ-701), an enzyme replacement therapy for ENPP1 Deficiency; an initial pivotal data readout from the ENERGY 3 study (children ages 1–12) is anticipated in 1H 2026, with a potential launch in 2027.

Skeletal conditions: Phase 1 healthy volunteer data for BMN 333 (long-acting C-type natriuretic peptide) showed PK AUC levels >3x those observed in other long-acting CNP studies, with no safety signals. BioMarin plans to initiate a registration-enabling pivotal Phase 2/3 trial in pediatric achondroplasia in 1H 2026 (potential 2030 launch if supportive). VOXZOGO: Phase 2 CANOPY data in younger children presented at the May Pediatric Endocrine Society meeting showed significantly reduced tibial bowing vs placebo, sustained with multi-year treatment. For hypochondroplasia, BioMarin expects topline pivotal data in 1H 2026 and plans regulatory submissions in 2H 2026 (potential launch 2027).

Regulatory filing catalyst: PALYNZIQ (PKU) adolescent (12–17) label expansion applications are planned for 2H 2025 in the U.S. and Europe (potential approval in 2026).

Other pipeline: BMN 351 (Duchenne muscular dystrophy) initial data expected by year-end; BMN 349 (AATD-associated liver disease) Phase 2 expected to begin 1H 2026; BMN 390 (PKU) discontinued due to immunogenicity threshold not met.