APT-F2
- Generic Name
- APT-F2
BioMarin Acquires Inozyme Pharma for $270 Million, Adding Late-Stage Enzyme Therapy for Rare Genetic Disorder
• BioMarin Pharmaceutical has entered into a definitive agreement to acquire Inozyme Pharma for $270 million, strengthening its enzyme therapy portfolio with the addition of INZ-701.
• INZ-701 is a Phase 3 enzyme replacement therapy being developed for ENPP1 Deficiency, a rare genetic condition affecting blood vessels, soft tissues, and bones with no currently approved treatments.
• The first pivotal data readout for INZ-701 in children is expected in early 2026, with potential regulatory approval and market launch in 2027.
RIBOMIC's Umedaptanib Pegol Shows Promise in Phase IIa Achondroplasia Trial
• RIBOMIC's umedaptanib pegol demonstrated a positive impact on annualized growth rate in children with achondroplasia in a Phase IIa trial.
• In the low-dose cohort, two out of five patients showed an increase in height growth rate of +4.6 cm and +3.3 cm/year compared to baseline.
• The observed growth rate surpasses the average of +1.7 cm/year reported for Voxzogo, a currently approved achondroplasia treatment.
• RIBOMIC is proceeding with a high-dose cohort study, with results expected in September 2025, and long-term evaluation of the low-dose cohort.
Umedaptanib Pegol Shows Promise in Phase IIa Trial for Achondroplasia
• RIBOMIC's umedaptanib pegol demonstrates a positive impact on the growth rate of pediatric patients with achondroplasia in a Phase IIa trial.
• Two out of five patients in the low-dose cohort experienced a significant increase in height growth rate compared to pre-treatment observations.
• The observed growth rate with umedaptanib pegol surpassed the average growth rate reported for Voxzogo, a currently approved achondroplasia treatment.
• A high-dose cohort study is underway, with results expected in September 2025, and no safety concerns have been reported in the ongoing trials.
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