BioMarin Reshapes Pipeline: Focused Strategy for Greater Impact
In a strategic move to streamline its pipeline, BioMarin has decided to offload BMN 293, a preclinical gene therapy aimed at treating hypertrophic cardiomyopathy, a condition causing heart muscles to thicken. Although BMN 293 showed promise in preclinical studies with functional improvements in MYBPC3, the company has chosen to discontinue its development.
This decision follows a broader shift in BioMarin’s approach, as they focus resources on three key assets with the highest potential impact:
1. BMN 351 – A next-generation oligonucleotide for Duchenne muscular dystrophy, with phase 1 data expected by year-end.
2. BMN 349 – An oral small molecule for Alpha-1 antitrypsin deficiency-associated liver disease, entering first-in-human studies in 2024.
3. BMN 333 – A long-acting peptide for multiple growth disorders, slated for early 2025.
Additionally, BioMarin will scale back its haemophilia A gene therapy, Roctavian, to the U.S., Germany, and Italy, optimizing for long-term profitability while reducing costs.
BioMarin’s refined focus underscores its commitment to advancing treatments that offer the greatest potential benefits for patients.