🛑 BioMarin Ends PKU Program BMN 390 After Immunogenicity Shortfall — Eyes Next Moves BioMarin has quietly pulled the plug on BMN 390, a preclinical PKU (phenylketonuria) therapy once positioned to improve on its existing Palynziq® treatment. Despite its promise—including the use of the novel POEGMA polymer to reduce immune reactions and improve injection tolerability—BMN 390 did not meet immunogenicity benchmarks and will not move forward. Key points: ✅ BMN 390 was intended as a successor to Palynziq with better tolerability and lower viscosity. ❌ Discontinued following a strategic R&D review after failure to meet target thresholds. 🔄 BioMarin confirmed that employees were redeployed within the organization. 🧪 The company remains committed to its PKU portfolio, including the adolescent expansion of Palynziq and gene therapy efforts like BMN 307, alongside R&D in genetic diseases including achondroplasia and alpha-1 antitrypsin deficiency. This move reinforces BioMarin’s disciplined pipeline curation—prioritizing only assets with clear clinical and differentiating potential. The dependable PKU franchise, led by Palynziq, continues to grow (Q2 2025 sales up ~20% YoY at $106M), with a label expansion filing for adolescents expected by year-end. reddit.com 14biomarin.com 14fiercebiotech.com 14fiercebiotech.com 1reddit.com 1investors.biomarin.com #BioMarin #PKU #BMN390 #Palynziq #DiscontinuedProgram #RareDisease #GeneticTherapy #PipelineStrategy