BioMarin’s (BMRN) Phase 3 Trial of BMN 401 for ENPP1 Deficiency Meets One Co-Primary Endpoint

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best low priced stocks to buy for the next 3 years. On May 18, BioMarin’s Phase 3 ENERGY 3 trial of BMN 401 for children with ENPP1 deficiency met only one of its two co-primary endpoints. Treatment led to statistically significant increases in plasma inorganic pyrophosphate/PPi concentration through week 52 compared to conventional therapy.

However, the drug failed to improve Radiographic Global Impression of Change/RGI-C scores, a critical measure of rickets severity. No positive trends were observed in secondary endpoints like growth Z-scores or Rickets Severity Scores, though the treatment was well-tolerated with no new safety signals.

BioMarin’s (BMRN) Phase 3 Trial of BMN 401 for ENPP1 Deficiency Meets One Co-Primary Endpoint

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Management expressed disappointment that the increased PPi levels did not yield clinical benefits for the 27 pediatric participants. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is evaluating the data to determine its next steps and plans to present detailed results at an upcoming medical meeting.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) develops and commercializes therapies for serious and life-threatening medical conditions and rare diseases. The company’s product pipeline includes Valoctocogene roxaparvovec, Vosoritide, and BMN 307.

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