On Saturday, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) released new Phase 3 PEGASUS study results on Saturday.

The data show that its therapy Palynziq (pegvaliase-pqpz) significantly reduced blood phenylalanine (Phe) levels in adolescents with phenylketonuria (PKU), a rare metabolic disorder.

The trial enrolled 55 patients aged 12–17, with 36 receiving Palynziq and 19 continuing on diet alone. After 72 weeks, 45% of Palynziq patients achieved at least a 50% reduction in Phe from baseline, many reaching guideline-recommended or normal levels.

Safety results were consistent with adult data. An extension phase is ongoing, and BioMarin plans regulatory submissions in the second half of 2025.

Also Read: BioMarin Discontinues Preclinical Drug Candidate, Advances BMN 333 Toward 2030

However, analysts flagged competitive risks from PTC Therapeutics' (NASDAQ:PTCT) newly approved Sephience, a daily oral therapy for PKU patients aged one month and older.

William Blair noted Sephience's broader label, easier administration, faster onset, and favorable safety profile could limit Palynziq's uptake. Analyst Sami Corwin expects only a modest revenue lift for BioMarin from the adolescent indication and warned Sephience could dampen longer-term growth forecasts.

Separately, William Blair cited potential headwinds for BioMarin's Voxzogo franchise, with rival navepegritide facing a U.S. FDA decision by Nov. 30 and a European filing anticipated this quarter.

Price Action: BMRN stock is down 3.46% at $55.77 at the last check on Monday.

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