BioMarin Reports Encouraging Phase 3 Results for PALYNZIQ® (pegvaliase-pqpz) in Teenagers with Phenylketonuria at the 15th International Congress of Inborn Errors of Metabolism | Intellectia.AI
BioMarin Reports Encouraging Phase 3 Results for PALYNZIQ® (pegvaliase-pqpz) in Teenagers with Phenylketonuria at the 15th International Congress of Inborn Errors of Metabolism
Written by Emily J. Thompson, Senior Investment Analyst
Efficacy of PALYNZIQ: The Phase 3 PEGASUS study showed a 49.7% reduction in mean blood phenylalanine (Phe) levels in adolescents aged 12-17 treated with PALYNZIQ, with nearly half of the participants achieving significant reductions in Phe concentrations compared to diet alone.
Regulatory Submission Plans: BioMarin is on track to submit the PEGASUS study data to global health authorities in the second half of 2025, aiming to expand the approved use of PALYNZIQ to include adolescents with phenylketonuria (PKU).
BMRN
$51.77+Infinity%1D
Analyst Views on BMRN
Wall Street analysts forecast BMRN stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for BMRN is 87.50 USD with a low forecast of 55.00 USD and a high forecast of 120.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
18 Analyst Rating
Wall Street analysts forecast BMRN stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for BMRN is 87.50 USD with a low forecast of 55.00 USD and a high forecast of 120.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
14 Buy
4 Hold
0 Sell
Strong Buy
Current: 52.120
Low
55.00
Averages
87.50
High
120.00
Current: 52.120
Low
55.00
Averages
87.50
High
120.00
Leerink
Outperform -> Market Perform
downgrade
$60
2025-12-03
Reason
Leerink
Price Target
$60
2025-12-03
downgrade
Outperform -> Market Perform
Reason
Leerink downgraded BioMarin to Market Perform from Outperform with a $60 price target.
Leerink
Outperform -> Market Perform
downgrade
$82 -> $60
2025-12-03
Reason
Leerink
Price Target
$82 -> $60
2025-12-03
downgrade
Outperform -> Market Perform
Reason
Leerink downgraded BioMarin to Market Perform from Outperform with a price target of $60, down from $82. BioMarin's new leadership has taken decisive actions, including workforce reductions, ending the Roctavian program, restructuring the pipeline, and acquiring Inozyme, which support more credible long-term financial targets, but competitive pressure in achondroplasia and a sizable pipeline gap make future M&A central to the story, leaving uncertainty about whether these steps are enough to drive meaningful upside from here, the analyst tells investors in a research note.
Stifel
Buy -> Hold
downgrade
$73 -> $61
2025-11-06
Reason
Stifel
Price Target
$73 -> $61
2025-11-06
downgrade
Buy -> Hold
Reason
Stifel downgraded BioMarin to Hold from Buy with a price target of $61, down from $73.
Stifel
Paul Matteis
Buy
to
Hold
downgrade
$73 -> $61
2025-11-06
Reason
Stifel
Paul Matteis
Price Target
$73 -> $61
2025-11-06
downgrade
Buy
to
Hold
Reason
Stifel analyst Paul Matteis downgraded BioMarin to Hold from Buy with a price target of $61, down from $73. Even from what feels like an "impaired valuation," it is become harder for the firm to see BioMarin significantly outperforming over the next 12 months, barring a takeout, the analyst tells investors. This is due to competitive concerns for Voxzogo, which are "valid" given key opinion leader checks are "concerning," underlying Voxzogo growth trends that "aren't great" even before competition, and the view that the path to actually disproving the bear case is unclear.
About BMRN
BioMarin Pharmaceutical Inc. is a global biotechnology company engaged in the development of genetic discovery into medicines that make an impact on the life of each patient. The Company has eight commercial therapies and a clinical and preclinical pipeline. Its commercial products include Vimizim (elosulfase alpha), Naglazyme (galsulfase), Palynziq (pegvaliase-pqpz), Brineura (cerliponase alfa), Aldurazyme (laronidase), Roctavian (valoctocogene roxaparvovec), Kuvan (sapropterin dihydrochloride), and Voxzogo (vosoritide). Vimizim is an enzyme replacement therapy for the treatment of MPS IVA, a lysosomal storage disorder. The Roctavian is an adeno-associated virus (AAV5) vector gene therapy designed to restore factor VIII plasma concentrations in patients with severe hemophilia A. Its key clinical programs include Vosoritide, BMN 333, BMN 390, BMN 351, BMN 349, and INZ-701. Its late-stage enzyme replacement therapy, INZ-701, is being developed for the treatment of ENPP1 deficiency.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
