Biomarin Pharmaceutical Inc Stock Price, News & Analysis

BioMarin (NASDAQ:BMRN) announced positive Phase 3 PEGASUS trial results for PALYNZIQ® in treating adolescents with phenylketonuria (PKU). The study demonstrated a 49.7% decrease in mean blood phenylalanine (Phe) levels compared to just 0.3% with diet alone.

Key achievements include: 51.6% of PALYNZIQ-treated patients achieved blood Phe levels ≤600 µmol/L, and 45.2% of participants achieved ≥50% reduction in blood Phe from baseline. The safety profile aligned with adult studies, though 5.6% experienced serious adverse events (anaphylaxis) leading to discontinuation.

BioMarin plans to submit these results to global health authorities in the second half of 2025 to expand PALYNZIQ's indication to include adolescent treatment.

BioMarin Pharmaceutical (NASDAQ: BMRN) has announced its participation in three major healthcare investor conferences in September 2025:

• Cantor Global Healthcare Conference on September 3 at 8:00 AM EST
• Wells Fargo Healthcare Conference on September 4 at 8:45 AM EST
• Morgan Stanley Global Healthcare Conference on September 9 at 11:30 AM EST

The company will provide live audio webcasts of the presentations through their investor relations website, with archived versions available for limited time after each event.

BioMarin (NASDAQ:BMRN) reported strong Q2 2025 results with total revenues of $825 million, up 16% year-over-year. The company achieved GAAP EPS of $1.23 (+124% Y/Y) and Non-GAAP EPS of $1.44 (+50% Y/Y).

Key highlights include VOXZOGO revenue growth of 20% and Enzyme Therapies revenue growth of 15%. The company completed the acquisition of Inozyme in July 2025, adding BMN 401 to its portfolio. Additionally, BMN 333, their long-acting CNP for achondroplasia, showed promising results in healthy volunteer studies.

Based on strong performance, BioMarin raised its full-year 2025 guidance for Total Revenues, Non-GAAP Operating Margin, and Non-GAAP Diluted EPS. The company ended Q2 with $1.9 billion in cash and investments.

BioMarin Pharmaceutical (Nasdaq: BMRN) has appointed Ian T. Clark to its Board of Directors, effective August 1, 2025. Clark, the former CEO of Genentech (2010-2016), brings significant industry experience, having overseen the launch of 15 new drugs during his tenure. Prior to Genentech, he held senior positions at major pharmaceutical companies including Novartis, Sanofi, Ivax, and G.D. Searle.

Clark currently serves on the boards of several biotech companies including Olema Oncology, Takeda Pharmaceutical, and Guardant Health. He is also an advisor to KKR & Co. Inc. His appointment brings valuable expertise in drug development and commercialization to BioMarin's board.

BioMarin Pharmaceutical (NASDAQ: BMRN) has scheduled its second quarter 2025 financial results conference call and webcast for Monday, August 4, 2025, at 4:30 p.m. ET. The company's President and CEO, Alexander Hardy, will lead the discussion of Q2 2025 financial performance and provide business updates.

Investors and interested parties can access the live audio webcast through BioMarin's investor relations website. A replay will be available for one week after the call. The company has provided both domestic and international dial-in numbers for telephone access to the conference call.

BioMarin Pharmaceutical (Nasdaq: BMRN) has completed its acquisition of Inozyme Pharma (Nasdaq: INZY) in an all-cash transaction valued at approximately $270 million, or $4.00 per share. The tender offer, which expired on June 30, 2025, resulted in approximately 70% of Inozyme shares being validly tendered.

The strategic acquisition strengthens BioMarin's enzyme therapies portfolio by adding INZ-701, a late-stage enzyme replacement therapy. This investigational treatment targets ENPP1 Deficiency, a rare genetic condition affecting blood vessels, soft tissues, and bones. INZ-701 is designed to restore pyrophosphate and adenosine levels and has potential applications in additional indications including ABCC6 Deficiency and calciphylaxis.

Following the merger completion, Inozyme shares have ceased trading on the Nasdaq Global Select Market and will be delisted. The Boston-based Inozyme, with approximately 50 employees, brings expertise in the PPi-Adenosine Pathway research and development to BioMarin's established portfolio of eight commercial therapies.

BioMarin (NASDAQ:BMRN) presented compelling five-year Phase 3 results for ROCTAVIAN®, their gene therapy treatment for severe hemophilia A, at the ISTH 2025 Congress. The GENEr8-1 trial demonstrated sustained efficacy with mean Factor VIII activity remaining in the mild hemophilia range (24.0 IU/dL one-stage assay), and 73.5% of participants maintaining FVIII levels in the mild-to-normal range.

Key outcomes after five years include: 81.3% of participants remaining off prophylaxis, mean annualized bleeding rate of just 0.6 bleeds/year, and 77.8% of participants experiencing zero treated bleeds during year five. The study confirmed consistent safety with no new signals, no FVIII inhibitors, no thromboembolic events, and no treatment-related malignancies.

BioMarin Pharmaceutical (BMRN) has announced the acquisition of Inozyme Pharma for $4.00 per share in an all-cash transaction valued at approximately $270 million. The deal adds INZ-701, a Phase 3 enzyme replacement therapy being developed for ENPP1 Deficiency, to BioMarin's enzyme therapies portfolio. INZ-701 could become the first treatment for this rare genetic condition that affects blood vessels, soft tissues, and bones. The first Phase 3 pivotal data readout in children is expected in early 2026, with potential launch in 2027. The transaction, unanimously approved by both companies' boards, is expected to close in Q3 2025. Phase 1/2 study results showed a favorable safety profile with improvements in pyrophosphate levels, bone mineralization biomarkers, and quality of life.

BioMarin presented new data for VOXZOGO® (vosoritide) showing promising results in treating children with achondroplasia and other skeletal conditions. Key findings include:

Treatment demonstrated significant reduction in tibial bowing compared to placebo in children with achondroplasia, with sustained improvement over several years. Modeling indicated potential final height increases of 21.7 cm in girls and 26.4 cm in boys compared to untreated children when treated early and continuously.

A large retrospective study of over 600 patients with hypochondroplasia revealed higher rates of comorbidities, surgeries, and doctor visits compared to those without the condition. Additionally, early data from a Phase 2 trial showed promising increases in annualized growth velocity for girls with Turner syndrome, even in those who previously had suboptimal results with human growth hormone.

BioMarin has completed enrollment in its pivotal Phase 3 study for VOXZOGO in hypochondroplasia, with topline data expected in 2026 and potential launch in 2027.