Biomarin Pharmaceutical Inc Stock Price, News & Analysis

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.

BioMarin (BMRN) reported strong Q1 2025 financial results with total revenues of $745 million, up 15% year-over-year. The company achieved GAAP EPS of $0.95 (+107% Y/Y) and Non-GAAP EPS of $1.13 (+59% Y/Y). Growth was primarily driven by VOXZOGO, which saw 40% revenue growth, reaching $214 million. Enzyme Therapies revenue grew 8% to $484 million. The company maintained its 2025 guidance, projecting total revenues of $3.1-3.2 billion. Key pipeline developments include positive Phase 3 PALYNZIQ results for adolescents and completed enrollment in VOXZOGO's hypochondroplasia study. Operating margins improved significantly, with GAAP operating margin at 30.0% (+16.4 points Y/Y) and Non-GAAP at 35.7% (+11.9 points Y/Y). Cash position strengthened to $1.8 billion.

BioMarin Pharmaceutical (NASDAQ: BMRN) has scheduled its first quarter 2025 financial results conference call and webcast for Thursday, May 1, 2025, at 4:30 p.m. ET. The event will be hosted by Alexander Hardy, President and Chief Executive Officer, who will discuss Q1 2025 financial performance and provide a business update.

Participants can join via phone using U.S./Canada dial-in (888-596-4144) or International dial-in (646-968-2525) with Conference ID 4327591. A live audio webcast will be available through BioMarin's investor website. A replay will be accessible for one week following the call.

BioMarin Pharmaceutical (Nasdaq: BMRN) announced positive Phase 3 PEGASUS trial results for PALYNZIQ® (pegvaliase-pqpz) in treating adolescents with phenylketonuria (PKU). The study met its primary efficacy endpoint, showing statistically significant reduction in blood Phe levels among patients aged 12-17 compared to diet alone.

The safety profile aligned with known results, and PALYNZIQ remains the first and only enzyme therapy approved for adult PKU treatment. BioMarin plans to present detailed findings at an upcoming medical meeting and will submit to global health authorities later this year to expand PALYNZIQ's label for adolescent use.

BioMarin (BMRN) presented new data for two key medicines at the 2025 ACMG Annual Meeting. A Japanese study of VOXZOGO in children under 3 with achondroplasia showed strong treatment adherence with no reported treatment-related adverse events across 63 children followed for up to 23.7 months.

The PALYNZIQ OPAL study demonstrated significant blood Phe level reduction from 1029 μmol/L at baseline to 293 μmol/L at week 96, representing a 67.8% decrease. Quality of life improvements were observed through PKU-QOL and PKU-SSIS measurements. Additional analyses from the Phase 3 PRISM trial showed that maintaining lower blood Phe levels led to improvements in attention and mood.

The ATLAS study revealed an increased proportion of PKU patients achieving target blood Phe levels ≤360 μmol/L across 19 U.S. clinics, with Phase 3 results in adolescents expected later this year.

BioMarin Pharmaceutical (NASDAQ: BMRN) has announced its upcoming participation at the TD Cowen 45th Annual Healthcare Conference in Boston, MA. The company's management will deliver a presentation on Tuesday, March 4, 2025, at 6:50 AM PT / 9:50 AM ET.

The presentation will be accessible through a live audio webcast via the company's investor relations website at investors.biomarin.com. For those unable to attend live, an archived version of the presentation will be made available on BioMarin's website for a time after the conference.

BioMarin Pharmaceutical (Nasdaq: BMRN) has appointed Timothy P. Walbert to its Board of Directors, effective Feb. 24, 2025. Walbert, the former chairman, president and CEO of Horizon Therapeutics until its acquisition by Amgen in 2023, brings over 30 years of pharmaceutical industry experience.

Walbert's extensive background includes leadership roles at IDM Pharma (acquired by Takeda), NeoPharm, Abbott (now AbbVie), Pharmacia, Merck, Pfizer, and Wyeth Ayerst. He currently serves as senior advisor at Amgen and holds board positions at Sagimet Biosciences, Mirum Pharmaceuticals, and Century Therapeutics.

The appointment aligns with BioMarin's new strategy focused on innovation, growth, and value creation, with CEO Alexander Hardy highlighting Walbert's operational expertise and patient-centered approach in commercializing medicines.