Biomarin Pharmaceutical Inc Stock Price, News & Analysis

BioMarin Pharmaceutical (NASDAQ:BMRN) presented promising results from a Phase 2 trial of VOXZOGO™ (vosoritide) for treating achondroplasia in children under five. Over 52 weeks, children on VOXZOGO (n=43) showed a height Z-score improvement of 0.30 SD and an annualized growth velocity increase of 0.92 cm/year compared to placebo (n=32). The company plans discussions with health authorities in H2 2022 to expand access for younger patients. The safety profile was consistent with older patients, with fewer serious adverse events in the Voxzogo group, suggesting early treatment benefits.

BioMarin Pharmaceutical announced significant results from its Phase 1/2 study of valoctocogene roxaparvovec, showing a 95% reduction in mean annualized bleed rate (ABR) and 96% reduction in Factor VIII usage after six years for the 6e13 vg/kg cohort. The 4e13 vg/kg cohort exhibited similar success with a 91% reduction in mean ABR over five years. BioMarin plans to present data at the ISTH 2022 Congress and expects to resubmit its Biologics Licensing Application (BLA) to the FDA by the end of September 2022, while the EMA's Marketing Authorization Application is currently under review.

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) has appointed Humaira Serajuddin as Senior Vice President, Chief Marketing Officer, and Erin Burkhart as Group Vice President, Chief Accounting Officer. These strategic hires are aimed at supporting the company's growth and navigating towards sustainable profitability. Serajuddin brings over 20 years of pharmaceutical experience, including successful product launches, while Burkhart has extensive knowledge in corporate finance and accounting, having previously worked at Eli Lilly. Both leaders are expected to drive BioMarin's anticipated growth and transformation in the coming years.

BioMarin Pharmaceutical announced the presentation of findings from their ongoing studies of valoctocogene roxaparvovec, an investigational gene therapy for severe hemophilia A, at the WFH 2022 World Congress in Montreal from May 8-11. The data includes updates on treatment durability, hepatotoxicity, and quality of life for patients. The global phase 3 GENEr8-1 study showed positive two-year results. BioMarin aims to improve shared decision-making for patients with severe hemophilia A, as current treatments often don't eliminate bleeding risks.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced that CEO Jean-Jacques Bienaimé will present at the BofA Securities Healthcare Conference on May 12, 2022, at 8:40 am PT/11:40 am ET in Las Vegas. Interested individuals can access the live webcast on the BioMarin website. A replay will be available for 90 days post-event. BioMarin specializes in developing therapies for serious rare diseases, with a portfolio of seven commercial products and various clinical candidates.

BioMarin reported record total revenues of $519 million for Q1 2022, an 11% increase year-over-year, driven by strong demand for Voxzogo and other products. Notably, Voxzogo generated $20 million in net product revenues, with full-year guidance revised to $100-125 million. GAAP net income surged to $120.8 million, attributed to a $89 million gain from a Rare Pediatric Disease Voucher. The company's pipeline includes valoctocogene roxaparvovec for hemophilia A, with regulatory reviews ongoing in Europe and the U.S.

BioMarin Pharmaceutical Inc. (BMRN) will host a conference call on April 27, 2022, at 4:30 p.m. ET to discuss its first quarter 2022 financial results and provide a general business update. The call can be accessed via dial-in numbers or through a live audio webcast available on the company's website. Interested parties will also have access to a replay of the call for one week post-event. BioMarin specializes in developing therapies for rare genetic diseases and currently has a portfolio of seven commercialized products.

BioMarin Pharmaceutical has published significant results from its Phase 3 GENEr8-1 study of valoctocogene roxaparvovec for severe hemophilia A in the New England Journal of Medicine. The study indicates an 84% reduction in the mean annualized bleeding rate (ABR) and a 99% reduction in factor VIII utilization compared to standard prophylactic therapy, both statistically significant (p < 0.001). Moreover, 90% of participants experienced fewer or no treated bleeds post-infusion. Safety data shows no new adverse events, reinforcing the potential of this gene therapy to transform treatment protocols for hemophilia A.

BioMarin Pharmaceutical reported a GAAP net loss of $57.9 million for Q4 2021, contrasting with a net income of $22.1 million in Q4 2020. Total revenues slightly declined by 1% year-over-year, totaling $449.8 million in Q4 and $1,846.3 million for the full year.

Despite this, the company anticipates a positive outlook for 2022 with projected total revenues between $2,050 to $2,150 million. The launch of Voxzogo is underway, and regulatory approvals for valoctocogene roxaparvovec gene therapy are anticipated.