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WASHINGTON, D.C. - BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), a $10.37 billion biotech company with an impressive 81% gross profit margin, announced Tuesday that its hemophilia A gene therapy ROCTAVIAN maintained efficacy and safety five years after treatment, according to data presented at the International Society on Thrombosis and Haemostasis Congress. InvestingPro data shows the company maintains a perfect Piotroski Score of 9, indicating strong financial health.
The Phase 3 GENEr8-1 trial showed that 81.3% of participants remained off prophylactic treatment after five years, with mean factor VIII activity levels remaining in the mild hemophilia range. The mean annualized bleeding rate for treated bleeds was 0.6 bleeds per year, with 77.8% of participants experiencing zero treated bleeds during year five. Despite these positive clinical results, BioMarin’s stock is currently trading near its 52-week low, presenting what InvestingPro analysis suggests is an undervalued opportunity.
Factor VIII activity levels were nearly stable compared to year four measurements, with mean activity at 24.0 IU/dL using the one-stage assay and 13.7 IU/dL with the chromogenic assay. Approximately 73.5% of participants maintained factor VIII levels in the mild hemophilia to normal range.
No new safety signals emerged during the five-year follow-up period. Throughout the trial, no participants developed factor VIII inhibitors or experienced thromboembolic events, and there were no treatment-related malignancies reported.
"I am impressed that the strong efficacy and safety results for ROCTAVIAN are sustained five years after individuals with severe hemophilia A received gene therapy," said Andrew Leavitt, M.D., director of the University of California San Francisco’s Adult Hemophilia Treatment Center.
The one-time infusion therapy works by delivering a functional gene designed to enable the body to produce factor VIII independently, potentially reducing the need for ongoing prophylaxis.
ROCTAVIAN received U.S. Food and Drug Administration approval in June 2023 and European Commission conditional marketing authorization in August 2022.
The information is based on a press release statement from BioMarin Pharmaceutical. With revenue growth of 19.36% over the last twelve months and a moderate P/E ratio of 19.68, the company shows strong fundamentals. For deeper insights into BioMarin’s financial health and growth prospects, access the comprehensive Pro Research Report available on InvestingPro, which covers over 1,400 top US stocks with expert analysis and actionable intelligence.
In other recent news, BioMarin Pharmaceutical Inc. has made significant strides with its recent acquisition of Inozyme Pharma for approximately $270 million. This strategic move is expected to strengthen BioMarin’s enzyme therapy portfolio, particularly with the addition of INZ-701, a Phase 3 candidate for treating ENPP1 Deficiency. Analysts from TD Cowen have reiterated a Buy rating with a $120 price target, emphasizing the acquisition as a promising addition to BioMarin’s pipeline, with pivotal trial data expected in 2026 and potential regulatory approval in 2027. Bernstein also raised its price target for BioMarin to $95, maintaining an Outperform rating, citing the acquisition’s alignment with the company’s existing business units and research strengths.
Stifel analysts have maintained a Buy rating with a $91 price target, highlighting BioMarin’s growth potential through its drug Voxzogo and the recent acquisition. The firm’s analysis suggests that BioMarin’s current stock price reflects a conservative outlook, with 2026 anticipated as a pivotal year for the company. Evercore ISI, maintaining an Outperform rating with a $105 target, described the acquisition as a savvy, low-risk transaction that complements BioMarin’s enzyme therapy business. The acquisition is seen as a strategic fit that aligns with BioMarin’s commitment to developing therapies for rare genetic diseases.
BioMarin’s CEO, Alexander Hardy, emphasized the acquisition’s potential to improve care for individuals with serious genetic conditions, while Inozyme’s CEO, Douglas A. Treco, expressed optimism about INZ-701’s transformative potential. The acquisition, subject to regulatory approval and other conditions, is expected to close in the third quarter of 2025. Investors will be closely monitoring BioMarin’s integration of INZ-701 into its portfolio and its ongoing strategic development efforts.
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