Fannie Mae, Freddie Mac shares tumble after conservatorship comments
SAN RAFAEL, Calif. - BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), a biotechnology company with a market capitalization of $12.96 billion and impressive gross margins of nearly 80%, announced on Monday that its Phase 3 PEGASUS trial for PALYNZIQ (pegvaliase-pqpz) met its primary efficacy endpoint, significantly reducing blood phenylalanine (Phe) levels in adolescents with phenylketonuria (PKU) compared to a Phe-restricted diet alone. The safety results were in line with the established profile of the medication, which is already approved for adult use. According to InvestingPro data, the company has demonstrated strong revenue growth of 18% over the last twelve months.
The PEGASUS trial involved 55 participants aged 12-17 and aimed to evaluate both the safety and efficacy of PALYNZIQ against diet management alone. The study, conducted over a primary treatment phase and an extension phase, will have its detailed results presented at a future medical meeting and submitted to global health authorities later this year, seeking to expand the treatment’s label to include adolescents. InvestingPro analysis shows seven analysts have recently revised their earnings expectations upward for BioMarin, suggesting growing confidence in the company’s pipeline developments. Subscribers can access 8 additional exclusive ProTips and comprehensive financial analysis through the platform’s Pro Research Report.
Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin, expressed the company’s commitment to advancing treatments for PKU, noting the importance of providing options for adolescents transitioning to adult living.
PALYNZIQ is a PEGylated enzyme therapy designed to substitute the deficient PAH enzyme in PKU patients, allowing for the breakdown of Phe. The treatment is currently approved in several regions for adults and individuals over 15 in Japan, with a safety profile primarily comprising immune-mediated responses, including anaphylaxis.
PKU is a genetic condition that leads to toxic accumulation of Phe in the blood, potentially causing severe neurological complications. Early diagnosis and treatment are critical, and while a strict diet can manage the condition, adherence is challenging for many, especially as they age.
BioMarin, a biotechnology firm founded in 1997, focuses on developing therapies for genetically defined diseases. The company’s announcement of the PEGASUS trial results is a significant step towards potentially offering younger PKU patients a new treatment option. With a strong financial health score rated as "GREAT" by InvestingPro, robust liquidity metrics, and current trading levels below Fair Value, BioMarin appears well-positioned to continue its growth trajectory. For detailed valuation analysis and comprehensive insights, investors can explore the company’s full Pro Research Report, part of InvestingPro’s coverage of over 1,400 US equities.
The information in this article is based on a press release statement from BioMarin Pharmaceutical Inc.
In other recent news, BioMarin Pharmaceutical Inc. reported a strong fourth quarter of 2024, with total revenues reaching $747 million, surpassing the consensus estimate of $712 million. This performance prompted Oppenheimer analysts to upgrade BioMarin’s stock rating from Perform to Outperform, setting a new price target of $98.00. Analysts at Cantor Fitzgerald maintained their Overweight rating on the stock, with a price target of $90.00, highlighting BioMarin’s projected revenue growth of 9-12% for 2025, expected to reach $3.1 billion to $3.2 billion. The company’s earnings are anticipated to grow significantly, with a projected increase of 20-25% year-over-year to $4.20-4.40 per share.
BioMarin also announced the appointment of Timothy P. Walbert to its Board of Directors, expanding the board from eleven to twelve members. Walbert, the former CEO of Horizon Therapeutics, brings extensive industry experience to the company. Additionally, BioMarin plans to present new data for its drugs Voxzogo and Palynziq at the upcoming ACMG Annual Clinical Genetics Meeting. Citi analysts have maintained a Neutral rating on BioMarin’s stock, with a price target of $82.00, as the company continues to focus on its research and development efforts. These developments reflect BioMarin’s ongoing strategic initiatives and its potential for future growth in the pharmaceutical sector.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.