News & Events zu BioMarin Pharmaceutical Inc. (BMRN)BMRNBioMarin Pharmaceutical Inc.

BioMarin Enters Exclusive License Agreement with GC Biopharma and Hanmi Pharm

BioMarin has entered into an exclusive worldwide license agreement with GC Biopharma and Hanmi Pharm to develop and commercialize HM15421/GC1134A, an investigational long-acting enzyme replacement therapy for Fabry disease. HM15421/GC1134A is a human recombinant alpha-galactosidase A enzyme therapy currently being studied in a Phase 1/2 clinical trial in people living with Fabry disease and has the potential to be the first subcutaneous enzyme replacement therapy for Fabry disease. Under the terms of the agreement, BioMarin will make an upfront payment of $70M. GC and Hanmi are also jointly eligible to receive up to an additional $405M upon the completion of certain development and commercial milestones, and tiered royalties. The candidate received Orphan Drug Designation from the FDA in 2024. Following the ongoing Phase 1/2 study, BioMarin will assume sole responsibility for all subsequent clinical development, manufacturing and commercialization of HM15421/GC1134A outside of Korea.

BioMarin Appoints Cara Bauer as Chief People Officer

BioMarin Pharmaceutical announced that Cara Bauer, Ph.D., has been named Executive Vice President, Chief People Officer. Bauer will assume responsibility for the company's Human Resources organization, effective Oct. 5, succeeding Amy Wireman, who is retiring. Bauer joins BioMarin from Kyverna Therapeutics where she served as Chief Human Resources Officer.

BioMarin Presents POMBILITI + OPFOLDA Data in Japan

BioMarin Pharmaceutical announced new data evaluating the efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease were presented at the 31st Annual Congress of the World Muscle Society, in Hiroshima, Japan. These results from the PROPEL open-label extension study of POMBILITI + OPFOLDA, which were shared in a late-breaking presentation, demonstrated durable motor function outcomes and relative stabilization of pulmonary function over long-term follow-up. The analysis included data from 82 individuals who received POMBILITI + OPFOLDA continuously from baseline observed in the initial PROPEL study to year five. For the enzyme replacement therapy-experienced cohort, the mean duration of prior ERT at baseline was 7.6 years. These data represent long-term benefits for people with LOPD followed in the study, including both ERT-experienced and ERT-naive cohorts. "These five-year results demonstrate that POMBILITI and OPFOLDA can provide durable, long-term benefits for people living with late-onset Pompe disease, including those who had been on enzyme replacement therapy for years before enrolling in the study," said Greg Friberg, Chief Research & Development Officer. "Sustaining motor function and limiting pulmonary decline for up to five years in a progressive disease like LOPD is clinically meaningful, and we remain committed to continuing to share long-term evidence to inform the care of people with this rare genetic condition." No new safety signals were identified. 51.2% of participants experienced treatment-related adverse events, the majority of which were manageable. Five individuals discontinued treatment for safety reasons, including two due to serious treatment-related adverse events. Additional presentations at WMS included data evaluating nivudirsen an investigational antisense oligonucleotide for ambulatory boys ages 4 to 10 with Duchenne muscular dystrophy amenable to exon 51 skipping. Included in these presentations was an update from the ongoing Phase 1/2 351-201 study, which was previously shared at the 19th International Congress on Neuromuscular Diseases in July. Results from 351-201 showed treatment with nivudirsen was generally well tolerated and led to consistently positive outcomes across multiple measures of therapeutic benefit, including expression of near full-length dystrophin.