SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced it has entered into an exclusive worldwide license agreement with GC Biopharma Corp. and Hanmi Pharm Co., Ltd. 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 (a-Gal 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.
“This agreement reflects our growing commitment to the Fabry community, building on the acquisition of Amicus earlier this year,” said Alexander Hardy, President and Chief Executive Officer of BioMarin. “HM15421/GC1134A has the potential to offer a new way of treating Fabry disease, and we believe it could be an important option for patients. As we continue to add to our clinical portfolio, both through internal research and external programs, HM15421/GC1134A is an excellent fit and could allow us to reach a new generation of patients with Fabry beyond those with mutations amenable to treatment with GALAFOLD.”
Under the terms of the agreement, BioMarin will make an upfront payment of $70 million. GC and Hanmi are also jointly eligible to receive up to an additional $405 million upon the completion of certain development and commercial milestones, and tiered royalties.
In preclinical studies, HM15421/GC1134A demonstrated differentiated efficacy across key measures, including renal function, vascular health and peripheral nervous system outcomes. The candidate received Orphan Drug Designation from the U.S. Food and Drug Administration (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. GC and Hanmi will retain commercialization rights for HM15421/GC1134A in Korea.
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by inherited genetic changes in the GLA gene, which hampers activity of the enzyme alpha-galactosidase A (a-Gal A). Reduced a-Gal A activity leads to lysosomal accumulation of globotriaosylceramide (GL-3), which is believed to cause the various symptoms of Fabry disease, including pain, kidney failure and increased risk of heart attack and stroke. The symptoms can be severe, differ from person to person, and begin at an early age.
About HM15421/GC1134A
HM15421/GC1134A is an investigational, long-acting enzyme replacement therapy for Fabry disease, a rare, X-linked lysosomal storage disorder caused by deficient alpha-galactosidase A activity. HM15421/GC1134A is designed to extend half-life and improve stability relative to currently available enzyme replacement therapies, supporting subcutaneous dosing. HM15421/GC1134A is currently being evaluated in a Phase 1/2 clinical trial.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.
|
Contacts |
|
|
Investors |
Media |
|
Traci McCarty |
Erin Rau |
|
BioMarin Pharmaceutical Inc. |
BioMarin Pharmaceutical Inc. |
|
(415) 455-7558 |
(925) 683-9622 |