BioMarin licenses long-acting Fabry enzyme replacement therapy HM15421/GC1134A, while Ferring agrees to acquire fertility biotech Gameto.
Two deals announced this week show established drugmakers adding specialty assets in very different corners of medicine. BioMarin paid $70 million upfront for exclusive worldwide rights to an investigational long-acting therapy for Fabry disease, with up to $405 million in further milestones.1
Ferring Pharmaceuticals signed an agreement to acquire Gameto, a cell-engineering company focused on women’s reproductive health, in a transaction worth up to $1.3825 billion if development, regulatory and commercial milestones are met.2
BioMarin entered an exclusive worldwide license agreement with GC Biopharma and Hanmi Pharm for HM15421/GC1134A, an investigational, long-acting enzyme replacement therapy for Fabry disease, the company announced Oct. 29.1 BioMarin pays $70 million upfront.
GC Biopharma and Hanmi are jointly eligible for up to $405 million in development and commercial milestones, plus tiered royalties on net sales; the royalty rates were not disclosed.1
“This agreement reflects our growing commitment to the Fabry community, building on the acquisition of Amicus earlier this year,” says Alexander Hardy, president and chief executive officer of BioMarin.
Following the ongoing Phase 1/2 study, BioMarin assumes sole responsibility for clinical development, manufacturing and commercialization outside Korea, where GC Biopharma and Hanmi retain commercialization rights.1 The release does not say who funds the current study, and it gives no closing conditions or timeline.1
The candidate is a human recombinant alpha-galactosidase A enzyme designed for a longer half-life and better stability, features intended to support subcutaneous dosing. BioMarin says it has the potential to be the first subcutaneous enzyme replacement therapy for Fabry disease.1
The company reports differentiated preclinical efficacy in renal function, vascular health and peripheral nervous system outcomes, and FDA granted the candidate orphan drug designation in 2024.1 Hardy says HM15421/GC1134A is “an excellent fit and could allow us to reach a new generation of patients with Fabry.”
Fabry disease is an X-linked lysosomal storage disorder caused by changes in the GLA gene. Reduced alpha-galactosidase A activity allows globotriaosylceramide to build up in cells, which is believed to drive symptoms that include pain, kidney failure and increased risk of heart attack and stroke.
Symptoms can be severe, vary between patients and begin early in life.1 A published review reported an annual incidence of 1 in 100,000 and cautioned that this may underestimate true prevalence.3
The program is in an early-stage trial, and the release reports no clinical efficacy or safety results. The efficacy claims cited so far are preclinical and company-reported, and whether a subcutaneous formulation improves outcomes for patients has not been tested.1
Ferring signed an agreement on Oct. 27 to acquire Gameto, a biotechnology company that uses cell engineering for women’s reproductive health, starting with infertility and expanding into ovarian disease and menopause.2 The transaction is worth up to $1.3825 billion, with payments contingent on development, regulatory and commercial milestones.
The upfront payment was not disclosed.2,4 Completion is subject to customary and other closing conditions, and the companies will operate independently until then. No closing date was given.2
“Ferring has been committed to helping people build families for more than 50 years,” says Jean-Frédéric Paulsen, chairman of the board and chief executive officer of Ferring Pharmaceuticals. “By combining Gameto’s scientific innovation with our expertise and global reproductive-medicine platform, we see an opportunity to advance new approaches in fertility care and reach more patients worldwide.”
Ferring’s release states that Gameto’s egg-maturation program is investigational in the U.S. and has not been approved by FDA.2 BioPharma Dive reports that the lead product, Fertilo, uses stem cell-derived ovarian support cells to grow eggs outside the body, is in Phase 3 testing in the U.S., and is cleared for use in Australia and parts of Latin America.
Gameto says the approach could remove the need for hormone injections and shorten fertility treatment cycles to “as little as three days.” The company has raised $127 million since its 2020 founding.4
“This is a defining moment for Gameto and for our mission to transform reproductive health,” says Dina Turner, PhD, chief executive officer and co-founder of Gameto. “By combining our pioneering cell-engineering platform with Ferring’s deep expertise and global reach, we have an extraordinary opportunity to accelerate innovation, expand access and bring more modern fertility treatment options to women and families around the world.”
The size of the upfront payment, the expected closing date and the share of the $1.3825 billion tied to regulatory milestones were not disclosed.2,4
