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Bolsters its enzyme therapies portfolio, adding a late-stage enzyme replacement therapy, INZ-701.
May 16, 2025
By: Charlie Sternberg
BioMarin Pharmaceutical Inc. has entered into a definitive agreement to acquire Inozyme Pharma Inc. for $4.00 per share in an all-cash transaction for a total consideration of approximately $270 million.
The transaction has been unanimously approved by the Boards of Directors of both companies and is expected to close in the third quarter of 2025, subject to regulatory approval, successful completion of a tender offer and other customary closing conditions.
The acquisition will strengthen BioMarin’s enzyme therapies portfolio, adding a late-stage enzyme replacement therapy, INZ-701, which is currently being assessed for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency, a rare, serious and progressive genetic condition that affects blood vessels, soft tissues and bones. The condition is associated with increased cardiovascular mortality risk across all age groups, especially in infants. It is also associated with severe rickets and osteomalacia in children and adults. Data from the first Phase 3 pivotal study of INZ-701 in children is expected in early 2026, with potential regulatory approval in 2027.
“BioMarin has been deeply committed to advancing enzyme therapies for children and adults living with serious genetic conditions for more than 25 years, and today’s agreement builds on our legacy,” said Alexander Hardy, President and CEO of BioMarin. “This acquisition brings to BioMarin an important medicine that has the potential to be the first treatment for children and adults with ENPP1 Deficiency, improving care for people living with this serious condition.”
“Today’s announcement gives greater hope to patients who may benefit from INZ-701, a potentially transformative therapy that aims to address the underlying causes and systemic impacts of ENPP1 Deficiency,” said Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme. “BioMarin has paved the way over the past two and a half decades, successfully launching five first-in-disease enzyme therapies. I’d like to thank the team at Inozyme and our partners for their outstanding work and dedication, as we pass this important potentially life-changing therapy to the leading innovator in genetically defined conditions.”
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