BioMarin Pharmaceutical and Inozyme Pharma announced that BioMarin has entered into a definitive agreement to acquire Inozyme for $4 per share in an all-cash transaction for a total consideration of about $270 million.
The Boards of Directors at both companies unanimously approved the deal. Subject to regulatory approval, successful completion of a tender offer, and other customary closing conditions, it is expected to close in the third quarter of 2025.
INZ-701: This 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 related to 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.
INZ-701 is a potential first-in-class and subcutaneous enzyme replacement therapy that is being developed for infants, pediatric and adult patients with ENPP1 Deficiency across a continuum of phenotypes. Along with ongoing Phase 3 pivotal study in children, Inozyme is currently enrolling infants in a pivotal study, and a supportive study for adolescents and adults is being planned.
In a Phase 1/2 study of adults living with ENPP1 Deficiency, INZ-701 showed a favorable safety profile, with no serious adverse events attributed to INZ-701. Improvements in pyrophosphate levels, bone mineralization biomarkers, and quality of life were all observed, suggesting a prospect for benefit in patients.
ENPP1 Deficiency is a lifelong, rare, progressive, multisystemic condition caused by mutations in the ENPP1 gene, leading to loss of ENPP1 enzymatic activity that results in low pyrophosphate, upregulation of fibroblast growth factor-23, and phosphate wasting. And the condition affects blood vessels, soft tissues and bones. It is associated with high risk of cardiovascular mortality in patients of all ages, especially infants. It is also associated with severe rickets and osteomalacia in children and adults. Patients require considerable multidisciplinary lifelong medical and surgical management of complications. Currently, there are no approved therapies for ENPP1 Deficiency.
Advisors: Goldman Sachs is acting as exclusive financial advisor to BioMarin, and Cooley is serving as legal counsel. Centerview Partners is acting as exclusive financial advisor to Inozyme, and Goodwin Procter LLP is serving as legal counsel.
KEY QUOTES:
“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. 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. As BioMarin continues our transformation and delivers on our corporate strategy, we will continue to evaluate external innovation alongside internal innovation. We are in a strong financial position to bring in additional assets as we accelerate the development of medicines for patients with significant unmet need.”
Alexander Hardy, President and Chief Executive Officer of BioMarin
“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. 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.”
Douglas A. Treco, Ph.D., Chief Executive Officer and Chairman of Inozyme
