BioMarin Pharmaceutical Inc. and Inozyme Pharma, Inc. (Nasdaq: INZY) announced today that BioMarin has entered into a definitive agreement to acquire Inozyme 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 Chief Executive Officer 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. 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.""
""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., Chief Executive Officer 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."" INZ-701 and ENPP1 Deficiency
INZ-701 is a potential first-in-class, subcutaneous enzyme replacement therapy that is being developed for infants, pediatric and adult patients with ENPP1 Deficiency across a continuum of phenotypes. In addition to the 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 demonstrated 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 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. 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.