BioMarin Expands Enzyme Therapy Portfolio by Acquiring Inozyme Pharma

BioMarin Pharmaceutical Inc., a biotechnology company renowned for developing innovative therapies for genetic conditions, has announced its intention to acquire Inozyme Pharma, a clinical-stage biopharmaceutical company. The acquisition, valued at approximately $270 million, will involve an all-cash transaction at $4.00 per share. The boards of both companies have unanimously approved the deal, which is expected to conclude in the third quarter of 2025, pending regulatory approval and fulfillment of customary closing conditions.

This strategic acquisition will enhance BioMarin's portfolio of enzyme replacement therapies by adding INZ-701, Inozyme's promising late-stage therapy. INZ-701 is currently under development for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency. ENPP1 Deficiency is a rare and progressive genetic disorder that impacts blood vessels, soft tissues, and bones, leading to a heightened risk of cardiovascular complications. It also causes severe rickets and osteomalacia in both children and adults.

The first pivotal data readout from a Phase 3 study of INZ-701 in children is anticipated in early 2026, with a potential market launch in 2027. This therapy could become the first-of-its-kind treatment for ENPP1 Deficiency, offering significant hope to patients affected by this serious condition. Alexander Hardy, President and CEO of BioMarin, expressed the company's long-standing commitment to developing enzyme therapies for genetic disorders. He emphasized that acquiring INZ-701 aligns with BioMarin's strategy to innovate and address unmet medical needs.

Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme, highlighted the potential of INZ-701 to transform treatment for ENPP1 Deficiency. He acknowledged BioMarin's success in launching first-in-disease enzyme therapies and expressed confidence in their ability to bring INZ-701 to market.

INZ-701 is a pioneering subcutaneous enzyme replacement therapy intended for use across a range of patients, from infants to adults, with ENPP1 Deficiency. Inozyme is actively enrolling infants in a pivotal study, while also planning a supportive study for adolescents and adults. Results from a Phase 1/2 study in adults showed that INZ-701 had a favorable safety profile and led to improvements in vital biomarkers and quality of life, suggesting its potential benefit for patients.

ENPP1 Deficiency is caused by mutations in the ENPP1 gene, which lead to reduced enzymatic activity, low pyrophosphate levels, and phosphate regulation issues. Patients with the disorder face a significant risk of cardiovascular mortality, especially in infancy, and require extensive lifelong medical management due to the lack of approved therapies.

As part of the acquisition agreement, BioMarin will initiate a cash tender offer for all outstanding Inozyme shares. The transaction's completion is contingent upon customary closing conditions, including a majority tender of Inozyme shares and regulatory clearances. Once finalized, Inozyme shares not tendered will be converted into the right to receive the same cash offer.

BioMarin has reiterated its financial guidance for 2025, excluding the transaction's accounting impact, and aims to achieve a 40% Non-GAAP Operating Margin by 2026. The acquisition is supported by financial and legal advisors from both companies, ensuring a smooth transition.

BioMarin, headquartered in San Rafael, California, has a history of effectively translating genetic discoveries into therapeutic solutions. With multiple commercialized therapies and a robust pipeline, the company is committed to redefining treatment possibilities for genetic conditions globally. Inozyme, based in Boston, focuses on therapies targeting the PPi-Adenosine Pathway, with INZ-701 leading their clinical development efforts.