BioMarin Expands Enzyme Therapies Portfolio with Inozyme Acquisition
BioMarin Pharmaceutical Inc. has entered into a definitive agreement to acquire clinical-stage biotech Inozyme Pharma, Inc. for approximately $270 million in an all-cash transaction. The move is set to significantly expand BioMarin’s enzyme therapy franchise. The deal, announced jointly by both companies on Friday, brings under BioMarin’s umbrella the promising investigational therapy INZ-701, currently in Phase 3 development as a potential first-ever treatment for ENPP1 Deficiency, a devastating and progressive genetic disorder affecting multiple organ systems.
The acquisition represents a strategic fit for BioMarin, complementing its established expertise in developing first-in-disease enzyme replacement therapies. Under the terms of the agreement, BioMarin will pay $4.00 per share in cash for all outstanding shares of Inozyme through a tender offer expected to close in the third quarter of 2025, pending customary regulatory approvals and closing conditions.
A Transformative Enzyme Replacement Therapy for an Orphan Disease
At the heart of this transaction lies INZ-701, a subcutaneous enzyme replacement therapy designed to address the underlying pathophysiology of ENPP1 Deficiency. This ultra-rare condition, caused by mutations in the ENPP1 gene, leads to deficient ectonucleotide pyrophosphatase/phosphodiesterase 1 activity, resulting in critically low levels of pyrophosphate and subsequent severe clinical manifestations across all age groups.
The clinical burden of ENPP1 Deficiency is particularly profound, with infants facing high cardiovascular mortality risk and patients of all ages experiencing debilitating skeletal complications, including rickets and osteomalacia. Currently, no approved therapies exist that target the root cause of this multisystem disorder, leaving patients to rely on supportive care and symptom management.
Alexander Hardy, President and Chief Executive Officer of BioMarin, emphasised the strategic importance of this acquisition:
“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.”
Clinical Development Pathway and Expected Milestones
The acquisition comes at a pivotal moment in INZ-701’s development trajectory. Inozyme has made significant progress advancing the therapy through clinical trials, with Phase 1/2 data in adult patients demonstrating a favourable safety profile and encouraging signals of biochemical and clinical activity. Most notably, treatment with INZ-701 resulted in increased pyrophosphate levels, improvements in bone mineralisation biomarkers, and positive quality-of-life measures, all without any serious treatment-related adverse events reported.
BioMarin anticipates several near-term value inflection points for the program. The first pivotal data readout from the ongoing Phase 3 study in pediatric patients is expected in early 2026, potentially positioning the therapy for regulatory approval as early as 2027. The development program is comprehensive, with ongoing enrollment in a pivotal study in infants and plans to initiate supportive studies in adolescents and adults, reflecting the therapy’s potential applicability across the full spectrum of ENPP1 Deficiency presentations.
Douglas A. Treco, Ph.D., Chief Executive Officer and Chairman of Inozyme, commented on the transaction’s significance:
“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.”
Financial and Strategic Considerations
The $270 million acquisition price represents a premium to Inozyme’s recent trading levels, reflecting both the potential of INZ-701 and the strategic value BioMarin places on strengthening its rare disease portfolio. Importantly, BioMarin reaffirmed its previously provided full-year 2025 financial guidance, indicating the transaction’s manageable impact on its strong financial position.
The deal structure includes a cash tender offer for all outstanding Inozyme shares at $4.00 per share, with the transaction not subject to any financing contingency. Following the successful completion of the tender offer, the remaining shares will be acquired through a merger at the same price per share. The transaction has received unanimous approval from both companies’ Boards of Directors.
Goldman Sachs & Co. LLC served as the exclusive financial advisor to BioMarin, with legal counsel provided by Cooley LLP. Inozyme engaged Centerview Partners LLC as its exclusive financial advisor and Goodwin Procter LLP for legal representation.
Expanding BioMarin’s Rare Disease Leadership
This acquisition marks another strategic step in BioMarin’s ongoing efforts to maintain its position as a leader in innovative therapies for genetically defined conditions. The company’s proven track record in developing and commercialising enzyme replacement therapies – including five first-in-disease treatments – positions it well to maximise the potential of INZ-701 should it gain regulatory approval.
The addition of INZ-701 complements BioMarin’s existing rare disease portfolio and pipeline while addressing a significant unmet medical need. With its global commercial infrastructure and deep expertise in bringing specialised therapies to market, BioMarin is uniquely positioned to ensure broad patient access should INZ-701 demonstrate safety and efficacy in ongoing and planned clinical trials.
Looking Ahead to INZ-701’s Clinical Development
As the transaction progresses toward expected closing in the third quarter of 2025, the pharmaceutical industry will be watching closely for updates on INZ-701’s clinical development. The therapy represents not only a potential breakthrough for ENPP1 Deficiency patients but also validates BioMarin’s strategy of targeted business development to complement its internal innovation efforts.
For patients and families affected by ENPP1 Deficiency, this acquisition brings renewed hope for the first disease-modifying treatment for this serious condition. For BioMarin, it represents another opportunity to deliver on its mission of transforming lives through genetic science, while for Inozyme shareholders, it provides recognition of the value created through years of innovative research and clinical development.
To learn more, please visit www.biomarin.com and www.inozyme.com.
Original Source: Press Release – BioMarin Strengthens Enzyme Therapy Business with Acquisition of Inozyme Pharma (May 16, 2025 SAN RAFAEL, Calif. and BOSTON, May 16, 2025 / PRNewswire/)
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