ENPP1 inhibitor(Aculeus)

Acquisition is Strong Strategic Fit for BioMarin, Adding INZ-701, a Phase 3 Enzyme Replacement Therapy Being Developed for Treatment of ENPP1 Deficiency First Pivotal Data Readout in Children Expected in Early 2026 with Potential Launch in 2027; Additional Clinical Programs to Expand to Patients of all Ages Potential First-in-Disease Treatment for ENPP1 Deficiency Conference Call and Webcast Scheduled Today at 8:45 a.m. ET SAN RAFAEL, CA and BOSTON, MA, USA I May 16, 2025 I BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) 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. Terms of the Transaction Under the terms of the merger agreement, BioMarin will promptly commence a cash tender offer to acquire all of the outstanding shares of Inozyme common stock at a price of $4.00 per share. Inozyme’s Board of Directors unanimously recommends that Inozyme’s stockholders tender their shares in the tender offer. The consummation of the tender offer is subject to customary closing conditions, including the tender of at least a majority of the outstanding shares of Inozyme, the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and other customary conditions. Following the successful completion of the tender offer, a wholly owned subsidiary of BioMarin will merge with Inozyme and the outstanding Inozyme shares not tendered in the tender offer will be converted into the right to receive the same $4.00 per share in cash paid in the tender offer. The transaction is not subject to any financing condition. BioMarin also today reaffirmed previously provided full-year 2025 financial guidance, excluding the impact of the accounting treatment of this transaction in accordance with Generally Accepted Accounting Principles (GAAP) upon closing, as well as its plan to achieve 40% Non-GAAP Operating Margin in 2026. Advisors Goldman Sachs & Co. LLC is acting as exclusive financial advisor to BioMarin, and Cooley LLP is serving as legal counsel. Centerview Partners LLC is acting as exclusive financial advisor to Inozyme, and Goodwin Procter LLP is serving as legal counsel. Conference Call BioMarin will host a conference call and webcast to discuss the acquisition today, May 16, 2025, at 8:45 a.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com . About BioMarin BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that offer new possibilities for people living with genetically defined conditions around the world. To learn more, please visit www.biomarin.com . About Inozyme Inozyme Pharma is a clinical-stage biopharmaceutical company, with approximately 50 employees based in Boston. The company is dedicated to developing innovative therapeutics that target the PPi-Adenosine Pathway, a key regulator of bone health and blood vessel function. Disruptions in this pathway underlie a range of severe diseases, including ENPP1 Deficiency. Our lead investigational therapy, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy designed to restore pyrophosphate and adenosine levels. INZ-701 is currently in late-stage clinical development in ENPP1 Deficiency, with the potential to expand into additional indications where deficiencies in the Pyrophosphate-Adenosine Pathway contribute to disease pathology, including ABCC6 Deficiency and calciphylaxis. Through our pioneering work, we aim to transform treatment options for patients affected by these devastating conditions. To learn more, please visit www.inozyme.com . SOURCE: BioMarin Pharmaceutical