In a significant move to expand its rare disease portfolio, BioMarin Pharmaceutical announced Friday its plans to acquire Boston-based Inozyme Pharma in an all-cash transaction valued at $270 million. The deal, which has received unanimous approval from both companies’ boards of directors, is expected to close between July and September 2025.
The acquisition will provide BioMarin with access to Inozyme’s experimental enzyme replacement therapy, INZ-701, which targets rare genetic disorders ENPP1 deficiency and ABCC6 deficiency. These life-threatening conditions occur when the body fails to produce sufficient amounts of an enzyme crucial for creating inorganic pyrophosphate, a molecule that prevents mineral deposits in soft tissues.
Without proper treatment, patients can experience severe complications including bone weakness, pain, and dangerous arterial blockages that may lead to strokes, tissue death, or multiple organ failure. The mortality rate exceeds 50% in newborns and infants with severe forms of these deficiencies, and currently, no FDA-approved treatments exist for these conditions.
INZ-701 is designed to replace the missing enzyme and restore inorganic pyrophosphate production. Results from a pivotal late-stage trial in children with ENPP1 deficiency are anticipated in 2026, potentially leading to FDA approval by 2027. The company has already completed a Phase 2 study for ABCC6 deficiency patients and a Phase 1 trial for a related blood vessel calcification condition associated with kidney failure.
Industry analysts have responded positively to the announcement. Leerink Partners analyst Joseph Schwartz noted that the acquisition “fits like a glove” with BioMarin’s existing business model, which includes other enzyme replacement therapies such as Vimizim, Naglazyme, and Palynziq. Similarly, Stifel analyst Paul Matteis described the deal as a “very strong strategic fit,” highlighting BioMarin’s established commercial infrastructure as ideal for maximizing the potential of INZ-701.
Though ENPP1 deficiency affects only approximately 10,000 patients globally, analysts believe the treatment area could develop into a valuable commercial opportunity for BioMarin. However, RBC Capital Markets analyst Luca Issi offered a more measured perspective, suggesting that while the acquisition makes strategic sense, it may not significantly impact BioMarin’s overall performance.
The transaction terms specify that BioMarin will acquire Inozyme shares at $4 each, representing a substantial 180% premium over Inozyme’s closing price of $1.42 on May 15. BioMarin’s financial position remains strong, with approximately $1.3 billion in cash, cash equivalents, and short-term investments as of March 2025.
BioMarin CEO Alexander Hardy emphasized the company’s commitment to growth through both internal and external innovation, stating they are well-positioned financially to acquire additional assets while advancing treatments for patients with unmet medical needs. This acquisition follows BioMarin’s strategic restructuring in 2024, which included workforce reductions and the elimination of several drug development programs.
The market responded positively to the announcement, with BioMarin’s shares trading up more than 1% to nearly $60 by late Friday morning. The company maintains its ambitious goal of achieving $4 billion in annual revenue by 2027, and this acquisition appears to align with that strategic objective.
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