In a significant move to expand its rare disease portfolio, BioMarin Pharmaceutical has announced its intention to acquire Boston-based Inozyme Pharma in a $270 million all-cash transaction. The deal, which received unanimous approval from both companies’ boards, is expected to close in the third quarter of 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 enzymes needed to generate inorganic pyrophosphate, a crucial molecule that prevents mineral deposits in soft tissues.
Without proper treatment, patients can experience severe complications including bone weakness, pain, and arterial blockages that may lead to strokes and organ failure. The conditions are particularly devastating in newborns and infants, with mortality rates exceeding 50% in severe cases. Currently, there are no FDA-approved treatments for these disorders.
INZ-701 aims to address this unmet medical need by replacing the missing enzyme and stimulating inorganic pyrophosphate production. Results from a pivotal late-stage trial in pediatric ENPP1 deficiency patients are anticipated in 2026, potentially leading to FDA approval by 2027. The drug has already completed Phase 2 testing in ABCC6 deficiency patients and a Phase 1 study for a related condition affecting blood vessel calcification in kidney failure patients.
Industry analysts have responded positively to the announcement. Leerink Partners analyst Joseph Schwartz described the acquisition as fitting “like a glove” with BioMarin’s existing business model, which includes several marketed enzyme replacement therapies such as Vimizim, Naglazyme, and Palynziq. The deal comes at a strategic time for BioMarin, following recent corporate restructuring that included workforce reductions and research program cuts.
While ENPP1 deficiency affects only approximately 10,000 patients globally, analysts believe the market opportunity could prove valuable 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.
Under the agreement terms, BioMarin will acquire Inozyme shares at $4 each, representing a substantial 180% premium over the company’s closing price of $1.42 on May 15. BioMarin appears well-positioned to finance the acquisition, with approximately $1.3 billion in cash and investments reported at the end of March 2025.
BioMarin CEO Alexander Hardy emphasized the company’s strong financial position and commitment to expanding its portfolio through both internal development and external acquisitions. The company has previously announced plans to achieve annual revenue of $4 billion by 2027.
The acquisition reflects an ongoing trend in the pharmaceutical industry of larger companies acquiring smaller biotechnology firms with promising late-stage assets. BioMarin’s established commercial infrastructure and expertise in rare diseases position it well to maximize the potential of Inozyme’s developmental therapy.
The market responded moderately positively to the announcement, with BioMarin’s shares trading up by just over 1% following the news, reaching nearly $60 per share. The transaction underscores BioMarin’s continued focus on rare disease treatments and its strategy to strengthen its pipeline through strategic acquisitions.
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