In a significant move to expand its rare disease portfolio, BioMarin Pharmaceutical announced Friday its intention to acquire Boston-based Inozyme Pharma in an all-cash transaction valued at $270 million. The acquisition, which received unanimous approval from both companies’ boards, is expected to be finalized between July and September 2025.
The deal will strengthen BioMarin’s position in the rare disease market by adding INZ-701, an experimental enzyme replacement therapy, to its pipeline. This therapeutic candidate targets two rare conditions: ENPP1 deficiency and ABCC6 deficiency. These
life-threatening disorders occur when the body fails to produce sufficient amounts of an enzyme crucial for generating inorganic pyrophosphate, a molecule that prevents mineral buildup in soft tissues.
Without adequate inorganic pyrophosphate, 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 conditions are particularly severe in newborns and infants, with mortality rates exceeding 50% in the most serious cases. Currently, there are no FDA-approved treatments for these conditions.
INZ-701’s development program includes a late-stage study in children with ENPP1 deficiency, with results anticipated in 2026. If
successful, FDA approval could come as early as 2027. The company has already completed a Phase 2 study in ABCC6 deficiency patients and a Phase 1 trial for a related condition involving blood vessel calcification in kidney failure patients.
Market analysts have responded positively to the announcement. Leerink Partners analyst Joseph Schwartz noted that the acquisition aligns perfectly with BioMarin’s existing business model, particularly given the company’s established presence in enzyme replacement therapies with products like Vimizim, Naglazyme, and Palynziq. Stifel analyst Paul Matteis emphasized BioMarin’s strong commercial infrastructure as an advantage in maximizing the potential of INZ-701.
While ENPP1 deficiency affects only approximately 10,000 patients globally, analysts believe the market opportunity could be
substantial. 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 premium of roughly 180% over Inozyme’s closing price of $1.42 on May 15. BioMarin’s financial position appears solid, with approximately $1.3 billion in cash and short-term investments as of March 2025, and the company has previously outlined plans to achieve $4 billion in annual revenue by 2027.
BioMarin CEO Alexander Hardy emphasized the company’s commitment to combining external and internal innovation, stating they remain well-positioned financially to acquire additional assets while advancing treatments for patients with unmet medical needs. This acquisition follows BioMarin’s recent corporate restructuring, which included workforce reductions and the discontinuation of several drug development programs.
The market responded positively to the announcement, with BioMarin’s shares trading up by more than 1% to nearly $60 by late Friday morning. The acquisition represents a strategic step in BioMarin’s ongoing transformation and expansion of its rare disease treatment portfolio, particularly in areas where the company’s existing expertise in enzyme replacement therapies can be leveraged
effectively.
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