In a significant move within the rare disease space, BioMarin Pharmaceutical announced Friday its intention to acquire Boston-based Inozyme Pharma in a $270 million all-cash transaction. The deal, which has received unanimous approval from both companies’ boards, is expected to be finalized between July and September 2025.
The acquisition will bring under BioMarin’s umbrella an experimental enzyme replacement therapy, INZ-701, which targets rare genetic conditions ENPP1 deficiency and ABCC6 deficiency. These
life-threatening disorders occur when the body fails to produce sufficient enzymes necessary for creating 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 dangerous artery blockages that may lead to strokes or organ failure. The conditions are particularly severe in infants and newborns, with mortality rates exceeding 50% in the most serious cases. Currently, there are no FDA-approved treatments for these conditions.
INZ-701’s development timeline shows promise, with late-stage trial results in pediatric ENPP1 deficiency patients expected in 2026, potentially leading to FDA approval by 2027. The therapy has already completed Phase 2 testing for ABCC6 deficiency and Phase 1 trials for a related blood vessel calcification condition associated with kidney failure.
Market analysts have responded positively to the announcement. Leerink Partners’ Joseph Schwartz noted that the acquisition aligns perfectly with BioMarin’s existing portfolio, which includes enzyme replacement therapies such as Vimizim, Naglazyme, and Palynziq. The deal comes at an opportune time for BioMarin, following its recent corporate restructuring that included workforce reductions and program cuts.
While ENPP1 deficiency affects only approximately 10,000 patients globally, analysts believe the market potential could be substantial. BioMarin’s established commercial infrastructure in rare diseases positions the company well to maximize the therapy’s value.
However, not all market observers share the same level of enthusiasm. RBC Capital Markets analyst Luca Issi, while acknowledging the strategic logic of the acquisition, suggested it may not significantly impact BioMarin’s overall performance.
The transaction values Inozyme shares at $4 each, representing a substantial 180% premium over the company’s closing price of $1.42 on May 15. BioMarin’s financial position appears solid, with
approximately $1.3 billion in cash and equivalents reported at the end of March 2025.
BioMarin CEO Alexander Hardy emphasized the company’s commitment to combining external and internal innovation, noting their strong financial foundation for pursuing additional acquisitions. The company maintains its ambitious goal of achieving $4 billion in annual revenue by 2027.
The market responded moderately positively to the announcement, with BioMarin’s shares trading up roughly 1% to nearly $60 following the news.
The acquisition represents a strategic expansion of BioMarin’s rare disease portfolio and demonstrates the company’s continued focus on addressing unmet medical needs through both internal development and strategic acquisitions. With BioMarin’s established expertise in commercializing rare disease treatments, the company appears well-positioned to advance INZ-701’s development and potential market introduction, should clinical trials prove successful.
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