In a significant move within the rare disease drug development space, BioMarin Pharmaceutical announced Friday its intention to acquire Boston-based Inozyme Pharma for $270 million in an 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 conditions known as ENPP1 and ABCC6 deficiencies. These life-threatening disorders 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.
Patients suffering from these conditions face severe health
complications, including bone weakness, pain, and dangerous artery blockages that can result in strokes, tissue death, or multiple organ failure. The mortality rate exceeds 50% in newborns and infants with severe forms of these deficiencies. Currently, there are no
FDA-approved treatments for these conditions.
INZ-701’s development timeline suggests potential FDA approval could come as early as 2027, with late-stage study results in children with ENPP1 deficiency expected next year. The therapy has already completed Phase 2 studies for ABCC6 deficiency and Phase 1 trials for a related blood vessel calcification condition associated with kidney failure.
Industry analysts have responded positively to the acquisition. Leerink Partners’ Joseph Schwartz noted that the deal “fits like a glove” with BioMarin’s existing business model, which includes other enzyme replacement therapies such as Vimizim, Naglazyme, and Palynziq. While ENPP1 deficiency affects only approximately 10,000 patients globally, analysts believe the treatment area could represent a valuable commercial opportunity for BioMarin.
The transaction arrives at a strategic time for BioMarin, following a period of corporate restructuring that included workforce reductions and project consolidation last year. CEO Alexander Hardy emphasized the company’s strong financial position and commitment to pursuing both external and internal innovation opportunities.
Under the agreement terms, 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 appears solid, with approximately $1.3 billion in cash and investments reported at the end of March, and projections targeting $4 billion in annual revenue by 2027.
However, not all market observers share equal enthusiasm about the deal’s impact. RBC Capital Markets analyst Luca Issi, while
acknowledging the strategic logic of the acquisition, suggested it may not significantly influence BioMarin’s overall performance.
Stifel analyst Paul Matteis highlighted BioMarin’s established commercial infrastructure as a key advantage in maximizing the potential of INZ-701, describing the acquisition as a “very strong strategic fit” for the company’s existing operations.
The market’s initial response was modest but positive, with BioMarin’s shares trading up by just over 1% to nearly $60 following the announcement. The deal represents a significant step in BioMarin’s ongoing transformation and demonstrates the company’s commitment to expanding its rare disease treatment portfolio through strategic acquisitions.
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