BioMarin Pharmaceutical announced Friday its intention to acquire Boston-based Inozyme Pharma in a $270 million all-cash transaction, expanding its presence in the rare disease treatment space. The acquisition, which has received unanimous approval from both companies’ boards of directors, is expected to finalize between July and September 2025.
The deal will bring under BioMarin’s control 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 amounts of an enzyme necessary for creating inorganic pyrophosphate, a molecule that prevents mineral deposits from accumulating in soft tissues.
Without proper levels of this molecule, patients can experience 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, these disorders lack FDA-approved treatments.
Clinical trials for INZ-701 are progressing, with results from a pivotal late-stage study in children with ENPP1 deficiency anticipated next year. If successful, FDA approval could be secured by 2027. The therapy has already completed Phase 2 testing for ABCC6 deficiency and a Phase 1 trial 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 portfolio, which includes 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 significant commercial opportunity for BioMarin.
The strategic fit was also highlighted by Stifel analyst Paul Matteis, who emphasized BioMarin’s established commercial infrastructure and expertise in maximizing the potential of specialized therapeutics. 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 announcement comes during a period of transformation for BioMarin, which underwent workforce reductions and project streamlining last year. CEO Alexander Hardy emphasized the company’s strong financial position and commitment to pursuing both external and internal innovation to develop treatments for patients with unmet medical needs.
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. The deal follows BioMarin’s previously announced goal of achieving $4 billion in annual revenue by 2027, supported by its healthy financial position, which included
approximately $1.3 billion in cash, cash equivalents, and short-term investments as of March 2025.
BioMarin’s stock responded modestly to the announcement, trading up just over 1% to nearly $60 per share by late Friday morning. The acquisition represents a strategic move to strengthen BioMarin’s position in the rare disease market while leveraging its existing expertise in enzyme replacement therapies and commercial
infrastructure.
Be First to Comment