Inozyme Pharma Acquisition Finalized: Strategic Focus Shifts to Clinical Development

The acquisition of Inozyme Pharma Inc by BioMarin Pharmaceutical was formally completed in July, marking a significant consolidation in the rare disease therapeutics sector. The transaction, valued at $270 million, provided shareholders with a final payout of $4.00 per share. All attention has now shifted to the future development of the flagship asset, BMN 401.

Strategic Rationale and Financial Terms

BioMarin Pharmaceutical acquired all outstanding shares of Inozyme Pharma at a fixed price of $4.00 per share. The strategic nature of this move was recently underscored during BioMarin’s presentation at the Cantor Global Healthcare Conference. Company executives confirmed that the continued advancement of BMN 401—previously known as INZ-701—is now a central priority.

Gregory Friberg, Executive Vice President at BioMarin, characterized the therapy as a “first-in-class and first-in-disease solution” for ENPP1 Deficiency. This rare genetic disorder can lead to severe health complications in infants and children.

Upcoming Clinical Milestones

A pivotal moment for the program is approaching. BioMarin confirmed that the ENERGY-3 study, which is evaluating the therapy in pediatric patients, is expected to report topline data in the first half of next year. This timeline aligns with previous communications from Inozyme.

Notably, patient recruitment for the ENERGY-3 trial was already completed in January 2025. The dosing phase of the study is scheduled to conclude in January 2026, with comprehensive data readouts anticipated in the first quarter of that same year.

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Pre-Acquisition Strategic Moves

Even before the acquisition was finalized, Inozyme had been strategically channeling its resources toward its ENPP1 Deficiency program. In a move to extend its financial runway into the first quarter of 2026, the company implemented a workforce reduction of approximately 25% in March 2025.

The acquisition by BioMarin effectively paves the way for INZ-701’s development under a new name, BMN 401. It leverages BioMarin’s established expertise and commercial infrastructure in bringing enzyme replacement therapies for rare genetic conditions to market. The transaction, which received unanimous approval from the boards of both companies, highlights the significant potential of INZ-701 to address a serious unmet medical need.

Implications for Shareholders

With the acquisition complete, Inozyme Pharma no longer operates as an independent public entity, and its stock has been delisted from public exchanges. The $4.00 per share acquisition price represents the final valuation for former shareholders.

The ongoing integration and subsequent development of BMN 401 under BioMarin’s stewardship will be closely monitored by the biotechnology investment community. BioMarin is currently projecting a potential regulatory approval for BMN 401 in 2027.