BioMarin Acquires Inozyme for $270M, Expanding Pipeline with Phase 3 Enzyme Replacement Therapy

BioMarin Pharmaceutical Inc. has made a significant move in its business development strategy, announcing the acquisition of Inozyme Pharma Inc. for $270 million. This deal marks the first major acquisition under the leadership of James Sabry, BioMarin's chief business officer, and represents a shift towards external innovation for the California-based biopharmaceutical company.

Strategic Acquisition Aligns with BioMarin's Core Strengths

The acquisition of Inozyme brings a promising phase 3 enzyme replacement therapy, INZ-701, into BioMarin's pipeline. This aligns closely with BioMarin's historical expertise in developing and commercializing enzyme replacement therapies, such as Aldurazyme and Naglazyme.

BioMarin CEO Alexander Hardy emphasized the strategic nature of the acquisition, stating that the company will "continue to evaluate external innovation alongside internal innovation." This move is consistent with BioMarin's stated goal of pursuing opportunities in areas where it has "a strength and a right to win."

INZ-701: A Potential Breakthrough for ENPP1 Deficiency

Inozyme's lead candidate, INZ-701, is currently in phase 3 clinical trials for the treatment of ENPP1 deficiency, a rare genetic disorder associated with increased cardiovascular mortality risk in infants. The therapy aims to address the underlying cause of the disease by replacing the missing or mutated enzyme.

Enrollment for the phase 3 trial of INZ-701 was completed in January, with topline data expected early next year. The treatment has shown promise in reversing the effects of pathologic mineralization, including calcification of arteries, tendons, and ligaments.

Challenges and Opportunities in Rare Disease Market

Despite the potential of INZ-701, Inozyme has faced challenges in the public market, with its share price declining 70% in the year leading up to the acquisition. Douglas Treco, Ph.D., Inozyme's CEO, acknowledged the low visibility and awareness of ENPP1 deficiency among physicians but highlighted the "predictive genetic prevalence" of the rare disease.

BioMarin's acquisition at $4 per share, more than double Inozyme's closing price before the announcement, suggests confidence in the therapy's potential. The company's extensive experience in rare disease markets positions it well to address the challenges of diagnosis and awareness that Treco identified as crucial for the success of INZ-701.