BioMarin Acquires Inozyme for $270M, Securing Phase 3 Prospect in Sabry's First M&A Move

20 May 2025
BioMarin, a biopharmaceutical company based in California, has announced its decision to acquire Inozyme for $4 per share, totaling approximately $270 million. This acquisition marks BioMarin's strategic shift towards external innovation as it seeks to expand its business development initiatives. BioMarin's move comes after hiring James Sabry, M.D., Ph.D., former Roche dealmaker, as chief business officer, signaling a departure from its traditional focus on early-stage deals.

Inozyme, the target company, has recently completed enrollment in a phase 3 trial of its enzyme replacement therapy, INZ-701. The therapy is aimed at treating children suffering from ENPP1 deficiency, a rare condition linked to increased cardiovascular mortality in infants. The phase 3 trial is expected to yield its topline results early next year, potentially demonstrating the effectiveness of INZ-701 in reversing pathological mineralization, including the calcification of arteries, tendons, and ligaments among affected individuals.

Despite promising interim biomarker and antibody data shared by Inozyme, the company has faced challenges in gaining investor confidence. Over the past year, its stock price has plummeted by 70%, underscoring the difficulties of public investment in the biopharma sector. However, BioMarin's decision to purchase Inozyme at more than double its closing price ahead of the acquisition announcement indicates a strong belief in the potential of INZ-701.

Douglas Treco, Ph.D., CEO of Inozyme, has acknowledged the limited visibility and awareness of ENPP1 deficiency among healthcare professionals. However, he asserts that the genetic prevalence of the rare disease is predictable and emphasizes Inozyme's efforts to identify patients and create a comprehensive registry. Treco believes that the ultimate measure of success will be the market performance of INZ-701, contingent on enhanced diagnosis and awareness.

BioMarin is well-positioned to take on the responsibility of increasing diagnoses and awareness for ENPP1 deficiency. The company has a proven track record in developing and marketing enzyme replacement therapies, with products like Aldurazyme and Naglazyme. BioMarin's acquisition of Inozyme aligns with its strategy to focus on areas where it holds a significant advantage and expertise, particularly in enzyme replacement therapies.

Alexander Hardy, CEO of BioMarin, has reiterated the company's commitment to evaluating both external and internal innovation, a sentiment he expressed during an earnings call. Hardy's statement underscores BioMarin's intention to pursue at least one business development deal annually, highlighting the company's dedication to expanding its portfolio and reinforcing its market position.

In summary, BioMarin's acquisition of Inozyme represents a pivotal move in the company's business development strategy, emphasizing external innovation and acquisition. As BioMarin prepares to integrate INZ-701 into its pipeline, it is poised to leverage its expertise in enzyme replacement therapies to drive awareness and improve diagnosis of ENPP1 deficiency, potentially transforming the landscape for patients suffering from this rare condition.

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