BioMarin Expands Rare Disease Portfolio with $270M Acquisition of Inozyme Pharma

BioMarin Pharmaceutical has announced its acquisition of Inozyme Pharma, a Boston-based rare disease specialist, for $270 million in an all-cash deal. The acquisition, expected to close in the third quarter of 2025, will bolster BioMarin's enzyme therapy portfolio and expand its presence in the rare disease market.

Strategic Acquisition Strengthens BioMarin's Enzyme Expertise

The centerpiece of the acquisition is Inozyme's lead candidate, INZ-701, an investigational enzyme replacement therapy currently in late-stage development for ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency. This rare and progressive disease, caused by mutations in the ENPP1 gene, can affect multiple organs and presents differently in each patient.

INZ-701 is designed to replace the deficient ENPP1 enzyme, potentially addressing a significant unmet medical need. The therapy combines the active site of the ENPP1 enzyme with a specific fragment of a human antibody, allowing for distribution throughout the body. Preclinical data have shown promise in preventing abnormal mineralization associated with the disease.

BioMarin CEO Alexander Hardy stated, "As BioMarin continues our transformation and delivers on our corporate strategy, we will continue to evaluate external innovation alongside internal innovation. We are in a strong financial position to bring in additional assets as we accelerate the development of medicines for patients with significant unmet need."

Expanding Treatment Pipeline for Rare Diseases

In addition to ENPP1 deficiency, Inozyme is also testing INZ-701 for ABCC6 deficiency and calcific uremic arteriopathy, both rare diseases with no currently approved treatments. The acquisition aligns with BioMarin's existing portfolio of enzyme replacement therapies, including Vimizim, Naglazyme, and Palynziq.

Results from a late-stage study of INZ-701 in children with ENPP1 deficiency are expected next year, with potential FDA approval as early as 2027. A Phase 2 study in patients with ABCC6 deficiency and a Phase 1 trial for a related blood vessel-calcifying disease have already been completed.

Financial Implications and Market Outlook

BioMarin will pay $4 for each outstanding share of Inozyme, representing a premium of approximately 180% over Inozyme's closing price on May 15, 2025. The deal has received unanimous approval from both companies' boards of directors.

The acquisition comes after a period of restructuring for BioMarin, which included workforce reductions and the discontinuation of several drug projects in the previous year. However, the company reported strong first-quarter earnings for 2025, with total revenues of $745 million, representing a 15% year-on-year increase.

While analysts from Leerink Partners and Stifel view the acquisition positively, describing it as a "step in the right direction" and a "very strong strategic fit," others, such as RBC Capital Markets, suggest that the deal is "unlikely to move the needle" for BioMarin as a whole.

As BioMarin integrates Inozyme's assets and expertise, the company aims to leverage its commercial infrastructure and rare disease experience to maximize the value of INZ-701 and other pipeline candidates. The acquisition reinforces BioMarin's commitment to addressing unmet needs in the rare disease space and supports its goal of reaching $4 billion in annual revenue by 2027.