In a significant move within the rare disease space, BioMarin Pharmaceutical announced Friday its intention to acquire Boston-based Inozyme Pharma in a $270 million all-cash transaction. The deal, which has received unanimous approval from both companies’ boards, is expected to be finalized between July and September 2025.
The acquisition positions BioMarin to expand its rare disease portfolio by gaining control of Inozyme’s experimental enzyme replacement therapy, INZ-701. This therapeutic candidate targets two rare conditions: ENPP1 deficiency and ABCC6 deficiency, both of which can have life-threatening consequences for patients.
These disorders occur when the body fails to produce sufficient amounts of an enzyme crucial for creating inorganic pyrophosphate, a molecule that prevents mineral deposits from forming in soft tissues. Without adequate 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, there are no FDA-approved treatments for these conditions. INZ-701 aims to address this unmet medical need by replacing the missing enzyme and stimulating inorganic pyrophosphate production. The therapy is progressing through clinical trials, with late-stage study results in children with ENPP1 deficiency expected in 2026,
potentially leading to FDA approval by 2027. Additionally, Phase 2 studies for ABCC6 deficiency were completed last summer, along with 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 business model, which already includes several 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 valuable commercial opportunity for BioMarin.
The transaction values Inozyme shares at $4 each, representing a substantial 180% premium over the company’s closing price of $1.42 on May 15. BioMarin, which reported approximately $1.3 billion in cash and equivalents at the end of March, views this acquisition as aligned with its strategic goals, including its target of achieving $4 billion in annual revenue by 2027.
However, not all analysts share the same level of enthusiasm. RBC Capital Markets’ Luca Issi, while acknowledging the strategic logic of the acquisition, suggested it may not significantly impact BioMarin’s overall performance.
BioMarin CEO Alexander Hardy emphasized that the company remains well-positioned financially to pursue additional assets while accelerating the development of treatments for patients with significant unmet needs. The acquisition follows BioMarin’s recent corporate restructuring, which included workforce reductions and the elimination of several drug development programs.
The deal represents a strategic step for BioMarin as it continues to strengthen its position in the rare disease market. With its established commercial infrastructure and expertise in enzyme replacement therapies, the company appears well-equipped to maximize the potential of Inozyme’s late-stage asset. The market responded positively to the announcement, with BioMarin’s shares trading up more than 1% to nearly $60 following the news.
Be First to Comment