BioMarin is expanding its focus on rare diseases with a $270-million all-cash deal to acquire Boston-based Inozyme Pharma. The move is BioMarin's first major acquisition since launching a new growth strategy last year, and its first under James Sabry, the former Roche dealmaker now leading business development for the company.
The transaction, announced Friday, will see BioMarin pay $4 per share for Inozyme, strengthening its enzyme replacement therapy portfolio — one of three pillars in its plan to reach $4 billion in annual revenue by 2027.
Friday's deal centres on Inozyme's lead investigational treatment INZ-701, currently in Phase III development for ENPP1 deficiency, a rare genetic condition affecting blood vessels, soft tissues and bones that can cause increased cardiovascular mortality and severe skeletal complications. If approved, the subcutaneous therapy would become the first treatment option for patients with the progressive disease.
Initial pivotal data from paediatric testing in the ENERGY trial is expected in early 2026, with a potential commercial launch in 2027, according to a release Friday. Inozyme recently said alongside its latest financial results that interim results from the study showed positive trends in safety, phosphate levels and immunogenicity.
Early clinical results also appear promising. In Phase I/II trials with adult ENPP1 deficiency patients, BioMarin said INZ-701 demonstrated a favourable safety profile with improvements in pyrophosphate levels, bone mineralisation biomarkers and patient-reported quality-of-life measures.
INZ-701 is being developed across populations and a continuum of phenotypes. Currently, Inozyme is enrolling infants in a pivotal trial, and a supportive study for adolescents and adults is being planned.
"Today's agreement builds on our legacy… [of] advancing enzyme therapies for children and adults living with serious genetic conditions," said BioMarin CEO Alexander Hardy. The transaction, unanimously approved by both companies' boards, is expected to close in the third quarter.
Last August, BioMarin laid off over 200 workers and narrowed the commercial focus for haemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox), which was struggling to achieve profitability.