Biomarin Pharmaceutical Inc. has announced its acquisition of Inozyme Pharma, Inc. for approximately $270 million. This strategic move is expected to further strengthen Biomarin's enzyme therapies portfolio, with the addition of Inozyme's late-stage enzyme replacement therapy, inz-701. This therapy is currently being evaluated for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency, a rare genetic condition affecting blood vessels, soft tissues, and bones. Biomarin aims to leverage this acquisition to potentially launch inz-701 in 2027, following the anticipated release of pivotal data in early 2026.
Inz-701 is a potential first-in-class, subcutaneous enzyme replacement therapy being developed for infants, pediatric, and adult patients with ENPP1 deficiency. In a phase 1/2 study of adults with ENPP1 deficiency, inz-701 demonstrated a favorable safety profile and showed improvements in pyrophosphate levels, bone mineralization biomarkers, and quality of life.
The terms of the acquisition involve Biomarin commencing a cash tender offer to acquire all outstanding shares of Inozyme common stock at a price of $4.00 per share. Inozyme's board of directors has unanimously recommended that stockholders tender their shares in the offer.
Biomarin also reaffirmed its previously provided full-year 2025 financial guidance, excluding the impact of the accounting treatment of this transaction, as well as its plan to achieve a 40% non-GAAP operating margin in 2026.
This acquisition reflects Biomarin's commitment to advancing enzyme therapies for individuals with serious genetic conditions, with the company's president and CEO, Alexander Hardy, emphasizing the potential of inz-701 to be the first treatment for children and adults with ENPP1 deficiency. Inozyme's CEO, Douglas A. Treco, expressed gratitude to the team and partners for their work on this potentially life-changing therapy and highlighted Biomarin's successful track record in launching first-in-disease enzyme therapies.
Biomarin's acquisition of Inozyme aligns with its mission of translating the promise of genetic discovery into impactful medicines. The company has a history of innovation, with eight commercial therapies and a robust clinical and preclinical pipeline.
Inozyme Pharma, a clinical-stage biopharmaceutical company dedicated to developing therapeutics targeting the PPI-adenosine pathway, will bring its lead investigational therapy, inz-701, to Biomarin's portfolio. In addition to ENPP1 deficiency, inz-701 has the potential to expand into additional indications where deficiencies in the pyrophosphate-adenosine pathway contribute to disease pathology.
Biomarin's strategic move to acquire Inozyme Pharma reflects its commitment to addressing unmet medical needs and advancing innovative therapies for patients with genetically defined conditions. Following these announcements, the company's shares moved 1.96%, and are now trading at a price of $59.41. Check out the company's full 8-K submission here.
