The drug brought by biomarin’s $270 million inozeme acquisition could be the first drug for rare diseases

BioMarin Pharmaceutical is building a pipeline by acquiring Inozyme Pharma for $270 million, the deal focuses on enzyme replacement therapy, which could become the first rare disease approved by the FDA, which is often a rare disease that is fatal to have it.

The terms of the acquisition agreement announced Friday that they called on Biomarin to pay $4 in cash per share for Boston-based Inosyme. While this is a premium of $1.42 on Thursday, it is still below the stock’s 52-week high of $6.24. Inosym’s stock price has been on a downward trend since its 2020 IPO, with a price of $16 per share, raising $112 million as biotech is ready to promote the clinic through its leading program INZ-701.

INZ-701 has reached a 3-stage test pyrophosphatase/phosphodiesterase 1 (ENPP1) defect, which is caused by mutations to the ENPP1 gene. ENPP1 enzyme plays a key role in processes such as bone mineralization, soft tissue calcification, and immunomodulation. This lack of enzyme can cause calcium accumulation in the arteries, which shrinks blood vessels and leads to cardiovascular disease, which can become fatal, especially in infants. Calcification can also affect bones and joint tissue.

INZ-701 is a fusion protein used to replace the ENPP1 enzyme as a weekly subcutaneous injection. Inozyme said all participants are expected to complete a one-year dose in January next year, an open-label phase 3 test that is evaluating treatment for children aged 1 to 13 years. Preliminary data for the first quarter of 2026 are expected. To date, INZ-701 is safe and tolerant. The therapy is also in the Phase 2 test of ABCC6 deficiency and calcification Phase 1 test.

The acquisition makes the breathless room and the breathless room. In March, Biotech announced a strategic repositioning. Patients currently in the ABBC6 deficiency trial will continue to receive treatment, but future trials of the disease and calcium matrix will be postponed. Inoseme cut 25% of its workforce to keep cash going until the first quarter of 2026.

In a note sent to investors, Leerink Partners analyst Joseph Schwartz said Inosyme’s therapy is related to Biomarin’s existing infrastructure. He added that the $270 million price left with a lot of firepower to pursue more deals. William Blair analyst Sami Corwin has endorsed encouraging data from INZ-071 ​​so far, but her research notes say additional work is needed to discover qualified patients so that bioMarin can build strong confidence on the 670 US patients identified by Inozyme.

Biomarin, based in San Rafael, California, has been well established in enzyme replacement therapy. The company’s first product is Naglazyme, an enzyme replacement therapy approved in 2005 for the treatment of mucopolysaccharide content VI. Biomarin’s highest product is Vimizim, which was approved in 2014 as an enzyme replacement therapy with mucopolysaccharide content IVA. Last year, Vimizim’s revenue was $739.8 million. Biomarin CEO Alexander Hardy spoke on a conference call on Friday, describing the Inoseme therapy as a “strong strategic fit.”

“Like five of our six enzyme therapies, this will be a treatment for a disease,” Hardy said. “Adding this research therapies to our enzyme therapies portfolio is a natural complement to our late pipeline, and if the data is supported, we will leverage regulatory and commercialized proprietary technologies from countries around the world to provide patients with global footprint access to INZ-701.”

Biomata estimates that the total addressable population of INZ-701 is between 2,000 and 2,500 patients, Hardy said. He believes that the company believes that most patients will be found outside the U.S., outside the U.S., are biomarin’s experience at Naglazyme, and that the company’s ability to identify patients helps to develop the product’s market. According to current estimates of the patient population, INZ-701’s BioMarin project, INZ-701, can achieve peak sales of $400 million to $600 million by the mid-2030s.

Bioforest and the board of directors have approved the deal, which are expected to close in the third quarter of this year, but are subject to customary approval and tenders for most Inozyme stocks.

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