The acquisition of inozyme of $ 270 million in biomarin brings a drug that could become the first for a rare disease
Biomarin Pharmaceutical builds its pipeline with the acquisition of Izyme Pharma in an agreement of $ 270 million centered on enzymatic replacement therapy which could become the first treatment approved by the FDA of a rare disease which is often fatal for born infants.
The terms of the acquisition agreement announced on Friday the call for Biomarin to pay $ 4 in cash for each part of ino -based Boston. Although this is a bonus in the closing price of $ 1.42 Thursday, it is still below the highest of $ 6.24. The Izozyme equity price has been on a mainly downward trend since its IPO of 2020, at a price of $ 16 per share, raised $ 112 million while biotechnology was preparing to qualify for the clinic with its main program, inz-701.
Inz-701 has reached phase 3 tests for pyrophosphatase / phosphodiesterase 1 (ENP1) deficiency, which is caused by mutations in the app1 gene. The enzyme enpp1 plays a key role in processes such as bone mineralization, calcification of soft tissues and immune modulation. The deficiency of this enzyme can lead to an accumulation of calcium in the arteries, shrink blood vessels and cause cardiovascular problems that can become fatal, especially in infants. Calcification can also affect the bones and joint tissues.
Inz-701, a fusion protein designed to replace the enzyme enPI1, is administered as subcutaneous injection once a week. A phase 3 test opened during therapy evaluation in children aged 1 to 13. In his report in the first quarter of 2025, the financial results published earlier this week, Inozyme said that he planned to finish a one -year dosage for all participants next January; Preliminary data is expected in the first quarter of 2026. To date, Inz-701 has been safe and well tolerated by patients. This therapy is also in phase 2 tests for deficiency in ABCC6 and in phase 1 tests for calciphylaxis.
The acquisition gives the inozyme a certain breathing room. In March, biotechnology announced a strategic reviritation. Patients currently in the Abbc6 deficiency trial would continue to be treated, but the future trials of this disorder and calciphylaxis would be postponed. Inozyme has reduced 25% of its workforce to make your money last in the first quarter of 2026.
In a note sent to investors, Leerink Partners’ analyst Joseph Schwartz said that inozyme therapy corresponded to the existing Biomarin infrastructure. He added that the price of $ 270 million leaves biomarin with a lot of firepower to continue more transactions. William Blair’s analyst Sami Corwin has so far recognized encouraging data for IN-Z-071, but his research note has declared that additional work to find eligible patients are necessary to give the company’s confidence that biomarin can rely on the 670 American patients that Inozyme has identified.
Biomarin, based in San Rafael, California, is already well established in enzymatic replacement therapies. The first product of the company was naglazyme, enzymatic replacement therapy approved in 2005 for the treatment of mucopolysaccharidose VI. The best biomarin product is vimizim, approved in 2014 as enzymatic replacement therapy for mucopolysaccharidose IVA. Vimizim generated $ 739.8 million in income last year. Speaking at a conference call on Friday, the CEO of Biomarin, Alexander Hardy, described inozyme therapy as “a strong strategic adjustment”.
“Like five of our six enzymatic therapies, it would be a first treatment in the disease,” said Hardy, who joined Biomarin last year after being CEO of Genentech. “The addition of this investigation therapy to our portfolio of enzymatic therapies is a natural addition to our pipeline at a late stage and, if the data is supported, we take advantage of our regulatory and commercial know-how in countries around the world to provide access to Inz-701 to patients through our global footprint.”
Biomarine estimates that the total addressable population for Inz-701 is between 2,000 and 2,500 patients, said Hardy. The company thinks that most of these patients are outside the United States who were biomarin experience with the Naglazyme, and the company’s ability to identify patients has helped develop the market for this product, he said. Based on current patient population estimates, Biomarine Inz-701 projects could reach 400 to 600 million dollars in advanced sales in the mid-2030s.
Boards of directors of Biomarin and Inozyme have approved the transaction, which companies expect to close in the third quarter of this year, subject to customary approvals and the call for tenders for the majority of inozymes.
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