Biomarin Pharmaceutical builds his pipeline with the acquisition of Inozyme Pharma in a $ 270 million deal targeted on an enzyme alternative remedy that might be the primary therapy permitted by the FDA for a uncommon illness that’s typically deadly for infants who had been born with it.

Situations of the acquisition settlement introduced on Friday below work for Biomarin to pay $ 4 money for every share in Boston primarily based inozyme. Though that may be a premium for the $ 1.42 closure worth on Thursday, it’s nonetheless beneath the best level of 52 weeks of the shares of $ 6.24. The inventory worth of Inozyme has largely downward pattern for the reason that IPO of 2020, priced at $ 16 per share, collected $ 112 million when the biotech ready for the clinic with Lead program Inz-701.

Inz-701 has reached section 3 exams for pyrophosphatase/phosphodiesterase 1 (Enpp1) deficiency, which is attributable to mutations to the Enpp1 gene. The Enpp1 enzyme performs a key position in processes equivalent to bone mineralization, calcification of soppy tissue and immune modulation. A deficiency of this enzyme can result in calcium construction in arteries, decreasing blood vessels and result in cardiovascular issues that may change into deadly, particularly with infants. The calcification may affect bones and joint tissue.

Inz-701, a merger protein designed to switch the Enpp1 enzyme, is run as a one-off subcutaneous injection. An open label section 3 take a look at is underway to judge the remedy in youngsters between the ages of 1 and 13 years. In its report of the primary quarter of 2025 monetary outcomes that had been launched earlier this week, Inozyme mentioned that it anticipated to finish a yr of dosage for all contributors in January; Provisional knowledge is predicted within the first quarter of 2026. Up to now, Inz-701 has been protected and effectively tolerated by sufferers. This remedy can also be in section 2 exams on ABCC6 deficiency and in section 1 exams on calciphylaxis.

The acquisition offers Inozyme a respiration house. In March the biotech introduced a strategic repeat capability. Sufferers presently handled within the ABBC6 deficiency research would nonetheless be handled, however future research on this situation and calciphylaxis can be postponed. Inozyme decreased 25% of his workforce to let his cash final within the first quarter of 2026.

In a memorandum despatched to buyers, Leerink companions analyst Joseph Schwartz mentioned that Inozyme's remedy matches in with the prevailing biomarin infrastructure. He added that the value of $ 270 million Biomarin leaves with a lot firepower left to pursue extra offers. William Blair analyst Sami Corwin acknowledged the encouraging knowledge for Inz-071 thus far, however her analysis invoice mentioned that additional work is required find eligible sufferers to provide the corporate belief that biomarin can construct on the 670 US sufferers who’ve recognized inozyme.

San Rafael, biomarin established in California, is already effectively established in enzyme alternative therapies. The primary product of the corporate was NaglazyMe, an enzyme alternative remedy permitted in 2005 for the therapy of mucopolysaccharidosis VI. The most effective product of Biomarin is Vimizim, permitted in 2014 as an enzyme alternative remedy for mucopolysaccharidosis IVA. Vimizim generated $ 739.8 million in earnings final yr. Throughout a convention name Friday, Biomarin CEO Alexander Hardy described the Inozyme remedy as 'a robust strategic match'.

“Like 5 of our six enzyme therapies, it might be a first-in-disease therapy,” mentioned Hardy, who got here to Biomarin final yr after a CEO of Genentech. “Including this analysis remedy to our enzyme remedy portfolio is a pure addition to our late stage pipeline, and, as knowledge is supported, we’ll use our know-how in authorized and commercialization in international locations around the globe to offer entry to Inz-701 to sufferers in our world footprint.”

Biomarin estimates that the whole addressable inhabitants for Inz-701 is between 2,000 and a pair of,500 sufferers, Hardy mentioned. The corporate believes that almost all of these sufferers will probably be discovered exterior the US, that the expertise of Biomarin was with Naglazyme, and the power of the corporate to establish sufferers, the marketplace for that product has develop, he mentioned. Primarily based on the present estimates of the affected person inhabitants, biomarin tasks Inz-701 can attain $ 400 million to $ 600 million in peak gross sales in opposition to the center of the 2030.

The biomarin and inozyme boards of drivers have permitted the transaction, which the businesses count on to shut within the third quarter of this yr, topic to ordinary approvals and the tendering of the vast majority of the Inozyme shares.

Photograph: Getty pictures