BioMarin Pharmaceutical is build up its pipeline with the acquisition of Inozyme Pharma in a $270 million deal centered on an enzyme substitute remedy that might change into the primary FDA-approved therapy for a uncommon illness that’s typically deadly to infants born with it.
Phrases of the acquisition settlement introduced Friday name for BioMarin to pay $4 in money for every share of Boston-based Inozyme. Whereas that’s a premium to the $1.42 closing worth on Thursday, it’s nonetheless under the inventory’s 52-week excessive of $6.24. Inozyme’s inventory worth has been on a principally downward pattern since its 2020 IPO, priced at $16 per share, raised $112 million because the biotech ready to advance to the clinic with lead program INZ-701.
INZ-701 has reached Part 3 testing for pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency, which is brought on by mutations to the ENPP1 gene. The ENPP1 enzyme performs a key function in processes equivalent to bone mineralization, soft-tissue calcification, and immune modulation. Deficiency of this enzyme can result in calcium buildup in arteries, narrowing the blood vessels and resulting in cardiovascular issues that may change into deadly, significantly in infants. The calcification may have an effect on bones and joint tissue.
INZ-701, a fusion protein engineered to exchange the ENPP1 enzyme, is run as a once-weekly subcutaneous injection. An open-label Part 3 check underway is evaluating the remedy in kids between the ages of 1 and 13. In its report of first quarter 2025 monetary outcomes launched earlier this week, Inozyme mentioned it expects to finish one-year dosing for all individuals subsequent January; preliminary knowledge are anticipated within the first quarter of 2026. Up to now, INZ-701 has been protected and nicely tolerated by sufferers. This remedy can be in Part 2 testing for ABCC6 deficiency and in Part 1 testing for calciphylaxis.
The acquisition offers Inozyme some respiratory room. In March, the biotech introduced a strategic reprioritization. Sufferers at present within the ABBC6 deficiency trial would proceed to be handled, however future trials on this dysfunction and calciphylaxis can be postponed. Inozyme reduce 25% of its workforce to make its money final into the primary quarter of 2026.
In a word despatched to buyers, Leerink Companions analyst Joseph Schwartz mentioned Inozyme’s remedy suits with BioMarin’s current infrastructure. He added that the $270 million worth leaves BioMarin with loads of firepower left to pursue extra offers. William Blair analyst Sami Corwin acknowledged the encouraging knowledge for INZ-071 thus far, however her analysis word mentioned further work find eligible sufferers is required to present the agency confidence that BioMarin can construct on the 670 U.S. sufferers that Inozyme has recognized.
San Rafael, California-based BioMarin is already nicely established in enzyme substitute therapies. The corporate’s first product was Naglazyme, an enzyme substitute remedy authorized in 2005 for treating mucopolysaccharidosis VI. BioMarin’s prime product is Vimizim, authorized in 2014 as an enzyme substitute remedy for mucopolysaccharidosis IVA. Vimizim generated $739.8 million in income final yr. Talking 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 could be a first-in-disease therapy,” mentioned Hardy, who joined BioMarin final yr after serving as CEO of Genentech. “Including this investigational remedy to our enzyme therapies portfolio is a pure addition to our late-stage pipeline, and, if knowledge supported, we’ll leverage our regulatory and commercialization know-how in nations internationally to offer entry to INZ-701 to sufferers throughout our international footprint.”
BioMarin estimates the entire addressable inhabitants for INZ-701 is between 2,000 and a pair of,500 sufferers, Hardy mentioned. The corporate believes most of these sufferers will probably be discovered outdoors of the U.S. That was BioMarin’s expertise with Naglazyme, and the corporate’s skill to determine sufferers helped develop the marketplace for that product, he mentioned. Primarily based on present estimates of the affected person inhabitants, BioMarin initiatives INZ-701 might obtain $400 million to $600 million in peak gross sales by the mid-2030s.
The BioMarin and Inozyme boards of administrators have authorized the transaction, which the businesses count on to shut within the third quarter of this yr, topic to customary approvals and the tender of nearly all of Inozyme shares.
Picture: Getty Photos
