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Inozyme Gets $49M From Sanofi, Novo to Tackle Rare Infant Disease

Xconomy Boston — 

Sanofi and Novo Nordisk this morning have put their venture arms to work. The two joined up with two venture firms to back a $49 million round for a fledgling startup, Inozyme Pharmaceuticals. The company aims to develop a therapy for an ultra-rare infant disorder—and possibly some more prevalent diseases too.

The startup, based in Cambridge, MA, is based on the work of Yale University pathologist Demetrios Braddock. In December 2015, Braddock’s lab published a paper in Nature Communications describing a way to treat the underlying cause of a rare, deadly disease called generalized arterial calcification of infants (GACI), which has no effective therapies. Braddock, Yale pharmacology department chairman Joseph Schlessinger, and healthcare venture capitalist Axel Bolte formed Inozyme shortly thereafter, with Bolte as CEO.

GACI is a genetic disease affecting about 1 every 391,000 births, according to the National Institutes of Health. Babies GACI have a genetic mutation that results in low levels of pyrophosphatase, an enzyme that stops calcium from building up in blood vessels. As a result, babies with GACI have high blood pressure and heart problems, and often die at an early age. Those who survive can go on to develop a rare type of rickets, a disorder marked by defective bone mineralization.

There are no approved, effective treatments for GACI. Bisphosphonates—a type of osteoporosis drug—have been studied, though different studies have come to different conclusions about their effectiveness, and they can lead to other issues if taken over a long period of time. Researchers at the Children’s Hospital of Philadelphia have been testing a different treatment in which pregnant mothers use the osteoporosis drug etirdonate, for instance, while the affected baby is in utero.

Braddock’s work at Yale centered on a gene, ENPP1, which provides the instructions for making pyrophosphatase, and is mutated in patients with GACI. In the Nature Communications paper, his team injected GACI mice with a replacement version of the enzyme, which helped reduce the calcifications that characterize the disease.

Inozyme was formed to turn that work into a GACI drug, a protein that’s regularly injected into babies—and perhaps other diseases where calcifications are a major problem. “We haven’t yet decided which those could be yet. There’s quite a selection,” Bolte says. Inozyme aims to start with rare diseases, but more prevalent chronic disorders could eventually become targets as well. Chronic kidney disease, for instance, affects 31 million Americans, according to the American Kidney Fund. People with CKD are at risk of calcifications in their veins and arteries, which can lead to cardiovascular problems and death. Inozyme’s lead candidate, INZ-701, is meant to treat calcifications in the circulatory system, bones, and kidneys.

Inozyme has now secured the cash to take INZ-701 through a human proof of concept study that should begin in 2019, according to Bolte.

Longitude Capital led the funding round, and was joined by New Enterprise Associates, Novo Ventures, and Sanofi Ventures.