Inozyme Pharma Raises $67 Million to Address Rare Mineralization Disorders

Inozyme Pharma said it completed a $67 million venture round to advance its lead INZ-701 into clinical development for severe disorders of calcification associated with deficiencies in the enzyme ENPP1, including Generalized Arterial Calcification of Infancy (GACI) and autosomal recessive hypophosphatemic rickets type 2 (ARHR2).

Photo: Axel Bolte, CEO and co-founder of Inozyme Pharma

Both GACI and ARHR2 are disorders that result from mutations in the ENPP1 gene, leading to ENPP1 deficiency.

INZ-701 is an enzyme replacement therapy in development for the treatment of mineralization disorders of the circulatory system, bones, and kidneys. In preclinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time normalizing bone mineralization.

Pivotal bioVenture Partners and Sofinnova Investments led the Series A2 round. Cross-over investors, including RA Capital Management, Cowen Healthcare Investments, and Rock Springs Capital, as well as the company’s previous investors, Longitude Capital, NEA, Novo Holdings, and Sanofi Ventures, also joined this round.

Inozyme also announced that it is expanding the INZ-701 development program by adding ABCC6 deficiency as a second indication. Similar to mutated ENPP1, mutations in the ABCC6 gene are linked to calcification disorders resulting from low pyrophosphate and can lead to a condition known as pseudoxanthoma elasticum (PXE). The company has also broadened its pipeline by adding a second research program targeting an undisclosed rare bone disease.

“With the addition of a second indication for INZ-701 and a second rare bone disease program, we are expanding our footprint in these underserved disorders,” said Axel Bolte, CEO and co-founder of Inozyme Pharma. “This latest round of financing enables us to broaden our development pipeline and to bring INZ-701 through clinical proof of concept.”

The company said it has already demonstrated preclinical proof of concept for INZ-701 in GACI and ARHR2. The company is currently completing the IND-enabling studies for INZ-701 and expects to initiate clinical trials in 2020.

Inozyme also announced the addition of Rob Hopfner, managing partner of Pivotal bioVenture Partners, to its Board of Directors, along with Sarah Bhagat, principal at Sofinnova Investments.