Inozyme bags $67M to usher rare bone disorder drug into the clinic

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Inozyme's lead asset in IND-enabling studies and is slated to enter the clinic in 2020. (Nadine Doerlé)

Inozyme Pharma has been aiming for the clinic since it came out of stealth in 2017 and now, with a $67 million round, it’s ready to go. The funding will advance its lead program into clinical trials for rare mineralization disorders in babies and children. 

With technology licensed from Yale University, Inozyme is developing treatments for genetic disorders in which mineral imbalances cause bones to weaken and soft tissues to harden from calcium buildup. Its lead asset, INZ-701, is an enzyme replacement therapy designed to compensate for a lack of plasma pyrophosphate, which is thought to cause mineralization disorders. The drug has been shown in mouse models to ward off calcification of the heart, aorta and coronary arteries. 

The Boston-based company will move INZ-701 into the clinic for generalized arterial calcification of infancy (GACI) and a form of rickets known as autosomal recessive hypophosphatemic rickets type 2. Both are extremely rare and caused by a mutation in the ENPP1 gene. GACI has no approved treatment and typically leads to death, from heart attack or stroke during infancy. 

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INZ-701 is in IND-enabling studies and is slated to enter the clinic in 2020, the company said in a statement. 

RELATED: Sanofi, Novo back rare disease startup Inozyme in $49M round 

Inozyme has added a new research program for a rare bone disease it’s keeping quiet for now, and will also test INZ-701 in conditions caused by a deficiency in the enzyme ABCC6. 

“Similar to mutated ENPP1, mutations in the ABCC6 gene are linked to calcification disorders resulting from low pyrophosphate (PPi) and can lead to a condition known as pseudoxanthoma elasticum,” the company said in the statement. 

“This latest round of financing enables us to broaden our development pipeline and to bring INZ-701 through clinical proof of concept. We are excited to evolve our investor base by adding five highly regarded new investors who are committed to helping us achieve our goals, and we appreciate the continued support of our existing investors as Inozyme continues to grow,” said Inozyme CEO Axel Bolte in the statement. 

Last May, Inozyme hired on David Thompson to take the newly created position of chief scientific officer as it prepared to transition INZ-701 into the clinic.  

Pivotal bioVenture Partners and Sofinnova Investments led the series A2, with RA Capital Management, Cowen Healthcare Investments, and Rock Springs Capital, Longitude Capital, NEA, Novo Holdings, and Sanofi Ventures also pitching in. The round brings Inozyme’s total raised to $116 million.

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