Benzinga | Jun 9, 2021 07:31AM EDT

Inozyme Announces Acceptance of First European Clinical Trial Application for Phase 1/2 Clinical Trial of INZ-701 in ABCC6 Deficiency

Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced the acceptance of its Clinical Trial Application (CTA) from the National Agency for the Safety of Medicines and Health Products (ANSM) in France to allow initiation of its Phase 1/2 clinical trial of INZ-701, as a potential treatment for ABCC6 Deficiency. Inozyme plans to initiate its Phase 1/2 trial in mid-2021. This CTA was submitted and accepted as part of ANSM's Fast Track procedure designed to reduce processing times for clinical trial authorization requests for innovative medical products.

ABCC6 Deficiency is a rare, inherited disorder that can present as generalized arterial calcification of infancy (GACI) type 2 in infants and as pseudoxanthoma elasticum (PXE) in children and adults. This is one of several disorders with a significant decrease in plasma pyrophosphate (PPi) levels, a potent regulator of mineralization. In patients with ABCC6 Deficiency, the abnormal calcification caused by low PPi can result in vision loss and life-threatening cardiovascular complications, among other morbidities. There is no approved treatment for ABCC6 Deficiency.

"The acceptance of this CTA marks another important regulatory milestone for Inozyme and a key advancement for INZ-701 as a potential treatment for people living with ABCC6 Deficiency," said Axel Bolte, MSc, MBA, co-founder, president, and chief executive officer of Inozyme Pharma. "We are well-positioned to execute on our planned clinical study and the notable level of interest from the ABCC6 Deficiency community underscores the urgent need for therapeutic options. I want to express my thanks to the team at Inozyme and our external collaborators, all of whom have been instrumental in our continued progress."

INZ-701 is an ENPP1 enzyme replacement therapy (ERT) 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 PPi and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time correcting bone abnormalities in Enpp1-deficient mice. In Abcc6-deficient mice, subcutaneous administration of INZ-701 (2 and 10 mg/kg every other day for two or eight weeks) led to a dose-dependent increase in plasma PPi levels at both two and eight weeks after initiation of treatment, leading to significantly lower levels of soft tissue mineralization.