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New Data Reported From Trials of Drug Developed at Yale Pathology to Treat Rare, Often Fatal Neonatal Disorder

March 01, 2023

Clinical trial data from an enzyme-therapy drug developed at Yale Pathology to treat a rare and often fatal neonatal calcification disorder were recently reported. The drug, INZ-701, was designed and validated in the laboratory of Demetrios Braddock, MD, PhD, associate professor of pathology at Yale School of Medicine.

Inozyme Pharma announced positive topline pharmacokinetic, pharmacodynamic and safety data from the ongoing Phase 1/2 clinical trials of INZ-701 in ENPP1 Deficiency and ABCC6 Deficiency. Dr. Braddock is the scientific founder of Inozyme Pharma and a member of its Scientific Advisory Board.

ENPP1 Deficiency is associated with generalized arterial calcification of infancy (GACI), a rare and often fatal neonatal calcification disorder characterized by arterial calcifications in the medium and large arteries. ABCC6 Deficiency develops when mutations in the ABCC6 gene can cause an inability to regulate pyrophosphate levels in the blood. This disorder can manifest as either GACI Type 2 or Pseudoxanthoma Elasticum (PXE).

Half of infants with GACI will die within six months of birth; it is hoped that the positive trial data will show a direct and meaningful impact on patients and their families.

The findings also noted:

  • Treatment with INZ-701 led to plasma pyrophosphate (PPi) levels returning to normal at all dose levels in GACI patients, and in the highest dosing level in PXE patients.
  • PPi levels saw rapid, significant and sustained increase in the highest dose cohort to levels similar to those seen in a healthy subjects study
  • INZ-701 was generally well-tolerated and exhibited a favorable safety profile in both trials

The findings were shared as “highly encouraging” and said they “warrant continued clinical development of INZ-701 in ENPP1 Deficiency and ABCC6 Deficiency.”

Based on the positive results, clinical trials in infants and children with GACI will begin in March 2023, and a Phase 3 pivotal study planned in infants and children with GACI for later this year.

Submitted by Terence P. Corcoran on March 02, 2023