Inozyme Pharma Inc. (INZY) Presents Preclinical Data Suggesting Utility of INZ-701 as a Potential Treatment for ABCC6 Deficiency

May 7, 2021 7:30 AM EDT

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Inozyme Pharma, Inc. (Nasdaq: INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today presented preclinical data suggesting the utility of its lead clinical development candidate, INZ-701, as a potential treatment for ABCC6 Deficiency. The data, presented at the virtual European Calcified Tissue Society Annual Congress (ECTS, May 6-8), are the first to show that an enzyme replacement therapy (ERT) increased plasma pyrophosphate (PPi) levels and reduced calcification in an animal model of ABCC6 Deficiency.

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 significant decrease in plasma 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.

“In patients with ABCC6 Deficiency, the reduced levels of PPi that lead to pathological mineralization suggest an overlap between ENPP1 and ABCC6 Deficiencies,” explained Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific Officer of Inozyme Pharma. “This supports the rationale for an enzyme replacement therapy aimed at raising PPi to treat these serious genetic disorders. The data show that INZ-701 increased plasma PPi levels and prevented abnormal calcification in an ABCC6-deficient mouse model, demonstrating its potential for treating patients with PXE, a chronic form of ABCC6 Deficiency with no approved therapeutic options.”

This study was performed in collaboration with Thomas Jefferson University. Subcutaneous administration of INZ-701 (2 and 10 mg/kg every other day for two or eight weeks) was initiated in ABCC6-deficient mice at five to six weeks of age, the time where initiation of ectopic mineralization in this model is observed. INZ-701 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. Histopathologic examination of tissue biopsies from vehicle-treated mice revealed extensive mineralization in the muzzle skin containing vibrissae, a biomarker of the mineralization process in this model. Compared to vehicle-treated mice, a quantitative calcium assay demonstrated that the amount of calcium in muzzle skin biopsies was reduced by 68% and 74% in mice receiving INZ-701 at dose levels of 2 and 10 mg/kg, respectively (p < 0.01).

“It is encouraging to see an ENPP1 enzyme replacement having an effect on tissue calcification in this PXE animal model. The patients suffering from this disease currently have no treatment options and collaborating with Inozyme to use our in-house expertise on this disease with their drug discovery efforts is exciting,” said Jouni Uitto, M.D., Ph.D., Professor and Chair of Dermatology and Cutaneous Biology, and Biochemistry and Molecular Biology, at Thomas Jefferson University. "This study will help Inozyme further characterize the therapeutic potential of INZ-701 in PXE and other manifestations of ABCC6 Deficiency, which may offer hope to patient communities that have been waiting many years for a viable treatment option.”



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