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inozyme pharma Inozyme Pharma Reports Q1 2021 Financial Results and Provides Business Highlights May 12 , 2021 - Filed Clinical Trial Application for INZ - 701 for ABCC6 Deficiency in Europe ; on track to initiate Phase 1/2 clinical trial in mid - 2021- - Published peer - reviewed preclinical data supporting INZ - 701 as a potential treatment for ENPP1 Deficiency in Journal of Bone and Mineral Research - - Presented data from first - ever Burden of Illness Study for ENPP1 and ABCC6 Deficiencies at multiple medical conferences - - Cash , cash equivalents , and investments expected to enable continued operations into the fourth quarter of 2022 - BOSTON , May 12 , 2021 ( GLOBE NEWSWIRE ) -- Inozyme Pharma , Inc. ( Nasdaq : INZY ) , a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization , today reported financial results for the first quarter ended March 31 , 2021 and provided recent business highlights . " We closed 2020 with the clearance of our IND for INZ - 701 by the FDA and our CTA by the MHRA of the UK , both for ENPP1 Deficiency . Since then , we have continued to execute on our plan to deliver a potential therapeutic option for patients with ENPP1 and ABCC6 Deficiencies , " said Axel Bolte , MSc , MBA , co - founder , president , and chief executive officer of Inozyme Pharma . " We recently reported key findings from the Burden of Illness Study in patients with both ENPP1 Deficiency and ABCC6 Deficiency . Notably , we found patients in different age groups are impacted by these deficiencies in distinct ways , illuminating an age - based shift that reflects the progression of these debilitating genetic diseases . We expect site activation in the United States for our Phase 1/2 clinical trial site for ENPP1 Deficiency in June 2021 and enrollment of the first patient shortly thereafter . Subject to receiving regulatory clearance , we also expect to initiate our planned Phase 1/2 clinical trial of INZ - 701 for ABCC6 Deficiency in mid - 2021 . " Mr. Bolte continued , “ Our current cash position allows us to appropriately resource each of the key functions necessary to execute on our goals , and is expected to enable our operations into the fourth quarter of 2022. " Recent Business Highlights • Filed Clinical Trial Application for INZ - 701 for ABCC6 Deficiency in Europe - The Company expects to initiate a Phase 1/2 clinical trial in mid - 2021 and to provide preliminary safety and biomarker data by the end of 2021 . • Published data supporting INZ - 701 as a potential treatment for ENPP1 Deficiency - Peer - reviewed article in the Journal of Bone and Mineral Research shows INZ - 701 increased plasma pyrophosphate ( PPI ) levels , improved disease markers , and decreased mortality in an Enpp1 - deficient mouse model . Preclinical findings support increase of PPI levels as a predictive marker of therapeutic benefit . • Data presented on utility of INZ - 701 as a potential treatment for ABCC6 Deficiency - The Company and research collaborators from Thomas Jefferson University presented data from a preclinical study examining INZ - 701 for the potential treatment for ABCC6 Deficiency / Pseudoxanthoma elasticum ( PXE ) at multiple medical conferences . • Data presented from Burden of Illness Study for ENPP1 and ABCC6 Deficiencies – The Company presented data from the first - ever burden of illness study in patients with ENPP1 and ABCC6 Deficiencies at multiple medical conferences . Upcoming Anticipated Milestones The Company also announced the following anticipated milestones for the INZ - 701 clinical development program , subject to COVID - 19 - related restrictions : • ENPP1 Deficiency • June 2021 : Site activation for Phase 1/2 clinical trial • Mid - 2021 : Enrollment for Phase 1/2 clinical trial • Mid - 2021 : Initiate prospective natural history study • H2 2021 : Report preliminary safety and biomarker data from Phase 1/2 clinical trial • ABCC6 Deficiency • Mid - 2021 : Site activation and enrollment for Phase 1/2 clinical trial o By the End of 2021 : Report preliminary safety and biomarker data from Phase 1/2 clinical trial