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FDA Fast Tracks iECURE's ECUR-506 for Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency Treatment

Wednesday, May 08, 2024

IECURE, a gene editing company focusing on mutation-agnostic in vivo gene insertion therapies for liver disorders, announced today that its ECUR-506 program received Fast Track designation from the U.S. Food and Drug Administration (FDA) for treating neonatal onset Ornithine Transcarbamylase (OTC) deficiency.

The Fast Track designation expedites the review process for therapeutics targeting serious conditions with unmet medical needs, facilitating more frequent communication with the FDA and potentially enabling accelerated approval.

"This Fast Track designation underscores the urgent need for effective treatments for neonatal onset OTC deficiency and reflects the promising preclinical data we've generated for ECUR-506," said Joe Truitt, CEO of iECURE. "These benefits may expedite the availability of ECUR-506 for physicians, which is crucial given the time-sensitive nature of this condition."

ECUR-506 has previously received Rare Pediatric Disease and Orphan Drug designations from the FDA, as well as Orphan designation from the European Commission.

IECURE's OTC-HOPE study, a Phase 1/2 trial in newborn males with genetically confirmed neonatal onset OTC deficiency, is currently enrolling participants at Great Ormond Street Hospital for Children in the United Kingdom. Additional trial sites in the United States and Australia will commence enrollment later this year. The study primarily aims to evaluate the safety and tolerability of ECUR-506 following a single dose, with secondary objectives including pharmacokinetics, efficacy, and exploratory endpoints related to disease markers, developmental milestones, and quality of life.

ECUR-506 utilizes a gene editing approach involving the delivery of two adeno-associated virus (AAV) capsids, each carrying different payloads. It consists of an ARCUS® nuclease vector targeting gene editing in the PCSK9 gene locus and a donor vector inserting the functional OTC gene. The PCSK9 site serves as the insertion point for the OTC gene, potentially enabling permanent expression of a healthy gene. ECUR-506 marks the first clinical meganuclease-based in vivo gene insertion program and the first use of ARCUS® nuclease for in vivo gene insertion in infants.

The OTC-HOPE study is a Phase 1/2 clinical trial of ECUR-506 in newborn males with neonatal onset OTC deficiency. It will assess varying doses of ECUR-506 and enroll newborn males up to seven months old with severe OTC deficiency. The primary objective is to evaluate the safety and tolerability of ECUR-506 administration, along with pharmacokinetics, efficacy, and potential effects on disease markers, developmental milestones, and quality of life.



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