Sarepta Therapeutics Submits Clinical Trial Application For SRP-1005-101 For Huntington's Disease Treatment To New Zealand Medicines And Medical Devices Safety Authority; Intends To Start Trial Of SRP-1005 In Q2

Benzinga · 4d ago

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the submission of its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT, to Medsafe, the New Zealand Medicines and Medical Devices Safety Authority. Pending approval, Sarepta expects to initiate this first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026. SRP-1005 is an investigational small interfering RNA (siRNA) therapeutic for the treatment of Huntington's Disease.

INSIGHTT is a Phase 1, multi-center, dose escalation study that will evaluate the safety and tolerability of subcutaneous dosing of SRP-1005 in approximately 24 participants. SRP-1005 leverages an advanced TfR1 (transferrin receptor protein 1) approach that uses monovalent fragment antigen binding (fAb) designed for efficient delivery to the central nervous system. Subcutaneous delivery aims to allow the therapy to stay below transferrin saturation and achieve constant and robust penetration across the blood brain barrier. Preclinical data with SRP-1005 have demonstrated the potential for significant protein knockdown in key deep brain regions, including the putamen and caudate, as well as the temporal and frontal cortexes.