Latest DB-OTO Results Demonstrate Clinically Meaningful Hearing Improvements in Nearly All Children with Profound Genetic Hearing Loss in CHORD Trial

Core Insights - Regeneron Pharmaceuticals announced updated data for its investigational gene therapy DB-OTO, showing significant improvements in hearing among children with profound genetic hearing loss due to otoferlin gene variants [1][6][12] - The Phase 1/2 CHORD trial demonstrated that 10 out of 11 children assessed post-treatment exhibited notable hearing improvements, with some achieving near-normal hearing levels [3][4] Group 1: Trial Results - The first participant in the trial showed improvement in hearing to near-normal levels across key speech frequencies, with positive auditory brainstem responses corroborating these findings [3] - Among the 11 participants with at least one post-treatment assessment, 10 demonstrated improved hearing at various decibel levels, with three out of five participants showing significant improvements in average hearing thresholds [4] - The first child treated at 10 months of age exhibited progress in speech and development, highlighting the therapy's potential impact on quality of life [1][2] Group 2: Safety and Tolerability - The surgical procedure for administering DB-OTO was well tolerated, with no serious adverse events related to the therapy reported [5] - Five participants experienced transient vestibular adverse events post-surgery, which resolved within six days [5] Group 3: Regulatory Designations - DB-OTO has received multiple designations from the U.S. FDA, including Orphan Drug and Fast Track designations, indicating its potential significance in treating rare pediatric diseases [6] - The investigational therapy is currently under clinical investigation, with its safety and efficacy not yet evaluated by regulatory authorities [6] Group 4: Background on Hearing Loss - Congenital deafness affects approximately 1.7 out of every 1,000 children born in the U.S., with genetic causes accounting for about half of these cases [7] - Otoferlin-related hearing loss is ultra-rare and results from variants in the OTOF gene, leading to a lack of functional otoferlin protein essential for auditory nerve communication [7] Group 5: CHORD Trial Overview - The CHORD trial is a Phase 1/2, multicenter, open-label study evaluating the safety, tolerability, and preliminary efficacy of DB-OTO in children with otoferlin variants [8][9] - The trial is currently enrolling participants across the U.S., U.K., and Spain, with a focus on infants, children, and adolescents [9]