REGN Reports Encouraging Data on Gene Therapy for Genetic Hearing Loss

Core Insights - Regeneron Pharmaceuticals, Inc. (REGN) announced updated data on its investigational gene therapy DB-OTO from the CHORD study, which targets profound genetic hearing loss due to variants of the otoferlin (OTOF) gene [1][9] Group 1: DB-OTO Gene Therapy - DB-OTO is a cell-selective, dual adeno-associated virus (AAV) vector gene therapy designed to provide durable hearing to individuals with profound, congenital hearing loss caused by OTOF gene variants [2] - The CHORD study is a registrational phase I/II multicenter, open-label study evaluating the safety, tolerability, and efficacy of DB-OTO in infants, children, and adolescents with OTOF-related hearing loss, currently enrolling participants under 18 years across the U.S., U.K., Spain, and Germany [3] - The study consists of two parts: Part A involves a single intracochlear infusion of DB-OTO in one ear, while Part B involves administering DB-OTO in both ears at the selected dose from Part A [4] Group 2: Study Results - Out of 12 participants aged 10 months to 16 years, 11 experienced clinically meaningful hearing improvements, with three achieving normal hearing levels; eight participants with longer follow-up showed stability or continued improvement in hearing [5][7] - Among three participants who completed speech assessments, all showed substantial improvement, with one able to identify one- and two-syllable words without visual cues [6] - The trial met its primary endpoint, with nine participants showing hearing improvements at a threshold of ≤70 decibel hearing level (dBHL) at week 24 [6] Group 3: Regulatory and Market Context - DB-OTO has received Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations from the FDA, with the European Medicines Agency also granting Orphan Drug Designation [7] - REGN plans to submit regulatory filings for DB-OTO in the U.S. later this year, pending discussions with the FDA [8] Group 4: Portfolio Diversification - REGN is diversifying its portfolio as its lead drug Eylea faces competition from Roche's Vabysmo, which has seen significant uptake [10] - Eylea HD sales surged 29% in the second quarter due to increased demand, helping to counter the decline in Eylea sales [13] - The company's oncology franchise has been bolstered by recent FDA approvals for Libtayo and linvoseltamab-gcpt, as well as Ordspono for treating certain types of lymphoma [15][16][17]