DB-OTO Results in the New England Journal of Medicine Showcase Dramatic and Sustained Improvements in Hearing and Speech Perception in Children with Profound Genetic Hearing Loss

Nearly all participants (11 of 12) experienced clinically meaningful hearing improvements, including three who achieved normal hearing; eight with longer follow-up showed stability or continued improvement in their hearing

Among three who completed speech assessments, all showed significant improvement with one able to identify one- and two-syllable words with no visual cues and respond to distant sounds and speech in noisy environments

Latest DB-OTO data presented at AAO-HNSF; U.S. regulatory submission planned later this year, pending discussions with the FDA

TARRYTOWN, N.Y., Oct. 12, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced updated data for their investigational gene therapy DB-OTO for profound genetic hearing loss due to variants of the otoferlin (OTOF) gene were published in The New England Journal of Medicine and presented during an oral presentation at the annual American Academy of Otolaryngology-Head and Neck Surgery (AAO-HNSF) meeting. These latest results from the pivotal CHORD trial show 11 out of 12 participants have experienced clinically meaningful hearing improvements, including 3 who achieved normal hearing levels. Additionally, eight participants with longer follow-up showed stability or continued improvement in their hearing, and among three who completed speech assessments, all showed significant improvement.

“Until now, genetic OTOF-related hearing loss was considered permanent, which is why many of us have dedicated our careers to this field,” said Lawrence R. Lustig, M.D., Chair of the Department of Otolaryngology-Head and Neck Surgery at the Columbia University College of Physicians and Surgeons and a trial investigator. “This registrational data set showcases consistent, rapid and robust responses to DB-OTO, and for those followed to later timepoints, we’ve seen hearing stability as well as continued improvement in understanding of speech. These results are even more poignant when viewed by the families – as one of the parents said, their situation is now ‘unimaginable’ from one year ago. This truly represents a new era in the treatment of hearing loss.”