New data showed Regeneron’s experimental gene therapy restored hearing in nearly all children in a clinical trial, with some achieving normal hearing levels.
Shares of Regeneron Pharmaceuticals rose nearly 0.2% in premarket trading on Monday after the drugmaker said its experimental gene therapy for hearing loss helped nearly all children in a clinical trial regain their hearing, including some who achieved normal hearing levels.
The company said it plans to seek U.S. regulatory approval later this year following discussions with the Food and Drug Administration. Hearing improved in 11 of 12 children in the trial, with three reaching normal hearing. Improvements continued or remained stable among patients with longer follow-up.
Speech tests also showed encouraging progress. Three participants followed for nearly a year demonstrated significant gains in speech recognition and the ability to respond to sounds, even in noisy environments.
Lawrence Lustig, chair of otolaryngology at Columbia University and a lead investigator, said the data showed “consistent, rapid and robust responses” to the therapy, with hearing stability and continued improvement in speech over time. Families described the changes as “unimaginable” compared with a year earlier.
The one-time treatment, known as DB-OTO, delivers a working copy of the OTOF gene that is missing or defective in children with this rare form of congenital deafness. It is administered through a single infusion into the inner ear, and most participants began showing improvement within weeks.
Regeneron reported that DB-OTO was well-tolerated, with no serious side effects associated with treatment. The therapy has been granted Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations from the FDA.
DB-OTO is being developed as a one-time gene therapy for otoferlin-related hearing loss, an “ultra-rare” condition estimated to affect 20 to 50 newborns annually in the U.S., according to a statement by the company.
On Stocktwits, retail sentiment for Regeneron was ‘bullish’ amid a 250% surge in 24-hour message volume.
Regeneron’s stock has declined 20% so far in 2025.
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