03:57 AM EDT, 10/13/2025 (MT Newswires) -- Regeneron Pharmaceuticals ( REGN ) said Sunday that 11 out of 12 children with profound genetic hearing loss who were treated with the DB-OTO investigational gene therapy showed "clinically meaningful" improvements in hearing during a phase 1/2 clinical that met its primary endpoint.

Almost all the patients responded within weeks of treatment and three of the 12 achieved normal hearing levels, the company said.

"This truly represents a new era in the treatment of hearing loss," said Lawrence R. Lustig, one of the trial investigators.

Regeneron said it plans a US regulatory submission for DB-OTO in 2025, pending discussions with the US Food and Drug Administration.

Among all 12 participants, both DB-OTO and the associated surgical procedure were well tolerated, though two participants experienced serious unrelated adverse events, the company said.