Latest DB-OTO Results Show Dramatically Improved Hearing to Normal Levels in a Child with Profound Genetic Deafness within 24 Weeks and Initial Hearing Improvements in a Second Child at 6 Weeks

Preliminary data detailed in an ASGCT oral presentation include results for one of the youngest children in the world to receive a gene therapy for genetic deafness

Ongoing Phase 1/2 CHORD trial is currently enrolling infants and children in the U.S., UK and Spain

TARRYTOWN, N.Y., May 08, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the investigational gene therapy DB-OTO improved hearing to normal levels in one child (dosed at 11 months of age) within 24 weeks, and initial hearing improvements were observed in a second child (dosed at 4 years of age) at a 6-week assessment. Both children were born with profound genetic deafness due to variants of the otoferlin gene, and the child dosed at 11 months of age is one of the youngest in the world to receive a gene therapy for genetic deafness. The results are from the ongoing Phase 1/2 CHORD trial, which is currently enrolling infants and children and were detailed during an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) annual conference.

“The opportunity of providing the full complexity and spectrum of sound in children born with profound genetic deafness is a phenomenon I did not expect to see in my lifetime,” said Lawrence R. Lustig, M.D., Chair of Columbia University’s Department of Otolaryngology - Head & Neck Surgery and a clinical trial investigator. “These impressive results showcase the revolutionary promise of DB-OTO as a potential treatment for otoferlin-related deafness, and we are excited to see how this translates into an individual’s development, especially since early intervention is associated with better outcomes for speech development. With the DB-OTO CHORD trial now enrolling participants in sites across the U.S. and Europe, we’re part of the beginning of a new era of gene therapy research that looks to create treatment options that address the root cause of profound genetic deafness.”

In the trial, both children received a single intracochlear injection of DB-OTO in one ear. The surgical procedure leverages the same approach used for cochlear implants, which is amenable for use in young infants. Hearing improvements were assessed by pure tone audiometry (PTA) and auditory brainstem response (ABR). PTA is considered by auditory experts to be the gold standard measurement of hearing and is measured through behavioral confirmation of sound (e.g., turning head towards sound) emitted at different intensity levels (measured in decibels or dB). ABR corroborates these behavioral responses, serving as an objective confirmation of hearing function, by measuring electrical brainstem responses to sound emitted at different dBs.