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 - Seite 2

At baseline, both participants had no behavioral (PTA) or electrophysiological (ABR) responses at maximum sound levels (≥100 dB). Following treatment with DB-OTO, both children showed auditory responses at the first efficacy assessment of 4 weeks.

As presented at ASGCT, the first participant dosed in the trial was 16 months of age at the 24-week assessment and showed:

• Improvement of hearing to normal levels across key speech frequencies, with an average 84 dB improvement from baseline and one frequency measure reaching 10 dB in hearing level per PTA. Across all tested frequencies, an average 80 dB improvement from baseline was observed.
• Positive ABR responses, with best frequency reaching 45 dB.

The second participant dosed in the trial was 4 years of age at the 6-week assessment and experienced consistent results to the first participant at the same timepoint, including:

• Initial improvement of hearing with responses to loud sounds, which was observed across key speech frequencies, with an average 19 dB improvement from baseline and one frequency measure reaching 80 dB in hearing level per PTA. Across all tested frequencies, an average 16 dB improvement from baseline was observed.
• Positive ABR responses, with best frequency reaching 75 dB.

Both the surgical procedure (delivery and post-operation) and DB-OTO were well tolerated, and there were no related adverse events or serious adverse events following treatment.

DB-OTO received Orphan Drug, Rare Pediatric Disease and Fast Track Designations from the U.S. Food and Drug Administration and Orphan Drug Designation was granted by the European Medicines Agency. The potential use of DB-OTO for otoferlin-related hearing loss is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority.

About Otoferlin-related Hearing Loss
Congenital deafness (hearing loss present at birth) is a significant unmet medical need that affects approximately 1.7 out of every 1,000 children born in the U.S. Although approximately half of these cases have genetic causes, otoferlin-related hearing loss is ultra-rare. This specific condition is caused by variants in the otoferlin gene, which impairs the production of the OTOF protein that is critical for the communication between the sensory cells of the inner ear and the auditory nerve. While hearing aids and cochlear implants can amplify sound to improve hearing for individuals with a range of hearing loss, these devices do not currently restore the full spectrum of sound.