Regeneron Pharmaceuticals announced updated positive data from its ongoing Phase 1/2 CHORD trial of DB-OTO, a gene therapy for children with profound genetic hearing loss caused by otoferlin gene variants. Ten of eleven children treated with DB-OTO showed marked hearing improvements, with some achieving near-normal hearing levels. The first child dosed, now 19 months old, continues to demonstrate speech and developmental progress 72 weeks after treatment.
This news holds immense potential for children born with otoferlin-related hearing loss, a condition currently without effective treatment options. The demonstrated hearing improvements, especially the progression towards normal hearing thresholds in some participants, suggest that DB-OTO could fundamentally alter the lives of these children by enabling them to access the auditory world. Furthermore, the observation of speech and developmental progress in the first treated child reinforces the potential for this therapy to facilitate normal development. This progress offers hope for improved quality of life and integration into mainstream society.
The CHORD trial includes children aged 10 months to 16 years, some treated in one ear and others in both. Improvements were measured using pure tone audiometry (PTA) and auditory brainstem responses (ABR). Several children reached near-normal or normal hearing levels (≤40 dBHL and ≤25 dBHL, respectively), with ABR results confirming the PTA findings. Importantly, the therapy and surgical procedure were well-tolerated, with only transient, resolvable vestibular side effects reported in some participants.
These results suggest a promising future for DB-OTO and potentially revolutionize the treatment of otoferlin-related hearing loss. Continued positive data from the CHORD trial could lead to regulatory approval and make this transformative therapy available to a population with a significant unmet medical need. The success of DB-OTO might also spur further research into gene therapies for other forms of genetic hearing loss, broadening the therapeutic landscape for hearing-impaired individuals.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
