Regeneron Pharmaceuticals, Inc. (REGN), a biotechnology company, announced updated results from the Phase 1/2 CHORD trial of DB-OTO, an investigational gene therapy designed to treat profound genetic hearing loss caused by variants of the otoferlin or OTOF gene.
This trial is evaluating the safety, tolerability, and preliminary efficacy of DB-OTO in children with otoferlin-related hearing loss which includes 12 participants aged 10 months to 16 years, with some receiving DB-OTO in one ear and others bilaterally.
The latest data shows that 10 out of 11 children with at least one post-treatment assessment demonstrated significant improvements in hearing.
The trial results showed near-normal hearing levels and positive speech perception in some participants, with no significant safety concerns related to DB-OTO.
Regeneron remains focused on advancing DB-OTO as a potential treatment for otoferlin-related hearing loss, and the therapy has received Orphan Drug and Rare Pediatric Disease designations from the U.S. FDA.
REGN closed Monday's (Feb.24 2025) trading at $713.25 up by 1.84 percent. In premarket trading Tuesday the stock is down by 0.17 percent at $712.01 on the Nasdaq.
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