Otovia Therapeutics, Inc.

Several small clinical trials have demonstrated that gene therapies have restored hearing in children with a rare form of genetic deafness. In an update at the Association for Research in Otolaryngology’s midwinter meeting in Anaheim, CA, Regeneron reported Monday that 10 of 11 children saw notable improvements in hearing after receiving its gene therapy for a form of deafness caused by mutations in the gene that encodes otoferlin. The Tarrytown, NY-based biotech also said that three of five children it followed for 24 weeks reached “nearly normal” or normal hearing levels. “One of these participants — the participant was in the shower — and they were perceiving something, and they could not understand what was happening,” Jonathon Whitton, head of Regeneron’s auditory global program, said in an interview. “What they realized was they never heard water in the shower before.” Meanwhile, Chinese researchers using a therapy developed by Otovia Therapeutics said Monday at the meeting that hearing was restored in all nine participants enrolled in an investigator-initiated otoferlin gene therapy trial, including one adult patient. Regeneron, Otovia and several other companies are working on gene therapies that treat hearing loss as a result of mutations in the gene for otoferlin, a key signaling protein between the ear and the brain. Back in May, Regeneron disclosed that two children had their hearing restored to the normal range about six months after receiving the therapy, which it gained from its 2023 acquisition of Decibel Therapeutics , where Whitton previously led clinical research. In its new update, Regeneron said it had dosed a total of 12 participants, though only 11 have had their first four-week assessment. Three participants received the gene therapy in both ears. One participant in Regeneron’s study did not experience a change in their hearing in the nearly six months following treatment. Some children in the study experienced side effects such as nausea, dizziness and vomiting from the surgery to receive the gene therapy in the days after the procedure. Whitton said the procedure is similar to the one used for cochlear implants in kids. Regeneron’s study is taking place in the US, Spain and the UK. Regeneron is talking to the FDA about the potential of using its current study to apply for approval of the gene therapy, according to Whitton. The form of deafness caused by mutations to the otoferlin gene is exceedingly rare, and Regeneron estimates there are less than 50 people per year in the US who are born with it. In an abstract published on ARO’s website, investigators at several institutions in China who are testing Otovia’s candidate described the gene therapy’s changes to hearing as “rapid” and taking “one month to restore most hearing.” They also reported that the therapy was more effective for younger participants, with those between the ages of 5 and 8 experiencing the most hearing restoration. An Otovia scientist told Endpoints News via email that the study has completed enrollment with 11 patients. Otovia was incubated by Fosun Health Capital in 2022, and the biotech said it’s “actively preparing for seed round financing to support our lead program.” The results from Otovia’s candidate follow those presented in May by a separate group of scientists in China, where researchers are able to quickly generate clinical data using investigator-initiated studies. In May, Yilai Shu and other researchers at Fudan University in Shanghai shared that 10 of 11 children who were born deaf had their hearing restored in an investigator-initiated study using a gene therapy developed by Refreshgene Therapeutics, a biotech based in the same city. Shu’s group this week also shared new analyses depicting how speech and hearing developed in children after they received the gene therapy and how outcomes from the experimental therapy compare to cochlear implants. Eli Lilly is also developing an otoferlin gene therapy and last year reported that it restored hearing in an 11-year-old boy who received the therapy. A French biotech called Sensorion is also doing work in this area. In December, it said it had dosed three patients in a clinical trial with its otoferlin gene therapy.