An 11-year-old boy who received Eli Lilly’s experimental gene therapy for hearing loss last October showed signs of restored hearing 30 days after treatment, the pharma giant announced on Tuesday.
The therapy, dubbed AK-OTOF, is designed to deliver a functional copy of a gene called otoferlin. Without otoferlin, auditory signals received by the ear aren’t transmitted to the brain. Lilly estimates that 200,000 people worldwide experience hearing loss caused by mutations to otoferlin.
The company is testing AK-OTOF in a Phase I/II trial enrolling 14 patients, according to the clinical trials registry. The first child dosed, an 11-year-old born with hearing loss, achieved restored hearing “across all tested frequencies, achieving thresholds of 65 to 20 dB HL,” including results within normal hearing range at some frequencies, according to Lilly.
Lilly said it will release more details and additional data from a second patient at this year’s Association for Research in Otolaryngology in February. The company said both the therapy and the surgical procedure were well-tolerated with “no serious adverse events.”
AK-OTOF was the lead candidate at Akouos before it was bought by Eli Lilly in late 2022 for $487 million, plus $123 million in contingent value rights.
Regeneron read out results in October suggesting its rival gene therapy DB-OTO led to improved auditory responses in a child under 2 years old through 6 weeks. The therapy is in an early-stage study in the US, Spain and the UK.
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