Akouos, a wholly owned subsidiary of Eli Lilly and Company, announced positive initial clinical results from the Phase 1/2 AK-OTOF-101 study, which demonstrated pharmacologic within 30 days of AK-OTOF administration in the first participant, an individual with a decade-plus history of profound hearing loss.
Results, including initial data from a second participant to receive AK-OTOF, will be presented during the Late Breaking Presidential Symposium at the 2024 Association for Research in Otolaryngology (ARO) MidWinter Meeting.
“Gene therapy for hearing loss is something physicians and scientists around the world have been working toward for over 20 years,” said Professor John Germiller, M.D., Ph.D., attending surgeon and Director of Clinical Research in the Division of Otolaryngology at Children’s Hospital of Philadelphia, and a principal investigator of the AK-OTOF-101 Clinical Trial who administered AK-OTOF to this participant. “These initial results show that it may restore hearing better than many thought possible.”
“Children with OTOF-mediated hearing loss are often born with profound hearing loss, yet only a small fraction have undergone genetic testing to receive a definitive diagnosis,” said Dr. Oliver Haag otolaryngologist, Head of Otolaryngology at Barcelona Children’s Hospital Sant Joan de Déu, and an investigator in Akouos’s AK-OTOF-NHS-002 Natural History Study in which the first individual to receive AK-OTOF was participating. “The AK-OTOF-101 Clinical Trial and AK-OTOF-NHS-002 Natural History Study demonstrate the power of international collaboration in the development of new medicines for rare genetic conditions. It is gratifying to see this collaborative effort provide benefit to the first participant to receive AK-OTOF.”
Hearing loss is the most common sensory condition, and with no approved pharmacologic treatments to restore hearing, represents a significant area of unmet need in medicine. Millions of individuals worldwide have disabling hearing loss because one of their genes generates an incorrect or incomplete version of a protein the ear requires for hearing.
“We are grateful to the participants, their families, the investigators and other collaborators who are working together with us on this pioneering trial,” said Manny Simons, Ph.D., M.B.A., CEO of Akouos and SVP, Gene Therapy at Lilly. “These initial results highlight the potential impact genetic medicines could have on individuals with OTOF-mediated hearing loss and reinforce our mission to make healthy hearing available to all.”
👉 Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at the Association for Research in Otolaryngology 2024 MidWinter Meeting | Eli Lilly and Company
