In a significant advancement for medical science and hearing loss treatment, Akouos, Inc., a wholly owned subsidiary of Eli Lilly and Company (NYSE: LLY), has unveiled encouraging initial clinical results from its Phase 1/2 AK-OTOF-101 study. This groundbreaking research, which demonstrated pharmacologic hearing restoration within 30 days of AK-OTOF administration in the first participant, marks a pivotal moment in the pursuit of gene therapy for hearing loss. The participant, an individual with a decade-plus history of profound hearing loss, experienced a remarkable improvement in hearing across all tested frequencies. These promising findings are set to be presented at the 2024 Association for Research in Otolaryngology (ARO) MidWinter Meeting during the Late Breaking Presidential Symposium.

The AK-OTOF-101 study represents a leap forward in the field of otolaryngology, focusing on participants with OTOF-mediated hearing loss, a genetic condition that has been largely unaddressed by current medical treatments. The study’s first participant, an 11-year-old with profound hearing loss from birth, achieved hearing thresholds within the normal range at some frequencies, showcasing the transformative potential of AK-OTOF. The surgical procedure and investigational therapy were well tolerated, with no serious adverse events reported, underscoring the safety and efficacy of this novel approach.

Professor John Germiller, an 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, emphasized the significance of these results. He noted that gene therapy for hearing loss has been a goal for over two decades, and the initial outcomes suggest a level of restoration previously thought impossible.

The AK-OTOF-101 trial involves a single, unilateral intracochlear administration of AK-OTOF, with hearing restoration assessed by behavioral audiometry and auditory brainstem response (ABR). This method provides a clinically accepted and objective measure of hearing sensitivity. Participants receive AK-OTOF at a dose designed to optimize safety and effectiveness, with the hope of restoring high-acuity physiologic hearing.

Dr. Oliver Haag, a pediatric otolaryngologist and Head of Otolaryngology at Sant Joan de Deu Hospital in Barcelona, highlighted the importance of genetic testing in diagnosing OTOF-mediated hearing loss. He praised the international collaboration that made the AK-OTOF-101 Clinical Trial and AK-OTOF-NHS-002 Natural History Study possible, offering hope to those affected by this rare genetic condition.

Hearing loss is the most common sensory condition globally, affecting millions with disabling consequences. The AK-OTOF gene therapy, developed by Akouos, aims to deliver a healthy version of the gene responsible for auditory function directly to target cells within the inner ear. This innovative treatment has the potential not only to restore hearing but also to enable high-acuity physiologic hearing for those with OTOF-mediated hearing loss.

Emmanuel Simons, CEO of Akouos and SVP, Gene Therapy at Lilly, expressed gratitude to the participants, their families, and the researchers involved in this pioneering trial. He highlighted the potential impact of genetic medicines on individuals with OTOF-mediated hearing loss and reinforced Akouos’s mission to make healthy hearing accessible to all.

The initial results from the AK-OTOF-101 study by Akouos, Inc. represent a monumental step in the treatment of hearing loss through gene therapy. This innovative approach, which has shown promising outcomes in restoring hearing in individuals with profound hearing loss, could redefine the management of sensory conditions. The well-tolerated surgical procedure and the investigational therapy’s efficacy highlight the potential of AK-OTOF as a safe and effective treatment option. As this research progresses, it could pave the way for addressing a wide range of genetic hearing loss conditions, offering new hope to millions affected worldwide.

  AK-OTOF, Akouos, Dr. Oliver Haag, Eli Lilly and Company, Emmanuel Simons, hearing restoration, John Germiller, Lilly