Akouos Says FDA Clears IND Application For AK-OTOF For OTOF-mediated Hearing Loss

(RTTNews) - Genetic medicine company Akouos, Inc. (AKUS) announced Tuesday that it has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 1/2, first in human, pediatric clinical trial of AK-OTOF, a gene therapy intended for the treatment of patients with otoferlin gene (OTOF)-mediated hearing loss.

Currently, there are no approved pharmacologic treatment options for individuals with OTOF-mediated hearing loss, a form of sensorineural hearing loss caused by mutations in the OTOF gene.

Approximately 20,000 individuals are affected in the United States and Europe. In April 2021, the FDA granted both Orphan Drug Designation and Rare Pediatric Disease Designation for AK-OTOF.

AK-OTOF is a dual adeno-associated viral (AAV) vector-based gene therapy intended to treat patients with OTOF-mediated hearing loss by delivering transgenes encoding OTOF to the inner hair cells (IHCs) of the cochlea.

The advancement of AK-OTOF into clinical development is supported by nonclinical data demonstrating administration of AK-OTOF in Otof knockout mice results in durable expression of human otoferlin protein sufficient for sustained restoration of auditory function.

The Phase 1/2 clinical trial is designed to evaluate the safety and tolerability of escalating doses of AK-OTOF administered unilaterally to trial participants with OTOF-mediated hearing loss. It is also designed to assess efficacy through clinical measures such as ABR, which is an objective, clinically accepted endpoint.

Based on interactions with FDA during the 30-day IND review period, the Company expects the first two participants will be as young as seven years of age, and that subsequent participants will be as young as two years of age at the time of administration.