By Chris Wack


Akouos Inc. said Tuesday that it has received clearance from the U.S. Food and Drug Administration for its investigational new drug 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-mediated hearing loss.

AK-OTOF is a dual adeno-associated viral vector-based gene therapy intended to treat patients with OTOF-mediated hearing loss by delivering transgenes encoding OTOF to the inner hair cells of the cochlea, the precision genetic medicine company said.

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, as assessed by translationally relevant assessments.

In both mice and non-human primates, AK-OTOF was systemically and locally well tolerated, and no adverse effects were observed in clinical pathology, otic pathology, systemic histopathology, or auditory or cochlear function, Akouos said.

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, the company said.

Akouos shares were up 11% to $3.89 in premarket trading.


Write to Chris Wack at chris.wack@wsj.com


(END) Dow Jones Newswires

09-13-22 0727ET