The FDA has signed offAkouos Inc’sAKUS Investigational New Drug application to initiate a Phase 1/2 trial of AK-OTOF, gene therapy for otoferlin gene (OTOF)-mediated hearing loss.
OTOF-mediated hearing loss is a form of sensorineural hearing loss caused by mutations in the otoferlin gene, which encodes otoferlin. This protein enables the inner hair cells of the cochlea to release neurotransmitter vesicles in response to stimulation by sound to activate auditory neurons.
The Phase 1/2 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 will also assess efficacy through clinical measures such as ABR, an objective, clinically accepted endpoint.
Nonclinical data demonstrated that AK-OTOF in Otof knockout mice results in durable expression of human otoferlin protein sufficient for sustained restoration of auditory function.
In both mice and non-human primates, AK-OTOF was systemically and locally well tolerated, and no adverse effects were observed.
The company plans to provide an update on clinical trial initiation activities later this year.
Price Action: AKUS shares are down 5.14% at $3.32 on the last check Tuesday.
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