BOSTON, MASSACHUSSETTS – Akouos, a precision genetic medicine company developing gene therapies to restore and preserve hearing, has filed for an initial public offer (IPO) with the Securities and Exchange Commission, eyeing a $100M IPO. The filing comes barely 3 months after he company completed its Series B funding round, which raised $105M.
The company’s lead program, AK-OTOF, is a gene therapy focused on restoring hearing in individuals with sensorineural hearing loss due to mutations in the otoferlin (OTOF) gene.
Normal otoferlin function enables the sensory cells of the ear (hair cells) to release neurotransmitter in response to stimulation by sound to activate auditory neurons. Without functional otoferlin protein, auditory signals received by the ear cannot be transmitted to the brain.
AK-OTOF uses an “adeno-associated viral (AAV) vector to deliver a healthy copy of the OTOF gene to cochlear hair cells, with the goal of restoring long-term physiologic hearing following a single administration to the inner ear”. AK-OTOF is intended to treat individuals with sensorineural hearing loss due to mutations in the OTOF gene, who typically have severe hearing loss in both ears from birth, by promoting the expression of normal, functional otoferlin protein in affected cells of the cochlea. Mutations in the OTOF gene are reported to be a major cause of genetic hearing loss, affecting an estimated 200,000 individuals worldwide.
“… Based on feedback from the U.S. Food and Drug Administration, or FDA, we are designing our Phase 1/2 trial to include ABR as an efficacy endpoint. We believe that this will enable us to quickly determine a clinical response and potentially result in rapid advancement towards a pivotal trial. There are no pharmacologic therapies currently approved for the treatment or management of OTOF-mediated hearing loss, or any other form of sensorineural hearing loss. We believe our product candidate has the potential to restore physiologic hearing and provide long-lasting benefits to these individuals and their families. We plan to submit an investigational new drug application, or IND, for AK-OTOF for OTOF-mediated hearing loss to the FDA in 2021, and we expect to report preliminary clinical data in 2022.”
–Company comments in SEC filing
The company was contacted regarding its IPO filing, but declined to offer comments at this time. The company’s SEC form S1 filing can be viewed here.