akouos gene therapy hearing loss treatment

Akouos More Than Doubles Original IPO Goal, Raises $213M in First Day of Trading

BOSTON, MASSACHUSSETTS — After originally filing for an initial public offer (IPO) with the US Securities and Exchange Commission (SEC) earlier this month, eyeing a $100M IPO, gene therapy startup Akouos significantly exceeded its original expectations on Friday by raising $213M.

The company sold 12.5 million shares for $17 each, above its anticipated range of $14-$16 per share, and saw its share prices rising in the first hours of trading to reach $22 per share by noon.

 

Using Gene Therapy to Restore Hearing

 

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. 

Akouos is planning to use $75 million of its IPO proceeds for clinical trials and another $35 million into preclinical R&D, based upon documents the company provided to the SEC and hopes for FDA approval to begin clinical trial in 2021.


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