BOSTON, April 13, 2021 (GLOBE NEWSWIRE) — Akouos, Inc. (NASDAQ: AKUS), a precision genetic drugs firm devoted to creating potential gene therapies for people residing with disabling listening to loss worldwide, as we speak introduced that the U.S. Meals and Drug Administration (FDA) has granted each Orphan Drug Designation (ODD) and Uncommon Pediatric Illness Designation (RPDD) for AK-OTOF, a gene remedy meant for the remedy of otoferlin gene-mediated listening to loss. Akouos anticipates that it’s going to submit an investigational new drug software (IND) for AK-OTOF within the first half of 2022.
Otoferlin gene (OTOF)-mediated listening to loss is a type of sensorineural listening to loss brought on by mutations within the OTOF gene. The OTOF gene encodes otoferlin, a protein that permits the inside hair cells of the cochlea to launch neurotransmitter vesicles in response to stimulation by sound to activate auditory neurons. Most people with OTOF-mediated listening to loss have Extreme-to-Profound sensorineural listening to loss from start and roughly 20,000 people are affected in the USA and Europe. AK-OTOF is designed to deal with the underlying explanation for OTOF-mediated listening to loss by means of supply of a transgene utilizing a twin vector know-how that leads to expression of regular, purposeful otoferlin protein within the affected cells, specifically inside hair cells, within the cochlea.
“There are presently no pharmacologic remedy choices for people with OTOF-mediated listening to loss, or for some other type of sensorineural listening to loss. The nonclinical information reported so far for AK-OTOF show sturdy restoration of auditory perform and help future medical growth.” stated Jen Wellman, chief working officer of Akouos. “We consider these are the primary Orphan Drug and Uncommon Pediatric Illness designations granted by FDA for a genetic type of listening to loss, and symbolize an vital milestone for the sector of inside ear genetic medicines. The receipt of each designations may assist us speed up growth of AK-OTOF, a remedy that we consider has the potential to revive physiologic listening to and supply long-lasting advantages to those people and their households.”
The Workplace of Orphan Merchandise Improvement of FDA grants ODD to medicine and biologics meant for the remedy, analysis, or prevention of uncommon illnesses, or situations affecting fewer than 200,000 folks in the USA. The designation affords Akouos the potential for sure advantages, together with as much as seven years of post-approval market exclusivity, help within the drug growth course of, tax credit for medical growth, and exemptions from sure FDA charges.
Uncommon pediatric illness designation is granted by FDA to encourage growth of therapies for critical or life-threatening uncommon illnesses wherein the illness manifestations primarily have an effect on people aged from start to 18 years. Underneath the Precedence Assessment Voucher program, and topic to FDA approval of AK-OTOF for the remedy of OTOF-mediated listening to loss, Akouos could also be eligible to obtain one precedence evaluation voucher, which may then be redeemed to obtain precedence evaluation for a subsequent advertising software for a distinct product or bought or transferred to a different sponsor.
Akouos is a precision genetic drugs firm devoted to creating gene therapies with the potential to revive, enhance, and protect high-acuity physiologic listening to for people residing with disabling listening to loss worldwide. Leveraging its precision genetic drugs platform that includes a proprietary adeno-associated viral (AAV) vector library and a novel supply strategy, Akouos is concentrated on creating precision therapies for types of sensorineural listening to loss. Headquartered in Boston, Akouos was based in 2016 by leaders within the fields of neurotology, genetics, inside ear drug supply, and AAV gene remedy.
Statements on this press launch about future expectations, plans and prospects, in addition to some other statements relating to issues that aren’t historic details, might represent “forward-looking statements” throughout the that means of The Non-public Securities Litigation Reform Act of 1995. These statements embody, however usually are not restricted to, statements referring to the timing of our IND submission for AK-OTOF and the potential receipt of a precedence evaluation voucher and different advantages from the ODD and RPDD. The phrases “anticipate,” “consider,” “proceed,” “may,” “estimate,” “anticipate,” “intend,” “might,” “plan,” “potential,” “predict,” “mission,” “ought to,” “goal,” “will,” “would,” and related expressions are meant to determine forward-looking statements, though not all forward-looking statements comprise these figuring out phrases. Precise outcomes might differ materially from these indicated by such forward-looking statements on account of numerous vital components, together with: our restricted working historical past; uncertainties inherent within the growth of product candidates, together with the initiation and completion of nonclinical research and medical trials; whether or not outcomes from nonclinical research will probably be predictive of outcomes or success of medical trials; the timing of and our capacity to submit purposes for, and acquire and keep regulatory approvals for, our product candidates; our expectations relating to our regulatory technique; our capacity to appreciate the anticipated advantages of ODD and RPDD; the potential for adjustments in regulatory necessities; our capacity to fund our working bills and capital expenditure necessities with our money, money equivalents, and marketable securities; the potential benefits of our product candidates; the speed and diploma of market acceptance and medical utility of our product candidates; our estimates relating to the potential addressable affected person inhabitants for our product candidates; our commercialization, advertising, and manufacturing capabilities and technique; our capacity to acquire and keep mental property safety for our product candidates; our capacity to determine further merchandise, product candidates, or applied sciences with vital industrial potential which are in step with our industrial aims; the impression of presidency legal guidelines and rules; dangers associated to aggressive packages; the potential that our inner manufacturing capabilities and/or exterior manufacturing provide might expertise delays; the impression of the COVID-19 pandemic on our enterprise, outcomes of operations, and monetary situation; our capacity to keep up and set up collaborations or acquire further funding; and different components mentioned within the “Danger Components” part included within the Firm’s Annual Report on Kind 10-Ok for the fiscal 12 months ended December 31, 2020 filed with the Securities and Alternate Fee, and in different filings that the Firm makes with the Securities and Alternate Fee sooner or later. Any forward-looking statements contained on this press launch communicate solely as of the date hereof, and the Firm expressly disclaims any obligation to replace any forward-looking assertion, whether or not on account of new info, future occasions or in any other case.
Katie Engleman, 1AB
Courtney Turiano, Stern Investor Relations