BOSTON, Aug. 11, 2021 (GLOBE NEWSWIRE) — Akouos, Inc. (NASDAQ: AKUS), a precision genetic medication firm devoted to creating potential gene therapies for people dwelling with disabling listening to loss worldwide, right now introduced that the European Medicines Company (EMA) Committee for Orphan Medicinal Merchandise (COMP) issued a constructive opinion on the corporate’s utility for orphan drug designation for AK-OTOF, a gene remedy supposed for the remedy of otoferlin gene-mediated listening to loss. The constructive opinion was subsequently adopted by the European Fee. AK-OTOF was beforehand granted Orphan Drug Designation (ODD) and Uncommon Pediatric Illness Designation (RPDD) by the U.S. Meals and Drug Administration for this identical indication.
Otoferlin gene (OTOF)-mediated listening to loss is a type of sensorineural listening to loss attributable to mutations within the OTOF gene. The OTOF gene encodes otoferlin, a protein that permits the internal 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 delivery and roughly 20,000 people are affected in america and Europe. AK-OTOF is designed to deal with the underlying reason behind OTOF-mediated listening to loss via supply of a transgene utilizing a twin vector expertise that leads to expression of regular, purposeful otoferlin protein within the affected cells, particularly internal hair cells, within the cochlea.
“Extreme to Profound sensorineural listening to loss from delivery attributable to mutations within the OTOF gene is a excessive unmet want, particularly on condition that there are presently no authorised pharmacologic remedy choices obtainable,” stated Katie Wachtel, M.S. and vp of regulatory affairs of Akouos. “The orphan drug designation granted by the European Fee is a crucial step in the direction of advancing the worldwide improvement of AK-OTOF, a gene remedy with potential to revive physiologic listening to and supply long-lasting profit to people with OTOF-mediated listening to loss. Together with the ODD and RPDD designations beforehand granted by FDA, the orphan drug designation for AK-OTOF within the European Union might speed up our improvement of AK-OTOF and our progress in the direction of our mission of wholesome listening to obtainable to all.”
Orphan drug designation within the European Union is granted by the European Fee primarily based on an opinion issued by the EMA COMP. An orphan drug designation offers a number of advantages, together with payment reductions, regulatory help, and the likelihood to use for a centralized European Union advertising authorization. Advertising and marketing authorization for an orphan drug can result in a ten-year interval of market exclusivity.
Akouos is a precision genetic medication firm devoted to creating gene therapies with the potential to revive, enhance, and protect high-acuity physiologic listening to for people dwelling with disabling listening to loss worldwide. Leveraging its precision genetic medication platform that comes with a proprietary adeno-associated viral (AAV) vector library and a novel supply method, 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, internal ear drug supply, and AAV gene remedy.
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Statements on this press launch about future expectations, plans and prospects, in addition to another statements concerning issues that aren’t historic information, might represent “forward-looking statements” throughout the which means of The Non-public Securities Litigation Reform Act of 1995. These statements embrace, however aren’t restricted to, statements referring to the initiation, plans, and timing of our future scientific trials and our analysis and improvement applications, and the potential receipt of exclusivity and different advantages from Orphan Drug Designation within the European Union and Orphan Drug Designation and Uncommon Pediatric Illness Designation in america. The phrases “anticipate,” “consider,” “proceed,” “might,” “estimate,” “count on,” “intend,” “might,” “plan,” “potential,” “predict,” “undertaking,” “ought to,” “goal,” “will,” “would,” and comparable expressions are supposed to determine forward-looking statements, though not all forward-looking statements include these figuring out phrases. Precise outcomes might differ materially from these indicated by such forward-looking statements because of varied vital components, together with: our restricted working historical past; uncertainties inherent within the improvement of product candidates, together with the initiation and completion of nonclinical research and scientific trials; whether or not outcomes from nonclinical research will probably be predictive of outcomes or success of scientific trials; the timing of and our skill to submit purposes for, and acquire and preserve regulatory approvals for, our product candidates; our expectations concerning our regulatory technique; our skill 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 scientific utility of our product candidates; our estimates concerning the potential addressable affected person inhabitants for our product candidates; our commercialization, advertising, and manufacturing capabilities and technique; our skill to acquire and preserve mental property safety for our product candidates; our skill to determine extra merchandise, product candidates, or applied sciences with vital business potential which are per our business targets; the affect of presidency legal guidelines and rules and any modifications in such legal guidelines and rules; dangers associated to aggressive applications; the potential that our inner manufacturing capabilities and/or exterior manufacturing provide might expertise delays; the affect of the COVID-19 pandemic on our enterprise, outcomes of operations, and monetary situation; our skill to keep up and set up collaborations or acquire extra funding; and different components mentioned within the “Threat Elements” included within the Firm’s Quarterly Report on Kind 10-Q for the three months ended March 31, 2021 filed with the Securities and Trade Fee, and in different filings that the Firm makes with the Securities and Trade Fee sooner or later. Any forward-looking statements contained on this press launch converse solely as of the date hereof, and the Firm expressly disclaims any obligation to replace any forward-looking assertion, whether or not because of new data, future occasions or in any other case.
Katie Engleman, 1AB
Courtney Turiano, Stern Investor Relations