Akouos Receives Orphan Drug and Rare Pediatric Disease Designations for AK-OTOF for the Treatment of Otoferlin Gene-Mediated Hearing Loss Nasdaq:AKUS
BOSTON, April 13, 2021 (GLOBE NEWSWIRE) – Akouos, Inc. (NASDAQ: AKUS), an accurate genetic medicine company dedicated to developing potential gene therapies for people with hearing loss worldwide, announced today that the U.S. Food and Drug Administration (FDA) has adopted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) awarded for AK-OTOF, a gene therapy intended for the treatment of Otoferlin gene-mediated hearing loss. Akouos expects to submit a research application for new drugs (IND) for AK-OTOF in the first half of 2022.
Otoferlin gene (OTOF) mediated hearing loss is a form of sensorineural hearing loss caused by mutations in the OTOF gene. The OTOF gene codes for otoferlin, a protein that enables the inner hair cells of the cochlea to release neurotransmitter vesicles in response to sound stimulation to activate auditory neurons. Most people with OTOF-mediated hearing loss have severe to profound sensorineural hearing loss from birth, and approximately 20,000 people are affected in the United States and Europe. AK-OTOF is designed to treat the underlying cause of OTOF-mediated hearing loss by delivering a transgene using a dual vector technology that results in expression of normal, functional otoferlin protein in the affected cells, namely the inner hair cells, in the cochlea.
“There are currently no pharmacological treatment options for individuals with OTOF-mediated hearing loss, or for any other form of sensorineural hearing loss. The non-clinical data reported for AK-OTOF to date demonstrate sustainable recovery of auditory function and support future clinical development. “said Jen Wellman, Akouos chief operating officer.” We believe these are the first orphan drug and rare pediatric disease designations awarded by the FDA for a genetic form of hearing loss, and that this is an important milestone in the field of genetic medicines for the inner ear. Receiving both designations could help us accelerate the development of AK-OTOF, a therapy that we believe has the potential to restore physiological hearing and provide long-term benefits to these individuals and their families . “
The FDA’s Office of Orphan Products Development grants ODD to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. The designation offers Akouos the potential for certain benefits, including up to seven years of post-approval market exclusivity, drug development assistance, clinical development tax credits and exemptions from certain FDA fees.
The FDA grants the rare pediatric disease designation to encourage the development of treatments for serious or life-threatening rare diseases, where the symptoms primarily affect individuals from birth to 18 years of age. Under the Priority Review Voucher Program, and subject to FDA approval of AK-OTOF for the treatment of OTOF-mediated hearing loss, Akouos may qualify for one Priority Review voucher, which can then be redeemed for priority review for a subsequent marketing application for another product or sold or transferred to another sponsor.
Akouos is a precision genetic medicine company dedicated to the development of gene therapies with the potential to restore, improve and preserve highly acute physiological hearing for people with hearing loss around the world. Using its precision genetic medicine platform that includes a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is committed to developing precision therapies for sensorineural hearing loss forms. Headquartered in Boston, Akouos was founded in 2016 by leaders in neurotology, genetics, inner ear drug delivery and AAV gene therapy.
Statements in this press release about future expectations, plans and prospects, as well as other statements relating to matters that are not historical facts, may be “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include, but are not not limited to, statements regarding the timing of our IND submission for AK-OTOF and the possible receipt of a priority review voucher and other benefits from the ODD and RPDD. The words’ anticipate ‘,’ believe ‘,’ continue ‘,’ can ‘,’ estimate ‘,’ expect ‘,’ plan ‘,’ can ‘,’ plan ‘,’ potential ‘,’ predict ‘,’ project “,” “Should”, “purpose”, “will”, “should” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results could differ materially from those indicated by such forward-looking statements as a result of several important factors, including: our limited business history; uncertainties inherent in the development of product candidates, including the initiation and completion of non-clinical studies and clinical trials; whether results from non-clinical studies will be predictive of the results or success of clinical studies; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for our product candidates; our expectations regarding our regulatory strategy; our ability to realize the expected benefits of ODD and RPDD; the potential for changes in regulatory requirements; our ability to fund our operating and capital expenditures with our cash, cash equivalents and marketable securities; the potential benefits of our product candidates; the speed and degree of market acceptance and clinical utility of our product candidates; our estimates of the potential addressable patient population for our product candidates; our commercialization, marketing and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations; risks related to competing programs; the potential that our internal manufacturing capabilities and / or external manufacturing supply will be delayed; the impact of the COVID-19 pandemic on our business, results of operations and financial condition; our ability to maintain and establish partnerships or obtain additional funding; and other factors discussed in the “ Risk Factors ” section of the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2020, filed with the Securities and Exchange Commission, and in other documents the Company makes with the Securities and Exchange Commission in the future. All forward-looking statements in this press release speak only as of the date here, and the company expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.
Katie Engleman, 1AB
Courtney Turiano, Stern Investor Relations