Akouos Receives FDA Clearance for IND Application for AK-OTOF, a Gene Therapy for the Treatment of OTOF-Mediated Hearing Loss

Akouos, Inc.

-IND for AK-OTOF is the first to receive FDA clearance for a genetic form of hearing loss and the first for an AAV vector therapy with the potential to treat an inner ear condition

-Akouos plans to initiate a Phase 1/2 pediatric clinical trial, including children as young as two years old in the dose escalation phase (Part A), to evaluate AK-OTOF for the treatment of OTOF– mediated deafness

-Based on auditory brainstem response data from non-clinical studies, a administration of AK-OTOF has the potential to provide lasting restoration of hearing function

BOSTON, Sept. 13, 2022 (GLOBE NEWSWIRE) — Akouos, Inc. (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for people living with disabling hearing loss worldwide, today announced that it has received clearance from the United States Food and Drug Administration (FDA) for its investigational new drug (IND) application to initiate a first-in-man, Phase 1/2 pediatric clinical trial , of AK-OTOF, a gene therapy for the treatment of patients with the otoferlin gene (OTOF)-mediated hearing loss. Currently, there are no approved pharmacological treatment options for people with OTOF-mediated hearing loss, a form of sensorineural hearing loss caused by mutations in the OTOF embarrassed.

“The FDA’s AK-OTOF IND clearance is an important step toward achieving our mission to make healthy hearing accessible to everyone,” said Manny Simons, Ph.D., MBA, Co-Founder, President and CEO of Akouos. “This first-in-man clinical trial for AK-OTOF is groundbreaking and highlights Akouos’ leadership in the field – we expect this to be the first clinical trial for a genetic condition in the inner ear, the first in which AAV gene therapy is delivered to the inner ear, and the first for any inner ear condition to begin in a pediatric population.

“We are excited to advance AK-OTOF into clinical development. There is a significant unmet need for OTOF-mediated hearing loss, as individuals typically have severe to profound sensorineural hearing loss from birth, and there are currently no approved pharmacological options,” said Jen Wellman, Chief Operating Officer of Akouos. “This clinical trial is designed not only to assess the safety and potential benefits of AK-OTOF for people with OTOF-mediated hearing loss, but also to help us demonstrate the applicability of our new delivery approach to a wide range of inner ear conditions. We look forward to sharing what we have learned from this pioneering work. »

OTOF– mediated hearing loss is a form of sensorineural hearing loss caused by mutations in the otoferlin gene, which encodes otoferlin, a protein that allows the inner hair cells of the cochlea to release neurotransmitter vesicles in response to sound stimulation to activate auditory neurons. people with OTOF– mediated hearing loss have bilateral hearing loss that is usually severe to profound and congenital, presenting with an absent or very abnormal auditory brainstem response (ABR) from birth. About 20,000 people are affected in the United States and Europe. In April 2021, the FDA granted both Orphan Drug Designation and Rare Pediatric Disease Designation to AK-OTOF.

AK-OTOF is an adeno-associated virus (AAV)-based dual gene therapy intended to treat patients with OTOF-mediated hearing loss by delivering transgene coding OTOF inner hair cells (IHC) of the cochlea. A single unilateral intracochlear administration of AK-OTOF is intended to induce normal full-length functional otoferlin protein expression in IHCs, which has the potential to lead to the recovery of auditory function.

The advancement of AK-OTOF in clinical development is supported by non-clinical data demonstrating the administration of AK-OTOF in otof knockout mice result in sustained expression of the human protein otoferlin sufficient for sustained restoration of auditory function, as assessed by translationally relevant ABR assessments. In mice and nonhuman primates, AK-OTOF was systemically and locally well tolerated, and no adverse effects were observed in clinical pathology, otic pathology, systemic histopathology, or auditory or cochlear function.

The Phase 1/2 clinical trial is designed to evaluate the safety and tolerability of increasing doses of AK-OTOF administered unilaterally to trial participants with OTOF-mediate hearing loss; it is also designed to assess efficacy by clinical measures such as ABR, which is an objective and clinically accepted criterion. Given both the early onset of serious manifestations and the need for rapid intervention due to anatomical and developmental considerations, eligible participants in the clinical trial will be pediatric. Based on interactions with the FDA during the IND’s 30-day review period, the company expects the first two participants to be as young as seven years old and subsequent participants to be as young as two. years at the time of administration.

The Company expects to provide an update on clinical trial initiation activities for AK-OTOF later this year.

About Akouos
Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve and preserve high-acuity physiological hearing for people living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Boston-based Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery and AAV gene therapy.

Forward-looking statements
Statements in this press release regarding future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, timing of our future clinical trials for AK-OTOF and the potential benefits of AK-OTOF. The words “anticipate”, “believe”, “contemplate”, “continue”, “could”, “estimate”, “expect”, “intend”, “could”, “may”, ” plan”, “potential”, “predict”, “project”, “should”, “target”, “should”, “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain not these identifying words. Actual results may differ materially from those indicated by these forward-looking statements due to a variety of important factors, including: our limited operating history; the uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether the results of non-clinical studies will be predictive of the results or success of clinical trials; the timing and our ability to submit applications and obtain and maintain regulatory approvals for our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure needs with our cash, cash equivalents and marketable securities; the potential benefits of our product candidates; the rate 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 sales, marketing and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify other products, product candidates or technologies that have significant commercial potential and are consistent with our business objectives; the impact of governmental laws and regulations and any changes to such laws and regulations; risks related to competitive programs; the possibility that our internal manufacturing capabilities and/or our external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in “Risk Factors” included in the Company’s Quarterly Report on Form 10-Q for the three months ended June 30, 2022, filed with the Securities and Exchange Commission on August 15, 2022, and in other documents that the company with the Securities and Exchange Commission in the future. All forward-looking statements contained in this press release speak only as of the date hereof, 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
[email protected]

Courtney Turiano, Stern Investor Relations
[email protected]

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