Decibel Therapeutics Presents Promising Preclinical Findings on DB-OTO, Gene Therapy Product Candidate Designed to Provide Hearing to Individuals with Otoferlin Mutations

BOSTON, Feb. 24, 2021 (GLOBE NEWSWIRE) -- Decibel Therapeutics DBTX, a clinical-stage company developing treatments to restore and improve hearing and balance, presented preclinical data supporting the company's gene therapy product candidate, DB-OTO, a potential transformative treatment for individuals with hearing loss due to mutation of the otoferlin gene. These and other data were presented by the company at the 44^th Annual MidWinter Meeting of the Association for Research in Otolaryngology (ARO), which was held virtually February 20–24, 2021.

Decibel's DB-OTO product candidate is designed to use dual AAV vectors and a cell-selective promoter to express functional otoferlin in hair cells of the inner ear, with the goal of enabling the ear to transmit sound to the brain and enable hearing. DB-OTO is intended to be delivered to patients using the surgical approach employed by otologists and pediatric otolaryngologists during a standard cochlear implantation procedure. Exploratory studies highlighted in the presentations utilizing the delivery procedure in non-human primates showed successful distribution and expression across the cochlear length. In these studies, DB-OTO and the surgical administration were generally well tolerated. The company presented data which suggest hair cell selective expression of otoferlin may enable greater efficacy and durability and minimize potential toxicity. In addition to these data, Decibel presented preclinical findings demonstrating that DB-OTO restored normal hearing sensitivity in animal models of otoferlin deficiency. Decibel is developing DB-OTO in collaboration with Regeneron Pharmaceuticals and expects to submit an IND or CTA in 2022.

"This is an exciting time for hearing and balance therapeutics, as we build preclinical evidence to support advancing gene therapies for the inner ear. We believe the data we shared at ARO, including potential benefits of cell-selective expression and utilization of an established surgical procedure, if shown in humans, would provide a competitive advantage for DB-OTO and support our approach to developing highly targeted gene therapies for hearing and balance," said John Lee, Executive Vice President, Pharmaceutical Development and Interim Chief Scientific Officer.

A selection of Decibel's presentations from ARO are available on the company's website:

• A podium presentation by Adam Palermo, Ph.D., Decibel's Vice President, Head of Genomics and Computational Biology, provided an overview of preclinical DB-OTO data from mice and non-human primates. The presentation can be found here.

• A podium presentation by Gabriela Pregernig, Ph.D., Associate Director of Computational Biology at Decibel, highlighted potential mechanisms of ubiquitous promoter-driven AAV toxicity in the ear. The presentation can be found here.

• Peter Weber, MBA, M.D., Decibel's Senior Medical Advisor, delivered a podium presentation that described the surgical approach for DB-OTO. He presented data in which dual vector AAV and Decibel's cell-selective promoter drove highly selective expression of GFP in hair cells of non-human primates. Details can be found here.

• A poster by Lars Becker, Ph.D., Associate Director of Discovery at Decibel, demonstrated that a variety of capsids are able to transduce inner hair cells of the inner ear in non-human primates and that the presence of neutralizing antibodies was not observed to impact transduction. The presentation can be found here.
  

About Decibel Therapeutics

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibel's pipeline, including its lead gene therapy product candidate, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all.

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Decibel's strategy, future operations, prospects, plans, objectives of management, the therapeutic potential for Decibel's product candidates and preclinical programs, the potential benefits of cell-selective expression and intracochlear injection, the expected timeline for submitting investigational new drug applications and expectations regarding the translation of preclinical findings to human disease, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "objective," "ongoing," "plan," "predict," "project," "potential," "should," or "would," or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Decibel may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities and the initiation and completion of preclinical studies and clinical trials; the timing of and Decibel's ability to submit and obtain approval to initiate clinical development of its program candidates; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; whether Decibel's cash resources are sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; uncertainties related to the impact of the COVID-19 pandemic on Decibel's business and operations; as well as the risks and uncertainties identified in Decibel's filings with the Securities and Exchange Commission (SEC), including those risks detailed under the caption "Risk Factors" in the final prospectus for Decibel's initial public offering filed with the SEC on February 12, 2021. In addition, the forward-looking statements included in this press release represent Decibel's views as of the date of this press release. Decibel anticipates that subsequent events and developments will cause its views to change. However, while Decibel may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Decibel's views as of any date subsequent to the date of this press release.

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