BOSTON - Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today reported financial results for the second quarter ended June 30, 2023 and provided a corporate update.
'This week we announced a definitive agreement with Regeneron Pharmaceuticals to acquire Decibel, which we believe reflects the tremendous promise of gene therapy for hearing loss,' said Laurence Reid, Ph.D., Chief Executive Officer of Decibel. 'We remain highly focused on executing on the CHORD clinical trial and excited by the potential benefit that DB-OTO may provide to people with otoferlin-related hearing loss. With six active clinical trial sites at prestigious institutions across three countries, we continue to work towards our goal of addressing this unmet medical need.'
Pipeline Highlights and Upcoming Milestones
Gene Therapies for Congenital, Monogenic Hearing Loss
Phase 1/2 CHORDTM Dose Escalation Clinical Trial of DB-OTO in Pediatric Patients
DB-OTO is an AAV-based dual-vector gene therapy product candidate designed to be delivered one time to selectively express functional otoferlin (OTOF) in the inner hair cells of individuals with OTOF deficiency with the goal of enabling the ear to transmit sound to the brain and enable durable, physiological hearing. Decibel has been developing DB-OTO in collaboration with Regeneron Pharmaceuticals.
In May 2023, Decibel announced the initiation of CHORD, a global Phase 1/2 dose escalation clinical trial of DB-OTO in pediatric patients. Decibel has opened clinical trial sites in the U.S., the U.K. and Spain and has commenced patient screening activities. DB-OTO received Orphan Drug Designation from the European Medicines Agency (EMA) Committee on Orphan Medicinal Products (COMP) in May 2023, adding to its Orphan Drug and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration.
Continued Progress with IND-Enabling Activities to Support the Advancement of AAV.103 for GJB2-Related Hearing Loss Program
In March 2023, Decibel achieved a pre-investigational new drug (IND) milestone under its collaboration agreement with Regeneron Pharmaceuticals related to the initiation of manufacturing of AAV.103, Decibel's gene therapy product candidate designed to restore hearing in individuals with mutations in the gap junction beta-2 (GJB2) gene, the leading cause of autosomal recessive, non-syndromic, congenital hearing loss worldwide.
Otoprotection Therapeutic
Received FDA Breakthrough Therapy Designation for DB-020
In July 2023, Decibel received FDA Breakthrough Therapy Designation for DB-020, a novel, proprietary formulation of sodium thiosulfate (STS), designed to protect against hearing loss in cancer patients receiving cisplatin chemotherapy. This was supported by Decibel's Phase 1b clinical trial in which 87% of treated patients experiencing ototoxicity in their placebo-treated ear were partially or completely protected from ototoxicity. Breakthrough Therapy Designation is intended to expedite the development and review of medicines intended to treat serious or life-threatening conditions that have preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available treatments. A Breakthrough Therapy Designation conveys all the fast-track program features, more intensive FDA guidance on the drug development program, an organizational commitment involving senior FDA managers and eligibility for rolling review and priority review.
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 gene therapy for the inner ear, the expected timeline for initiating clinical trials, reporting clinical trial results and achieving other anticipated milestones, expectations regarding the translation of preclinical findings to human disease, the sufficiency of Decibel's existing cash resources for the period anticipated, the occurrence of any event, change, or other circumstance that could give rise to the termination of the Agreement and Plan of Merger with Regeneron Pharmaceuticals, Inc., the prospective benefits of the proposed acquisition, the value of potential contingent consideration amounts and timing of the closing for the transaction, 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: risks related to our proposed acquisition by Regeneron, 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,, 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 Decibel's Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2023 and in other filings Decibel may make with the SEC. 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.
Contact:
Chris Railey
Email: chris@tenbridgecommunications.com
Tel: 617-834-0936
DECIBEL THERAPEUTICS, INC. 
