BOSTON, MASSACHUSSETTS — Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported financial results for the third quarter ended September 30, 2021 and provided a corporate update.
“Decibel continues to work towards bringing transformative treatments to patients with hearing and balance disorders as we move to the end of a successful 2021 and look forward to 2022,” said Laurence Reid, Ph.D., Chief Executive Officer of Decibel.
“We remain on track to initiate a Phase 1/2 clinical trial of DB-OTO in pediatric patients in 2022 and report the results of an interim analysis of our Phase 1b clinical trial of DB-020 in patients receiving cisplatin chemotherapy in the first half of 2022. We are incredibly pleased to have received Orphan Drug and Rare Pediatric Disease designations for DB-OTO, reiterating the importance of our approach to gene therapies for congenital, monogenic hearing loss. Beyond DB-OTO, we have continued to leverage our precision gene therapy platform to advance our GJB2, stereocilin and regeneration gene therapy programs.”
— Laurence Reid, Ph.D., Decibel Therapeutics CEO
Gene Therapies for Congenital, Monogenic Hearing Loss
- Received Orphan Drug and Rare Pediatric Disease Designations for DB-OTO: In September 2021, Decibel announced that the U.S. Food and Drug Administration (FDA) granted both Orphan Drug Designation and Rare Pediatric Disease Designation for DB-OTO for the treatment of otoferlin-related congenital hearing loss.
- On Track to Achieve DB-OTO Key Milestones in 2022: Decibel expects to submit an investigational new drug application (IND) with the FDA and/or a Clinical Trial Application (CTA) in Europe for DB-OTO and initiate a Phase 1/2 clinical trial of DB-OTO for pediatric patients with congenital hearing loss due to an otoferlin deficiency in 2022.
- Announces AAV.104 Gene Therapy Program for Restoration of Hearing in Patients with Congenital Hearing Impairment Due to Recessive Mutations in the Stereocilin (STRC) Gene: AAV.104 aims to restore hearing to individuals with a STRC deficiency, the second most common cause of autosomal recessive, non-syndromic, congenital hearing loss. The Company estimates that the prevalence of individuals with this form of hearing loss in the United States and the major markets in Europe is approximately 70,000. STRC is a large, extracellular, structural protein expressed in outer hair cells of the cochlea. Functional outer hair cells amplify sound within the ear, a process required for normal hearing sensitivity and frequency selectivity. Despite the absence of the STRC protein and observed hearing loss in patients carrying STRC mutations, third-party research has shown that the outer hair cells remain intact and viable for gene therapy. AAV.104 is designed to express STRC selectively in outer hair cells, thus providing STRC specifically in its natural cellular location, a strategy that has the potential to restore expression of the protein and hearing. Decibel is working in collaboration with Regeneron to develop AAV.104 and plans to share preclinical data on this program at an upcoming scientific meeting.
Gene Therapies for Hair Cell Regeneration
- On Track to Announce AAV.201 Program Target: Decibel continues to advance AAV.201, its gene therapy program for regeneration of hair cells in the vestibule for the treatment of bilateral vestibulopathy (BVP). Decibel plans to announce the program target(s) for AAV.201 in 2022.
Otoprotection Therapeutic
- On Track to Report Interim Results from Phase 1b Proof-of-Concept Trial of DB-020 for the Treatment of Cisplatin-Induced Hearing Loss: Decibel expects to report interim results from the ongoing Phase 1b clinical trial of DB-020 in patients with cisplatin-induced hearing loss, a serious and debilitating condition for which there are no approved therapies, in the first half of 2022. Cisplatin, a commonly used chemotherapy agent, is known to cause hearing loss, tinnitus and speech recognition difficulty. DB-020 comprises a proprietary formulation of sodium thiosulfate which has been optimized for delivery to the ear. By locally disabling cisplatin in the cochlea, DB-020 is designed to protect hearing without interfering with cisplatin’s anti-cancer activity.
- Granted Key U.S. Patent Covering DB-020 Formulation: In July 2021, the United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 11,071,751, “Hypertonic pharmaceutical compositions containing an anti-platinum chemoprotectant agent,” a foundational patent and component of Decibel’s DB-020 intellectual property portfolio.
Research Highlights:
- Announced Foundational Study of Noise-Related Inner Ear Damage: In September 2021, Decibel announced the publication of new findings in Cell Reports from a study on noise-related inner ear damage conducted in collaboration with the University of Maryland School of Medicine and the Karolinska Institute. This study demonstrates how Decibel’s platform is built to provide a molecular characterization of the cells of the inner ear to enable the identification of therapeutics that counter the underlying molecular pathology of noise-induced hearing loss in the future.
Third Quarter 2021 Financial Results:
- Cash Position: As of September 30, 2021, cash, cash equivalents and available-for-sale securities were $172.4 million.
- Research and Development Expenses: Research and development expenses were $9.0 million for the third quarter of 2021, compared to $4.7 million for the third quarter of 2020. The increase in research and development expenses for the third quarter of 2021 was driven primarily by an increase in costs associated with IND/CTA enabling activities for DB-OTO.
- General and Administrative Expenses: General and administrative expenses were $5.7 million for the third quarter of 2021, compared to $2.4 million for the same period in 2020. The increase in general and administrative expenses for the third quarter of 2021 was primarily attributable to increases in professional fees, personnel costs and directors’ and officers’ insurance costs incurred as a result of becoming a public company.
Financial Guidance:
Based on its current operating and development plans, Decibel believes that its existing cash, cash equivalents and available-for-sale securities will fund its pipeline programs and operating expenses into 2024.
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 program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of creating a world of connection for people with hearing and balance disorders.
Source: Decibel Therapeutics
