On March 18, 2022 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 fourth quarter and full year ended December 31, 2021 and provided a corporate update (Press release, Decibel Therapeutics, MAR 18, 2022, View Source [SID1234610323]).
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"In 2021, Decibel made significant progress across our pipeline and towards our goal to bring transformative treatments to patients with hearing and balance disorders. We are on track to meet a number of important milestones in 2022, including the initiation of the first clinical trial of our lead gene therapy candidate, DB-OTO, designed to treat congenital hearing loss resulting from an otoferlin deficiency," said Laurence Reid, Ph.D., Chief Executive Officer of Decibel. "Among the milestones expected in 2022, we look forward to reporting the results of the interim analysis of our Phase 1b clinical trial of DB-020 in patients receiving cisplatin chemotherapy. We were also pleased that our gene therapy research and development collaboration with Regeneron was extended, that we expanded our preclinical gene therapy pipeline, and that we augmented our excellent Board of Directors."
Pipeline Highlights and Upcoming Milestones:
Gene Therapies for Congenital, Monogenic Hearing Loss
On Track to Achieve DB-OTO Key Milestones in 2022: Decibel expects to submit an investigational new drug application (IND) to the U.S. Food and Drug Administration and/or a Clinical Trial Application (CTA) in Europe for DB-OTO in 2022. Decibel also anticipates initiating a Phase 1/2 clinical trial of DB-OTO for pediatric patients with congenital hearing loss due to an otoferlin deficiency in 2022.
Presented New Preclinical Data at the 45th Annual Association for Research in Otolaryngology Conference (ARO) for DB-OTO and AAV.104 Programs: In February 2022, Decibel presented preclinical DB-OTO data as well as preclinical data highlighting the identification of proprietary, cell-selective promoters for three of its gene therapy pipeline programs at ARO. New DB-OTO data further characterized expression of otoferlin over time following DB-OTO administration in mice and non-human primates consistent with the expression previously reported in mice that achieved stable functional recovery. Decibel also shared functional recovery and tolerability data associated with DB-OTO dosing over a 10-fold dose range in mice. Decibel also presented data on how the Company deploys its platform to develop novel, proprietary, cell-selective promoters and the potential advantages that cell-selective expression may confer to its gene therapy preclinical programs.
Announced AAV.104 Gene Therapy Program for Restoration of Hearing in Patients with Congenital Hearing Impairment Due to Recessive Mutations in the Stereocilin (STRC) Gene: In November 2021, Decibel announced the target for its discovery stage program, AAV.104, which aims to restore hearing to individuals with an STRC deficiency, the second most common cause of autosomal recessive, non-syndromic, congenital hearing loss. The AAV.104 program is designed to express STRC selectively in outer hair cells, thus providing STRC specifically in its natural cellular location, a strategy that we believe has the potential to restore expression of the protein and hearing.
Product Candidate Selection for AAV.103 Program Anticipated in 2022: Decibel intends to select a product candidate for its AAV.103 program to restore hearing to individuals with a gap junction beta-2 (GJB2) deficiency, the most common cause of autosomal recessive, non-syndromic, congenital hearing loss.
Gene Therapies for Hair Cell Regeneration
Plans to Announce AAV.201 Program Target(s): Decibel continues to advance gene therapy programs for regeneration of hair cells in the vestibule for the treatment of bilateral vestibulopathy (BVP). Decibel plans to announce the program target(s) for its AAV.201 program in 2022. Our AAV.201 program is an AAV-based gene therapy that utilizes a proprietary supporting cell-selective promoter to express ATOH1 in combination with an additional factor. Our AAV.201 program aims to restore balance by promoting regeneration of Type I and Type II hair cells in the vestibular system.
Otoprotection Therapeutic
Interim Results from Phase 1b Proof-of-Concept Trial of DB-020 for the Treatment of Cisplatin-Induced Hearing Loss Expected in the First Half of 2022: Decibel expects to report the results of an interim analysis 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 that has been optimized for delivery to the ear. DB-020 is designed to protect hearing without interfering with cisplatin’s anti-cancer activity by locally disabling cisplatin in the cochlea.
Research Highlights:
Publication of Foundational Study of Noise-related Inner Ear Damage: In September 2021, Cell Reports published findings from a study of noise-related inner ear damage by the University of Maryland School of Medicine in collaboration with Decibel and the Karolinska Institute. The study’s aim was to generate an atlas of gene expression changes across inner ear cell types following noise damage. The study represents the first such comprehensive analysis and provides a foundational dataset that can be mined for future studies of the inner ear.
Corporate Highlights:
Strengthened Board of Directors with Key Appointments: In December 2021, Decibel announced the appointments of Alison Finger, MBA, and Saraswathy Nochur, Ph.D. to its Board of Directors. Previously, in June 2021, William (Bill) H. Carson, M.D., was appointed as Chairman of the Board of Directors.
Extended Research Term Under Strategic Collaboration with Regeneron to Discover and Develop Gene Therapies for Hearing Loss: In November 2021, Decibel announced that Regeneron extended the research term of its collaboration to discover and develop gene therapies for hearing loss. The research term was extended to November 15, 2023, and Regeneron is obligated to pay Decibel the associated extension fee of $10 million in the fourth quarter of 2022. Through the collaboration, Regeneron provides Decibel with broad access to its proprietary suite of technologies to support Decibel’s goal of discovering new medicines for congenital, monogenic hearing loss. Decibel retains worldwide development and commercialization rights to the product candidates being developed in the collaboration and is obligated to pay Regeneron tiered royalties based on net sales.
Fourth Quarter and Full Year 2021 Financial Results:
Cash Position: As of December 31, 2021, cash, cash equivalents and available-for-sale securities were $162.3 million, compared to $54.3 million as of December 31, 2020. The increase in cash, cash equivalents and available-for-sale securities was primarily due to net proceeds of $125.0 million from the company’s IPO in February 2021 and net proceeds of $27.4 million from the second closing of our Series D fundraising in February 2021 offset by operating expenses.
Research and Development Expenses: Research and development expenses were $8.0 million for the fourth quarter of 2021, compared to $7.9 million for the same period in 2020. Research and development expenses were $29.8 million for the full year 2021, compared to $25.3 million for the full year 2020. The increase in research and development expenses for the full year 2021 was primarily due to higher development costs related to manufacturing activities and IND-enabling studies for DB-OTO.
General and Administrative Expenses: General and administrative expenses were $4.9 million for the fourth quarter of 2021, compared to $5.1 million for the same period in 2020. General and administrative expenses were $20.4 million for the full year 2021, compared to $14.2 million for the full year 2020. The increase in general and administrative expenses for the full year 2021 was primarily attributable to higher costs associated with operating as a public company, including D&O insurance and accounting and legal fees, as well as higher wages due to increased headcount.
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.