On August 11, 2023 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 second quarter ended June 30, 2023 and provided a corporate update (Press release, Decibel Therapeutics, AUG 11, 2023, View Source [SID1234634272]).
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"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 (FDA).
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.
Corporate Update:
Announced definitive agreement for Regeneron Pharmaceuticals to acquire Decibel:
On August 9, 2023, Decibel and Regeneron announced the entry into an agreement for Regeneron to acquire Decibel to build on the existing collaboration, and accelerate and further resource key gene therapy programs for hearing loss. The proposed acquisition values Decibel at a total equity value of approximately $109 million based on the amount payable at closing, and a total equity value of up to approximately $213 million if the contingent value right (CVR) milestones are achieved.
Second Quarter 2023 Financial Results:
Cash Position: As of June 30, 2023, cash, cash equivalents and available-for-sale securities were $78.3 million, compared to $104.6 million as of December 31, 2022.
Research and Development Expenses: Research and development expenses were $12.8 million for the second quarter of 2023, compared to $11.2 million for the same period in 2022. The increase in research and development expenses for the second quarter 2023 was primarily due to higher clinical development and related costs for the Company’s Phase 1/2 clinical trial of DB-OTO, additional regulatory and clinical costs for DB-020, and higher personnel-related costs due to increased headcount, wages and stock-based compensation.
General and Administrative Expenses: General and administrative expenses were $6.2 million for the second quarter of 2023, compared to $5.9 million for the same period in 2022. The increase in general and administrative expenses for the second quarter 2023 was primarily due to increased facility-related costs due to decreased sublease income and higher personnel-related costs due to increased headcount, wages and stock-based compensation, partially offset by decreased professional fees year over year.