Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced authorization of its Clinical Trial Application (CTA) by the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial in pediatric patients of DB-OTO, its lead gene therapy product candidate.
DB-OTO is being developed in collaboration with Regeneron Pharmaceuticals, Inc. and is a cell-selective adeno-associated virus (AAV) gene therapy designed to provide durable hearing in individuals with profound, congenital hearing loss caused by mutations of the otoferlin gene.
The U.K. CTA is part of an international regulatory strategy for clinical development of DB-OTO, which also includes a regulatory clearance in October 2022 for an Investigational New Drug (IND) application in the United States and a CTA submitted to the Spanish Agency of Medicines and Medical Devices.
“This CTA marks the second regulatory clearance for DB-OTO and what we believe to be the first in Europe for any gene therapy targeting otoferlin. This is an important milestone as we execute our international clinical development strategy, which addresses pediatric patients of diverse ages, including infants two years of age and younger,” said Laurence Reid, Ph.D., Chief Executive Officer at Decibel. “We believe DB-OTO has transformative potential for individuals with otoferlin-related hearing loss, and we intend to initiate the clinical trial in the first half of 2023. We expect to share initial data from the first cohort of patients in the first quarter of 2024.”
DB-OTO received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration in 2021. Currently, there are no approved pharmacologic treatment options for individuals with otoferlin-related hearing loss.
The Phase 1/2 dose escalation clinical trial is designed to evaluate the safety, tolerability and efficacy of DB-OTO in pediatric patients with congenital hearing loss due to an otoferlin deficiency. In addition to safety and tolerability endpoints, the auditory brainstem response (ABR), an objective, clinically accepted, physiologic measure of hearing sensitivity, will be used as an efficacy endpoint in the clinical trial. The ABR, which was used to characterize dose-response of DB-OTO after intra-cochlear delivery in translational studies in animal models, provides an opportunity to rapidly assess hearing functionality and sensitivity.
The Company intends to provide an update on the design of the clinical trial in mid-2023.