Decibel Therapeutics (NASDAQ:DBTX) said its clinical trial application (CTA) received approval from the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) for starting a phase 1/2 trial in children of gene therapy DB-OTO.
DB-OTO, which is being developed in collaboration with Regeneron Pharmaceuticals (NASDAQ:REGN), is a cell-selective adeno-associated virus (AAV) gene therapy designed to provide durable hearing in patients with profound, congenital hearing loss caused by mutations of the otoferlin gene, the company added.
Decibel noted that a one-time administration of DB-OTO resulted in production of otoferlin protein and durable auditory brainstem responses to sound in a congenitally deaf rodent disease model.
“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,” said Decibel CEO Laurence Reid.
CTA authorization provides approval to expand the planned phase 1/2 trial to the U.K. in patients two years of age and younger. The company plans to start the trial in H1 2023.
The company’s application had received clearance in the U.S. in October 2022 to start a study of the gene therapy. Decibel has also filed a CTA with the Spanish drug regulator.