Sensorion, a pioneering clinical-stage biotech company dedicated to the development of new therapies to restore, treat, and prevent hearing loss disorders, today announced that it has submitted an Application Initial clinical trial (CTA) for OTOF-GT for the UK Medicines and Health Agency. The Healthcare Products Regulatory Authority (MHRA).The Phase 1/2 (Audiogene) clinical trial aimed to evaluate the safety, tolerability and efficacy of intracochlear implantation of OTOF-GT for the treatment of gene-mediated hearing loss of otoferlin in pediatric patients under 31 months of age. The CTA submission followed extensive preclinical studies evaluating the safety and efficacy of OTOF-GT and the successful production of a gene therapy drug product for clinical trial.Sensorion’s OTOF-GT dual AAV gene therapy development program aims to restore hearing in patients with the OTOF mutation, who have severe to severe non-syndromic pre-verbal neural hearing loss. Otoferlin is a protein expressed in inner hair cells (IHCs) found in the cochlea and is essential for signaling to the auditory nerve. Otoferlin-related hearing loss is responsible for up to 8% of all cases of congenital hearing loss, with approximately 20,000 affected in the United States and Europe1. OTOF-GT previously received Orphan Drug Designation from the US Food and Drug Administration (FDA)2 and the European Medicines Agency (EMA)3 and Rare Patient Designation from the FDA in the first quarter of this year 4 year 2022.
“This first gene therapy CTA filing is a major milestone for our OTOF-GT program and Sensorion’s broader gene therapy franchise. This is the first of several planned CTA filings and we hope to commence patient recruitment soon, subject to the CTA approval. We look forward to continuing to work with clinicians, regulatory authorities and patient groups to address unmet and underserved medical needs in the intricate hearing space.”
Nawal Ouzren, Chief Executive Officer of Sensorion.
Sensorion plans to submit the CTA in Europe in the coming weeks.“There are currently no approved drug for patients with mutations of the gene encoding for otoferlin. Our goal is to transform the standard of care for OTOF newborns, by reducing dependence on cochlear implants, which would transform their quality of life. We believe that gene therapy has the potential to offer permanent solutions to patients with diseases caused by the OTOF mutation and other inner ear diseases and we are really excited to move OTOF-GT forward towards clinical development.”
Géraldine Honnet, Chief Medical Officer of Sensorion.
