Sensorion announced that it has received approval for its Clinical Trial Application (CTA) to initiate a Phase 1/2 clinical trial of SENS-501 (OTOF-GT), in France. The conclusion of the Part I of the assessment report according to regulation EU 536/2014 covering France, Italy and Germany is that the conduct of the clinical trial is acceptable. The phase 1/2 clinical trial (Audiogene) aims to evaluate the safety, tolerability, and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing impairment in paediatric patients aged 6 to 31 months at the time of gene therapy treatment.

Targeting the first years of life, the time period when the auditory system plasticity is optimal, will maximize the chances of these young children with pre-lingual hearing loss to acquire normal speech and language. Audiogene will also assess the clinical safety, performance, and usability of the administration device system under development in partnership with EVEON. The design of the study will consist of two cohorts of 2 doses followed by an expansion cohort at the selected dose.

While the safety will be the primary endpoint for the dose escalation cohort, the auditory brainstem response (ABR) will be the primary efficacy endpoint of the dose expansion cohort. The CTA approval follows extensive preclinical studies assessing the safety and efficacy of SENS-501 and successful manufacturing of the gene therapy Drug Product for the clinical trial. Sensorion will communicate about the first patient in the course of H2 2024.

Sensorion?s SENS-501 (OTOF-GT) dual vector AAV gene therapy development program aims to restore hearing in patients with mutations in the gene coding for otoferlin protein who suffer from severe to profound sensorineural prelingual non-syndromic hearing loss. Sensorion?s lead gene therapy program has been developed as part of its collaboration focused on the genetics of hearing with the Institut Pasteur, which has been initiated in 2019. The Genetics and Physiology of the Hearing Unit of the Institut Pasteur, led by Professor Christine Petit, MD, PhD, has developed world-class expertise over the last 25 years in the molecular physiology and physiopathology of the hearing system.

Recent advances, conducted alongside Saaid Safieddine, PhD, have led to the development of the gene therapy product SENS-501.