Sensorion To Focus on Paediatric Patients For Gene Therapy

Pioneering clinical-stage biotechnology company Sensorion announces 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 Part I of the evaluation report according to Regulation EU 536/2014 of 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, as per pharmabiz.

Audiogene is also evaluating the clinical safety, efficacy and usability of the delivery device system being developed in collaboration with EVEON. The study design consists of two 2-dose cohorts followed by a dose-selected extension cohort. While safety is the primary endpoint in the dose-escalation cohort, auditory brainstem response (ABR) is the primary efficacy endpoint in the dose-escalation cohort..

Nawal Ouzren, Sensorion's chief executive officer, said: "Securing approval to start the phase 1/2 clinical trial for SENS-501 marks a significant milestone for Sensorion and confirms our position of a leading company in the emerging field of gene therapies for hearing loss. Hearing loss caused by mutations of the gene encoding for otoferlin is a challenging disorder and there are no approved curative treatments for children with congenital deafness, so this is a significant unmet medical need. We look forward to continuing to work with healthcare providers, regulatory authorities, patient groups to address unmet and underserved medical needs in the hearing space.