Sensorion Submits Clinical Trial Application for Lead Gene Therapy Candidate OTOF-GT in the UK

Regulatory News:

Sensorion (FR0012596468 – ALSEN) (Paris:ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, today announced that it has submitted a first Clinical Trial Application (CTA) for OTOF-GT to the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

The phase 1/2 clinical trial (Audiogene), aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of OTOF-GT, for the treatment of otoferlin gene-mediated hearing loss in pediatric patients aged up to 31 months. The CTA submission follows extensive preclinical studies assessing the safety and efficacy of OTOF-GT and successful manufacturing of the gene therapy Drug Product for the clinical trial.

Sensorion’s OTOF-GT dual AAV vector gene therapy development program aims to restore hearing in patients with mutations in OTOF who suffer from severe to profound sensorineural prelingual non syndromic hearing loss. Otoferlin is a protein expressed in the inner hair cells (IHC) present in the cochlea and is critical for the transmission of the signal to the auditory nerve. Otoferlin related hearing loss is responsible for up to 8% of all cases of congenital hearing loss, with around 20,000 people affected in the US 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 Pediatric Disease Designation from the FDA in Q4 2022.

Nawal Ouzren, Chief Executive Officer of Sensorion, commented: “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.”

Géraldine Honnet, Chief Medical Officer of Sensorion, added: “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.”