Announcement • Jun 11
Sensorion Achieves Key Regulatory Milestones for Sens-601 for Gjb2-Related Hearing Loss and Selects Program as Lead Gene Therapy Candidate
Sensorion has selected SENS-601 which addresses GJB2-related hearing loss as its lead program. A Clinical trial application has been filed in Canada and France to evaluate the safety, tolerability and efficacy of intra-cochlear administration of SENS-601, for the treatment of GJB2 gene-mediated hearing loss in paediatric patients. Sensorion has recently submitted Clinical Trial Applications for SENS-601 (Hearconnex) in Canada, and in France, with Fast Track procedure granted by the French Agency (ANSM). The trial is intended to evaluate the safety, tolerability and efficacy of intra-cochlear administration of SENS-601, for the treatment of GJB2 gene-mediated hearing loss in pediatric patients. Hearconnex will also assess the clinical safety, performance and ease-of-use of the delivery system developed by Sensorion. The submissions follow productive pre-submission alignment discussions with the regulatory authorities, and the Company has high confidence in the completeness and scientific quality of the dossier. The Fast Track procedure granted in France provides a significantly reduced assessment period compared to the standard pathway. The IND submission with the FDA in the U.S. and the submission in Australia are on track by year-end 2026. Sensorion has decided to discontinue the clinical development activity for SENS-501 for OTOF-related hearing loss after conducting a strategic review of its therapeutic candidate pipeline and is consequently ending recruitment in the Audiogene trial. OTOF-related hearing loss is an ultra-rare condition, and the recent availability of a gene therapy demonstrating meaningful clinical efficacy in this indication has notably changed the development environment for SENS-501. In this context, the Board and management concluded that concentrating Sensorion’s resources on SENS-601, where the unmet need is an order of magnitude larger and no approved therapy is currently available, is the right strategic decision for the Company and its stakeholders. Sensorion extends gratitude to the clinical teams, investigators, and patients of the Audiogene trial for their contribution to advancing gene therapy for hearing loss. The scientific and operational platform built through this work, developed in close collaboration with Prof. Christine Petit’s team (Institut reConnect, Institut de l’Audition, Institut Pasteur, Inserm, CNRS), are directly transferrable to SENS-601 and provide a unique and foundational head start for the program. In accordance with applicable regulatory requirements, Sensorion remains fully committed to ensuring the long-term follow-up of all patients enrolled in the Audiogene trial. The cash runway is extended until the end of 2027, supporting the execution of the clinical advancement of SENS-601 and the generation of first in human data. SENS-601 (GJB2-GT) is an innovative AAV-based gene therapy program developed in collaboration with Prof. Christine Petit's team (Institut reConnect, Institut de l'Audition, Institut Pasteur, Inserm, CNRS) to treat hearing loss linked to mutations in the GJB2 gene, which plays a critical role in maintaining the ionic balance necessary for sound transduction in the inner ear. GJB2 mutations represent the most common cause of genetic congenital deafness, responsible for approximately 50% of autosomal recessive non-syndromic hearing loss. Recent research has also established that GJB2 mutations are found in early onset forms of severe presbycusis in adults, which appear to be monogenic potentially treatable by gene therapy. As such, SENS-601 has the potential to address three distinct pathologies: paediatric congenital deafness, progressive forms of hearing loss in children, and early onset of presbycusis in adults. With no approved gene therapy currently available for GJB2-related hearing loss, SENS-601 has the potential to be among the first gene therapy addressing GJB2 mutations. This program is partially funded by the French State as part of the France 2030 investment plan (ConnexGene project, with Bpifrance). SENS-501 (OTOF-GT) is an innovative gene therapy program developed to treat a specific form of congenital deafness linked to mutations in the OTOF (otoferlin) gene. This gene plays a key role in the transmission of auditory signals between the hair cells of the inner ear and the auditory nerve. When this gene is defective, affected individuals are born with severe to profound hearing loss. The aim of SENS-501 (OTOF-GT) is to restore hearing by introducing a functional copy of the OTOF gene directly into hair cells via viral vector technology (AAV). This therapy aims to restore the normal process of converting sound into electrical signals, enabling patients to regain their hearing ability. This gene therapy for patients suffering from otoferlin deficiency has been developed in the framework of RHU AUDINNOVE, a consortium composed of Sensorion with the Necker Enfants Malades Hospital, the Institut Pasteur, and the Fondation pour l’Audition. The project is partially financed by the French National Research Agency, through the “investing for the future” program (ref: ANR-18-RHUS-0007). The OTOF gene targeted by the Audiogene trial was discovered in 1999 at the Institut Pasteur, by Prof. Christine Petit's team (Institut reConnect, Institut de l'Audition, Institut Pasteur), who also unravelled the pathophysiology of the corresponding deafness (DFNB9).
