Following Regeneron approval, Sensorion selects new lead hearing loss therapy

Sensorion has selected SENS-601 — an AAV-based gene therapy that addresses GJB2-related hearing loss — as its lead program, abandoning its OTOF-related hearing loss candidate in the wake of a rival approval.

The company has filed a clinical trial application 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 pediatric patients. The IND submission with the FDA in the U.S. and the submission in Australia are on track by year-end 2026.

In conjunction with this, Sensorion has decided to discontinue the clinical development activity for SENS-501 for OTOF-related hearing loss and ending recruitment in the Audiogene trial. Back in April, the FDA granted accelerated approval to Regeneron’s Otarmeni, an AAV vector-based gene therapy indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss associated with molecularly confirmed biallelic variants in the OTOF gene. Regeneron also announced it’s decision to make the therapy available for free in the U.S.

Given that OTOF-related hearing loss is an ultra-rare condition, Sensorion will concentrate the company’s resources on SENS-601, “where the unmet need is an order of magnitude larger and no approved therapy is currently available.” GJB2 mutations represent the most common cause of genetic congenital deafness, responsible for approximately 50% of autosomal recessive non-syndromic hearing loss. As such, SENS-601 has the potential to address three distinct pathologies: pediatric congenital deafness, progressive forms of hearing loss in children, and early onset of presbycusis in adults.