Sensorion has reported a six-month update from the Audiogene Phase I/II clinical trial’s cohort 2 assessing SENS-501, its gene therapy candidate for otoferlin-mediated congenital deafness.

The study’s recent data indicate that early improvements in pure-tone audiometry, seen at month three in two of three patients receiving a higher SENS-501 dose (cohort 2), persisted at the six-month follow-up evaluation.

This aligns with earlier observations from cohort 1, where initial auditory pathway activation signs appeared at a lower dose, supporting evidence of a dose-response relationship in the two cohorts.

According to Sensorion, all six participants in the dose-escalation phase tolerated the surgical and intra-cochlear administration of the gene therapy well, with no serious adverse events or side effects reported.

The trial is designed to assess the efficacy, safety and tolerability of intra-cochlear SENS-501 injection for otoferlin gene-mediated hearing loss in infants and toddlers aged six to 31 months at treatment time.

It includes two dosing cohorts followed by an expansion group receiving the selected dose.

The primary endpoint in the dose escalation phase is safety while auditory brainstem response will serve as the main efficacy endpoint during expansion. The delivery system developed by Sensorion is also being evaluated for clinical safety and ease of use.

The company is considering adding a third dose level to the trial and will consult regulatory authorities accordingly.

Sensorion chairman and interim CEO Amit Munshi said: “The data generated through Audiogene validates the key components of our gene therapy platform, from surgical delivery and safety through dose escalation to the observation of a dose-response relationship.

“This growing body of clinical and procedural experience, together with the relationships we have built with leading clinical centres, regulatory agencies, and our partners, provides further confidence in the advancement of SENS-601, our potential first-in-human gene therapy programme targeting GJB2-related hearing loss.”

In February 2025, Sensorion’s Audiogene Phase I/II trial of the SENS-501 gene therapy programme received a favourable recommendation from the Data Monitoring Committee (DMC) to continue.