Sensorion, a clinical-stage biotechnology company specializing in hearing loss therapies, has announced the completion of enrollment for the first cohort of its Phase I/II Audiogene clinical trial. The trial is evaluating the safety, tolerability, and efficacy of SENS-501, a gene therapy, for treating hearing loss caused by mutations in the Otoferlin (OTOF) gene in infants and toddlers. This milestone marks a significant step forward in addressing a critical unmet need for young children with congenital deafness.
The Audiogene trial targets infants and toddlers aged 6 to 31 months who have not received cochlear implants. By focusing on this age group, when brain plasticity is at its peak, the trial aims to maximize the potential for these children to develop normal speech and language skills following hearing restoration. The trial design includes two cohorts receiving different doses of SENS-501, followed by an expansion cohort at the selected dose.
Safety and Tolerability
According to Sensorion, the surgical procedure for intra-cochlear administration of SENS-501 was well-tolerated in all three subjects in the first cohort, with no serious adverse events reported. Furthermore, initial data from the first two toddlers treated showed encouraging behavioral improvements. These findings suggest that SENS-501 has a favorable safety profile in this young patient population.
Nawal Ouzren, CEO of Sensorion, expressed enthusiasm about the trial's progress, stating, "I am very pleased with the progress Sensorion has realised in its Phase 1/2 gene therapy clinical trial... The surgery of the infant was uneventful, and no serious adverse events were reported. I am very excited about the KOL event we plan in early 2025, with leading field experts, to present and comment on a fuller set of data measurements."
SENS-501: AAV-Based Gene Therapy for OTOF-Related Hearing Loss
SENS-501 is an innovative gene therapy that utilizes an Adeno-Associated Virus (AAV) vector to deliver a functional copy of the OTOF gene directly into the hair cells of the inner ear. The OTOF gene encodes otoferlin, a protein crucial for transmitting auditory signals from the inner ear to the auditory nerve. Mutations in this gene result in severe to profound congenital hearing loss.
The goal of SENS-501 is to restore the normal auditory signal conversion process, enabling patients to regain their hearing ability. This approach holds significant promise for families affected by this rare form of genetic deafness, offering a potential alternative to cochlear implants, particularly in very young children.
Trial Design and Endpoints
The Audiogene trial's primary endpoint for the initial dose-escalation phase is safety. The primary efficacy endpoint for the expansion phase is auditory brainstem response (ABR), a measure of auditory nerve function. The trial will also assess the clinical safety, performance, and ease of use of Sensorion's delivery system.
Sensorion anticipates completing the recruitment of the second cohort by the end of H1 2025. The company plans to host a Key Opinion Leader (KOL) event in early 2025 to present and discuss the first cohort's safety data, qualitative and quantitative efficacy measurements, and the next steps for the Audiogene trial, including planned interactions with the U.S. Food and Drug Administration.
