Sensorion Shares Audiogene Phase 1/2 Gene Therapy Trial Data

In recent developments from the biotechnology sector, Sensorion, a clinical-stage company based in Montpellier, France, has released promising updates on its ongoing Audiogene clinical trial. Sensorion is pioneering efforts to develop innovative treatments for hearing loss disorders, and the latest data focuses on the safety profile of its gene therapy product, SENS-501, targeting a specific type of congenital deafness caused by mutations in the otoferlin (OTOF) gene.

The Audiogene trial is in Phase 1/2, assessing the safety and initial efficacy of SENS-501 through intra-cochlear injections in infants and toddlers aged between 6 to 31 months, who have not undergone cochlear implantation. The primary goal in this early stage is to evaluate the safety of the gene therapy when administered directly into the cochlea, the auditory region of the inner ear. Sensorion's approach targets young children to exploit the brain's plasticity, which is most receptive to language development early in life. The hope is that this treatment can significantly influence speech and language acquisition, markedly impacting the lives of those affected by pre-linguistic hearing loss.

The trial's first two patients experienced smooth procedures without any serious adverse events, suggesting a strong safety profile for SENS-501. Additionally, both patients showed encouraging signs of behavioral improvements, a promising indicator of the treatment's potential efficacy. Sensorion plans to host a Key Opinion Leader (KOL) event in early 2025 to delve deeper into these findings, discuss qualitative and quantitative efficacy measures, and outline future plans, including interactions with the U.S. Food and Drug Administration (FDA).

By the year's end, Sensorion aims to complete the enrollment for its first cohort and proceed with recruiting participants for the second cohort by mid-2025. Géraldine Honnet, M.D., Sensorion’s Chief Medical Officer, expressed satisfaction with the trial's progress and its potential to redefine treatment standards for otoferlin deficiency. She emphasized the importance of the trial's design, which not only seeks to restore hearing in a uniformly affected population but also aims to foster language development and overall quality of life improvements.

Professor Natalie Loundon, the Coordinating Investigator of the Audiogene study, shared her optimism regarding the preliminary safety data. She highlighted the potential of gene therapy to transform treatment paradigms for children with DFNB9 mutations, which are linked to congenital hearing loss. The upcoming data updates are highly anticipated, especially as they will provide insights from the lowest dose administered in the trial.

The Audiogene trial is structured to evaluate both the safety and efficacy of SENS-501, focusing initially on the safety of variable doses and later on the efficacy as measured by auditory brainstem responses. Furthermore, the trial will assess the performance of Sensorion's delivery system for the gene therapy.

SENS-501, also known as OTOF-GT, is designed to treat deafness related to otoferlin gene mutations by introducing a functional version of the OTOF gene directly into the ear's hair cells using viral vector technology. By correcting the genetic defect, this therapy aims to restore the natural hearing process, offering significant hope to those affected by this rare genetic condition.

Sensorion's efforts are part of a larger initiative within the RHU AUDINNOVE consortium, which includes esteemed institutions like Necker Enfants Malades Hospital and the Institut Pasteur. Partially funded by the French National Research Agency, this project underscores the commitment to scientific advancement in addressing genetic hearing loss.

In summary, Sensorion's progress with SENS-501 in the Audiogene trial represents a significant step forward in the field of gene therapy for hearing loss, with early results underscoring both safety and the beginnings of clinical efficacy. This study could pave the way for groundbreaking treatments that not only restore hearing but also enhance speech and language development in young children affected by congenital deafness.