Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company specializing in the development of novel therapies to restore, treat and prevent hearing loss disorders, announced today latest data updates confirming the safety of SENS-501 in the two first patients injected in the Company’s Audiogene study. This is a Phase 1/2 clinical trial evaluating SENS-501 in patients aged 6 to 31 months and naïve of cochlear implant, who are suffering from a specific form of congenital deafness linked to mutations in the OTOF (otoferlin) gene.
The Audiogene clinical trial assesses, as primary endpoint in the first part of the dose escalation study, the safety of an intra-cochlear injection of SENS-501 in infants and toddlers aged 6 to 31 months at the time of gene therapy treatment. Sensorion’s objective in targeting the first years of life, when brain plasticity is optimal, is to maximize the chances of these young children with pre-linguistic hearing loss to acquire normal speech and language, thus, potentially transforming these toddlers’ lives. For the first two toddlers treated in the first cohort, SENS-501 gene therapy product and surgical procedure were well tolerated: the intra-cochlear administration of the gene therapy product was uneventful, and no serious adverse events were reported. In addition, encouraging behavioural improvements were observed in both toddlers. Sensorion plans on hosting a KOL event in early 2025 to further comment and discuss the first cohort safety data, qualitative and quantitative efficacy measurements, and next steps for Audiogene Phase 1/2 clinical trial, including planned interactions with the U.S. Food and Drug Administration. Further details regarding the event shall be disclosed in due course.
The Company continues to anticipate the completion of the first cohort enrollment by the end of the year and the recruitment of the second cohort by the end of H1 2025.
Géraldine Honnet, M.D., Chief Medical Officer of Sensorion, said: “I’m very pleased with the progress made in the Audiogene Phase 1/2 clinical trial evaluating SENS-501, a potentially game changing hearing loss therapeutic being developed in collaboration with the Institut Pasteur. Today’s data results confirm an excellent safety profile for the first toddlers treated with SENS-501 and I am happy to report early signs of encouraging behavioural changes in both patients. We look forward to treating the third patient of the first cohort imminently, thus achieving an important development milestone for Sensorion. We will host a KOL call in early 2025 and I am looking forward to presenting further safety and efficacy data for patients included in our first cohort.
I am confident that Sensorion’s differentiated clinical approach will set new standards in the field of gene therapy for otoferlin deficiency as the study has indeed been designed to assess whether SENS-501 can demonstrate not only hearing restoration in a very homogeneous population of infants and toddlers but also enable language acquisition and development, and the resulting significant improvements in quality of life. I would like to reiterate my gratitude to the parents of the treated patients for their trust, and to the participating investigators for their ongoing commitment to the Audiogene study and conviction in the potential of SENS-501 to create a new treatment paradigm for this debilitating form of deafness.”
Professor Natalie Loundon, M.D., Director of the Center for Research in Pediatric Audiology, Pediatric Otolaryngologist and Head and Neck Surgeon, Necker Enfants Malades, AP-HP, in Paris, France, Coordinating Investigator of the Audiogene clinical study, commented: “The preliminary results provide satisfactory data on the safety of SENS-501 for patients. Gene therapy represents real hope for a therapeutic treatment and improvement hearing, speech acquisition and quality of life for children born deaf due to DFNB9 mutations. I look forward to the next data update on Audiogene’s first cohort, where patients will have received the lowest dose investigated for the restoration of hearing in DFNB9 patients and I’m excited to continue our work on the Audiogene clinical study.”