Sensorion Receives Approval for Phase 1/2 Trial of SENS-501 Gene Therapy in Europe

Sensorion, a clinical-stage biotechnology firm renowned for its innovative approaches to hearing disorder treatments, has been granted approval to launch the Phase 1/2 clinical trial of its lead gene therapy candidate, SENS-501 (OTOF-GT), in France. This marks a significant advancement in the field of genetic therapies for hearing loss, as SENS-501 is designed to address severe to profound sensorineural hearing loss caused by mutations in the otoferlin gene.

The clinical trial, known as Audiogene, will focus on the safety, tolerability, and efficacy of SENS-501 in pediatric patients aged between 6 to 31 months. The treatment aims to capitalize on the optimal plasticity of the auditory system during early childhood, thereby increasing the likelihood of affected children developing normal speech and language skills. The trial will also evaluate a novel administration device system, developed in collaboration with EVEON, ensuring the safe and effective delivery of the therapy.

The approval to proceed with the trial follows a comprehensive review of preclinical studies that demonstrated the safety and efficacy of SENS-501. Sensorion anticipates sharing updates on the first patient enrolled in the trial in the second half of 2024.

Nawal Ouzren, CEO of Sensorion, highlighted the significance of this approval, stating it solidifies Sensorion's leading position in the burgeoning area of gene therapy for hearing loss. The absence of curative treatments for children with congenital deafness due to otoferlin gene mutations underscores the pressing need for innovative solutions like SENS-501.

Professor Natalie Loundon, Coordinating Investigator of the Audiogene clinical study and a leading expert in pediatric audiology, emphasized the potential of SENS-501 to offer hope to children born with deafness due to otoferlin gene defects. The single-injection therapy requires a sophisticated hospital setup and a skilled team adept in gene therapy administration.

The development of SENS-501 is a result of Sensorion's strategic partnership with the Institut Pasteur, initiated in 2019, focusing on the genetics of hearing. The Genetics and Physiology of Hearing Unit at the Institut Pasteur, under the leadership of Professor Christine Petit, has been instrumental in advancing the understanding of the molecular physiology of hearing, leading to the creation of SENS-501.

Otoferlin, a protein crucial for transmitting acoustic signals to the auditory nerves, is linked to a significant percentage of congenital hearing loss cases. SENS-501 has received Orphan Drug Designation from both the US FDA and the EMA, reflecting its potential to benefit patients with rare conditions.

Preclinical data presented by Sensorion suggest that the dual AAV vector used in SENS-501 could safely and efficiently translate into clinical applications. The therapy has shown promising results in a DFNB9 mouse model, demonstrating the restoration of auditory brainstem response. Additionally, Sensorion, in partnership with EVEON, has developed an optimal surgical procedure and administration device system, which has proven effective in Non-Human Primates.

Denis Le Squer, Executive Director of "Fondation Pour l’Audition," expressed pride in the support provided to French innovation and the teams at Institut de l'Audition and the Clinical Center for Research in Pediatric Audiology, which has now culminated in the Audiogene trial.

Sensorion's commitment to addressing hearing loss disorders extends beyond gene therapy. The company is also engaged in the development of small molecule programs and is working on identifying biomarkers to enhance the diagnosis of hearing loss conditions. Its Phase 2 product, SENS-401 (Arazasetron), is being studied for its potential in treating Cisplatin-Induced Ototoxicity and Sudden Sensorineural Hearing Loss, in addition to its application in cochlear implant patients.

Sensorion's dedication to pioneering research and development in hearing loss therapies positions it at the forefront of medical innovation, offering hope to those affected by hearing disorders and their families.