Sensorion Granted Approval for Phase 1/2 Trial of SENS-501 Gene Therapy

A pioneering clinical-stage biotechnology company, Sensorion, has received the green light to initiate a Phase 1/2 clinical trial for its gene therapy candidate, SENS-501 (OTOF-GT), in France. This therapy is designed to address hearing loss caused by mutations in the otoferlin gene, a condition that affects a significant number of individuals annually.

The trial, known as Audiogene, will focus on the safety, tolerability, and efficacy of SENS-501 in treating pediatric patients aged between 6 to 31 months at the time of treatment. The decision to target this age group is rooted in the understanding that the auditory system's plasticity is at its peak during the first years of life, offering the best chance for children with pre-lingual hearing loss to develop normal speech and language skills.

The study is structured with two cohorts receiving different doses, followed by an expansion cohort at the selected dose. Safety will be the primary endpoint for the initial dose escalation cohort, while the auditory brainstem response (ABR) will serve as the primary efficacy endpoint for the dose expansion cohort. The approval to proceed with the clinical trial comes on the heels of extensive preclinical studies that have demonstrated the safety and efficacy of SENS-501.

Sensorion's collaboration with the Institut Pasteur has been instrumental in the development of SENS-501. The Genetics and Physiology of Hearing Unit at the Institut Pasteur, led by Professor Christine Petit, has been at the forefront of research into the molecular physiology and pathophysiology of the hearing system for the past 25 years. Their work has been pivotal in advancing the understanding of the otoferlin gene's role in congenital deafness and the development of potential gene therapies.

Otoferlin is a protein crucial for the transmission of acoustic signals to the auditory nerves, and its deficiency can lead to congenital hearing loss. SENS-501 has received Orphan Drug Designation from both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), highlighting its potential as a treatment for a rare condition with significant unmet medical needs.

Preclinical data presented by Sensorion suggest that the dual AAV vector approach to delivering the otoferlin gene could be both safe and effective. The company has also developed an optimal surgical procedure and an administration device system in partnership with EVEON, which has shown promising results in Non-Human Primates (NHPs).

The Audiogene clinical trial is a significant step forward in the treatment of genetic deafness and brings hope to affected children and their families. It is a testament to the collaborative efforts of Sensorion, the Institut Pasteur, and healthcare providers in addressing a critical medical need.

Sensorion's commitment to developing novel therapies for hearing loss disorders is evident through its unique R&D technology platform. The company is also working on the identification of biomarkers to improve the diagnosis of these conditions. In addition to its gene therapy programs, Sensorion has clinical-stage small molecule programs aimed at treating and preventing hearing loss disorders, including a Phase 2 product, SENS-401, which is being studied for its potential in treating Cisplatin-Induced Ototoxicity and Sudden Sensorineural Hearing Loss.

The development of SENS-501 and the Audiogene clinical trial represent a significant milestone in the field of gene therapy for hearing loss. It underscores the importance of collaboration between academia, industry, and healthcare providers in advancing medical science and improving the lives of those affected by debilitating conditions.