Sensorion Reveals Positive Clinical Data in Gene Therapy and Small Molecule Programs at World Audiology Congress

Sensorion, a leading clinical-stage biotech firm focusing on hearing loss therapies, has unveiled promising new data from its SENS-501 and SENS-401 clinical programs at the 36th World Congress of Audiology in Paris, France. The company, known for developing innovative treatments to combat hearing disorders, shared findings from both gene therapy and small molecule initiatives.

Professors Natalie Loundon, Catherine Birman, Yann Nguyen, Stephen O’Leary, and Christophe Vincent, all esteemed experts in otorhinolaryngology, presented the significant medical advancements.

### Gene Therapy Developments

The SENS-501 program, aimed at addressing otoferlin deficiency—a prevalent cause of congenital deafness—showed positive initial results. Prof. Catherine Birman shared that the first patient in the Phase 1/2 clinical trial experienced well-tolerated surgery with no safety concerns. Notably, early observations indicated changes in the child's behavior and vocalizations, suggesting potential efficacy. This gene therapy targets children aged 6 to 31 months, capitalizing on the optimal plasticity of the auditory system during early development to enhance speech and language acquisition. The trial is structured with two dose cohorts followed by an expansion cohort, focusing on safety and auditory brainstem response (ABR) as primary endpoints.

### Small Molecule Advancements

The SENS-401 small molecule program yielded notable preliminary data for two key applications:

#### Cisplatin-Induced Ototoxicity (NOTOXIS)

Prof. Yann Nguyen discussed the Phase 2a trial results for SENS-401 in preventing hearing loss due to cisplatin ototoxicity in chemotherapy patients. The findings suggest that a cumulative cisplatin dose significantly impacts ototoxicity severity. SENS-401 demonstrated a favorable safety profile over extended administration periods, with preliminary data hinting at an otoprotective effect beyond a cisplatin dose of 300 mg/m². The NOTOXIS trial, a multicenter, randomized, controlled study, evaluates the efficacy of SENS-401 in adult patients post-chemotherapy, assessing ototoxicity rates, pure tone audiometry changes, and drug tolerability.

#### Cochlear Implantation

Prof. Stephen O’Leary and Prof. Christophe Vincent presented final data from the Phase 2a trial of SENS-401 for preserving residual hearing post-cochlear implantation. The analysis revealed clinically significant benefits in patients treated with SENS-401 compared to the control group. This multicentric, randomized, controlled open-label trial involves administering SENS-401 to adult patients seven days before and 42 days after cochlear implantation. The study aims to evaluate SENS-401 presence in the cochlea (perilymph) and its effects on hearing preservation.

### Sensorion’s Ongoing Research

Sensorion is committed to pioneering new treatments for hearing loss, a field with significant unmet medical needs globally. The company leverages a robust R&D platform to understand inner ear diseases' pathophysiology and etiology better, enabling precise target selection for drug candidates. Sensorion's portfolio includes two gene therapy programs: SENS-501, targeting otoferlin deficiency, and GJB2-GT, aimed at hearing loss due to GJB2 gene mutations. Additionally, the company focuses on identifying biomarkers to enhance the diagnosis and treatment of these conditions.

The small molecule portfolio features SENS-401, which is advancing in Phase 2 studies for cisplatin-induced ototoxicity and cochlear implantation. A Phase 2 study for sudden sensorineural hearing loss (SSNHL) concluded in January 2022.

### Conclusion

Sensorion's latest clinical data underscore its innovative approach to tackling hearing loss through both gene therapy and small molecules. The promising results from the SENS-501 and SENS-401 programs reflect the company's commitment to addressing various hearing impairments and improving patients' quality of life.