Sensorion's SENS-501 Shows Promise in Gene Therapy for OTOF-Related Hearing Loss
核心洞察
Sensorion's SENS-501 Audiogene trial treated its first patient, showing initial safety and behavioral changes, with cohort completion expected by year-end.
Final data from the SENS-401 Phase 2a trial indicates it preserves residual hearing after cochlear implantation, showing better outcomes at multiple frequencies.
Preliminary data from the SENS-401 NOTOXIS trial suggests a potential protective effect against Cisplatin-Induced Ototoxicity (搜索), especially at higher doses.
Sensorion, a clinical-stage biotechnology company specializing in hearing loss (搜索) therapies, has reported significant progress in its clinical programs. The company presented updates on its gene therapy program SENS-501 and its small molecule candidate SENS-401 at the World Congress of Audiology (WCA) in Paris.
SENS-501 Audiogene Trial: First Patient Treated
The Phase 1/2 Audiogene clinical trial of SENS-501 (OTOF (搜索)-GT) has treated its first patient, a child with severe to profound hearing loss (搜索) due to mutations in the OTOF gene. The OTOF gene encodes otoferlin, a protein crucial for transmitting auditory signals from inner ear hair cells to the auditory nerve. Sensorion's gene therapy aims to restore hearing by delivering a functional copy of the OTOF gene directly into hair cells using an adeno-associated virus (AAV) vector.
The first patient, enrolled in Australia under the care of Professor Catherine Birman, tolerated the intra-cochlear injection of SENS-501 well, with no serious adverse events reported. Although an upper respiratory infection at the time of the first efficacy assessment (ABR at Month 1) affected reliable hearing sensitivity measurements, early observations noted changes in the child’s behavior and vocalizations.
Sensorion anticipates completing the first cohort of three patients by the end of 2024 and publishing initial efficacy data by year-end. Completion of the second cohort is planned for the first half of 2025.
SENS-401: Preserving Residual Hearing After Cochlear Implantation
Sensorion also announced final results from its Phase 2a clinical trial of SENS-401 for preserving residual hearing after cochlear implantation. The study, conducted in collaboration with Cochlear Limited, was a multicenter, randomized, controlled, open-label trial.
The study met its primary endpoint, confirming the presence of SENS-401 in the perilymph at levels compatible with potential therapeutic efficacy seven days after the start of treatment. Final results indicated that SENS-401 treatment led to systematically better preservation of residual hearing at all frequencies compared to the control group seven weeks post-treatment (six weeks post-implantation). This protective effect persisted eight weeks after cessation of treatment (14 weeks post-implantation). Notably, 40% of SENS-401-treated patients showed complete hearing preservation, compared to 0% in the control group.
Stephen O’Leary, Head of Otorhinolaryngology at the University of Melbourne, noted that the results support the conclusion that SENS-401 preserves residual hearing in patients undergoing cochlear implantation, which is crucial for maintaining and improving the quality of hearing.
SENS-401: Preventing Cisplatin-Induced Ototoxicity
Preliminary data from the NOTOXIS Phase 2a trial of SENS-401 in preventing Cisplatin-Induced Ototoxicity (搜索) (CIO) were also presented. The study is a multi-center, randomized, controlled, and open-label trial in adult subjects with neoplastic disease. Preliminary analyses were conducted on 16 patients (7 in the SENS-401 group, 9 in the control group).
Preliminary results suggest a potential trend toward an otoprotective effect of SENS-401, particularly beyond a cisplatin dose of 300 mg/m². The incidence of ototoxicity in the control group was consistent with published data, while most SENS-401-treated participants showed only mild ototoxicity, despite receiving higher cumulative doses of cisplatin.
Yann Nguyen, ENT Surgeon at Pitié Salpêtrière Hospital in Paris, expressed satisfaction with the drug's safety profile and the otoprotective trend suggested by the preliminary data.
Financial Position
Sensorion reported a strong financial position, with €87.3 million in cash and cash equivalents as of June 30, 2024, sufficient to fund operations until the end of 2025.