Sensorion Makes Progress in Trial for OTOF Hearing Loss Gene Therapy
The data monitoring committee reviewed safety data from the trial’s first cohort, which included 3 patients, and deemed the findings favorable.
Sensorion’s phase 1/2 AUDIOGENE clinical trial (NCT06370351), which is evaluating investigational adeno-associated virus (AAV) vector-based gene therapy SENS-501 (also known as OTOF-GT) for the treatment of otoferlin (OTOF) gene-mediated hearing loss, has received clearance from its data monitoring committee (DMC) to move onto dosing its second dose cohort.1
The DMC reviewed safety data from the trial’s first cohort, which included 3 patients, and deemed the findings favorable. As such, it has recommended that Sensorion proceed with dosing the second cohort, which will utilize a higher dose and include 3 additional patients. The trial is open to children aged 6 to 31 months and involves intracochlear administration of SENS-501. The administration method was characterized as “well-tolerated” in the first cohort.
Sensorion expects that recruitment of patients for the second cohort will be complete before the midway point of this year. Pending the results of the second cohort, a dose will be selected from the 2 doses evaluated and the trial will move onto an expansion cohort at this dose.
“On behalf of the Sensorion team, I wish to thank the DMC members for their review of Audiogene’s safety data generated to date and I am pleased with the Committee’s recommendation to proceed with the trial without modifications,” Nawal Ouzren, MSc, the CEO of Sensorion, said in a statement.1
According to the clinicaltrials.gov page for AUDIOGENE, which was most recently updated on September 26, 2024, the trial is currently recruiting at locations in Westmead, Australia, and Paris, France. The study, which was launched on June 21, 2024, is expected to enroll approximately 12 patients in total. In addition to evaluating the safety of the gene therapy itself, Audiogene will also evaluate the safety and efficacy of the surgical procedure for intracochlear injection and a device system developed by EVEON that is used for administration.2 Efficacy of SENS-501 will be assessed in the dose expansion portion via auditory brainstem response.
In December 2024, Sensorion reported initial data from the first 2 patients treated in AUDIOGENE’s first cohort.3 The company stated that no serious adverse events occurred and that encouraging behavioral improvements were seen in both children.
“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,” Géraldine Honnet, MD, the chief medical officer of Sensorion, said in a statement.3 “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 behavioral changes in both patients... We will host a key opinion leader 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.”
SENS-501 is intended to deliver a functional copy of the OTOF gene via a dual AAV vector for assembly in place.4 The gene therapy product has previously been granted orphan drug designation (ODD) and rare pediatric disease designation from the FDA and ODD from the European Medicines Agency.5,6
