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- Accelerating development of the OTOF-GT and GJB2-GT gene therapy programs in collaboration with Institut Pasteur
- Advancing SENS-401 joint clinical development program with Cochlear Ltd. for hearing preservation in patients scheduled for cochlear implantation with first patient enrollment expected by mid-2022
- Sensorion will continue the proof-of-concept clinical study for Cisplatin-Induced Ototoxicity following a thorough analysis of the secondary endpoints of the AUDIBLE-S SENS-401 study
- In line with a disciplined capital allocation approach, Sensorion will explore partnering opportunities for SENS-401 in SSNHL
- Cash position of €50 million at year-end provides extended cash runway until end of Q2 2023
Regulatory News:
Sensorion (PARIS:ALSEN) (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing disorders, announces today its full-year 2021 financial results and provides an update on its business activities and outlook for 2022.
“In 2021, Sensorion made progress across its gene therapy and small molecule programs aimed at treating hearing loss disorders,” said Nawal Ouzren, CEO of Sensorion. “We made good progress with our gene therapy programs, notably OTOF-GT for the treatment of pediatric deafness due to otoferlin deficiency and GJB2-GT for the treatment of GJB2-driven deafness in collaboration with Institut Pasteur. In parallel, we have been expanding our gene therapy CMC platform by strengthening our development lab capabilities and reinforcing our in-house expertise.
“In our small molecule portfolio, we are advancing our existing partnership with Cochlear Limited with a clinical trial of our drug candidate SENS-401 for hearing preservation in patients scheduled for cochlear implantation. We see a compelling unmet medical need and a potential clinical development pathway for SENS-401 in Cisplatin-Induced Ototoxicity and will conduct our proof-of-concept clinical study, with a view to exploring partnership opportunities.
“During the year, we also further strengthened our Board of Directors with the appointments of Scott D. Myers as Chairman, and Dr. Aniz Girach as Independent Board Member. Both have decades of relevant, world-class scientific, medical and governance experience, and have already provided important support and guidance to Sensorion.”
Key developments in 2021: science and operational
Gene therapy programs
- OTOF-GT
Sensorion received scientific advice from regulatory agencies on the preclinical and clinical development plans for OTOF-GT, the Company’s dual vector AAV gene therapy program for the treatment of children born with hearing loss caused by Otoferlin deficiency.
The European Medicines Agency’s advisors welcomed the ongoing Natural History Study, Audioferline (NCT04202185), a component of the AUDINNOVE project coordinated by researchers at Hôpital Necker-Enfants malades (Necker Hospital) in partnership with Sensorion. Sensorion is expanding the study across Europe to document the natural course of disease progression in otoferlin deficiency patients, define clinically meaningful endpoints suitable for market approval, and identify the patient populations that would benefit the most from Sensorion’s OTOF-GT treatment. The Natural History Study will enable Sensorion to select the most relevant and clinically meaningful endpoints and clinical trial design as OTOF-GT progresses into the clinic.
At the Association for Research in Otolaryngology (ARO) 45th MidWinter Meeting in February 2022, Sensorion presented a poster on OTOF gene therapy. The data indicated potential for safe and efficient clinical translation of gene therapy for Otoferlin delivered by a dual AAV vector. The chosen capsid of AAV-OTOF in both mouse and non-human primate (NHP) models targets Inner Hair Cells (IHCs) and not Outer Hair Cells. Otof de novo expression in IHCs in a DFNB9 mouse model (OTOF-KO) demonstrates long-term expression of Otoferlin and hearing restoration up to one year post injection. In NHPs, the surgical procedure similar to cochlear implantation has been optimized to achieve an effective transduction rate of the targeted IHCs at levels compatible with therapeutic intervention in humans.
Sensorion goal is to start producing toxicological batches for OTOF-GT at intended clinical-scale volumes by mid-2022. The company is on track to file a Clinical Trial Application (CTA) for its OTOF-GT program in H1 2023.
- GJB2-GT
On February 15, 2021, Sensorion announced its largest gene therapy program to date, a collaboration with Institut Pasteur, targeting the GJB2 gene in pediatric and adult deafness. Research by Institut Pasteur demonstrated that anomalies in GJB2 are both the most common cause of congenital deafness as well as a wide contributor of severe age-related hearing loss in adults. Although the types of GJB2 Mutation in children and adults may differ, gene therapy could potentially provide solutions for both.
Sensorion’s GJB2 gene therapy programs have the potential to address three pathologies related to GJB2 mutations: early onset of presbycusis in adults, progressive forms of hearing loss in children, and pediatric congenital deafness. Sensorion plans to select a candidate by mid-2022.
- SONOVA
In September 2021, Sensorion announced the signing of an important multi-year collaboration with Sonova, a leading international player in the hearing solutions market. The collaboration aims to create new diagnostic and therapeutic solutions for hearing loss and expands a commitment made between the two companies in December 2020, when Sonova acquired a 3.7% stake in Sensorion.
Part of the collaboration is a jointly funded study of natural history in age-related progressive hearing loss (presbycusis) in adults. It will involve the collection of disease information and samples via selected Sonova Audiological Care stores. The collaboration could lead to the introduction of genetic analysis to the routine diagnosis of progressive hearing loss in adults and subsequently open the way for improved care through a combination of advanced therapeutic interventions and traditional hearing solutions including hearing aids. Sonova and Sensorion will jointly fund the study with €7.0 million, split 70 /30%, respectively, between the two companies.
- USHER-T1-GT
During 2021, Sensorion completed the preclinical proof-of-concept study for its gene therapy approach in Usher’s syndrome type 1G (USHER-GT). This study was designed to test whether a gene therapy approach would be effective in older mice, thereby opening the possibility of extended treatment windows for clinical studies.
The results of the completed study show that there is a full restoration of the vestibular function, but that audition cannot be restored at a satisfactory level. In line with a focused approach to capital allocation, Sensorion has decided to terminate this program. The Master Research Agreement provides us the possibility to explore other opportunities with Institut Pasteur.
SENS-401
- SENS-401 Cochlear
At the beginning of 2021, Sensorion…
Full story available on Benzinga.com
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