Sensorion Announces Completion of Enrollment of First Cohort of Audiogenic Gene Therapy Phase 1/2 Clinical Trial Investing.com
MONTPELLIER, France–(BUSINESS WIRE)–Regulatory news:
Sensorion (FR0012596468 “ ALSEN) a pioneering clinical biotechnology company specializing in the development of novel therapies for the restoration, treatment and prevention of hearing loss disorders, today announces the completion of patient enrollment in the first cohort of the Audiogene clinical gene therapy Phase 1/2 trial.
Recruitment of the first group (three patients) was completed as planned, and the third patient received an injection of the gene therapy product SENS-501 in December 2024. All patients treated in the first group tolerated the surgical procedure well: intra-cochlear administration of the gene therapy product was without problems and no serious side effects were reported. Audiogene is the first gene therapy clinical trial looking at a unique homogeneous population of infants and young children (aged 6 to 31 months at the time of injection) who did not have cochlear implants. Audiogene’s clinical trial design is intended to evaluate the ability of the SENS-501 gene therapy product not only to restore hearing, but also to enable infants and young children to acquire and develop normal speech.
Nawal Ouzren, Chief Executive Officer of Sensorion, said: I am very pleased with the progress Sensorion has made in its Phase 1/2 gene therapy clinical trial. The injection into the third and final patient from the first cohort, less than a year after the clinical trial application was approved, is a major achievement for Sensorion. The newborn’s surgery was uneventful and no serious side effects were reported. I am very excited about the KOL event we are planning in early 2025, with leading experts in the field, to present and comment on a more complete set of data measurements. I look forward to advancing SENS-501 and evaluating its ability to restore hearing and enable normal speech acquisition and development in treated young children and infants.
About the trial of Audiogene
Audiogene aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing loss in infants and young children aged 6 to 31 months at the time of gene therapy treatment. Targeting the early years of life, when brain plasticity is optimal, increases the chances that these young children with pre-linguistic hearing loss will acquire normal speech and language. The study consists of two two-dose groups followed by an extended dose-selected group. While safety will be the primary endpoint of the first part of the dose escalation study, the auditory brainstem response (ABR) will be the primary efficacy endpoint of the second part of the extension. Audiogene will also evaluate the clinical safety, performance and ease of use of the delivery system developed by Sensorion.
About SENS-501
SENS-501 (OTOF-GT) is an innovative gene therapy program developed to treat a specific form of congenital deafness associated with mutations in the OTOF (otoferlin) gene. This gene plays a key role in the transmission of auditory signals between the hair cells of the inner ear and the auditory nerve. When this gene is faulty, affected individuals are born with severe to profound hearing loss.
The goal of SENS-501 (OTOF-GT) is to restore hearing by introducing a functional copy of the OTOF gene directly into hair cells via viral vector technology (AAV). This therapy aims to re-establish the normal process of converting sound into electrical signals, allowing patients to regain their ability to hear.
Currently in clinical research, this gene therapy program offers significant hope for families affected by this rare form of genetic deafness. SENS-501 (OTOF-GT) embodies a commitment to scientific innovation in the field of hearing, with the potential to dramatically improve the quality of life of patients suffering from genetic deafness.
This gene therapy for patients suffering from otoferlin deficiency was developed within the framework of RHU AUDINNOVA, a consortium made up of Sensorion with the Hospital Necker Enfants Malades, the Institut Pasteur and the Fondation pour l’Audition. The project is partially financed by the French National Research Agency, through the program Investing for the future (ref: ANR-18-RHUS-0007).
About Sensorion
Sensorion is a pioneering clinical-stage biotechnology company specializing in the development of novel therapies for the restoration, treatment and prevention of hearing loss disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear-related diseases, enabling it to select the best targets and mechanisms of action for drug candidates.
It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed as part of a broad strategic collaboration focused on hearing genetics with the Pasteur Institute. SENS-501 (OTOF-GT) currently in Phase 1/2 clinical trial development, targets deafness caused by mutations in the gene encoding otoferlin, and GJB2-GT targets hearing loss associated with mutations in the GJB2 gene to potentially address significant loss hearing segments in adults and children. The company is also working to identify biomarkers to improve the diagnosis of these underserved diseases.
Sensorion’s portfolio also includes small molecule clinical programs for the treatment and prevention of hearing loss disorders. Sensorion’s clinical phase portfolio includes one Phase 2 product: SENS-401 (Arazasetron) which is progressing in the planned Phase 2 proof-of-concept clinical study of SENS-401 in cisplatin-induced ototoxicity (CIO) and, with partner Cochlear Limited, has completed the SENS-401 study in patients scheduled for cochlear implantation. A phase 2 study of SENS-401 was also completed in sudden sensorineural hearing loss (SSNHL) in January 2022.
www.sensorion.com
Mark: SENSORY
ISIN: FR0012596468
Mnemonic: ALSEN
Waiver
This press release contains certain forward-looking statements regarding Sensorion and its business. Such forward-looking statements are based on assumptions that Sensorion believes to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, and such forward-looking statements are subject to a number of risks, including the risks set forth in the 2023 full-year report published on March 14, 2024 and available on our website and the development of economic conditions, financial markets and the markets in which Sensorion operates. Forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause Sensorion’s actual results, financial conditions, performance or achievements to differ materially from such forward-looking statements. This press release and the information it contains do not constitute an offer to sell or subscribe for, or the solicitation of an offer to buy or subscribe for, Sensorion shares in any country. Communicating this press release in certain countries may violate local laws and regulations. Any recipient of this press release must inform themselves of such local restrictions and comply with them.
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Source: Sensorion
