Sensorion Submits Clinical Trial Application for Lead Gene Therapy Candidate OTOF-GT in the UK
10 Juillet 2023 - 7:30AM
Business Wire
Regulatory News:
Sensorion (FR0012596468 – ALSEN) (Paris: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 loss disorders, today announced that it
has submitted a first Clinical Trial Application (CTA) for OTOF-GT
to the UK’s Medicines and Healthcare products Regulatory Agency
(MHRA).
The phase 1/2 clinical trial (Audiogene), aims to evaluate the
safety, tolerability and efficacy of intra-cochlear injection of
OTOF-GT, for the treatment of otoferlin gene-mediated hearing loss
in pediatric patients aged up to 31 months. The CTA submission
follows extensive preclinical studies assessing the safety and
efficacy of OTOF-GT and successful manufacturing of the gene
therapy Drug Product for the clinical trial.
Sensorion’s OTOF-GT dual AAV vector gene therapy development
program aims to restore hearing in patients with mutations in OTOF
who suffer from severe to profound sensorineural prelingual non
syndromic hearing loss. Otoferlin is a protein expressed in the
inner hair cells (IHC) present in the cochlea and is critical for
the transmission of the signal to the auditory nerve. Otoferlin
related hearing loss is responsible for up to 8% of all cases of
congenital hearing loss, with around 20,000 people affected in the
US and Europe1. OTOF-GT previously received Orphan Drug Designation
from the US Food and Drug Administration (FDA)2 and the European
Medicines Agency (EMA)3 and Rare Pediatric Disease Designation from
the FDA in Q4 2022.
Nawal Ouzren, Chief Executive Officer of Sensorion,
commented: “This first gene therapy CTA filing is a major milestone
for our OTOF-GT program and Sensorion’s broader gene therapy
franchise. This is the first of several planned CTA filings and we
hope to commence patient recruitment soon, subject to the CTA
approval. We look forward to continuing to work with clinicians,
regulatory authorities and patient groups to address unmet and
underserved medical needs in the intricate hearing space.”
Géraldine Honnet, Chief Medical Officer of Sensorion,
added: “There are currently no approved drug for patients with
mutations of the gene encoding for otoferlin. Our goal is to
transform the standard of care for OTOF newborns, by reducing
dependence on cochlear implants, which would transform their
quality of life. We believe that gene therapy has the potential to
offer permanent solutions to patients with diseases caused by the
OTOF mutation and other inner ear diseases and we are really
excited to move OTOF-GT forward towards clinical development.”
Sensorion plans to submit the CTA in Europe in the coming
weeks.
About Sensorion
Sensorion is a pioneering clinical-stage biotech company, which
specializes in the development of novel therapies to restore, treat
and prevent 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 in the framework of its broad
strategic collaboration focused on the genetics of hearing with the
Institut Pasteur. OTOF-GT targets deafness caused by mutations of
the gene encoding for otoferlin and GJB2-GT targets hearing loss
related to mutations in GJB2 gene to potentially address important
hearing loss segments in adults and children. The Company is also
working on the identification of biomarkers to improve diagnosis of
these underserved illnesses. Sensorion’s portfolio also comprises
clinical-stage small molecule programs for the treatment and
prevention of hearing loss disorders. Sensorion’s clinical-stage
portfolio includes one Phase 2 product: SENS-401 (Arazasetron)
progressing in a planned Phase 2 proof of concept clinical study of
SENS-401 in Cisplatin-Induced Ototoxicity (CIO) and, with partner
Cochlear Limited, in a study of SENS-401 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
Disclaimer
This press release contains certain forward-looking statements
concerning Sensorion and its business. Such forward looking
statements are based on assumptions that Sensorion considers to be
reasonable. However, there can be no assurance that such
forward-looking statements will be verified, which statements are
subject to numerous risks, including the risks set forth in the
2022 full year financial report published on March 30, 2023, and
available on our website and to the development of economic
conditions, financial markets and the markets in which Sensorion
operates. The 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 actual results, financial
conditions, performance or achievements of Sensorion to be
materially different from such forward-looking statements. This
press release and the information that it contains do not
constitute an offer to sell or subscribe for, or a solicitation of
an offer to purchase or subscribe for, Sensorion shares in any
country. The communication of this press release in certain
countries may constitute a violation of local laws and regulations.
Any recipient of this press release must inform oneself of any such
local restrictions and comply therewith.
_______________________________ 1 Rodríguez-Ballesteros M,
Reynoso R, Olarte M, Villamar M, Morera C, Santarelli R, Arslan E,
Medá C, Curet C, V�lter C, Sainz-Quevedo M, Castorina P, Ambrosetti
U, Berrettini S, Frei K, Tedín S, Smith J, Cruz Tapia M, Cavallé L,
Gelvez N, Primignani P, Gómez-Rosas E, Martín M, Moreno-Pelayo MA,
Tamayo M, Moreno-Barral J, Moreno F, del Castillo I. A multicenter
study on the prevalence and spectrum of mutations in the otoferlin
gene (OTOF) in subjects with nonsyndromic hearing impairment and
auditory neuropathy. Hum Mutat. 2008 Jun;29(6):823-31. doi:
10.1002/humu.20708. PMID: 18381613. 2 FDA Orphan Drug Designations
and Approvals
https://www.accessdata.fda.gov/scripts/opdlisting/oopd/listResult.cfm
3 EU Community Register of orphan medicinal products
https://ec.europa.eu/health/documents/community-register/html/o2698.htm
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Investor Relations Noémie Djokovic, Investor Relations
and Communications (Europe/France)
ir.contact@sensorion-pharma.com
International Media Relations Consilium Strategic
Communications Jessica Hodgson/Sue Stuart/Isabelle Abdou +44 7561
424788 Sensorion@consilium-comms.com
Ulysse Communication Pierre-Louis Germain / 00 33 (0)6 64
79 97 51 plgermain@ulysse-communication.com
Bruno Arabian / 00 00(0)6 87 88 47 26
barabian@ulysse-communication.com
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