Sensorion Submits Clinical Trial Application for OTOF-GT, its Lead Gene Therapy Candidate, in Europe
July 19 2023 - 1:30AM
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 Clinical Trial Application (CTA) to initiate a
Phase 1/2 clinical trial of OTOF-GT in Europe (France, Italy and
Germany).
On July 10th, 2023, Sensorion submitted a first 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 administration
of OTOF-GT, for the treatment of otoferlin gene-mediated hearing
loss in pediatric patients aged from 6 to 31 months. The CTAs
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, Sensorion's Chief Executive Officer,
commented: "Our gene therapy programs, OTOF-GT being the most
advanced project, offer great hope for many children suffering from
congenital deafness, who today cannot benefit from approved
curative treatments. With this filing in Europe, we are taking the
final steps in preparing for the start of the clinical trial for
OTOF-GT and consolidating our position as one of the leading
players in the field of gene therapies applied to hearing loss. We
are fully focused on the development timetable and on revealing the
value of this portfolio of innovative drug candidates, while
respecting our commitments to patients, academic, hospital partners
and shareholders."
Géraldine Honnet, Chief Medical Officer of Sensorion,
added: “We are excited to advance OTOF-GT, our first gene therapy
program, to the clinic to address unmet medical need in the complex
hearing space. Pending regulatory authority approvals, we are
looking forward to recruiting the first patients aged from 6 to 31
months and offer them the potential of an innovative treatment to
correct their hearing loss due to otoferlin deficiency. Hearing
loss caused by mutations of the gene encoding for otoferlin is a
debilitating disorder with currently no approved drugs.”
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
Label: SENSORION ISIN: FR0012596468 Mnemonic:
ALSEN
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
Press Relations 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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