- Sensorion has received approval to initiate Phase 1/2
clinical trial of SENS-501 (OTOF-GT) in France as first
country
- SENS-501 is Sensorion’s lead gene therapy candidate. It aims
at restoring hearing in patients with mutations in the OTOF gene,
suffering from severe to profound hearing loss. It has been
developed in the frame of the strategic partnership with the
Institut Pasteur
- Audiogene, the Phase 1/2 clinical trial sponsored by
Sensorion aims to evaluate the safety, tolerability and efficacy of
SENS-501 in young children
Regulatory News:
Sensorion (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, today announces that it has
received approval for its Clinical Trial Application (CTA) to
initiate a Phase 1/2 clinical trial of SENS-501 (OTOF-GT), in
France. The conclusion of the Part I of the assessment report
according to regulation EU 536/2014 covering France, Italy and
Germany is that the conduct of the clinical trial is
acceptable.
The phase 1/2 clinical trial (Audiogene) aims to evaluate the
safety, tolerability, and efficacy of intra-cochlear injection of
SENS-501 for the treatment of OTOF gene-mediated hearing impairment
in paediatric patients aged 6 to 31 months at the time of gene
therapy treatment. Targeting the first years of life, the time
period when the auditory system plasticity is optimal, will
maximize the chances of these young children with pre-lingual
hearing loss to acquire normal speech and language. Audiogene will
also assess the clinical safety, performance, and usability of the
administration device system under development in partnership with
EVEON. The design of the study will consist of two cohorts of 2
doses followed by an expansion cohort at the selected dose. While
the safety will be the primary endpoint for the dose escalation
cohort , the auditory brainstem response (ABR) will be the primary
efficacy endpoint of the dose expansion cohort. The CTA approval
follows extensive preclinical studies assessing the safety and
efficacy of SENS-501 and successful manufacturing of the gene
therapy Drug Product for the clinical trial. Sensorion will
communicate about the first patient in the course of H2 2024.
Nawal Ouzren, Sensorion's Chief Executive Officer, said:
"Securing approval to start the phase 1/2 clinical trial for
SENS-501 marks a significant milestone for Sensorion and confirms
our position of a leading company in the emerging field of gene
therapies for hearing loss. Hearing loss caused by mutations of the
gene encoding for otoferlin is a challenging disorder and there are
no approved curative treatments for children with congenital
deafness, so this is a significant unmet medical need. We look
forward to continuing to work with healthcare providers, regulatory
authorities, patient groups to address unmet and underserved
medical needs in the hearing space.”
Professor Natalie Loundon, M.D., Director of the
Center for Research in Pediatric Audiology, Pediatric
Otolaryngologist and Head and Neck Surgeon, Necker Enfants Malades,
AP-HP, in Paris, France, Coordinating Investigator of the Audiogene
clinical study, commented: “SENS-501 represents a hope for
numerous children born with deafness linked to a defect in the
otoferlin gene and for whom no curative treatment exists today. The
administration of a single-injection therapy to very young children
requires state-of-the-art hospital technical platform and health
care givers team familiar with the handling of gene therapies. In
keeping with our philosophy of supporting innovation wherever
children's medical needs go unmet, we are delighted to be fully
involved from the very beginning in this pioneering clinical
trial.”
Sensorion’s SENS-501 (OTOF-GT) dual vector AAV gene therapy
development program aims to restore hearing in patients with
mutations in the gene coding for otoferlin protein who suffer from
severe to profound sensorineural prelingual non-syndromic hearing
loss. Sensorion’s lead gene therapy program has been developed as
part of its collaboration focused on the genetics of hearing with
the Institut Pasteur, which has been initiated in 2019.
The Genetics and Physiology of the Hearing Unit of the Institut
Pasteur, led by Professor Christine Petit, MD, PhD, has developed
world-class expertise over the last 25 years in the molecular
physiology and physiopathology of the hearing system. Recent
advances, conducted alongside Saaid Safieddine, PhD, have led to
the development of the gene therapy product SENS-501.
Professor Christine Petit, Professor at the Institut Pasteur
and Professor Emeritus at the Collège de France, France, Winner of
the Kavli Prize in 2018, commented: “This regulatory green
light for the inclusion of patients in France in one of the world's
first gene therapy trials in the field of hearing represents a
major achievement for the teams at the Institut de l'Audition
(Institut Pasteur center), and a crucial milestone in our strategic
collaboration with Sensorion. The SENS-501 program, which aims to
correct the deficiency of a gene responsible for congenital
deafness in order to restore hearing, is based on very solid
pioneering research elucidating the role of otoferlin and the
pathogenic processes elicited by otoferlin defect. Its success will
pave the way for other potential therapeutic innovations, based on
gene therapy in many forms of deafness and for thousands of
patients.”
Otoferlin is a protein expressed in the inner hair cells (IHC)
present in the cochlea and is critical for hearing by ensuring the
transmission of the acoustic signals to the auditory nerves.
Otoferlin related hearing loss is responsible for up to 8% of all
cases of congenital hearing loss, affecting around 20,000 people
per year in the US and Europe1. SENS-501 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.
Sensorion presented preclinical data that indicated the
potential for safe and efficient clinical translation of gene
therapy for otoferlin delivered by a dual AAV vector. SENS-501
administration in a DFNB9 mouse model (OTOF-KO) showed long-term de
novo expression of otoferlin in inner hair cells (IHCs) and ABR
(auditory brainstem response) restoration. Sensorion has also
developed, in Non-Human Primates (NHPs), an optimal surgical
procedure, similar to cochlear implantation, and an administration
device system, in partnership with EVEON, which demonstrated an
effective transduction rate of the targeted IHCs in NHPs. EVEON
designs and manufactures custom medical devices for the preparation
and delivery of drugs.
Denis Le Squer, Executive Director for the French non-profit
foundation for hearing “Fondation Pour l’Audition”, added: “The
launch of the Audiogene clinical trial is a major step forward for
deaf children with otoferlin defects and their parents but also
brings hope to people with genetic deafness. We are very proud that
our long-time support to French innovation, supported by Sensorion,
and to the teams of Prof. Petit at Institut de l'Audition, center
of the Institut Pasteur, and Prof. Loundon, at the Clinical Center
for Research in Pediatric Audiology at AP-HP Necker hospital,
translates now into a trial.”
This gene therapy for patients suffering from otoferlin
deficiency has been developed in the framework of RHU AUDINNOVE, a
consortium composed of Sensorion with the Necker Enfants Malades
Hospital, the Institut Pasteur, and the Fondation pour l’Audition.
The project is partially financed by the French National Research
Agency, through the “investing for the future” program (ref:
ANR-18-RHUS-0007).
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. SENS-501 (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 Noemie Djokovic, Investor Relations
and Communications Associate ir.contact@sensorion-pharma.com
International Media Relations Ulysse Communication
Pierre-Louis Germain / Bruno Arabian +33 (0)6 64 79 97 51 / +33
(0)6 87 88 47 26 plgermain@ulysse-communication.com
barabian@ulysse-communication.com
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