- First patient treated in SENS-501 Audiogene clinical study;
well on-track for first cohort recruitment completion by year-end
2024
- Final data to be presented from the Phase 2a study of
SENS-401 in the preservation of residual hearing after Cochlear
Implantation at the World Congress of Audiology (WCA) on September
20, 2024
- On-track for preliminary efficacy and safety data
presentation of SENS-401 Phase 2a in Cisplatin-Induced Ototoxicity
at the World Congress of Audiology (WCA) on September 20,
2024
- Cash on-hand of c.€87m to support planned operations into
end of 2025
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 loss disorders, today reports its half-year
results as of June 30, 2024, as well as its outlook for the
remainder of 2024.
Nawal Ouzren, Chief Executive Officer of Sensorion, said:
“Over the first half of 2024, Sensorion has made steady positive
progress, reaching several key milestones in its development plan.
Most notably, with SENS-501, our most advanced gene therapy
program, we have made significant regulatory and clinical advances.
After receiving a green light from European regulators in January
2024, I am thrilled to announce that the first patient has been
recruited to the study and injected. We look forward to updating
you on the progress with this program in the coming months.
We have also continued to advance our Phase 2a clinical programs
for SENS-401. Firstly, we unveiled new positive efficacy data for
SENS-401 in the prevention of residual hearing loss after cochlear
implantation, with full data to be presented at the World Congress
of Audiology, later this month. Secondly, we have continued to
recruit adult patients to the NOTOXIS study in Cisplatin-Induced
Ototoxicity.
On the financial front, the steady execution of our clinical
development roadmap has attracted new investment into Sensorion,
with new recognised specialist international healthcare investors
taking part in the two private placements completed last February
and April, for a total amount of over €65 million. With these
additional resources, we are well-funded to the end of 2025.
Overall, I am delighted with the advances we are making, and I look
forward to sharing future updates over the course of 2024, which
will be rich in terms of clinical data on our lead programs.”
Pipeline Highlights and Upcoming Milestones
During the first half of 2024, Sensorion continued to develop
innovative therapies to restore hearing, treat and prevent hearing
loss, to potentially transform the patients’ quality of
hearing.
Gene Therapies for Hereditary Monogenic Hearing Loss
Sensorion continues to advance its gene therapy programs,
developed as part of its collaboration with the Institut Pasteur,
initiated in 2019 and extended for an additional 5-year period in
January 2024. The framework agreement for a research partnership
grants Sensorion an option to obtain an exclusive license to
develop and commercialize gene therapy drug candidates for the
restoration of hearing. The company has notably made progress in
its lead gene therapy program, SENS-501, with a first patient
recruited and injected.
- SENS-501 (OTOF-GT): several milestones achieved with
Clinical Trial Application (CTA) approval in Europe and First
Patient Included (FPI) and injected. FPI initial safety data
communication planned at the WCA on September 20, 2024
Sensorion’s SENS-501 (OTOF-GT) dual AAV vector gene therapy
development candidate aims at restoring hearing in patients with
mutations in OTOF gene who suffer from severe to profound
sensorineural prelingual non syndromic hearing loss. The 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. Sensorion’s lead gene
therapy program, SENS-501, has been developed as part of its
collaboration focused on the genetics of hearing with the Institut
Pasteur which was initiated in 2019 and extended for an additional
5-year period in January 2024.
On January 19, 2024, Sensorion announced the approval to
initiate the Phase 1/2 clinical trial of SENS-501, Audiogene, in
Europe with France as first country. 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. 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. The design of
the study consists of two cohorts of two 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. Audiogene will also assess the clinical
safety, performance, and usability of the administration device
system developed by Sensorion.
On September 11, 2024, Sensorion received the European Medicines
Agency decision agreeing on a Pediatric Investigation Plan (PIP)
for SENS-501. A PIP is a development plan aimed at ensuring that
the necessary data are obtained to support the marketing
authorization of a medicine in the pediatric population in the
European Union. All applications for marketing authorization for
new medicines require the results of studies as described in an
agreed PIP.
Sensorion will communicate initial safety data about the first
patient at the WCA, on September 20, 2024, and is on track for the
recruitment of the first cohort by end of 2024. The recruitment
completion of the two first cohorts of patients is expected in H1
2025.
OTOCONEX, the Company’s Natural History Study to document the
natural course of disease progression in otoferlin deficiency
patients and in children with hearing loss related to GJB2
mutations, is running across Europe and plays an important role in
identifying patients as early as possible.
- GJB2-GT: progressing CTA/IND-enabling studies towards
Clinical Trial Applications Submission in H2 2025
Sensorion's GJB2 gene therapy program, developed in
collaboration with the Institut Pasteur, has the potential to
address three forms of hearing loss related to GJB2 mutations:
early onset of presbycusis in adults, progressive forms of hearing
loss in children, and pediatric congenital deafness. Although the
types of GJB2 mutations in children and adults may differ, gene
therapy offers potential solutions for both populations.
On April 6, 2023, Sensorion announced the candidate selection
for GJB2-GT during its R&D Day focusing on gene therapy. The
candidate is designed with a specific adeno-associated virus (AAV)
capsid and includes specific regulatory sequences to safely target
key cells in the ear that normally express GJB2 and avoids
ototoxicity.
Sensorion has developed the non-GMP process development of the
GJB2 candidate up to 50L scale. Process and analytical methods are
under transfer to the CDMO for the GMP production of the clinical
batch. Sensorion is advancing the candidate into CTA/IND-enabling
activities for anticipated submission in H2 2025.
The Company plans on providing updates on preclinical activities
related to GJB2-GT including additional Proof-of-Concept data at
the European Society of Cell & Gene Therapy (ESGCT), taking
place on October 22-25, 2025, Rome, Italy.
SENS-401
Sensorion is developing SENS-401 (Arazasetron) in three
indications and two Proof of Concept Phase 2a clinical trials are
ongoing, including a study designed to assess SENS-401 for residual
hearing preservation during cochlear implantation in partnership
with Cochlear Limited; the final results of which will be reported
on September 20, 2024, during the World Congress of Audiology. The
second study is a proof-of-concept trial in Cisplatin-Induced
Ototoxicity (CIO).
SENS-401 is an orally available small molecule that aims to
protect and preserve inner ear tissue from damage responsible of
hearing impairment. SENS-401 has been granted Orphan Drug
Designation by the EMA in Europe for the treatment of sudden
sensorineural hearing loss (SSNHL), and by the FDA in the U.S. for
the prevention of platinum-induced ototoxicity in pediatric
population.
- Milestones achieved for SENS-401 in the prevention of
residual hearing loss after cochlear implantation with primary
endpoint achieved and positive secondary efficacy endpoints data.
Full final data readout to be presented at the WCA, on September
20, 2024
Sensorion has advanced its small molecule SENS-401 in a
multicentric, randomized, controlled open-label Phase 2a trial
aimed at evaluating the presence of SENS-401 in the cochlea
(perilymph) after 7 days of twice-daily oral administration in
adult patients prior to cochlear implantation due to moderately
severe to profound hearing impairment. Patients start treatment
with SENS-401 7 days before implantation and continue to receive
SENS-401 for a further 42 days.
On February 1, 2024, Sensorion announced the completion of
patient inclusion in the Phase 2a POC clinical trial. Following
this announcement, Sensorion communicated in March 2024, that a
total of 25 patients have been implanted with a cochlear implant:
16 in the treated arm and 9 in the control non-treated arm. The
presence of SENS-401 in the perilymph at a level compatible with
potential therapeutic efficacy has been confirmed in 100% of the
patients sampled, 7 days after the start of the treatment,
confirming that the primary endpoint was met.
On July 13, 2024, Professor Stephen O'Leary, M.D., Ph.D., the
study principal investigator, presented new medical data and
analysis of results from the SENS-401 Phase 2a clinical trial at
the 17th International Conference on Cochlear Implants and Other
Implantable Technologies (CI2024) in Vancouver, Canada. The study
design included a number of secondary endpoints, notably the change
of hearing threshold from baseline to the end of the treatment
period in the implanted ear at several frequencies. Study entry
criteria required patients to have a pure tone audiometry (PTA)
threshold of 80 dB or better (i.e., ≤80 dB) at 500 Hz, defined as
indicating a minimal level of residual hearing. The descriptive
results show that the administration of SENS-401 reduced hearing
loss following cochlear implantation. Six weeks post cochlear
implantation (corresponding to end of SENS-401 treatment), the data
indicate that the mean hearing loss induced by the surgery at 500
Hz is 19 dB for patients treated with SENS-401 (N=16) compared to
32 dB for control group of SENS-401 untreated patients (N=8).
Similar clinically meaningful difference is observed for the mean
of the three following frequencies (250, 500 and 750 Hz) with 16 dB
in the SENS-401 treated group compared to 31 dB in the control
group. These good descriptive results remained clinically
meaningful over time and up to the last study visit fourteen weeks
after cochlear implantation and confirm the key role of SENS-401 in
preserving residual hearing.
The Company plans on communicating the results of the final
analysis at the WCA, held in Paris, France, on September 19-22,
2024.
- SENS-401 in Cisplatin-Induced Ototoxicity progresses with
patient recruitment progressing at a steady pace and positive Data
Safety Monitoring Board (DSMB) recommendation to pursue the
study
Cisplatin and other platinum compounds are essential
chemotherapeutic agents for many malignancies. Unfortunately,
platinum-based therapies cause ototoxicity, or hearing loss, which
is permanent, irreversible and particularly harmful to 50-60% of
adult patients and 90% of pediatric patients who survive
cancer.
The NOTOXIS Proof-of-Concept (POC) Phase 2a trial is a
multicenter, randomized, controlled, open-label study designed to
assess the efficacy of SENS-401 in preventing cisplatin-induced
ototoxicity in adult patients with neoplastic disease, four weeks
after completion of cisplatin-based chemotherapy. The trial
assesses several endpoints, including the rate and severity of
ototoxicity, changes in pure tone audiometry (PTA) (dB) throughout
the study compared to before cisplatin treatment, and
tolerability.
Eligible participants are randomized on Day 1 to either Arm A or
Arm B in ratio 1:1. In Arm A, patients receive 43.5mg of oral
SENS-401 one week before the start of the chemotherapy, continues
throughout the entire chemotherapy duration, and extends for up to
four weeks post-chemotherapy. This study is conducted in comparison
to a control group of patients receiving chemotherapy alone, Arm B.
The patients entering the study are to receive high doses of
cisplatin, exceeding 70mg/m2 per treatment cycle and totaling at
least 210 mg/m2 over the course of their chemotherapy regimen.
On December 18, 2023, Sensorion announced that over one-third of
the required study population had been recruited and that
preliminary safety data for patients exposed to a daily dose of
43.5 mg SENS-401 administered b.i.d. for up to 11 weeks indicated a
favorable profile.
On July 23, 2024, Sensorion announced that the independent Data
Safety Monitoring Board (DSMB) had undertaken a review of the
safety data for the patients participating in the NOTOXIS POC Phase
2a clinical trial. The DSMB recommended the continuation of the
study and confirmed the absence of any concern as to the safety of
SENS-401 when administered in adult patients receiving a daily dose
of 43.5 mg, administered twice daily, over a period of up to 23
weeks. The patient enrolment continues to progress at a steady
pace, in 13 clinical centers open to date. Sensorion will report
preliminary safety and efficacy data of the Phase 2a POC clinical
trial of SENS-401 CIO at the WCA, to be held on September 19-22,
2024, in Paris, France.
Expected future milestones and estimated timelines:
- September 2024 – SENS-401 in combination with cochlear
implantation: Final data readout
- September 2024 – SENS-401 in Cisplatin-Induced Ototoxicity:
Preliminary safety and efficacy data
- September 2024 – SENS-501: FPI initial safety data
- October 2024 (ESGCT) – GJB2-GT: Update on additional
Proof-of-Concept efficacy and safety data
- H2 2024 – SENS-501: Enrollment completion of the first cohort
in Audiogene
- H1 2025 – SENS-501: Enrollment completion of the second cohort
in Audiogene
- H1 2025 – SENS-401 in Cisplatin-Induced Ototoxicity: Patient
recruitment completion
- H2 2025 – GJB2-GT: Clinical Trial Applications filing
First-half 2024 financial highlights
Cash & Cash Equivalents amounted to 87.3 million as of June
30, 2024, compared to €37.0 million as of December 31, 2023.
- Research And Development (R&D) Expenses
R&D expenses increased by 19% from €12.3 million as of June
30, 2023, to €14.7m as of June 30, 2024, primarily due to an
increase in gene therapy programs with the start of GJB2-GT
manufacturing.
- General And Administrative (G&A) Expenses
G&A expenses were €3.8 million for half-year 2024, compared
to €2.6 million for the same period in 2023.
Net loss was -€13,9 million for half-year 2024, compared to
€12.3 million for half-year 2023.
Based on its forecasted expenses and a cash position of €87.3
million as of June 30, 2024, the Company expects to be able to fund
its operations until end of 2025.
Financial structure
The half-year accounts on June 30, 2024, drawn up according to
IFRS standards, were approved by the Board of Directors on
September 17, 2024.
The simplified income statement as of June 30, 2024, is as
follows:
In thousands of Euros – IFRS standards
30.06.2024
30.06.2023
Operating income
3,332
2,647
Research & Development expenses
14,660
12,271
General & Administrative expenses
3,791
2,572
Total operating expenses
18,451
14,844
Operating loss
-15,119
-12,196
Financial result
1,323
130
Pre-tax current income
-13,796
-12,066
Corporate Income Tax
-98
-222
Net loss
-13,895
-12,288
The simplified balance sheet as of June 30, 2024, is as
follows:
In thousands of Euros – IFRS standards
30.06.2024
31.12.2023
Non-current Assets
3,536
3,236
Other Current Assets
10,649
6,293
Cash & cash equivalent
87,344
36,974
Total Assets
101,529
46,503
Equity
83,462
33,276
Non-current Liabilities
3,897
2,950
Current Liabilities
14,110
10,278
Total Liabilities
101,529
46,503
First-Half 2024 certified accounts
On September 17, 2024, the Board of Directors approved the
Company’s first half-year results as of June 30, 2024. The
Company’s statutory auditors have completed their limited review
and are in the process of issuing an unqualified report, which
shall be available in the coming days.
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) currently being
developed in a Phase 1/2 clinical trial, 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
2023 full year report published on March 14, 2024, 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, et al., 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.
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Investor Relations Noémie Djokovic, Investor Relations
and Communication Associate ir.contact@sensorion-pharma.com
Press Relations Ulysse Communication Bruno Arabian / 00
33(0)6 87 88 47 26 barabian@ulysse-communication.com Nicolas Entz /
00 33 (0)6 33 67 31 54 nentz@ulysse-communication.com
Grafico Azioni Sensorion (EU:ALSEN)
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