- Milestone achieved for SENS-501 gene therapy program with
Clinical Trial Application approval in Europe (France as first
country)
- Communication on first patient expected in H2 2024
- GJB2 gene therapy program advancing towards Clinical Trial
Applications filing expected in Q2 2025
- Primary endpoint met in Phase 2a clinical trial of SENS-401
in the preservation of residual hearing following cochlear
implantation
- Presence of SENS-401 detected in 100% of patients sampled at
levels compatible with potential therapeutic efficacy
- Final data readout expected in Q3 2024
- Continued progress for SENS-401 in Phase 2a clinical trial
in the prevention of Cisplatin-Induced Ototoxicity
- Preliminary safety and efficacy data readout expected at the
World Congress of Audiology in September 2024
- Completion of two financing rounds, raising €35 million in
August 2023 and €50.5 million in February 2024, enabling the
company to extend its cash runway through the end of Q2
2025
Regulatory News:
Sensorion (Paris:ALSEN) (FR0012596468 –
ALSEN) a pioneering clinical-stage biotechnology company which
specializes in the development of novel therapies to restore, treat
and prevent hearing loss disorders, today reports full-year 2023
financial results and business update.
Nawal Ouzren, Chief Executive Officer of Sensorion, said:
“Over the past months, Sensorion has successfully achieved all the
major milestones on its roadmap. Our pioneering gene therapy,
SENS-501, is entering clinical development, and our small molecule,
SENS-401, has met primary endpoint in its most advanced indication:
residual hearing preservation after cochlear implantation, in a
Phase 2a study. At the same time, we have succeeded in securing
financing for our operations until mid-2025 with the support of
international and long-term investors. Sensorion is well equipped
to meet its operational objectives for 2024, to continue bringing
innovative therapeutic solutions to patients in the field of
hearing, and to create value for its shareholders.”
Pipeline Highlights and Upcoming Milestones
In 2023, Sensorion continued to develop innovative therapies to
restore hearing, to treat and prevent hearing loss, and potentially
transform quality of life for patients suffering from hearing loss
disorders.
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 exclusive option to obtain an exclusive license
to develop and commercialize drug candidates in gene therapy for
the restoration of hearing. The company has made significant
progress in its gene therapy pipeline with the submission and
approval of clinical trial application for SENS-501 in Europe, and
the candidate selection for GJB2-GT. The company has expanded its
technical development capabilities over the period along with
pilot-scale non-GMP (Good Manufacturing Practices) manufacturing
capacity.
- SENS-501: Milestone achieved with Audiogene Clinical Trial
Application approval in Europe. First patient communication
anticipated in H2 2024
Sensorion’s SENS-501 (OTOF-GT) dual AAV vector gene therapy
development product 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.
In the first half of 2023, Sensorion achieved a major
development milestone by completing the preclinical package, and
successfully producing the GMP dual AAV SENS-501 clinical batches
at 200L scale. The production will enable the Company to cover the
needs for the envisioned Phase 1/2 gene therapy clinical trial.
Following the positive feedback from Scientific Advice meetings
with regulatory agencies EMA (European Medicines Agency), ANSM
(French National Agency for Medicines and Health Products Safety)
and the UK’s MHRA (Medicines and Healthcare product Regulatory
Agency), Sensorion has submitted a Clinical Trial Application (CTA)
to initiate a Phase 1/2 clinical trial of SENS-501 to the UK’s MHRA
on July 10, 2023, and in Europe on July 19, 2023. In parallel,
Sensorion submitted a Medical Device Application for the injection
system.
On January 19, 2024, Sensorion announced the approval to
initiate the Phase 1/2 clinical trial of SENS-501, Audiogene, in
Europe (France as first country). The CTA approval follows
extensive preclinical studies assessing the safety and efficacy of
SENS-501 and successful GMP 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. 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 at two
different doses followed by an expansion cohort at the selected
dose. While the safety will be the primary endpoint for the dose
escalation cohorts, the auditory brainstem response (ABR) will be
the primary efficacy endpoint of the dose expansion cohort.
Sensorion will communicate on the first patient in the course of
H2 2024 and expects the recruitment completion of the first two
cohorts of patients in H1 2025.
- GJB2-GT: Moving forward towards Clinical Trial Applications
submission in Q2 2025
Sensorion's GJB2 gene therapy program, initiated in 2021 and
developed in collaboration with the Institut Pasteur, has the
potential to address three pathologies 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.
On April 6, 2023, Sensorion announced the candidate selection
for GJB2-GT during its gene therapy focused R&D Day. The
candidate designed with a specific adeno-associated virus (AAV)
capsid safely targets key cells in the ear that normally express
GJB2.
Sensorion has acquired bioreactors and has completed the non-GMP
process development of the GJB2 candidate up to 50L scale. Process
and analytical methods will be transferred to the CDMO selected by
the Company for the GMP production of the clinical batch. Sensorion
is advancing the candidate into IND/CTA enabling activities for
future clinical development and anticipates the submission of the
Clinical Trial Applications in Q2 2025.
SENS-401: Sensorion’s small molecule for the prevention of
hearing loss
SENS-401(Arazasetron) is an orally available small molecule that
aims to protect and preserve inner ear tissue from damage
responsible for 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.
Sensorion is developing SENS-401 in two Proof of Concept (POC)
Phase 2a clinical trials. The first one is designed to assess
SENS-401 for residual hearing preservation during cochlear
implantation in partnership with Cochlear Limited (Cochlear), the
global leader in implantable hearing solutions, and the second one
is a trial assessing SENS-401 in Cisplatin-Induced Ototoxicity
prevention (CIO) in adult patients undergoing a cisplatin-based
chemotherapy.
- SENS-401 to prevent residual hearing loss after cochlear
implantation. Milestone achieved with primary endpoint met. Final
data readout planned in Q3 2024
Sensorion is developing SENS-401 in a multicentric, randomized,
controlled open-label Phase 2a clinical 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 continue to receive SENS-401 for a further 42
days after implantation.
In June 2023, Sensorion released preliminary data from its Phase
2a study, showing that SENS-401 was detected in the perilymph of
all five adult patients treated with the product. Levels of
SENS-401 were considered consistent with potential therapeutic
effects after seven days of repeated oral treatment.
In July 2023, the Company reported further analysis during its
KOL webinar suggesting that SENS-401 treated patients demonstrated
a clinically significant 21 dB improvement in the preservation of
their residual hearing compared to the control group six weeks
after cochlear implantation at 500 Hz. Indeed, in the
SENS-401-treated group (N=5), the loss of residual hearing was only
12 dB, contrasting with a larger loss of 33 dB observed in the
control group of four participants not treated with SENS-401. These
findings reinforce the hypothesis that SENS-401, by crossing the
labyrinthine barrier to reach the cochlear compartment, has a
positive effect on the preservation of residual hearing.
On February 1, 2024, Sensorion announced the completion of
patient inclusion in the Phase 2a POC clinical trial, with 28
patients recruited in the study in total. Following this
announcement, Sensorion communicated on March 11, 2024, that a
total of 28 patients have been randomized and 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. The study is now completed, the follow-up of the last
patients is still ongoing and the secondary endpoints including
results on the preservation of the residual hearing will be
available and analyzed later this year. The Company plans to
publish the complete readout of the study in Q3 2024.
- SENS-401 to prevent Cisplatin Induced Ototoxicity (CIO)
progressing as planned with efficacy and safety preliminary data
expected in September 2024
Cisplatin and other platinum compounds are essential
chemotherapeutic agents for many malignancies. Unfortunately,
platinum-based therapies cause ototoxicity and hearing loss, which
are permanent, irreversible and particularly harmful in about 60%
of adult and pediatric patients treated. This indication represents
a very significant unmet need for patients and is an attractive
market with more than 500,000 patients forecast in 2025 in the G7
countries.
SENS-401 is progressing in NOTOXIS, a multicenter, randomized,
controlled, open-label Phase 2a clinical study, designed to
evaluate the efficacy of SENS-401 to prevent CIO in adult patients
with a neoplastic disease four weeks after the completion of
cisplatin-based chemotherapy. The trial assesses several outcome
measures, including the rate and severity of ototoxicity, the
change from baseline in Pure Tone Audiometry (PTA) (dB) throughout
the study and the tolerance.
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 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.
In December 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 consistent with previously reported data for
patients exposed for up to 7 weeks.
Recruitment is progressing at a sustained path, with eleven
clinical centers open as of December 18, 2023. Sensorion’s
management team will communicate preliminary efficacy and safety
data of the POC Phase 2a clinical study of SENS-401 in CIO during
the World Congress of Audiology, held on September 19-22, 2024, in
Paris, France.
Strengthening the Board of Directors and senior
leadership
On March 31, 2023, Sensorion appointed Khalil Barrage as interim
Chairman of the Board. Mr. Barrage has served on the Board of
Sensorion for over three years, supporting with several capital
raises and providing leadership on the Company’s evolution towards
becoming a gene therapy-focused company. He has been a managing
director at Invus Group, a major investor in Sensorion, for nearly
20 years, heading its Public Equity group and serves on the Boards
of Elevate Bio, Valerio Therapeutics, Orthobond and Protagenic
Therapeutics.
On August 3, 2023, following the completion of the private
placement, Redmile Group (represented by Natalie Berner) became a
board member in replacement of Bpifrance Investissement, whose
permanent representative was Jean-François Morin.
On December 12, Sensorion announced the appointment of CMC
(Chemistry, Manufacturing and Control) expert Bernd Schmidt as
Chief Technical Officer. He brings over 20 years of industry
experience in the pharmaceutical sector, covering a broad range of
innovative medicines at different stages in development and post
launch.
On January 25, 2024, Sensorion announced the nomination of Dr.
Federico Mingozzi as Non-Executive Director to the Board of
Directors. Mr. Mingozzi brings over 25 years of experience in gene
therapy, immunology, as well as biochemistry and molecular biology
in academia and industry.
2024 Outlook
As of December 31, 2023, the Company had €37.0 million in cash
and cash equivalents. Based on this cash position, the net proceeds
from the €50.5 million financing announced on February 9, 2024, and
its forecasted expenses, the company expects it will be able to
finance its operations up until the end of Q2 2025.
In 2024, Sensorion plans on pursuing developments across its
portfolio of innovative programs with the aim of treating,
restoring and preventing hearing disorders and improving the
quality of life of patients suffering from hearing disorders. The
Company plans to communicate about the first patient enrolled in
Audiogene, its Phase 2a clinical trial of its gene therapy product,
SENS-501, in H2 2024. The Company is on track to submit Clinical
Trial Applications for its second gene therapy, GJB2-GT, in H1
2025, with ongoing IND/CTA-enabling preclinical activities. In
parallel, SENS-401, Sensorion’s orally available small molecule, is
progressing in two Phase 2a clinical trials. Final data readout is
expected in Q3 2024 for its trial of SENS-401 in the prevention of
residual hearing loss following cochlear implantation, and
preliminary safety and efficacy are expected in September 2024
during the World Congress of Audiology for its second ongoing
clinical trial of SENS-401, in the prevention of Cisplatin-Induced
Ototoxicity.
Expected next clinical milestones:
- Q3 2024 – SENS-401 in combination with cochlear implantation:
Final data readout
- H2 2024, World Congress of Audiology – SENS-401 in
Cisplatin-Induced Ototoxicity: Preliminary safety and efficacy
data
- H2 2024 – SENS-501: First patient communication
- H1 2025 – SENS-501: Enrollment of the first two cohorts of
Phase 1/2 Audiogene trial completed
- Q2 2025 – GJB2-GT: Clinical Trial Applications Submission
Full Year 2023 financial highlights
Cash & Cash Equivalents amounted to €37.0 million as at
December 31, 2023, compared to €26.2 million at December 31,
2022.
- Research And Development (R&D) Expenses
R&D expenses decreased by 0.7 % from €22.9 million in 2022
to €22.8 million in 2023.
- General And Administrative (G&A) Expenses
G&A expenses were €5.3 million for 2023, compared to €5.2
million for 2022.
Net loss was -€22.1 million for 2023, compared to -€23.2 million
for 2022.
Based on cash and cash equivalents of €37.0 million at 31
December 2023, and the net proceeds of a €50.5 million offering of
new shares announced on February 9, 2024, amounting to c. €47
million, the Company has sufficient net working capital to meet its
cash requirements beyond the next twelve months, i.e. until the end
of Q2 2025.
Financial results
The annual accounts as at December 31, 2023, were prepared
according to IFRS standards and approved by the Board of Directors
on March 13, 2024.
The simplified income statement as of December 31, 2023, is as
follows:
In thousands of Euros – IFRS standards
31.12.2023
31.12.2022
Operating income
5,698
5,006
Research & Development expenses
-22,756
-22,925
General & Administrative expenses
-5,252
-5,217
Total operating expenses
-28,008
-28,142
Operating loss
-22,310
-23,137
Financial result
544
-72
Corporate Income Tax
-297
0
Net loss
-22,063
-23,209
The simplified balance sheet at December 31, 2023, is as
follows:
In thousands of Euros – IFRS standards
31.12.2023
31.12.2022
Non-current Assets
3,236
3,176
Other Current Assets
6,293
9,565
Cash & cash equivalent
36,974
26,204
Total Assets
46,503
38,945
Equity
33,276
21,885
Non-current Liabilities
2,950
3,467
Current Liabilities
10,278
13,593
Total Liabilities
46,503
38,945
2023 certified accounts
On March 13, 2024, the Board of Directors approved the Company’s
full year results as of December 31, 2023. The Full Year Financial
Report can be found on Sensorion’s website
(https://www.sensorion.com/en/home/) in the investor section under
financial information. The full year accounts of 2023 have been
subject to a limited review by the Company’s statutory auditors and
an unqualified report is being issued.
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 is currently
developed in a Phase 1/2 clinical study, 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 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
2023 full year financial 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, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240313961079/en/
Investor Relations Noémie Djokovic, Investor Relations
and Communications Associate ir.contact@sensorion-pharma.com
Press Relations Ulysse Communication Pierre-Louis Germain
/ +33 (0)6 64 79 97 51 plgermain@ulysse-communication.com Bruno
Arabian / +33(0)6 87 88 47 26 barabian@ulysse-communication.com
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