Sensorion Sas · EURONEXT:ALSEN
Sensorion Sas — Sensorion — Investor Presentation (November 2022)
37 slides from Sensorion Sas's investor presentation, November 2022
Slide 1: Sensorion Corporate Presentation November 2022
—_~ | & Sensorion
Slide 2: Sensorion
1 SENSORION Copyright by Sensorion - 2022 - All RightsReserved
Slide 3: Disclaimer
DISCLAIMER • This document has been prepared by Sensorion (the “Company”) and is provided for • This document contains certain forward-looking statements. These statements are not information purposes only. This document does not purport to contain comprehensive guarantees of the Company’s future pe
Slide 4: Investment Highlights
Investments Highlights • Sensorion is focused on innovative treatments that can restore, treat and prevent hearing loss − Its oral small molecule asset SENS-401 currently in clinical development in the following indications: • Sensorion and Cochlear Ltd. CTA approved for SENS-401 in patients schedul
Slide 5: The inner ear: one of the most delicate organs in the human body
The inner ear: one of the most delicate organs in the human body Inner Ear Biology Limited number of hair cells: KEY FACTS • 3,500 Inner Hair Cells • 12,000 Outer Hair Cells Hair cells do not naturally regenerate According to the WHO*: ~ 400m people affected by disabling hearing loss worldwide inclu
Slide 6: Our strategy: Restore, Treat & Prevent hearing loss
Our strategy: RESTORE, TREAT & PREVENT hearing loss Causes of hearing loss Idiopathic SMALL MOLECULE APPROACH 25% Genetic • Phase 2 study completed with SENS-401 to TREAT Sudden Sensorineural Hearing Loss 50% Exploring partnering opportunities Environmental • Phase 2a study with SENS-401 to PREVENT
Slide 7: Our critical strategic alliances from bench to bedside
Our critical strategic alliances from bench to bedside French Military Biomedical Research Institute • Access to a military population at risk of noise-induced hearing loss • EU reference center for monogenic forms of deafness CLINICAL • Strong medical network, strict monitoring and precise, regular
Slide 8: Our pipeline: a comprehensive portfolio to Restore, Treat & Prevent hearing loss
Our pipeline: a comprehensive portfolio to RESTORE, TREAT & PREVENT hearing loss Next milestones Product Indication Discovery In-vivo POC Pre-clinical Phase 1 Phase 2 Phase 3 (estimated timelines) Hearing preservation SENS-401 after cochlear First results in 1H 2023 PREVENT implantation SENS-401 Cis
Slide 9: An experienced team, Board of Directors and SAB
An experienced team, Board of Directors and SAB NAWAL OUZREN GÉRALDINE HONNET STEPHANIE FILIPE LAURENT DESIRE CHRISTINE LE BEC Chief Executive Officer Chief Medical Officer Head of PMO Preclinical Development Director Head of CMC Gene Therapy SENSORION SENSORION SENSORION SENSORION SENSORION (Since
Slide 10: We have established internal capabilities to ensure successful execution
We have established internal capabilities to ensure successful execution PRECLINICAL CAPABILITIES CLINICAL CMC GENE THERAPY REGULATORY FOR SMALL MOLECULES EXPERIENCE FACILITIES EXPERTISE & GT PROGRAMS • In Vitro platform: assays • 400 people enrolled in Sensorion • Process development lab: non- • Mu
Slide 11: SENS-401: Treat and Prevent
2 SENS-401 TREAT AND PREVENT Copyright by Sensorion - 2022 - All RightsReserved
Slide 12: SENS-401: Multiple indications to treat and prevent hearing loss
SENS-401: Multiple indications to treat and prevent hearing loss Product Indication Discovery In vivo POC Preclinical Phase 1 Phase 2 Phase 3 Hearing preservation PREVENT SENS-401 after cochlear implantation Cisplatin SENS-401 Induced Ototoxicity TREAT Sudden SENS-401 Sensorineural Hearing Loss* *”P
Slide 13: Sudden Sensorineural Hearing Loss SSNHL
TREAT Sudden Sensorineural Hearing Loss SSNHL WHAT IS SSNHL? The sudden onset of a significant hearing loss due to dysfunction of the cells of the cochlea and central auditory structures. Hearing loss develops over less than 72 hrs, hearing sensitivity is reduced by at least 30 dB (1,000 fold) in th
Slide 14: SENS-401 SSNHL program: phase 2 design
TREAT SENS-401 SSNHL program: phase 2 design A RANDOMIZED, MULTICENTER, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL 50 clinical sites globally 1 Primary objective Audiometry 15 dB improvement vs. Placebo 115 patients enrolled Timing Control group: +/- oral corticosteroids Q1 2019 June 20 & Q1 2022 center
Slide 15: SENS-401 shows a clinically meaningful effect in a large sub-population
SENS-401 shows a clinically meaningful effect at Day 84 in a large TREAT sub-population PTA improvement from baseline compared to placebo on per protocol idiopathic SSNHL 5 Day 7 Day 14 Day 28 Day 84 Estimate PTA change from baseline (dB) High dose N= 21 N= 23 N= 22 N= 17 0 Low dose N= 26 N= 26 N= 2
Slide 16: SENS-401 effect more pronounced in profound hearing loss sub-group
SENS-401 effect is more pronounced in a profound hearing loss sub-group TREAT (PTA ≥ 80 dB) PTA improvement from baseline compared to placebo 0 Day 7 Day 14 Day 28 Day 84 Estimate PTA change from baseline (dB) -5 High dose N= 11 N= 11 N= 9 N= 9 -10 Low dose N= 11 N= 11 N= 9 N= 9 Placebo N= 14 N= 15
Slide 17: Responder rate is always better in the treated groups compared to placebo
TREAT Responder rate is always better in the treated groups compared to placebo Responder analysis on ITT population Population showing an improvement greater than 30 dB 100% n Day 7 Day 14 Day 28 Day 84 High Dose 13 19 21 17 75% % responders Low Dose 13 21 22 21 Day 28 50% Placebo 11 18 20 18 Day 8
Slide 18: SENS-401 induces complete PTA recovery in 50% of patients
TREAT SENS-401 induces complete PTA recovery in 50% of patients Complete PTA recovery 70 p=0.1452 * p=0.0230 60 p=0.1230 p=0.2707 % patients with complete PTA Complete PTA 50 Placebo High Dose Low Dose recovery (n/n total) 40 Day 28 9/28 12/23 13/26 recovery 30 Day 84 8/26 8/18 14/22 20 10 0 Day 28
Slide 19: SENS-401 SSNHL phase 2 results summary
TREAT SENS-401 SSNHL phase 2 results summary Exploring partnering opportunities AUDIBLE-S SECONDARY ENDPOINT RESULTS • Safe and well tolerated in 115-patient SSNHL study; primary endpoint not met • SENS-401 shows a clinically meaningful and statistically significant effect on PTA change over time in
Slide 20: SENS-401 to preserve residual hearing after cochlear implantation
PREVENT SENS-401 to preserve residual hearing after cochlear implantation COMBINATION OF COCHLEAR IMPLANT WITH SENS-401 TO PREVENT CELL-DEATH POST COCHLEAR IMPLANT PROCEDURE HEALTHY AGEING 36,450 Growing understanding of the link between Implants sold by Cochlear® healthy hearing and healthy ageing
Slide 21: SENS-401 proof-of-concept clinical study design approved in France and Australia
PREVENT PREVENT SENS-401 proof-of-concept clinical study design approved in France and Australia with first patient enrolled in Sept. 2022 A PHASE IIA, MULTICENTER, RANDOMIZED, CONTROLLED, OPEN-LABEL STUDY SCREENING PERIOD EVALUATION PERIOD D1 D8 D49 D105 CI switch on Randomization Treated Arm: SENS
Slide 22: Cisplatin administration damages the inner ear and leads to hearing loss
Cisplatin administration for chemotherapeutic treatment of cancer PREVENT damages the inner ear and leads to hearing loss, tinnitus and dizziness WHAT IS CIO? Hearing loss caused by cisplatin administration as chemotherapeutic treatment. Risk factors include young age as well as individual and cumul
Slide 23: SENS-401 CIO NOTOXIS to prevent ototoxicity induced by cisplatin
PREVENT SENS-401 CIO NOTOXIS to prevent ototoxicity induced by cisplatin SIGNIFICANTLY REDUCES CISPLATIN-INDUCED HEARING LOSS AND OUTER HAIR CELL DEATH IN PRE-CLINICAL MODELS TREATMENT PROTOCOL SENS-401 6.6 mg/kg, 13.2 mg/kg or placebo were administered Significant improvement versus placebo to rats
Slide 24: SENS-401 amended Phase 2a proof-of-concept study design approved in France
PREVENT PREVENT SENS-401 amended Phase 2a proof-of-concept clinical study design approved in France in October 2022 A Phase 2a, Multicenter, Randomized, Controlled, Open-label Study to Evaluate the Efficacy of SENS-401 to Prevent the Ototoxicity induced by Cisplatin in Adult Subjects with a Neoplast
Slide 25: SENS-401 program next steps
PREVENT SENS-401 program next steps SENS-401 CIO NOTOXIS CTA First patient enrolled in SENS-401 CIO amendment approved 2H 2022 NOTOXIS 2H 2022 SENS-401 in combination with cochlear SENS-401 CIO NOTOXIS implants first results 1H 2023 first results 1H 2023 Copyright by Sensorion - 2022 - All RightsRes
Slide 26: Gene Therapy: Restore
3 GENE THERAPY RESTORE Copyright by Sensorion - 2022 - All RightsReserved
Slide 27: Strategic R&D collaboration with Institut Pasteur on genetics of hearing
RESTORE Strategic R&D collaboration with Institut Pasteur on genetics of hearing 2 PROGRAMS INITIATED UNDER THE COLLABORATION AGREEMENT WITH INSTITUT PASTEUR Rare disease, high unmet medical need Master Research Master Research Project ProjectAgreement “OTOF” Agreement “OTOF” Sensorion has an Agreem
Slide 28: Sensorion's gene therapy programs to treat rare auditory diseases
RESTORE Sensorion’s gene therapy programs to treat rare auditory diseases 2 PROGRAMS INITIATED UNDER THE STRATEGIC COLLABORATION AGREEMENT WITH INSTITUT PASTEUR OTOFERLIN DEFICIENCY GJB2-RELATED HEARING LOSS We have identified three forms of hearing loss associated with GJB2 • Patients with mutation
Slide 29: OTOF gene encodes otoferlin, a key Ca2+ sensor protein
RESTORE OTOF Gene encodes otoferlin, a key Ca2+ sensor protein Iow Ca2+ Increase in Ca2+ SNARE Proteins VAMP Ca2+ Ca2+ Ca2+ Ca2+ Vesicle Ca2+-Channel Otoferlin Pre-synaptic membrane neurotransmitters release restricted SNARE interaction Otoferlin SNARE interaction SNARE-complex binding primed vesicl
Slide 30: Dual AAV-OTOF resulted in IHC-specific expression and hearing restoration in DFNB9 mice
Dual AAV-OTOF resulted in IHCs specific expression and hearing RESTORE restoration in DFNB9 mice Target cell specific protein expression Hearing restoration in DFNB9 mice • Dual AAV-OTOF injection into the cochlea leads to IHC specific de novo • Durable hearing restoration in Otof-/- mice by dual AA
Slide 31: Optimized surgical procedure leads to IHC-specific transduction in mature NHP cochlea
RESTORE Optimized surgical procedure leads to IHC specific AAV-delivered transgene transduction in mature NHP cochlea AAV vector distribution in cochlea of NHP RWM Optimization Surgical approach • Delivery of the AAV transgenes to • Surgical procedure is similar to IHCs and not OHCs in NHP cochlear
Slide 32: Otoferlin Audinnove consortium provides privileged access to patients and surgeons
RESTORE Otoferlin “Audinnove” consortium provides privileged access to patients and surgeons Audinnove consortium received Hospital-University Research (RHU) prize: • The consortium is eligible to receive up to €9.7m to develop a gene therapy program addressing otoferlin deficiency AUDINNOVE CONSORT
Slide 33: OTOF gene therapy program status
RESTORE OTOF gene therapy program status POC data in mouse & POC Submission of European Natural preliminary data in NHPs History Study OTOCONEX Product development and Delivery of batches for toxicology manufacturing agreement study mid-2022 Advice from regulatory Clinical Trial Application 1H 2023
Slide 34: Connexin 26: a gap-junction protein encoded by GJB2 gene
RESTORE CONNEXIN 26: a gap-junction protein encoded by GJB2 gene and responsible for tissue homeostasis - mutations in the gene lead to deafness • GJB2 is the gene encoding for the Connexin 26 protein; one of 20 known connexins in humans and almost endemic to the cochlea (together with Cx30); a hexa
Slide 35: GJB2 gene therapy program next steps
RESTORE GJB2 gene therapy program next steps Submission of European Natural Submission of Natural History Study History Study OTOCONEX in collaboration with Sonova Candidate selection 2H 2022 Preclinical IND enabling studies Copyright by Sensorion - 2022 - All RightsReserved 35
Slide 36: Sensorion potential newsflow
Sensorion potential newsflow [estimated timelines] • Mid-2022 – OTOF-GT: delivery of batches for toxicology study • 2H 2022 – OTOF-GT: EMA’s positive opinion for ODD • 2H 2022 – SENS-401 CIO: NOTOXIS CTA study amendment approval • 2H 2022 – GJB2-GT candidate selection • 1H 2023 – SENS-401 in combina
Slide 37: Thank you
THANK YOU Nawal Ouzren Chief Executive Officer E: contact@sensorion-pharma.com