Sensorion Sas · EURONEXT:ALSEN

Sensorion Sas — Sensorion — Investor Presentation (May 2023)

41 slides from Sensorion Sas's investor presentation, May 2023

Slide 1: Unlocking the potential of advanced therapies for hearing loss
Unlocking the potential of advanced therapies for hearing loss May 2023
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: Sensorion: Overview
Sensorion: Overview • Sensorion currently develops two Gene Therapy (GT) programs in the ear, targeting monogenic forms of deafness with pediatric and adult onset: • OTOF-GT caused by mutations of the gene encoding for otoferlin, EU & US ODD, US RPDD • GJB2-GT related to mutations in GJB2 gene (cand
Slide 5: We Want People With Inner Ear Hearing Disorders To Live Life With Unlimited Connections
We Want People With Inner Ear Hearing Disorders To Live Life With Unlimited Connections 1. Make OTOF-GT the Standard of Care • Replacing cochlear implantation • Transforming lives for babies 2. Develop GJB2-GT and Other GT Products • Addressing adolescent and adult populations 3. Enjoy Life to the m
Slide 6: Our Vision: A Global Franchise Establishing Leadership In The Hearing Space
Our Vision: A Global Franchise Establishing Leadership In The Hearing Space Portfolio Product Pipeline (Institut Pasteur, Other…) GJB2-GT OTOF-GT SENS-401 Ecosystem • EU Natural History Key Opinion Centers of Patient Groups Partners • US Studies Leaders Excellence • ROW Copyright by Sensorion - 2023
Slide 7: Together With Best-In-Class Partners We Can Transform the Current Standard of Care
Together With Best-In-Class Partners We Can Transform the Current Standard of Care • EU reference center for monogenic forms of deafness • Natural History Study currently running for TRANSLATIONAL CLINICAL all monogenic forms of deafness; extension • Interdisciplinary approach to the RESEARCH mechan
Slide 8: Sensorion is Well Positioned to Transform the Hearing Landscape
Sensorion is Well Positioned to Transform the Hearing Landscape - Institut Pasteur Partnership Provides GT Pipeline GENE THERAPY SMALL MOLECULE: SENS-401 Otoferlin deficiency (OTOF-GT) Sudden Sensorineural Hearing Loss (SSNHL) CTA enabling studies AUDIBLE-S Ph2 study completed • Hearing restoration
Slide 9: Sensorion's Portfolio Of Advanced Hearing Loss Therapies
Sensorion’s Portfolio Of Advanced Hearing Loss Therapies Upcoming Product Indication Discovery In-vivo POC Preclinical Phase 1 Phase 2 Phase 3 Milestones (estimated) Clinical Trial OTOF-GT* Otoferlin Deficiency Application Q2 2023 Adult onset RESTORE GJB2-GT* Candidate Selected (presbycusis) GJB2-GT
Slide 10: An Experienced Leadership Team and SAB
An Experienced Leadership Team and SAB NAWAL OUZREN GÉRALDINE HONNET DAVID LAWRENCE STEPHANIE FILIPE LAURENT DESIRE CHRISTINE LE BEC Chief Executive Officer Chief Medical Officer Chief Financial Officer Head of PMO Head of Preclinical Development Head of CMC Gene Therapy SENSORION SENSORION SENSORIO
Slide 11: We Have Established Strong Internal Capabilities To Ensure Successful Execution
We Have Established Strong Internal Capabilities To Ensure Successful Execution PRECLINICAL CAPABILITIES CLINICAL CMC GENE THERAPY REGULATORY FOR SMALL MOLECULES EXPERIENCE FACILITIES EXPERTISE & GT PROGRAMS • Cell Model Platform: assays • 400 people enrolled in • Process development: non- • Develop
Slide 12: Our Team has Significant Experience in Gene Therapy Clinical Development
Our Team has Significant Experience in Gene Therapy Clinical Development The team has been involved in 15+ programs … using different technologies… from preclinical to BLA filing… 10 4 1 15 1 1 Preclinical Clinical BLA filing Gene therapy Cell Gene editing (AAVs / LVs) therapy … across different org
Slide 14: Sensorion's Gene Therapy Programs Target Rare Auditory Diseases
Sensorion’s Gene Therapy Programs Target Rare Auditory Diseases FIRST PROGRAMS RESULTING FROM THE INSTITUT PASTEUR COLLABORATION OTOFERLIN DEFICIENCY GJB2-RELATED HEARING LOSS • Patients with mutations in OTOF suffer from severe to profound We have identified three forms of hearing loss associated w
Slide 15: Aiming To Develop Best-in Class And First-in Class Gene Therapy
Aiming To Develop Best-in Class And First-in Class Gene Therapy CRITERIA SENSORION AAV capsid selected for high-level of target cells specificity GT product showing high level of target cells transduction Limited off-target tissue biodistribution Surgical approach developed and mastered by ENTs surg
Slide 16: Gene Therapy Pediatric Indications Have Blockbuster Sales Potential
Gene Therapy Pediatric Indications Have Blockbuster Sales Potential OTOF-GT is the perfect pilot program • OTOF-GT will be the pilot program demonstrating that GT is a relevant • Well understood biology and pathology of the otoferlin medical approach for the inner ear deficiency • OTOF-GT will estab
Slide 17: OTOF Gene Encodes Otoferlin, A Key Ca2+ Sensor Protein
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 vesicle contai
Slide 18: Dual AAV-OTOF Resulted In IHCs Specific Expression And Hearing Restoration In DFNB9 Mice
Dual AAV-OTOF Resulted In IHCs Specific Expression And Hearing Restoration In DFNB9 Mice Target cell specific protein expression Hearing restoration in DFNB9 mice • Durable hearing restoration in Otof-/- mice by dual AAV- • Dual AAV-OTOF injection into the cochlea leads to IHC OTOF directly delivere
Slide 19: Optimized Surgical Procedure Leads To IHC Specific AAV-delivered Transgene Transduction In Mature NHP Cochlea
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 implant
Slide 20: Otoferlin Audinnove Consortium Provides Privileged Access To Patients And Surgeons
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 CONSORTIUM MEMB
Slide 21: OTOF Gene Therapy Program Status – Progressing On Track
OTOF Gene Therapy Program Status – Progressing On Track 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 Appli
Slide 22: Connexin 26: A Gap-junction Protein Encoded By GJB2 Gene
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 hexamer of 6 p
Slide 23: Candidate Selected Prevents Hearing Loss in Relevant Mouse Model
Candidate Selected Prevents Hearing Loss in Relevant Mouse Model PROOF OF CONCEPT IN PROGRESSIVE MOUSE MODEL Conditional knock-out mouse model leading to 2 phenotypes ABR threshold - 1 month after birth ABR threshold - 6 months after birth HEARING LOSS PREVENTION CORRELATES WITH CONNEXIN 26 EXPRESSI
Slide 24: GJB2 Gene Therapy Program Next Steps
GJB2 Gene Therapy Program Next Steps Submission of European Natural Submission of Natural History Study History Study OTOCONEX in collaboration with Sonova Candidate selection Q2 2023 Preclinical IND enabling studies Copyright by Sensorion - 2023 - All Rights Reserved 24
Slide 26: 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 27: SENS-401 - A Portfolio With Potential Blockbuster Value
SENS-401 - A Portfolio With Potential Blockbuster Value SSNHL CIO From treatment to prevention SENS-401 Value SENS-401 SSNHL clinical data and insight derisk further development of SENS-401 in other indications Copyright by Sensorion - 2023 - All Rights Reserved 27
Slide 28: Sudden Sensorineural Hearing Loss Affects More Than 200,000 Patients Per Year
Sudden Sensorineural Hearing Loss (SSNHL) Is A Severe Disease Affecting More Than 200,000 Patients Per Year 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, heari
Slide 29: SENS-401 SSNHL Program: Phase 2 Design
SENS-401 SSNHL Program: Phase 2 Design A RANDOMIZED, MULTICENTER, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL 50 clinical sites globally 1 Primary endpoint Audiometry 15 dB improvement vs. Placebo 115 patients enrolled Timing Control group: +/- oral corticosteroids Q1 2019 June 20 & Q1 2022 center Jan 21
Slide 30: SENS-401 Plasmatic Exposure
SENS-401 Plasmatic Exposure Plasmatic concentration (Pre-dose at Day 14 and Day 28) Legend ng/mL SENS-401 High dose SENS-401 Low dose Placebo Copyright by Sensorion - 2023 - All Rights Reserved 30
Slide 31: SENS-401 Induces Complete PTA Recovery In 50% Of Patients
SENS-401 Induces Complete PTA Recovery In 50% Of Patients Complete PTA recovery 70 p=0.1452 * p=0.0230 Complete PTA p=0.1230 p=0.2707 Placebo High Dose Low Dose 60 recovery (n/n total) % patients with complete PTA 50 Day 28 9/28 12/23 13/26 40 recovery 30 Day 84 8/26 8/18 14/22 20 10 Legend 0 SENS-4
Slide 32: Although Primary Endpoint Not Met, Data Supports And Informs Further Clinical Development
Although Primary Endpoint Not Met, Data Supports And Informs Further Clinical Development PTA improvement from baseline compared to placebo on per protocol idiopathic SSNHL 5 Estimate PTA change from baseline (dB) Day 7 Day 14 Day 28 Day 84 High N= 21 N= 23 N= 22 N= 17 0 dose Low N= 26 N= 26 N= 26 N
Slide 33: SENS-401 Effect Is More Pronounced In A Profound Hearing Loss Sub-group
SENS-401 Effect Is More Pronounced In A Profound Hearing Loss Sub-group (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) High N= 11 N= 11 N= 9 N= 9 -5 dose Low -10 N= 11 N= 11 N= 9 N= 9 dose Placebo N= 14 N= 15 N= 15
Slide 34: SENS-401 SSNHL Phase 2 Results Summary
SENS-401 SSNHL Phase 2 Results Summary Seeking Partners For Late-Stage Development And Commercialization AUDIBLE-S SECONDARY ENDPOINT RESULTS • Complete PTA recovery is achieved in 50% of the SENS-401 treated patients • SENS-401 shows a clinically meaningful and statistically significant effect on P
Slide 35: Cisplatin Administration Damages the Inner Ear and Leads to Hearing Loss
Cisplatin Administration for Chemotherapies 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 cumulative cisplatin doses. CIO lea
Slide 36: SENS-401 Phase 2a Proof-of-Concept Study First Data Expected H2 2023
SENS-401 Phase 2a Proof-of-Concept Study First Data Expected H2 2023 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 Neoplastic Disease SCREENING PERIOD (between -28 and RA
Slide 37: SENS-401 To Preserve Residual Hearing After Cochlear Implantation
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 KEY FIGURES Growing understanding of the link between healthy hearing and healthy ageing 36,450 Implants sold by Cochle
Slide 38: SENS-401 Study Commenced In Sept. 2022 First Data Expected Mid-2023
SENS-401 Study Commenced In Sept. 2022 First Data Expected Mid-2023 A Phase 2a, Multicenter, Randomized, Controlled, Open-label Study SCREENING PERIOD EVALUATION PERIOD D1 D8 D49 D105 CI switch on Randomization SENS- Treated Arm: SENS-401 b.i.d Follow-up 401 Arm A COCHLEAR up to IMPLANTATION EFFICAC
Slide 39: SENS-401 Program Key Milestones, Data Readouts Mid 2023
SENS-401 Program Key Milestones, Data Readouts Mid 2023 SENS-401 CIO NOTOXIS CTA First patient enrolled in SENS-401 amendment approved Oct 2022 CIO NOTOXIS Dec 2022 SENS-401 combo with cochlear implants SENS-401 CIO NOTOXIS - preliminary results mid 2023 - preliminary results H2 2023 Copyright by Se
Slide 40: Sensorion Newsflow
Sensorion Newsflow [estimated timelines] • Mid-2022 – OTOF-GT: delivery of batches for toxicology study • H2 2022 – OTOF-GT: EMA’s positive opinion for ODD • H2 2022 – SENS-401 CIO: NOTOXIS CTA study amendment approval • H2 2022 – OTOF-GT: FDA approval for RPDD • H2 2022 – OTOF-GT: FDA approval for
Slide 41: Thank You
THANK YOU E: contact@sensorion-pharma.com

Sensorion — Investor Presentation (May 2023) on 6ix

Sensorion Sas — Sensorion — Investor Presentation (May 2023)

Slide 1: Unlocking the potential of advanced therapies for hearing loss

Slide 2: Sensorion vision

Slide 3: Disclaimer

Slide 4: Sensorion: Overview

Slide 5: We Want People With Inner Ear Hearing Disorders To Live Life With Unlimited Connections

Slide 6: Our Vision: A Global Franchise Establishing Leadership In The Hearing Space

Slide 7: Together With Best-In-Class Partners We Can Transform the Current Standard of Care

Slide 8: Sensorion is Well Positioned to Transform the Hearing Landscape

Slide 9: Sensorion's Portfolio Of Advanced Hearing Loss Therapies

Slide 10: An Experienced Leadership Team and SAB

Slide 11: We Have Established Strong Internal Capabilities To Ensure Successful Execution

Slide 12: Our Team has Significant Experience in Gene Therapy Clinical Development

Slide 13: Gene Therapy Programs

Slide 14: Sensorion's Gene Therapy Programs Target Rare Auditory Diseases

Slide 15: Aiming To Develop Best-in Class And First-in Class Gene Therapy

Slide 16: Gene Therapy Pediatric Indications Have Blockbuster Sales Potential

Slide 17: OTOF Gene Encodes Otoferlin, A Key Ca2+ Sensor Protein

Slide 18: Dual AAV-OTOF Resulted In IHCs Specific Expression And Hearing Restoration In DFNB9 Mice

Slide 19: Optimized Surgical Procedure Leads To IHC Specific AAV-delivered Transgene Transduction In Mature NHP Cochlea

Slide 20: Otoferlin Audinnove Consortium Provides Privileged Access To Patients And Surgeons

Slide 21: OTOF Gene Therapy Program Status – Progressing On Track

Slide 22: Connexin 26: A Gap-junction Protein Encoded By GJB2 Gene

Slide 23: Candidate Selected Prevents Hearing Loss in Relevant Mouse Model

Slide 24: GJB2 Gene Therapy Program Next Steps

Slide 25: Small Molecule Programs

Slide 26: SENS-401: Multiple Indications To Treat And Prevent Hearing Loss

Slide 27: SENS-401 - A Portfolio With Potential Blockbuster Value

Slide 28: Sudden Sensorineural Hearing Loss Affects More Than 200,000 Patients Per Year

Slide 29: SENS-401 SSNHL Program: Phase 2 Design

Slide 30: SENS-401 Plasmatic Exposure

Slide 31: SENS-401 Induces Complete PTA Recovery In 50% Of Patients

Slide 32: Although Primary Endpoint Not Met, Data Supports And Informs Further Clinical Development

Slide 33: SENS-401 Effect Is More Pronounced In A Profound Hearing Loss Sub-group

Slide 34: SENS-401 SSNHL Phase 2 Results Summary

Slide 35: Cisplatin Administration Damages the Inner Ear and Leads to Hearing Loss

Slide 36: SENS-401 Phase 2a Proof-of-Concept Study First Data Expected H2 2023

Slide 37: SENS-401 To Preserve Residual Hearing After Cochlear Implantation

Slide 38: SENS-401 Study Commenced In Sept. 2022 First Data Expected Mid-2023

Slide 39: SENS-401 Program Key Milestones, Data Readouts Mid 2023

Slide 40: Sensorion Newsflow

Slide 41: Thank You