Sensorion Sas · EURONEXT:ALSEN
Sensorion Sas — Sensorion — Investor Presentation (November 2023)
43 slides from Sensorion Sas's investor presentation, November 2023
Slide 1: Unlocking the potential of advanced therapies for hearing loss
Unlocking the potential of advanced therapies for hearing loss November 2023
Slide 2: Sensorion vision
1 SENSORION Our vision is to help people with inner ear hearing disorders to live life with unlimited Copyright by Sensorion - 2023 - All Rights Reserved connections
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: • SENS-501 (OTOF-GT) caused by mutations of the gene encoding for otoferlin, EU & US ODD, US RPDD, CTA filed in the UK and in Europe • G
Slide 5: 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 SENS-501 SENS-401 Ecosystem • EU Natural History Key Opinion Centers of Patient Groups Partners • US Studies Leaders Excellence • ROW Copyright by Sensorion - 202
Slide 6: 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 7: 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) – SENS-501 Sudden Sensorineural Hearing Loss (SSNHL) CTA filed (UK MHRA & Europe) AUDIBLE-S Ph2 study completed • H
Slide 8: 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) OTOF-GT* UK & Europe CTA Otoferlin Deficiency SENS-501 Approval H2 2023 Adult onset IND-enabling RESTORE GJB2-GT* (presbycusis) precli
Slide 9: We Have Established Internal Capabilities to Ensure Successful Execution
We Have Established Internal Capabilities to Ensure Successful Execution PRECLINICAL - CLINICAL CMC GENE THERAPY REGULATORY PATIENT SMALL MOLECULES & EXPERIENCE FACILITIES EXPERTISE ACCESS GT PROGRAMS • Cell Model Platform: assays • 400 people enrolled in • Process development: non- • Develop regula
Slide 10: 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 11: Gene Therapy Programs
2 GENE THERAPY PROGRAMS Copyright by Sensorion - 2023 - All Rights Reserved
Slide 12: 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 13: 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 14: Gene Therapy Pediatric Indications Have Blockbuster Sales Potential
Gene Therapy Pediatric Indications Have Blockbuster Sales Potential CTA Submitted in UK (MHRA) and Europe SENS-501 (OTOF-GT) is the perfect pilot program • SENS-501 will be the pilot program demonstrating that GT is a relevant • Well understood biology and pathology of the otoferlin medical approach
Slide 15: 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 16: SENS-501 Leads to Long-term Hearing Recovery in a Translational Model
SENS-501 Leads to Long-term Hearing Recovery in a Translational Model of Otoferlin Deficiency Long-term hearing restoration Hearing restoration corrlates with de novo OTOF expression AAV-mOTOF injected in mice before hearing onset SENS-501 injected in mice after hearing onset O/IHC: Outer/Inner Hair
Slide 17: SENS-501 Leads to Restoration of Efficient Sound Processing in Behavioural Test
SENS-501 Leads to Restoration of Efficient Sound Processing in Behavioural Test Behavior test based on hearing recovery implemented in mouse White noise pulse (2-20 kHz, 115 dB) Wild type Non-injected Otof ko/ko Restored Otof ko/ko Olivier et al. ASGCT 2023 link Copyright by Sensorion - 2023 - All R
Slide 18: Dedicated Surgical Approach for Gene Therapy
Dedicated Surgical Approach for Gene Therapy Non-Human Primates injected through the round window membrane (RWI) with or without stapedotomy (stap) 1 Fenestration (Round window membrane) Used for cochlear implant Surgical approach • Overpressure • Limited volume • Backflow • Surgical procedure is si
Slide 19: 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 20: SENS-501 (OTOF) Gene Therapy Program Status – Progressing
SENS-501 (OTOF) Gene Therapy Program Status – Progressing Submission of European Natural Preclinical package completed History Study OTOCONEX GMP Drug product successfully Injection device system manufactured development completed CTA Submission CTA Approvals UK MHRA & Europe H2 2023 Copyright by Se
Slide 21: 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 22: Selected Candidate Prevents Hearing Loss in Relevant Mouse Model
Selected Candidate 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 23: 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 23
Slide 24: Small Molecule Programs
3 SMALL MOLECULE PROGRAMS Copyright by Sensorion - 2023 - All Rights Reserved
Slide 25: 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 26: SENS-401 - a Portfolio With Potential Blockbuster Value
SENS-401 - a Portfolio With Potential Blockbuster Value CI + SSNHL CIO For treatment And prevention Blockbuster Potential SENS-401 SSNHL clinical data and insight derisk further development of SENS-401 in other indications Copyright by Sensorion - 2023 - All Rights Reserved 26
Slide 27: Sudden Sensorineural Hearing Loss (SSNHL) is a Severe Disease
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 28: 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 29: 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 29
Slide 30: 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 31: 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 32: 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 33: SENS-401 Study Commenced In Sept. 2022, Positive Preliminary Results Reported
SENS-401 Study Commenced In Sept. 2022 Positive Preliminary Results Reported 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 IMPLANTATIO
Slide 34: SENS-401 is Detected in the Perilymph of the First 5 Treated Patients
SENS- 401 is Detected in the Perilymph of the First 5 Treated Patients at Levels Consistent with Those Observed in Animals Perilymph Concentrations Data Rat Guinea Pig Cat Human Minimal perilymph concentration in animals • Plasma concentrations of SENS-401 in humans at steady state: dosing of 43.5 m
Slide 35: Residual Low Frequency Hearing Benefits for Cochlear Implant Users
Residual Low Frequency Hearing Benefits for Cochlear Implant Users Initial shift* Second shift* (2-4 weeks postoperative) (3-6 months postoperative) between surgery and initial activation of the device • Attributed to perioperative factors • Attributed to intracochlear fibrosis, excitotoxic changes
Slide 36: SENS-401 Preserves Early Loss of Residual Hearing
SENS- 401 Preserves Early Loss of Residual Hearing - As Shown in all Five Patients Treated so far Mean 3 Frequency PTA Change (250, 500 And 750 Mean Change from Baseline At 500 Hz, Hz), in the Implanted Ear, in the Implanted Ear, from Baseline to 6 Weeks After Implantation 6 Weeks After Implantation
Slide 37: SENS-401 Also Preserves Post-Operative Hearing
SENS- 401 Also Preserves Post-Operative Hearing - As Measured at the End of the Treatment Period Change Of 500 Hz Hearing Threshold Mean 3-frequency PTA Change (250, 500 And 750 Hz) in the in the Implanted Ear, from Baseline to 6 Weeks Implanted Ear, From Baseline yo 6 Weeks After Implantation After
Slide 38: SENS-401 CI Preliminary Results Conclusion
SENS-401 CI Preliminary Results Conclusion Preliminary key results are promising and suggest that SENS-401 can cross the labyrinthine barrier to target cochlear hair cells. Six weeks post-cochlear implantation, the residual hearing loss whether assessed at 500 Hz or across an average of 3 consecutiv
Slide 39: 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 40: 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 41: SENS-401 Program Key Milestones, Data Readouts in 2023
SENS-401 Program Key Milestones, Data Readouts in 2023 SENS-401 with cochlear implants First patient enrolled in SENS-401 - Positive Preliminary Results Reported CIO NOTOXIS Dec 2022 SENS-401 with cochlear implants SENS-401 CIO NOTOXIS - Final data readout H1 2024 - preliminary results H2 2023 Copyr
Slide 42: Sensorion Newsflow
Sensorion Newsflow [estimated timelines] • H2 2022 – SENS-501: EMA & FDA ODD, FDA RPDD • Q2 2023 – GJB2-GT: Candidate selection • Mid-2023 – SENS-401 in combination with cochlear implantation: preliminary results • Q2 2023 – SENS-501: Clinical Trial Application (CTA) first submission (MHRA) • July 2
Slide 43: Thank You
THANK YOU E: contact@sensorion-pharma.com