Sensorion Sas · EURONEXT:ALSEN
Sensorion Sas — Sensorion — Investor Presentation (February 2022)
35 slides from Sensorion Sas's investor presentation, February 2022
Slide 1: February 2022
February 2022 11
Slide 2: Disclaimer
DISCLAIMER • This document has been prepared by Sensorion (the "Company") and is provided for information purposes only. This document does not purport to contain comprehensive • This document contains certain forward-looking statements. These statements are not guarantees of the Company’s future pe
Slide 3: Sensorion
1 SENSORION Copyright by Sensorion - 2022 - All Rights Reserved 3
Slide 4: Investments Highlights
Investments Highlights • Sensorion is focused on innovative treatments that can restore, treat and prevent hearing loss − Three novel gene therapy programs targeting monogenic forms of deafness: • caused by a mutation of the gene encoding for Otoferlin • related to mutation in GJB2 gene FINANCIAL OV
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*: ~ 466m people affected by disabling hearing loss worldwide ~ 700
Slide 6: Our strategy: Restore, Treat & Prevent hearing loss
Our strategy: RESTORE, TREAT & PREVENT hearing loss Causes of hearing loss GENE THERAPY APPROACH • Exclusive collaboration signed with Institut Pasteur in Gene Therapy to Idiopathic RESTORE auditory functions 25% • Program to RESTORE hearing in Otoferlin deficiency (DFNB9 deafness), Genetic one of t
Slide 7: Our critical strategic alliances from bench to bedside
Our critical strategic alliances from bench to bedside French Military Biomedical Research Institute CLINICAL • Access to a military population at risk of noise-induced hearing loss • EU reference center for monogenic forms of deafness RESEARCH • Strong medical network, strict monitoring and precise
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) Sudden Secondary endpoints TREAT SENS-401 sensorineural Mid-March 2022 hearing loss Clinical Trial Cisp
Slide 9: An experienced team, board of directors and SAB
An experienced team, board of directors and SAB NAWAL OUZREN GÉRALDINE HONNET NORA YANG OTMANE BOUSSIF STEPHANIE FILIPE Chief Executive Officer Chief Medical Officer Chief Scientific Officer Chief Technical Officer Head of PMO SENSORION SENSORION SENSORION SENSORION SENSORION (Since 2017) (Since 202
Slide 10: We have established internal capabilities to ensure successful execution
We have established internal capabilities to ensure successful execution PRECLINICAL CAPABILITIES CLINICAL EXPERIENCE CMC GENE THERAPY REGULATORY EXPERTISE FOR SMALL MOLECULES FACILITIES & GT PROGRAMS • In Vitro platform: assays • 400 people enrolled in • Process development lab: • Multiple regulato
Slide 11: Gene Therapy: Restore
3 GENE THERAPY RESTORE Copyright by Sensorion - 2022 - All Rights Reserved 11
Slide 12: Strategic R&D collaboration with Institut Pasteur on genetics of hearing
RESTORE Strategic R&D collaboration with Institut Pasteur on genetics of hearing 3 PROGRAMS INITIATED UNDER THE COLLABORATION AGREEMENT WITH INSTITUT PASTEUR Rare disease, high unmet medical need Master Research Project Agreement “OTOF” Sensorion Agreement has an option Project Agreement “GJB2” on a
Slide 13: Sensorion's first gene therapy programs to treat rare auditory diseases
RESTORE Sensorion’s first gene therapy programs to treat rare auditory diseases 3 PROGRAMS INITIATED UNDER THE STRATEGIC COLLABORATION AGREEMENT WITH INSTITUT PASTEUR OTOFERLIN DEFICIENCY GJB2-RELATED HEARING LOSS USHER SYNDROME TYPE 1 • Patients with mutations in OTOF suffer from We have identified
Slide 14: 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 primed vesicle containing restricted SNARE interaction otoferlin SNARE interaction SNARE-com
Slide 15: Dual AAV-OTOF targeting IHCs resulted in durable hearing restoration in mature DFNB9 mice
Dual AAV-OTOF targeting IHCs resulted in durable hearing RESTORE restoration in mature DFNB9 mice HEARING RESTORATION TROPISM • Data showing 5-months AAV-OTOF efficacy in DFNB9 mouse model: the expression of Otof protein in mature Otof-/- • The chosen capsid of AAV-OTOF targets mice injected with du
Slide 16: AAV-OTOF vector transduces IHCs with high efficiency and specificity in mature NHP cochlea
RESTORE AAV-OTOF vector transduces IHCs with high efficiency and specificity in mature NHP cochlea 1 4 IHC 2 5 • The AAV-OTOF delivers transgenes to IHCs and not OHCs in non-human-primates • Transduction efficiency reaches more than 50% along the Apex to Base axis in the cochlea • The surgical proce
Slide 17: Otoferlin Audinnove consortium provides privileged access to patients and surgeons
Otoferlin “Audinnove” consortium provides privileged RESTORE 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 • Audio
Slide 18: OTOF gene therapy program status
RESTORE OTOF gene therapy program status PoC data in mouse Advice from regulatory PoC data in mouse authorities Submission of PoC preliminary data in European Natural History Non-Human Primates Study OTOCONEX Product Development and Clinical Trial Application H1 2023 Manufacturing Agreement Copyrigh
Slide 19: Connexin 26: a gap-junction protein encoded by GJB2 gene
CONNEXIN 26: a gap-junction protein encoded by GJB2 gene RESTORE and responsible for tissue homeostasis mutations in the gene leads 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 hexam
Slide 20: GJB2 gene therapy program next steps
RESTORE GJB2 gene therapy program next steps PoC data in mouse Submission of European Natural Submission of Natural History Study History Study OTOCONEX in collaboration with Sonova Candidate selection mid 2022 Preclinical IND enabling studies Copyright by Sensorion - 2022 - All Rights Reserved 20
Slide 21: USH1G gene therapy restored vestibular functions
RESTORE USH1G gene therapy restored vestibular functions PROOF OF CONCEPT IN A KNOCK-OUT MOUSE MODEL BY INSTITUT PASTEUR Mouse displacement recordings Cochlear stereocilia Vestibular stereocilia in Open Field Test Normal mice Control mice treated Control mice untreated Ush1g-/-mice Ush1g-/- untreate
Slide 22: USH1G gene therapy restored hearing functions
RESTORE USH1G gene therapy restored hearing functions PROOF OF CONCEPT IN A KNOCK-OUT MOUSE MODEL BY INSTITUT PASTEUR Mechanoelectrical transduction (MET) currents recorded ex vivo (recording of peak amplitude of the MET currents) USH1G-/-mice Treated USH1G-/-mice Source: Emptoz et al. 2017 (link) T
Slide 23: USH1G gene therapy program status
RESTORE USH1G gene therapy program status PoC data in mouse PoC data in mouse with an extended therapeutic window Sensorion and Institut Pasteur to discuss next steps H1 2022 Copyright by Sensorion - 2022 - All Rights Reserved 23
Slide 24: SENS-401: Treat and Prevent
2 SENS-401 TREAT AND PREVENT Copyright by Sensorion - 2022 - All Rights Reserved 24
Slide 25: SENS-401: multiple indications with one compound
SENS-401: multiple indications with one compound Sudden Sensorineural Cisplatin Induced Cochlear Implantation Hearing Loss Ototoxicity 5-HT3 antagonist & modulates calcineurin activity Disrupted Ca2+ Calcineurin Activation Neurodegenerative cascades: INSULT homeostasis Excitotoxicity NFAT translocat
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 Sudden TREAT SENS-401 sensorineural hearig loss* Cisplatin SENS-401 PREVENT induced ototoxicity Hearing preservation SENS-401 after cochlear implantation *Pat
Slide 27: Sudden sensorineural hearing loss and cisplatin induced ototoxicity can lead to permanent hearing loss
Sudden sensorineural hearing loss and cisplatin induced ototoxicity can lead to permanent disabling hearing loss WHAT IS SSNHL? WHAT IS CIO? The sudden onset of a significant hearing loss due to dysfunction of the cells of Hearing loss caused by cisplatin administration as chemotherapeutic treatment
Slide 28: SENS-401 developed to treat sudden sensorineural hearing loss
TREAT SENS-401 developed to treat sudden sensorineural hearing loss SENS-401 DEMONSTRATED SAFETY SENS-401 MARKET EXCLUSIVITY IN PHASE 1 SENS-401 MARKET EXCLUSIVITY • 36 healthy volunteers enrolled in a double-blind, randomized, • Strong IP with 2 patent families multiple ascending dose design (7 day
Slide 29: SENS-401 SSNHL Program: Phase 2 design
TREAT TREAT 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 Q1 2019 June 2020 Q1 2022 center Positive Final openings DSMB resu
Slide 30: SENS-401 SSNHL Program: Phase 2 results
RESTOR TREAT E SENS-401 SSNHL Program: Phase 2 results AUDIBLE-S PRIMARY ENDPOINT RESULTS • Safe and well tolerated in 115-patient SSNHL study; primary endpoint not met • A sub-analysis in participants with hearing threshold > 80dB (severe hearing loss) showed a better response compared to placebo a
Slide 31: SENS-401 Cisplatin Induced Ototoxicity preclinical results led to POC clinical study submission
PREVENT SENS-401 Cisplatin Induced Ototoxicity preclinical results led to POC clinical study submission (NOTOXIS study) SIGNIFICANTLY REDUCES CISPLATIN-INDUCED HEARING LOSS AND OUTER HAIR CELL DEATH IN PRE-CLINICAL MODELS TREATMENT PROTOCOL Significantly more SENS-401 6.6 mg/kg, 13.2 mg/kg or placeb
Slide 32: SENS-401 clinical study submission to preserve residual hearing post cochlear implantation
PREVENT SENS-401 clinical study submission to preserve residual hearing post cochlear implementation COMBINATION OF COCHLEAR IMPLANT WITH SENS-401 TO PREVENT CELL-DEATH POST COCHLEAR IMPLANT PROCEDURE HEALTHY AGEING Growing understanding of the link between healthy hearing and healthy ageing 31,600
Slide 33: SENS-401 program next steps
RESTORE SENS-401 program next steps Secondary endpoints data readout SENS-401 in SSNHL Mid-March 2022 CTA submission for SENS-401 study to preserve residual hearing post cochlear implementation H1 2022 Receive CTA approval for NOTOXIS H1 2022 Start enrolment in the NOTOXIS study Copyright by Sensori
Slide 34: Sensorion potential newsflow
Sensorion potential newsflow [estimated timelines] • Mid-March 2022 – Secondary endpoints data readout for the SENS-401 Phase 2 clinical study in SSNHL • April 28, 2022 – Full year financial results • H1 2022 – Start of the SENS-401 NOTOXIS study in CIO • H1 2022 – CTA submission for SENS-401 study
Slide 35: Thank you
THANK YOU Nawal Ouzren Chief Executive Officer E: contact@sensorion-pharma.com 30