Sensorion Sas · EURONEXT:ALSEN
Sensorion Sas — Sensorion — Investor Presentation (March 2021)
38 slides from Sensorion Sas's investor presentation, Mar 8, 2021
Slide 1: Corporate Presentation March 2021
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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: Investment Highlights
INVESTMENT HIGHLIGHTS Sensorion is focused on innovative treatments that can restore, treat and prevent hearing loss Three novel gene therapy programs targeting unmet needs in Otoferlin Deficiency, GJB2-related hearing loss and Usher FINANCIAL OVERVIEW Syndrome Type 1 Date Established...............
Slide 4: Sensorion
1 SENSORION 4
Slide 5: Management Team
MANAGEMENT TEAM EDWIN MOSES NAWAL OUZREN GÉRALDINE HONNET JUERGEN HEITMANN Chairman of the Board Chief Executive Officer Chief Medical Officer Chief Business Officer SENSORION SENSORION SENSORION SENSORION (Since 2020) (Since 2017) (Since 2020) (Since 2017) Chairman of the Board SHIRE GENETHON TAKED
Slide 6: Sensorion is building up a gene therapy franchise with Institut Pasteur
SENSORION Sensorion is building up a gene therapy franchise in collaboration with Institut Pasteur Management team highly experienced in gene therapy and drug development FINANCIAL OVERVIEW Date Established............................................... 2009 RESTORE, TREAT and PREVENT in the field o
Slide 7: Strategy: Restore, Treat & Prevent Hearing Loss
STRATEGY: RESTORE, TREAT & PREVENT HEARING LOSS GENE THERAPY APPROACH Exclusive collaboration signed with Institut Pasteur in Gene Therapy to RESTORE auditory functions Program to RESTORE hearing in Otoferlin deficiency (DFNB9 deafness), one of the most common forms of congenital deafness Program to
Slide 8: Institut Pasteur is leading the way in the genetics of hearing
INSTITUT PASTEUR IS LEADING THE WAY IN THE GENETICS OF HEARING CHRISTINE PETIT MD, PhD Chair of Genetics and Cellular Physiology, GENETICS AND PHYSIOLOGY OF HEARING Professor at College de France UNIT AT INSTITUT PASTEUR LED BY PROFESSOR Professor at Institut Pasteur (Paris) CHRISTINE PETIT Head of
Slide 9: Broad strategic R&D collaboration with Institut Pasteur on genetics of hearing
SENSORION HAS ENTERED INTO A BROAD STRATEGIC R&D COLLABORATION WITH INSTITUT PASTEUR ON GENETICS OF HEARING SENSORION HAS A RIGHT OF FIRST REFUSAL ON ALL GENE THERAPY PROGRAMS IN THE FIELD OF INNER EAR AT INSTITUT PASTEUR Project Agreement “OTOF” Project Agreement “Usher T1” Master Sensorion Project
Slide 10: Scientific Advisory Board
SCIENTIFIC ADVISORY BOARD Pr Christine Petit Chair of the Scientific Advisory Board Pr Alain Fischer Dr Diane Lazard Dr Hernán López-Schier Pr Paul Avan Dr Rob Dow Professor at College de France ENT Surgeon Senior Group Leader and Physicist and Medical Doctor in >37 years of experience in the 2009-2
Slide 11: The inner ear is one of the most delicate organs in the human body
THE INNER EAR IS ONE OF THE MOST DELICATE ORGANS IN THE HUMAN BODY Inner Ear Biology KEY FACTS ~466m PATIENTS AFFECTED BY HEARING LOSS WORLDWIDE* Every human is born with a specific number of sensory hair cells 3,500 Inner Hair Cells ~900m PATIENTS PROJECTED TO BE AFFECTED BY 2050* 12,000 Outer Hair
Slide 12: Pipeline: building an attractive pipeline in the hearing space
PIPELINE: BUILDING AN ATTRACTIVE PIPELINE IN THE HEARING SPACE Next milestones Product Indication Discovery In vivo PoC Pre-clinical Phase 1 Phase 2 Phase 3 (estimated timelines) TREAT Sudden SENS-401 sensorineural Phase 2 topline results hearing loss (Q4 2021) Cisplatin Start of natural history SEN
Slide 13: Gene Therapy Restore
2 GENE THERAPY RESTORE 13
Slide 14: 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 t
Slide 15: 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 16: Dual AAV OTOF gene therapy - mechanism of action
RESTORE DUAL AAV OTOF GENE THERAPY - MECHANISM OF ACTION vector functional otoferlin containing with 6 C2 domains N-Terminal AAV-otof (N-Ter) coding sequence recombination transcription splicing vector containing Translation to functional otoferlin C-Terminal AAV-otof (C-Ter) coding sequence Copyrig
Slide 17: Pre-clinical OTOF gene therapy proof of concept restores cochlear receptor function
PRE-CLINICAL OTOF GENE THERAPY PROOF OF RESTORE CONCEPT DURABLY RESTORES COCHLEAR RECEPTOR FUNCTION IN A KNOCK-OUT MOUSE MODEL Immunostained inner hair cells in wild type, Otof -/- and Otof -/- injected with dual AAV2 quadY-F OTOF vector expression of Otof protein in cochlear receptors Normal mice O
Slide 18: 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 Natural history: cl
Slide 19: OTOF gene therapy program status
RESTORE OTOF GENE THERAPY PROGRAM STATUS PoC data in mouse PoC preliminary data in Non- Human Primates Product Development and Manufacturing Agreement Next step: Advice from regulatory authorities Copyright by Sensorion - 2021 - All RightsReserved 19
Slide 20: Connexin 26 is a gap-junction protein encoded by GJB2 gene
CONNEXIN 26 IS A GAP-JUNCTION PROTEIN ENCODED BY RESTORE GJB2 GENE 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 21: GJB2 identified in Institut Pasteur's systematic process for early severe presbycusis
GJB2 HAS BEEN IDENTIFIED AS PART OF INSTITUT PASTEUR’S RESTORE DELIBERATE AND SYSTEMATIC PROCESS TO IDENTIFY MONOGENIC FORMS OF EARLY ONSET OF SEVERE PRESBYCUSIS 105 122 120 mARHL sARHL controls TOTAL COHORTS Severe presbycusis is a bilateral progressive loss of Auditory scoring: Auditory scoring: 9
Slide 22: GJB2 gene therapy program next steps
RESTORE GJB2 GENE THERAPY PROGRAM NEXT STEPS Natural history study Candidate selection Preclinical IND enabling studies Copyright by Sensorion - 2021 - All RightsReserved 22
Slide 23: USH1G gene encodes SANS, essential for mechanoelectrical transduction
RESTORE USH1G GENE ENCODES "SANS", AN ESSENTIAL PROTEIN FOR MECHANOELECTRICAL TRANSDUCTION Sans protein Tip link Tip link MET channel MET channel Myosin 7a cdhr23 K+, Ca2+ cdhr15 Harmonin b Stereocilia on top of hair cells Adapted from Mathur and Yang. 2014 Adapted from Emptoz et al. 2017 (link) Tip
Slide 24: USH1G gene therapy restored hearing & vestibular functions
RESTORE USH1G GENE THERAPY RESTORED HEARING & 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-/
Slide 25: USH1G gene therapy restored hearing & vestibular functions (cont.)
RESTORE USH1G GENE THERAPY RESTORED HEARING & VESTIBULAR FUNCTIONS (CONT.) PROOF OF CONCEPT IN A KNOCK-OUT MOUSE MODEL BY INSTITUT PASTEUR Thesensitivity The sensitivityofofthe the transductioncurrent transduction current Mechanoelectrical transduction (MET) currents recorded responsetotohair respon
Slide 26: USH1G gene therapy program status
RESTORE USH1G GENE THERAPY PROGRAM STATUS PoC data in mouse In progress: PoC data in mouse with an extended therapeutic window Next step: PoC Non-Human Primates study Next step: Advice from regulatory authorities Copyright by Sensorion - 2021 - All RightsReserved 26
Slide 27: SENS-401 Treat and Prevent
3 SENS-401 TREAT AND PREVENT 27
Slide 28: SENS-401 mechanism of action targets multiple indications
SENS-401 MECHANISM OF ACTION CREATES THE OPPORTUNITY TO TARGET MULTIPLE INDICATIONS WITH ONE COMPOUND Disrupted Ca2+ Calcineurin Neurodegenerative cascades: INSULT homeostasis Activation NFAT translocation: oxidative stress, survival, inflammation Excitotoxicity pathways Neuro Inflammation Cell deat
Slide 29: SENS-401: multiple indications pursued to treat and prevent hearing loss
SENS-401: MULTIPLE INDICATIONS PURSUED TO TREAT AND PREVENT HEARING LOSS ORALLY AVAILABLE SMALL MOLECULE 5HT3 RECEPTOR ANTAGONIST & CALCINEURIN INHIBITOR – ESTIMATED TIMELINES Indication 2020 2021 Sudden Sensorineural Hearing Loss (SSNHL)* Phase 2 read-out Start of Clinical trial Cisplatin induced o
Slide 30: SSNHL 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 31: 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 days) O
Slide 32: Daily administration of SENS-401 reduces auditory deficit in rats
TREAT DAILY ADMINISTRATION OF SENS-401 REDUCES AUDITORY DEFICIT IN RATS A daily oral administration of SENS-401 (13.2 mg/kg bid) reduces auditory deficit and improves recovery MODEL BENEFIT Randomized treatment post-noise induced trauma Regulatory threshold for efficacy (>10 dB improvement) (2h expo
Slide 33: SENS-401 Phase 2 to treat SSNHL
TREAT SENS-401 PHASE 2 TO TREAT SSNHL A RANDOMIZED, MULTICENTER, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL 50 clinical sites globally 1 Primary endpoint Audiometry 10dB improvement vs. placebo Enrollment 258 patients Timing Q1 2019 June 2020 Q4 2021 center Positive Final openings DSMB results Review D-
Slide 34: SENS-401 pre-clinical proof of concept in cisplatin induced hearing loss
PREVENT SENS-401 PRE-CLINICAL PROOF OF CONCEPT IN CISPLATIN INDUCED HEARING LOSS Significantly SIGNIFICANTLY REDUCES CISPLATIN-INDUCED HEARING LOSS more surviving AND OUTER HAIR CELL DEATH IN PRE-CLINICAL MODELS outer hair cells were present after SENS-401 treatment TREATMENT PROTOCOL compared with
Slide 35: Collaboration with Cochlear Ltd
PREVENT COLLABORATION WITH COCHLEAR® LTD 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 34,000 In Q4 2017 Sensorion and Cochlear® entered into Implants sol
Slide 36: Potential Newsflow
SENSORION Potential Newsflow [Estimated timelines] Q1 2021 – Final pre-clinical data for SENS-401 in cochlear implant study H1 2021 – Start of CIO Natural History Clinical Trial H1 2021 – Discussions with regulatory authorities on potential OTOF clinical study H1 2021 – Confirmatory pre-clinical PoC
Slide 37: Investment Highlights
INVESTMENT HIGHLIGHTS Sensorion is focused on innovative treatments that can restore, treat and prevent hearing loss Three novel gene therapy programs targeting unmet needs in Otoferlin Deficiency, GJB2-related hearing loss and Usher FINANCIAL OVERVIEW Syndrome Type 1 Date Established...............
Slide 38: Thank You
THANK YOU Nawal Ouzren Chief Executive Officer E: contact@sensorion-pharma.com 30