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Corporate Presentation January 2017 Caring for inner ear disability

Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

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Page 1: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Corporate PresentationJanuary 2017

Caring for inner ear disability

Page 2: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

• This document has been prepared by Sensorion (the "Company") and is provided for information purposes only. This document does not purport to contain comprehensive or complete

information about the Company and is qualified in its entirety by the business, financial and other information that the Company is required to publish in accordance with the rules,

regulations and practices applicable to companies listed on Euronext Paris. No reliance may be placed for any purposes whatsoever on the information or opinions contained in this

document or on its accuracy or completeness.

• This presentation does not constitute an offer to sell, a solicitation of, or an invitation to subscribe for or to buy, securities of Sensorion in any jurisdiction

• The information and opinions contained in this document are provided as of the date of this document only and may be updated, supplemented, revised, verified or amended, and thus

such information may be subject to significant changes. The Company is not under any obligation to update the information or opinions contained herein which are subject to change

without prior notice.

• The information contained in this document has not been subject to independent verification. No representation, warranty or undertaking, express or implied, is made as to the

accuracy, completeness or appropriateness of the information and opinions contained in this document. The Company, its subsidiaries, its advisors and representatives accept no

responsibility for and shall not, under any circumstance, be held liable for any loss or damage that may arise from the use of this document or the information or opinions contained

herein.

• This document contains information on the Company’s markets and competitive position, and more specifically, on the size of its markets. This information has been drawn from various

sources or from the Company’s own estimates which may not be accurate and thus no reliance should be placed on such information.

• This document contains certain forward-looking statements. These statements are not guarantees of the Company's future performance. These forward-looking statements

relate to the Company's future prospects, developments and marketing strategy and are based on analyses of earnings forecasts and estimates of amounts not yet

determinable. Forward-looking statements are subject to a variety of risks and uncertainties as they relate to future events and are dependent on circumstances that may or

may not materialize in the future. Forward-looking statements cannot, under any circumstance, be construed as a guarantee of the Company's future performance and the

Company’s actual financial position, results and cash flow, as well as the trends in the sector in which the Company operates, may differ materially from those proposed or

reflected in the forward-looking statements contained in this document. Important factors that could cause actual results to differ materially from the results anticipated in

the forward-looking statements include those discussed or identified in the “Risk Factors” section of our “Document de référence” registration document filed with the

Autorité des marchés financiers on July 28, 2016 under n°R.16-069 (a copy of which is available on www.sensorion-pharma.com). Even if the Company’s financial position,

results, cash-flows and developments in the sector in which the Company operates were to conform to the forward-looking statements contained in this document, such

results or developments cannot be construed as a reliable indication of the Company's future results or developments. The Company does not undertake any obligation to

update or to confirm projections or estimates made by analysts or to make public any correction to any prospective information in order to reflect an event or circumstance

that may occur after the date of this document.

• Certain figures and numbers appearing in this document have been rounded. Consequently, the total amounts and percentages appearing in the tables may not necessarily equal the

sum of the individually rounded figures, amounts or percentages.

• All persons accessing this document must agree to the restrictions and limitations set out above.

Disclaimer

Page 2

Page 3: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Disabling Diseases Without Effective TreatmentSignificant $10B+ market opportunity*

Vertigo

InnerEar

Middle Ear

Hearing Loss - Tinnitus

Existing marketed drugs

have limited efficacy and

various side effects

No product license approval

to date

Page 3

Vestibule

Cochlea

* Source: Alcimed, Sensorion

Page 4: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

“Pure player” in therapeutics for inner ear disorders (vertigo, hearing loss, and tinnitus)

– $10B untapped market opportunity*

Key Assets

– First-in-class small molecules administered orally

– De-risked pipeline entering phase 2 stage

Selection of drug candidates with clinical data

» SENS-111 for treatment of acute vertigo

» SENS-401 for treatment of sudden sensorineural hearing loss

7 patents families (composition of matter & method of use)

– Technology platform to support pipeline expansion and pharma collaboration

Execution capabilities to reach near term milestone events

– Publicly-traded company with strong balance sheet and experienced leadership team

Corporate Highlights

Page 4* Source: Alcimed, Sensorion

Page 5: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Significant Market Opportunity

* Based on estimated number of treatments per patient per year – excluding the age-related hearing loss segment; Source: Alcimed, Sensorion

Symptomatic treatment of crisis 123 million patients worldwide

Tinnitus(41 million

patients)$2.6B

Vertigo crisis(82 million

patients)$2.5B

$5.1B

Market size*

Anti-lesional curative treatment 21 million patients worldwide

$6.4B

Cancer treatment

toxicity(2 million patients)

$2.6B

$1.8B

Vertigo(8 million patients)

Hearing loss (11 million patients)

$2.0B

Market size*

$10B+ Global Market Potential

Page 5

Page 6: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Well Positioned Versus Competition to Address the Market

Page 6

Oral, IV, subQ drugs easy to take or give

Severe pathologies

Milder pathologies

Inner ear market

Invasive transtympanic injection by ENT specialists

Page 7: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Efficient, In-house Screening Platform Dedicated to Inner Ear

Page 7

15y+ academic & pharma know-how

Comprehensive toolbox to explore vestibular

& cochlear applications

AAALAC certified in-house platform

In vitro target assay

Functional cellular

Ex vivo assay

Toxicology – Manufacturing CMC*

Pharmacodynamics – In vivo disease models

Pharmacokinetics Local & Systemic

Functional cellular – ex vivo assay

In vitro target assay

First in inner ear patient with the best drug candidates

* Outsourced

Page 8: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Phase 2-ready PipelineScreening platform for next generation drugs

Page 8

1

2

3

Screening platform

Reproducible process for next generation products(Internal development & academic/pharma collaborations)

Phase2

Phase1

Product SENS-111Histamine H4 antagonistTreatment of acute vertigo

Product SENS-401Anti-lesional MOATreatment of sudden sensorineural hearing loss

Product Series SENS-300Undisclosed MOAPrevention of drug-induced ototoxicity

US IND granted

Orphan ODD in EU

Candidateselection

Product pipeline

(small molecules)

In vitro target assay

Functional cellular

Ex vivo assay

Toxicology – Manufacturing CMC*

Pharmacodynamics – In vivo disease models

Pharmacokinetics Local & Systemic

Functional cellular – ex vivo assay

In vitro target assay

Potential drug candidates

* Outsourced

Page 9: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Latest development in the histaminergic pathway applied to vertigo treatment

– First-in-class Histamine H4 Receptor antagonist (H4Ra) administered orally and non-sedative

Small molecule new chemical entity covered by composition of matter patent and use patents in inner ear disorders

until 2028 before patent term extension

Attractive drug profile

– MOA: restore balance by reducing neuronal activity in vestibules

– Positive preclinical comparative studies in acute vertigo

– Clinical tolerance demonstrated in 227 healthy volunteers up to 500 mg to date

– Pharmacokinetic profile allowing once-daily dosing

– Preliminary signal of activity on vertigo endpoints measured in clinical phase 1b study

– US IND granted by FDA

On-going implementation of an international Phase 2 study in Acute Unilateral Vestibulopathy with first patient

inclusion expected in Q1.2017

SENS-111 in Acute Vertigo

Page 9* Source: Alcimed, Sensorion

Page 10: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Study objectives

– Evaluate the safety of single and repeated ascending doses of SENS-111

– Determine the pharmacokinetic profile of SENS-111

– Document the effect of a routine vestibular stress test - caloric test

Phase 1 study design

– Randomized placebo controlled in 100 healthy volunteers

– Part A: Single oral dosing from 100 mg to 500 mg

– Part B: 4 to 7 days of daily oral dosing from 50 mg to 250 mg

Vestibular stress test: caloric test

– Routine test for ENT doctors to assess the vestibular function

– Unilateral irrigation of one auditory canal with cold water to induce a transient

vestibular dysfunction lasting for 2 to 3 minutes

– Recording of Vertigo sensation using visual analogic scales (VAS) and Nystagmus

using VideoNystagmoGraphy (VNG)

Page 10

SENS-111Phase 1b study successfully completed

Caloric test

Page 11: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Results*

– Safety

Well tolerated (16% mild adverse events on SENS-111 vs 36% on placebo)

No sedation

– Pharmacokinetics

Linear with doses up to 200 mg/day,

Long elimination half-life (24-48h)

Vestibular stress test

– Induced mild vertigo for 2 to 3 minutes

– Decreased duration of vertigo by 30% (28 sec decrease from 99 sec at baseline,

p<0.05)

– Activity in the 0 to 500-700 ng/mL concentration range

Conclusions

– SENS-111 is well tolerated and can be given once-daily

– Signal of anti-vertigo activity related to plasma concentration consistent with preclinical data

– Large dose window described in human to guide phase 2 dose selection

Page 11

SENS-111Phase 1b study successfully completed

seconds

* Presented at AAO-HNSF, September 18-21, 2016 - San Diego, USA and EACPT, October 6-9, 2016 – Opatija, Croatia

Page 12: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Acute unilateral vestibulopathy

– Pure vestibular dysfunction with sudden occurrence of severe acute vertigo lasting 4-7 days in patients aged between 30

and 60 years (no previous vestibular history, no hearing loss)

– Leads to immediate severely disabling postural imbalance with a tendency to fall

– Can lead to long term complications (40-50%): dizziness, imbalance, abnormal gait, unsteadiness

– Incidence of 3.5 to 15.5 per 100,000 people

– Not properly addressed by drug treatment:

Need for safe (non sedative) and effective drugs

Patient population of the SENS-111 Phase 2 study

Page 12

“AUV is assumed to be an ideal model for vestibular diseases. If this trial shows a benefit of any

agent, the drug is also going to be used in other diseases which lead to dizziness and vertigo”

Pr. Michael Strupp from Ludwig-Maximilians-University Munich, Germany

(KOL event, Sensorion webcast, November 29, 2016)

Page 13: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Primary objective

– To demonstrate the efficacy of SENS-111 100 mg and 200 mg versus placebo in the treatment of Acute Unilateral Vestibulopathy (AUV)

Secondary objectives

– Optimal dose regimen, safety, tolerability, plasma exposure, long term recovery, health economics & quality of life

Primary endpoint

– Vertigo intensity (visual analogic scale) measured by AUC of the VI-VAS in standing position

Other endpoints

– Worst vertigo intensity, Romberg test, nystagmus, nausea, disability (Dizziness Handicap Inventory and Vestibular Disorders Activities of Daily

Living Scale), time to unassisted walk, Health economics (Work Productivity and Activity Impairment questionnaire), PK, safety

Page 13

SENS-111 Phase 2 study objectives & End-points

A multicenter, randomized, double-blind, placebo-controlled study

to assess the efficacy and safety of 2 dose regimens of orally administered SENS-111 (100 mg and 200 mg)

given during 4 days in patients suffering from Acute Unilateral Vestibulopathy

Page 14: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Page 14

SENS-111 Phase 2 study design

Screening Treatment Follow-up

Randomization

D1 D2 D3 D4 D5 D14 D28

Visit Visit Visit Visit Visit Visit Visit

Dosing

Treatment arm – Dose 2

Placebo arm

Treatment arm – Dose 1 25 clinical sites in Europe,

USA, Korea

Correctly powered with 𝛼=5% and β=85% to detect 20% improvement vs placebo– Targeted sample size of 207

patients

– Interim analysis planned

Planning– Start: Q1 2017

– End: Q4 2018

Page 15: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

First-in-class small molecule administered orally

– SENS-401 is one enantiomer of SENS-218 (azasetron) a racemate drug only marketed in Asia in

chemotherapy-induced nausea and vomiting (CINV)

– Patent estate

“Use of 5HT3 antagonists in lesional vestibular disorders” patent granted

Additional compound-related patents filed

Attractive drug profile

– Anti-lesional MOA protecting against nerve degeneration and synaptic uncoupling

– Positive preclinical studies* in noise-induced cochlear lesions leading to hearing loss

– Excellent clinical tolerance demonstrated with racemate SENS-218 in Asian and Caucasian

populations

SENS-401 for Sudden Sensorineural Hearing Loss

Page 15

* Presented at the Society for Neuroscience Annual Meeting 2016, November 12-16, 2016 - San Diego, USA

Page 16: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

SENS-401Preclinical Data in Noise-induced Cochlear Lesions

Page 16

A daily oral administration of SENS-401 reduces auditory deficit and improves recovery

Randomized treatment post-noise induced trauma (2h exposure at 120 dB) in rats receiving either placebo or SENS-401 PO

Source: Dyrhfjeld-Johnsen et al 2016 SFN abstract,

*

* p< 0.05

Page 17: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Page 17Source: Dyrhfjeld-Johnsen et al 2016 SFN abstract,

Cochleograms

Placebo SENS-401

At D14 after audiometry, the cochleae were removed, and inner and outer hair cells were labelled and counted to obtain a cochleogram

A daily oral administration of SENS-401 reduced hair cell loss

SENS-401Preclinical Data in Noise-induced Cochlear Lesions

*

Cell count

Significant hair cell loss Limited hair cell loss

Histology of hair cell layers

* p< 0.001

Page 18: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Orphan Drug Designation (ODD) granted in Europe for SENS-401 in Sudden Sensorineural Hearing Loss

– Considered by ENT doctors as a case of emergency

– Rapid loss of hearing > 30 dB usually in one ear occurring either at once or over few days

– Secondary to the destruction of inner ear cells and their connections

– Often classified as idiopathic and affects typically adults in their 40s and 50s (prevalence of 4 in 10,000 people in EU i.e.

approx. 205,000 patients)

– No medicine approved

– About half of the patients will not recover their hearing or at least only partially

Active talks with EU & US Regulatory authorities

– Define path forward to Phase 2 program in patients suffering from sudden sensorineural hearing loss

SENS-401 Next Steps in Sudden Sensorineural Hearing Loss

Page 18

Page 19: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Founded in 2009 as an academic spin-off from INSERM, France

IPO in 2015 (Alternext Paris: ALSEN)

– Current share price: €4.95 (as of January 3, 2017)

– Market cap: €34m (as of January 3, 2017)

Financials

– Cash position: €9m (as of June 30, 2016) + €3m (equity line in August 2016)

– Equity line with potential additional funding of €19m

– H1 2016 cash used: €5m

Headquartered in Montpellier, France

– 17 Full time employees (o/w 13 in R&D)

Contact

– Laurent Nguyen, CEO / Paul Bikard, CFO

– E-mail: [email protected]

– Website: www.sensorion-pharma.com

Corporate overview

Page 19

Shareholding structure as of December 31, 2016

34,4%

43,1%

14,4%

4,8% 3,3% Innobio (Bpifrance)

Free float

Inserm Transfert Initiative

Founders and managers

Business Angels

Page 20: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Experienced Leadership Team in Life Sciences to Move Forward

Laurent NguyenChief Executive Officer MD, MPH

20+ years at Roche, Pierre Fabre, Merck KGaA, Hoechst-Roussel

– Solid Marketing & Sales and BD&L (Buy- & Sell-side) experience

Pierre Attali Chief Medical OfficerMD, MSc, Board certified in HGE

30+ years at Synthelabo, Sanofi, BioAlliance Pharma/Onxeo

– 10+ NCE/new formulations registered in EU/US

PhD in Neuroscience and post-doctoral research (Irvine-CA,

Harvard Medical School-Boston, USA)

– 15+ years research in CNS and inner-ear

Jonas Dyhrfjeld-JohnsenHead of PharmacologyPhD

20+ years as auditor (Coopers & Lybrand-PWC, Andersen-E&Y)

and CFO (Transgène, Prestwick Chemical)

– Solid Administration & Finance experience of SMEs

Paul BikardAdministration & Finance Director MSc Lyon Business school

Page 20

Page 21: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Significant Milestones & Investment Catalysts

* Presentation at international scientific conferences ** Subject to R&D progress and/or refinancingPage 21

Corporate & Clinical Platform

SENS-218: Phase 1 completed

SENS-401: selection of one enantiomer of SENS-218 (SFN*)

– Orphan Drug Designation granted in EU

SENS-111:

– preclinical comparative data (IEB*)

– FDA IND clearance

– phase 2 initiation in vertigo

Equity line (€3m exercised, left €19m)

2016

€8m raised during the IPO

SENS-218: Preclinical POC in hearing loss (SFN*, ARO*)

€8m private placement

SENS-111: Phase 1b study completed (AAO-HNS*, EACPT*)

Equity line (€3m exercised, left €22m)

Q2-Q42015

SENS-111: phase 2 patient recruitment in vertigo

SENS-401: clinical development in SSNHL hearing loss

SENS-300: selection of a drug candidate in drug-induced

ototoxicity

2017** Next generation programs

Pipeline expansion

Academic & Pharma collaborations

Page 22: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

“Pure player” in therapeutics for inner ear disorders (vertigo, hearing loss, and tinnitus)

– $10B untapped market opportunity*

Key Assets

– First-in-class small molecules administered orally

– De-risked pipeline entering phase 2 stage

Selection of drug candidates with clinical data

» SENS-111 for treatment of acute vertigo

» SENS-401 for treatment of sudden sensorineural hearing loss

7 patents families (composition of matter & method of use)

– Technology platform to support pipeline expansion and pharma collaboration

Execution capabilities to reach near term milestone events

– Publicly-traded company with strong balance sheet and experienced leadership team

Invest and/or partner with us to change the gamePage 22

Why invest in Sensorion?

* Source: Alcimed, Sensorion

Page 23: Corporate Presentation January 2017 - sensorion-pharma.com · Latest development in the histaminergic pathway applied to vertigo treatment – First-in-class Histamine H4 Receptor

Thank You

Caring for inner ear disability