Company Presentation - Seeking Alpha

September 2018

Santhera Pharmaceuticals Company Presentation

Company Presentation | September 2018

Disclaimer

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This presentation is not and under no circumstances to be construed as a solicitation, offer, or recommendation, to buy or sell securitiesissued by Santhera Pharmaceuticals Holding AG. Santhera Pharmaceuticals Holding AG makes no representation (either express or implied)that the information and opinions expressed in this presentation are accurate, complete or up to date. Santhera Pharmaceuticals Holding AGdisclaims, without limitation, all liability for any loss or damage of any kind, including any direct, indirect or consequential damages, whichmight be incurred in connection with the information contained in this presentation.

This presentation expressly or implicitly contains certain forward-looking statements concerning Santhera Pharmaceuticals Holding AG and itsbusiness. Certain of these forward-looking statements can be identified by the use of forward-looking terminology or by discussions ofstrategy, plans or intentions. Such statements involve certain known and unknown risks, uncertainties and other factors, which could causethe actual results, financial condition, performance or achievements of Santhera Pharmaceuticals Holding AG to be materially different fromany expected results, performance or achievements expressed or implied by such forward-looking statements. There can be no guaranteethat any of the research and/or development projects described will succeed or that any new products or indications will be brought tomarket. Similarly, there can be no guarantee that Santhera Pharmaceuticals Holding AG or any future product or indication will achieve anyparticular level of revenue. In particular, management’s expectations could be affected by, among other things, uncertainties involved in thedevelopment of new pharmaceutical products, including unexpected preclinical and clinical trial results; unexpected regulatory actions ordelays or government regulation generally; the Company’s ability to obtain or maintain patent or other proprietary intellectual propertyprotection; competition in general; government, industry, and general public pricing and other political pressures. Santhera PharmaceuticalsHolding AG is providing the information in this new release as of the date of the publication, and does not undertake any obligation to updateany forward-looking statements contained herein as a result of new information, future events or otherwise.


Santhera at a Glance

• International pharma company headquartered in Switzerland (SIX: SANN) with regional

presences in Europe and North America

• Pipeline in rare disease therapeutic areas with three clinical drug candidates in five indications

• Steadily growing revenue stream

1H 2018: CHF 16 million (+48% y-o-y)

• Headcount 116 (August 2018)

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Northern Europe Cluster (NEC)

Western Europe Cluster (WEC)

Central Europe Cluster (CEC)

Southern Europe Cluster (SEC)

Headquarters


Key Financials First Half-Year 2018

4

(reviewed, IFRS, consolidated, in CHF million)

1H 2018

Net sales (+48% y-o-y) 16.0

Operating expenses –39.9

Operating result –26.3

Net result –27.4

Operating cash flow –22.2

Freely available liquid funds (30.6.2018)(cash & cash equivalents and short-term financial assets)

34.8

Restricted cash, in addition to freely available liquid funds 6.0

Net change in cash & cash equivalents –23.1


Our therapeutic focus

We are focusing on the development of treatments for neuro-ophthalmological,

neuromuscular and pulmonary diseases that have a high unmet medical need

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THEIR FUTURE – OUR FOCUSTHEIR FUTURE – OUR FOCUS

Focus on rare diseasesFocus on rare diseases

Neuro-ophthalmological

Neuro-ophthalmological

PulmonaryPulmonaryNeuromuscularNeuromuscular


Our product pipeline

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*Raxone® (Santhera Pharmaceuticals) is the tradename for idebenone. Raxone (150 mg idebenone) is currently approved for the treatment of visual impairment in adolescent and adult patients with LHONGC: glucocorticoid

Santhera Pipeline Drug Preclin. Phase I Phase II Phase III Filing Market

Neuro-ophthalmological Diseases

Leber’s Hereditary Optic Neuropathy Idebenone* Raxone®

Neuromuscular Diseases

Duchenne Muscular Dystrophy (GC non- users) Idebenone

Duchenne Muscular Dystrophy (GC users) Idebenone

Congenital Muscular Dystrophy Omigapil

Pulmonary Diseases

Cystic Fibrosis POL6014

Alpha-1 Antitrypsin Deficiency POL6014

To be explored

Non-Cystic Fibrosis Bronchiectasis POL6014

Primary Ciliary Dyskinesia POL6014


Raxone® sales in LHON and guidance for 2018

• Raxone® is sold in more than 20 European countries

• Regional roll-out progressing: first Asian submission in South Korea

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0

5

10

15

20

25

30

35

2015 2016 2017 2018

Rax

on

e® N

et P

rod

uct

Sal

es(i

n C

HF

mill

ion

s)

1H 2018:16.0

+48% y-o-y

FY 2018:30-32 est.

22.9

19.0


Active in-licensing / partnering strategy for high-quality, late-stage rare disease assets with a short time to market

Strategies for further growth

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Further develop and grow our rare disease business in neuro-ophthalmology, neuromuscular

and pulmonary towards leadership

Progress pipeline assets and advance towards regulatory approval

Grow sales of Raxone® for the treatment of LHON and expand commercial reach

Raxone® (idebenone) in Leber’s Hereditary Optic Neuropathy (LHON)Neuro-ophthalmological Diseases


Raxone® is the first and only approved treatment for LHON

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• LHON, a rare mitochondrial disease

resulting in progressive and severe

vision loss

• Most common in males with a disease

onset between 15 – 35 years of age

• Within 1 year > 90% of patients

experience vision loss in both eyes

• Approved in EU, Norway, Iceland,

Liechtenstein and Israel

LHON visionNormal vision

Days, weeks or months

Raxone® is the first and only available treatment in LHON and can lead to stabilization or recovery of vision


Raxone® improves vision in patients with LHON

Clinical data have shown3-5:

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1 in 2 patients who received idebenone

experienced a CRS, with vision remaining

below logMAR 1.0**

1 in 2 patients who have lived with LHON

achieved a CRR after idebenone treatment

1.0 logMAR(20/200)

CRS CRR

Prevention of further vision loss by clinically relevant stabilization (CRS) and improvement of visual acuity by a clinically relevant recovery (CRR) are important and meaningful outcomes for patients with LHON

Prevention of further vision loss by clinically relevant stabilization (CRS) and improvement of visual acuity by a clinically relevant recovery (CRR) are important and meaningful outcomes for patients with LHON

*Clinical data has shown:


Neuro-ophthalmology

Neuro-ophthalmology

Outlook neuro-ophthalmology business

• Projected to reach profitability in 2018 for neuro-ophthalmology business (including costs for post approval studies)

• Anticipated peak sales potential for Europe: CHF ~50 million p.a.

• LEROS: An open-label, interventional Phase IV study in Europe and USA to assess the long-term efficacy and safety of Raxone® in LHON

• Protection through Orphan Drug Status in Europe until 4Q 2025

• Expansion of marketing authorizations to countries outside Europe

Raxone® for the treatment of LHON is approved in Israel

Marketing authorization application submitted in South Korea

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Idebenone in Duchenne MuscularDystrophy (DMD)Neuromuscular Diseases


Urgent medical need for new therapies in DMD

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• Increasing respiratory muscle weakness

in DMD leads to:

‒ Decreased lung volumes and flow rates

‒ Decreased ability to cough effectively and

clear airways from mucus

‒ Increased risk of airway infections

• There are no approved pharmacological

therapies for treating respiratory

decline

• ~35,000 patients combined in US and

Europe

As respiratory function declines, assisted ventilation is required to alleviate symptoms


Available clinical data with idebenone in DMD

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The Phase III DELOS study

40% of patients 10 years and older are not using glucocorticoids and were eligible for DELOS:

• Treatment duration: 52 weeks• Completed• First positive Phase III trial in DMD• Data extensively published

The Phase III DELOS study

40% of patients 10 years and older are not using glucocorticoids and were eligible for DELOS:

• Treatment duration: 52 weeks• Completed• First positive Phase III trial in DMD• Data extensively published

Comparative analysis* of DELOS outcome with new data from natural history studies showed that:

• Treatment effect with idebenone observed in DELOS trial can be linked to a delay in the initiation of assisted ventilation by three years

* Presented at the 15th International Congress on Neuromuscular Diseases (ICNMD), 2018

Comparative analysis* of DELOS outcome with new data from natural history studies showed that:

• Treatment effect with idebenone observed in DELOS trial can be linked to a delay in the initiation of assisted ventilation by three years

* Presented at the 15th International Congress on Neuromuscular Diseases (ICNMD), 2018

Patients with DMD not using glucocorticoidsPatients with DMD not using glucocorticoids

Patientsnot using GCs

Idebenone 900mg daily

Placebo

Compassionate use in DMD after completion of the DELOS study:

• Long-term data demonstrate persistence of the idebenone treatment effect on pulmonary function outcomes

* Publication planned for fall 2018

Compassionate use in DMD after completion of the DELOS study:

• Long-term data demonstrate persistence of the idebenone treatment effect on pulmonary function outcomes

* Publication planned for fall 2018


www.SIDEROSdmd.com

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FVC: forced vital capacity; GC: glucocorticoid; RCT: randomized controlled trial

Population Patients ≥ 10 y in respiratory function decline

Study design Interventional, placebo controlled, Phase 3, RCT

TreatmentsParallel group assignment to idebenone 300mg orally 3 time daily, or placebo

Treatment duration

18 months

Primary endpoint

Change from baseline in forced vital capacity percent predicted (FVC %p) at 18 months

Status Recruiting

A Phase III double-blind study with idebenone in patients with DMD taking glucocorticoidsA Phase III double-blind study with idebenone in patients with DMD taking glucocorticoids

Placebo

Idebenone 900mg daily 1:1

≥ 266 patientsusing GCs

Open-label extension

offered

> 60 sites across the EU, U.S. and Israel

13 EU countries 18 states across US1 non-EU country

Status: June 2018


U.S. Expanded Access Program: BreatheDMD

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Population DMD patients ≥ 10 years in respiratory decline

ObjectiveProvide access to treatment with idebenone for patients with DMD in the US

Key endpoints Safety, tolerability, effectiveness and QoL data

Status Enrolling

More information

www.breathedmd.com

WA

OR

CA

MT

ID

NV

AZ

UT

WY

CO

NM

TX

OK

KS

NE

SD

ND

MN

IA

MO

AR

LA

MSAL

GA

FL

SC

TN

NC

IL

WIMI

OHIN

KY

WV VA

PA

NY

ME

VTNH

NJDE

MD

MA

Up to 35 sites across the US

Supported by:

Up to 250 DMD

patients Idebenone 300mg orally 3 times daily

A U.S. Expanded Access Program (EAP) in patients with DMDA U.S. Expanded Access Program (EAP) in patients with DMD


Santhera’s disease awareness campaigns in DMD

Dedicated websites providing information on respiratory function care

• US website: www.takeabreathdmd.com

• European website: www.breatheduchenne.com

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NeuromuscularNeuromuscularOutlook: neuromuscular diseases pipeline

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Idebenone in DMD

• Compassionate use programs

‒ Roll-out Expanded Access Program in US

‒ Continuation of Early Access to Medicines Scheme (EAMS) in UK

• Prepare for EU and US regulatory filing, initially for patients not using glucocorticoids

• Continue SIDEROS study in GC users; study readout expected 2H 2020

Omigapil in CMD

• Successful completion of Phase I clinical trial with omigapil in patients with CMD

‒ Favorable pharmacokinetic and safety/tolerability profile

• Discuss new study design with clinical expert team

• Obtain input from US and EU regulators on development plan for 2 CMD subtypes

POL6014 in Cystic FibrosisPulmonary Diseases


Cystic fibrosis, a rare inherited lung disease

• CF is a progressive, genetic disease leading to thick mucus in the lung (airway obstruction)

• This results in persistent lung infections, chronic inflammation and loss of respiratory function

• The disease is diagnosed in young children, about 70,000 patients live in US & EU

• Current treatments do not specifically address the chronic, underlying inflammation

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Infection

Respiratory Failure

Inflammation

Genetic Defect

Obstruction

Respiratory Failure


Targeting elastase to treat chronic lung inflammation

• Inflammation causes excessive production of human neutrophil elastase (hNE)

Elevated hNE levels play a central role in lung tissue damage

• POL6014 is a reversible, competitive and selective inhibitor of hNE

Compound has been rationally designed for potency and selectivity

Administered via inhalation to achieve high concentrations in the lung while limiting systemic exposure and associated adverse events

• POL6014 presents an opportunity for a pipeline in a product

Santhera licensed POL6014 worldwide for development in CF and additional indications

Chronic inflammation also present in other neutrophilic lung diseases such as non-cystic fibrosis bronchiectasis (NCFB), alpha-1 antitrypsin deficiency (AATD), primary ciliary dyskinesia (PCD) and chronic obstructive pulmonary disease (COPD)

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POL6014

eFlow Nebulizer


Outlook: pulmonary pipeline

POL6014 in CF

• Single ascending dose in healthy volunteers and CF patients completed

• Start multiple ascending dose trial in CF patients (4Q 2018)

• Apply for Orphan Drug Designations for CF in EU and US (2H 2018)

• Prepare for Phase II efficacy trial (2019)

POL6014 in other pulmonary diseases

• Explore opportunities in other pulmonary diseases with clear rationale for elastase inhibition

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PulmonaryPulmonary

Outlook and Strategy for Growth


Active in-licensing / partnering strategy for high-quality, late-stage rare disease assets with a short time to market

Strategies for further growth

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Progress pipeline assets and advance towards regulatory approval

Grow sales of Raxone® for the treatment of LHON and expand commercial reach

THEIR FUTUREOUR FOCUSThank you for your attention and interest in Santhera

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