Giovanni StropoliSanthera HQ, Liestal, Switzerland

Advancing Mitochondrial Medicine

Disclaimer

This presentation is not and under no circumstances to be construed as a solicitation, offer, or recommendation, to buy or sell securities

issued 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 AG

disclaims, without limitation, all liability for any loss or damage of any kind, including any direct, indirect or consequential damages, which

might 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

its business. Certain of these forward-looking statements can be identified by the use of forward-looking terminology or by discussions of

strategy, plans or intentions. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause

the actual results, financial condition, performance or achievements of Santhera Pharmaceuticals Holding AG to be materially different from

any expected results, performance or achievements expressed or implied by such forward-looking statements. There can be no guarantee

that any of the research and/or development projects described will succeed or that any new products or indications will be brought to

market. Similarly, there can be no guarantee that Santhera Pharmaceuticals Holding AG or any future product or indication will achieve any

particular level of revenue. In particular, management’s expectations could be affected by, among other things, uncertainties involved in the

development of new pharmaceutical products, including unexpected preclinical and clinical trial results; unexpected regulatory actions or

delays or government regulation generally; the Company’s ability to obtain or maintain patent or other proprietary intellectual property

protection; competition in general; government, industry, and general public pricing and other political pressures. Santhera Pharmaceuticals

Holding AG is providing the information in this new release as of the date of the publication, and does not undertake any obligation to update

any forward-looking statements contained herein as a result of new information, future events or otherwise.

2

Santhera Pharmaceuticals

• Founded as a Biotech Merger in 2004 in Liestal Basel Biozentrum Spin-off Myocontract and Heidelberg based Graffinity

• Currently about 25 people, planning idebenone introduction in EU

• Clinical Development in Orphan Diseases(mitochondrial/neuromuscular)

• Direct Distribution of Niche Pharmaceutical Products

3

3

Pipeline with idebenone (Raxone®) in three indications with high unmet medical need

4

Primary progressive MS (ppMS):

Phase 2 study in collaboration with NIH

Duchenne Muscular Dystrophy (DMD):

Positive Phase 3 study outcome,

NDA/MAA filing in preparation

Leber’s Hereditary Optic Neuropathy (LHON):

MAA under review in EU

Raxone®: trademark in EUCatena®: alternative trademark

Inherited form of blindness:

Prevalence ~ 2 in 100’000

~ 2’500 patients diagnosed in past 5 years in EU

Genetic disease with clear diagnosis and family pattern

Predominantly young males in all ethnic groups affected

Rapid loss of central vision by functional loss and

degeneration of retinal nerve cells

No treatment available

LHON – an inherited form of blindness

5

1 2 3 4 50

RECOVERY

Off - chart

STABILISATION

ONSETYEARS

VA

NATURAL HISTORY

The therapeutic objective:

– stabilize the disease

– promote clinically relevant recovery

Potential window of opportunity for treatment up to ~ 5 years

Therapeutic objectives

6

50% of LHON patients treated with Raxone®

experience clinically relevant recovery of vision

7

Data from Expanded Access Program

The animation simulates

the average treatment

response in EAP

Raxone® efficacy typically seen within12 months from treatment start

8

Data from Expanded Access Program

Majority of patients who show improved vision do so within first 12 months of treatment

Raxone® in Leber’s Hereditary Optic Neuropathy (LHON): pathway to its availability

9

Leber’s Hereditary Optic Neuropathy (LHON):

Marketing Authorization Application (MAA)

under review in EU

Temporary Authorization in France,

CHMP Decision for Europe awaited

Authorization for Temporary Use (ATUc) granted in 2014

Use of Raxone® in hospital-treated patients with LHON

Supply of Raxone® fully reimbursed by the government program

Marketing Authorization Application (MAA) for Europe filed in May 2014 (validated in June 2014)

Decision by EMA’s CHMP expected in 1H 2015

Market exclusivity due to Orphan Drug protection

10

Raxone® slows the loss of respiratory function in DMD patients not using steroids

First successful Phase 3 study in DMD

Efficacy demonstrated in primary endpoint (Peak Expiratory Flow)

Supportive evidence of efficacy from

other respiratory function endpoints

clinical observations directlyrelated to morbidity and mortality

Treatment was safe and well tolerated

11

European introduction plans

12

BRITAINIRELAND

PORTUGAL SPAIN

FRANCE

GERMANYGERMANY

AUSTRIAAUSTRIA

HUNGARY

GREECE

ITALY

SWEDEN

FINLAND

SLOVENIA

SLOVAKIA

ROMANIA

BULGARIA

POLAND

CROATIA

CZ

NORWAY

CH

DENMARK

NETH.

BELG.

LUX.

ESTONIA

LATVIA

LITHUANIA

Santhera presence build-up in 4 regional clusters

Potential national /regional distribution agreements for other EU countries

Currently staffing for Europe (and RoW) product introduction

Advancing mitochondrial medicine towards a first treatment in:

Thank you for your attention and support

LHON

DMD

ppMS

13