Santhera Pharmaceuticals Company Presentation September 2018
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 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) Northern Europe Cluster (NEC) Central Europe Cluster (CEC) Western Europe Cluster (WEC) Southern Europe Cluster (SEC) Headquarters 3
Key Financials First Half-Year 2018 (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 4
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 Focus on rare diseases Neuroophthalmological Neuromuscular Pulmonary THEIR FUTURE OUR FOCUS 5
Our product pipeline 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) Duchenne Muscular Dystrophy (GC users) Congenital Muscular Dystrophy Idebenone Idebenone Omigapil Pulmonary Diseases Cystic Fibrosis Alpha-1 Antitrypsin Deficiency Non-Cystic Fibrosis Bronchiectasis Primary Ciliary Dyskinesia POL6014 POL6014 POL6014 POL6014 To be explored *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 LHON GC: glucocorticoid 6
Raxone sales in LHON and guidance for 2018 Raxone Net Product Sales (in CHF millions) 35 30 25 20 15 10 5 19.0 22.9 FY 2018: 30-32 est. 1H 2018: 16.0 +48% y-o-y 0 2015 2016 2017 2018 Raxone is sold in more than 20 European countries Regional roll-out progressing: first Asian submission in South Korea 7
Strategies for further growth Grow sales of Raxone for the treatment of LHON and expand commercial reach Further develop and grow our rare disease business in neuroophthalmology, neuromuscular and pulmonary towards leadership Progress pipeline assets and advance towards regulatory approval Active in-licensing / partnering strategy for high-quality, late-stage rare disease assets with a short time to market 8
Raxone (idebenone) in Leber s Hereditary Optic Neuropathy (LHON) Neuro-ophthalmological Diseases
Raxone is the first and only approved treatment for LHON 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 Normal vision Days, weeks or months LHON vision Raxone is the first and only available treatment in LHON and can lead to stabilization or recovery of vision 10
Raxone improves vision in patients with LHON Clinical Prevention data of have further shown vision 3-5 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: * 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 CRS 1.0 logmar (20/200) CRR 11
Outlook neuro-ophthalmology business Projected to reach profitability in 2018 for neuro-ophthalmology business (including costs for post approval studies) Neuroophthalmology 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 12
Idebenone in Duchenne Muscular Dystrophy (DMD) Neuromuscular Diseases
Urgent medical need for new therapies in DMD Increasing respiratory muscle weakness in DMD leads to: As respiratory function declines, assisted ventilation is required to alleviate symptoms 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 14
Available clinical data with idebenone in DMD Patients with DMD not using glucocorticoids The Phase III DELOS study 40% of patients 10 years and older are not using glucocorticoids and were eligible for DELOS: Patients not using GCs Idebenone 900mg daily Placebo 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 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 15
www.siderosdmd.com A Phase III double-blind study with idebenone in patients with DMD taking glucocorticoids 266 patients using GCs 1:1 Idebenone 900mg daily Placebo Open-label extension offered Population Study design Treatments Treatment duration Primary endpoint Status Patients 10 y in respiratory function decline Interventional, placebo controlled, Phase 3, RCT Parallel group assignment to idebenone 300mg orally 3 time daily, or placebo 18 months Change from baseline in forced vital capacity percent predicted (FVC %p) at 18 months Recruiting > 60 sites across the EU, U.S. and Israel 13 EU countries 1 non-eu country 18 states across US Status: June 2018 FVC: forced vital capacity; GC: glucocorticoid; RCT: randomized controlled trial 16
U.S. Expanded Access Program: BreatheDMD A U.S. Expanded Access Program (EAP) in patients with DMD Up to 250 DMD patients Population Objective Idebenone 300mg orally 3 times daily DMD patients 10 years in respiratory decline Provide access to treatment with idebenone for patients with DMD in the US Up to 35 sites across the US WA MT ND MN OR ID WI SD WY IA NE NV UT CA CO KS MO OK AR AZ NM TX LA IL MS MI IN TN AL KY OH GA WV PA SC FL VA NY NC MD VT NH NJ DE ME MA Key endpoints Safety, tolerability, effectiveness and QoL data Supported by: Status Enrolling More information www.breathedmd.com 17
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 18
Outlook: neuromuscular diseases pipeline Neuromuscular 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 19
POL6014 in Cystic Fibrosis Pulmonary 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 Obstruction Infection Genetic Defect Respiratory Failure Inflammation 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 21
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 POL6014 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) eflow Nebulizer 22
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) Pulmonary POL6014 in other pulmonary diseases Explore opportunities in other pulmonary diseases with clear rationale for elastase inhibition 23
Outlook and Strategy for Growth
Strategies for further growth Grow sales of Raxone for the treatment of LHON and expand commercial reach Progress pipeline assets and advance towards regulatory approval Active in-licensing / partnering strategy for high-quality, late-stage rare disease assets with a short time to market 25
THEIR FUTURE OUR FOCUS Thank you for your attention and interest in Santhera