Company Presentation. January 2019

Transcription

1 1 Company Presentation January 2019

2 FORWARD LOOKING STATMENTS This presentation contains forward-looking statements that provide Saniona s expectations or forecasts of future events such as new product developments, regulatory approvals and financial performance. Such forward looking statements are subject to risks, uncertainties and may be impacted by inaccurate assumptions. This may cause actual results to differ materially from expectations and it may cause any or all of Saniona s forward-looking statements here or in other publications to be wrong. Factors that may affect future results include currency exchange rate fluctuations, delay or failure of development projects, loss or expiry of patents, production problems, breaches or terminations of contracts, government-mandated or market driven price decreases, introduction of competing products, exposure to product liability claims and other lawsuits, changes in reimbursement rules, changes of laws regulations or interpretation thereof, and unexpected cost increases. Saniona undertakes no obligation to update forward looking statements. 2

3 Investment Highlights 1 Go-2-Market opportunity with Tesomet in orphan indications - Tesomet may be Phase 3-ready in 2019, with potential market approval in Phase 2a Prader Willi Syndrome (PWS) results - Phase 2a for Hypothalamic Obesity initiation 2 Additional value driver from late stage partnership with Medix - Tesofensine in Phase 3 for obesity developed by Medix in Mexico - Medix owns rights for Mexico and Argentina - Saniona eligible for low double-digit royalties - Validating for Tesomet - Significant interest from RoW markets - Tesofensine is an off-patent compound that has been clinically tested in multiple indications 3 3 Unique platform fuels early stage pipeline and generates cash for the company - SAN711 in preclinical development for Neuropathic pain & Itching - CAD-1883 in Phase 1 for ataxia and tremor Cadent partnership - BI program in preclinical development for Schizophrenia Boehringer Ingelheim partnership - Several partnerships and three spin-outs

4 Proprietary Pipeline Near term news flow and value generation Product Indication Preclinical Phase 1 Phase 2 Milestone Tesomet tesofensine + metoprolol (monoamine reuptake inhibitor + beta blocker) Prader Willi Syndrome Hypothalamic obesity Ph2a data Q1 19 Ph2b start 2019 Ph2a start Q1 19 SAN711 (GABA α3 PAM) Neuropathic pain Itching Ph1 start H1 19 IK Program Inflammation, IBD 4

5 Partnered pipeline Near term news flow and non-dilutive cash Product Indication Preclinical Phase 1 Phase 2 Phase 3 Tesofensine Obesity CAD-1883 Essential tremor Ataxia Spinout Minority stake Royalties Not disclosed Schizophrenia Upfront: 5M Milestones: 85M Royalties NS2359 Cocaine Addiction NS2359 off patent; financed by US grants 5

6 Tesomet: Potential Orphan Drug with Blockbuster Potential MoA creates multiple opportunities within metabolic diseases and eating disorders Potential best-in-class profile for weight related metabolic diseases via unmatched weight loss and benign side effect profile 6

7 Tesomet - lead indication: Prader-Willi syndrome Genetic disease caused by mutations/deletion of genes on chromosome 15 Chronic feeling of extreme hunger (hyperphagia) no matter how much the patient eats Other symptoms and characteristics Mental retardation and behavioural problems Low metabolic rate (50% of normal) Sensitive to some medicines (½ dose prescribed) Medical need Acute life-threating hyperphagia (choking, bowel rupture) Life-threatening obesity } Short life expectation (average in 30s) No effective treatment available today Economic and social costs Quality of life for patients and families Family stress and loss of income Care and medical costs (USD K per year) 7

8 COMPOSITION Tesomet: tesofensine + metoprolol Tesofensine, in preclinical models and clinical trials, has shown efficacy and safety Reduction in food intake Weight loss efficacy Effects on glycemic parameters relevant for type 2 diabetes Excellent safety and tolerability TESOFENSINE Effective weight loss drug METOPROLOL Beta blocker to control slight increase in heart rate Tesomet = tesofensine + beta blocker (metroprolol) Neutralizes slight heart rate increase observed with tesofensine Allows for strong intellectual property protection through

9 Tesomet packs all benefits of tesofensine & controls for heart rate 9

10 Tesomet: Phase 2a Study in PWS adults Study set up Phase 2a study initiated in April 2017 Exploratory randomized, double-blind, placebocontrolled 12 weeks study in 9 patients - Tesomet 6 - Placebo 3 Positive effect on key efficacy endpoints - Reduced craving for food - Weight loss PK and Safety - No SAE - AE mainly CNS related - Half-life longer than expected in PWS patients 10

11 Hyperphagia score Tesomet: Phase 2a Study in PWS adults Results PWS hyperphagia score and weight loss (data show mean and SD) Week 8 Week 13 Tesomet 5.00 % (n=5) 6.75 % (n=2) 10 Placebo 0.46 % (n=2) 0.75 % (n=2) 5 Second part of PWS study in adolescents ongoing days of treatment placebo treatment 11

12 Tesomet: Go-2-Market opportunity in orphan indications Potential for market entry within 4 years Total investment of $30-40M - >$4B opportunity Prader Willi Syndrome Positive Phase 2a in PWS adults Phase 2a in adolescent patients ongoing Life-threatening hyperphagia and obesity Prevalence: 1/ Estimated market size: ~3B USD Hypothalamic Obesity Phase 2 study preparations Life-threatening hyperphagia and obesity Prevalence: 1/( ,000) Estimated market size: >1B USD Prader Willi Syndrome Hypothalamic obesity Phase 2a Dose finding Phase 3 Phase 2a Phase 3 FDA filing FDA filing 12

13 PWS opportunity has blockbuster potential Accessible market value equals 3 Billion USD (Analyst estimates) Premium pricing potential Orphan drug status will ensure premium pricing Majority of drugs with less that 10,000 patients in the US tend to be priced above 200K USD per year Large commercial opportunity No drugs approved for treating hyperphagia Low investment Clear endpoint with short studies (Phase 3: 100 patients / 6 months) Straightforward commercialization (most patients are managed by specialists in central centers) 13

14 Tesofensine: Successful Phase 3 Study in Mexico by Partner Medix Medix to file for registration in Mexico and Argentina Phase 3 Study Design Randomized, double-blind, placebo controlled trial in Mexico 372 patients enrolled: - N=124: placebo - N=124: 0.25mg tesofensine - N=124: 0.50mg tesofensine - 24 weeks treatment period and 12 week follow up - All patients prescribed an energy restricted diet of 1,500-2,000 kcal and physical activity of minutes All endpoints met: Primary endpoint: percent change in bodyweight compared to baseline at 24 weeks Secondary endpoints include: - Proportions of patients achieving a weight loss of >5 and 10 percent, respectively - Metabolic including glycemic endpoints - Quality of life 14

15 Medix partnership With ~50% market share, Medix is market leader in the $250M Rx Obesity Market in Mexico Regional deal structure Medix holds the rights to tesofensine & Tesomet in Mexico & Argentina Medix finances clinical studies and commercialization Saniona receives double digit royalties Saniona retains rights to rest of the world including exclusive rights to Medix clinical data Medix could be on the market in Mexico in 2020 and in Argentina one year later Mexico Phase 3 Cofepris review Commercialization Argentina Argentina NDA Commercialization 15

16 Tesofensine: Phase 2 Study Study methodology & results Methodology Randomized, double-blind, placebo controlled trial in five Danish obesity management centers Enrolled 203 patients Energy restricted diet with a daily energy deficit of 300kcal in addition to physical activity of minutes Results At 24 weeks patients had lost 11.2 % in bodyweight at 0.5 mg per day compared to 2.0% for placebo Tesofensine well tolerated Adverse effects similar to placebo with an increase in heart rate compared to baseline Primary endpoint: percentage change in bodyweight compared to baseline at 24 weeks 16

17 Tesofensine strong efficacy in bodyweight and appetite Phase 2b study Reduction in bodyweight compared to baseline Reduction in appetite/craving for food Change from baseline (total score) % reduction % reduction % reduction 12.6% reduction Placebo 0.25 mg 0.5 mg 1 mg 17

18 Weight loss treated vs placbo Tesofensine could double weight loss compared to competitors Results at 48 weeks suggest tesofensine could be used as an alternative to surgery Apples to oranges comparison of reduction in bodyweight versus competing drugs* 48 weeks open label 14% 10.0% 9.0% 8.0% 9.2% 7.0% 6.0% 5.0% 5.2% 6.0% 6.6% 4.0% 3.0% 2.4% 3.1% 2.0% 1.0% 0.0% Xenical, 3x120mg, 4 years Belvig, 2x10mg, 1 year Contrave, 2x360/32mg, 56 weeks Victoza, 3mg, 56 weeks Qsymnia, 7.5/46mg, 56 weeks Tesofensine, 0,50mg, 24 weeks Tesofensine 0,50 mg, 48 weeks open label extension 18 *Results from competing drugs taken from their respective studies and have been adjusted for their respective placebo results. Results from competing trials are not directly comparable.

19 Weight loss in kg vs baseline Additional weight loss in open label extension up to 58 weeks 0 TIPO-1 Placebo TIPO-4 (open label extension) 0.5 mg tesofensine mg tesofensine -15 Drug holiday 0.5 mg tesofensine Time (weeks) 19 9

20 Tesofensine and Tesomet are supported by a large safety database Tesofensine, the key active ingredient in Tesomet, is well studied and has been safe and well tolerated More than 1700 subjects treated to date. 1 Phase 3 study, N= 372 (248 tesofensine) 8 Phase 2 studies, N = 1310 (1000 tesofensine) 2 Phase 2 studies* (one ongoing), N=78 18 Phase 1 studies, N = 391 (325 tesofensine) 3 Phase 1 studies* (one ongoing), N= *Indications include Type 2 diabetes and Prader Willi syndrome

21 Tesomet/tesofensine development strategy: Geographies EU & US: PWS and HO Rest of the world: Metabolic disease 21

22 Ion channel platform is rapidly fuelling early stage pipeline Leverage research platform through partnerships and develop at least one candidate to Phase 2 internally Product/Target Indication Preclinical Research Preclinical Development Phase 1 Phase 2 CAD-1883 Essential tremor Ataxia SAN711 Neuropathic pain Itching Not disclosed Schizophrenia IK Program Inflammation, IBD Kv7 program Pain, epilepsy, UI Nicotine α6 Program Parkinson s Disease Spinout Minority stake Royalties Upfront: 5M Milestones: 85M Royalties 22

23 Financial statements 9 month 2018, 9 month 2017, 2017 and accumulated since the company became operations in 2012 Income statement MSEK 9 months 18 9 months Q Net sales Operating expenses Operating profit/loss Financial items Tax on net profit Profit/loss Other comprehensive income Total comprehensive income Balance sheet MSEK Sep 18 Sep 17 Dec-17 Non-current assets Current receivables Cash and cash equivalent Total assets Equity Total liabilities Total equity and liabilities Cash flows MSEK 9 months 18 9 months Q Operating activities Investing activities Financing activities Cash flow

24 Near term value inflection points 1 Go-2-Market opportunity with Tesomet in orphan indications Completion of second part of Phase 2a in adolescents with PWS Initiate Phase 2a in hypothalamic obesity Initiate Phase 2b PWS dose range finding study 2 Additional value drivers from Medix tesofensine collaboration Tesofensine: Phase 3 top-line data in obesity Tesofensine NDA filing in Mexico Tesofensine NDA filing in Argentina 3 Research platform and early stage pipeline SAN711: Initiation of Phase 1 for chronic pain and itching Potential new collaborations 24

25 Management Team Jørgen Drejer, Ph.D. CEO, Co-Founder and Board Member Previously, Executive Vice President, Research Director and co-founder of NeuroSearch A/S. Board member of 2Curex AB and Ellegaard Göttingen Minipigs ApS Formerly board member of NSGene A/S, Origio A/S, Delta and Zgene A/S. Thomas Feldthus, M.Sc., MBA CFO and Co-Founder Previously, CFO and co-founder of Symphogen A/S, Investment manager of Novo A/S, Corporate Development manager of Novo Nordisk A/S, board member of Saniona AB and Leukotech A/S. Palle Christophersen, Ph.D. CSO and Co-Founder Previously, Vice President and member of the NeuroSearch A/S VP management group. Director of in Vitro Pharmacology, NeuroSearch A/S 25

26 Board of Directors J. Donald debethizy Chairman of the Board Don currently serves as Board member of Albumedix A/S, Argenx N.V., Newron Pharmaceuticals SpA, Noxxon Pharma NV and Proterris, Inc. Previously, he served as President and CEO of Santaris Pharma A/S, Executive Chairman of Contera Pharma ApS, CEO of Targacept, Inc. He previously served as Board member of Asceneuron SA, Biosource Inc., Enbiotix Inc., LigoCyte Pharmaceuticals Inc., Rigontec GmbH, Serendex Pharmaceuticals A/S and Targacept Inc. Claus Braestrup Board Member, Former Chairman of the Board Claus currently serves as a Board member of Bavarian Nordic A/S and Evotec AG. Previously, he was the CEO of H. Lundbeck A/S, CEO of Nordic Biotech General Partner II ApS, Chairman of the Board of Directors of Probiodrug AG, and member of the Board of Santaris Pharma A/S, Ataxion Inc., Evolva Holding SA and Gyros AB. Anna Ljung Board Member Anna currently serves as as the CFO of Moberg Pharma AB where she prepared the company for its 2011 initial public offering onto the OMX Small Cap list. She previously served as CFO of Athera Biotechnologies AB and Lipopeptide AB and has worked as an independent consultant in the field of technology licensing Carl Johan Sundberg Board Member Carl currently serves as a Professor at the Department of Physiology & Pharmacology at Karolinska Institutet, Sweden. He also serves as a Board member of Cobra Biologics Holding AB. Previously, he served as a Board member of Alfta Rehab Holding AB, Karolinska Development AB, KI Management AB, and KI Management Partners AB. Jørgen Drejer, Ph.D. CEO, Co-Founder and Board Member 26 Previously, Executive Vice President, Research Director and co-founder of NeuroSearch A/S. Board member of 2Curex AB and Ellegaard Göttingen Minipigs ApS Formerly board member of NSGene A/S, Origio A/S, Delta and Zgene A/S.

27 Saniona AB Baltorpvej 154 DK-2750 Ballerup Denmark Tel: Web: saniona.com 27