Company Presentation. February 2019

Transcription

1 1 Company Presentation February 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 2020, 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 Unique platform fuels early stage pipeline and generates cash for the company - SAN711 in preclinical development for Neuropathic pain & Itching - CAD-1883 in Phase 2 for tremor and Phase 1 for ataxia Cadent partnership - Boehringer Ingelheim program in preclinical development for Schizophrenia 3

4 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 4

5 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/3 start 2019/20 Ph2a start Q1 19 SAN711 (GABA α3 PAM) Neuropathic pain Itching Ph1 start IK Program Inflammation, IBD Candidate selection 5

6 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 6

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

10 Tesomet/tesofensine development strategy: Geographies EU & US: PWS and HO Rest of the world: Metabolic disease 10

11 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 11

12 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) 12

13 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 13

14 Hyperphagia score Tesomet: Phase 2a Study in PWS adults Results PWS hyperphagia score (data show mean and SD) 20 PWS weight loss 15 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 14

15 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) 15

16 Tesomet/tesofensine development strategy: Geographies EU & US: PWS and HO Rest of the world: Metabolic disease 16

17 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 17

18 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 18

19 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 19

20 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 20

21 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 21 *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.

22 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) 22 9

23 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

24 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 24

25 SAN711 for neuropathic pain and Itching with orphan potential Phase 1 ready in Q Class GABA Modulator (selective α3, first in class, small molecule) Neuropathic pain Status Phase 1 ready in Q Indication Prevalence Market Medical Need MoA Neuropathic pain: +10 billion patients Itching: e.g. chronic kidney disease associated pruritus 4.5 million patients in the US Orphan itch indications: prurigo nodularis; brachioradial pruritus Neuropathic pain: +10 million globally ex back-pain Neuropathic pain: USD 6 billion Standard pain killers not working Narcotic analgesics only used in cancer due to tolerance development and abuse liability Antiepileptic and antidepressants have some effect Experimental drugs Efficacy and reduction of adverse effects 50 % achieves no pain relief and the rest obtains partial response only Only 25 % obtains a pain reduction of 50 % or more (score) Restores the endogenous pain control system in the spinal cord Itching Prurigo Nodularis 25 Patent Until 2038

26 SAN711 mode-of-action Regulates spinal cord neurons pain and itch signalling to the brain Normal Injury spinal disinhibition SAN711 increases the efficacy of dysfunctional inhibitory GABAergic neurons in spinal cord, which control relay of pain signals to the brain 26 26

27 SAN711 effective in neuropathic pain model Efficacy maintained after prolonged treatment SAN711 alleviate nerve-injury induced neuropathic pain in rats SAN711 does not lead to sedation in rats 100 Total dist. (30 min.) Veh 3 mg/kg 10 mg/kg 30 mg/kg SAN711 reduces sciatic nerve lesion induced mechanical hypersensitivity in rats with an effect similar to morphine The analgesic effect of SAN711 is maintained after prolonged treatment for 7 days while the analgesic effect of morphine is completely lost due to development of tolerance SAN711 does not lead to sedative effects in rats exploring a novel environment 27

28 IK Program for inflammation and fibrosis in IBD with orphan potential Candidate selection H. xerocytos Class KCa3.1 inhibitor (first in class, small molecule) Status: Preclinical to be initiated (Phase 1 in 2020) Indications Colitis: 1 million patients in 7 MM Crohn s disease: 1.5 million patients in 7 MM Heriditary xerocytosis (orphan): 40,000 patients in USA Crohns disease Market Colitis/Chron s: USD 5 billion (2018) Sulfasalazine, ASA, mabs Medical need: Current treatment insufficient to control flare ups, disease progression, and prevention of fibrosis +30 % of colitis patients and 80 % of Crohns develop chronic changes needing surgical intervention to resolve potentially life-threatening intestinal obstructions Colitis MoA: Downregulates immune response by inhibiting calcium entry to immune cells IP:

29 IK program mode-of-action Downregulates immune response by inhibiting calcium entry to immune cells K Ca 3.1 is expressed in all major immunological and reactive cell types involved in IBD K Ca 3.1 strengthens calcium-signalling via a positive control loop SAN903 inhibits K Ca 3.1, dampens pathological responses, and acts on acute/chronic symptoms Basic mechanism Cell types Cellular effect Therapeutic effect Orai, Trp, etc. K Ca 3.1 T-cells, macrophages Cytokine release Ca 2+ channel Effector cell K + channel T-cells, B-cells, macrophages Proliferation Anti-inflammatory T-cells, B-cells, macrophages Migration Fibroblasts, myofibroblasts Collagen release Anti-fibrotic Epithelial cells Salt and H 2 0 transport Anti-diarrhetic 29

30 SAN903 effective in IBD models Healthy large intestines Inflamed large intestines 30 SAN903-treated large intestines

31 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

32 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 Data from open label extension studies Initiate Phase 2a in hypothalamic obesity Initiate Phase 2b/3 PWS study 2 Additional value drivers from Medix tesofensine collaboration Tesofensine NDA filing in Mexico Approval and launch in Mexico Tesofensine NDA filing in Argentina 3 Research platform and early stage pipeline SAN711: Initiation of Phase 1 for chronic pain and itching IK program: Candidate selection Potential new collaborations 32

33 Saniona AB Baltorpvej 154 DK-2750 Ballerup Denmark Tel: Web: saniona.com 33