Company presentation. June 2017

Transcription

1 1 Company presentation June 2017

2 Clinical stage company focusing on drugs for diseases of the central nervous system, autoimmune diseases and metabolic diseases 3 clinical stage candidates for obesity, metabolic diseases and cocaine addiction Several partnerships with top names in the field World-class research team focused on ion channels with over 25 years of experience Selected partners 2

3 1. Introduction 2. Portfolio I. Clinical stage programs II. Pre-clinical programs 3. Financials and outlook 3

4 Pipeline Product or Target Indication Preclinical research Preclinical development Clinical Phase 1 Clinical Phase 2 Clinical Phase 3 Tesofensine monotherapy Obesity Tesomet Type 2 diabetes Tesomet Prader-Willis syndrome NS2359 Cocaine addiction GABA-A α2/α3 Neuropathic pain Boehringer Ingelheim program Schizophrenia IK Inflammation, IBD Luc Therapeutics program Ataxia Proximagen program Neurological disorders Nicotinic α6 program Parkinson's disease 4

5 Corporate milestones Recent pipeline milestones /2012 Saniona operational 06/2016 U Penn (TRC) initiates Phase 2a study for NS2359 in cocaine addiction 08/2016 Saniona signs partnership with Boehringer Ingelheim for Schizophrenia / Heavily oversubscribed IPO 10/2016 Saniona and Proximagen research collaboration 01/2017 Saniona reports positive top line results from the Tesomet Phase 2a study in type 2 diabetes / Saniona moves to Nasdaq First North Premier 04/2017 Saniona initiates Phase 2a study for Tesomet in Prader-Willi syndrome / Saniona moves to Nasdaq Stockholm Small Cap 04/2017 Saniona obtains research milestone from The Michael J. Fox Foundation for Parkinson's Research 04/2017 Medix receives approval to initiate Phase 3 study in obesity 5 5

6 1. Introduction 2. Portfolio I. Clinical stage programs II. Pre-clinical programs 3. Financials and outlook 6

7 Tesofensine treats Obesity as a CNS disorder Saniona is collaborating with Medix on tesofensine for obesity in Mexico Triple mode of action for unique weight loss Tesofensine is a triple monoamine inhibitor which targets dopamine, serotonin and noradrenaline to stimulate weight loss in obese patients DESCRIPTION Tesofensine is a triple monoamine reuptake inhibitor Food cravings DOPAMINE PLEASURE REWARD CENTER - Humans feel pleasure by eating as it stimulates dopamine - Obese patients crave food and suger due to under stimulation - Tesofensine reduces the craving for food by stimulating the reward center INDICATION (TARGET GEOGRAPHY) Obesity (Mexico) 1 - Prevalence in Mexico: 28.1% Appetite Fat burn SEROTONIN SATIETY CENTER - Humans achieve satiety by eating as it stimulates this center - Obese patients have continuous hunger due to under stimulation - Tesofensine normalizes appetite by stimulating the satiety center NORADRENALINE PATHWAYS - Humans respond to stress by mobilizing energy from fat and increasing the heart rate, both functions are controlled by noradrenaline - Obese patients accumulate fat due to reduced fat burn - Tesofensine increases fat burn, but also heart rate MARKET SIZE The prescription medicine market for obesity in Mexico is around USD 250 million 2 STAGE OF DEVELOPMENT Regulatory approval to start Phase III in 2017 PARTNER 7 (1) World Health Organisation estimate of prevalence for both sexes in (2) Medix estimate.

8 Tesofensine could double weight loss compared to competitors Results at 48 weeks suggest tesofensine could be used as an alternative to surgery Reduction in bodyweight compared to baseline Phase 2b study methodology Randomised, double-blind, placebo controlled trial in five Danish obesity management centers 203 patients were enrolled: - 52 received placebo - 52 assigned to 0.25mg tesofensine group - 50 assigned to 0.50mg tesofensine group - 49 assigned to 1.00mg tesofensine group Energy restricted diet with a daily energy deficit of 300kcal in addition to physical activity of minutes Primary endpoint: percentage change in bodyweight compared to baseline at 24 weeks Phase 2b trial results Placebo 2.0% average reduction 0.25mg tesofensine 6.5% average reduction 0.50mg tesofensine 11.2% average reduction 1.00mg tesofensine 12.6% average reduction Adverse effects similar to placebo with an increase in heart rate compared to baseline Follow up results An open label study was conducted to follow patients for a further 24 weeks after the Phase 2b trial. At 48 weeks patients had lost 14-15% in bodyweight compared to baseline. The results at 48 weeks suggest tesofensine could be competitive to surgery which usually result in 15-20% reduction in bodyweight 8 *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.

9 Placebo controlled weight loss Tesofensine could double weight loss compared to competitors Results at 48 weeks suggest tesofensine could be used as an alternative to surgery Reduction in bodyweight versus competing drugs* Tesofensine 0.5 mg, 48 weeks open label: 14-15% compared to baseline Phase 2b study methodology Randomised, double-blind, placebo controlled trial in five Danish obesity management centers 203 patients were enrolled: - 52 received placebo - 52 assigned to 0.25mg tesofensine group - 50 assigned to 0.50mg tesofensine group - 49 assigned to 1.00mg tesofensine group 10,0% 9,0% 8,0% 7,0% 6,0% 5,0% 5.2% 6.0% 6.6% 9.2% Energy restricted diet with a daily energy deficit of 300kcal in addition to physical activity of minutes Primary endpoint: percentage change in bodyweight compared to baseline at 24 weeks Phase 2b trial results Placebo 2.0% average reduction 0.25mg tesofensine 6.5% average reduction 0.50mg tesofensine 11.2% average reduction 1.00mg tesofensine 12.6% average reduction 4,0% 3,0% 2,0% 1,0% 0,0% 2.4% Xenical, 3x120mg, 4 years 3.1% Belvig, 2x10mg, 1 year Contrave, 2x360/32mg, 56 weeks Victoza, 3mg, 56 weeks Qsymnia, 7.5/46mg, 56 weeks Tesofensine, 0,50mg, 24 weeks Adverse effects similar to placebo with an increase in heart rate compared to baseline Follow up results An open label study was conducted to follow patients for a further 24 weeks after the Phase 2b trial. At 48 weeks patients had lost 14-15% in bodyweight compared to baseline. The results at 48 weeks suggest tesofensine could be competitive to surgery which usually result in 15-20% reduction in bodyweight 9 *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.

10 Medix Phase 3 study Medix to initiate Phase 3 in 2017 and could potentially be on the market before 2020 Randomised, double-blind, placebo controlled trial in Mexican population 372 patients to be enrolled: 124 will receive placebo 124 will receive 0.25mg tesofensine 124 will receive 0.50mg tesofensine All patients are prescribed an energy restricted diet with a daily energy of 1,500-2,000 kcal in addition to a physical activity of minutes Primary endpoint: percentage change in bodyweight compared to baseline at 24 weeks Secondary endpoints include: proportions of patients achieving a weight loss of more than 5 and 10 percent respectively, metabolic including glycaemic endpoints, quality of life. 10

11 EFFECT INDICATIONS COMPOSITION Tesomet - Potential blockbuster drug with orphan drug prospects Tesomet s mechanism of action creates multiple opportunities within metabolic diseases Tesomet Indications and treatment effects Potential best-in-class profile combining unmatched weight loss with a benign side effect profile for the treatment of weight related metabolic diseases DESCRIPTION Tesomet is a fix-dosed combination of tesofensine and metoprolol TESOFENSINE Effective weight loss drug TYPE 2 DIABETES PRADER WILLI METOPROLOL Beta blocker to control increased heart rate FATTY LIVER (NASH) INDICATIONS Type 2 diabetes 1 - Global prevalence: ~370 million Prader Willi 2 - Global prevalence ~0.4million Fatty liver (NASH) 3 - US prevalence +10 million Chronic condition characterized by elevated blood glucose Rare genetic disorder which among other things causes a chronic feeling of hunger leading to excessive eating Common disease characterised by a build up of fat in the liver potentially leading to liver damage MARKET SIZE Type 2 diabetes 4 Prader Willi Fatty liver USD 23bn NA NA Reduction of fat in the liver leading to normalisation of diabetic symptoms and long term remission Reduction in the chronic feeling of hunger to normal levels Reduction of fat build-up in the liver allowing a reversal of disease progression STAGE OF DEVELOPMENT Phase IIa recruitment completed PROPRIETARY PROJECT 11 (1) Estimate based on International Diabetes Federation s total estimate of diabetics in the world in 2015 and the average estimate of split between type 1 and 2 diabetes based on: Tuomilehto J. The emerging global epidemic of type 1 diabetes. Curr Diab Rep 2013; 13: and Centers for Disease Control and Prevention, National diabetes statistics report: estimates of diabetes and its burden in the United States, Atlanta, GA: US Department of Health and Human Services, 2014 (2) Butler MG, Hanchett JM, Thompson T. Clinical findings and natural history of Prader-Willi syndrome. In: Management of Prader-willi Syndrome, Butler MG, Lee PDK, Whitman BY (Eds), Springer, New York 2006 (3) G. Vernon; A. Baranova; Z. M. Younossi, The Epidemiology and Natural History of Non-alcoholic Fatty Liver Disease and Non-alcoholic Steatohepatitis in Adults ( (4) Datamonitor, 2014.

12 Retrospective analysis on pre-diabetics in TIPO-1 study Tesofensine induces weight loss and reduces HbA1c in overweight pre-diabetics Pre-diabetics obtain significant weight loss Pre-diabetics obtain normalization of HbA1C 12

13 Tesomet Phase 2a study to prove efficacy and safety Result: Tesomet neutralize increase in heart rate and maintain extraordinary weight loss Phase 2a high level development plan Tesomet meets primary endpoint 60 type 2 diabetes patients with two arms: placebo versus Tesomet Heart rate reduced with of 4.3 bpm (p= versus placebo) 3 months treatment No diet nor exercise introduced Primary endpoint Demonstrate that Metoprolol can counteract tesofensine s heart rate effects Tesomet reduce body weight, blood pressure and liver fat Body weight reduced with 3.5 kg (3.5%) (p< versus placebo) Waist circumference reduced with 2.29 cm (p<0.01 versus placebo) Secondary endpoints Numerical reduction in blood pressure Systolic reduced by 3.1 mmhg (p=0.152) ENDPOINT RELEVANT INDICATION Diastolic reduced by 2.2 mmhg (p=0.138) Sustained efficacy of Tesomet on weight loss Type 2 diabetes, Prader Willi, Fatty liver Numerical reduction in liver fat content of 8.3% whereas placebo Effect on liver fat Fatty liver and type 2 diabetes increased with 11.1% (p=0.0625) Effect on glycemic endpoints Type 2 diabetes Glycemic secondary efficacy endpoints not significantly reduced in this rather short 12-week study. 13

14 Additional indication added following positive Phase 2a results Saniona initiated a Phase 2a trial in Prader Willi Syndrome in Q Prader Willi A rare genetic disorder Chronic feeling of extreme hunger Excessive eating and life-threatening obesity Incidence is 1 in 15,000 live births No cure available today Expensive for society and burdensome for affected families Limited numbers of ongoing programs in the pipeline Small Phase 2a study initiated in April 2017 Exploratory randomized, double-blind, placebocontrolled 12 weeks study in patients 3:2 - randomization Tesomet (tesofensine 0.5 mg + metoprolol 50 mg) Placebo Split in two parts with safety evaluation First part: adult patients Second part: adolescents Results expected one year from initiation Tesomet A promising candidate for PWS Tesomet could provide substantial benefits to patients Potential for orphan drug status, breakthrough designation and expedited review Relatively small and manageable clinical program: - Small Proof of Concept study may serve as Phase 2 (Zafgen n=17) - Phase 3 trials likely to be a few hundred patients (Zafgen n=108, 6 months) 14

15 Next step for Tesomet Prepare for long term Phase 2b and Phase 3 studies by Saniona or together with a partner Increase data package in order to enable long term studies with combination product Long term preclinical studies on combination Phase 1 clinical study on the fixed dose combination pill Indications and patient population for consideration Type 2 diabetes or subgroup of type 2 diabetes patients (e.g. maximum x years from diagnosis) Obesity or obese diabetics Binge eating, Nash Prader Willi (potential phase 3) Major Phase 2b design elements Multi arm study 6 months study Diet and Exercise 15

16 Dopamine levels NS2359 Potential first drug for cocaine addiction Phase IIa study initiated in June 2016 at the University of Pennsylvania NS2359 blunts highs and subsequent lows associated with cocaine NS2359 administered together with cocaine NS2359 normalizes dopamine levels in addicts and blunts highs and lows after cocaine Speed of increase and level of dopamine causes the feeling of euphoria NS cocaine Cocaine DESCRIPTION NS2359 is a triple monoamine reuptake inhibitor which blocks the reuptake of dopamine, norepinephrine and serotonin INDICATION Cocaine addiction million cocaine dependents in the US Normal Speed of drop in dopamine causes the feeling of a crash MARKET SIZE The global market for first class treatment for cocaine dependence is estimated at USD 1.2bn 2 Addict Time STAGE OF DEVELOPMENT Phase IIa initiated in 2016 Withdrawl symptoms Cravings Feeling of Euphoria PARTNER 16 (1) National Survey on Drug Use and Health. (2) National Institute on Drug Abuse (NIDA).

17 Phase IIa study being conducted at world class institution University of Pennsylvania TRC is among the top sites for clinical trials in drug addiction Phase I data supports NS2359 s efficacy Phase IIa high level development plan Phase I data in 24 psychostimulant cocaine users shows NS2359 reduces the attractiveness of cocaine to addicts in multiple factors 80 cocaine addicts with two arms: placebo versus NS weeks treatment "Amount you would pay" 39% 100% Primary endpoint "Like to use" 32% 100% Abstinence from cocaine during the last 2 weeks of treatment "Feeling stimulated" 58% 100% Secondary endpoints "Desire" 36% 100% Reduce craving for cocaine and withdraw symptoms Reduce amount of alcohol consumption "Drug liking" 23% 100% Reduce smoking Improved cognitive ability "Feeling high" 53% 100% Trial site 0% 20% 40% 60% 80% 100% Cocaine NS cocaine In addition to normalising the heart rate Heart rate

18 1. Introduction 2. Portfolio I. Clinical stage programs II. Pre-clinical programs 3. Financials and outlook 18

19 Boehringer deal new preclinical collaboration Further validation of Saniona s partnering approach FOCUS Develop novel compounds for the treatment of schizophrenia DRUG TARGET Unique Saniona ion channel research program FINANCIALS Saniona will receive: - EUR 5m (SEK ~ 47.4m) in upfront payment - Research funding - Up to EUR 90m (SEK ~853m) in milestone payments - Royalties on worldwide net sales of any resulting products under the collaboration Active preclinical programs Program Indication Partner Boehringer GABAA α2/α3 IK program Ataxion Proximagen Nicotinic α6 Schizophrenia Neuropathic pain Inflammation, IBD Ataxia Neurological disorders Parkinson s disease Added 29 August

20 Pipeline financing and commercial rights distribution Saniona commercial rights GABAA α2/α3 Pain IK program IBD A free ride NS2359 cocaine addiction Saniona finance Tesomet metabolic Tesomet Prader-Willi Tesomet obesity Tesofensine obesity 3 rd party finance Risk sharing 3 rd party commercial rights Clinical stage programs Pre-clinical programs 20

21 1. Introduction 2. Portfolio I. Clinical stage programs II. Pre-clinical programs 3. Financials and outlook 21

22 Strategy and financing Focus internal investments on most advanced programs Evaluate potential of Tesomet in various indications and preparing for late stage clinical trials: - Phase 2 in T2D a disruptive concept in a very large market - Prader Willis an orphan metabolic disorder with no treatment today - Binge eating a relative large CNS based disorder with poor treatment options today - NASH (fatty liver) the next projected metabolic syndrome epidemic Select and develop one additional clinical candidate Partners pave the way for two clinical programs which could reach the market within 3-5 years Medix conducts Phase 3 for tesofensine in obesity to obtain market approval in the first 2 countries University of Penn conducts Proof of Concept on NS2359 for cocaine addictions based on external grants Funded research engine secures long term value and financial support to internal activities Research collaborations create long term value as projects develop to clinical stage Research collaborations also provide funding for internal activities until market approval - Operational costs - Investments in internal pipeline 22

23 Financial position Income statement MSEK Acummulated Net sales Operating expenses Operating profit/loss Financial items Tax on net profit Profit/loss Other comprehensive income Total comprehensive income Balance sheet MSEK Dec-16 Dec-15 Dec-14 Dec-13 Dec-12 Non-current assets Current receivables Cash and cash equivalent Total assets Equity Total liabilities Total equity and liabilities Cash flows MSEK Acummulated Operating activities Investing activities Financing activities Cash flow

24 Ambitions for 2017 Pipeline ambitions Current status Report top-line data on Tesomet Phase 2a Initiate a Phase 2a study on Tesomet for Prader-Willi syndrome Initiate Phase 3 for tesofensine in Mexico together with Medix Selection of clinical candidate and initiation of preclinical development for at least one of the current drug discovery programs January April + Regulatory approval, April 2017 Business development ambitions Additional partnering on clinical assets Collaboration or spin-outs on other programs and platform 1 Spin-out, May 2017 Corporate ambitions List Saniona on Nasdaq Stockholm Listed in June

25 Summary 1 Clinical stage company with 3 programs in Phase 2 clinical trials - Tesomet for T2D financed by Saniona - Tesomet for Prader-Willi financed by Saniona - NS2359 for cocaine addiction based on grants 2 New partnerships with significant potential - 4 new partnerships - Spin-out of Initiator Pharma and distribution of shares according to Lex ASEA - Spin-out of Scandion Oncology 3 Many future potential inflection points - Medix initiates Phase 3 for tesofensine in obesity - Selection of clinical candidate for internal development - Safety read out and results for Prader Willi study as well as future plans for Tesomet 25 - Interim data for NS2359 in cocaine addiction - World class research team and several programs for partnering or spin-outs