Company presentation June 2017 1 | Clinical stage company focusing - - PowerPoint PPT Presentation
Company presentation June 2017 1 | 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
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June 2017
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Selected partners
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
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1. Introduction 2. Portfolio
I. Clinical stage programs II. Pre-clinical programs
3. Financials and outlook
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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
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08/2016 – Saniona signs partnership with Boehringer Ingelheim for Schizophrenia
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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 04/2017 – Saniona initiates Phase 2a study for Tesomet in Prader-Willi syndrome 04/2017 – Medix receives approval to initiate Phase 3 study in obesity 04/2017 – Saniona obtains research milestone from The Michael J. Fox Foundation for Parkinson's Research
2016 2017 2014
04/2014 - Heavily
05/2016 - Saniona moves to Nasdaq First North Premier 06/2017 - Saniona moves to Nasdaq Stockholm Small Cap 09/2012 – Saniona
06/2016 – U Penn (TRC) initiates Phase 2a study for NS2359 in cocaine addiction
2017 2012
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1. Introduction 2. Portfolio
I. Clinical stage programs II. Pre-clinical programs
3. Financials and outlook
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(1) World Health Organisation estimate of prevalence for both sexes in 2014. (2) Medix estimate.
PARTNER DESCRIPTION
Tesofensine is a triple monoamine reuptake inhibitor
INDICATION (TARGET GEOGRAPHY)
Obesity (Mexico)1
28.1%
STAGE OF DEVELOPMENT
Regulatory approval to start Phase III in 2017
MARKET SIZE
The prescription medicine market for obesity in Mexico is around USD 250 million2
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
Food cravings DOPAMINE PLEASURE REWARD CENTER
reward center
Appetite SEROTONIN SATIETY CENTER
center
Fat burn NORADRENALINE PATHWAYS
increasing the heart rate, both functions are controlled by noradrenaline
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Results at 48 weeks suggest tesofensine could be used as an alternative to surgery
Randomised, double-blind, placebo controlled trial in five Danish
203 patients were enrolled:
Energy restricted diet with a daily energy deficit of 300kcal in addition to physical activity of 30-60 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
Reduction in bodyweight compared to baseline
*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.
Phase 2b study methodology
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2.4% 3.1% 5.2% 6.0% 6.6% 9.2%
0,0% 1,0% 2,0% 3,0% 4,0% 5,0% 6,0% 7,0% 8,0% 9,0% 10,0%
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
Placebo controlled weight loss
Results at 48 weeks suggest tesofensine could be used as an alternative to surgery
Randomised, double-blind, placebo controlled trial in five Danish
203 patients were enrolled:
Energy restricted diet with a daily energy deficit of 300kcal in addition to physical activity of 30-60 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
Reduction in bodyweight versus competing drugs*
Tesofensine 0.5 mg, 48 weeks open label: 14-15% compared to baseline
*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.
Phase 2b study methodology
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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 20-40 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.
Medix to initiate Phase 3 in 2017 and could potentially be on the market before 2020
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TESOFENSINE METOPROLOL
Effective weight loss drug Beta blocker to control increased heart rate
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
TYPE 2 DIABETES
Chronic condition characterized by elevated blood glucose Reduction of fat in the liver leading to normalisation of diabetic symptoms and long term remission
COMPOSITION INDICATIONS EFFECT
Tesomet’s mechanism of action creates multiple opportunities within metabolic diseases
PROPRIETARY PROJECT DESCRIPTION
Tesomet is a fix-dosed combination of tesofensine and metoprolol
INDICATIONS
Type 2 diabetes1
~370 million Prader Willi2
~0.4million Fatty liver (NASH)3
+10 million
STAGE OF DEVELOPMENT
Phase IIa recruitment completed
MARKET SIZE
Type 2 diabetes4 USD 23bn Prader Willi NA Fatty liver NA
(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: 795–804 and Centers for Disease Control and Prevention, National diabetes statistics report: estimates of diabetes and its burden in the United States, 2014. 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 (http://www.medscape.com/viewarticle/746578_5) (4) Datamonitor, 2014.
FATTY LIVER (NASH)
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 Reduction in the chronic feeling
Reduction of fat build-up in the liver allowing a reversal of disease progression
PRADER WILLI
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Tesofensine induces weight loss and reduces HbA1c in overweight pre-diabetics
Pre-diabetics obtain significant weight loss Pre-diabetics obtain normalization of HbA1C
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Result: Tesomet neutralize increase in heart rate and maintain extraordinary weight loss
Secondary endpoints
Fatty liver and type 2 diabetes
RELEVANT INDICATION
Type 2 diabetes Type 2 diabetes, Prader Willi, Fatty liver Sustained efficacy of Tesomet on weight loss
ENDPOINT
Effect on glycemic endpoints Effect on liver fat
Phase 2a high level development plan
60 type 2 diabetes patients with two arms: placebo versus Tesomet
3 months treatment
No diet nor exercise introduced
Primary endpoint
Demonstrate that Metoprolol can counteract tesofensine’s heart rate effects
Tesomet meets primary endpoint
Heart rate reduced with of 4.3 bpm (p=0.0038 versus placebo)
Tesomet reduce body weight, blood pressure and liver fat
Body weight reduced with 3.5 kg (3.5%) (p<0.0001 versus placebo)
Waist circumference reduced with 2.29 cm (p<0.01 versus placebo)
Numerical reduction in blood pressure
Systolic reduced by 3.1 mmHg (p=0.152)
Diastolic reduced by 2.2 mmHg (p=0.138)
Numerical reduction in liver fat content of 8.3% whereas placebo increased with 11.1% (p=0.0625)
Glycemic secondary efficacy endpoints not significantly reduced in this rather short 12-week study.
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Saniona initiated a Phase 2a trial in Prader Willi Syndrome in Q2 2017
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, placebo- controlled 12 weeks study in 20-30 patients 3:2 - randomization Tesomet (tesofensine 0.5 mg + metoprolol 50 mg) Placebo Split in two parts with safety evaluation First part: 10-15 adult patients Second part: 10-15 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:
6 months)
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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
Prepare for long term Phase 2b and Phase 3 studies by Saniona or together with a partner
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(1) National Survey on Drug Use and Health. (2) National Institute on Drug Abuse (NIDA).
PARTNER DESCRIPTION
NS2359 is a triple monoamine reuptake inhibitor which blocks the reuptake of dopamine, norepinephrine and serotonin
INDICATION
Cocaine addiction1
STAGE OF DEVELOPMENT
Phase IIa initiated in 2016
MARKET SIZE
The global market for first class treatment for cocaine dependence is estimated at USD 1.2bn2
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
Phase IIa study initiated in June 2016 at the University of Pennsylvania
Withdrawl symptoms Cravings Feeling of Euphoria 1 2 3 4 5 6 7 8 9 10 1 2 3 4 5 6 7 8 9 10 Dopamine levels NS2359 + cocaine Cocaine Time Normal Addict
Speed of increase and level of dopamine causes the feeling of euphoria Speed of drop in dopamine causes the feeling of a “crash”
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Phase I data supports NS2359’s efficacy
80 cocaine addicts with two arms: placebo versus NS2359
8 weeks treatment
Primary endpoint
Abstinence from cocaine during the last 2 weeks of treatment
Secondary endpoints
Reduce craving for cocaine and withdraw symptoms
Reduce amount of alcohol consumption
Reduce smoking
Improved cognitive ability
Trial site
53% 23% 36% 58% 32% 39% 100% 100% 100% 100% 100% 100% 0% 20% 40% 60% 80% 100% "Feeling high" "Drug liking" "Desire" "Feeling stimulated" "Like to use" "Amount you would pay"
Cocaine NS2359 + cocaine
88 123 20 40 60 80 100 120 Heart rate
Phase I data in 24 psychostimulant cocaine users shows NS2359 reduces the attractiveness of cocaine to addicts in multiple factors
In addition to normalising the heart rate
University of Pennsylvania TRC is among the top sites for clinical trials in drug addiction
Phase IIa high level development plan
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1. Introduction 2. Portfolio
I. Clinical stage programs II. Pre-clinical programs
3. Financials and outlook
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Further validation of Saniona’s partnering approach Active preclinical programs
Boehringer GABAA α2/α3 IK program Ataxion Proximagen Nicotinic α6 Schizophrenia Neuropathic pain Inflammation, IBD Ataxia Neurological disorders Parkinson’s disease Program Indication Partner
Added 29 August 2016
FOCUS
Develop novel compounds for the treatment of schizophrenia
DRUG TARGET
Unique Saniona ion channel research program
FINANCIALS
Saniona will receive:
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IK program IBD
NS2359
cocaine addiction
Tesofensine
Tesomet
Saniona finance 3rd party finance Saniona commercial rights 3rd party commercial rights A free ride
GABAA α2/α3 Pain
Risk sharing
Clinical stage programs Pre-clinical programs
Tesomet
Prader-Willi
Tesomet
metabolic
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1. Introduction 2. Portfolio
I. Clinical stage programs II. Pre-clinical programs
3. Financials and outlook
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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
Partners pave the way for two clinical programs which could reach the market within 3-5 years
Research collaborations create long term value as projects develop to clinical stage Research collaborations also provide funding for internal activities until market approval
Funded research engine secures long term value and financial support to internal activities
Evaluate potential of Tesomet in various indications and preparing for late stage clinical trials:
Select and develop one additional clinical candidate
Focus internal investments on most advanced programs
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Income statement
MSEK 2016 2015 2014 2013 2012 Acummulated Net sales 74.9 13.6 21.7 13.3 7.9 131.5 Operating expenses
Operating profit/loss 4.2
Financial items 0.8
0.5
0.1 Tax on net profit
6.3 1.8 0.4 0.6 6.4 Profit/loss 2.2
Other comprehensive income
0.3 0.0
0.0
Total comprehensive income 1.1
Balance sheet
MSEK Dec-16 Dec-15 Dec-14 Dec-13 Dec-12 Non-current assets 2.7 2.3 2.1 2.0 0.1 Current receivables 14.8 8.4 3.7 1.1 0.8 Cash and cash equivalent 53.3 47.0 9.7 0.9 7.2 Total assets 70.8 57.7 15.5 4.0 8.0 Equity 54.3 52.9 8.8
Total liabilities 16.5 4.7 6.7 6.9 9.6 Total equity and liabilities 70.8 57.7 15.5 4.0 8.0
Cash flows
MSEK 2016 2015 2014 2013 2012 Acummulated Operating activities 8.0
7.1
Investing activities
Financing activities
17.6
84.4 Cash flow 7.2 36.9 8.7
7.1 53.8
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Pipeline ambitions
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
Current status
January 2017
Business development ambitions
Additional partnering on clinical assets Collaboration or spin-outs on other programs and platform List Saniona on Nasdaq Stockholm Selection of clinical candidate and initiation of preclinical development for at least one of the current drug discovery programs
Corporate ambitions
April 2017
Spin-out, May 2017
Regulatory approval, April 2017
Listed in June 2017
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Clinical stage company with 3 programs in Phase 2 clinical trials
New partnerships with significant potential
Many future potential inflection points