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Company Presentation February 2019 1 | FORWARD LOOKING STATMENTS - - PowerPoint PPT Presentation

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Company Presentation February 2019 1 | FORWARD LOOKING STATMENTS This presentation contains forward- looking statements that provide Sanionas expectations or forecasts of future events such as new product developments, regulatory approvals

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Company Presentation February 2019

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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.

FORWARD LOOKING STATMENTS

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Investment Highlights

Go-2-Market opportunity with Tesomet in orphan indications

1

  • Tesomet may be Phase 3-ready in 2020, with potential market approval in 2022
  • Phase 2a Prader Willi Syndrome (PWS) results
  • Phase 2a for Hypothalamic Obesity initiation

3

Additional value driver from late stage partnership with Medix

2

  • 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

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
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Tesomet: Go-2-Market opportunity in orphan indications

Prader Willi Syndrome

Positive Phase 2a in PWS adults Phase 2a in adolescent patients ongoing Life-threatening hyperphagia and obesity Prevalence: 1/40.000 Estimated market size: ~3B USD

2018 2019 2020 2021 2022 Prader Willi Syndrome Hypothalamic

  • besity

Hypothalamic Obesity

Phase 2 study preparations Life-threatening hyperphagia and obesity Prevalence: 1/(50.000-100,000) Estimated market size: >1B USD

Phase 2a Dose finding Phase 3 FDA filing Phase 2a Phase 3 FDA filing

Potential for market entry within 4 years – Total investment of $30-40M - >$4B opportunity

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Proprietary Pipeline

Near term news flow and value generation

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Product Indication Preclinical Phase 1 Phase 2 Milestone

Tesomet tesofensine + metoprolol

(monoamine reuptake inhibitor + beta blocker)

Prader Willi Syndrome Ph2a data Q1 19 Ph2b/3 start 2019/20 Hypothalamic

  • besity

Ph2a start Q1 19 SAN711

(GABA α3 PAM)

Neuropathic pain Itching Ph1 start IK Program Inflammation, IBD Candidate selection

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Partnered pipeline

Near term news flow and non-dilutive cash

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Product Indication Preclinical Phase 1 Phase 2 Phase 3

Tesofensine Obesity CAD-1883 Essential tremor Ataxia Not disclosed Schizophrenia NS2359 Cocaine Addiction

Upfront: 5M € Milestones: 85M € Royalties Spinout Minority stake Royalties NS2359 off patent; financed by US grants

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

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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

Tesomet = tesofensine + beta blocker (metroprolol)

  • Neutralizes slight heart rate increase observed with tesofensine
  • Allows for strong intellectual property protection through 2036

TESOFENSINE METOPROLOL

Effective weight loss drug Beta blocker to control slight increase in heart rate COMPOSITION

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Tesomet: Potential Orphan Drug with Blockbuster Potential

Potential “best-in-class” profile for weight related metabolic diseases via unmatched weight loss and benign side effect profile MoA creates multiple opportunities within metabolic diseases and eating disorders

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Tesomet/tesofensine development strategy: Geographies

EU & US: PWS and HO Rest of the world: Metabolic disease

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Tesomet: Go-2-Market opportunity in orphan indications

Prader Willi Syndrome

Positive Phase 2a in PWS adults Phase 2a in adolescent patients ongoing Life-threatening hyperphagia and obesity Prevalence: 1/40.000 Estimated market size: ~3B USD

2018 2019 2020 2021 2022 Prader Willi Syndrome Hypothalamic

  • besity

Hypothalamic Obesity

Phase 2 study preparations Life-threatening hyperphagia and obesity Prevalence: 1/(50.000-100,000) Estimated market size: >1B USD

Phase 2a Dose finding Phase 3 FDA filing Phase 2a Phase 3 FDA filing

Potential for market entry within 4 years – Total investment of $30-40M - >$4B opportunity

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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 Economic and social costs Quality of life for patients and families Family stress and loss of income Care and medical costs (USD 100-300K per year) Short life expectation (average in 30s) No effective treatment available today

}

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Tesomet: Phase 2a Study in PWS adults

Study set up Exploratory randomized, double-blind, placebo- controlled 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

Phase 2a study initiated in April 2017

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Tesomet: Phase 2a Study in PWS adults

Results

PWS hyperphagia score

(data show mean and SD)

Week 8 Week 13 Tesomet 5.00 % (n=5) 6.75 % (n=2) Placebo 0.46 % (n=2) 0.75 % (n=2)

Second part of PWS study in adolescents ongoing

20 40 60 80 100 5 10 15 20

days of treatment Hyperphagia score

placebo treatment

PWS weight loss

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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)

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Tesomet/tesofensine development strategy: Geographies

EU & US: PWS and HO Rest of the world: Metabolic disease

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Medix partnership

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 With ~50% market share, Medix is market leader in the $250M Rx Obesity Market in Mexico 2018 2019 2020 2021 2022 Mexico Argentina

Phase 3 Cofepris review Commercialization Argentina NDA Commercialization

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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 20-40 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
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Tesofensine: Phase 2 Study

Study methodology & results

Methodology

Randomized, double-blind, placebo controlled trial in five Danish

  • besity management centers

Enrolled 203 patients 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

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

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Tesofensine strong efficacy in bodyweight and appetite

Phase 2b study

  • 60.00
  • 50.00
  • 40.00
  • 30.00
  • 20.00
  • 10.00

0.00 10.00 Placebo 0.25 mg 0.5 mg 1 mg

Reduction in appetite/craving for food

Change from baseline (total score)

Reduction in bodyweight

compared to baseline

2.0% reduction 6.5% reduction 11.2% reduction 12.6% reduction

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Tesofensine could double weight loss compared to competitors

Results at 48 weeks suggest tesofensine could be used as an alternative to surgery

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

Weight loss treated vs placbo

“Apples to oranges” comparison of reduction in bodyweight versus competing drugs*

48 weeks open label

*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.

̴ 14%

Tesofensine 0,50 mg, 48 weeks

  • pen label extension
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Additional weight loss in open label extension up to 58 weeks

2 4 6 8 10 12 14 16 18 20 22 24 26 28 30 32 34 36 38 40 42 44 46 48 50 52 54 56 58

Time (weeks)

9

  • 5
  • 10
  • 15

Weight loss in kg vs baseline

Drug holiday 0.5 mg tesofensine 0.5 mg tesofensine 0.5 mg tesofensine Placebo

TIPO-1 TIPO-4 (open label extension)

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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

8 Phase 2 studies, N = 1310 (1000 tesofensine) 1 Phase 3 study, N= 372 (248 tesofensine) 2 Phase 2 studies* (one ongoing), N=78

More than 1700 subjects treated to date.

18 Phase 1 studies, N = 391 (325 tesofensine) 3 Phase 1 studies* (one ongoing), N=105

*Indications include Type 2 diabetes and Prader Willi syndrome

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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 Upfront: 5M € Milestones: 85M € Royalties Spinout Minority stake Royalties

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SAN711 for neuropathic pain and Itching with orphan potential

Class GABA Modulator (selective α3, first in class, small molecule) Status Phase 1 ready in Q1 2019 Indication 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 Prevalence Neuropathic pain: +10 million globally ex back-pain Market 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

Medical Need Efficacy and reduction of adverse effects

  • 50 % achieves no pain relief and the rest obtains partial response
  • nly
  • Only 25 % obtains a pain reduction of 50 % or more (score)

MoA Restores the endogenous pain control system in the spinal cord Patent Until 2038

Phase 1 ready in Q1 2019

Neuropathic pain Itching Prurigo Nodularis

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SAN711 mode-of-action

SAN711 increases the efficacy of dysfunctional inhibitory GABAergic neurons in spinal cord, which control relay of pain signals to the brain

Regulates spinal cord neurons’ pain and itch signalling to the brain

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Injury – spinal disinhibition Normal

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SAN711 effective in neuropathic pain model

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

Efficacy maintained after prolonged treatment

Veh 3 mg/kg 10 mg/kg 30 mg/kg

Total dist. (30 min.)

20 40 60 80 100

SAN711 alleviate nerve-injury induced neuropathic pain in rats SAN711 does not lead to sedation in rats

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IK Program for inflammation and fibrosis in IBD with orphan potential

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 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 MoA: Downregulates immune response by inhibiting calcium entry to immune cells IP: 2039

Candidate selection

Crohns disease Colitis

  • H. xerocytos
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IK program mode-of-action

KCa3.1 is expressed in all major immunological and reactive cell types involved in IBD KCa3.1 strengthens calcium-signalling via a positive control loop SAN903 inhibits KCa3.1, dampens pathological responses, and acts on acute/chronic symptoms

Downregulates immune response by inhibiting calcium entry to immune cells

Cell types Basic mechanism Cellular effect Therapeutic effect

T-cells, macrophages T-cells, B-cells, macrophages T-cells, B-cells, macrophages Fibroblasts, myofibroblasts Epithelial cells

Cytokine release ↓ Proliferation ↓ Migration ↓ Collagen release ↓ Salt and H20 transport ↓

Anti-inflammatory Anti-fibrotic Anti-diarrhetic

KCa3.1 Orai, Trp, etc.

Ca2+ channel K+ channel Effector cell

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SAN903 effective in IBD models

Healthy large intestines Inflamed large intestines SAN903-treated large intestines

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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 17 2017 2012 - Q3 2018 Net sales 52.7 16.1 20.7 204.9 Operating expenses

  • 72.6
  • 56.7
  • 77.9
  • 318.1

Operating profit/loss

  • 19.9
  • 40.6
  • 57.2
  • 113.2

Financial items

  • 0.5
  • 0.9

0.9 0.5 Tax on net profit 2.7 7.1 7.1 16.2 Profit/loss

  • 17.7
  • 34.4
  • 49.2
  • 96.5

Other comprehensive income 0.7 0.1

  • 1.0
  • 1.1

Total comprehensive income

  • 17.0
  • 34.3
  • 50.2
  • 97.6

Balance sheet

MSEK Sep 18 Sep 17 Dec-17 Non-current assets 9.0 15.2 7.8 Current receivables 15.3 9.5 18.3 Cash and cash equivalent 37.3 40.9 22.3 Total assets 61.6 65.5 48.4 Equity 50.1 53.3 37.6 Total liabilities 11.5 12.2 10.7 Total equity and liabilities 61.6 65.5 48.4

Cash flows

MSEK 9 months 18 9 months 17 2017 2012 - Q3 2018 Operating activities

  • 15.0
  • 39.9
  • 57.3
  • 97.9

Investing activities 1.4

  • 6.2
  • 6.0
  • 9.7

Financing activities 29.1 33.2 33.2 146.6 Cash flow 15.5

  • 12.9
  • 30.1

39.1

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Near term value inflection points

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 Additional value drivers from Medix tesofensine collaboration Tesofensine NDA filing in Mexico Approval and launch in Mexico Tesofensine NDA filing in Argentina Research platform and early stage pipeline SAN711: Initiation of Phase 1 for chronic pain and itching IK program: Candidate selection Potential new collaborations

1 3 2

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Saniona AB Baltorpvej 154 DK-2750 Ballerup Denmark Tel: +45 70705225 Web: saniona.com