Company Presentation March 2019 1 | FORWARD LOOKING STATMENTS This - - PowerPoint PPT Presentation

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Company Presentation March 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

An advanced-stage pipeline in the drug tesofensine Tesofensine is progressing toward regulatory approval in the $250M obesity market in Mexico. Other markets are being evaluated Tesomet (a combination of tesofensine and metoprolol) has demonstrated dramatic reductions in craving for food and weight in the rare genetic eating disorder Prader Willi Syndrome and will be progressed to Phase 2b targeting a billion dollar market Tesomet is also being studied in patients with Hypothalamic Obesity, another rare eating disorder with limited treatment options representing a billion dollar market opportunity Discovered and is advancing four first in class ion channel modulators through early clinical and late preclinical development Two internally addressing large market segments as well as rare diseases with high medical need Two with partners for treatment of schizophrenia and ataxia respectively Continue to look for strategic partners where shareholder value can be accelerated Experienced management team with a globally recognized track record of developing product candidates for CNS related disorders and conditions

“Saniona has two drugs that work!”

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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 Ph2b/3 start 2019/20 Hypothalamic

• besity

Ph2a readout Q1 20 SAN711

(GABA α3 PAM)

Neuropathic pain Itching Ph1 initiation Q3 19 IK Program Inflammation, IBD Candidate selection Q2 19

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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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Tesofensine – How does it work?

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Tesomet = Tesofensine + “betablocker”

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Tesofensine: 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 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 1800 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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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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2018 2019 2020 2021 2022 Prader Willi Syndrome Hypothalamic

• besity

Open label

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

Potential for market entry within 4 years >$4B opportunity Hypothalamic Obesity

Phase 2 study initiated in March 2019 Proof of principle from previous compound Life-threatening hyperphagia and obesity Prevalence: 1/(50.000-100,000) Estimated market size: >1B USD

Phase 2a Phase 2b Phase 3 FDA filing Phase 2a Phase 3 FDA filing

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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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Causes of death among 312 individuals with PWS

Source: HHS Public Access, Merlin G. Butler et al

Category N Mean Age SD Respiratory Failure 94 24.6 16 Cardiac 50 32.1 14 GI 30 21.4 16 Infection 29 35.7 16 Obesity 22 30.7 12 Pulmonary Embolism 19 34.1 12 Choking 18 30.1 17 Accident 17 25.0 16 Renal Failure 7 34.2 11 Neurologic 6 18.0 21 Hypothermia 3 30.8 14 Drug Reaction 3 25.1 9 Cancer 4 39.7 27

Merlin G. Butler et al: “individuals with PWS show high rates of choking, accidents and GI-perforation presumably related to uncontrolled hyperphagia and food seeking behaviors contributing to about one third

• f all reported deaths and about one half of the deaths in childhood”

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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

Results from Phase 2a study in February 2018 (0.5 mg per day) Conclusion: Second part of Phase 2a study to be conducted at quarter of dose (0.125 mg per day)

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Tesomet: Phase 2a Study in PWS adults

Strong effect on hyperphagia and weight (0.5 mg per day)

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)

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: Phase 2a Study in PWS adolescents

Study set up Exploratory randomized, double-blind, placebo- controlled 12 weeks study in 9 adolescents

• Tesomet 5
• Placebo 4

Reduction in hyperphagia in both treatment and placebo group

• No significant difference

in hyperphagia and weight PK and Safety

• Well tolerated
• -Long half-life in PWS

patients confirmed

• Plasma level did not

reach target

Second part of Phase 2a study conducted at quarter of dose (0.125 mg per day) Study continued to open label extension at higher dose (0.25 mg per day)

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Key learnings from PWS studies

Tesomet provides strong reduction in both hyperphagia and weight at 0.5 mg per day, but dose may be too high in some patients Tesofensine’s long half-life in PWS patients leads to very high plasma level when given at 0.5 mg per day (optimal dose for obese patients) Early onset of both hyperphagia and weight reduction suggests that Tesomet will be effective at lower doses where tesofensine has a plasma level similar to the optimal doses in obese patients The optimal dose in obesity is 0.5 mg per day, where the optimal plasma concentration is about 10 ng / ml Tesomet at 0.125 mg per day does not reach the target plasma level where tesofensine reduces appetite and significant weight loss A dose of 0.25 mg per day is estimated to provide the target plasma level of 10 ng / ml

Conclusion

Tesomet a promising highly effective treatment option for control of hyperphagia and weight in PWS patients The optimal dose is expected to be between 0.25 mg and 0.5 mg per day

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Hypothalamic Obesity (HO)

CHOP Pituitary Brain Tumor Day March 16, 2019

Cause: Benign tumor formation on the pituitary and hypothalamus Diagnosis: Histological examination of a surgical specimen Prevalence: 1 in 50000 Symptoms: Post-surgical obesity and behavioral problems (hyperphagia, memory, attention, impulse control, motivation, and socialization) Treatment: Surgical ablation of tumors; no approved treatments post-surgery

Craniopharyngioma

HO: Acquired rare disease as a consequence of damage to “appetite center” (hypothalamus)

Sibutramine was effective: Zafgen received EU orphan designation for Beloranib

Program now abandoned

Combined Dietary and Pharmacological Weight Management in Obese Hypopituitary Patients

Henriette Mersebach,* Marianne Klose,* Ole L. Svendsen,* Arne Astrup,† and Ulla Feldt-Rasmussen*

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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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Financial statements - million SEK

2018, 2017 and accumulated since the company became operations in 2012

Income statement MSEK 2018 2017 2012 - 2018 Net sales 54.9 20.7 207.1 Operating expenses

• 109.1
• 77.9
• 354.6

Operating profit/loss

• 54.2
• 57.2
• 147.5

Financial items 5.9 0.9 6.9 Tax on net profit 7.2 7.1 20.7 Profit/loss

• 41.1
• 49.2
• 119.8

Other comprehensive income 0.6

• 1.0
• 1.2

Total comprehensive income

• 40.4
• 50.2
• 121.0

Balance sheet MSEK Dec/18 Dec/17 Non-current assets 12.4 7.8 Current receivables 16.0 18.3 Cash and cash equivalent 54.7 22.3 Total assets 83.1 48.4 Equity 39.5 37.6 Total liabilities 43.6 10.7 Total equity and liabilities 83.1 48.4 Cash flows MSEK 2018 2017 2012 - 2018 Operating activities

• 22.9
• 57.3
• 105.8

Investing activities 0.9

• 6.0
• 10.1

Financing activities 46.7 33.2 164.3 Cash flow 24.7

• 30.1

48.4

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Major milestones 2018/Q1 2019 – all achieved

Dec 2017 Dec 2017 Apr 2018 Jan 2018 Jun 2018 Jul 2018 Mar 2018 Feb 2018 NEWS/activity SAN711: selection of new candidate Scandion spin out PWS Ph2 initiated PWS Ph2 second part initiated Tesomet extended tox study Tesomet tablet Ph1 study init. Tesomet – teso+met combi Ph1 init. Boehringer milestone schizophrenia Cadent Ph1 initiation Tesofensine Medix Ph3 recruitment NS2359 Cocaine addiction ongoing STATUS March 2019 Preclinical devl. complete - Ph1 ready IPO completed Nov 2018 Part 1 successfully reported Part 2 double blind completed Successfully completed Successfully completed Last cohort recruited Preclinical devl. Ongoing Cadent 40MUSD finance + Ph2 init. Successful completion of Ph3 Interim conclusion – continue Ph2 MILESTONE

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Near term value inflection points

Go-2-Market opportunity with Tesomet in orphan indications Data from open label extension studies of Phase 2a in adolescents with PWS Initiate Phase 2b/3 PWS study Results from Phase 2a in hypothalamic obesity 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

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