Pioneering Ion Chanel Development For The Treatments of Rare - - PowerPoint PPT Presentation

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Pioneering Ion Chanel Development For The Treatments of Rare Diseases

Corporate presentation

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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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Saniona Executive Leadership Team

Jørgen Drejer, Ph.D.

CSO, Co-Founder and Board Member

Thomas Feldthus, M.Sc., MBA

CFO and Co-Founder

Rami Levin, MBA

President, CEO and Board Member

Board member of 2Curex AB Previously, Executive Vice President, Research Director and co-founder of NeuroSearch A/S, Department Head and Project Manager at Novo Nordisk. Board member of Scandion Oncology A/S Previously, CFO and co-founder of Symphogen A/S, Investment manager of Novo A/S, Corporate Development manager of Novo Nordisk A/S Previously, President Sobi Inc., Vice President Marketing and EMD Serono Inc. Managing Director Merck Serono in Scandinavia, Global Marketing Director Merck Serono, Geneva, Switzerland

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Saniona Investment Highlights

Clinical stage Biopharmaceutical company focused

• n rare diseases with high unmet medical need

Addressing unmet needs in rare CNS diseases Late stage treatment in development for two rare eating disorders: Prader Willi Syndrome (PWS) and Hypothalamic Obesity (HO) PWS: Will begin phase 2b in H2, 2020 HO: Phase 2 results available in Q2, 2020 Unique ion-channel drug discovery platform SAN711 – For rare neuropathic itching disorders (e.g. brachioradial pruritus), entering phase 1 SAN903 – For rare inflammatory and fibrotic disorders (e.g. idiopathic pulmonary fibrosis) Delivering additional value through strategic partnerships Tesofensine for obesity CAD-1883 for essential tremor and Ataxia GABAa5 for schizophrenia An experienced executive leadership team with a combined experience of over 80 years in the industry both in the US and the EU Experience in research, development and commercialization of rare disease drugs, both in the US and EU

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Prader-Willi Syndrome (PWS)

“Therefore, patients are more available to other activities, and life as a whole becomes easier for the patients and their families.” Dóra Török Primary Investigator “Weight gain, hyperphagia and obsession with food are the greatest burden on both patients with Prader-Willi syndrome and their families. Tesomet helps to control weight and appetite and it decreases preoccupation with food.”

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Prader-Willi Syndrome, a Debilitating, Rare Genetic Disorder

Patient Population Birth incidence: 1 in 15000 Cause Absence of paternally expressed genes at Chromosome 15 (q11-q13) Disease Characteristics Hyperphagia, insatiable hunger Short life expectancy, median 30-40 years1 Complications from hyperphagia Obesity related comorbidities Intellectual disabilities, physical deficiencies, behavioral problems Significant burden on caregivers and families

1Manzardo, A., Loker, J., Heinemann, J. et al. Survival trends from the Prader–Willi Syndrome Association

(USA) 40-year mortality survey. Genet Med 20, 24–30 (2018) doi:10.1038/gim.2017.92

USA 8,000 Europe 13,000 Japan 3,300

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Tesomet - a true Triple Monoamine Uptake Inhibitor Controls Eating

Tesomet increases levels of monoamines by blocking re- uptake Reduces hyperphagia by controlling appetite and craving for food Increases metabolic rate Addresses significant unmet needs in PWS Tesomet

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Significant reduction in hyperphagia scores in phase 2a PWS study

Adults: Tesomet 0.5 mg reduced the hyperphagia score to zero in a double-blind study Adolescents: Hyperphagia is down to low single digits at 0.25 mg per day during OLE 2

N=2 N=5 N=6 N=6 N=2 N=2 N=3 N=2

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• 6
• 4
• 2

2 4 6

• 2.6

2.9 3.6 0.4

• 5.6

% change in body weight (adults & adolescents)

change in weight (%) Tesomet Placebo

Significant reduction in body weight in phase 2a PWS study

The 0.5 mg in adults showed promising efficacy The 0.125 mg in adolescents did not show efficacy

• n primary endpoint
• likely due to too low exposures of tesofensine

Double blinded phases of the study: Extension phases of the study:

A weight reduction was seen in OLE 2 where patients were at the target plasma levels of tesofensine

adults adolescents

0.5 mg (n=9) 0.125 mg (n=9) 0.25 mg (n=3) 0.125 mg (n=1)

OLE-2

ANCOVA, Change from baseline to Day 91, LOCF

3 months 3 months 3 months

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• 7%
• 6%
• 5%
• 4%
• 3%
• 2%
• 1%

0% 1% 2% 3% 4% 5% 6% 7%

• 1

2 3 4 5 6 7 8 9 10 11 12

% change in body weight Plasma level (ng/ml)

Monthly change in body weight by plasma level in individual patients

Significant correlation between change in body weight and plasma level

0.125 mg 0.25 mg 0.125 mg 0.25 mg

All individual patients experienced dose-dependent monthly weight loss – highly statistically significant correlation between weight loss and plasma concentration

p=0.003 (intercept) p=0.005 (slope) Random Co-Efficient Analysis PK versus % Monthly Weight Change

Expected 95% conf. limits 95% pred. limits

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Conclusions of phase 2a results of Tesomet in Prader Willi Syndrome

Study Design

• 18 patients dosed and followed

up to 9 months

• Three doses investigated:

0.125mg, 0.25mg and 0.5mg

Main Efficacy Findings

• Significant reduction in

hyperphagia score

• 2.6% reduction in body weight
• n the 0.25mg dose by the end of

the study

• Good correlation between

efficacy, dose and plasma level

Main Safety Findings

• 0.125mg and 0.25mg doses

were safe and well tolerated

• Higher than expected drop-out

rates were observed at a dose of 0.5mg

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Study Overview 16-week dose finding study; 36-week open label extension with dose adjustment In total: about 150 patients over a 2-3-year period and a total investment of $30M

Pivotal PWS Clinical Program with Overlapping Program Design

2020 2021 2022

FDA filing

Phase 2b Phase 3

Tesomet

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Hypothalamic Obesity (HO)

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Hypothalamic Obesity, an Acquired Eating Disorder

Patient Population 50% of patients acquire HO post craniopharyngioma1 Prevalence ~1 in 50,000-100,0002 Disease characteristics Post surgical obesity and hyperphagia, insatiable hunger Memory impairment, attention, impulse control Depression and suicide

1 Roth, C., Hypothalamic Obesity in Craniopharyngioma Patients, J. Clin. Med. 2015, 4(9), 1774-1797; https://doi.org/10.3390/jcm4091774 2Inferred given prevalence of craniopharyngioma cited by Garnett, M.R., Puget, S., Grill, J. et al. Craniopharyngioma. Orphanet J Rare Dis 2, 18

(2007) doi:10.1186/1750-1172-2-18

USA 3,400 – 6,800 Europe 5,500 – 11,000 Japan 1,300 – 2,600

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Tesomet: Phase 2a Study in Hypothalamic Obesity

Study Overview

• Primary endpoint: safety
• Secondary endpoints: Change in bodyweight compared to baseline at 24 weeks, appetite

score, metabolic including glycemic endpoints, quality of life

• Single centre, randomized, double-blind placebo controlled trial
• 21 patients enrolled

Placebo Placebo crossover Tesomet* 24-weeks double blind 24-weeks open label

2020 2021

Phase 3

FDA FDA filing

2022

Phase 2 Phase 3 *0. 50mg tesofensine + 50mg metroprolol

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Unique Ion-Channel Drug Discovery Platform

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Proprietary Pipeline: Multiple Value Drivers

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Product Indication Preclinical Phase 1 Phase 2a Phase 2b Next steps

Tesomet tesofensine + metoprolol

(monoamine reuptake inhibitor + beta blocker)

Prader-Willi Syndrome Ph2b start 2020 Hypothalamic Obesity Ph2a completion Q2 2020 SAN711

(GABA α3 PAM)

Rare neuropathic itching disorders Phase 1 SAN903

(IK channel blocker)

Rare inflammatory disorders Filing IND

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Overview of Strategic Partnerships

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Partnered Pipeline and Milestones

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Product Indication Preclinical Phase 1 Phase 2 Phase 3

Tesofensine Obesity CAD-1883 Essential tremor Ataxia

Upfront: 5M € Milestones: 85M € Royalties Spinout Minority stake Royalties Expected approval and launch in H2 2020

GABAa5 Schizophrenia

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Conclusions

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Expected Corporate Milestones

TIMING EVENT H2 2020

• Tesomet PWS: Initiate phase 2b
• Tesomet HO: Topline data
• Approval/launch: tesofensine Mexico
• tesofensine NDA filing in Argentina

H1 2020

• Tesomet PWS: IND filing

PARTNER EVENT

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Saniona Investment Highlights

4. Delivering additional value through strategic partnerships 2. Late stage treatment in development for two rare eating disorders: Prader Willi Syndrome and Hypothalamic Obesity 3. Unique ion-channel drug discovery platform 5. An experienced executive leadership team with a combined experience of

• ver 80 years in the industry both in the US and the EU

1. Clinical stage Biopharmaceutical company focused on rare diseases with high unmet medical need

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