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VICORE PHARMA A Rare Disease Company with a Focus on Patients with Fibrotic Lung Disease

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Page 1: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

VICORE PHARMA

A Rare Disease Company with a Focus on Patients with Fibrotic Lung Disease

Page 2: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

FORWARD LOOKING STATEMENT

This presentation may contain certain forward-looking statements and forecasts based onuncertainty, since they relate to events and depend on circumstances that will occur in the futureand which, by their nature, will have an impact on Vicore Pharma’s business, financial conditionand results of operations. The terms “anticipates”, “assumes”, “believes”, “can”, “could”,“estimates”, “expects”, “forecasts”, “intends”, “may”, “might”, “plans”, “should”, “projects”, “will”,“would” or, in each case, their negative, or other variations or comparable terminology are usedto identify forward-looking statement. There are a number of factors that could cause actualresults and developments to differ materially from those expressed or implied in a forward-looking statement or affect the extent to which a particular projection is realized. Factors thatcould cause these differences include, but are not limited to, implementation of Vicore Pharma’sstrategy and its ability to further grow, risks associated with the development and/or approval ofVicore Pharma’s products candidates, ongoing clinical trials and expected trial results, the abilityto commercialize C21, technology changes and new products in Vicore Pharma’s potential marketand industry, the ability to develop new products and enhance existing products, the impact ofcompetition, changes in general economy and industry conditions and legislative, regulatory andpolitical factors.

No assurance can be given that such expectations will prove to have been correct. Vicore Pharmadisclaims any obligation to update or revise any forward-looking statements, whether as a resultof new information, future events or otherwise.

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Page 3: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

INVESTMENT CASE

Fibrotic lung diseases – devastating with need of better therapies

Two complementary programs for treatment of fibrotic lung disease• VP01 – C21 (new modality) in phase II

• VP02 – IMiD (reformulated) entering phase I

Differentiated programs addressing fibrosis, vasculopathy and cough –improving quality of life

Diversification through two programs with multiple indications –idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough

Large market potential

Highly experienced leadership

Existing shareholders include HealthCap (28%), Robur, AP4, Handelsbanken and HBM Healthcare

Page 4: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

Vicore is well positioned to develop novel therapies for fibrotic lung disease

Page 5: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

THE TEAM

First class drug development team

Page 6: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

CARL-JOHAN DALSGAARD, CEOMD, PhD, Karolinska Institute with post-doc experience from Harvard Former Head of Therapy Area Pain Control, AstraZeneca R&D10 years of experience from senior management, AstraZeneca19 years of experience as Venture Partner at HealthCap

OLA CAMBER, CMC MSc, PhD, Uppsala UniversityFormer Director Pharmaceutics & Biopharmaceuticals, PharmaciaFormer Director Astra Zeneca, Pre-formulation & BiopharmaceuticalsMore than 30 years of experience in drug development

KICKI JOHANSSON, HEAD OF DRUG DEVELOPMENTMSc Pharm, PhD Former Senior Project Leader/VP AstraZenecaAccountable for the development of over 40 new compoundsApproximately 30 years’ experience of drug development

JOHANNA GRÄNS, REGULATORY AFFAIRS MANAGERMSc, PhD, University of Gothenburg Extensive experience in preclinical R&DResearch experience in drug metabolism

HANS JEPPSSON, CFOPhD, Finance with post-doc research at UC BerkeleyCross-disciplinary background in finance and medicineFormer equity research analyst at Danske BankProfessor in Finance at Gothenburg School of Economics and Law

ROHIT BATTA, CMO MBBS, King’s College London, MFPMMedical doctor with extensive industry experience in Rare DiseasesJoins us from GSK, where he led the global medical and clinical development of the world’s first paediatric gene therapy

GÖRAN TORNLING, SENIOR MEDICAL ADVISORMD, PhD, Karolinska InstitutetMedical doctor and Pulmonologist with more than 20 years of clinical experienceFormer Director Clinical Strategy, AZ and responsible for IPF study designs

JOHAN RAUD, CSO MD, PhD, Karolinska InstitutetFormer Director Inflammation research AstraZenecaInvestment Manager KIF25 years of experience in drug development

THE VICORE TEAM

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NINA CARLÈN, CHIEF ADMINISTRATIVE OFFICEREducation from Berghs School of CommunicationMore than 15 years of marketing and communicationsResponsible for HR and company administration

CHRISTIAN HALL, INVESTOR RELATIONSMSc, Stockholm School of Economics Experience as sell side analyst at SwedbankIR consultant at Oxenstierna & Partners

MINMI FLENSBURG; CLINICAL OPERATIONSDMV, PhD Experience from Novo, Lundbeck and several biotech companiesSuccessfully leading drug candidates through phase I-IV

Page 7: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

FIBROTIC LUNG DISEASE

Rare diseases with large medical needs

Page 8: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

IDIOPATHIC PULMONARY FIBROSIS (IPF)A rare interstitial pulmonary disease with unknown etiologyHigh morbidity with shortness of breath and cough with a life expectancy of 3-5 years after diagnosis

Two drugs on the marketEsbriet and Ofev only slows disease progression and show significant side effects, despite that reached sales of $ 2.3 billon in 2018

PrevalenceUS: 150,000EU: 100,000Male>female

Page 9: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

IPF COUGHA dry persistent cough In IPF a severe debilitating cough correlates to disease progression, morbidity and mortality

The pictures below shows a herniated diaphragm due to IPF cough

Prevalence for IPF with severe dry coughUS: 60,000EU: 40,000Male>female

Severe dry cough is thesymptom that impacts quality of life the mostCurrently no therapyis available

The IPF patient wants to feel better, stop coughing and live longer – in that order

Page 10: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

SYSTEMIC SCLEROSIS (SSc)

A rare autoimmune disease with heterogeneous clinical manifestationsThe disease is chronic and has frequently a progressive course, can affect every organ with significant disability, disfigurement and mortality

Prevalence for severe disseminated diseasewith ILDUS: 36,000EU: 21,000Female>male

Treatment optionsOfev (nintedanib) is recently approved for SSc/lung fibrosis (ILD)

Page 11: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

PIPELINE

Two phase II programs and one program with proven clinical efficacy

Page 12: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

Fibrosis

Exploratory Preclinical Phase I Phase II

cvSystemic Sclerosis

Idiopathic PulmonaryFibrosis

VICORE PIPELINE

12

VP01 (C21)

VP02 (IMiD)

FOLLOW-ON MOLECULES (AT2R)

Idiopathic PulmonaryFibrosis

Page 13: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

VP01 (C21)

First in class small molecule AT2R agonist

Page 14: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

C21 MODE OF ACTION

C21 is the first small molecule angiotensin II type 2 receptor (AT2R)agonist and also a low affinity thromboxane receptor (TP) antagonist

C21 AT2R

TP

AntagonistLow affinity

AgonistHigh affinity

Antifibrotic

Vasodilation

Reduced platelet activation

Anti-inflammatory

C21 has dual mode of actions, both of which are relevant for IPF and SSc

CounterbalanceAT1R

Page 15: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

AT2R EXPRESSION IN MAN

According to the Human Protein Atlas, AT2R is primarily expressed in lung in healthy individuals

8%

0%

4%

6%

2%

10%

IPF SScControl

Total AT2r quantificationIn idiopathic systemic sclerosis (IPF) and systemic sclerosis (SSc), AT2R is dramatically upregulated suggesting a crucial role in counteracting fibrosis

Parra 2014

AT2R is expressed in human lung with a significant up-regulation in fibrotic disease

Page 16: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

Wollin 2014Heckmann 2016Rathinasabapathy 2018

Esbriet PamrevlumabOfev C21

C21 IN THE BLEOMYCIN IPF MOUSE MODEL

16

Reduction of fibrosis score (∆ compared with placebo) when drugs are given as prevention

-23%-38% -41%

-65%

PIRFENIDONEROCHE

50MG/KG

NINTEDANIBB-I

60MG/KG

ANTI-CTGFFIBROGEN30MG/KG 0.03MG/KG

C21 effect is at least comparable to standard of care

C21

Esbriet PamrevlumabOfev

Page 17: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

C-21 IN THE MONOCROTALINE PH MOUSE MODEL

20

10

5

0

15

100

60

40

20

80

4

2

1

0

3

4

2

1

0

3

% InterstitialLung Fibrosis

Right Ventricularsystolic pressure

Relative AT2Receptor Expression

Relative TGF-βGene Expression

Representative images of lung fibrosis

C21 reverses cardiopulmonary fibrosis and reduce PH

Page 18: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

C21 IS ALSO A TP-RECEPTOR ANTAGONIST

In addition to the NO-mediated vasodilation via AT2R C21 also exhibit an AT2R independent vasodilation in human resistance vessels via the thromboxane receptor

C21 can address vasculopathy through both AT2R and TP receptor

Vicore, data on file

Page 19: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

VP01 – STATUS

PreclinicalEfficacy

Strong effects in models of IPF and pulmonary hypertensionReduce expression of profibrotic growth factorsDilates small resistance vessels

Safety 3 months toxicology data in mouse, dog and non human primate

CMC Established production method for C21Initially formulated as a solutionCapsule formulation under development

Phase I Completed 2019200 mg daily is safe and well toleratedConfirmed dose sufficient for AT2R engagement

Phase II IPF(PoC)

Clinical trial application (CTA) to be filed 2019Double blind, randomized and placebo controlled120 patient safety and lung function studyPowered to pick up a functional difference

Phase II SSc(PoM)

CTA filed 2019Mechanistic study to capture vascular effects

IPR Orphan drug status in IPF, 7 years in the US and 10 years in EU

Page 20: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

VP02 (IMiD)

Effective therapy targeted directly to the lung

Page 21: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

IMiDs

• IMiDs belong to a well established drug class which includesthalidomide, lenalidomide and pomalidomide

• Controlled studies and case reports confirm that systemic thalidomideresolves IPF cough

• However, peripheral neuropathy and other systemic side effects have limited the use

• IMiDs are also powerful immunomodulators and anti-inflammatory suggesting an effect also on the underlying disease

• To capture the lung effect and reduce systemic exposure Vicore is developing a formulation that delivers the IMiD directly to the lung

Page 22: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

Heckmann 2016Rathinasabapathy 2018

Esbriet PamrevlumabOfev C21

IMiDs IN BLEOMYCINE IPF MOUSE MODEL

22

Reduction of fibrosis score (∆ compared with placebo) when drugs are given as treatment

Heckmann 2016Rathinasabapathy 2018Choe 2010

-1% -10%-41% -45% -50%

PIRFENIDONEROCHE

50MG/KG

NINTEDANIBB-I

60MG/KG

ANTI-CTGFFIBROGEN30MG/KG 0.03MG/KG 4MG/KG

Esbriet PamrevlumabOfev C21 Thalidomide

IMiDS are at least comparable to standard of care

Page 23: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

IMiDs IN IPF – CLINICAL EVIDENCE

Toby MaherLondon

Treated individual cases

Lin ZhangXi’an

Open label placebo controlled studySignificant improvement in lung function

Significant reduction of cytokines

Maureen HortonBaltimore

Double blind placebo controlledcross over study

Significant reduction of coughImprove quality if life

Letitia Kawano-DouradoSao Paulo

Treated individual cases

Rebecca HarafToledo

Published case report

Consistent feedback from multiple centres – IMiDs work, however safety challenges remain

Page 24: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

IMiDS IMPROVE QUALITY OF LIFE IN IPF

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Quality of life, as measured by the specificSt George’s Respiratory Questionnaire (SGRQ), is improved by IMiD treatment

An absolute change of 7 is considered clinically relevant

No other approved or investigational drug even shows stabilization of SGRQ

Change in SGRQ scores

Thalidomide12 w; n=20

∆=11.2; p=0.001

Nintedanib52 w; n=700 and 500∆=2.05; p=0.0095

0

10

10

Δ Improvement

Δ Deterioration

2012

IMiDS are the only drug class that improves Quality of life

Page 25: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

DRUG FORMULATION FOR DIRECT LUNG DELIVERY

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IMiDs in amorphous form are loaded in nanopores of biodegradable microspheres

After lung deposition there is an immediate release of 30% and a sustained release of the remaining 70% as the microspheres degrade

Direct pulmonary delivery is expected to reduce systemic exposure by 50-85%

Page 26: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

VP02 – STATUS

PreclinicalEfficacy

Strong effects in models of IPFReduction of TNF and other cytokine release

Safety Systemic safety well knownLocal lung tolerability to be established

CMC Established production method for APIInhalation presentation in nanoporous microspheres ongoing, evaluating PK

Phase I Planned for 2020PK will determine probability of success

Clinical Tablet form with systemic exposure have shown dramatic effects on cough and quality of life, as well as on lung function

IPR Patent application for product filed in May 2018

Page 27: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

MARKET

Page 28: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

Price for one year’s treatment is close to $100,000

Combined global sales exceeded $2.3 billion in 2018

75% of the sales are in the US

Allied Market Research estimates annual sales of $3.6 billion in 2023

THE PULMONARY FIBROSIS MARKET

28

Global IPF sales by brand

$0

$250

$500

$750

$1 000

$1 250

2014 2015 2016 2017 2018

Mill

ion

Esbriet Ofev

Company reports, Bloomberg

Ofev will be launched 2019/20Global dSSc/ILD sales by brand

There are no drugs for IPF cough on the marketGlobal ILD/cough sales by brand

Page 29: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

FINANCIAL

Page 30: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

FINANCIAL INFORMATION

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Listed on Nasdaq Stockholm main market on Sep 27, 2019 STOCK

HealthCap VII L.P. 27.8%Göran Wessman 8.3%Swedbank Robur 6.3%Fourth Swedish National Pension Fund 4.9%HBM Healthcare Investments (Cayman) Ltd 4.6%Kjell Stenberg 3.6%Unionen 3.4%Pomona-gruppen AB 2.5%Shaps Capital 2.3%Alfred Berg 2.2%Handelsbanken Funds 2.1%Other 31.8%Total number of shares (42,374,714) 100%

LARGEST SHAREHOLDERS

June 30, 2019

SEK730 million (approx. €68 million)MARKET CAPSept 30, 2019

SEK193 million (approx. €18 million) CASH

June 30, 2019

Page 31: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

NEAR TERM TENTATIVE MILESTONES

Filing of CTA* for mechanistic SSc phase II study with C21 – October 1

Capsule formulation of C21 – October 1

Filing of CTA for PoC** in IPF with C21

Filing of CTA for open label extension

Selection of candidate formulation for VP02

Filing of CTA for phase I inhalation with IMiD

Progress in backup chemistry program

*Clinical Trial Application**Proof of Concept

Page 32: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

SUMMARY

Page 33: VICORE PHARMA...idiopathic pulmonary fibrosis (IPF), systemic sclerosis (SSc), IPF cough ... Former Head of Therapy Area Pain Control, AstraZeneca R&D 10 years of experience from senior

Vicore is well positioned to develop novel therapies for

fibrotic lung disease