VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING...

31
VICORE PHARMA A Rare Disease Company with a Focus on Patients with Fibrotic Lung Disease

Transcript of VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING...

Page 1: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

VICORE PHARMA

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

Page 2: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

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.

2

Page 3: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

Patients with fibrotic lung diseaseFOCUS

Addressing fibrosis and vasculopathy; improving cough and quality of lifeDIFFERENTIATION

Two clinical stage differentiated and complementary assets;VP01 – C21 (new modality) in phase II, VP02 – IMiD (reformulated) in preclinical development

PIPELINE

Listed on Nasdaq Stockholm main market since Sep 27, 2019 SHARE

Team of experienced drug developersPEOPLE

Idiopathic pulmonary fibrosis (IPF), IPF cough and systemic sclerosis (SSc)INDICATIONS

Unmet medical needs – every day mattersGUIDING PRINCIPLE

VICORE – AT A GLANCE

Existing shareholders include HealthCap (27%), Robur, AP4, Handelsbanken and HBM HealthcareSHAREHOLDERS

3

Page 4: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

THE TEAM

First class drug development team

4

Page 5: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

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 the University of Gothenburg

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

5

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

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

Page 6: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

FIBROTIC LUNG DISEASE

Rare diseases with large medical needs

6

Page 7: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

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 slows disease progression, but show significant side effects. Despite that, they reached sales of $2.3 billon in 2018

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

7

Page 8: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

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

8

Page 9: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

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)

9

Page 10: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

PIPELINE

Two phase II programs and one program with proven clinical efficacy

10

Page 11: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

VICORE PIPELINE

Page 12: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

VP01 (C21)

First in class small molecule AT2R agonist

12

Page 13: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

VP01 (C21)

13

Completed 2019; 200 mg daily is safe and well tolerated in man

Well tolerated in toxicology studies

Strong preclinical fibrosis data, pulmonary hypertension data and dilatation of resistance vessels

10,000 x higher affinity for AT2R compared with AT1R

First in class small molecule angiotensin II type 2 receptor (AT2R) agonist with ODD for IPF (EU, US)CLASS

SAFETY

PHASE I

PRECLINICAL

SELECTIVITY

Clinical trial application (CTA) to be filed early 2020Powered to pick up a functional differencePHASE II IPF (PoC)

Phase II ongoingMechanistic study to capture vascular effectsPHASE II SSc (PoM)

Established production method for C21Initially formulated as a solution, switch to capsule formulation in phase IICMC

Page 14: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

C21 MODE OF ACTIONC21 is the first small molecule angiotensin II type 2 receptor (AT2R) agonist and also a low affinity thromboxane receptor (TP) antagonist

C21 has dual mode of actions, both of which are relevant for IPF and SSc14

TP-receptor AT2-receptor

Fibrosis

VasocontractionInflammation

C21

+ -C21 is the first drug candidate to stimulate the angiotensin-receptor AT2R. At the same time, C21 has a blocking effect on the thromboxane receptor (TP)

These properties oc C21 counteract fibrosis, inflammation and vascular contraction, all of which contribute to the onset of IPF

Reduce/counteract

Page 15: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

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 SSc-NSIPControl

Total AT2r quantificationIn idiopathic pulmonary fibrosis (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 disease15

Page 16: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

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 PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

C21 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 PH17

Page 18: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

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

18

Page 19: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

VP02 (IMiD)

Effective therapy targeted directly to the lung

19

Page 20: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

IMiD (IMMUNOMODULATORY DRUG)

Includes pomalidomide, lenalidomide, and thalidomideCLASS

Clinical systemic side effects include sedation, sensory neuropathy and gastrointestinal (GI) effectsSAFETY

Local pulmonary deliveryPRESENTATION

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

Strong preclinical fibrosis data and a potent TNF inhibitorPRECLINICAL

Broad anti-inflammatory and antifibrotic actionSELECTIVITY

Inhalation presentation in nanoporous microspheres ongoing, evaluating PK. Phase I planned for late 2020. STATUS

20

Page 21: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

Heckmann 2016Rathinasabapathy 2018

Esbriet PamrevlumabOfev C21

IMiDs IN BLEOMYCINE IPF MOUSE MODEL

21

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 22: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

IMiDs ARE EFFECTIVE IN IPF COUGH

22

Reduction of cough is a unique feature of IMiDS

In a double-blind crossover study, thalidomide had a dramatic effect on cough frequency

Unlike other chronic cough, IPF do not respond to placebo treatment

0

20

40

60

80

100

End ofPeriod 1…

End ofPeriod 2…

Coug

h VA

S

p<0.001

0

20

40

60

80

100

End ofPeriod 1…

End ofPeriod 2…

Coug

h VA

S

2012

Page 23: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

IMiDS IMPROVE QUALITY OF LIFE IN IPF

23

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 in IPF

Page 24: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

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

24

Page 25: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

DRUG FORMULATION FOR DIRECT LUNG DELIVERY

25

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 PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

MARKET

26

Page 27: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

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

27

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

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

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

Page 28: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

FINANCIAL

28

Page 29: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

FINANCIAL INFORMATION

29

Listed on Nasdaq Stockholm main marketSTOCK

HealthCap VII L.P. 27.4%Göran Wessman 7.6%Swedbank Robur 6.6%Fourth Swedish National Pension Fund 6.4%HBM Healthcare Investments (Cayman) Ltd 4.8%Unionen 3.3%Kjell Stenberg 3.1%Pomona-gruppen AB 2.5%Shaps Capital 2.4%Länsförsäkringar 2.4%Handelsbanken Funds 2.2%Other 31.4%Total number of shares (50,174,714) 100.0%

LARGEST SHAREHOLDERS

Dec 31, 2019

SEK 605 million (approx. €57 million)MARKET CAPFeb 28, 2020

SEK265 million (€25 million) FINANCIAL POSTION

Dec 31, 2019

Page 30: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

SUMMARY

30

Page 31: VICORE PHARMAvicorepharma.com/wp-content/uploads/2020/03/vicore-pharma-mar... · FORWARD LOOKING STATEMENT This presentation may contain certain forward-looking statements and forecasts

Vicore is well positioned to develop novel therapies for

fibrotic lung disease