Vicore Pharma Holding AB · OMXSTO:VICO
Vicore Pharma Holding AB — Investor Presentation (January 2021)
45 slides from Vicore Pharma Holding AB's investor presentation, January 2021
Slide 1: Vicore Pharma AB
Vicore Pharma AB A Rare Disease Company with a Focus on Patients with Fibrotic Lung Disease January 2021
Slide 2: Forward looking statement
Forward looking statement This presentation may contain certain forward-looking statements and forecasts based on uncertainty, since they relate to events and depend on circumstances that will occur in the future and which, by their nature, will have an impact on Vicore Pharma’s business, financial
Slide 3: Vicore – at a glance
Vicore – at a glance Focus Patients with fibrotic lung disease Pipeline Two differentiated assets with 3 phase II studies lined up addressing >$1bn markets Indications Idiopathic pulmonary fibrosis (IPF), IPF cough, systemic sclerosis (SSc) and COVID-19 Differentiation Addressing fibrosis, inflammat
Slide 4: Pipeline
Pipeline Two programs with proven clinical efficacy 4
Slide 5: Pipeline
Pipeline Preclini Project Indication Phase I Phase II Phase III Next event cal Idiopathic Reduced TGF upregulation in IPF lung Pulmonary slices. Started to enroll patients in Q4 Fibrosis (IPF) 2020 Raynaud’s Mechanistic phase II study determining phenomena/ effects on resistance vessels. Top-line VP
Slide 6: C21 (VP01 project)
C21 (VP01 project) First-in-class with unique mechanism of action 6
Slide 7: C21 A first-in-class drug
C21 A first-in-class drug, A first in classstimulating the body's drug, stimulating repair the body's system, repair reducing tissue system, damage reducing anddamage tissue promoting healing healing and promoting 7
Slide 8: Overview of the renin-angiotensin system
Overview of the renin-angiotensin system The RAS system is target for blockbuster drugs 8
Slide 9: C21 – mode of action
C21 – mode of action C21 is a first in class small molecule stimulating the angiotensin II type 2 receptor Angiotensin II has 2 receptors with opposite actions Vasoconstriction Vasodilation Inflammation Anti-inflammation Fibrosis Anti-fibrosis In disease ANG II binding to AT1R dominates C21 countera
Slide 10: C21 First-in-class anti-fibrotic for IPF
C21 First-in-class anti-fibrotic for IPF 10
Slide 11: IPF - a rare interstitial pulmonary disease with unknown etiology
IPF - a rare interstitial pulmonary disease with unknown etiology Male predominance Alveolus in pulmonary hypertension Scarring • 250,000 in US and EU Congested, enlarged Progressive disease vessels Enlarged with • 3-5 years life expectancy increased pressure • Loss of lung function Deformation Alve
Slide 12: C21 in bleomycin fibrosis treatment model
C21 in bleomycin fibrosis treatment model Reduction of fibrosis compared to competitors Esbriet Ofev Pamrevlumab C21 50mg/kg 60mg/kg 30mg/kg 0.03mg/kg -1% -10% -41% -45% Heckmann 2016 Rathinasabapathy 2018 Esbriet: pirfenidone, Roche; Ofev: nintedanib; B-1; pamrevlumab, anti-CTGF C21 reduces fibrosi
Slide 13: Robust effects in human IPF tissue
Robust effects in human IPF tissue Effects of C21 on TGFb1, a master regulator of fibrosis, in IPF lung tissue Vicore, data on file Vicore, data on file C21, at clinical concentrations, attenuate TGFb1 protein and mRNA expression 13
Slide 14: Effect on PH in the rat Sugen-hypoxia model
Effect on PH in the rat Sugen-hypoxia model The Sugen model is gold standard for pulmonary hypertension • Long-term model with vascular remodeling C21 has a unique profile • Reduces vascular remodeling • Thereby reduces pulmonary artery pressure • And improves cardiac function Vicore, data on file C
Slide 15: C21 product development
C21 product development Product protection Non-clinical safety CMC • Orphan drug designation and • 3-month toxicology in rat, dog, • Developed as an immediate- formulation patent applications non-human primate release dry capsule Phase I Phase II • 3 phase I studies up to 200 mg bid • Positive phase
Slide 16: IPF study design
IPF study design A phase II, multicenter, open-label, single-arm trial investigating the safety, efficacy and pharmacokinetics of C21 in 60 subjects with IPF Treatment Treatment Treatment Screen period 1 period 2 period 3 FU 4 weeks 4 weeks 12 weeks 12 weeks 12 weeks Safety Efficacy Efficacy • Prima
Slide 17: Impact of treating early in IPF
Impact of treating early in IPF C21 targets a 67% reduction in FVC decline • In an untreated patient, mild disease will progress to severe in 7 years • With treatment – the patient will remain in mild disease Lung volume (L) 5 Mild: 80-100% normal 4 Reduction by age 67% reduction in mild IPF Moderat
Slide 18: C21 positioning in IPF
C21 positioning in IPF First line Standard of care Second line Add on to standard of care pirfenidone Third line C21 nintedanib Standard of care GLPG 1690 failures PRM 151 TD-139 pamrevlumab With efficacy data on par or better than SoC and safety data superior to SoC, C21 can challenge pirfenidone a
Slide 19: VP03 – C21 follow-up compounds
VP03 – C21 follow-up compounds Rationale Properties • C21 only has orphan drug • Similar receptor designation for product selectivity protection (7-10 years) as C21 Chemistry • Improved properties • There is a limited space Status for novel chemistry • Initiating scale-up chemistry • New proprietary
Slide 20: C21 First-in-class anti-fibrotic for pulmonary SSc
C21 First-in-class anti-fibrotic for pulmonary SSc 20
Slide 21: Pulmonary fibrosis in systemic sclerosis
Pulmonary fibrosis in systemic sclerosis SSc is a rare autoimmune disease with heterogeneous clinical manifestations Female predominance Oral: Pulmonary: • 60,000 in US and EU Xerostomia Reduced aperture Interstitial lung disease Pulmonary artery hypertension Mucocutaneous telangiectasia Cardiac: Up
Slide 22: SSc/Raynaud’s phenomenon study design
SSc/Raynaud’s phenomenon study design A phase II, double-blind, crossover trial investigating the safety and effects on acute blood flow regulation of C21 in 12 subjects with SSc Screen Treatment Washout Treatment FU 2 weeks Active or placebo 2 weeks Placebo or active 2 weeks • Patients with SSc hav
Slide 23: C21 A novel treatment for COVID-19
C21 A novel treatment for COVID-19 23
Slide 24: COVID-19
COVID-19 COVID-19 is SARS-CoV-2 respiratory infection • It primarily affects the upper airways • Dry cough and fever are the main initial symptoms • After a few days, some patients deteriorate rapidly • The disease changes from an upper respiratory tract infection to involve the lungs as well • Alve
Slide 25: The CoV-2 virus creates an imbalance in the RAS
The CoV-2 virus creates an imbalance in the RAS The virus hijacks the enzyme ACE2 for cellular entry and triggers an imbalance in RAS leading to a cytokine storm in the lungs (ARDS) • Direct stimulation of AT2R may bypass the incapacitated ACE2 enzyme to counteract the inflammatory drive C21 bypasse
Slide 26: C21 in COVID-19 study design
C21 in COVID-19 study design A randomized, double-blind, placebo-controlled phase II trial investigating the efficacy of VP01 in COVID-19 infection VP01 100 mg BID n=50 FU Screen Randomization 1 week Placebo n=50 • The study will measure disease progression by the means of C-reactive protein, diseas
Slide 27: COVID-19 study results
COVID-19 study results Demographics • The treatment groups were well balanced with regard to baseline and treatment including corticosteroid regimens Oxygen supplementation • There was a statistically significant reduced risk of being on oxygen after treatment was stopped and this risk reduction inc
Slide 28: Reduced need of oxygen support
Reduced need of oxygen support 40 Number of patients being on oxygen 39 Placebo 37 30 C21 p=0.057 25 p=0.014 20 18 p=0.002 10 14 11 8 1 Day 1 Day 7 Day 8 Day 14 C21 gradually lowered the risk for patients needing oxygen supplementation 28
Slide 29: VP02 Inhaled IMiD for IPF cough
VP02 Inhaled IMiD for IPF cough 29
Slide 30: VP02 IMiD inhalation
VP02 IMiD inhalation A clinically proven drug in IPF and IPF cough, formulated for inhalation to avoid systemic side effects 30
Slide 31: IPF cough
IPF cough IPF cough is a debilitating symptom • A severe dry cough up to 50 times/hour • Affects up to 40% of IPF patients • Correlates with morbidity and mortality • The single symptom with highest impact on quality of life • No effective treatment exists (eg anti-tussives) “Debilitating is the bes
Slide 32: VP02 – Clinical evidence with IMiDs in IPF
VP02 – Clinical evidence with IMiDs in IPF Maureen Horton Toby Maher Baltimore London • Double-blind, placebo-controlled, crossover study • Treated individual cases • Significant reduction of cough • Improve quality of life Lin Zhang Xi’an • Open-label, placebo- controlled study • Significant improv
Slide 33: Oral thalidomide significantly improves IPF cough and quality of life
Oral thalidomide for 3 months significantly improves IPF cough and quality of life Cough VAS Quality of Life scores 100 15 Δ Deterioration Δ Improvement 10 80 5 60 VAS 0 40 -5 20 -10 0 -15 0 3 Thalidomide Months 12 w; n=20 D=11.2; p=0.001 • The systemic dosing gives rise to side effects such as peri
Slide 34: VP02 – the principle of targeted delivery
VP02 – the principle of targeted delivery Inhaled Systemic Local delivery is well tolerated Systemic delivery is poorly with low systemic exposure tolerated with high risk and improved safety of side effects Drug delivered directly to the lungs, reduces the risk of adverse effects 34
Slide 35: Novel drug formulation for direct lung delivery
Novel drug formulation for direct lung delivery IMiDs in amorphous form are loaded in Microspheres are nanopores of perforated allowing biodegradable rapid release of dry microspheres powder inhalation Microspheres have After lung consistent size and deposition there form (3mm) is an immediate relea
Slide 36: Direct lung delivery
Direct lung delivery IMiD concentration in awake rat after intra-tracheal administration NLAB Silica particles give a 10-20 times difference between local and systemic exposure 36
Slide 37: Phase I IMiD Inhalation
Phase I IMiD Inhalation A phase I, single ascending and multiple ascending trial investigating the safety and pharmacokinetics of inhaled IMiD in healthy subjects SAD SAD SAD SAD SAD SAD dose 1 dose 2 dose 3 dose 4 dose 5 dose 6 MAD MAD MAD MAD dose 2 dose 3 dose 4 dose 5 • Start the single ascendin
Slide 38: The team
The team First class drug development team 38
Slide 39: The Vicore Team
The Vicore Team CARL-JOHAN DALSGAARD, CEO HANS JEPPSSON, CFO • MD, PhD, Karolinska Institute with post-doc experience from Harvard • PhD, Finance with post-doc research at UC Berkeley • Former Head of Therapy Area Pain Control, AstraZeneca R&D • Cross-disciplinary background in finance and medicine
Slide 40: Board of Directors
Board of Directors MICHAEL WOLFF JENSEN, CHAIRMAN OF THE BOARD SARA MALCUS, BOARD MEMBER • Member since 2020 • Member since 2018 • Law degree from University of Copenhagen • PhD in Immunology and Inflammatory Medicine at University of • Chief legal officer at Ascendis Pharma Gothenburg • 20 years of
Slide 41: Scientific Advisors
Scientific Advisors PROF. JOANNA PORTER PROF. TOBY MAHER • Clinical expert • Clinical expert • Joanna Porter is a Professor in the Department of Medicine at UCL • Toby Maher is British Lung Foundation Chair in Respiratory Research and a Consultant in Respiratory and General Medicine. She is the and
Slide 42: Financial information
Financial information ycore pharma 42
Slide 43: The pulmonary fibrosis market
The pulmonary fibrosis market Global IPF sales by brand $1 700 • Combined global sales exceeded 2.8 BUSD $1 450 in 2019* $1 200 • Price for one year’s treatment is close to $100,000 Million $950 $700 • 75% of the sales are in the US • Thelansis estimates the market for IPF in $450 8MM to reach 4.3 B
Slide 44: Financial information
Financial information FINANCIAL POSITION 361 MSEK (35 MEUR) Sep 30, 2020 STOCK Listed on Nasdaq Stockholm main market MARKET CAP Approx. 1,900 MSEK (190 MEUR) Dec 31, 2020 HealthCap VII L.P. 25.9% Swedbank Robur 9.9% Fourth Swedish National Pension Fund 7.5% Göran Wessman 7.2% HBM Healthcare Investm
Slide 45: Vicore is well positioned to develop novel therapies for fibrotic lung disease
Vicore is well positioned to develop novel therapies for fibrotic lung disease 45