Molecure Sa · GPW:MOC
Molecure Sa — OncoArendi Therapeutics — Investor Presentation (November 2021)
40 slides from Molecure Sa's investor presentation, Nov 4, 2021
Slide 1: Investor & Analyst Presentation
INVESTOR & ANALYST PRESENTATION November 2021
Slide 2: Disclaimer
OncoArendi Therapeutics SA ("OAT") and its Board and employees make no warranties or assurances regarding the accuracy or completeness of any representations or other materials contained herein. The information and data contained herein reflect the current conditions and assessments of the market si
Slide 3: Company Leadership
COMPANY LEADERSHIP MARCIN SŁAWOMIR SZUMOWSKI BRONIAREK PhD, MBA BOARD MEMBER, CFO CHAIRMAN OF THE BOARD & CEO More than 20 years of experience in financial Entrepreneur and investor with 20-year experience in markets, including the investment sector the life science industry, including 2 successful
Slide 4: R&D Leaders
R&D LEADERS PhD MARTA PhD NANCY EXPERT DRUG DUDEK MEDICAL DIRECTOR VAN OSSELAER DEVELOPMENT Clinical oncologist experienced in chemotherapy of solid Clinical and preclinical pharmacologist with 25 years of tumors and hematological malignancies. In parallel to scientific, people and project managemen
Slide 5: Company Overview
COMPANY OVERVIEW A biopharma company focused Our mission is to challange current Listed on Warsaw Stock Exchange; on discovering and developing medical solutions to treat patients new drugs for cancer, fibrotic with uncurable diseases 50M USD raised to date; and inflammatory diseases +33M USD in par
Slide 6: Our Vision
OUR VISION WE WILL BE THE MOST RECOGNIZED POLISH BIOTECHNOLOGY COMPANY WORLDWIDE DISCOVERING, DEVELOPING AND COMMERCIALIZING BREAKTHROUGH SMALL MOLECULE DRUGS OF THE FUTURE INTERACTING WITH NOVEL PROTEIN & RNA TARGETS
Slide 7: Investment Highlights
INVESTMENT HIGHLIGHTS A diversified pipeline in immuno-oncology with lead Phase I ready OATD-02 RNA targeting discovery platform with disruptive potential Breakthrough innovation fueled by academic and industrial partnerships worldwide Innovative science validated by high value partnering deal (Ph I
Slide 8: Three Engines of Growth
THREE ENGINES OF GROWTH OATD-02 leading a diversified pipeline RNA - Collaboration targeting with discovery engine 8
Slide 9: Current Pipeline
CURRENT PIPELINE MOLECULAR TARGET LEAD IN VIVO CLINICAL PRECLINICAL REGULATORY TARGET INDICATIONS VALIDATION OPTIMIZATION POC CANDIDATE DEVELOPMENT PROCESS PHASE I PHASE II HIT-TO-LEAD SELECTION PROPRIATORY PIPELINE OATD-02 IMMUNO- OATD-02 ARG1/ARG2 ONCOLOGY 2ND GENERATION IMMUNO- OATD-02 ONCOLOGY (
Slide 10: Recent Activities
RECENT ACTIVITIES Selected the CRO for performing the FIH multi-center study with OATD-02 Selected 30+ non-druggable clinically validated mRNA targets for modeling Discovered an early lead molecule in the USP-7 discovery program Initiated two new discovery programs (CHIT1 selective & USP-XX) Analyse
Slide 11: Growth Catalysts (2022) and Anticipated Milestones
GROWTH CATALYSTS (2022) AND ANTICIPATED MILESTONES First patients dosed in OATD-02 multi-center first-in-human trial mRNA Discovery Platform PoC + new multi-target antiviral program Exclusive license for the bio-informatics tools for in-silico screening & modeling Clinical candidate selection in the
Slide 12: OATD-02 Program
OATD-02 PROGRAM Current value driver 12
Slide 13: OATD-02 Is the First in Class Dual ARG1-ARG2 Inhibitor
OATD-02 IS THE FIRST IN CLASS DUAL ARG1-ARG2 INHIBITOR ARG1 and ARG2, have been validated as promising therapeutic targets for cancer, affecting both tumor immunity and metabolism. OAT is the only company offering arginase inhibitors with high intracellular activity proven in the preclinical develop
Slide 14: Competitive Landscape Demonstrates Our Therapeutic Target Is Attractive
COMPETITIVE LANDSCAPE DEMONSTRATE OUR THERAPEUTIC TARGET IS ATTRACTIVE AND VERY WELL VALIDATED 3 active competitors in Oncology with small molecules: • Incyte / Calithera INCB-001158 – Phase II • AstraZeneca AZD-0011 – Preclinical • MSD – Discovery 2 companies developing small molecule arginase inhi
Slide 15: OATD-02 Showed a Superior Efficacy Over INCB-001158
OATD-02 SHOWED A SUPERIOR EFFICACY OVER INCB-001158 IN COLORECTAL AND AGGRESSIVE KIDNEY CANCER MODELS Final tumor weight Syngeneic CT26 model Renca syngeneic model Regulatory T lymphocytes of colorectal carcinoma Foxp3+ within CD4+ T cells ✱ 2500 TGI TGI Vehicle 4000 40 (mm3, mean with SEM) 27% -3%
Slide 16: OATD-02 Can Directly Target Cancers Whose Metabolism Depend on ARG2
OATD-02 CAN DIRECTLY TARGET CANCERS WHOSE METABOLISM DEPEND ON ARG2 K562 xenograft model of human chronic myeloid leukemia high ARG2 expression 1750 Arg2 has low expression in healthy (mm3, mean with SEM) 1500 individuals and is highly increased in Tumor volume 1250 multiply cancers such as: AML, Gl
Slide 17: OATD-02 Has Potential for a Higher Therapeutic Window
OATD-02 HAS POTENTIAL FOR A HIGHER THERAPEUTIC WINDOW COMPARED TO INCB-001158 Similar low activity High activity important for therapeutic efficacy towards liver cells Inhibition of Inhibition of Inhibition of Inhibition of the urea cycle human ARG1 human ARG2 immunosuppressing macrophages in human
Slide 18: Superiority of OATD-02 Compared to INCB-001158
SUPERIORITY OF OATD-02 COMPARED TO INCB-001158 Biological effect INCB-001158 OATD-02 Extracellular activity hARG1 80 nM hARG1 20 nM Low/none Tregs, ARG2 dependent cancer cells, tumor Intracellular Activity associated fibroblasts Efficacy in syngeneic models average high Efficacy in xenograft models
Slide 19: OATD-02 First Dual Arginase Inhibitor On-Track to Reach Cancer Patients
OATD-02 FIRST DUAL ARGINASE INHIBITOR ON-TRACK TO REACH CANCER PATIENTS We achieved competitive advantage in drug-like parameters and mechanism of action: • favorable therapeutic window of OATD-02 with improved safety and tolerability • better infiltration in TME enhancing therapeutic efficacy • pos
Slide 20: OATD-02 Proposed Clinical Set-up
OATD-02 PROPOSED CLINICAL SET-UP An open-label dose-escalation monotherapy study in patients with selected advanced metastatic solid tumors: colorectal cancer, platinum-resistant serous ovarian cancer, pancreatic ductal cancer, renal cell carcinoma, relapsed/refractory acute myeloid leukaemia Tumor-
Slide 21: OATD-02 Clinical Study Endpoints and Design
OATD-02 CLINICAL STUDY ENDPOINTS AND DESIGN Bayesian Optimal Interval (BOIN design): based on pre-defined number of patients Primary endpoints: Dose escalation safety, tolerability, /de-escalation flexibility DLT & MTD Secondary endpoints: Excretion of orotic acid - PK/PD, PAD as biomarker of urea -
Slide 22: OATD-02 Take-Home Messages
OATD-02 TAKE-HOME MESSAGES High intracellular activity (ARG2) First-in-class potential confirmed in preclinical development The first nanoliposomal encapsula Next generation approach to ted OATD-02 drug form obtained maximize the value of the program 22
Slide 23: USP-7 Program
» OncoArend ‘Therapeutics USP-7 PROGRAM
Slide 24: OAT-4828 Is Our Newly Discovered Lead USP7 Allosteric Inhibitor
OAT-4828 IS OUR NEWLY DISCOVERED LEAD USP7 ALLOSTERIC INHIBITOR The project is at the Lead Optimization Stage In vitro profile of OAT-4828: • Good activity in biochemical (IC50< 30nM) and cellular assays (EC50<350nM (SW480) • Highly selective vs other DUBs (USP47, USP2, USP15, USP21, USP9x) • hERG>
Slide 25: USP7 Allosteric Inhibitors Are Tested for Immunomodulatory Properties
USP7 ALLOSTERIC INHIBITORS ARE TESTED FOR THEIR IMMUNOMODULATORY PROPERTIES Enzymatic tests O A T -4 8 2 8 in h ib itio n o f W N T / c a t e n in 5 [R L U ] E C 5 0 0 .3 3 [ M ] 0 d e c re a s e Cellular assays -5 T O P /F O P -1 0 -1 5 0 .0 0 1 0 .0 1 0 .1 1 1 0 1 0 0 WE ARE HERE MoA experimen
Slide 26: YKL-40 Program
» OncoArend ‘Therapeutics YKL-40 PROGRAM
Slide 27: OAT-3912 Advanced Lead Compound with Drug-Like Properties
OAT-3912 ADVANCED LEAD COMPOUND WITH DRUG-LIKE PROPERTIES • Selective towards other chitinase enzymes • PK properties allowing to study our lead compound in the context of immuno-oncology • No adverse effects observed • Nanomolar activity towards murine and human proteins 27
Slide 28: Current Activities Aim at Understanding Biology of YKL-40 and OAT-3912
CURRENT ACTIVITIES AIM AT UNDERSTANDING BIOLOGY OF YKL-40 AND OAT-3912 Preliminary data shows efficacy in animal model of cancer and now we focus on MoA to better choose specific therapeutic indication Our recent data and current literature confirms that OAT-3912 has highest chances of positively mo
Slide 29: Discovery Platform of Future Medicines
DISCOVERY PLATFORM OF FUTURE MEDICINES Future value driver 29
Slide 30: Major Breakthroughs in Drug Discovery
MAJOR BREAKTHROUGHS IN DRUG DISCOVERY 2021 COVID-19 Vaccine 2020 Evrysdi 2017 Luxturna RNA technology 2017 Kymriah 2014 Keytruda 2002 Humira 1998 Herceptin 1995 Saquinavir 1928 Penicillin 1921 Insulin 1899 Aspirin 30
Slide 31: Discovering Medicines of the Future: Small Molecules Targeting RNA
DISCOVERING MEDICINES OF THE FUTURE: SMALL MOLECULES TARGETING RNA 31
Slide 32: Revolution in Medicine
REVOLUTION IN MEDICINE The number of potential therapeutic targets (proteins) for traditional small molecules is limited Targeting RNA offers access to thousands of new therapeutic targets for future medicines 32
Slide 33: Targeting RNA Is a New Strategic Direction for OncoArendi
TARGETING RNA IS A NEW STRATEGIC DIRECTION FOR ONCOARENDI I NATURAL SYNERGY • OncoArendi’s expertise in the development of small molecules • World-class RNA-focused research teams in Poland • Exclusive partnership & research collaboration agreement with the International Institute of Molecular and C
Slide 34: Access to a Unique Bioinformatics Platform
ACCESS TO A UNIQUE BIOINFORMATICS PLATFORM OUTPERFORMING STANDARD METHODS USED IN DISCOVERY OF DRUGS TARGETING RNA Exclusive collaboration with International Institute of Molecular and Cell Biology in Warsaw PROF. JANUSZ BUJNICKI • > 18,000 citations, h-index > 70 • Among TOP 2% of all scientists in
Slide 35: Exclusive Collaboration with the International Institute of Molecular and Cell Biology
EXCLUSIVE COLLABORATION WITH THE INTERNATIONAL INSTITUTE OF MOLECULAR AND CELL BIOLOGY IN WARSAW – SMALL MOLECULES TARGETING RNA >20 confirmed hit compounds screening results of > 100,000s small molecules Q2 2023 3 targets selected for structural analysis Q2 2022 30 targets selected for computationa
Slide 36: Financial Results
» OncoArend ‘Therapeutics FINANCIAL RESULTS
Slide 37: Financial Results Q1-Q3 2021
FINANCIAL RESULTS Q1-Q3 2021 PLN m Q1-Q3 2020 Q1-Q3 2021 R&D spending Revenue 1,34 0,87 Cash position Oct 31st PLN 12,4m Costs 3,55 7,82 PLN 107m (~$27m) Sold projects costs 0,00 2,31 of which grants Financing available until New started projects 0,00 0,46 PLN 4m EBITDA -2,09 -6,83 end 2023* CAPEX 2
Slide 38: Summary
SUMMARY On the aniversary of the deal with Galapagos, after a year of investments in human resources, infrastructure and IT tools, along with engagement in new key research collaborations, OncoArendi is positioned for fast expansion and progression of its pipeline through in-licensing and internal g
Slide 39: Better Medicines Inspired by Science
BETTER MEDICINES INSPIRED BY SCIENCE
Slide 40: Glossary
GLOSSARY Arg –Arginase enzymes 1 nad 2 – our biological target for OATD-02 PK - pharmacokinetic - studies performed to determine how compound is metabolized, distributed and cleared in our bodies IND - Investigational New Drug CT 26 - a murine colorectal carcinoma cell line Renca – a murine renal co
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