Slide 1: Development Strategy Update 2023-2025
Development Strategy Update 2023-2025 Fate can be altered 29 June 2023
Slide 2: Experienced company leadership
Experienced company leadership Marcin Szumowski Sławomir Broniarek Nicolas Beuzen Chairman of the Board & CEO Chief Financial Officer Business Development Director R&D leaders Samson Fung Zbigniew Zasłona Barbara Dymek Chief Scientific Officer R&D Operations Director Chief Medical Officer 29/06/2023
Slide 3: Development Strategy Update for 2023-2025
Development Strategy Update for 2023-2025 2 assets in clinical trials Progress in the development of current programs and expansion of new early stage projects AI invaids biotechnology Main strategic BD goal: Expected partnering for at least one of clinical programs and one major partnering transact
Slide 4: Development areas as a foundation for future growth
Development areas as a foundation for future growth Clinical progression of two most advanced assets, OATD-01 and OATD-02 Machine learning and Generative Artificial Intelligence (GenAI)-driven drug discovery platform for small molecule drugs modulating the function of mRNA and previously unexplored
Slide 5: Strategic goals for 2023-2025
Strategic goals for 2023-2025 1. Clinical development of OATD-01 to demonstrate therapeutic efficacy (a Phase II clinical proof-of-concept) in patients with pulmonary sarcoidosis (final report in mid-2025). Determining the safety, tolerability and maximum tolerated dose (MTD) and the Phase II recomm
Slide 6: Balanced pipeline as a trigger for commercialization
Balanced pipeline as a trigger for commercialization Current status Clinical Candidate 2025 estimate Selection Molecular Target Lead In Vivo Preclinical Regulatory Target Indications Validation Optimization POC Development Process Phase I Phase II Hit-to-lead PROTEIN TARGETS OATD-01 Sarcoidosis, oth
Slide 7: OATD-01: Disease modifying potential in sarcoidosis
OATD-01: Disease modifying potential in sarcoidosis 29.06.2023 7
Slide 8: OATD-01 | a macrophage modifier
OATD-01 | a macrophage modifier CHIT1 plays a key role in macrophages biology OATD-01 is a potent Chitotriosidase (CHIT1) inhibitor OATD-01 enters Phase II PoC in sarcoidosis this year, MoA could transform treatment of inflammatory & fibrotic diseases 29.06.2023 8
Slide 9: OATD-01 efficacious in granulomatous inflammation model in mice
OATD-01 | is efficacious in Multiwalled carbon nanotube-induced granulomatous inflammation model in mice OATD-01 suppressed development of organized granulomatous structures in lungs and reduced macrophage inflammation protein (CCL4 in the broncho-alveolar lavage fluid - BALf) in the lungs 40 days p
Slide 10: OATD-01 attenuates lung fibrosis in mice
OATD-01 | attenuates lung fibrosis in mice Bleomycin OATD-01 Pirfenidone Control (2 U/kg) (30 mg/kg, b.i.d) (250 mg/kg, b.i.d) OATD-01 reduces features of pulmonary fibrosis in bleomycin induced model B C D Control Bleomycin OATD-01 Pirfenidone Control Bleomycin OATD-01 Pirfenidone Control Bleomycin
Slide 11: OATD-01 efficacious in multiple models of NASH in mice and rats
C on tr ol (n NAFLD activity score 0 2 4 6 o di se ST as A Controle) M (no disease) STAM + ST O A 29.06.2023 A M TD ✱✱✱✱ ST ve A -0 1 . h M + 30 STAM veh. O m A and rats TD g/ kg ✱✱✱ -0 1 + OATD-01 STAM qd 1030 mg/kg qd 0 ✱✱✱✱ m g/ kg STAM + OATD-01 qd 100 mg/kg qd N W D NAS score [scale 0-9] 0 2 4
Slide 12: OATD-01 | Pre-clinical and phase I data support further development
OATD-01 | Pre-clinical and phase I data strongly support further development Compelling Data • Efficacy proven in multiple animal models • Convincing translational data • Excellent pharmacological profile • Demonstrated safety in animals and humans (129 subjects exposed) High unmet need in sarcoidos
Slide 13: OATD-01 significant market opportunity for disease-modifying profile
OATD-01 | significant market opportunity for disease-modifying profile Global sarcoidosis treatment sales with current treatment options are estimated at about US$188m* as steroids and other immunosuppressive drugs are inexpensive However, the market is likely to completely change in the coming year
Slide 14: OATD-01 | Phase 2 in sarcoidosis
OATD-01 | Phase 2 in sarcoidosis Double-blind, randomized, placebo-controlled multi- center study to assess the safety and efficacy of an oral inhibitor of CHIT1 (OATD-01) in patients with active pulmonary sarcoidosis. *IND *CTA *FDA & EMA* Intermediate Results *FPFV *LPLV and *CSR 20 to 30 submissi
Slide 15: OATD-02: A first in class dual arginase inhibitor for cancer applications
OATD-02: A first in class dual arginase inhibitor for cancer applications 15
Slide 16: OATD-02 | first in class dual ARG1-ARG2 inhibitor
OATD-02 | first in class dual ARG1-ARG2 inhibitor Molecure is the only company offering a dual arginase (ARG) inhibitor with high intracellular activity Highly potent, orally active dual ARG1/ARG2 OATD-02 inhibitor with intracellular activity Predicted favorable human Restored arginase-suppressed ph
Slide 17: OATD-02 exhibits dose-dependent therapeutic effect in multiple tumor models
OATD-02 | exhibits dose-dependent therapeutic effect in a multiple tumor models Significant dose-dependent effect was demonstrated in multiple models (here CT26) Syngeneic CT26 model of colorectal carcinoma OATD-02 in tumors Arginine in tumors (nmol/g, mean with SEM) 20 1000 (nmol/g, mean with SEM)
Slide 18: OATD-02 improved antitumour efficacy of immune-checkpoint inhibitors
OATD-02 | improved the antitumour efficacy of immune-checkpoint inhibitors OATD-02 enhanced the efficacy of anti-PD-1 antibody OATD-02 significantly improved the efficacy of anti-PD-1 antibody from TGI 64% to 87% (p = 0.0450) OATD-02 prolonged the life of tumour-bearing animals by 5 days in monother
Slide 19: OATD-02 | Addressing cancer with unmet need
OATD-02 | Addressing cancer with unmet need Pancreatic ductal cancer (advanced, inoperable) • current standard treatments comprise chemotherapy combinations (taxanes, gemcitabine, platinum derivates, irinotecan) • ICI* (e.g., Keytruda, Opdivo, Yervoy) have not yet demonstrated significant efficacy •
Slide 20: OATD-02 | Phase I clinical trial
OATD-02 | Phase I clinical trial Design: Open-label single-arm Patient population (30-40 pts): dose-escalation monotherapy Relapsed/refractory advanced study (Bayesian design, 2.5-30mg) and/or metastatic solid tumors Colorectal cancer, platinum- resistant serous ovarian cancer, pancreatic ductal can
Slide 21: OATD-02 administered to the second cohort of patients
OATD-02 | administered to the second cohort of patients Sixth cohort Min. 3-6 patients Fifth cohort Min. 3-9 patients 30 mg Fourth cohort Min. 3-9 patients 25 mg Third cohort 71-85 week for 6 pts Min. 3 patients 2 weeks for SRC 20 mg Second cohort 48-69 week for 9 pts Min. 3 patients 2 weeks for SRC
Slide 22: A promising pre-clinical pipeline
A promising pre-clinical pipeline 22
Slide 23: USP7 inhibitor | T cell modifier with proven anti-cancer potential
USP7 inhibitor | T cell modifier with proven anti-cancer potential Validated translational potential • Deubiquitinating enzymes (DUBs) reverse the post translational modification of proteins by removing ubiquitin • Enhanced expression of DUBs can be seen in both cancer and some inflammatory states M
Slide 24: mRNA Platform | discovering medicines of the future
mRNA Platform | discovering medicines of the future Small molecules targeting RNA to prevent downstream RNA translation BOUND SMALL MOLECULE GENE OF INTEREST 2D RNA STRUCTURE - NO TRANSLATION - UTRs of RNA OF DRUGGABLE REGIONS 3D STRUCTURE NO PATHOLOGICAL PROTEIN STRUCTURAL BIOINFORMATICS OMIC DATA
Slide 25: Our approach
Our approach • Selection of functional regions of mRNA for undruggable protein targets • Identify locally stable and robustly folded sequences in the functional regions of the RNA of interest with the potential to form pockets accessible by small molecules • These secondary structures are not influe
Slide 26: AI-driven drug discovery | following market trends
AI-driven drug discovery | following market trends Early signals that AI companies increase by 2025 development efficiency Biopharma target to validated lead ~50% ~24-36 months o reduction of time and cost from therapeutic target validation to High value Leading AI companies 40-60% clinical candidat
Slide 27: Commercialization and milestones
Commercialization and milestones 29.06.2023 27
Slide 28: Commercialization models
Commercialization models Partnering – main form of revenue generation Program Preffered potential models and assumptions o exclusive global license for further development and commercialization $/$$ Clinical stage assets o development the 2nd asset through Ph2 PoC when first asset commercialized $
Slide 29: Summary of milestones
Summary of milestones Potential milestones targeted by 2025 Protein Targets RNA Platform BD/Financial/Operational o Two clinical assets in PhI/II o In vitro PoC reached for o At least one clinical stage multi center clinical trials in 2-3 high value mRNA program partnered in patients targets a high-
Slide 30: Finance
Finance 29.06.2023 30
Slide 31: Planned expenditures to fuel growth | H2 2023-2025
Planned expenditures to fuel growth | H2 2023-2025 In total ~PLN 250 m (USD 62,5 m*) Clinical phase programs ~PLN 100 m | ~USD 25 m* Discovery and preclinical development programs ~PLN 75 m | ~USD 19 m* mRNA platform (including AI tools) ~PLN 25 m | ~USD 6 m* General, administrative & BD expenses ~P
Slide 32: Financing strategy
Financing strategy Molecure assumes that the implementation of the updated strategy will be financed from: • Existing cash and cash equivalents (approx. PLN 50 m SD 12,5 m**, estimated for June 30, 2023). • Existing (already granted) and future non-dilutive grants and subsidies (at least PLN 75 Fina
Slide 33: Molecure | leading the vibrant Polish biotech sector
Molecure | leading the vibrant Polish biotech sector 7 small molecules aimed at modulating Catalyst rich novel and attractive period ahead targets Highly effective in Efficient drug repeatedly progressing discovery with early-stage assets into limited cash burn clinical development 29.06.2023 33
Slide 34: Molecure SA makes no warranties or assurances regarding accuracy
Molecure SA (”MOC") 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 situation as at t