Molecure Sa · GPW:MOC
Molecure Sa — Investor Presentation (April 2024)
29 slides from Molecure Sa's investor presentation, Apr 10, 2024
Slide 1: Fate can be altered
Fate can be altered April 10, 2024
Slide 2: Presenting Team
Presenting Team Marcin Szumowski Zbigniew Zasłona Chairman of the Board & CEO Chief Scientific Officer Samson Fung Sławomir Broniarek Chief Medical Officer Chief Financial Officer 10.04.2024 2
Slide 3: Summary | Molecure on the path of continued growth
Summary | Molecure on the path of continued growth OATD-01: FDA and MHRA approval for OATD-02: Ph1 clinical Implementation of Ph2 clinical trial, new application for trial: progression to 20 mg AI tools in drug approval in the countries of the EU and cohort. Increase in PD discovery; First AI- Norwa
Slide 4: Molecure | Balanced pipeline
Molecure | Balanced pipeline 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, other OATD-01 OATD-01 CSR*
Slide 5: OATD-01: Disease modifying potential in sarcoidosis
OATD-01: Disease modifying potential in sarcoidosis 10.04.2024 5
Slide 6: 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. Central Bioethic Application FPFV (US/UK) EU approval / Intermediate FDA MHRA LPLV
Slide 7: OATD-01 | current status
OATD-01 | current status First patient dosed in Phase II clinical trial at Edinburgh Hospital in the UK Study active in UK and US (1 active site in each country) Approval from the U.S. FDA and Central Bioethics Committee Clinical trial in Europe: o Approval of Medicines and Healthcare products Regul
Slide 8: OATD-02: A first in class dual arginase inhibitor for cancer applications
OATD-02: A first in class dual arginase inhibitor for cancer applications 8
Slide 9: OATD-02 | Phase I clinical study overview
OATD-02 | Phase I clinical study overview Patient population: Relapsed/refractory advanced and/or metastatic solid tumors Design: First-In-Human (FIH) OATD-02 Colorectal cancer, platinum-resistant serous Open-label single-arm dose-escalation ovarian cancer, pancreatic ductal cancer, renal monotherap
Slide 10: OATD-02 | start of 4th cohort
OATD-02 | start of 4th cohort Sixth cohort o After each dose level the decision is taken by Min. 3-6 patients Sponsor with help of SRC* to continue the study. Fifth cohort o Screening for next dose level must not start before Min. 3-9 patients SRC recommendation. 30 mg o Only one patient can be enro
Slide 11: OATD-02 | Swimmer plot – all enrolled patients
OATD-02 | Swimmer plot – all enrolled patients* DLT phase (4 weeks) 10.04.2024 *cut-off date 04 April 2024 11
Slide 12: OATD-01 and OATD-02: to learn more about mechanism of action (MoA)
OATD-01 and OATD-02: to learn more about mechanism of action (MoA) 12
Slide 13: OATD-01 | New publication
OATD-01 | New publication o Examples presented in this review demonstrate that protein glycosylation regulates metabolism-driven immune responses in macrophages, with implications for fibrotic processes and granuloma formation. o Targeting proteins that regulate glycosylation, such as fucosyltransfe
Slide 14: OATD-02 MoA | proof of intracellular activity
OATD-02 MoA | proof of intracellular activity o collaboration with a research group from the University of Mannheim to use Mass Spectrometry Imaging (MSI) to assess metabolites Conclusions: the metabolic activity of OATD-02 demonstrates significant changes in tissue distribution of important metabol
Slide 15: A promising pre-clinical pipeline
A promising pre-clinical pipeline 15
Slide 16: USP7 inhibitor | T cell activator with proven anti-cancer potential
USP7 inhibitor | T cell activator with proven anti-cancer potential Validated translational potential o The USP7 inhibitor program is at an advanced stage of optimization of the lead compound (medicinal chemistry work is focused on the search for an advanced lead compound with improved pharmacologic
Slide 17: mRNA Platform | discovering medicines of the future
mRNA Platform | discovering medicines of the future Small molecules targeting mRNA to prevent downstream mRNA translation DEL PoC achieved Docking (virtual 3D In silico Druggable In silico 3D Docking Active X-ray screening) Optimized Small Validation structure Sequence Secondary structure Small Lead
Slide 18: mRNA platform | first step - druggable region selection
mRNA platform | first step - druggable region selection SHAPE-MaP Predicted robust structure Shannon entropy In vitro In vitro – in cellulo Conservation Region with a stable structure Publications CLIP-seq Protein coding RNA isoforms from patient’s tissues and cell lines contain the target region Fu
Slide 19: mRNA platform | wet lab screening cascade – biophysical tests
mRNA platform | wet lab screening cascade – biophysical tests 5.5M compounds AI-based preselection VS SPR ~ 30 HITs with KD < 100 µM 500k 376 VS HITs 5 Different chemotypes RNA-Targeted Library 6 HITs with KD ≤ 10 µM 10.04.2024 19
Slide 20: mRNA Platform | Proof-of-Concept
mRNA Platform | Proof-of-Concept We confirmed that small molecules designed to target mRNA inhibit translation of the encoded protein relevant in cancer. Cmpd no. 1 OAT-5932 Cmpd no. 2 OAT-5944 Cmpd no. 3 OAT-5953 Cmpd no. 4 OAT-5954 target (normalized to β-actin) target (normalized to β-actin) targ
Slide 21: Finance
Finance 10.04.2024 21
Slide 22: Financial results
Financial results Assets & Liabilities changes (PLN m) PLN m 2023 2022 0,92 Revenue 1 328 1 638 1,62 40,71 7,65 Grants 1 309 1 613 Other 19 25 168,55 Cost incl: 23 174 18 407 138,03 General & Adm 12 842 13 947 Early stage programs 10 332 4 460 EBIT -21 846 -16 769 2022 Total Project Change in Cash R
Slide 23: R&D expenses, CAPEX and employment
R&D expenses, CAPEX and employment PLN m 2023 2022 R&D expenses incl.: 40.71 25.67 Cash position (March 2024) Clinical phase programs 30.63 12.92 >PLN 54m Discovery and preclinical development 10.08 12.75 programs Early programs 10.33 4.46 114 employees General and administrative expenses, 12.84 13.
Slide 24: Grants
Grants Funding Qualified cost Program requested Institution Filing Decision Protest filing Current status [PLN m] [PLN m] Agreement signed mRNA platform 52 33 PARP 05.2023 Positive (10.2023) - (12.2023) OATD-01 16 8.2 PARP 07.2023 Due Q3 2024 - - To be filed within USP21 66 47 PARP 05.2023 Rejected
Slide 25: Finance
Finance Molecure intends to use four possible sources of financing its activites: transaction revenues such as up-front payment(s) from one or more licensing agreements, grants and subsidies, share issue (SPO) and/or debt instruments. R&D and SG&A expenses in total Financing of Molecure’s strategic
Slide 26: Business Development and plans
Business Development and plans 10.04.2024 26
Slide 27: BIO Europe Spring 2024
BIO Europe Spring 2024 Number of Discussions per program: 36 o OATD-01 - 20 one-on- o OATD-02 - 6 one o mRNA – 4 meetings o other - 6 Notes: 10 high o The pipeline is perceived as very interesting and with high potential priority Follow-Ups o Good traction with some new and returning Big Pharma comp
Slide 28: Molecure | Key milestones 2024-2025
Molecure | Key milestones 2024-2025 OATD-01 OATD-02 o Reaching the therapeutic dose (Ph2RD) o Interim data analysis by an independent (2025) unblinded committee after completion of dosing of approx. 50 patients with lung sarcoidosis (expected early 2025) AI o Last patient dosed in the KITE Phase II
Slide 29: Disclaimer
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