Molecure Sa · GPW:MOC
Molecure Sa — Conference Presentation (April 2025)
32 slides from Molecure Sa's conference presentation, Apr 2, 2025
Slide 1: Fate can be altered
Fate can be altered April 2, 2025
Slide 2: Presenting Team
Presenting Team Marcin Szumowski Zbigniew Zasłona Chairman of the Board & CEO Chief Scientific Officer Piotr Iwanowski Sławomir Broniarek Chief Medical Officer Chief Financial Officer 4/2/2025 2
Slide 3: Summary | Molecure on the path of continued growth
Summary | Molecure on the path of continued growth OATD-01 (in Phase 2 clinical development) OATD-02 (in Phase 1 clinical development) o Accelerating patient o Escalating the dose towards MTD: enrolment in the KITE study a more dynamic dose escalation o Strong translational data and beyond 20 mg/day
Slide 4: Molecure | Current pipeline
Molecure | Current pipeline Clinical Candidate Current status 2025 / 2026 Selection estimate 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, MASH, OATD-01 OATD-01
Slide 5: OATD-01: Phase 2 clinical study in pulmonary sarcoidosis
OATD-01: Phase 2 clinical study in pulmonary sarcoidosis 4/2/2025 5
Slide 6: OATD-01 | Phase 2 study in pulmonary sarcoidosis
OATD-01 | Phase 2 study in pulmonary 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 Application FPFV (UK) EU approval / FDA MHRA Interim analysis LPLV
Slide 7: OATD-01 | KITE: Boosting patient enrolment
OATD-01 | KITE: Boosting patient enrolment Study awarenesess & recognition Study protocol amendment Sep 2024: ERS (Austria); Nov 2024: WASOG (India); Modifications of in/ex criteria to ease patient Dec 2024: Panhellenic Pulmonology Congress recruitment & study procedures to ease study (Greece); Mar
Slide 8: OATD-01: Disease modifying potential in MASH
OATD-01: Disease modifying potential in metabolic dysfunction-associated steatohepatitis (MASH) 4/2/2025 8
Slide 9: OATD-01 | A window of opportunity
OATD-01 | A window of opportunity Sarcoidosis is the entry point to proving the clinical utility of targeting CHIT1 in other diseases with unmet medical needs Positive results in the Phase 2 proof-of-concept could pave the way to several other indications where chronic inflammation drives tissue rem
Slide 10: OATD-01 | MASH Market Forecast 7MM
OATD-01 | MASH Market Forecast 7MM With increasing disease prevalence, the MASH market holds immense untapped potential. Given the complex etiology of MASH, it is possible that each pipeline agent will have a place in the market CAGR 41.5% $30,0B Key Players: $25,0B - Approved $20,0B $15,0B $10,0B $
Slide 11: OATD-01 | Novel mechanism of action driving efficacy
OATD-01 | Novel mechanism of action driving efficacy 4/2/2025 UNPUBLISHED DATA 11
Slide 12: OATD-01 | Reverses metabolic processes in MASH
OATD-01 | Reverses metabolic processes in MASH MASH MASH + OATD-01 4/2/2025 UNPUBLISHED DATA 12
Slide 13: CHIT1 | Recent academic research on OATD-01 in MASH
CHIT1 | Recent academic research on OATD-01 in MASH Conclusions: o CHIT1 inhibition attenuates hepatic inflammation and fibrosis in mice with MASH-related liver fibrosis. o CHIT1 is upregulated in patients with liver fibrosis and MASH with progressive liver fibrosis and correlates with disease sever
Slide 14: CHIT1 | A novel approach using integrated RNA-seq data to screen for MASH biomarkers
CHIT1 | A novel approach using integrated RNA-seq data to screen for MASH biomarkers F0: no scarring (no fibrosis) F1: minimal scarring F2: scarring has occurred and extends outside the liver area (significant fibrosis) F3: fibrosis spreading and forming bridges with other fibrotic liver areas (seve
Slide 15: CHIT1 is upregulated in MASH patients and correlates with disease severity
CHIT1 is upregulated in MASH patients and correlates with disease severity Expression of CHIT1 in early stage versus late-stage liver fibrosis A Unique Immune-Related Gene Signature Represents Advanced Liver Fibrosis and Reveals Potential Therapeutic Targets. Biomedicines 2022, 10, 180 Circulating C
Slide 16: Depletion of CHIT1 activity prevents progression from simple steatosis to MASH
Depletion of CHIT1 activity prevents progression from simple steatosis to MASH and fibrosis Genotyped for CHIT1 (common 24-bp duplication mutation) were 200 NAFLD patients (110 MASH, 90 simple steatosis) and 100 controls (a case-control association study) o Compared was the frequency of mutant CHIT1
Slide 17: Potential in MASH | Summary
Potential in MASH | Summary CHIT1 is a therapeutic target upregulated in MASH that correlates with diseases severity o OATD-01 has been proven to have therapeutic effect in MASH models, including reversal of fibrosis (in-house, CRO, Galapagos and independent academic research data) o Patients with a
Slide 18: OATD-02: A first in class dual arginase inhibitor for cancer indications
OATD-02: A first in class dual arginase inhibitor for cancer indications 4/2/2025 18
Slide 19: OATD-02 | Phase 1 clinical study overview
OATD-02 | Phase 1 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 20: OATD-02 | First-in-Human study: towards more dynamic dose escalation
OATD-02 | First-in-Human study: towards more dynamic dose escalation 6th dose level group Min. 3-6 patients o After each dose level completed, the decision on 5th dose level group next step is taken by Sponsor under Min. 3-9 patients YY recommendation by Safety Review Committee mg/day 4th dose level
Slide 21: Reduced & focused pre-clinical pipeline
Reduced & focused pre-clinical pipeline 4/2/2025 21
Slide 22: Preclinical programs
Preclinical programs Protein targets mRNA targets o USP7 – advanced lead currently in the o X target – early program at the stage of toxicology study - results expected in hit-to-lead (obtained by VS) confirmed at June, proven immunomodulatory effect the cellular level, inducer of apoptosis for by M
Slide 23: Finance
Finance 4/2/2025 23
Slide 24: Financial results
Financial results Changes in assets & liabilities (PLN m) PLN m 2024 2023 Revenue 2,15 (2,9*) 3,67 Grants 1,39 (2,14) 1,65 Other 0,76 0,01 Cost incl: 34,48 35,59 General & Adm 11,03 12,90 Early stage programs 19,41 22,69 Motivation program 4,04 0 (32,33) / EBIT (31,92) (28,29**) Net Financial income
Slide 25: R&D expenses, CAPEX and employment
R&D expenses, CAPEX and employment PLN m 2024 2023 R&D expenses incl.: 37,55 53,34 Clinical stage programs 18,14 30,65 Cash position as of Discovery and preclinical development 19,41 22,69 31.03.2025 ~PLN 40m programs General and administrative expenses, (cash runway until H1 2026) 11,03 12,90 inclu
Slide 26: Grants
Grants Funding Eligble costs Program requested [PLN Institution Filing Decision Protest filing Current status [PLN m] m] Positive Agreement signed mRNA platform 52 33 PARP 05.2023 - (10.2023) (12.2023) OATD-01 16 8.2 PARP 07.2023 Rejected - Expected Resubmission OATD-01 USD 2.1m USD 2.2m NIH 04.2025
Slide 27: 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 28: Business Development and plans
Business Development and plans 4/2/2025 28
Slide 29: BD 2024 activities & BIO Europe Spring 2025
BD 2024 activities & BIO Europe Spring 2025 Partnering events attended: 100+ one-on- o BIO Europe Spring – March (Barcelona) one o BIO International – June (San Diego) meetings in o BIO Europe – November (Stockholm) 2024 o BIO Europe Spring – March 2025 (Milan) Notes: OATD-01: 70 OATD-02: 18 o The p
Slide 30: Molecure | Key milestones 2025/2026
Molecure | Key milestones 2025/2026 OATD-01 OATD-02 o PoC study (KITE) in pulmonary sarcoidosis: o Establishng the therapeutic dose (RP2D) Interim data analysis by the independent (projected 2H 2025) (unblinded) Data Monitoring Committee after completion of dosing of approx. 50 patients (H2 2025), t
Slide 31: Q&A
Q&A 4/2/2025 31
Slide 32: Molecure SA 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