Molecure Sa · GPW:MOC
Molecure Sa — Corporate Update (October 2024)
33 slides from Molecure Sa's corporate update, Oct 1, 2024
Slide 1: Fate can be altered
Fate can be altered October 1, 2024
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 10/1/2024 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 Patient enrolment in this PoC o Escalating the dose towards MTD: clinical trial in progress SRC recommendation to expand o Strong translational data and the 4th do
Slide 4: Molecure | Balanced pipeline
Molecure | Balanced pipeline Robust pipeline of novel, first in class differentiated assets. Several anticipated value creating milestones over next 2 years Clinical Candidate 2025 / 2026 Current status estimate Selection Molecular Target Lead In Vivo Preclinical Regulatory Target Indications Valida
Slide 5: OATD-01: Phase 2 clinical trial in sarcoidosis
OATD-01: Phase 2 clinical trial in sarcoidosis 10/1/2024 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 (US/UK) EU approval / FDA MHRA Interim analysis LP
Slide 7: OATD-01 | KITE study: Patient enrolment
OATD-01 | KITE study: Patient enrolment Challenging patient enrolment (rare disease, with a subset of patients eligible: low-risk treatment-naïve/currently untreated patients) Competitive landscape now more favourable (RESOLVE-Lung and PREDMETH studies recently completed enrolment) Implementation of
Slide 8: OATD-01: Disease modifying potential in MASH
OATD-01: Disease modifying potential in metabolic dysfunction-associated steatohepatitis (MASH) 10/1/2024 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 multiple other indications where chronic inflammation drives tissue re
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 10/1/2024 UNPUBLISHED DATA 11
Slide 12: OATD-01 | Reverses metabolic processes in MASH
OATD-01 | Reverses metabolic processes in MASH MASH MASH + OATD-01 10/1/2024 UNPUBLISHED DATA 12
Slide 13: CHIT1 | Late breaking report on OATD-01 in MASH
CHIT1 | Late breaking report 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 severity.
Slide 14: CHIT1 | 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 reserach data) o Patients with a
Slide 18: OATD-02: A first in class dual arginase inhibitor for cancer applications
OATD-02: A first in class dual arginase inhibitor for cancer applications 10/1/2024 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: expanding 4th cohort
OATD-02 | First-in-Human study: expanding 4th cohort o After each dose level completed, the decision on Sixth dose level group next step is taken by Sponsor under Min. 3-6 patients recommendation by Safety Review Committee Fifth dose level group Min. 3-9 patients 30 mg/day Fourth dose level group Mi
Slide 21: A promising pre-clinical pipeline
A promising pre-clinical pipeline 10/1/2024 21
Slide 22: USP7 inhibitor | Preclinical candidate selection
USP7 inhibitor | Preclinical candidate selection A research collaboration to facilitate the discovery and development of novel small molecule drugs targeting USP7 Molecure’s expertise combined Candidate USP7i developed for an intravenous with Avicenna’s AI-driven medicinal Risk/profit sharing cooper
Slide 23: mRNA Platform | Combination therapy leads to discovery program initiation
mRNA Platform | Very promising data in combination therapy leads to discovery program initiation* Protein MoA: induction of apoptosis of cancer cells Next steps: synthesis of more selective and potent analogues Control Apoptosis Compound Compound activator + apoptosis activator ELISA assay determine
Slide 24: mRNA Platform | Second early-stage program initiated
mRNA Platform | Second early-stage program initiated* Receptor MoA: inhibition of secretion Viability of a pro-inflammatory Viability [%] mediator Next step: synthesis of more selective and potent analogs Log compound concentration [µM] Hit compound derived from a DEL screenining campaign Compounds
Slide 25: Finance
Finance 10/1/2024 25
Slide 26: Financial results
Financial results PLN m H1 2024 H1 2023 Changes in assets & liabilities (PLN m) Revenue 0,31 1,79 10,29 Grants 0,04 1,79 26,14 1,47 Other 0,26 0,01 0,72 112,25 Cost incl: 15,38 17,14 95,65 General & Adm 4,98 6,01 Early stage programs 10,40 11,14 2023 Total Project Change in Cash Receivables Fixed As
Slide 27: R&D expenses, CAPEX and employment
R&D expenses, CAPEX and employment PLN m H1 2024 H1 2023 R&D expenses incl.: 20,69 20,29 Clinical stage programs 10,29 9,15 Cash position (end of Discovery and preclinical development 10,40 11,14 September 2024) programs General and administrative expenses, 4,98 6,01 >PLN 27m including business deve
Slide 28: 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) Waiting for OATD-01 16 8.2 PARP 07.2023 rejected - evaluation sheets Expected OATD-01 USD 2.2m USD
Slide 29: 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 30: Business Development and plans
Business Development and plans 10/1/2024 30
Slide 31: BIO International 2024
BIO International 2024 Number of discussions per program: 42 one-on- o OATD-01 – 28 – Main BD focus on initiating a clinial PoC MASH study with a partner in one paralel with the KITE study in lung sarcoidosis patients – discussions ongoing meetings o OATD-02 – 8 – BD progress pending MTD and PhII re
Slide 32: Molecure | Key milestones 2025/2026
Molecure | Key milestones 2025/2026 OATD-01 OATD-02 o Reaching the therapeutic dose (Ph2RD) o Interim data analysis by an independent (projected 1H 2026) unblinded committee after completion of dosing of approx. 50 patients with lung sarcoidosis (projected mid 2025) AI o Last patient dosed in the KI
Slide 33: 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