Molecure Sa · GPW:MOC
Molecure Sa — Investor Presentation (July 2022)
25 slides from Molecure Sa's investor presentation, Jul 21, 2022
Slide 1: Fate can be altered
Fate can be altered July 21, 2022 1
Slide 2: 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
Slide 3: Presenting Team
Presenting Team Samson Fung Zbigniew Zasłona Piotr Iwanowski MD PhD, MD, PhD Chief Medical Officer VP Research Biology Clinical Research & Operations Experienced pharmaceutical Biologist with extensive Expert executive with significant expertise experience in anti-inflammatory 20 years of experience
Slide 4: Developing first-in-class small molecule drugs to address challenging protein targets
Developing first-in-class small molecule drugs to address challenging protein targets 21/07/2022 4
Slide 5: Current pipeline
Current pipeline Clinical Candidate Selection Molecular Target Lead In Vivo Preclinical Regulatory Target Indications Validation Optimization POC Development Process Phase I Phase II Hit-to-lead mRNA TARGETS Undisclosed mRNA In progress INFLAMMATION/ONCOLOGY PROTEIN TARGETS OATD-01 Sarcoidosis, othe
Slide 6: Molecure now has global rights to OATD-01
Molecure now has global rights to OATD-01 1) Molecure regained all rights, associated data, intellectual property and inventory to OATD-01 2) Data generated to date confirm an attractive benefit-risk potential, warranting further development 3) Data and information generated and obtained by Galapago
Slide 7: Why sarcoidosis?
Why sarcoidosis? 21/07/2022 7
Slide 8: What is sarcoidosis
What is sarcoidosis • Sarcoidosis is a systemic inflammatory disease characterized by formation of immune granulomas in various organs • Granulomas are tight aggregates of immune cells consisting of centrally located, pathologically activated macrophages, epithelioid and giant cells, surrounded by T
Slide 9: Epidemiology
Epidemiology • Sarcoidosis is an idiopathic, global disease • Affecting both men and women • Variation in sarcoidosis incidence across ethnic groups is well documented, with African-Americans and northern Europeans having the highest rates of sarcoidosis incidence (up to 3x increase in incidence in
Slide 10: Clear need for new drugs for sarcoidosis
Clear need for new drugs for sarcoidosis Currently no cure for sarcoidosis and treatments only modify the granulomatous process and its clinical consequences. Furthermore, there is little prospective data to guide therapy strategies Systemic corticosteroids remain the standard of treatment and altho
Slide 11: Creating Significant Value with OATD-01
Creating Significant Value with OATD-01 A potential disease modifying agent for sarcoidosis 21/07/2022 11
Slide 12: OATD-01 – product profile and strengths
OATD-01 – product profile and strengths • Novel, single-digit nM, first-in-class small molecule chitinase inhibitor targeting a key mechanistic pathway involved in inflammation and tissue remodeling • Dual anti-inflammatory and anti-fibrotic activity through a single new chemical entity • Once-a-day
Slide 13: OATD-01 – scientific and regulatory milestones achieved to date
OATD-01 – scientific and regulatory milestones achieved to date • Efficacy proven in multiple animal models of sarcoidosis, idiopathic pulmonary fibrosis, (IPF) and non-alcoholic steatohepatitis (NASH) • Compelling translational data • Excellent pharmacological profile • Demonstrated safety in anima
Slide 14: CHIT1 is highly expressed in lungs of patients with sarcoidosis
CHIT1 is highly expressed in lungs of patients with sarcoidosis Expression of CHIT1 in lungs and serum of patients with confirmed sarcoidosis Lung sarcoidosis Serum Lung biopsies from sarcoidosis patients show very strong Biopsy 1. – diseased lungs Biopsy 2. – non-diseased with granulomas lungs expr
Slide 15: Efficacy of OATD-01 in MWCNT-induced granulomatous inflammation model in mice
Efficacy of OATD-01 in MWCNT-induced granulomatous inflammation model in mice Organized granulomatous structures 30 Day 10 10 **** 40 days post MWCNT+ESAT-6 Granulomatous inflammation (Ave counts/3lobes/3section) 8 CCL4 in BAL fluid 20 [pg/ml] 6 CHIT1 H&E 4 10 2 0 0 ) l 6 o kg ] T6 ol kg T- tr tr g/
Slide 16: OATD-01 – translational and clinical data to support a PoC study in sarcoidosis
OATD-01 – translational and clinical data to support a PoC study in sarcoidosis Very strong clinical Unmet medical need: no Clinical Phase I results correlation of CHIT1 activity effective therapy, advanced with the disease process disease is debilitating with 8% mortality •Full and sustained pharma
Slide 17: GLPG4716-CL-102 (NCT05030857) conclusions
GLPG4716-CL-102 (NCT05030857) conclusions Drug-drug Interaction and Food-effect Study With OATD-01 and Midazolam in Healthy Subjects Conclusions: • OATD-01 when co-administered with midazolam was well tolerated. • Repeated dosing of OATD-01 did not result in meaningful changes in midazolam exposure,
Slide 18: OATD-01 on-track to enter Phase 2 clinical study H1 2023
OATD-01 on-track to enter Phase 2 clinical study H1 2023 A double-blind, randomized, placebo-controlled multicentre study to assess the anti- granulomatous efficacy and safety of an oral inhibitor of CHIT1 (OATD-01) in patients with active pulmonary sarcoidosis • 6 to 10 out-patient sites in the US
Slide 19: Timelines & budget estimates for Phase 2 PoC
Timelines & budget estimates for Phase 2 PoC Submission package filing expected Q1 2023 KOL CTA First Patient Last Patient Clinical Study consultations Submission First Visit Last Visit Report Q3-Q4 2022 Q1 2023 Q3 2023 H2 2024 H1 2025 Estimated budget: $10 mln Potential sources of funding: • Founda
Slide 20: Key Opinion Leaders supporting the clinical development of OATD-01 in sarcoidosis
Key Opinion Leaders supporting the clinical development of OATD-01 in sarcoidosis • Prof. Michael Kreuter MD PhD • Head of the Center for Interstitial and Rare Lung Diseases in the Thoracic Clinic of the University Hospital Heidelberg • Marlies Wijsenbeek – Lourens MD PhD • Pulmonologist, Chair Cent
Slide 21: Prof. Michael Kreuter
Prof. Michael Kreuter Prof. Dr. Michael Kreuter is Head of the Center for Interstitial and Rare Lung Diseases in the Thoracic Clinic of the University Hospital Heidelberg. This is an ILD center certified by the European Reference Network (ERN) and a sarcoid expert center recognized by WASOG*. Prof.
Slide 22: Market Competition
Market • Currently the sarcoidosis market is fragmented due to the lack of standard of care • Annual cost or treatment varies form $280 for prednisone alone to over $30,000 for adalimumab • Disease modifying treatment (DMT) will warrant a premium price, estimated acceptable for the commercial payer
Slide 23: OATD-01 potential source of significant value
OATD–01 potential source of significant value 1) OATD-01 is a novel, first in class dual acting chitinase inhibitor 2) Potentially disease modifying, and could transform standard of care for patients with lung sarcoidosis – no current compelling treatment options 3) Efficacy proven in multiple anima
Slide 24: Questions & answers
Questions & answers 21/07/2022 24
Slide 25: Abbreviations used
Abbreviations used: •ADME: Absorption, Distribution, Metabolism, And Excretion •nM: Nanomolar •BALF: Broncho-alveolar Lavage Fluid •NOAEL: No-observed-adverse-effect Level •BID: Bis In Die – Twice A Day •ODD: Orphan Drug Designation •CHIT1: Chitotriosidase •PK/PD: Pharmacokinetics / Pharmacodynamics