Slide 1: First Half 2021 Financial and Corporate Update
First Half 2021 Financial and Corporate Update September 23, 2021
Slide 2: Disclaimer
Disclaimer Some of the statements contained in this presentation constitute forward-looking statements. Statements that are not historical facts are forward-looking statements. Forward-looking statements generally can be identified by the use of forward-looking terminology such as “may”, “will”, “ex
Slide 3: Poxel's Investment Highlights
Poxel’s Investment Highlights To discover, develop and commercialize innovative therapies for patients suffering • TWYMEEG® (Imeglimin) approved from serious chronic and rare diseases and launched for Type 2 Diabetes in with underlying metabolic pathophysiology Japan • Strategic focus on rare metabo
Slide 4: A New Chapter to Drive Shareholder Value
A New Chapter to Drive Shareholder Value Focus on Rare Metabolic Diseases and NASH Rare ALD* Metabolic Diseases PXL065 Type 2 Diabetes NASH D-TZD2 Platform PXL770 TWYMEEG® AMPK3 Platform (Imeglimin) External Partnered in Asia1 Opportunities with diabetes market leader in Japan • Phase 2a Biomarker P
Slide 5: Robust Mid-to-Late Stage Metabolic Pipeline
Robust Mid-to-Late Stage Metabolic Pipeline Focus on Rare Metabolic Diseases and NASH Discovery Approved/ Indication MOA PH 1 PH 2 PH 3 Partner/ Rights Upcoming Milestones /PC Marketed Type 2 Diabetes (T2D) • TWYMEEG approved for T2D in Japan on TWYMEEG® MRC June 23, 2021 T2D Japan / Asia1 Modulator
Slide 6: TWYMEEG (Imeglimin): Approved in Japan
TWYMEEG® (Imeglimin): Approved in Japan Partnered in Asia1 with Diabetes Market Leader, Sumitomo Dainippon Pharma • June 23rd Approval in Japan triggered: o 3rd and final tranche of EUR 13.5 million from IPF loan received June 30, 2021 o Milestone payment of ~EUR 13.2 million (USD 15.8 million)2 fro
Slide 7: Strategy to Pursue Treatments for Rare Diseases
Strategy to Pursue Treatments for Rare Diseases Poxel Molecules can Target Key Nodes in Pathways Driving Several Rare Diseases Why Rare Disease ? o High unmet needs, limited treatment options; > 90% of rare diseases are without an FDA approved treatment1 o Efficient and lower cost development: faste
Slide 8: Financial Update
Financial Update First Half 2021 8
Slide 9: Revenue
Revenue Mostly reflecting the Marketing approval milestone for Imeglimin in Japan H1 2021 H1 2020 (amounts in K€) 6 months 6 months Roivant Agreement 13 1 Mostly reflects the JPY 1,750 million milestone payment from Sumitomo Dainippon Pharma triggered by the marketing approval of 1 Sumitomo Agreemen
Slide 10: Statement of Comprehensive Income as of June 30, 2021
Statement of Comprehensive Income as of June 30, 2021* Net loss reduced at €8.0m as a result of increasing revenue Consolidated statement of loss June 30, 2021 June 30, 2020 (amounts in K€) 1 Mostly reflects the JPY 1,750 million 1 Revenue 13 274 6 372 marketing approval milestone for Imeglimin in J
Slide 11: Statements of Financial Position as of June 30, 2021
Statements of Financial Position as of June 30, 2021* Assets Consolidated Financial Position June 30, 2021 Dec. 31, 2020 (Amounts in k€) 1 Reflects marketing approval milestone for Intangible Assets 16 642 16 642 Imeglimin in Japan, paid in July Property, Plant & Equipment 1 968 2 224 Other non-curr
Slide 12: Statements of Financial Position: Shareholders' Equity and Liabilities
Statements of Financial Position as of June 30, 2021* Shareholders’ Equity and Liabilities (amounts in K€) June 30, 2021 Dec. 31, 2020 1 Mostly reflects the HY 2021 net loss 1 Total shareholders’ equity 21,625 26,879 Employee benefits 544 581 2 Total financial liabilities: €37m (mostly Non-current f
Slide 13: Statements of Cash Flow as of June 30, 2021
Statements of Cash Flow as of June 30, 2021* Consolidated statement of cash flows June 30, 2021 June 30, 2020 (amounts in K€) Cash and cash equivalents as of the opening 40 203 37 187 1 Includes the HY 2021 net loss and a date €10m increase in WC (mostly 1 Cash flows from operating activities -16 06
Slide 14: Key Financial & Shareholder Information
Key Financial & Shareholder Information Market data Shareholder ownership2 8.3% Andera Partners 19.7% Bpi France Ticker: POXEL 57.8% Floating 9.2% ISIN: FR0012432516 Founders Number of shares: 28,670,3581 5.0% Roivant Sciences Ltd Key financials Analyst coverage • As of 06/30/21 cash & cash equivale
Slide 15: Clinical Update
Clinical Update First Half 2021 15
Slide 16: Accelerating & Expanding Rare Metabolic Disease Programs
Rare Metabolic Diseases Accelerating & Expanding Rare Metabolic Disease Programs Starting with existing platforms: PXL065 - D-TZD’s* PXL770 - AMPK Activator *Deuterium-modified thiazolidinediones. 16
Slide 17: Two First-in-Class Advanced Lead Molecules
Two First-in-Class Advanced Lead Molecules PXL065 PXL770 • Deuterium stabilized R-stereoisomer of • Proprietary direct allosteric AMPK pioglitazone* activator • Preclinical: • Preclinical: o no (PPARg –driven) weight gain/fluid retention o Metabolic, anti-inflammatory, cytoprotective efficacy in NA
Slide 18: Adrenoleukodystrophy
Adrenoleukodystrophy A Not-so-Rare Orphan Neurometabolic Disease Genetics Prevalence Diagnosis • Monogenic, X-linked mutations in Estimated US Prevalence* • Newborn screening – ABCD1 gene 20,000 – 29,000 increasingly common (now • Gene encodes a transporter present >60% of newborns in US) in peroxis
Slide 19: Poxel Platforms – Potential to Target ALD Pathophysiology
Poxel Platforms – Potential to Target ALD Pathophysiology ABCD1: Transports VLCFA into peroxisome for degradation (ABCD2 can serve as an alternative peroxisomal transporter) Defective ABCD1 leads to accumulation of VLCFA ABCD1 in tissues VLCFA cause axonal degeneration and secondary demyelination D-
Slide 20: Both PXL065 and PXL770 Mediate Neurologic Benefits
Both PXL065 and PXL770 Mediate Neurologic Benefits ABCD1-Null Mouse (12 week Treatment) Electronic Microscopy of Sciatic Nerve Neurologic Tests (Balance Beam) WT ABCD1-null untreated ABCD1-null + PXL065 5 ✱ ✱✱ Score (Balance Beam) 4 3 Cell shape change 60 ✱ 6:0) 2 ✱ ✱✱ Stellate cell shape (% of tota
Slide 21: Planned Phase 2a Studies in ALD/AMN
Planned Phase 2a Studies in ALD/AMN PXL770 and PXL065 in Two Separate Identical Studies Key inclusion criteria • Males with AMN Screening PXL770 / PXL065 1 Dose Each Follow • Age 18-65 Period up • No active cerebral Treatment period disease 12 weeks • 12 patients each stable baseline multiple testin
Slide 22: ALD Opportunity Summary
ALD Opportunity Summary High Unmet Needs, Blockbuster Market Potential • Blockbuster market opportunity o US prevalence of 20,000-29,000; Global prevalence of 444,000 – 644,000 o Ability for premium pricing based upon other orphan drugs with similar prevalence o Potential Regulatory designations: •
Slide 23: NASH: PXL065 and PXL770
NASH PXL065 Non-Genomic Pathway D-TZD Modulator for Treatment of NASH Utilizing the 505(b)(2) Regulatory Pathway PXL770 Direct AMPK Activator 23
Slide 24: Non-Alcoholic Steatohepatitis (NASH)
Non-Alcoholic Steatohepatitis (NASH) No Approved Medicines - Poxel has Two Clinical Stage First-in-Class Oral Therapies Metabolic syndrome High Morbidity Dyslipidemia Excessive caloric intake Type 2 diabetes Cardiovascular events Sedentary lifestyle Obesity (leading cause of death) NASH Hepatic impa
Slide 25: PXL770 and PXL065: Novel, First-in-Class Product Candidates
PXL770 and PXL065: Novel, First-in-Class Product Candidates HALLMARKS OF NASH MΦ Lipid accumulation in Immune cells Hepatic stellate hepatocytes (macrophages - MΦ) Cellular damage-death cell activation Steatosis Inflammation Ballooning Fibrosis • First-in-Class - Novel Mechanisms • Daily oral admini
Slide 26: PXL065 Ongoing Phase 2 in Biopsy-Proven NASH Patients
PXL065 Ongoing Phase 2 in Biopsy-Proven NASH Patients Single Streamlined Study - 505(b)(2) Pathway; Designed to Select Ph3 Dose(s) Randomization PXL065 7.5 mg QD / 30 patients Week 36 1:1:1:1 PXL065 15 mg QD / 30 patients Key inclusion criteria PXL065 22.5 mg QD / 30 patients • Biopsy-proven NASH pa
Slide 27: PXL770 – NASH Summary
PXL770 – NASH Summary Phase 2a – Results in NAFLD Patients with Diabetes Liver Fat Content • First direct AMPK activator studied in (% Change from baseline) human disease Pbo 250 QD 250 BID 500 QD 0 • Well tolerated, acceptable safety profile -5 LS mean change from Baseline • Target engagement estab
Slide 28: Near-Term Milestones to Drive Poxel's Growth
Near-Term Milestones to Drive Poxel’s Growth NASH T2DM PXL065: PXL065: ALD/Other Enrollment of Results from DESTINY Ph 2 trial PXL065 & PXL770: DESTINY Ph 2 trial Cash PXL065 & PXL770: completed in Initiate Ph 2a September Results from Ph 2a biomarker POC trials biomarker POC trials in ALD/AMN in AL
Slide 29: Summary and Investment Highlights
Summary and Investment Highlights • TWYMEEG® (Imeglimin) approved for Type 2 Diabetes in Japan o Sumitomo, #1 diabetes company in Japan, launch Sept 2021 o Up to EUR 200 million (~USD 230 million)1 in potential sales-based payments and escalating double- digit royalties2 o US/Europe: exploring optio
Slide 30: Question & Answer Session
Question & Answer Session 25 minutes Participants can submit questions in the chat 30