Thx Pharma · EURONEXT:ALTHX
Thx Pharma — Theranexus — Investor Presentation (January 2023)
16 slides from Thx Pharma's investor presentation, January 2023
Slide 1: Investor Presentation January 2023
INVESTOR PRESENTATION January 2023 1
Slide 2: Speakers
Speakers Julien Mathieu Thierry VEYS CHARVERIAT LAMBERT Chief Business Co-founder, CSO Chief Financial Officer Development Officer & Deputy CEO Graduate in Science from the Ecole Polytechnique and Business Administration at University of Aix-Marseille Mines Paris-Tech the University of Birmingham MB
Slide 3: Profile and pipeline
Profile and pipeline THERANEXUS IS A CLINICAL STAGE COMPANY DEVELOPING FIRST-IN-CLASS OR FIRST TO MARKET INNOVATIONS TO TACKLE RARE NEUROLOGICAL DISORDERS 2013 2017 2019 2022 Launch of P1/2 21 Paris area Partnership with Spin off from Entered the trial of Batten-1 Lyon Beyond Batten an academic Euro
Slide 4: Juvenile Batten disease (CLN3) in brief
Juvenile Batten disease (CLN3) in brief 700-1,000 patients in the US1 Autosomal recessive No treatment registered 800 - 1,000 patients in the EU2 Founding effect localised in the Nordic countries Diagnosis in Loss of visual Cognitive decline Motor symptoms children acuity leading to and epileptic be
Slide 5: Batten-1 in CLN3: a strong molecular rationale
Batten-1 in CLN3: a strong molecular rationale Gangliosides Gb3Cer (e.g. GM1) iGb3Cer NEURONAL APOPTOSIS Globo series ↗ Lysosomal load AND (e.g. Gb3, Gb4) LacCer NEUROINFLAMMATORY Ceramides GlcCer PROCESS GCS Lacto & neo lacto series (e.g. Lc3, Lc4) Accumulation of Batten-1 glycosphingolipids in CLN
Slide 6: Batten-1 in CLN3: non-clinical validation of efficacy
Batten-1 in CLN3: non-clinical validation of efficacy Miglustat restores physiological levels of glycosphingolipids and reduces apoptosis in CLN3 models Ganglioside GM1 Globoside Gb4 CLN3 patient iPSC derived cortical neurons : • Reversion of GM1, Gb4, SCMAS by miglustat Similar conclusions in sever
Slide 7: Batten-1: proprietary drinkable formulation adapted to CLN3 patients
Batten-1: development of a proprietary drinkable formulation adapted to the CLN3 patient population • A drinkable solution that makes it easier to take and modulate the treatment Drinkable solution : – Suitable for pediatric patients as well as for patients presenting difficulties in swallowing –
Slide 8: Aim for a market approval by 2026
Development plan with the ambition of a market approval by 2026 2021 2022 2023 2024 2025 2026 IND granted NDA filing Dialogue with the FDA Phase 3 protocol (type C meetings) review by the FDA APPROVAL P1/2 OPEN LABEL SAFETY, PK & EFFICACY (6 PATIENTS) Safety extension study FPI LPI Completion of saf
Slide 9: Batten-1: global peak sales exceeding $500 million per year
Batten-1: global peak sales exceeding $500 million per year no. of currently Sales based on Sales based on Territories diagnosed diagn. patients Est. prevalence4 est. patients patients (USD m)1 USD m)1 United States ~7502 ~480 ~1’200 ~765 UE5 (Germany, France, Italy, Spain, UK) ~3403 ~50 ~500 ~75 No
Slide 10: Attractive competitive environment and strong market opportunities
Attractive competitive environment and strong market opportunities • Orphan designation obtained from the FDA (7 y. exclusivity) and the EMA (10 y. exclusivity) • Patent protection covering at least until 2037 • New proprietary drinkable formulation adapted to patients' needs • No competing treatmen
Slide 11: Project achievements and ambition
Project achievements and ambition ODD status for Batten-1 IND status for Batten-1 P1/2 launched and fully recruited Good tolerance of Batten-1 highlighted by P1/2 safety results Clinical sites selected for P3 H2. 2023 Start of the Phase 3 clinical trial with Batten-1 in children with CLN3
Slide 12: Appendices
Appendices a THERANEXUS
Slide 13: Theranexus' discovery platforms
Theranexus’ discovery platforms Neuronal Lysosomal hyperexcitability storage disorders platform platform Glial modulation of Neuron/glia Proteinopathies & TFEB platform neuronal activity metabolism autophagy Assessing neuron/glia Healthy & diseased iPSC interactions Identify TARGETS OF INTEREST IN R
Slide 14: Listing and Shareholding
Listing and Shareholding Financial data Capital structure • ISIN : FR0013286259 - Mnemo: ALTHX • Market: Euronext Growth • Share price on 30 December 2022: €1.75 • Market capitalisation: €9.3m • Coverage: ODDO BHF, Portzamparc • Liquidity contract: Portzamparc 14
Slide 15: Simplified income statement
Simplified income statement Controlled expenditure In K€ (French standards) S1 2022 S1 2021 Operating income 462 12 Other purchases and external charges 1 950 2 944 Wages and social charges 1 509 1 388 Depreciation and amortization on fixed assets 166 195 Other expenses 24 34 Operating result (3 187
Slide 16: Thank you for your attention!
THANK YOU FOR YOUR ATTENTION! Your contacts: Mathieu Charveriat Co-founder, CSO & deputy CEO mathieu.charveriat@theranexus.com / mobile: + 33 6 63 01 19 19 Thierry Lambert CFO thierry.lambert@theranexus.com / mobile: +33 6 03 86 27 88