Slide 1: Investor Presentation - Sachs Life Sciences Forum
INVESTOR PRESENTATION Sachs Life Sciences Forum March 8 & 9, 2023
Slide 2: Disclaimer
Disclaimer This document contains certain forward-looking statements concerning Theranexus and its business, including its prospects and product candidate development. Such forward-looking statements are based on assumptions that Theranexus considers to be reasonable. However, there can be no assura
Slide 3: Speakers
Speakers Mathieu Thierry CHARVERIAT LAMBERT Co-founder& CEO Chief Financial Officer Ecole Polytechnique and Business Administration at Mines Paris-Tech the University of Birmingham Doctor in Neuroscience and MBA (Master of Business Cell Biology from the Pierre Administration) from INSEAD and Marie C
Slide 4: Clinical-stage company developing drug candidates for rare neurological diseases
Theranexus is a clinical-stage company developing drug candidates targeting rare neurological diseases Lead Nonclinical Batten-1 Screening Selection PoC IND Phase I Phase II Phase III NDA Juvenile Batten disease (CLN3) Entering P3 P1-2 fully recruited & ongoing in H2 2023 New RNA based candidate TFE
Slide 5: A first highly promising asset in Juvenile Batten disease (CLN3)
A first highly promising asset in Juvenile Batten disease (CLN3) Use of an approved The only advanced Recent A proprietary liquid First-to-market in a compound with a A crucial clinical stage asset in understanding of the formulation adapted market with strong clinically validated partnership CLN3 m
Slide 6: CLN3: the mechanism of the disease was discovered thanks to recent academic work
CLN3 : the mechanism of the disease was discovered thanks to recent academic work funded by BBDF Autosomal recessive 700 - 1 000 patients in the US1 Founder effect located in the No treatment Loss of visual 800 - 1 000 patients in the EU2 Nordic countries approved acuity leading to to blindness 6-10
Slide 7: Batten-1 targets the synthesis of glycosphingolipids
Batten-1 targets the synthesis of glycosphingolipids which accumulation is toxic to neurons Miglustat (active ingredient of Batten-1) blocks the synthesis of GSL, leading to Reducing neuronal cell death, a reduction of their accumulation with the objective of stopping the progression of the This pha
Slide 8: 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 is more convenient to take and modulate the treatment Drinkable solution : – Suitable for pediatric patients as well as for patients presenting difficulties in swallowing
Slide 9: 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 10: 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 11: Theranexus: a strong value creation proposition for investors
Theranexus : a strong value creation proposition for investors Batten-1, a particularly attractive clinical-stage asset: Batten-1 registration in 2026 A well-understood mechanism with already demonstrated efficacy A high degree of protection (proprietary formulation + ODD) A facilitated access
Slide 12: Appendices
Appendices Xs THERANEXUSI
Slide 13: Listing and Shareholding
Listing and Shareholding Financial data Capital structure • ISIN : FR0013286259 - Ticker: ALTHX • Market: Euronext Growth • Share price on 22 February 2022: €1.91 • Market capitalization: €10.2m • Coverage: ODDO BHF, Portzamparc • Liquidity contract: Portzamparc 13
Slide 14: 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 15: Thanks for your attention!
THANKS FOR YOUR ATTENTION ! Your contacts : Mathieu Charveriat Chief Executive Officer mathieu.charveriat@theranexus.com / mobile : + 33 6 63 01 19 19 Thierry Lambert Chief Financial Officer thierry.lambert@theranexus.com / mobile : +33 6 03 86 27 88