Thx Pharma · EURONEXT:ALTHX
Thx Pharma — Theranexus — Investor Presentation (May 2023)
15 slides from Thx Pharma's investor presentation, May 2023
Slide 1: Investors Presentation May 2023
INVESTORS PRESENTATION May 2023
Slide 2: Speakers
Speakers Mathieu CHARVERIAT Thierry LAMBERT CEO and Co-founder Chief Financial Officer Ecole Polytechnique and Business Administration at the Mines Paris-Tech University of Birmingham Doctor in neuroscience and Cell Biology MBA (Master of Business from the Pierre and Marie Curie Administration) from
Slide 3: Theranexus, a clinical stage company developing drug candidates for rare neurological disorders
Theranexus, a clinical stage company... ...developing drug candidates for rare neurological disorders 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 and ongoing in early 2024 New ASO candidate1 in H
Slide 4: A particularly promising first active ingredient in juvenile Batten disease (CLN3)
A particularly promising first active ingredient in juvenile Batten disease (CLN3) Use of a registered The only active agent Recent discovery of A proprietary & First-to-market in a molecule with a A structuring in clinical development the disease patient adapted high potential clinically validated
Slide 5: CLN3: the mechanism of the disease has been elucidated thanks to recent academic work
CLN3: the mechanism of the disease has been elucidated thanks to recent academic work funded by BBDF In a nutshell Progression of the disease 700 to 1 000 800 to 1 000 No treatment registered Loss of patients in the US1 patients in the EU2 visual acuity => blindness Autosomal recessive transmission,
Slide 6: Batten-1 targets the synthesis of glycosphingolipids whose accumulation is toxic to neurons
Batten-1 targets the synthesis of glycosphingolipids whose accumulation is toxic to neurons Blockage of GSL synthesis by miglustat (active ingredient in Batten-1), leading to a reduction in their accumulation Reduction of cell death in the brain, with the objective of blocking disease Activity alrea
Slide 7: Batten-1: development of a proprietary drinkable formulation adapted to the CLN3 patient population
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 swallowing difficulties. ▶ Hig
Slide 8: FDA approval on efficacy endpoints and design of Phase 3 study
FDA approval on efficacy endpoints and design of Phase 3 study (our press release dated May 9th 2023) • Randomized double-blind study evaluating the efficacy of Batten-1 in 2 parallel groups over a 2-year treatment period, versus placebo • Target population: pediatric population of approximately 60
Slide 9: Development plan with Phase 3 results by the end of 2026 followed by market approval
Development plan with Phase 3 results by the end of 2026 followed by market approval 2021 2022 2023 2024 2025 2026 IND granted Dialogue with the FDA FDA approval of efficacy NDA filing (type C meetings) endpoints for Phase 3 (end of phase 2 meeting) P1/2 OPEN-LABEL SAFETY, PK AND EFFICACY (6 PATIENT
Slide 10: Batten-1: global peak sales exceeding $500 million per year
Batten-1: global peak sales exceeding $500 million per year Number of currently diagnosed Sales based on Estimated Sales based on est. Territories diagnosed patients patients patients prevalence4 (USD m)1 (USD m)1 United States ~7502 ~480 ~1 200 ~765 EU5 (Germany, France, Italy, ~3403 ~50 ~500 ~75 S
Slide 11: Theranexus: a strong value creation opportunity for investors
Theranexus: a strong value creation opportunity for investors Batten-1, a particularly attractive clinical asset: Batten-1 P3 results in 2026 A well understood and effective mechanism A high degree of protection (proprietary formulation + ODD) Easy access to a high potential market (>$500M/year): •
Slide 12: Appendix
Appendix Xs THERANEXUSI
Slide 13: Listing and shareholding
Listing and shareholding Financial data Capital structure Number of shares: 5,314,434 • ISIN : FR0013286259 - Mnemo : ALTHX Management & Employés • Market: Euronext Growth 12,0% • Share price on May 5th, 2023: €1.20. Auriga Partners • Market capitalization: 6.388 million euros 10,9% • Coverage: ODDO
Slide 14: Simplified income statement
Simplified income statement Controlled expenses In K€ (French standards) 2022 2021 Operating income 1,246 25 Other purchases and external expenses 3,967 5,591 Paris region Salaries and social charges 3,165 2,689 Depreciation and amortization 332 1,204 21 Lyon Other expenses 39 53 Operating result (6
Slide 15: Thank you for your attention!
THANK YOU 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