Thx Pharma · EURONEXT:ALTHX
Thx Pharma — Theranexus — Investor Presentation (September 2023)
17 slides from Thx Pharma's investor presentation, Sep 29, 2023
Slide 1: Investor Presentation
Innovative biopharmaceutical company specialized in the treatment of rare central nervous system disorders INVESTOR PRESENTATION (updated following Batten-1 phase 1-2 trial results after 12 months of treatment) September 29th 2023
Slide 2: Disclaimer
Disclaimer This presentation has been prepared by Theranexus S.A. (the "Company") and is provided for information purposes only. The information and opinions contained in this presentation are provided solely at the date of the presentation and may be updated, supplemented, reviewed, verified or ame
Slide 3: Speakers
Speakers Mathieu CHARVERIAT Thierry LAMBERT CEO & 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 I
Slide 4: Theranexus, a company with a Phase ready clinical asset
Theranexus, a company with a Phase 3 ready clinical asset ...developing drug candidates for rare neurological disorders Preclinical NDA Batten-1 Screening Selection proof of concept IND Phase 1 Phase 2 Phase 3 (approval) Juvenile Batten disease (CLN3) Entry into P3 P1-2 fully recruited and in progre
Slide 5: Batten-1, a particularly attractive clinical-stage asset
Batten-1, a particularly attractive clinical- stage asset
Slide 6: A particularly promising first clinical stage asset in juvenile Batten disease (CLN3)
A particularly promising first clinical stage asset in juvenile Batten disease (CLN3) A STRUCTURING USE OF A REGISTERED MOLECULE DENSE SHORT AND MEDIUM- PARTNERSHIP WITH A CLINICALLY VALIDATED TERM NEWSFLOW MECHANISM OF ACTION • BBDF: the leading • Phase 3 US/Europe patient foundation in • Proven ab
Slide 7: CLN3: the mechanism of the disease has been discovered thanks to recent academic work
CLN3: the mechanism of the disease has been discovered thanks to recent academic work financed by BBDF First symptoms between ages 4 and 6 700 to 1 000 800 to 1 000 No treatment registered Loss of patients in the U.S.1 patients in the EU2 visual acuity => blindness Autosomal recessive transmission,
Slide 8: Batten-1 (miglustat) targets glycosphingolipid synthesis, whose accumulation is toxic to neurons
Batten-1 (miglustat) targets glycosphingolipid synthesis, whose accumulation is toxic to neurons GSL reduction by miglustat already validated ..an activity also validated on ...leading to a reduction in humans for other indications... preclinical models in CLN3... of cell death in the brain Signific
Slide 9: Encouraging Phase 1/2 clinical results after 12 months of treatment
Encouraging Phase 1/2 clinical results after 12 months of treatment • 6 patients with juvenile Batten disease, aged 17 and Interim results at 12 months more • Decrease of neurofilament light chain (NFL) in the blood and cerebrospinal fluid of patients dosed • Study conducted by Batten disease refere
Slide 10: Batten-1: Phase 3 study design validated by the FDA and EMA
Batten-1: Phase 3 study design validated by the FDA and EMA FDA and EMA approvals obtained for design and efficacy endpoints of pivotal Phase 3 study Primary endpoint: Visual acuity • Randomized, double-blind study • A single Phase 3 study would Evaluated by : enable registration of the evaluating t
Slide 11: Development plan with Phase 3 results by the end of 2026, followed by marketing
Development plan with Phase 3 results by the end of 2026, followed by marketing authorization 2022 2023 2024 2025 2026 2027 IND granted Dialogue with the FDA Validation of Phase 3 efficacy Filing a marketing application for criteria by the FDA and EMA registration and commercialization P1/2 OPEN- LA
Slide 12: Batten-1: development of a proprietary oral solution for CLN3 patients
Batten-1: development of a proprietary oral solution for CLN3 patients A drinkable solution that makes treatment easier to take and modulate — Drinkable solution : ▶ Suitable for pediatric patients and patients presenting swallowing difficulties. ▶ High concentration, enabling treatment of all patie
Slide 13: Batten-1: global sales peak at over $500 million a year
Batten-1: global sales peak at over $500 million a year Market estimates obtained by an independent study commissioned by A favorable pricing environment for rare Theranexus, demonstrating a strong market opportunity diseases Currently diagnosed Sales based Sales based Territories patients / estimat
Slide 14: Financial information
Financial information
Slide 15: Listing and shareholders
Listing and shareholders Financial data Capital structure • ISIN: FR0013286259 - Mnemo: ALTHX • Market: Euronext Growth • Share price on September 28, 2023: 1.17 € • Market capitalization: 9.1 M€ • Coverage: Portzamparc • Liquidity contract: Portzamparc 15
Slide 16: Simplified income statement
Simplified income statement In thousands of euros (French accounting standards) H1-2023 H1-2022 2022 2021 Operating income 254 462 1,246 25 Other purchases and external charges 2480 1 950 3,967 5,591 Salaries and social charges 1520 1 509 3,165 2,689 Depreciation and amortization 168 166 332 1,204 O
Slide 17: Theranexus: a strong opportunity to create value for investors
Theranexus: a strong opportunity to create value for investors Innovative biopharmaceutical company A dynamic Batten-1 newsflow with many strengths over the next 3 years Batten-1: H1 2024 • A clinical asset approaching Phase 3, with a Launch of the phase 3 pivotal trial mechanism validated in humans