Thx Pharma · EURONEXT:ALTHX
Thx Pharma — Theranexus — Investor Access Presentation, September 27th-28th 2021
25 slides from Thx Pharma's conference presentation, Sep 27, 2021
Slide 1: Investor Access Presentation
INVESTOR ACCESS PRESENTATION September 27th-28th , 2021
Slide 2: Theranexus: A Uniquely Positioned Biotech in the CNS Space
THERANEXUS: A UNIQUELY POSITIONNED BIOTECH IN THE CNS SPACE Our speakers Franck Mouthon CEO & founder Thierry Lambert CFO Top researcher at leading research 5 years in Transaction Services with PWC UK organization CEA ACA-trained (Institute of Chartered Co-founder of Theranexus Accountants in Englan
Slide 3: A Strong and Diversified Clinical Pipeline
A STRONG AND DIVERSIFIED CLINICAL PIPELINE Candidate Pre-clinical Clinical trials selection trials Phase I Phase II Phase III A phase 2 –Parkinson’s Disease asset with positive clinical efficacy data in EDS Excessive Daytime Sleepiness Ph2a results THN 102 linked to Parkinson’s disease Published Q1-
Slide 4: Agenda
AGENDA 1 THN102 (PARKINSON’S DISEASE) 2 BBDF-101 (JUVENILE BATTEN DISEASE) 3 NEWSFLOW 4 FINANCIAL SITUATION
Slide 5: THN102 Clinical Development Success
THN102 CLINICAL DEVELOPMENT SUCCESS THN102 MODAFINIL: FLECAINIDE: Wake-promoting drug Drug Repurposed on a Registered in narcolepsy novel target Originally cardiology drug and Obstructive Sleep (used here at a low dose) as Apnea OPTIMIZED EFFICACY astrocytic network modulator P1b sleep ✓ P2a EDS in
Slide 6: THN102 Is a Uniquely Positioned Asset in EDS in Parkinson's Disease
THN102 IS A UNIQUELY POSITIONNED ASSET IN EDS IN PARKINSON’S DISEASE Excessive daytime sleepiness in More than 2 million patients (G7) Parkinson‘s disease One of the most debilitating symptoms of the disease 73 • The risk of falls increases by 20% per unit change on the ESS** in PD patients 40% % %
Slide 7: THN102 in EDS in Parkinson's Disease: Study Design
THN102 IN EDS IN PARKINSON’S DISEASE: STUDY DESIGN Randomised, double-blind, placebo-controlled, complete 3-way cross-over phase IIa trial to investigate safety and efficacy of two THN102 doses in subjects with excessive daytime sleepiness associated with Parkinson’s disease, PI: Prof JC Corvol, ICM
Slide 8: THN102 in EDS in Parkinson's Disease Patients: Clear Superiority vs. Placebo
THN102 IN EDS IN PARKINSON’S DISEASE PATIENTS: CLEAR SUPERIORITY VS. PLACEBO • Excessive daytime sleepiness (EDS) is assessed using the Epworth Sleepiness Scale (ESS) • The « normal » range of ESS scores is up to 10. ESS scores of 11-24 represent increasing levels of excessive daytime sleepiness (Jo
Slide 9: THN102 in EDS in Parkinson's Disease Patients: Improved Remission Rate
THN102 IN EDS IN PARKINSON’S DISEASE PATIENTS: IMPROVED REMISSION RATE WITH THN102 • Remission is generally defined as ESS< 11, as it is reported that the « normal » range of ESS scores is up to 10 (Johns, 1991 ; Chen at al, 1995 ; Johns and Hocking, 2004 ; Manni et al, 1999 ; Izci et al, 2008) Abse
Slide 10: THN102 in EDS in Parkinson's Disease Patients: A Large Market Potential
THN102 IN EDS IN PARKINSON’S DISEASE PATIENTS: A LARGE MARKET POTENTIAL 1 - A large patient pool 4 – No treatment currently on the market 73 73% convinced of 5 – Favourable pricing benchmarks 2 - KOLs40% already % the medical needs Typical prices > 10k$ per patient p.a. in the US % OF KOLS(1) INTERV
Slide 11: Partnership Strategy for THN102
THN102: PARTNERSHIP STRATEGY FOR THN102 Clinical trials Candidate Pre-clinical selection trials Phase I Phase II Phase III Market and dimension Excessive Daytime Sleepiness THN 102 linked to Parkinson’s disease No treatment to date Specialists in EDS or CNS Generalists and “big pharma” INTRINSIC COM
Slide 12: Agenda
AGENDA 1 THN102 (PARKINSON’S DISEASE) 2 BBDF-101 (JUVENILE BATTEN DISEASE) 3 NEWSFLOW 4 FINANCIAL SITUATION
Slide 13: BBDF-101: Discovery and Development Until the Agreement Between Theranexus and the Foundation
BBDF-101 : DISCOVERY AND DEVELOPMENT UNTIL THE AGREEMENT BETWEEN THERANEXUS AND THE FOUNDATION EPIDEMIOLOGY AND PHYSIOPATHOLOGY OF NCL3 Blindness Cognitive decline No registered treatment Diagnosis in children Loss of motor skills c. 3,000 patients Autosomal recessive (all NCL types) aged 4 to 8 Dis
Slide 14: BBDF-101 Ambition: Reduce Neuronal Death and Slow the Progress of the Disease
BBDF-101 AMBITION : REDUCE NEURONAL DEATH AND SLOW THE PROGRESS OF THE DISEASE Discovery by Dr Sardiello of Baylor College of Medicine NCL3 mutation Lysosomal Glucosyltransferase Autophagy Apoptosis functions BBDF 101 TFEB Miglustat Trehalose AKT NCL3 transgenic mice ↑ Cell survival Multiple article
Slide 15: Regulatory Achievements and Development Since Agreement with BBDF
REGULATORY ACHIEVMENTS AND DEVELOPMENT SINCE AGREEMENT WITH BBDF ✓ FDA validation of preclinical plan ✓ Waiver to file IND based on interim report of 9-months Orphan drug Ph1-2 vivo studies designation * Q4-2021 Type-C meeting & RPDD** ✓ RPDD secured in US Obtaining the IND ✓ and ODD in US & EU Sept
Slide 16: BBDF-101: Phase I-III Pivotal Program
BBDF-101 : PHASE I-III PIVOTAL PROGRAM • Adolescent/adult cohort of 6 patients over a period of 5 month : Ph1-2 (launching Q4 2021) • Pediatric cohort of 30 patients over a period of two years with an intermediate assessment at 12 month : Ph3 (launch H2-2022) • Open label: Evaluation based on compar
Slide 17: Competitive Environment and Market Opportunity
COMPETITIVE ENVIRONMENT AND MARKET OPPORTUNITY COMPARABLES COMPETITION IN CLINICAL DEVELOPMENT NCL3 AAV9 gene therapy (Amicus Therapeutics) • Aim = treat very young patients (3-10 yeas old) 6,000 cases USA 5,000 cases USA 500 cases USA 500 cases USA • Currently in P1/2 (completion expected Dec 2022)
Slide 18: Agenda
AGENDA 1 THN102 (PARKINSON’S DISEASE) 2 BBDF-101 (JUVENILE BATTEN DISEASE) 3 NEWSFLOW 4 FINANCIAL SITUATION
Slide 19: A Strong Newsflow Over the Next Months
A STRONG NEWSFLOW OVER THE NEXT 12 MONTHS THN 102 THN102 partnership agreement First patient In Ph1-2 : Q4-2021 BBDF 101 Launch of Ph3 : H2-2022 Continuing programs stemming from the discovery platform THN XX 19
Slide 20: Agenda
AGENDA 1 THN102 (PARKINSON’S DISEASE) 2 BBDF-101 (JUVENILE BATTEN DISEASE) 3 NEWSFLOW 4 FINANCIAL SITUATION
Slide 21: P&L 2020; Cash at June 30, 2021
P&L 2020 In K€ (french GAAP) 2019 2020 Operating income 617 315 REDUCED EXPENSES : END OF CLINICAL STUDIES ESPECIALLY ON THN102 Other purchases and external charges 5 426 3 568 Salaries and benefits 2 353 2 422 Depreciation and amortization 154 376 Other operating expenses 61 48 Operating result (7
Slide 22: Capital Markets Snapshot
CAPITAL MARKETS SNAPSHOT FINANCIAL DATA SHAREHOLDERS ISIN : FR0013286259 - Mnemo: ALTHX Number of shares : 4 686 513 Market : Euronext Growth 15.6% Mgt & employees Stock price as at Sept. 22th, 2021 : €8.2 Market cap : €38.4m 56.7% Free-float Brokers coverage : Bryan, Garnier & Co, Portzamparc, ODDO
Slide 23: Appendices
“—\”. Ra 4 AGENDA “> APPENDICES
Slide 24: NeuroLead: Strengthening the Lead Generation Platform
NEUROLEAD : STRENGTHENING THE LEAD GENERATION PLATFORM A NEW PLATFORM FOR DRUG CANDIDATE GENERATION FOCUSED NeuroLead ON MEDICAL AND INDUSTRIAL VALUE - Development of a drug candidate generating platform based on neuron-glia interactions - Prestigious partners: PLATFORM PLATFORM FIRST GENERATION Neu
Slide 25: Theranexus Organisation
THERANEXUS ORGANISATION Franck Mouthon CEO & founder Top researcher at leading research organization CEA 19 employees, mostly R&D Co-founder of Theranexus scientists, clinical operations President of France Biotech managers and business developers Mathieu Charvériat CSO & founder In-house vitro capa
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