Thx Pharma · EURONEXT:ALTHX
Thx Pharma — Theranexus — Investor Presentation (June 2019)
27 slides from Thx Pharma's investor presentation, June 2019
Slide 1: Investor Presentation
INVESTOR PRESENTATION LABORATOIRE EN 1ÈRE LIGNE CONTRE LES MALADIES NEUROLOGIQUES
Slide 2: Your Contacts
YOUR CONTACTS Franck MOUTHON Thierry LAMBERT Co-founder and Chairman CFO and CEO ● Franck Mouthon holds a degree in ● Thierry Lambert holds a degree in life sciences from the École Normale business administration from Supérieure Birmingham University and an MBA ● Joined the Life Sciences Department
Slide 3: A Disruptive Biotech in Central Nervous System Research
A DISRUPTIVE BIOTECH IN CENTRAL NERVOUS SYSTEM RESEARCH MAJOR ASSETS A POWERFUL TOOL A high-potential diversified portfolio A unique & versatile platform for generating drug candidates Phase 2 for Parkinson’s disease Two other high-need indications: Alzheimer’s disease and neuropathic pain Potential
Slide 4: Diversified Pipeline
DIVERSIFIED PIPELINE Clinical trials Candidate Pre-clinical selection trials Phase I Phase II Phase III Excessive Daytime Sleepiness THN 102 Ph2 results linked to Parkinson’s disease H2-2019 No treatment to date Program viability under 102 Ph2 definitive results Narcolepsy $2 billion THN assessment
Slide 5: Theranexus Platform: Proprietary, Scalable & Versatile
THERANEXUS PLATFORM: PROPRIETARY, SCALABLE & VERSATILE CNS DRUGS GLIAL CELL MODULATOR DRUG SEEN AS DRUG REPOSITIONED THE 1ST LINE-TREATMENT AS A MODULATOR Condition with a strong unmet need for improved efficacy (with the current arsenal of therapeutics) Action Theranexus Optimization CNS drugs libr
Slide 6: NeuroLead: Strengthening the Lead Generation Platform
NEUROLEAD :STRENGTHENING THE LEAD GENERATION PLATFORM A NEW PLATFORM FOR DRUG CANDIDATE GENERATION FOCUSED ON MEDICAL AND INDUSTRIAL VALUE NeuroLead - Development of a drug candidate generating platform A based on neuron-glia interactions soluti on - Prestigious partners: that PLATFORM ADVANTAGES PL
Slide 7: Table of Contents
TABLE OF CONTENTS AN OPPORTUNITY IN BATTEN DISEASE: PARTNERSHIP WITH 1 BEYOND BATTEN DISEASE FOUNDATION 2 ASSETS UNDER CLINICAL DEVELOPMENT 3 FINANCIALS AND NEWSFLOW
Slide 8: Batten disease or juvenile neuronal ceroid lipofuscinosis (NCL3)
Batten disease or juvenile neuronal ceroid lipofuscinosis (NCL3) – A rare genetic disease that is fatal between the ages of 20 and 30 EPIDEMIOLOGY AND PHYSIOPATHOLOGY OF NCL3 Blindness Cognitive decline No registered treatment Diagnosis in children Loss of motor skills c. 3,000 patients Autosomal re
Slide 9: The product – BBDF101 miglustat-trehalose combination
The product – BBDF 101 - miglustat-trehalose combination FOUNDATION BBDF101 Trehalose + Miglustat combination Eligible for Fast Track procedure (MA in 6 months) Eligible for pediatric ODD Ready to enter Phase I/III , Created in 2008 by Craig Benson Pre-IND meeting – Strong Exclusivity for 7.5 years
Slide 10: Competitive environment and market opportunity
Competitive environment and market opportunity COMPARABLES COMPETITION IN CLINICAL DEVELOPMENT 6,000 cases USA 5,000 cases USA 500 cases USA 500 cases USA 5,000 cases EU 1,800 cases EU 400 cases EU 250 cases EU Gaucher disease Pompe disease Hunter syndrome NCL2 NCL3 AAV9 gene therapy Amicus Therapeu
Slide 11: BBDF development plan
BBDF 101 development plan Signing of firm agreement Application for IND Registration of product – the Theranexus plans to market the between BBDF and (authorization to enter FDA has already confirmed product directly. The limited Theranexus (exclusivity of clinical trial phase) that the product woul
Slide 12: Strategic interest of BBDF101 for Theranexus
Strategic interest of BBDF101 for Theranexus SCIENTIFIC - Disease involving neuron/glia interactions CONVERGENCE - A pharmacology acting on both cell types in the brain CONSISTENCY AND - A rare terminal paediatric neurological disease COMPLEMENTARITY OF - A combination of 2 repositioned drugs THE PO
Slide 13: Table of Contents
TABLE OF CONTENTS AN OPPORTUNITY IN BATTEN DISEASE: PARTNERSHIP WITH 1 BEYOND BATTEN DISEASE FOUNDATION 2 ASSETS UNDER CLINICAL DEVELOPMENT 3 FINANCIALS AND NEWSFLOW
Slide 14: THN102: A drug candidate to combat sleepiness related to Parkinson's
THN102: A DRUG CANDIDATE TO COMBAT SLEEPINESS RELATED TO PARKINSON’S MODAFINIL FLECAINIDE THN 102 Parkinson’s disease Phase II clinical trial underway Design: Sleepiness, attention, cognition Double-blind study comparing 2 doses of THN102 Almost 1 million patients (G7) with a placebo in a three peri
Slide 15: THN201: A high-potential candidate for dementia
THN201: A HIGH-POTENTIAL CANDIDATE FOR DEMENTIA DONEPEZIL MEFLOQUINE THN 201 Neurocognitive disorders linked to Alzheimer’s disease Launch of Phase Ib clinical trial Under the CX-COG project funded by the Impaired memory, reasoning and orientation French “Fonds Unique Interministériel” (FUI AAP22) 1
Slide 16: THN101: Drug candidate ready for Phase II trials: pain
THN101: DRUG CANDIDATE READY FOR PHASE II TRIALS: PAIN AMITRIPTYLINE MEFLOQUINE THN 101 Neuropathic pain Preparation stage for Phase II clinical trial Chronic pain with occasional stabbing pain, sensations of burning Key efficacy criteria: pain scale or electric shocks Double-blind randomized study
Slide 17: 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” MULTIPLE OPPO
Slide 18: Strong interest among industry players for the first conditions targeted
STRONG INTEREST AMONG INDUSTRY PLAYERS FOR THE FIRST 3 CONDITIONS TARGETED DEVELOPPEMENT UP FRONT MILESTONES ROYALTIES CONDITION DATE SELLER BUYER PROFILE STAGE (M$) (M$) (M$) Parkinson’s 2018 Prexton Lundbeck NCE Phase II 123 993 - disease 2016 Cynapsus Sunovion LCM Phase III 624 - - Neuropathic 20
Slide 19: Table of Contents
TABLE OF CONTENTS AN OPPORTUNITY IN BATTEN DISEASE: PARTNERSHIP WITH 1 BEYOND BATTEN DISEASE FOUNDATION 2 ASSETS UNDER CLINICAL DEVELOPMENT 3 FINANCIALS AND NEWSFLOW
Slide 20: 2018 P&L
2018 P&L In K€ (French GAAP) 2017 2018 GOOD CONTROL OVER EXPENSES IN A CONTEXT OF Operating income 164 175 ACCELERATED CLINICAL DEVELOPMENTS Other purchases and external 1 477 4 969 charges Salaries and benefits 1 370 2 117 Depreciation and amortization 26 55 Other operating expenses 11 24 Operating
Slide 21: Investors Relations and Shareholders
INVESTORS RELATIONS AND SHAREHOLDERS DATA AS AT 26 JUNE 2019 SHAREHOLDERS ISIN : FR0013286259 - Mnemo: ALTHX 20,5% Founders Listed on Euronext Growth & employess Stock price : 5,3€ (on Jun. 18th 2019) 31,1% Floating Market Cap : 16 M€ Liquidity contract : Portzamparc 14,5% 4,9% Auriga Partners Cash
Slide 22: Publication Schedule for 2019
PUBLICATION SCHEDULE FOR 2019 P2 results: H2-2019 THN 102 P1b results: H2-2019 THN 201 Results of P1a: end of H1-2019 THN 101 Preparation stage of P2: pain H2-2019 IND for BBDF 101 in Batten disease BBDF 101 22
Slide 23: Appendices
OVERVIEW APPENDICES
Slide 24: Clinical development – Example: Brineura (BioMarin) for CLN2
Clinical development – Example: Brineura (BioMarin) for CLN2 CLN2 500 CLN2 cases US – 250 cases EU Phase I/II Pre- registration Diagnostic between 2 and 4 years old Duration: 2 years –extension study on going Autosomal recessive n=23 patients [Enrollment in six months] Dementia, motor deficit, visio
Slide 25: Transforming Research into Innovation
TRANSFORMING RESEARCH INTO INNOVATION PRINCIPLE: APPLICATION: Enhance neuron action with the modulation Combine medication that targets neurons with a of glial cells medication that optimizes neuroglial interaction Non-neural networks Astrocyte The modulation of glial connexins Connexin Connexin XX
Slide 26: Targeting Glial Cells: A Soaring Approach
TARGETING GLIAL CELLS : A SOARING APPROACH APPROACH MECHANISM OF ACTION SOCIETIES Oligodendrocytes & remyelination Microglia & neuroinflammation Astrocytes & metabolism Interactions neurons / Glia 26
Slide 27: Phase IIa trial: Narcolepsy
Phase IIa trial: NARCOLEPSY SAFETY AND EFFICACY OF THN102 IN SLEEPINESS IN NARCOLEPTIC PATIENTS DOUBLE-BLIND MULTICENTER STUDY COMPARING 3 TREATMENTS OVER 5 SITES IN FRANCE AND ONE IN BELGIUM INVESTIGATOR: PROFESSOR YVES DAUVILLIERS, CHU MONTPELLIER MOD300: PLACE STAB DOSE OF 300MG BO OF MODAFINIL P