Table of Contents Table of Contents 2 List of Tables 4 List of Figures 4 Introduction 5 Global Markets Direct Report Coverage 5 Charcot-Marie-Tooth Disease Type I A Overview 6 Therapeutics Development 7 Pipeline Products for Charcot-Marie-Tooth Disease Type I A - Overview 7 Pipeline Products for Charcot-Marie-Tooth Disease Type I A - Comparative Analysis 8 Charcot-Marie-Tooth Disease Type I A - Therapeutics under Development by Companies 9 Charcot-Marie-Tooth Disease Type I A - Therapeutics under Investigation by Universities/Institutes 10 Charcot-Marie-Tooth Disease Type I A - Pipeline Products Glance 11 Late Stage Products 11 Early Stage Products 12 Charcot-Marie-Tooth Disease Type I A - Products under Development by Companies 13 Charcot-Marie-Tooth Disease Type I A - Products under Investigation by Universities/Institutes 14 Charcot-Marie-Tooth Disease Type I A - Companies Involved in Therapeutics Development 15 Addex Therapeutics Ltd 15 Affectis Pharmaceuticals AG 16 Genzyme Corp 17 Lead Discovery Center GmbH 18 Pharnext SA 19 Charcot-Marie-Tooth Disease Type I A - Therapeutics Assessment 20 Assessment by Monotherapy Products 20 Assessment by Combination Products 21 Assessment by Target 22 Assessment by Mechanism of Action 24 Assessment by Route of Administration 26 Assessment by Molecule Type 27 Drug Profiles 29 (baclofen + naltrexone hydrochloride + sorbitol) - Drug Profile 29 Product Description 29 Mechanism Of Action 29 R&D Progress 29 ADX-71441 - Drug Profile 32 Product Description 32 Mechanism Of Action 32 R&D Progress 32 AFC-5128 - Drug Profile 36 Product Description 36 Mechanism Of Action 36 R&D Progress 36 Small Molecules for Charcot-Marie-Tooth Disease Type I A - Drug Profile 37 Product Description 37 Mechanism Of Action 37 R&D Progress 37 Small Molecules to Inhibit PMP22 for Charcot-Marie-Tooth Type 1 A - Drug Profile 38 Product Description 38 Mechanism Of Action 38 R&D Progress 38 Stem Cell Therapy for Charcot-Marie-Tooth Disease Type 1A - Drug Profile 39 Product Description 39 Mechanism Of Action 39 R&D Progress 39 Charcot-Marie-Tooth Disease Type I A - Dormant Projects 40 Charcot-Marie-Tooth Disease Type I A - Product Development Milestones 41 Featured News & Press Releases 41 Dec 20, 2016: Pharnext Completes Patient Enrollment in International Pivotal Phase 3 Trial of PXT3003 for Charcot-Marie-Tooth Disease Type 1A 41 Nov 17, 2016: Pharnext Announces that the Data Safety Monitoring Board Recommends Continuing the Ongoing Phase 3 Trial of PXT3003 for Charcot-Marie-Tooth Disease Type 1A 42 Oct 04, 2016: Pharnext Announces Support of the First Patient-Centered Charcot-Marie-Tooth Summit Organized by the Hereditary Neuropathy Foundation 42 Sep 01, 2016: Pharnext’s PXT3003 Featured in Multiple Presentations at the 6th International Charcot-Marie-Tooth and Related Neuropathy Consortium (CMTR) Meeting 43 Apr 25, 2016: Pharnext Announces the Opening of the First U.S. Study Site for its PLEO-CMT International Pivotal Phase 3 Trial in Charcot-Marie-Tooth Disease Type 1A 43 Feb 16, 2016: Pharnext Supports the Fight Against Rare Diseases through Development of Pleotherapy-based Treatments 44 Dec 15, 2015: Pharnext Initiates PLEO-CMT, an International Pivotal Phase 3 Trial, in Charcot-Marie-Tooth disease type 1A 44 Jun 30, 2015: Pharnext's PXT-3003 Featured in Multiple Presentations at 2015 Peripheral Nerve Society Biennial Meeting 45 Dec 17, 2014: Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A through Two Peer-Reviewed Publications 46 Oct 21, 2014: Addex And The Charcot-Marie-Tooth Association Enter Collaboration To Advance ADX71441 In Charcot-Marie-Tooth 1A disorder 47 Jun 23, 2014: The EMA and FDA Grant Orphan Drug Designation to Pharnext's PXT-3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A 48 Apr 24, 2013: Addex Receives Approval To Initiate Phase I First-In-man Clinical Study For ADX71441 48 Jan 22, 2013: Addex Receives Broad US Composition Of Matter Patent For ADX71441 49 Jan 07, 2013: Addex Therapeutics Announces Positive Data With ADX71441 In Pre-Clinical Transgenic Model Of Charcot-Marie-Tooth 1A Disease 49 Nov 22, 2011: Pharnext Completes Patient Recruitment For Phase II Study Of PXT3003 In Type 1A Charcot-Marie-Tooth Disease 50 Appendix 51 Methodology 51 Coverage 51 Secondary Research 51 Primary Research 51 Expert Panel Validation 51 Contact Us 51 Disclaimer 5

List of Tables Number of Products under Development for Charcot-Marie-Tooth Disease Type I A, H2 2016 7 Number of Products under Development for Charcot-Marie-Tooth Disease Type I A - Comparative Analysis, H2 2016 8 Number of Products under Development by Companies, H2 2016 9 Number of Products under Investigation by Universities/Institutes, H2 2016 10 Comparative Analysis by Late Stage Development, H2 2016 11 Comparative Analysis by Early Stage Development, H2 2016 12 Products under Development by Companies, H2 2016 13 Products under Investigation by Universities/Institutes, H2 2016 14 Charcot-Marie-Tooth Disease Type I A - Pipeline by Addex Therapeutics Ltd, H2 2016 15 Charcot-Marie-Tooth Disease Type I A - Pipeline by Affectis Pharmaceuticals AG, H2 2016 16 Charcot-Marie-Tooth Disease Type I A - Pipeline by Genzyme Corp, H2 2016 17 Charcot-Marie-Tooth Disease Type I A - Pipeline by Lead Discovery Center GmbH, H2 2016 18 Charcot-Marie-Tooth Disease Type I A - Pipeline by Pharnext SA, H2 2016 19 Assessment by Monotherapy Products, H2 2016 20 Assessment by Combination Products, H2 2016 21 Number of Products by Stage and Target, H2 2016 23 Number of Products by Stage and Mechanism of Action, H2 2016 25 Number of Products by Stage and Route of Administration, H2 2016 26 Number of Products by Stage and Molecule Type, H2 2016 28 Charcot-Marie-Tooth Disease Type I A - Dormant Projects, H2 2016 40 List of Figures Number of Products under Development for Charcot-Marie-Tooth Disease Type I A, H2 2016 7 Number of Products under Development for Charcot-Marie-Tooth Disease Type I A - Comparative Analysis, H2 2016 8 Number of Products under Development by Companies, H2 2016 9 Comparative Analysis by Early Stage Products, H2 2016 12 Assessment by Monotherapy Products, H2 2016 20 Assessment by Combination Products, H2 2016 21 Number of Products by Targets, H2 2016 22 Number of Products by Stage and Targets, H2 2016 22 Number of Products by Mechanism of Actions, H2 2016 24 Number of Products by Stage and Mechanism of Actions, H2 2016 24 Number of Products by Stage and Routes of Administration, H2 2016 26 Number of Products by Molecule Types, H2 2016 27 Number of Products by Stage and Molecule Types, H2 2016 27

Addex Therapeutics Ltd Affectis Pharmaceuticals AG Genzyme Corp Lead Discovery Center GmbH Pharnext SA