Table of Contents List of Tables List of Figures Introduction Global Markets Direct Report Coverage Phenylketonuria (PKU) - Overview Phenylketonuria (PKU) - Therapeutics Development Pipeline Overview Pipeline by Companies Products under Development by Companies Phenylketonuria (PKU) - Therapeutics Assessment Assessment by Target Assessment by Mechanism of Action Assessment by Route of Administration Assessment by Molecule Type Phenylketonuria (PKU) - Companies Involved in Therapeutics Development American Gene Technologies International Inc BioMarin Pharmaceutical Inc Censa Pharmaceuticals Inc Codexis Inc EryDel SPA Erytech Pharma SA MipSalus ApS Synlogic Inc Synthetic Biologics Inc Ultragenyx Pharmaceutical Inc Phenylketonuria (PKU) - Drug Profiles CDX-6114 - Drug Profile Product Description Mechanism Of Action R&D Progress CNSA-001 - Drug Profile Product Description Mechanism Of Action R&D Progress DTX-501 - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy for Phenylketonuria - Drug Profile Product Description Mechanism Of Action R&D Progress pegvaliase - Drug Profile Product Description Mechanism Of Action R&D Progress Phelimine - Drug Profile Product Description Mechanism Of Action R&D Progress Recombinant Enzyme for Phenylketonuria - Drug Profile Product Description Mechanism Of Action R&D Progress Recombinant Phenylalanine Ammonia Lyase Replacement for Phenylketonuria - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile Product Description Mechanism Of Action R&D Progress SYN-200 - Drug Profile Product Description Mechanism Of Action R&D Progress SYNB-1618 - Drug Profile Product Description Mechanism Of Action R&D Progress Phenylketonuria (PKU) - Dormant Projects Phenylketonuria (PKU) - Product Development Milestones Featured News & Press Releases Jan 05, 2018: Synlogic Provides Update on its Phenylketonuria Drug candidate SYNB1618 Dec 22, 2017: BioMarin Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28, 2018 Oct 24, 2017: Synlogic Receives Orphan Drug Designation for SYNB1618, a Synthetic Biotic Medicine for the Treatment of Phenylketonuria Sep 14, 2017: FDA Not Currently Planning to Hold Advisory Committee Meeting for BioMarins Pegvaliase Biologics License Application (BLA) Sep 05, 2017: BioMarin To Present Data On Pegvaliase At 13th International Congress of Inborn Errors of Metabolism 2017 Aug 29, 2017: FDA Accepts BioMarins Pegvaliase Biologics License Application and Grants Priority Review Designation Jun 30, 2017: BioMarin Submits Pegvaliase Biologics License Application to the U.S. FDA for Treatment of Phenylketonuria Mar 31, 2016: Promising results with breakthrough treatment of orphan disease PKU Mar 21, 2016: BioMarin Phase 3 Study of Pegvaliase for Phenylketonuria Meets Primary Endpoint of Blood Phenylalanine Reduction (p0.0001) Feb 2

List of Tables Number of Products under Development for Phenylketonuria (PKU), H1 2018 Number of Products under Development by Companies, H1 2018 Products under Development by Companies, H1 2018 Number of Products by Stage and Target, H1 2018 Number of Products by Stage and Mechanism of Action, H1 2018 Number of Products by Stage and Route of Administration, H1 2018 Number of Products by Stage and Molecule Type, H1 2018 Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H1 2018 Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H1 2018 Phenylketonuria (PKU) - Pipeline by Censa Pharmaceuticals Inc, H1 2018 Phenylketonuria (PKU) - Pipeline by Codexis Inc, H1 2018 Phenylketonuria (PKU) - Pipeline by EryDel SPA, H1 2018 Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H1 2018 Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H1 2018 Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H1 2018 Phenylketonuria (PKU) - Pipeline by Synthetic Biologics Inc, H1 2018 Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, H1 2018 Phenylketonuria (PKU) - Dormant Projects, H1 2018 List of Figures Number of Products under Development for Phenylketonuria (PKU), H1 2018 Number of Products under Development by Companies, H1 2018 Number of Products by Targets, H1 2018 Number of Products by Stage and Targets, H1 2018 Number of Products by Mechanism of Actions, H1 2018 Number of Products by Stage and Mechanism of Actions, H1 2018 Number of Products by Routes of Administration, H1 2018 Number of Products by Stage and Routes of Administration, H1 2018 Number of Products by Molecule Types, H1 2018 Number of Products by Stage and Molecule Types, H1 2018

American Gene Technologies International Inc BioMarin Pharmaceutical Inc Censa Pharmaceuticals Inc Codexis Inc EryDel SPA Erytech Pharma SA MipSalus ApS Synlogic Inc Synthetic Biologics Inc Ultragenyx Pharmaceutical Inc