Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — J.P. Morgan Healthcare Conference 2024
30 slides from Ptc Therapeutics's conference presentation, Jan 8, 2024
Slide 1: PTC Therapeutics – JP Morgan 2024
PTC Therapeutics – JP Morgan 2024 Matthew B. Klein, MD CEO PTC Therapeutics 2024 Patient Living with PKU
Slide 2: Forward-looking statements
PTC Therapeutics – JP Morgan 2024 This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including stateme
Slide 3: 2023
3 PTC Therapeutics – JP Morgan 2024 2023
Slide 4: Building the PTC of the Future
4 PTC Therapeutics – JP Morgan 2024 Building the PTC of the Future Focus Right Sizing OPEX Reduction Royalty Financing
Slide 5: PTC Mission Remains Patient Focused
PTC Therapeutics – JP Morgan 2024 PTC Mission Remains Patient Focused 5 Discover Develop Commercialize Transformative therapies for patients with rare diseases
Slide 6: Strategy Leverages Innovative Science
PTC Therapeutics – JP Morgan 2024 PTC Strategy Leverages Innovative Science, Passionate Team and Strong Cash Position 6 Innovative Science Ferroptosis and Inflammation Splicing
Slide 7: Strategy Leverages Therapeutic Expertise
PTC Therapeutics – JP Morgan 2024 PTC Strategy Leverages Innovative Science, Passionate Team and Strong Cash Position 7 Therapeutic Neurology Metabolism Expertise* Innovative Science * Unesbulin (PTC596) development discontinued
Slide 8: Global Commercial Infrastructure
PTC Therapeutics – JP Morgan 2024 PTC Strategy Leverages Innovative Science, Passionate Team and Strong Cash Position 8 Global Commercial Infrastructure Therapeutic Expertise* Innovative Science Europe United States Asia Pacific Latin America Middle East & North Africa
Slide 9: Robust Portfolio to Support Growth & Value Creation
PTC Therapeutics – JP Morgan 2024 Robust Portfolio to Support Growth & Value Creation 9 Research Splicing Platform Ferroptosis and Inflammation Platform SCA-3 MAP-tau Undisclosed (Movement Disorders) Undisclosed (Neurodegenerative Diseases) Undisclosed (Pediatric Neurodevelopment Disorders) Developm
Slide 10: Strong Commercial Performance and Growth in 2023
PTC Therapeutics – JP Morgan 2024 Unaudited DMD Franchise Net Product Revenue ~$610M Strong Commercial Performance and Growth in 2023 10 2023 Unaudited Total Revenue ~$946M
Slide 11: Strong R&D Execution and Value Creation in 2023
PTC Therapeutics – JP Morgan 2024 Strong R&D Execution and Value Creation in 2023 11 *Primary endpoint did not meet statistical significance Positive interim data for PIVOT-HD Highly statistically significant and meaningful APHENITY results Positive upright stability and fatigue scale results for MO
Slide 12: 2024 Revenue and OPEX Guidance
PTC Therapeutics – JP Morgan 2024 2024 Revenue and OPEX Guidance *Non-GAAP measure which excludes estimated non-cash, stock-based compensation expense of approximately $80 million. GAAP R&D and SG&A expense for the full year 2024 is anticipated to be 12 between $740 and $835 million. The Company ant
Slide 13: Key Expected Regulatory & Clinical Milestones in 2024
PTC Therapeutics – JP Morgan 2024 Key Expected Regulatory & Clinical Milestones in 2024 13 Q1 Q2 Q3 Q4 Sepiapterin PKU MAA Submission Sepiapterin PKU NDA Submission Upstaza BLA Submission t Topline Results Utreloxastat Interim Results PTC518
Slide 14: Sepiapterin PKU Program
PTC Therapeutics – JP Morgan 2024 Sepiapterin PKU Program Patient Living with PKU
Slide 15: APHENITY Results Demonstrate Meaningful Benefit of Sepiapterin
15 PTC Therapeutics – JP Morgan 2024 Phe, phenylalanine; PKU, phenylketonuria. APHENITY Results Demonstrate Meaningful Benefit of Sepiapterin in PKU Patients The primary endpoint was reached in the placebo-controlled portion of the study with statistically significant reductions in blood Phe levels
Slide 16: Sepiapterin Enables Increased Dietary Phe Intake
PTC Therapeutics – JP Morgan 2024 0 50 100 150 200 Baseline W1-W2 W3-W4 W5-W6 W7-W8 W9-W10 W11-W12 W13-W14 W15-W16 W17-W18 W19-W20 W21-W22 W23-W24 W25-W26 RDA* 40 Dietary Phe Intake and Blood Phe Levels Over Time Phe Consumption (mg/kg/day) 16 Sepiapterin Enables Increased Dietary Phe Intake in PKU
Slide 17: APHENITY Results Support Potential to Address Broad PKU Population
PTC Therapeutics – JP Morgan 2024 APHENITY Results Support Potential for Sepiapterin to Address Broad PKU Population 17 Therapy-Naive Patients Including Classical PKU Patients Who Are Not Well Controlled by Current Therapies Patients Who Have Failed on Current Therapies Greater than $1 Billion Poten
Slide 18: Global Regulatory Submissions and Launch Preparation Planned for 2024
PTC Therapeutics – JP Morgan 2024 Global Regulatory Submissions and Launch Preparation Planned for 2024 18 Global Regulatory Submissions Global Launch Sequence and Continued Launch Preparation
Slide 19: PTC518 HD Program
PTC Therapeutics – JP Morgan 2024 PTC518 HD Program
Slide 20: Key Attributes of PTC518 Drive Differentiation
PTC Therapeutics – JP Morgan 2024 Key Attributes of PTC518 Drive Differentiation 20 Orally bioavailable Titratable and reversible Reduces HTT mRNA and protein in the CNS and periphery Uniform lowering in key regions of the brain Highly selective and specific Not effluxed, penetrates blood brain barr
Slide 21: PIVOT-HD Study Design
PTC Therapeutics – JP Morgan 2024 PIVOT-HD Study Design 21 3 months 9 months 5 mg PTC518 R 10 mg PTC518 N = ~30 N = ~30 N = ~30 Part A Part B Placebo 5 mg PTC518 10 mg PTC518 Primary Endpoints • Safety and tolerability of PTC518 • Percent reduction in HTT mRNA and protein in blood Secondary Endpoint
Slide 22: 12-Week Interim Data Met Key Objectives
22 PTC Therapeutics – JP Morgan 2024 12-Week Interim Data Met Key Objectives PTC518 treatment resulted in dose-dependent lowering of HTT mRNA and protein levels in blood cells PTC518 demonstrated desired CSF exposure with higher concentrations of free drug in the CSF than plasma PTC518 was well tole
Slide 23: Month Data Update to Include Blood, CSF and Radiographic Biomarkers
PTC Therapeutics – JP Morgan 2024 12 Month Data Update to Include Blood, CSF and Radiographic Biomarkers 23 Safety and tolerability of PTC518 Percent reduction in HTT mRNA and protein in blood Percent reduction in mHTT protein in CSF Changes in neurofilament light chain (NfL) in plasma and CSF Chang
Slide 24: Vatiquinone FA Program
PTC Therapeutics – JP Morgan 2024 Vatiquinone FA Program Patient Living with FA
Slide 25: Meaningful Clinical Results in MOVE-FA
PTC Therapeutics – JP Morgan 2024 Meaningful Clinical Results in MOVE-FA 25 *Study primary endpoint Change from Baseline to Week 72 Analysis Placebo Vatiquinone Difference P-value mFARS Total* 2.83 1.22 -1.61 0.144 Upright Stability 2.99 1.73 -1.26 0.021 Bulbar 0.22 0.040 -0.18 0.044 Fatigue Scale (
Slide 26: Utreloxastat ALS Program
PTC Therapeutics – JP Morgan 2024 Utreloxastat ALS Program
Slide 27: Evidence Confirm Link Between ALS and Ferroptosis Pathway
PTC Therapeutics – JP Morgan 2024 Preclinical and Clinical Evidence Confirm Link Between ALS and Ferroptosis Pathway 27 Iron accumulation, a marker of ferroptosis, within spinal cord lesions has been reported as an early event in ALS pathogenesis1 Fe3+ Oxidized lipids and the 15-lipoxygenase end-pro
Slide 28: CardinALS Study Design
PTC Therapeutics – JP Morgan 2024 CardinALS Study Design 28 Key Endpoints • Change in ALS-FRS Scale • Respiratory Function • Survival Utreloxastat 250 mg BID Utreloxastat 250 mg BID 24 Weeks 28 Weeks Placebo-Controlled Phase Open-Label Extension Phase Placebo Study Timeline • Enrollment Completed: Q
Slide 29: 2023
29 PTC Therapeutics – JP Morgan 2024 2023
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30 PTC Therapeutics – JP Morgan 2024
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