Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — J.P. Morgan 2026 Healthcare Conference
25 slides from Ptc Therapeutics's conference presentation, Jan 12, 2026
Slide 1: PTC Therapeutics 2026
J.P. Morgan 2026 Healthcare Conference PTC Therapeutics 2026 Matthew B. Klein, MD CEO
Slide 2: 2025: A Year of Focus and Execution
J.P. Morgan 2026 Healthcare Conference FDA and EMA approvals of Sephience Advancement of early-stage R&D programs Strong start to Sephience global launch Drive revenue and effectively manage OpEx 2025: A Year of Focus and Execution 2
Slide 3: Forward-looking statements
J.P. Morgan 2026 Healthcare Conference 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 st
Slide 4: 2025 Revenue Performance Exceeded Guidance
J.P. Morgan 2026 Healthcare Conference 2025 Revenue Performance Exceeded Guidance 4 ~$823M* Product Revenue ~$588M * All revenue numbers are unaudited; revenue performance includes product and royalty revenue; royalty revenue is pending accounting treatment of Evrysdi
Slide 5: Sephience Launch Continues Strong Momentum
J.P. Morgan 2026 Healthcare Conference Sephience Launch Continues Strong Momentum 5 $92.5M Q4 Global Revenue* $81.6M US $10.9M ex-US 946 patients on commercial therapy worldwide** 1,134 Patient Start Forms in US** ** As of December 31, 2025 * Unaudited net revenue
Slide 6: 2026 Revenue and OpEx Guidance
J.P. Morgan 2026 Healthcare Conference 2026 Revenue and OpEx Guidance 6 YoY Product Revenue Growth $700 19-36% * -800M * Product revenue guidance excludes Evrysdi royalty and collaboration revenues; YoY based on $700-$800M guidance
Slide 7: 2026 Revenue and OpEx Guidance
J.P. Morgan 2026 Healthcare Conference 2026 Revenue and OpEx Guidance 7 * Product revenue guidance excludes Evrysdi royalty and collaboration revenues ** OpEx is a Non-GAAP measure that includes only R&D and SG&A expenses for full year 2026, and excludes estimated non-cash, stock-based compensation
Slide 8: 2026: Planning for Continued Success
J.P. Morgan 2026 Healthcare Conference 2026: Planning for Continued Success 8 Continue Sephience global launch momentum Move toward reaching cash flow breakeven Initiate votoplam HD Phase 3 trial Advance early-stage R&D programs
Slide 9: Sephience PKU Program
J.P. Morgan 2026 Healthcare Conference 9 Patient living w ith PKU Sephience PKU Program
Slide 10: Sephience Dual Mechanism of Action Supports Potential for Broad Uptake
J.P. Morgan 2026 Healthcare Conference Sephience Dual Mechanism of Action Supports Potential for Broad Uptake 10 * SR, sepiapterin reductase (enzyme that converts Sephience to BH2) ** DHFR, dihydrofolate reductase (enzyme that converts BH2 to BH4) 1. Precursor to BH4 SP Sephience 7.8-Dihydrobiopteri
Slide 11: Sephience Provides Significantly Greater Phe Reduction Compared With BH4
J.P. Morgan 2026 Healthcare Conference Sephience Provides Significantly Greater Phe Reduction Compared With BH4 11 -437.0 -256.6 -600 -500 -400 -300 -200 -100 0 100 Absolute least squares mean change in blood Phe concentration (µmol/L) Sephience BH4 70.3% Greater reduction with Sephience p < 0.0001
Slide 12: Sephience Provides Diet Liberalization, Cognitive and Quality of Life Benefits
J.P. Morgan 2026 Healthcare Conference Sephience Provides Diet Liberalization, Cognitive and Quality of Life Benefits 12 Improved cognitive function and mood Improved concentration & slow thinking, and decreased irritability Meaningful diet liberalization 69% of subjects reached age-adjusted protein
Slide 13: Sephience Differentiated Profile Enables Penetration Into All Key Market Segments
J.P. Morgan 2026 Healthcare Conference Sephience Differentiated Profile Enables Penetration Into All Key Market Segments 13 On Treatment Treatment Failures Treatment Naive 17K Patients in US TOTAL ADDRESSABLE MARKET INCLUDING ALL AGES AND SEVERITIES
Slide 14: 2025: Launch Initiated in US, EU and Canada
J.P. Morgan 2026 Healthcare Conference 2025: Launch Initiated in US, EU and Canada 14 UNIT ED STATES CANADA EUROPE
Slide 15: 2026: Expanding the Sephience Global Launch
J.P. Morgan 2026 Healthcare Conference 15 2026: Expanding the Sephience Global Launch NORTH AMERICA EUROPE ASIA LATAM Anticipated Sephience geographic footprint for 2026
Slide 16: Votoplam Huntington's Disease Program
J.P. Morgan 2026 Healthcare Conference 16 Votoplam Huntington’s Disease Program Patient living w ith HD
Slide 17: Phase 2 Placebo-Controlled Trial Achieved Key Efficacy and Safety Objectives
J.P. Morgan 2026 Healthcare Conference Phase 2 Placebo-Controlled Trial Achieved Key Efficacy and Safety Objectives 17 Long-term positive clinical trends at Month 24 relative to natural history and dose-dependent NfL lowering Continued favorable safety and tolerability profile with no treatment-rela
Slide 18: Votoplam Phase 3 Trial Expected to be Initiated by Novartis in H1 2026
J.P. Morgan 2026 Healthcare Conference Votoplam Phase 3 Trial Expected to be Initiated by Novartis in H1 2026 18 INVEST-HD Global Phase 3 Study • 3:2 randomization of votoplam: placebo • Target enrollment: ~770 participants in >30 countries • Primary endpoint: Change in cUHDRS from Baseline up to 36
Slide 19: Vatiquinone Friedreich's Ataxia Program
J.P. Morgan 2026 Healthcare Conference 19 Vatiquinone Friedreich’s Ataxia Program Patient living w ith FA
Slide 20: Vatiquinone Demonstrated Meaningful Slowing of Short- and Long-term Disease Progression
J.P. Morgan 2026 Healthcare Conference Vatiquinone Demonstrated Meaningful Slowing of Short- and Long-term Disease Progression 20 Vatiquinone Placebo Long-term Extension Studies 4.8-point benefit (p<0.0001) on mFARS** over 2 years in ambulatory and non-ambulatory adults 50% slowing (p<0.0001) of dis
Slide 21: Innovative Research Platforms
J.P. Morgan 2026 Healthcare Conference 21 Innovative Research Platforms
Slide 22: PTSeek Platform Provides Rapid and Reliable Identification of Splicing Therapy Candidates
J.P. Morgan 2026 Healthcare Conference PTSeek Platform Provides Rapid and Reliable Identification of Splicing Therapy Candidates 22 2005 2020 2023 Splicing Foundation Splicing Platform 2025 and beyond Targeted high-throughput screening Transcriptome-wide direct splicing interrogation Novel molecules
Slide 23: R&D Pipeline Includes Innovative Programs from Differentiated Scientific Platforms
J.P. Morgan 2026 Healthcare Conference R&D Pipeline Includes Innovative Programs from Differentiated Scientific Platforms 23 MSH3: Huntington’s Disease, Myotonic Dystrophy I Undisclosed: Neurodegenerative Diseases Undisclosed: Brain Tumors & Metastases Undisclosed: Sickle Cell Disease, β-Thalassemia
Slide 24: Well Positioned for Success in 2026 & Beyond
J.P. Morgan 2026 Healthcare Conference Well Positioned for Success in 2026 & Beyond 24 Strong Financial Position Robust Global Commercial Engine Innovative R&D Platforms
Slide 25: PTC Therapeutics 2026
J.P. Morgan 2026 Healthcare Conference PTC Therapeutics 2026 Matthew B. Klein, MD CEO
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