Ptc Therapeutics, Inc. · NASDAQ:PTCT

Ptc Therapeutics — Investor Presentation (July 2026)

28 slides from Ptc Therapeutics's investor presentation, July 2026

Slide 2: Forward Looking Statements
Forward Looking Statements 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 with respect t
Slide 3: PTC: A Global Leader in Rare Disease Drug Development and Commercialization
PTC: A Global Leader in Rare Disease Drug Development and Commercialization Robust Global Lead product Innovative R&D Strong Financial Commercial Sephience to Platforms and Position Infrastructure support near- and Pipeline mid-term growth Proven track record Strong initial global Pioneered field of
Slide 4: 2026 Strategic Priorities
2026 Strategic Priorities Continue Advance Sephience Advance Move toward Phase 3 global launch early-stage reaching cash votoplam HD momentum R&D programs flow breakeven program © 2026 PTC Therapeutics. All rights reserved. 4
Slide 5: 2026 Revenue and OpEx Guidance
2026 Revenue and OpEx Guidance Product Revenue 1 $850 - 950M Our Products4 45 - 62% YoY Growth Total Revenue2 $1.18 - 1.28B OpEx 3 $680 - 720M 1 Product revenue excludes Evrysdi royalty and collaboration revenues; YoY based on $850-950M guidance. 2 Total revenue includes Evrysdi royalty and collabor
Slide 7: Sephience Dual Mechanism of Action Supports Potential for Broad Uptake
Sephience Dual Mechanism of Action Supports Potential for Broad Uptake 1. Precursor to BH4 2. Independent Chaperone Function Misfolded Chaperone SR* PAH Enzyme Effect SP 7.8-Dihydrobiopterin (BH2) Sephience DHFR** SP BH4 BH4 BH4 Decreased Increased Enzyme Enzyme Activity Activity Tetrahydrobiopterin
Slide 8: Sephience Provides Significantly Greater Phe Reduction Compared With BH4
Sephience Provides Significantly Greater Phe Reduction Compared With BH4 Mean Absolute Change in Blood Phe from Baseline 100 Absolute least squares mean change in blood Phe concentration (µmol/L) Sephience BH4 0 -437.0 -256.6 -100 -200 -300 70.3% Greater reduction -400 with Sephience -500 p < 0.0001
Slide 9: Sephience Provides Diet Liberalization, Cognitive and Quality of Life Benefits
Sephience Provides Diet Liberalization, Cognitive and Quality of Life Benefits Meaningful diet Improved cognitive Improved liberalization function and mood quality of life 69% of subjects Improved concentration Reduced disease reached age- & slow thinking, and impact on emotional, adjusted protein R
Slide 10: Sephience Differentiated Profile Enables Penetration Into All Key Market Segments
Sephience Differentiated Profile Enables Penetration Into All Key Market Segments On Treatment Treatment Failures Treatment Naive T O TA L A D D R E S S A B L E M A R K E T INCLUDING ALL AGES AND SEVERITIES 58K Patients WW © 2026 PTC Therapeutics. All rights reserved. 10
Slide 11: Sephience: Strong Launch Momentum Continues
Sephience: Strong Launch Momentum Continues $151M 1,647 Patients Q2 2026 on commercial Global Revenue therapy worldwide* * As of June 30, 2026 © 2026 PTC Therapeutics. All rights reserved. 11
Slide 12: Sephience Global Launch Footprint Expansion Planned for 2026 and 2027
Sephience Global Launch Footprint Expansion Planned for 2026 and 2027 NORTH EUROPE ASIA AMERICA L AT A M Anticipated Sephience geographic footprint for 2026 © 2026 PTC Therapeutics. All rights reserved. 12
Slide 14: Phase 2 PIVOT-HD Trial Achieved Key Efficacy and Safety Objectives
Phase 2 PIVOT-HD Trial Achieved Key Efficacy and Safety Objectives PIVOT-HD study met Evidence of Continued evidence primary endpoint dose-dependent of favorable of blood HTT protein disease slowing safety profile lowering at Week 12 on cUHDRS in Stage 2 with no new AE with durable dose- participant
Slide 15: Dose-dependent Slowing of Disease Progression on cUHDRS
Dose-dependent Slowing of Disease Progression on cUHDRS at 24 months in Stage 2 Participants 0 Baseline of Composite UHDRS Mean (+/-SE) Change from -0.5 Votoplam Mean difference in 10 mg cUHDRS progression of Worsening Votoplam 52% (10mg) and 28% (5mg) -1.0 5 mg at Month 24 NH Cohort -1.5 Baseline 1
Slide 16: INVEST-HD Global Phase 3 Trial Initiated by Novartis
INVEST-HD Global Phase 3 Trial Initiated by Novartis Study Overview • Individuals with early symptomatic disease • 3:2 randomization of votoplam 10mg: placebo Finalizing plan to engage with FDA • Target enrollment: ~770 participants in >30 countries in 2H 2026 to • Primary endpoint: Change from base
Slide 18: Vatiquinone Demonstrated Meaningful Slowing of Short- and Long-term Disease Progression
Vatiquinone Demonstrated Meaningful Slowing of Short- and Long-term Disease Progression 72-week Placebo-Controlled MOVE-FA Trial* Long-term Extension Studies Mean change from Baseline in mFARS 50% slowing (p<0.0001) of Upright Stability score (+/- SE) Vatiquinone Placebo disease progression over 3 y
Slide 19: PROVE-FA Phase 3 Open-Label Study to Support NDA Resubmission
PROVE-FA Phase 3 Open-Label Study to Support NDA Resubmission Initiating in Q3 2026 Study Overview • Open-label study using matched natural history cohort from FACOMS disease registry • Target enrollment: 120 individuals with FA ages 7 to 21 • Primary endpoint: Change in mFARS from baseline to 24 mo
Slide 21: R&D Pipeline Includes Innovative Programs from Differentiated Scientific Platforms
R&D Pipeline Includes Innovative Programs from Differentiated Scientific Platforms Oral Small Molecule Inflammation/ Splicing Platform Ferroptosis Platform MSH3: Huntington’s Disease, Myotonic Dystrophy I Ferroptosis: Multisystem Atrophy (MSA) & Parkinson’s Disease Undisclosed: Brain Tumors & Metast
Slide 22: PTC303 MSH3 Small Molecule Splicing Program
PTC303 MSH3 Small Molecule Splicing Program • MSH3 is a genetic modifier of repeat expansion disorders such as HD and DM1 • Validated correlation between MSH3 reduction and HD onset and progression, supporting therapeutic potential of MSH3 reduction • PTC303 to enter clinic in 2027Redcution in MSH3
Slide 23: PTC612 NLRP3 Inhibitor Program
PTC612 NLRP3 Inhibitor Program • NLRP3 is an intracellular sensor protein that detects infection, stress or tissue damage • Upon detection, NLRP3 is activated, triggering the assembly and activation of the NLRP3 inflammasome leading to elevated levels of IL-1β and IL-18 • Aberrant NLRP3 activation i
Slide 24: PTC612: Greater Potency & Selectivity Compared to Other NLRP3 Inhibitors
PTC612: Greater Potency & Selectivity Compared to Other NLRP3 Inhibitors PTC612 Demonstrates >10x Potency and Selectivity vs Other NLRP3 Inhibitors Assay PTC612 Selnoflast1 DFV8902 Usnoflast3 AZD41444 THP-1 0.99 220 13 13 23-82 IL1β IC50 (nM) Assay PTC612 Vent-015 DFV8902 VTX-32326 VTX-27356 hWB 9.7
Slide 25: PTC844 DHODH Inhibitor Program
PTC844 DHODH Inhibitor Program • DHODH plays a critical role in inflammation by regulating the proliferation of immune cells and modulating cytokine production • Marketed DHODH inhibitors have limitations due to lack of specificity and associated toxicity • PTC844 is a more potent and selective DHOD
Slide 26: PTC844 Demonstrates Superior Potency and Selectivity Compared to Other DHODH Inhibitors
PTC844 Demonstrates Superior Potency and Selectivity Compared to Other DHODH Inhibitors PTC844 Vidofludimus Teriflunomide IC50=1nM IC50~5000nM IC50>10,000nM [nM] [nM] [nM] Phase 2a PK/PD study of PTC844 initiating Q3 2026 © 2026 PTC Therapeutics. All rights reserved. 26
Slide 27: Well Positioned for Success in 2026 & Beyond
Well Positioned for Success in 2026 & Beyond Lead product Robust Global Sephience Innovative Strong Commercial to support near- R&D Platforms Financial Infrastructure and mid-term and Pipeline Position growth © 2026 PTC Therapeutics. All rights reserved. 27

PTC Therapeutics — Investor Presentation (July 2026) on 6ix

Ptc Therapeutics — Investor Presentation (July 2026)

Slide 1: PTC Therapeutics

Slide 2: Forward Looking Statements

Slide 3: PTC: A Global Leader in Rare Disease Drug Development and Commercialization

Slide 4: 2026 Strategic Priorities

Slide 5: 2026 Revenue and OpEx Guidance

Slide 6: Sephience

Slide 7: Sephience Dual Mechanism of Action Supports Potential for Broad Uptake

Slide 8: Sephience Provides Significantly Greater Phe Reduction Compared With BH4

Slide 9: Sephience Provides Diet Liberalization, Cognitive and Quality of Life Benefits

Slide 10: Sephience Differentiated Profile Enables Penetration Into All Key Market Segments

Slide 11: Sephience: Strong Launch Momentum Continues

Slide 12: Sephience Global Launch Footprint Expansion Planned for 2026 and 2027

Slide 13: Votoplam

Slide 14: Phase 2 PIVOT-HD Trial Achieved Key Efficacy and Safety Objectives

Slide 15: Dose-dependent Slowing of Disease Progression on cUHDRS

Slide 16: INVEST-HD Global Phase 3 Trial Initiated by Novartis

Slide 17: Vatiquinone

Slide 18: Vatiquinone Demonstrated Meaningful Slowing of Short- and Long-term Disease Progression

Slide 19: PROVE-FA Phase 3 Open-Label Study to Support NDA Resubmission

Slide 20: Innovative Research Platforms

Slide 21: R&D Pipeline Includes Innovative Programs from Differentiated Scientific Platforms

Slide 22: PTC303 MSH3 Small Molecule Splicing Program

Slide 23: PTC612 NLRP3 Inhibitor Program

Slide 24: PTC612: Greater Potency & Selectivity Compared to Other NLRP3 Inhibitors

Slide 25: PTC844 DHODH Inhibitor Program

Slide 26: PTC844 Demonstrates Superior Potency and Selectivity Compared to Other DHODH Inhibitors

Slide 27: Well Positioned for Success in 2026 & Beyond

Slide 28: PTC Therapeutics