Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — Corporate Update (October 2026)
31 slides from Ptc Therapeutics's corporate update, Oct 29, 2026
Slide 1: PTC Therapeutics October 2026
© 2026 PTC Therapeutics. All rights reserved. PTC Therapeutics October 2026
Slide 2: Forward-looking statements
© 2026 PTC Therapeutics. All rights reserved. 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, inclu
Slide 3: PTC: A Global Leader in Rare Disease Drug Development and Commercialization
© 2026 PTC Therapeutics. All rights reserved. PTC: A Global Leader in Rare Disease Drug Development and Commercialization 1As of September 30, 2026 3 Robust global commercial infrastructure Proven track record of achieving regulatory authorization, gaining market access and commercializing rare dise
Slide 4: 2026 Strategic Priorities
© 2026 PTC Therapeutics. All rights reserved. 2026 Strategic Priorities 4 Continue Sephience global launch momentum Move toward reaching cash flow breakeven Advance Phase 3 votoplam HD program Advance early-stage R&D programs
Slide 5: 2026 Revenue and OpEx Guidance
© 2026 PTC Therapeutics. All rights reserved. 2026 Revenue and OpEx Guidance 5 Product Revenue1 OpEx $680 - 720M 3 $1.29 - 1.32B $960 - 990M 64 - 69% YoY Growth Total Revenue2 Our Products4 1 Product revenue excludes Evrysdi royalty and collaboration revenues; YoY based on $960-990M guidance. 2 Tota
Slide 6: Sephience PKU Program
Katie, living with PKU © 2026 PTC Therapeutics. All rights reserved. Sephience PKU Program 6
Slide 7: Sephience Dual Mechanism of Action Supports Potential for Broad Uptake
© 2026 PTC Therapeutics. All rights reserved. Sephience Dual Mechanism of Action Supports Potential for Broad Uptake 7 1. Precursor to BH4 SP Sephience 7.8-Dihydrobiopterin (BH2 ) Tetrahydrobiopterin (BH4 ) SR* DHFR** BH BH4 4 BH4 Sephience significantly increases intracellular BH4 levels 2. Indepen
Slide 8: Sephience Provides Significantly Greater Phe Reduction Compared With BH4
© 2026 PTC Therapeutics. All rights reserved. Sephience Provides Significantly Greater Phe Reduction Compared With BH4 8 -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
Slide 9: Sephience Provides Diet Liberalization, Cognitive and Quality of Life Benefits
© 2026 PTC Therapeutics. All rights reserved. Sephience Provides Diet Liberalization, Cognitive and Quality of Life Benefits 9 Improved cognitive function and mood Improved concentration & slow thinking, and decreased irritability Meaningful diet liberalization 69% of subjects reached age-adjusted p
Slide 10: Sephience Differentiated Profile Enables Penetration Into All Key Market Segments
© 2026 PTC Therapeutics. All rights reserved. Sephience Differentiated Profile Enables Penetration Into All Key Market Segments 10 On Treatment Treatment Failures Treatment Naive 58K Patients WW T O TA L A D D R E S S A B L E M A R K E T I N C L U D I N G A L L A G E S A N D S E V E R I T I E S
Slide 11: Sephience: Strong Launch Momentum Continues
© 2026 PTC Therapeutics. All rights reserved. Sephience: Strong Launch Momentum Continues * As of September 30, 2026 11 $458M Global revenue YTD in first full year of launch Broad penetration into all key market segments
Slide 12: Sephience Global Launch Footprint Expansion Planned for 2026 and 2027
© 2026 PTC Therapeutics. All rights reserved. Sephience Global Launch Footprint Expansion Planned for 2026 and 2027 12 N O R T H A M E R I C A E U R O P E A S I A L AT A M Anticipated Sephience geographic footprint for 2026
Slide 13: ST-920 Fabry Disease Program
© 2026 PTC Therapeutics. All rights reserved. ST-920 Fabry Disease Program 13
Slide 14: Fabry Disease is a Rare, Life-Shortening Disorder with Multiorgan Impact
© 2026 PTC Therapeutics. All rights reserved. • Lysosomal storage disorder causing serious organ damage, leading to premature death • Affects ~11,000 individuals (US) • Standard of care of enzyme replacement therapies (ERT) have several drawbacks − Frequent (up to biweekly) administration − Require
Slide 15: ST-920 Provides One-Time Administered Durable Enzyme Production
© 2026 PTC Therapeutics. All rights reserved. • AAV gene therapy that provides humanized gene enabling long-term production of the deficient α-Gal A enzyme • Favorable safety profile with no need for immunosuppression • Durable α-Gal A activity with longest follow up of over 5 years • Favorable effe
Slide 16: Votoplam Huntington's Disease Program
April, living with HD © 2026 PTC Therapeutics. All rights reserved. Votoplam Huntington’s Disease Program 16
Slide 17: Phase 2 PIVOT-HD Trial Achieved Key Efficacy and Safety Objectives
© 2026 PTC Therapeutics. All rights reserved. Phase 2 PIVOT-HD Trial Achieved Key Efficacy and Safety Objectives 17 17 Evidence of dose-dependent disease slowing on cUHDRS in Stage 2 participants at 24 months PIVOT-HD study met primary endpoint of blood HTT protein lowering at Week 12 with durable d
Slide 18: Dose-dependent Slowing of Disease Progression on cUHDRS at 24 months
© 2026 PTC Therapeutics. All rights reserved. Dose-dependent Slowing of Disease Progression on cUHDRS at 24 months in Stage 2 ParticipantsWorsening Results based on observed data SE = Standard Error Baseline 12 Month 24 Month Mean (+/-SE) Change from Baseline of Composite UHDRS 0 -0.5 -1.0 -1.5 NH C
Slide 19: INVEST-HD Global Phase 3 Trial Enrolling Worldwide
© 2026 PTC Therapeutics. All rights reserved. INVEST-HD Global Phase 3 Trial Enrolling Worldwide 19 19 • Individuals with early symptomatic disease • 3:2 randomization of votoplam 10mg: placebo • Target enrollment: ~770 participants in >30 countries • Primary endpoint: Change from baseline in cUHDRS
Slide 20: Vatiquinone Friedreich's Ataxia Program
Olivia, living with FA © 2026 PTC Therapeutics. All rights reserved. Vatiquinone Friedreich’s Ataxia Program 20
Slide 21: Vatiquinone Demonstrated Meaningful Slowing of Short- and Long-term Disease Progression
© 2026 PTC Therapeutics. All rights reserved. Vatiquinone Demonstrated Meaningful Slowing of Short- and Long-term Disease Progression 21 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)
Slide 22: PROVE-FA Open-Label Study to Support NDA Resubmission
© 2026 PTC Therapeutics. All rights reserved. PROVE-FA Phase 3 Open-Label Study to Support NDA Resubmission Initiated in Q3 2026 22 • Open-label study using matched natural history cohort from FACOMS disease registry • Target enrollment: 120 individuals with FA ages 7 to 21 • Primary endpoint: Chang
Slide 23: Innovative Research Platforms
© 2026 PTC Therapeutics. All rights reserved. Innovative Research Platforms 23
Slide 24: R&D Pipeline Includes Innovative Programs from Differentiated Scientific Platforms
© 2026 PTC Therapeutics. All rights reserved. R&D Pipeline Includes Innovative Programs from Differentiated Scientific Platforms 24 MSH3: Huntington’s Disease, Myotonic Dystrophy I Undisclosed: Alzheimer’s Disease Undisclosed: Brain Tumors & Metastases Undisclosed: Sickle Cell Disease, -Thalassemia
Slide 25: PTC303 MSH3 Small Molecule Splicing Program
© 2026 PTC Therapeutics. All rights reserved. • 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 2027 PTC303 MSH3 Sma
Slide 26: PTC612 NLRP3 Inhibitor Program
© 2026 PTC Therapeutics. All rights reserved. • 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 NLR
Slide 27: PTC612: Greater Potency & Selectivity Compared to Other NLRP3 Inhibitors
© 2026 PTC Therapeutics. All rights reserved. PTC612: Greater Potency & Selectivity Compared to Other NLRP3 Inhibitors 27 Assay PTC612 Selnoflast1 DFV8902 Usnoflast3 AZD41444 THP-1 IL1β IC50 (nM) 0.99 220 13 13 23-82 Assay PTC612 Vent-015 DFV8902 VTX-32326 VTX-27356 hWB IL1β IC50 (nM) 9.7 124 180 13
Slide 28: PTC844 DHODH Inhibitor Program
© 2026 PTC Therapeutics. All rights reserved. • 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
Slide 29: PTC844 Demonstrates Superior Potency and Selectivity Compared to Other DHODH Inhibitors
© 2026 PTC Therapeutics. All rights reserved. PTC844 Demonstrates Superior Potency and Selectivity Compared to Other DHODH Inhibitors 29 Phase 2a PK/PD study of PTC844 initiating Q3 2026 [nM] IC50=1nM IC50~5000nM [nM] IC50>10,000nM [nM] PTC844 Vidofludimus Teriflunomide
Slide 30: Well Positioned for Success in 2026 & Beyond
© 2026 PTC Therapeutics. All rights reserved. Well Positioned for Success in 2026 & Beyond 30 Robust global commercial infrastructure Strong financial position Lead product Sephience to support near-and mid-term growth Innovative R&D platforms and pipeline
Slide 31: PTC Therapeutics October 2026
© 2026 PTC Therapeutics. All rights reserved. PTC Therapeutics October 2026