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Corporate Presentation –November 2025 1 PTC Therapeutics November 2025 Pam and Kelsey living with PKU
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Corporate Presentation – November 2025 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 statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of regulatory and sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia and other regions; the effect of the European Commission’s adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna on other regulatory bodies; PTC's ability to use the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from its trials in Translarna; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington’s disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Kebilidi, Upstaza, Evrysdi, Tegsedi, Waylivraor vatiqunone.The forward-looking statements contained herein represent PTC's views only as of the date of this presentation and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this presentation except as required by law. Forward Looking Statements 2
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Corporate Presentation – November 2025 PTC Mission is Patient Focused 3 Commercialize Discover Develop Transformative therapies for patients with rare diseases
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Corporate Presentation – November 2025 PTC is Positioned for Sustainable Value Creation 4 Strong balance sheet and continued expense management enable near-term path to profitability Path to $2B topline revenuewith commercial launches and innovative R&D platforms Significant commercial opportunity for Sephience as the potential new standard of care provides foundation for growth
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Corporate Presentation – November 2025 Strong Cash Position Enables Future Revenue Growth and R&D Innovation 5 Reach CashflowBreakeven Without Additional CapitalSupport CommercialLaunches and Innovative R&D ProgramsFund BD Activitiesto ComplementProduct Portfolio
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Corporate Presentation – November 2025 6 SephiencePotentialto be the new standard of care for PKUOwen living with PKU
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Corporate Presentation – November 2025 Strong Start to Sephience Global Launch Launch metrics as of September 30, 2025 7 $19.6Mglobal revenue $14.4M US $5.2M ex-US 341 patients on commercial therapy worldwide 521 Patient Start Forms received from 141 unique prescribers in the US Broad initial uptake across all patient segments Additional Sephience marketing authorization reviews ongoing; Japan decision expected in Q4 2025
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Corporate Presentation – November 2025 Sephience Treatment Resulted in Clinically Significant Blood Phe Reduction 8 Overall Primary Analysis Population Mean % Blood PheReduction -70-60-50-40-30-20-10010SepiapterinN=49 63% 1%N=49 (p<0.0001) PlaceboSephience Classical PKU Patients Mean % Blood PheReduction586 464 -70-60-50-40-30-20-10010SepiapterinPlaceboN=6 69% 3%N=9 (p<0.001) Sephience
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Corporate Presentation – November 2025 Sephience Treatment Enabled Meaningful Diet Liberalization Baseline is the mean of the Month 1 values for the daily dietary Phe intake. Dashed lines indicate the RDA for dietary Phe intake (dark blue) and low-Phe protein substitute intake (light blue). Data cutoff: February 4, 20259 Mean Dietary Phe Intake Over Time Mean (SD) DailydietaryPheconsumption(mg/kg/day) BaselineW1–W2W3–W4W5–W6W7–W8W9–W10W11–W12W13–W14W15–W16W17–W18W19–W20W21–W22W23–W24W25–W26 117 106106106991029995969494929293 1251007550250 DietaryPheintake 80604020 0 Low-Pheproteinsubstituteintake(g) DietaryPhe intake,n 100150
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Corporate Presentation – November 2025 Sephience Treatment Resulted in Superior PheLowering Compared to Sapropterin (BH4) 10 -437.0-256.6 -600-500-400-300-200-1000100Mean Absolute Change in Blood Phe from Baseline Absolute least squares mean changein blood Phe concentration (µmol/L) Sephience (n = 58) Sapropterin (n = 56) 70.3% Greater reduction with sepiapterin p < 0.0001 AMPLIPHY was a crossover study comparing Phe lowering of Sephience vs sapropterin in 82 subjects
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Corporate Presentation – November 2025 Essential Pillars for Commercial Success in Place 11 Well aggregated patient community 104 Centers of Excellence treating vast majority of patientsNewborn screening in place Experienced PTC customer-facing teams
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Corporate Presentation – November 2025 12 Votoplam Huntington’s Disease Program April living with HD
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Corporate Presentation – November 2025 Key Attributes of Votoplam Drive Differentiation Orally bioavailable Favorable safety profile, with no treatment-related NfL spikes 13 Dose-dependent and durable lowering of blood HTT protein Dose-dependent clinical benefit across several key measures
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Corporate Presentation – November 2025 Phase 2 Placebo-Controlled Trial Achieved Key Objectives 14 Continued favorable safety and tolerability profile with no treatment-relatedNfL spikes Favorable and dose-dependent trends on clinical scales at Month 12 in Stage 2 subjects Long-term positive clinical trendsat Month 24 relative to natural history and dose-dependent NfL lowering Study met primary endpoint of blood HTT protein lowering at Week 12 with durable dose-dependent lowering at Month 12 FDA meeting for votoplam HD program planned for Q4 2025
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Corporate Presentation – November 2025 Development and Commercialization Collaboration Transaction closed in January 2025. Refer to press release dated December 2, 2024 for additional details. Novartis to fund development activities following completion of PIVOT-HD $1 billion upfront payment 40% U.S. profit share, double-digit tiered royalties on ex-U.S. sales Up to $1.9 billion in development, regulatory and sales milestones 15
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Corporate Presentation – November 2025 16 Vatiquinone Friedreich’s Ataxia ProgramOlivia living with FA
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Corporate Presentation – November 2025 Vatiquinone Demonstrated Meaningful Slowing of Short- and Long-term Disease Progression 17 VatiquinonePlacebo Mean Change from Baseline in mFARS Upright Stability score (+/- SE) 42% slowingp=0.021 4.8-point benefit (p<0.0001) on mFARS** over 2 years in ambulatory and non-ambulatory adults 50% slowing (p<0.0001) of disease progression over 3 years** in MOVE-FA long-term extension study Long-term Extension Studies72-week Placebo-Controlled Trial* *MOVE-FA did not meet its primary endpoint of statistically significant change in total mFARS at 72 weeks **Relative to matched FACOMS (Friedreich’s Ataxia Clinical Outcome Measures Study) natural history cohort
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Corporate Presentation – November 2025 18 Innovative Research Platforms
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Corporate Presentation – November 2025 Validated Splicing Platform Provides Sourceof Innovative and Valuable Therapies PTC has pioneered discovery and development of oral splicing therapies Additional early-stage programs targeting non-CNS indicationsPlatform innovations such as PTSeek™ accelerate discovery of novel splicing therapies Multiple active CNS programs advancing towards clinic 19
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Corporate Presentation – November 2025 Inflammation & Ferroptosis Programs Targeting CNS and Non-CNS Disorders Focused on novel targets key to inflammation and oxidative stress Active programs targeting CNS and non-CNS disorders Phase 2 ready DHODH inhibitor program for neuroinflammation indications NLRP3 inhibitor program entering IND-enabling studies Preclinical program targeting alpha synuclein for Parkinson’s disease Preclinical program targeting nrf2 activation for both CNS and peripheral indications 20
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Corporate Presentation – November 2025 PTC Vision 21 Path to $2 Billion Topline Revenue Highly Differentiated and Innovative R&D Platforms Business Development to Accelerate Revenue Growth Cash Flow Break Even
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Corporate Presentation – November 2025