EX-99.2 3 tmb-20220107xex99d2.htm EX-99.2

Exhibit 99.2

| PTC 2022J.P. Morgan Healthcare Conference Stuart W. Peltz, Ph.D., CEO

| Forward Looking StatementsThis 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 with respect to preliminary unaudited 2021 financial information with respect to 2021 total net revenue and2021 DMD franchise net product revenue, statements with respect to guidance relating to 2022 total net product revenue, 2022 DMD franchise net product revenue, 2022 operating expenditure guidance and future revenue guidance and 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 and other matters; expectations with respect to PTC's gene therapy platform, including any potential regulatory submissions and manufacturing capabilities; advancement of PTC's joint collaboration program in SMA, including any potential regulatory submissions, commercialization or royalty or milestone payments; PTC's expectations with respect to the licensing, regulatory submissions and commercialization of 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: expectations with respect to the COVID-19 pandemic and related response measures and their effects on PTC's business, operations, clinical trials, regulatory submissions and approvals, and PTC's collaborators, contract research organizations, suppliers and manufacturers; 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 PTC's gene therapy platform, including any regulatory submissions and potential approvals, manufacturing capabilities and the potential financial impact and benefits of its leased biologics manufacturing facility andthe potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; the enrollment, conduct, and results of ongoing studies under the SMA collaboration and events during, or as a result of, the studies that could delay or prevent further development under the program, including any regulatory submissions and commercialization with respect to Evrysdi; PTC's ability to utilize results from Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarnafor the treatment of nmDMDfollowed by an 18-month open-label extension, to support a marketing approval for Translarnafor the treatment of nmDMDinthe United States; PTC's ability to maintain its marketing authorization of Translarnafor the treatment of nmDMDin the European Economic Area (EEA) and other regions, including whether theEuropean Medicines Agency(EMA) determines in future annual renewal cycles that the benefit-risk balance of Translarnaauthorization supports renewal of such authorization; PTC's ability to fund, complete and timely submit to the EMA the results of Study 041, which is a specific obligation to continued marketing authorization in the EEA; expectations with respect to the commercialization of Tegsediand Waylivra; the enrollment, conduct and results of PTC's clinical trial for emvododstatfor COVID-19; 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 the lease agreement for its leased biologics manufacturing facility; 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 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 theSEC. 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, includingTranslarna,Emflaza,Evrysdi,Tegsedi,Waylivraor PTC-AADC.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.JPM Presentation 2022 –Private & Confidential2

| CommercializeProviding patients with access to transformative treatments DevelopEnduring innovation enginePTC Continually Innovates to Bring New Therapies to Patients3 DiscoverProven groundbreaking science

| 4Building a Pipeline to Produce a Therapy Every Three Years Discover Develop 2to 3yearsNew product every Commercialize

| PTC has a Growing Global Footprint5 ~1,200 employees 20 offices worldwide Footprint in >50 countries Founded in 1998

| Diversified Platform Drives Strong Portfolio6 Clinical Commercial Research Nonsense MutationLatAm CommercialDeflazacortMetabolicOncology US Ataluren Emvododstat AML UnesbulinDIPG UnesbulinLMS PTC923 PKU EmvododstatCOVID-19 Virology SCIENTIFIC PLATFORMS and RESEARCH SCA-3 MAP-Tau PTC518 HDSplicing Bio-e VatiquinoneMDAS VatiquinoneFA PTC857 ALS 3 Undisclosed Gene Therapy Angelman IRDs Cog Disorders FA PTC-AADC 8 Undisclosed AADC, aromatic L-amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID-19, coronavirus disease 2019; DIPG, diffuse intrinsic pontine glioma; FA, Friedreich’s ataxia; ALS, amyotrophic lateral sclerosis; HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizures; PKU, phenylketonuria; SCA-3, spinocerebellar ataxia type 3. Potential registrational studies Early-stage programs 3 Undisclosed 2 Undisclosed

| Multiple Platforms Provide Opportunity to Target Over 700,000 Patients by 20307ALS+ ~150KDMD/SMA/AADC+ ~50KMito Sz+ ~20KPKU+ ~58KFA+ ~25KHD+ ~135K 20212030 =10,000 pts GBA-PD+ ~190K AS+ ~75K ~50K~70K~128K~153K~288K~438K~628K~703KEstimated Global Prevalence

| Enduring Innovation Drives Value Creation 2026Key Drivers: Evrysdi®, PTC-AADC, Tegsedi®, Waylivra®, Translarna™, Vatiquinone, PTC923, Unesbulin $536MVatiquinone|PTC923 |PTC518 |PTC857 |Unesbulin|Emvododstat|GT-FA |GT-AS |Research 2030Key Drivers: Evrysdi®, PTC-AADC, Tegsedi®, Waylivra®, Vatiquinone, PTC923, Unesbulin, PTC518, PTC857 ~$3B ~$8BCommercial 8Clinical Unaudited total revenuePotential future revenues 2021

| Revenue Contribution of our Pipeline Grows9Potential future revenues Commercial Products + RoyaltiesCurrent Pipeline 57%43%20262025 64%36%20242023 88%12% 100%

| Completed Phase 1 healthy volunteer trial of PTC518; initiation of Phase 2 Completed Phase 1 HV trial of PTC857, Phase 2 in ALS to initiate 1Q22Enrollment update MIT-E, MOVE-FA?Initiation of registration- directed APHENITY Phase 3 trial for PTC923 in PKUCompletion of unesbulinPhase 1b oncology trialCompletion (?) of FITE-19 trial with results expected 1Q22 Evrysdinow approved in >65 countries including the EU and JapanWaylivraapproved in Brazil for treatment of FCSTranslarnalabel expansion in Brazil to include patients 2 years of age and up2 Rare Pediatric Disorder Designations6 Orphan Drug Designations DMD franchise continues to grow with new patients in existing geographies and geographic expansion for Translarnaand new patients and increased compliance for EmflazaEvrysdiis most prescribed SMA product and reached ~20% market share in the USTegsediCategory 1 pricingWaylivraCategory 1 pricing Completed Phase 1 healthy volunteer trial of PTC518Completed Phase 1 healthy volunteer trial of PTC857Completed enrollment of vatiquinoneMOVE-FA registration-directed trialInitiation of registration-directed APHENITY Phase 3 trial for PTC923 in PKUCompletion of unesbulinPhase 1b LMS trialCompletion of unesbulinPhase 1b DIPG trialSignificant Execution and Value Creation in 202110 Commercial Achievements Regulatory Achievements Clinical Achievements

| Strong Financial Performance Supports Innovation11Unaudited TotalRevenue$536MUnaudited DMD Franchise Net Product Revenue$424M*Non-GAAP measure which excludesestimated non-cash, stock-based compensation expense of approximately $115 million.GAAP R&D andSG&A expense for the full year 2022 is anticipated to be between $915 and $965 million. TotalRevenue Guidance$475-495MOPEX Guidance* $800-850M $700-750MDMD FranchiseNet Product Revenue Guidance

| Clinical Nonsense MutationLatAm CommercialDeflazacortMetabolicOncology US Ataluren Emvododstat AML UnesbulinDIPG UnesbulinLMS PTC923 PKU EmvododstatCOVID-19 Virology SCIENTIFIC PLATFORMS and RESEARCH SCA-3 MAP-Tau PTC518 HDSplicing Bio-e VatiquinoneMDAS VatiquinoneFA PTC857 ALS Gene Therapy Angelman IRDs Cog Disorders FA PTC-AADC 3 Undisclosed 8 Undisclosed 3 Undisclosed 2 Undisclosed Diversified Platform Drives Strong Portfolio12 Clinical Commercial Research Nonsense MutationLatAm CommercialDeflazacortMetabolicOncology US Ataluren Emvododstat AML UnesbulinDIPG UnesbulinLMS PTC923 PKU EmvododstatCOVID-19 Virology SCIENTIFIC PLATFORMS and RESEARCH SCA-3 MAP-Tau PTC518 HDSplicing Bio-e VatiquinoneMDAS VatiquinoneFA PTC857 ALS Undisclosed Gene Therapy Angelman IRDs Cog Disorders FA PTC-AADC 3 Undisclosed 8 Undisclosed 3 Undisclosed 2 Undisclosed AADC, aromatic L-amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID-19, coronavirus disease 2019; DIPG, diffuse intrinsic pontine glioma; FA, Friedreich’s ataxia; ALS, amyotrophic lateral sclerosis; HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizures; PKU, phenylketonuria; SCA-3, spinocerebellar ataxia type 3. Potential registrational studies Early-stage programs

| 13 Data Expected 4Q 2022 Vatiquinone Mitochondrial Disease Associated Seizures Data Expected 2Q 2023 Vatiquinone Friedreich Ataxia Data Expected YE 2022 PTC923 PKU Data Expected 1H 2022 Emvododstat COVID-19 Data ExpectedMid Year2022Study 041 Translarna (Ataluren) nmDMDFive Registration-Directed Clinical Trials Drives Near-Term Value

| Substantial Pipeline Progress Planned in 202214Study ResultsStudy Initiations GT-FAPh1Initiation DIPG Ph2 Initiation Study 041 2022 Q42022 Q22022 Q32022 Q1

| SCA-3 MAP-Tau PTC518 HDSplicing Clinical Commercial Research Nonsense MutationLatAm CommercialDeflazacortMetabolicOncology US Ataluren Emvododstat AML UnesbulinDIPG UnesbulinLMS PTC923 PKU EmvododstatCOVID-19 Virology AADC, aromatic L-amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID-19, coronavirus disease 2019; DIPG, diffuse intrinsic pontine glioma; FA, Friedreich’s ataxia; GBA, glucocerebrosidase; HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizures; PD, Parkinson’s disease; PKU, phenylketonuria; SCA-3, spinocerebellar ataxia type 3.SCIENTIFIC PLATFORMS and RESEARCH Potential registrational studies Early-stage programs Gene Therapy Angelman IRDs Cog Disorders FA PTC-AADC 3 Undisclosed 2 Undisclosed 8 UndisclosedDiversified Platform Drives Strong Portfolio15 Reducing Oxidative StressInhibition of 15 lipoxygenase (15-LO) blocks inflammation and oxidative stress response Bio-e VatiquinoneMDAS VatiquinoneFA PTC857 ALS Science of Bio-e•Differentiated therapy for neurological indications•High levels of oxidative stress and inflammation underpin multiple CNS disease pathologies•Bio-e platform targets the key enzymatic hubs that regulate oxidative stress and inflammatory pathways to modulate disease progression 3 Undisclosed

| 16 MIT-E:Registration-directed trial of vatiquinoneforMitochondrial Disease Associated Seizures

| Disease Mitochondrial disease associated seizures (MDAS)is the highly morbid condition of refractory seizures in patients with inherited mitochondrial disease Current Treatments No approved disease modifying treatments Mechanism of Action Vatiquinone targets 15-lipoxygenase, a regulator of the key energetic and oxidative stress pathways that underpin seizures in these patients17 ~20,000Global Prevalence Vatiquinonehas Potential to Show Clinically Differentiated Improvement for MDAS Patients

| Vatiquinonehas Potential to Show Clinically Differentiated Improvement for MDAS Patients18 Primary EndpointChangefrom baseline in frequency of observable motor seizuresPTC743 (vatiquinone) Placebo N = 30 24 weeks RN = 30 48 weeks PTC743 (vatiquinone)N = 60Open-Label ExtensionPlacebo-Controlled Primary Endpoint Trial Status•Enrolling•Data expected 4Q 2022

| 19MOVE-FA: Registration-directed trial of vatiquinone for Friedreich Ataxia

| Disease Friedreich ataxia (FA)is a rare,inherited, progressive disease resulting from mitochondrial dysfunction Current Treatments No approved disease modifying therapies Opportunity Vatiquinone targets 15-lipoxygenase, a regulator of key energetic and oxidative stress pathways that are disrupted in FA 20 ~25,000Global Prevalence Vatiquinonehas the Potential to Provide Improvement in Neurological Function

| Vatiquinonehas the Potential to Provide Improvement in Neurological Function 21 Primary EndpointChange in mFARSKey Secondary EndpointChange in FA-ADLOpen-Label ExtensionPlacebo-ControlledPTC743 (vatiquinone) Placebo N = ~60 72 weeks RN = ~60 24 weeks PTC743 (vatiquinone)N = ~120 Primary Endpoint Trial Status•Data expected in 2Q 2023 Enrollment complete

| 22CardinALS:Phase 2 trial of PTC857 for Amyotrophic Lateral Sclerosis

| 23 Disease Amyotrophic lateral sclerosis (ALS) is a rapidly progressing neurodegenerative disease caused by oxidative damage which leads to neuronal cell death and muscular atrophy Current Treatments No approved disease modifying therapies Mechanism of Action PTC857 inhibits pathways leading to oxidative damage and ferroptosis, resulting in protection of motor neurons ~150,000Global Prevalence PTC857 has Potential to Slow Disease Progression in ALS

| Primary EndpointsChange in ALSFRS-RSecondary EndpointsSafety and PK24Treatment PeriodPTC857 250 mg BID Placebo 24weeks Primary Endpoint RLong-Term Treatment Extension 28 weeks PTC857 250 mg BID Trial Status•Initiation in 2Q22Screening 8weeksPTC857 has Potential to Slow Disease Progression in ALSN = ~85N = ~170

| Clinical Commercial Research Nonsense MutationLatAm CommercialDeflazacortMetabolicOncology US Ataluren Emvododstat AML UnesbulinDIPG UnesbulinLMS PTC923 PKU EmvododstatCOVID-19 Virology AADC, aromatic L-amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID-19, coronavirus disease 2019; DIPG, diffuse intrinsic pontine glioma; FA, Friedreich’s ataxia; GBA, glucocerebrosidase; HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizures; PD, Parkinson’s disease; PKU, phenylketonuria; SCA-3, spinocerebellar ataxia type 3.SCIENTIFIC PLATFORMS and RESEARCH Potential registrational studies Early-stage programs Bio-e VatiquinoneMDAS VatiquinoneFA PTC857 ALS Gene Therapy Angelman IRDs Cog Disorders FA PTC-AADCDiversified Platform Drives Strong Portfolio 3 Undisclosed25 3 Undisclosed 2 Undisclosed 0 SCA-3 MAP-Tau PTC518 HDSplicing Splicing•Pioneers in splicing•Small molecule regulation of splicing events•Chemistry optimized for uniform distribution, blood brain barrier penetration and limited efflux5’ Splice Site 3’ Splice Site Isoform plex HTSpliceseqDatabases of Splicing Targets Isoform plex HTSpliceseqDatabases of Splicing Targets Leaders in splicing technology 8 Undisclosed

| 26 PIVOT HD:PTC518 forHuntington's Disease

| 27 Disease Huntington’s disease (HD) is a progressive brain disorder that causes uncontrolled movements and cognitive loss Current Treatments No approved disease modifying therapies Mechanism of Action PTC518 modulates splicing to induce degradation of HTT mRNA, reducing expression of the toxic HTT protein ~135,000Global Prevalence PTC518 Reduces HTT mRNA and Protein to Target the Proximal Cause of HD

| Crosses blood brain barrier and is not effluxed Dose-dependent reduction of HTT mRNA and protein Consistent pharmacology Generally well tolerated 28Results from Phase 1 Healthy Volunteer StudyPTC518 Reduces HTT mRNA and Protein to Target the Proximal Cause of HD

| 29PTC518 has the Potential to Reduce HTT Protein in Huntington Patients Trial Status•Trial to initiate in first quarter 3 months 9 months 5 mg PTC518 10 mg PTC518 Alternative Dose PTC518 R Primary EndpointN = ~30N = ~30N = ~30Part APart B •Safety and tolerability of PTC518 in Huntington disease patients•Percent reduction in HTT mRNA and protein in bloodPrimary endpointsPlacebo 5 mg PTC518 10 mg PTC518 Alternative Dose PTC518

| 30 •Safety and tolerability of PTC518 in Huntington’s disease patients•Percent reduction in HTT mRNA and protein in bloodPrimary endpoints •Percent reduction in HTT protein in CSF•Changes in neurofilament light chain (NfL) in plasma and CSF •Change in caudate, putamenal, ventricular volume on volumetric MRI imaging•Changes in clinical scales of motor and cognitive functionSecondary endpoints •Ambulatory Huntington’s patients ages 25 and older•CAG repeats 42-50 inclusive•Motor and Cognitive Function:•UHDRS-IS score of 100•UHDRS TFC score of 13•PINHDscore 0.18 -4.93•Multivariate calculation including SDMT, TMS, age, CAGInclusion CriteriaPIVOT HD Trial Will Target the “Goldilocks” Population

| 31 APHENITY:Registration-directed trial of PTC923 forPKU

| 32 Disease Phenylketonuria (PKU) is a metabolic condition caused by mutations to phenylalanine hydroxylase that can lead to cognitive disabilities and seizures Current Treatments Maority of patients do not initially respond or are not well controlled by standard of care Mechanism of Action PTC923 is a more bioavailable precursor than exogenously administered synthetic BH4 and has the potential to treat a broader range of PKU patients ~58,000Global Prevalence APHENITY is a Global Registration-Directed Trial of PTC923 for PKU

| APHENITY is a Global Registration-Directed Trial of PTC923 for PKU 33 Primary EndpointReduction in blood phenylalanine levels Primary Endpoint Trial Status•Initiated in 3Q21•Data expected YE 2022 R PTC923 Placebo 42 DaysOpen-Label ExtensionPlacebo-ControlledPTC923 12 Months PTC923 14DaysRun-in

| Clinical Commercial Research Nonsense MutationLatAm CommercialDeflazacortMetabolic US Ataluren PTC923 PKU EmvododstatCOVID-19 Virology AADC, aromatic L-amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID-19, coronavirus disease 2019; DIPG, diffuse intrinsic pontine glioma; FA, Friedreich’s ataxia; GBA, glucocerebrosidase; HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizures; PD, Parkinson’s disease; PKU, phenylketonuria; SCA-3, spinocerebellar ataxia type 3.SCIENTIFIC PLATFORMS and RESEARCH Potential registrational studies Early-stage programs SCA-3 MAP-Tau PTC518 HDSplicing Bio-e VatiquinoneMDAS VatiquinoneFA PTC857 ALS Diversified Platform Drives Strong Portfolio34 3 Undisclosed 8 Undisclosed Gene Therapy Angelman IRDs Cog Disorders FA PTC-AADC 2 Undisclosed Oncology Emvododstat AML UnesbulinDIPG UnesbulinLMS •Niche oncology indications•Small molecule inhibitors of cell proliferation:•Unesbulindisrupts tubulin function•Emvododstatinhibits pyrimidine biosynthesis•Targeting rare and difficult to treat cancers with poor prognosis Oncology 3 Undisclosed

| 35 Disease Leiomyosarcoma (LMS) is a rare and aggressive cancer with tumors found in smooth muscle Current Treatments Several chemotherapeutics are utilized but offer minimal meaningful clinical benefit Mechanism of Action Unesbulin is an oral small molecule tubulin inhibitor that arrests tumor cells in G2/M phase, including cancer stem cells by inhibiting tubulin polymerization ~4,000Diagnosed annually in US Unesbulinhas Opportunity to Provide Additional Progression Free Survival in LMSNot an actual LMS patient.

| 36Phase 1b study results300 mg was selected as RP2DUnesbulinwas well tolerated 21Day Treatment CyclesPhase 1b Study DesignAscending doses 200, 300 and 400 mg Unesbulin+ 1000mg/m2dacarbazineN = 29 •Patients with locally advanced or metastatic LMS intolerant or refractory to standard therapyInclusion Criteria•Any number of previous lines of treatments allowed Unesbulin has Opportunity to Provide Additional Progression Free Survival in LMS

| Unesbulinhas Opportunity to Provide Additional Progression Free Survival in LMS R 300 mg Unesbulin+ 1000mg/m2dacarbazinePlacebo + 1000 mg/m2 dacarbazine Primary EndpointPFS as determined by RECISTSecondary EndpointsOS, ORR, DCR, DOR 21 Day Treatment Cycle 24MonthsInterim AnalysisN ≈ 115N ≈ 23037Trial Status•Initiating in 2Q 2022

| Commercial Nonsense MutationLatAm CommercialDeflazacortMetabolicOncology US Ataluren Emvododstat AML UnesbulinDIPG UnesbulinLMS PTC923 PKU EmvododstatCOVID-19 Virology SCIENTIFIC PLATFORMS and RESEARCH SCA-3 MAP-Tau PTC518 HDSplicing Bio-e VatiquinoneMDAS VatiquinoneFA PTC857 ALS Gene Therapy Angelman IRDs Cog Disorders FA PTC-AADC 3 Undisclosed 8 Undisclosed 3 Undisclosed 2 Undisclosed 38Diversified Platform Drives Strong Portfolio Clinical Commercial Research Nonsense MutationLatAm CommercialDeflazacortMetabolicOncology US Ataluren Emvododstat AML UnesbulinDIPG UnesbulinLMS PTC923 PKU EmvododstatCOVID-19 Virology SCIENTIFIC PLATFORMS and RESEARCH SCA-3 MAP-Tau PTC518 HDSplicing Bio-e VatiquinoneMDAS VatiquinoneFA PTC857 ALS Gene Therapy Angelman IRDs Cog Disorders FA PTC-AADC 3 Undisclosed 8 Undisclosed 3 Undisclosed 2 Undisclosed AADC, aromatic L-amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID-19, coronavirus disease 2019; DIPG, diffuse intrinsic pontine glioma; FA, Friedreich’s ataxia; ALS, amyotrophic lateral sclerosis; HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizures; PKU, phenylketonuria; SCA-3, spinocerebellar ataxia type 3. Potential registrational studies Early-stage programs

| •First and only corticosteroid approved for DMD; approved for all US DMD patients >2yrs•Data show clinical benefit over prednisone•New patients, high adherence and fewer discontinuations •Evrysdinow approved in >65 countries•Continued strong uptake in the U.S. ~20%market share just over one year after launch•Potential for $325M in sales-based milestones •Innovative treatment for hATTRamyloidosis patients•Disease awareness and patient ID continuing•LATAM patients benefiting through early-access programs•Received Category 1 pricing, in final pricing negotiations •For treatment of familial chylomicronaemia syndrome (FCS)•LATAM patients benefiting through early-access programs•Received Category 1 pricing, in final pricing negotiations •Treatment for nonsense mutation DMD for ages 2 and older•Distributed in 50+ countries•Expanding patient base, high compliance, better access, strong geographic expansion Success Across Our Commercial Portfolio 39

| 2021 unaudited net product revenue40Continued Strong DMD Franchise GrowthUSD millions 34 81 29 92 101 139 188 145 171 190 192 236 2018174201542420192016201720202021263291331 +52% TranslarnaEmflaza

| PTC-AADC Gene Therapy Opportunity and Launch Preparation 41 Potential over $1B in cumulative revenueSuccessful patient finding is ongoing CHMP opinion expected in April PTC-AADC BLA submission expected in 2Q22 Development of virtual education: disease-specific webinars and congress symposiaEngaging with patient advocacy groups and payers Identification and preparation of expert pediatric neurosurgical centersContinued KOL engagement Treatment Centers Disease Education Regulatory Market Opportunity

| 42 Enduring Innovation Drives Value Creation 2026Key Drivers: Evrysdi®, PTC-AADC, Tegsedi®, Waylivra®, Translarna™, Vatiquinone, PTC923, Unesbulin $536M 2021Vatiquinone|PTC923 |PTC518 |PTC857 |Unesbulin|Emvododstat|GT-FA |GT-AS |Research 2030Key Drivers: Evrysdi®, PTC-AADC, Tegsedi®, Waylivra®, Vatiquinone, PTC923, Unesbulin, PTC518, PTC857 ~$3B ~$8BCommercial Clinical Unaudited total revenuePotential future revenues