Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — 2022 J.P. Morgan Healthcare Conference
42 slides from Ptc Therapeutics's conference presentation, Jan 10, 2022
Slide 1: PTC 2022 J.P. Morgan Healthcare Conference
PTC 2022J.P. Morgan Healthcare Conference Stuart W. Peltz, Ph.D., CEO
Slide 2: Forward Looking Statements
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
Slide 3: PTC Continually Innovates to Bring New Therapies to Patients
CommercializeProviding patients with access to transformative treatments DevelopEnduring innovation enginePTC Continually Innovates to Bring New Therapies to Patients3 DiscoverProven groundbreaking science
Slide 4: Building a Pipeline to Produce a Therapy Every Three Years
4Building a Pipeline to Produce a Therapy Every Three Years Discover Develop 2to 3yearsNew product every Commercialize
Slide 5: PTC has a Growing Global Footprint
PTC has a Growing Global Footprint5 ~1,200 employees 20 offices worldwide Footprint in >50 countries Founded in 1998
Slide 6: Diversified Platform Drives Strong Portfolio
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 Bi
Slide 7: Multiple Platforms Provide Opportunity to Target Over 700,000 Patients by 2030
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
Slide 8: Enduring Innovation Drives Value Creation
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,
Slide 9: Revenue Contribution of our Pipeline Grows
Revenue Contribution of our Pipeline Grows9Potential future revenues Commercial Products + RoyaltiesCurrent Pipeline 57%43%20262025 64%36%20242023 88%12% 100%
Slide 10: 2021 Accomplishments
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 1Q22Enrollment update MIT-E, MOVE-FA?Initiation of registration- directed APHENITY Phase 3 trial for PTC923 in PKUCompletion of unesbulinPhase 1b oncology
Slide 11: Strong Financial Performance Supports Innovation
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 anticipat
Slide 12: Diversified Platform Drives Strong Portfolio
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
Slide 13: Five Registration-Directed Clinical Trials Drives Near-Term Value
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 Clin
Slide 14: Substantial Pipeline Progress Planned in 2022
Substantial Pipeline Progress Planned in 202214Study ResultsStudy Initiations GT-FAPh1Initiation DIPG Ph2 Initiation Study 041 2022 Q42022 Q22022 Q32022 Q1
Slide 15: Diversified Platform Drives Strong Portfolio
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;
Slide 16: MIT-E: Registration-directed trial of vatiquinone for MDAS
16 MIT-E:Registration-directed trial of vatiquinoneforMitochondrial Disease Associated Seizures
Slide 17: Vatiquinone has Potential to Show Clinically Differentiated Improvement for MDAS Patients
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 e
Slide 18: Vatiquinone MDAS Trial Design
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 Prima
Slide 19: MOVE-FA: Registration-directed trial of vatiquinone for Friedreich Ataxia
19MOVE-FA: Registration-directed trial of vatiquinone for Friedreich Ataxia
Slide 20: Vatiquinone Potential to Improve Neurological Function in FA
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
Slide 21: Vatiquinone FA Trial Design
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 Tri
Slide 22: CardinALS: Phase 2 trial of PTC857 for ALS
22CardinALS:Phase 2 trial of PTC857 for Amyotrophic Lateral Sclerosis
Slide 23: PTC857 has Potential to Slow Disease Progression in ALS
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 oxid
Slide 24: PTC857 ALS Trial Design
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 =
Slide 25: Diversified Platform Drives Strong Portfolio
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 2
Slide 26: PIVOT HD: PTC518 for Huntington's Disease
26 PIVOT HD:PTC518 forHuntington's Disease
Slide 27: PTC518 Reduces HTT mRNA and Protein to Target the Proximal Cause of HD
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
Slide 28: Results from Phase 1 Healthy Volunteer Study
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
Slide 29: PTC518 has the Potential to Reduce HTT Protein in Huntington Patients
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 pati
Slide 30: PIVOT HD Trial Will Target the Goldilocks Population
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 volume
Slide 31: APHENITY: Registration-directed trial of PTC923 for PKU
31 APHENITY:Registration-directed trial of PTC923 forPKU
Slide 32: APHENITY is a Global Registration-Directed Trial of PTC923 for PKU
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
Slide 33: APHENITY Trial Design
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
Slide 34: Diversified Platform Drives Strong Portfolio
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, Fr
Slide 35: Unesbulin Opportunity in Leiomyosarcoma
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 cell
Slide 36: Unesbulin Phase 1b Study Results
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
Slide 37: Unesbulin LMS Phase 2/3 Trial Design
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 ≈ 23037Tria
Slide 38: Diversified Platform Drives Strong Portfolio
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 Angelm
Slide 39: Commercial Products Overview
•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 aft
Slide 40: Continued Strong DMD Franchise Growth
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
Slide 41: PTC-AADC Gene Therapy Opportunity and Launch Preparation
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
Slide 42: Enduring Innovation Drives Value Creation
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®, Vati
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