Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — Investor Presentation (September 2022)
42 slides from Ptc Therapeutics's investor presentation, Sep 7, 2022
Slide 1: PTC 2022 Corporate Presentation
PTC 2022 Corporate Presentation September 2022
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 statem
Slide 3: PTC Continually Innovates to Bring New Therapies to Patients
Commercialize Providing patients with access to transformative treatments Develop Enduring innovation engine PTC Continually Innovates to Bring New Therapies to Patients 3 Discover Proven groundbreaking science
Slide 4: Building a Pipeline to Produce a Therapy Every 2 to 3 Years
4 Building a Pipeline to Produce a Therapy Every 2 - 3 Years Discover Develop 2 to 3 years New product every Commercialize
Slide 5: PTC Has a Growing Global Footprint
PTC Has a Growing Global Footprint 5 1,300+ employees 20 offices worldwide Footprint in >50 countries Founded in 1998
Slide 6: Diversified Platform Drives Strong Portfolio
Diversified Platform Drives Strong Portfolio 6 Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Metabolic Oncology US A taluren Emvododstat AML Unesbulin DIPG Unesbulin LMS PTC923 PKU Emvododstat COVID - 19 Virology SCIENTIFIC PLATFORMS and RESEARCH SCA - 3 MAP - Tau PTC51
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 2030 7 ALS + ~1 50K DMD/SMA / AADC + ~ 50K Mito Sz + ~20 K PKU + ~ 58K FA + ~ 25K HD + ~ 135K 2021 2030 =10,000 pts GBA - PD + ~ 190K AS + ~75K ~ 50K ~ 70K ~ 128K ~ 153K ~ 288K ~ 438K ~ 628K ~ 703K Estimated Global Prevalence
Slide 8: Enduring Innovation Drives Value Creation
8 Enduring Innovation Drives Value Creation 2026 Key Drivers: Evrysdi ® , PTC - AADC , Tegsedi ® , Waylivra ® , Translarna ™ , Vatiquinone , PTC923, Unesbulin $539M 2021 Vatiquinone | PTC923 | PTC518 | PTC857 | Unesbulin | Emvododstat | GT - FA | GT - AS | Research 2030 Key Drivers: Evrysdi ® , PTC
Slide 9: Revenue Contribution of Our Pipeline Grows
Revenue Contribution of Our Pipeline Grows 9 Potential future revenues Commercial Products + Royalties Current Pipeline 57 % 43 % 2026 2025 64 % 36 % 2024 2023 88 % 12 % 100 %
Slide 10: Strong Financial Performance Supports Innovation
Strong Financial Performance Supports Innovation 10 Total Revenue $539 M DMD Franchise Net Product Revenue $423 M *Non - GAAP measure which excludes estimated non - cash, stock - based compensation expense of approximately $115 million. GAAP R&D and SG&A expense for the full year 2022 is anticipated
Slide 11: Diversified Platform Drives Strong Portfolio
Diversified Platform Drives Strong Portfolio 11 Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Metabolic Oncology US A taluren Emvododstat AML Unesbulin DIPG Unesbulin LMS PTC923 PKU Emvododstat COVID - 19 Virology SCIENTIFIC PLATFORMS and RESEARCH SCA - 3 MAP - Tau PTC5
Slide 12: Success Across Our Commercial Portfolio
Success Across Our Commercial Portfolio 12 • Treatment for nonsense mutation DMD for ages 2 and older • Distributed in 50+ countries • New patients in existing geographies and geographic expansion • First and only corticosteroid approved for DMD; approved for all US DMD patients >2yrs • Data show cl
Slide 13: Continued Strong DMD Franchise Growth
13 Continued Strong DMD Franchise Growth Net Product Revenue (USD Millions) 34 81 29 92 101 139 188 145 171 190 192 236 2018 2020 2015 2016 2017 2019 291 2021 174 263 331 423 +52% Emflaza Translarna
Slide 14: Upstaza Has the Potential to Provide Significant Benefit to AADC Deficiency Patients
Disease Aromatic L - amino acid decarboxylase deficiency (AADC - d) is a rare, highly morbid, and fatal childhood disease. Children with severe AADC deficiency never achieve motor development milestones. Current Treatments Upstaza is the first and only approved disease - modifying therapy for AADC -
Slide 15: Upstaza Gene Therapy for AADC Deficiency Approved by European Commission
15 Upstaza Gene Therapy for AADC Deficiency Approved by European Commission • Potential over $1B in cumulative revenue • Successful patient finding is ongoing Approved by EMA in July • PTC - AADC BLA submission expected in 4Q22 • Development of virtual education: disease - specific webinars and cong
Slide 16: Diversified Platform Drives Strong Portfolio
Diversified Platform Drives Strong Portfolio 16 Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Metabolic Oncology US A taluren Emvododstat AML Unesbulin DIPG Unesbulin LMS PTC923 PKU Emvododstat COVID - 19 Virology SCIENTIFIC PLATFORMS and RESEARCH SCA - 3 MAP - Tau PTC5
Slide 17: Substantial Pipeline Progress
Substantial Pipeline Progress Planned 17 Study Results Study Initiations 1Q 2023 3Q 2022 4Q 2022 1H 2022 2Q 2023 GT - FA Ph1 Initiation DIPG Ph2 Initiation Study 041 12 - week data
Slide 18: Three Registration-Directed Clinical Trials Drive Near-Term Value
18 Data Expected 1 Q 2023 Vatiquinone Mitochondrial Disease Associated Seizures Data Expected 2Q 2023 Vatiquinone Friedreich Ataxia Data Expected YE 2022 PTC923 PKU Three Registration - Directed Clinical Trials Drive Near - Term Value
Slide 19: Diversified Platform Drives Strong Portfolio
SCA - 3 MAP - Tau PTC518 HD Splicing Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Metabolic Oncology US A taluren Emvododstat AML Unesbulin DIPG Unesbulin LMS PTC923 PKU Emvododstat COVID - 19 Virology AADC, aromatic L - amino acid decarboxylase deficiency; AML; acute
Slide 20: MIT-E: Registration-directed trial of vatiquinone for Mitochondrial Disease Associated Seizures
20 MIT - E: Registration - directed trial of vatiquinone for Mitochondrial Disease Associated Seizures
Slide 21: Vatiquinone Has the 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
Slide 22: Vatiquinone Has the Potential to Show Clinically Differentiated Improvement for MDAS Patients
Vatiquinone Has the Potential to Show Clinically Differentiated Improvement for MDAS Patients 22 Primary Endpoint Cha nge from baseline in frequency of observable motor seizures PTC743 ( vatiquinone ) Placebo N = 30 24 weeks R N = 30 48 weeks PTC743 ( vatiquinone ) N = 60 Open - Label Extension Plac
Slide 23: MOVE-FA: Registration-directed trial of vatiquinone for Friedreich Ataxia
23 MOVE - FA: Registration - directed trial of vatiquinone for Friedreich Ataxia
Slide 24: Vatiquinone Has the Potential to Provide Improvement in Neurological Function
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 disrup
Slide 25: Vatiquinone Has the Potential to Provide Improvement in Neurological Function
Vatiquinone Has the Potential to Provide Improvement in Neurological Function 25 Primary Endpoint Change in mFARS Key Secondary Endpoint Change in FA - ADL Open - Label Extension Placebo - Controlled PTC743 ( vatiquinone ) Placebo N = ~ 60 72 weeks R N = ~ 60 24 weeks PTC743 ( vatiquinone ) N = ~120
Slide 26: CardinALS: Phase 3 trial of PTC857 for Amyotrophic Lateral Sclerosis
26 CardinALS : Phase 2 trial of PTC857 for Amyotrophic Lateral Sclerosis
Slide 27: PTC857 Has the Potential to Slow Disease Progression in ALS
27 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 ox
Slide 28: PTC857 Has the Potential to Slow Disease Progression in ALS
Primary Endpoints Change in ALSFRS - R Secondary Endpoints Safety and PK 28 Treatment Period PTC857 250 mg BID Placebo 24 weeks Primary Endpoint R Long - Term Treatment Extension 28 weeks PTC857 250 mg BID Trial Status • Initiated in 1Q 2022 Screening 8 weeks PTC857 Has the Potential to Slow Disease
Slide 29: Diversified Platform Drives Strong Portfolio
Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Metabolic Oncology US A taluren Emvododstat AML Unesbulin DIPG Unesbulin LMS PTC923 PKU Emvododstat COVID - 19 Virology AADC, aromatic L - amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID - 19, coronav
Slide 30: PIVOT HD: PTC518 for Huntington's Disease
30 PIVOT HD: PTC518 for Huntington's Disease
Slide 31: PTC518 Reduces HTT mRNA and Protein to Target the Proximal Cause of HD
31 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 HT
Slide 32: 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 32 Results from Phase 1 Healthy Volunteer Study PTC518 Reduces HTT mRNA and Protein to Target the Proximal Cause of HD
Slide 33: PTC518 Has the Potential to Reduce HTT Protein in Huntington Patients
33 PTC518 Has the Potential to Reduce HTT Protein in Huntington Patients Trial Status • Initiated in 1Q 2022 3 months 9 months 5 mg PTC518 10 mg PTC518 Alternative Dose PTC518 R Primary Endpoint N = ~30 N = ~ 30 N = ~ 30 Part A Part B • Safety and tolerability of PTC518 in Huntington disease patient
Slide 34: PIVOT HD Trial Target Population and Endpoints
34 • Safety and tolerability of PTC518 in Huntington’s disease patients • Percent reduction in HTT mRNA and protein in blood Primary Endpoints • Percent reduction in HTT protein in CSF • Changes in neurofilament light chain ( NfL ) in plasma and CSF • Change in caudate, putamenal , ventricular volum
Slide 35: APHENITY: Registration-directed trial of PTC923 for PKU
35 APHENITY: Registration - directed trial of PTC923 for PKU
Slide 36: APHENITY Is a Global Registration-Directed Trial of PTC923 for PKU
36 Disease Phenylketonuria (PKU) is a metabolic condition caused by mutations to phenylalanine hydroxylase that can lead to cognitive disabilities and seizures Current Treatments Majority of patients do not initially respond or are not well controlled by standard of care Mechanism of Action PTC923 i
Slide 37: APHENITY Is a Global Registration-Directed Trial of PTC923 for PKU
APHENITY Is a Global Registration - Directed Trial of PTC923 for PKU 37 Primary Endpoint Reduction in blood phenylalanine levels Primary Endpoint Trial Status • Data expected YE 2022 R PTC923 Placebo 42 Days Open - Label Extension Placebo - Controlled PTC923 12 Months PTC923 14 Days Run - in
Slide 38: Diversified Platform Drives Strong Portfolio
Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Metabolic US A taluren PTC923 PKU Emvododstat COVID - 19 Virology AADC, aromatic L - amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID - 19, coronavirus disease 2019; DIPG, dif fuse intrinsic pontine gl
Slide 39: Unesbulin Has the Opportunity to Provide Additional Progression-Free Survival in LMS
39 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 40: Unesbulin Has the Opportunity to Provide Additional Progression-Free Survival in LMS
40 Phase 1b study results 300 mg was selected as RP2D Unesbulin was well tolerated 21 Day Treatment Cycles Phase 1b Study Design Ascending doses 200, 300 and 400 mg u nesbulin + 1000mg/m 2 dacarbazine N = 29 • Patients with locally advanced or metastatic LMS intolerant or refractory to standard ther
Slide 41: Unesbulin Has the Opportunity to Provide Additional Progression-Free Survival in LMS
Unesbulin Has the Opportunity to Provide Additional Progression - Free Survival in LMS R 300 mg U nesbulin + 1000mg/m 2 dacarbazine Placebo + 1000 mg/ m 2 dacarbazine Primary Endpoint PFS as determined by RECIST Secondary Endpoints OS, ORR, DCR, DOR 21 Day Treatment Cycle 24 Months Interim Analysis
Slide 42: Enduring Innovation Drives Value Creation
42 Enduring Innovation Drives Value Creation 2026 Key Drivers: Evrysdi ® , Upstaza ™ , Tegsedi ® , Waylivra ® , Translarna ™ , Vatiquinone , PTC923, Unesbulin $539M 2021 Vatiquinone | PTC923 | PTC518 | PTC857 | Unesbulin | Emvododstat | GT - FA | GT - AS | Research 2030 Key Drivers: Evrysdi ® , Upst
PTC Therapeutics — Investor Presentation (September 2022) on 6ix