Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — 2021 J.P. Morgan Healthcare Conference
51 slides from Ptc Therapeutics's conference presentation, Jan 11, 2021
Slide 1: PTC 2021 J.P. Morgan Healthcare Conference
PTC 2021 J.P. Morgan Healthcare Conference Stuart W. Peltz, Ph.D., CEO
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 199 5. All statements contained in this release, other than statements of historic fact, are forward -looking statements, including statements with r
Slide 3: Translating Science to Transform Lives
Translating Science to Transform Lives 3
Slide 4: Significant Execution and Value Creation in 2020
Significant Execution and Value Creation in 2020 4 Clinical Regulatory Commercial Financial ✓ Initiated two potential registrational trials with vatiquinone in Mitochondrial Epilepsy & Friedreich ataxia ✓ Completed Translarna™ dystrophin trial for potential US accelerated approval ✓ Initiated trial
Slide 5: Diversified Platform Drives Strong Portfolio
Diversified Platform Drives Strong Portfolio 5 Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Gene Therapy Bio-e Metabolic Oncology US Dystrophin PTC-AADC PTC299 AML PTC596 DIPG PTC596 LMS Vatiquinone ME Vatiquinone FA PTC923 PKU PTC299 COVID-19 SCA-3 Undisclosed MAP-Tau
Slide 6: Enduring Innovation Engine
Enduring Innovation Engine 6
Slide 7: Diversified Platform Drives Strong Portfolio
Diversified Platform Drives Strong Portfolio 7 Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Gene Therapy Bio-e Metabolic Oncology US Dystrophin PTC-AADC PTC299 AML PTC596 DIPG PTC596 LMS Vatiquinone ME Vatiquinone FA PTC923 PKU PTC299 COVID-19 SCA-3 Undisclosed MAP-Tau
Slide 8: Bio-e Platform is a Novel Approach to Treating Rare Disorders
O OH O OH Oxidized Fatty Acids Enzyme Polyunsaturated Fatty Acids O OH Bio-e Platform is a Novel Approach to Treating Rare Disorders Polyunsaturated Fatty Acids Oxidized Fatty Acids Activated Enzyme Oxidative stress cellular viability & neurological function inflammation & oxidative stress decreased
Slide 9: Bio-e Platform is a Novel Approach to Treating Rare Disorders
Enzyme Polyunsaturated Fatty Acids O OH Bio-e Platform is a Novel Approach to Treating Rare Disorders Polyunsaturated Fatty Acids Oxidized Fatty Acids Activated Enzyme Oxidative stress Inhibited 9 PTC Compounds O O HO • Bio-e platform targets a family of oxidoreductase enzymes critical to generation
Slide 10: MIT-E: Registrational trial of vatiquinone for Mitochondrial Epilepsy
10 MIT-E: Registrational trial of vatiquinone for Mitochondrial Epilepsy
Slide 11: Mitochondrial Epilepsy is a Highly Morbid Disorder with High Unmet Need
Mitochondrial Epilepsy is a Highly Morbid Disorder with High Unmet Medical Need Disease • Mitochondrial epilepsy is the highly morbid condition of refractory seizures in patients with inherited mitochondrial disease Current Treatments • No approved disease modifying treatments for mitochondrial epil
Slide 12: Vatiquinone Reduced Seizure Frequency in Mitochondrial Epilepsy Patients
Vatiquinone Reduced Seizure Frequency and Improved Neurological Function in Mitochondrial Epilepsy Patients 12 Disruption of refractory status epilepticus Reduction in seizure frequency Decrease in seizure-related hospitalizations Decrease in disease-related mortality risk Data from previous studies
Slide 13: Vatiquinone Potential for Differentiated Improvement in Mitochondrial Epilepsy
Vatiquinone has Potential to Show Clinically Differentiated Improvement for Mitochondrial Epilepsy Patients 13 Primary Endpoint Change from baseline in frequency of observable motor seizures PTC743 (vatiquinone) Placebo N = 30 (planned) 24 weeks R N = 30 (planned) 48 weeks PTC743 (vatiquinone) N = 6
Slide 14: MOVE-FA: Registrational trial of vatiquinone for Friedreich Ataxia
14 MOVE-FA: Registrational trial of vatiquinone for Friedreich Ataxia
Slide 15: Friedreich Ataxia is a Highly Morbid Neuromuscular Disorder
Friedreich Ataxia is a Highly Morbid, Neuromuscular Disorder with no Approved Therapy Disease • Friedreich ataxia (FA) is a rare, inherited, progressive disease resulting from mitochondrial dysfunction that mainly affects the central nervous system and the heart Current Treatments • No approved dis
Slide 16: Vatiquinone Demonstrated Improvement in Friedreich Ataxia Patients
Vatiquinone Demonstrated Significant Improvement in Long-term Disease Severity & Neurological Function in Friedreich Ataxia Patients Clinical Study Summary • Double-blind, placebo-controlled with delayed start • N=63 subjects • Three US clinical sites • Key endpoint: FA disease rating scale (FARS) 1
Slide 17: Vatiquinone Potential to Improve Neurological Function
Vatiquinone has the Potential to Provide Improvement in Neurological Function 17 Primary and Key Secondary Endpoints Change from baseline in the Modified FA Rating Scale (mFARS) Score at Week 72 Improvement in activities of daily living (FA-ADL) Open-Label Extension Placebo-Controlled PTC743 (vatiqu
Slide 18: Diversified Platform Drives Strong Portfolio
Diversified Platform Drives Strong Portfolio 18 Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Gene Therapy Bio-e Metabolic Oncology US Dystrophin PTC-AADC PTC299 AML PTC596 DIPG PTC596 LMS Vatiquinone ME Vatiquinone FA PTC923 PKU PTC299 COVID-19 SCA-3 Undisclosed MAP-Ta
Slide 19: APHENITY: Registrational trial of PTC923 for PKU
19 APHENITY: Registrational trial of PTC923 for Phenylketonuria (PKU)
Slide 20: Phenylketonuria is a Serious Metabolic Condition with High Unmet Need
Phenylketonuria is a Serious Metabolic Condition with High Unmet Medical Need ~58,000 Global prevalence Disease • Phenylketonuria is a metabolic condition caused by mutations to phenylalanine hydroxylase that can lead to cognitive disabilities and seizures Current Treatments • Majority of patients d
Slide 21: Majority of PKU Patients not Addressed by Current Therapies
Majority of PKU Patients not Addressed by Current Therapies 21 ~ 25-30% of diagnosed patients ~60-75% of diagnosed patients
Slide 22: Majority of PKU Patients not Addressed by Current Therapies
Majority of PKU Patients not Addressed by Current Therapies 22 ~ 25-30% of diagnosed patients ~60-75% of diagnosed patients Up to 70% of Kuvan Failure Up to 60% of Initial Responders Are Poorly controlled on Kuvan Up to 30% of diagnosed patients
Slide 23: PTC923 Reduced Phenylalanine Compared to Kuvan in Phase 2
PTC923 Demonstrated Statistically Significant Differences in Reduction of Phenylalanine (Phe) Compared to Kuvan in Phase 2 Study 23 p< 0.0001
Slide 24: APHENITY is a Global Registrational Trial of PTC923
APHENITY is a Global Registrational Trial of PTC923 for Phenylketonuria 24 R PTC923 Placebo Primary Endpoint Reduction in blood of phenylalanine hydroxylase levels 42 Days ~12 sites in 15-19 countries Open-Label Extension Placebo-Controlled PTC923 12 Months Primary Endpoint Trial Status • Initiating
Slide 25: Diversified Platform Drives Strong Portfolio
Diversified Platform Drives Strong Portfolio 25 Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Gene Therapy Bio-e Metabolic Oncology US Dystrophin PTC-AADC PTC299 AML PTC596 DIPG PTC596 LMS Vatiquinone ME Vatiquinone FA PTC923 PKU PTC299 COVID-19 SCA-3 Undisclosed MAP-Ta
Slide 26: PTC518: Program for Huntington Disease
26 PTC518: Program for Huntington Disease
Slide 27: Huntington Disease is a Debilitating Neurological Disorder
Huntington Disease is a Debilitating Neurological Disorder with No Available Disease Modifying Treatments Disease • Huntington disease is a progressive brain disorder that causes uncontrolled movements and cognitive loss Current Treatments • No approved disease modifying therapies Opportunity • PTC5
Slide 28: Small Molecules Have a Critical Advantage for Pan Brain Distribution
Small Molecules Have a Critical Advantage for Pan Brain Distribution 28 HD Healthy Goh et al. Aus Psychiatry. 2018
Slide 29: Identification of a Novel Approach to Lower HTT
Identification of a Novel Approach to Lower HTT 29 HTT patient Ex 1 Ex Y Ex X Favored mRNA Ex 1 Ex Y Ex X Leads to toxic HTT protein (CAG)>35
Slide 30: Identification of a Novel Approach to Lower HTT
Identification of a Novel Approach to Lower HTT 30 HTT patient Ex 1 Ex Y Ex X Favored mRNA Ex 1 Ex Y Ex X Leads to toxic HTT protein (CAG)>35 Degraded through translation-linked mRNA decay Ex 1 Ex Y Ex X Mutant toxic HTT protein lowering Ex 1 Ex Y Ex X (CAG)>35 Small molecule assisted exon definitio
Slide 31: PTC518 Has Broad Tissue Distribution with Brain and Blood Correlation
PTC518 Has Broad Tissue Distribution with Strong Correlation between Brain and Blood 31 120 100 80 60 40 20 0 Vehicle 1 3 7 21 63 Brain % HTT Remaining Dose dependent HTT lowering in the brain in BACHD mice Dose (mg/kg)
Slide 32: PTC518 Has Broad Tissue Distribution with Brain and Blood Correlation
PTC518 Has Broad Tissue Distribution with Strong Correlation between Brain and Blood 32 120 100 80 60 40 20 0 Vehicle 1 3 7 21 63 Brain % HTT Remaining Dose dependent HTT lowering in the brain in BACHD mice Brain and blood HTT lowering Dose (mg/kg) *Data on file from multiple studies Measurements de
Slide 33: Potential Proof of Concept: HTT mRNA and Protein Reduction
Potential Proof of Concept: Ability to Define HTT mRNA and Protein Reduction Preliminary and confidential 33 Phase 1 trial in healthy volunteers is ongoing Single and multiple ascending doses Endpoints include safety, tolerability and pharmacokinetics Ability to define HTT mRNA and protein reduction
Slide 34: Diversified Platform Drives Strong Portfolio
Diversified Platform Drives Strong Portfolio 34 Clinical Commercial Research Nonsense Mutation LatAm Commercial Deflazacort Gene Therapy Bio-e Metabolic Oncology US Dystrophin PTC-AADC PTC299 AML PTC596 DIPG PTC596 LMS Vatiquinone ME Vatiquinone FA PTC923 PKU PTC299 COVID-19 SCA-3 Undisclosed MAP-Ta
Slide 35: Treating Rare Monogenic Disorders with Targeted Gene Therapy
Treating Rare Monogenic Disorders with Targeted Gene Therapy 35 Potential advantages of targeted therapy • Local administration lowers systemic immunogenicity and exposure • Low turnover cells may lead to improved durability • Leveraging stereotactic neurosurgery technologies to enable precise and a
Slide 36: Gene Therapy Potential Benefit to AADC Patients
Gene Therapy Has the Potential to Provide Significant Benefit to AADC 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 • N
Slide 37: PTC-AADC Treated Patients Make Significant and Sustainable Progress
PTC-AADC Treated Patients Make Significant and Sustainable Progress 37 Age 2 Age 3 Age 4.5 Untreated Post-Treatment
Slide 38: Preparing for PTC's First Gene Therapy Launch
Preparing for PTC’s First Gene Therapy Launch 38 US Regulatory PTC-AADC BLA submission expected in 1H21 Treatment Centers Identification and preparation of expert pediatric neurosurgical centers EU Regulatory PTC-AADC MAA submitted CHMP opinion expected in 1H21 Patient Finding Ongoing patient findin
Slide 39: Providing Patients Access to Transformative Treatments
Providing Patients Access to Transformative Treatments 39
Slide 40: Global Geographic Presence Supports Growing Product Portfolio
Global Geographic Presence Supports Growing Product Portfolio 40 ~1,000 employees Offices in 20 countries Footprint in >50 countries
Slide 41: DMD Commercial Franchise – A Growing Global Business
DMD Commercial Franchise – A Growing Global Business 41
Slide 42: DMD Commercial Franchise – A Growing Global Business
DMD Commercial Franchise – A Growing Global Business 42 • YE 2020 unaudited revenue of $192M • Treatment for nonsense mutation DMD for ages 2 and older • Distributed in over 50 countries worldwide • Dystrophin data in 1Q21 for potential US NDA submission
Slide 43: DMD Commercial Franchise – A Growing Global Business
DMD Commercial Franchise – A Growing Global Business 43 • YE 2020 unaudited revenue of $192M • Treatment for nonsense mutation DMD for ages 2 and older • Distributed in over 50 countries worldwide • Dystrophin data in 1Q21 for potential US NDA submission • YE 2020 unaudited revenue of $139M • First
Slide 44: Continued Strong DMD Franchise Growth
Continued Strong DMD Franchise Growth USD millions 34 81 145 171 190 29 92 101 2019 2020 2017 2015 2016 2018 263 174 331 +58% 291 Emflaza Net Sales Translarna Net Sales 139 192 44
Slide 45: Evrysdi's Strong Global Launch Brings Therapy to SMA Patients
Evrysdi’s Strong Global Launch Brings Therapy to SMA Patients with High Unmet Need 45 Patients treated across all SMA types Patients are treatment-naïve or switching from both Spinraza and Zolgensma Broadest range of age treated Differentiated product for SMA patients • The first at-home, oral treat
Slide 46: Multiple Platforms Provide Opportunity to Treat Over 500,000 Patients by 2030
Multiple Platforms Provide Opportunity to Treat Over 500,000 Patients by 2030 46 DMD SMA AADC ~50K ME ~20K PKU ~58K FA ~25K HD ~135K GBA-PD ~190K AS ~75K 2021 2030 AADC
Slide 47: Strong Financial Performance Supports Future Growth
Strong Financial Performance Supports Future Growth 47 Unaudited Net Product Revenue $333M DMD Franchise Net Product Unaudited Net Revenue $331M $1.1B Year-end Unaudited Cash Position 2020
Slide 48: Strong Financial Performance Supports Future Growth
Strong Financial Performance Supports Future Growth 48 DMD Franchise Net Product Revenue Guidance $355-375M OPEX Guidance* $725-755M Unaudited Net Product Revenue $333M DMD Franchise Net Product Unaudited Net Revenue $331M 2020 2021 $1.1B Year-end Unaudited Cash Position *Non-GAAP measure which excl
Slide 49: 2021 Potential Milestones to Generate Value
2021 Potential Milestones to Generate Value 49 AADC CHMP Opinion and BLA Submission PTC857 Data PTC518 Data Translarna Dystrophin Data PTC-FA First Patient Dosed PTC596 LMS and DIPG Data PTC923 PKU P3Trial Initiated PTC299 COVID-19 Data 1H 2021 2H 2021
Slide 50: PTC has Built a Strong, Sustainable Company to Execute on our Mission
PTC has Built a Strong, Sustainable Company to Execute on our Mission 50 Proven groundbreaking science Enduring innovation engine Providing patients access to transformative treatments Culture Operational Excellence
Slide 51: Translating Science to Transform Lives
Translating Science to Transform Lives 51
PTC Therapeutics — 2021 J.P. Morgan Healthcare Conference on 6ix