Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — 2020 J.P. Morgan Healthcare Conference
37 slides from Ptc Therapeutics's conference presentation, Jan 17, 2020
Slide 1: PTC 2020 JP Morgan Healthcare Conference
PTC 2020 JP Morgan Healthcare Conference Stuart W. Peltz, Ph.D., CEO
Slide 2: Forward looking statement
Forwar lookingstatement Th i s pr e s en tat i o n co n ta i ns for w ar d - lo o k in g s ta tem en t s wi t h in the m ean i ng o f T he P r i va te S e c u r i ti e s Li ti g a ti o n R efor m Ac t o f 1995 . Al l s ta tem en t s c on ta i ned in t h is pr e s en tat i on , o ther than s ta tem e
Slide 3: A global, commercial, diversified biopharmaceutical company for rare genetic disorders
A global, commercial, diversified, biopharmaceutical company focused on innovative therapies for rare genetic disorders 3
Slide 4: Global commercial capabilities & infrastructure
Global commercial capabilities & infrastructure Offices in 20 countries Footprint in >50 countries 800+ employees 4
Slide 5: Strong financial performance supports future growth
Strong financial performance supports future growth ~$306M 2019 Unaudited Net Revenue New product launches and approvals could also contribute 2020 revenue to $291M 2019 DMD Franchise Net Product Unaudited Net Revenue (e.g.,Tegsedi, commercial Waylivra EAP, PTC-AADC and risdiplam) $320–$340M 2020 DM
Slide 6: Significant success-based revenue through remaining risdiplam milestones and royalties
Significant success-based revenue through remaining risdiplam milestones and royalties Total remaining regulatory milestone-based payments Potential 2020 risdiplam milestone payments to PTC $72.5M Milestone Payment Up to NDA Filing in Japan $ 7,500,000 mid-teen royalties Total sales-threshold-based
Slide 7: Multiplatform approach builds diversified pipeline
Multiplatform approach builds diversified pipeline SCIENTIFIC PLATFORMS & RESEARCH * Investigator-initiated study with NYU 7 Potential registrational studies Commercial SMA AADC Clinical HD FA PTC857 GBA-PD Angelman Undisclosed IRDs Research Cog Disorders Undisclosed PTC299 AML PTC596 LMS PTC596 DIP
Slide 8: Sustainable innovation drives continuous value creation
Sustainable innovation drives continuous value creation 2023 DMD Franchise, PTC-AADC, risdiplam, Tegsedi™, Waylivra® 2020* DMD Franchise (risdiplam, Tegsedi™, Waylivra®, PTC-AADC) REVENUE Beyond 2023 Nonsense Mutation Gene Therapy Splicing Redox 2019 DMD Franchise (unaudited) $1.5B 2016 Translarna™
Slide 9: Multiple potential value driving events in 2020
Multiple potential value driving 1Q20 events in 2020 4Q19 2Q20 3Q20 4Q20 ales through early access program Clinical Regulatory Commercial 9 Analyst Day Nonsense Mutation Aniridia data US dystrophin data Splicing Risdiplam NDA filed SUNFISH Part 2 topline SUNFISH Part 2 data FIREFISH Part 2 topline R
Slide 10: Multiplatform approach builds diversified pipeline
Multiplatform approach builds diversified pipeline SCIENTIFIC PLATFORMS & RESEARCH * Investigator-initiated study with NYU 1 0 Potential registrational studies Commercial SMA AADC Clinical HD FA PTC857 GBA-PD Angelman Undisclosed IRDs Research Cog Disorders Undisclosed PTC299 AML PTC596 LMS PTC596 D
Slide 11: Translarna demonstrates long-term benefit in DMD patients
Translarna™ demonstrates long-term benefit in DMD patients EU5 nmDMD patients treated with Translarna Compliance STRIDE is a real-world, long-term registry of patients receiving Translarna Translarna treatment slowed disease progression in nmDMD compared to matched natural history patients 2.2% Tran
Slide 12: Growth opportunities in DMD franchise
Growth opportunities in DMD franchise Translarna • Increased penetration in existing territories Geographic expansion into new territories Increased awareness and earlier diagnosis Potential US NDA submission in 2020 2020 net DMD Franchise revenue guidance 2019 net DMD Franchise revenue • • $190M (u
Slide 13: Multiplatform approach builds diversified pipeline
Multiplatform approach builds diversified pipeline SCIENTIFIC PLATFORMS & RESEARCH * Investigator-initiated study with NYU 1 3 Potential registrational studies Commercial SMA AADC Clinical HD FA PTC857 GBA-PD Angelman Undisclosed IRDs Research Cog Disorders Undisclosed PTC299 AML PTC596 LMS PTC596 D
Slide 14: Splicing is highly selective with broad applicability
Splicing is highly selective with broad applicability Isoform plex Proprietary RNA-targeted small molecule library HTSpliceseq Transcriptome mRNA isoform detection platforms Exploiting U1-noncanonical exons 2020 and beyond Risdiplam SMN2 exon 7 inclusion Alternative splicing Familial dysautonomia EL
Slide 15: Risdiplam: an oral small molecule SMN2 splicing modifier
Risdiplam: An modifier oral small molecule selective SMA splicing SMN1 SMN2 DNA 6 6 7 8 7 8 pre-mRNA 6 7 8 6 7 8 Risdiplam mRNA 6 7 8 6 8 Protein 2b 7 1 2a 2b 3 4 5 6 1 2a 3 4 5 6 8 Functional Unstable 1 5
Slide 16: Risdiplam – most competitive commercial profile across broadest population
Risdiplam – Most population competitive commercial profile across broadest FIREFISH – Type 1 SMA • Studied in Type 1,2,3 patients from newborns to 60 years of age • No treatment-related safety findings leading to withdrawal 82% on high dose had a CHOP-INTEND ≥ 40 48% of all infants were able to main
Slide 17: Risdiplam – SMN protein in blood of SMA patients and healthy subjects
Risdiplam – Most competitive commercial profile population across broadest SMN protein in blood of SMA patients and healthy subjects • Studied in Type 1,2,3 patients from newborns to 60 years of age before and after 4 weeks of treatment 8 • No treatment-related safety findings leading to withdrawal
Slide 18: Toxic HTT protein aggregation causes extensive neuronal cell death
Toxic HTT death protein aggregation causes extensive neuronal cell Healthy HD Goh et al. Aus Psychiatry. 2018 1 8
Slide 19: Splicing modifiers reduce HTT protein in Huntington disease
Splicing modifiers reduce HTT protein in Huntington disease Healthy HD HD is a neurodegenerative disease caused by a toxic gain-of-function triplet repeat (CAG) expansion in the huntingtin gene (CAG)>35 (CAG)>35 Stop exon Stop exon Treatment with splicing modifier Untreated Ex 1 Ex X Ex Y Ex 1 Ex X
Slide 20: HD splicing small molecules have broad tissue distribution
HD splicing small molecules have broad tissue distribution Dose dependent HTT lowering in the brain in BACHD mice HD IND expected late 2020 • • • • Oral, crosses BBB Titratable Development candidate selected Ability to measure mRNA and protein in blood in healthy volunteers Brain 120 100 80 60 40 20
Slide 21: Multiplatform approach builds diversified pipeline
Multiplatform approach builds diversified pipeline SCIENTIFIC PLATFORMS & RESEARCH * Investigator-initiated study with NYU 2 1 Potential registrational studies Commercial SMA AADC Clinical HD FA PTC857 GBA-PD Angelman Undisclosed IRDs Research Cog Disorders Undisclosed PTC299 AML PTC596 LMS PTC596 D
Slide 22: Treating rare monogenic disorders with targeted gene therapy
Treating rare monogenic disorders with targeted gene therapy Potential advantages of targeted therapy • Local administration lowers systemic immunogenicity and exposure Low turnover cells may lead to improved durability • • Micro-dosing lowers manufacturing and patient burden Pipeline • • • • • PTC-
Slide 23: Internal gene therapy manufacturing capabilities
Internal gene therapy manufacturing capabilities • • In-house manufacturing begins in 2020 15-year lease on ~185,000 sq. ft. which includes a state-of-the-art biologics production facility with supporting research and operations buildings in NJ • Highly qualified staff in biologics manufacturing joi
Slide 24: AADC deficiency – rare disorder with significant unmet need
AADC deficiency – Rare disorder with significant unmet need Normal AADC • Rare progressive childhood disease, affecting approximately 5,000 patients globally Head Position 3-4 months Up • Children with severe AADC deficiency never achieve motor development milestones • Profound development failure w
Slide 25: PTC-AADC patients make significant and sustainable progress
PTC-AADC patients make significant and sustainable progress Untreated Post-Treatment Age 2 Age 3 Age 4.5 2 5
Slide 26: PTC-AADC demonstrates significant and durable motor improvements
PTC-AADC demonstrates significant and durable motor improvements across patient population 1-Year Results of Motor Development PDMS-21 through 12 months – Studies 1 & 2 5-Year Results of Motor Development PDMS-21 through 60 months - Study 1 150 150 100 100 50 50 0 60 0 20 40 60 80 100 120 20 40 80 1
Slide 27: Expertise in patient identification
Expertise in patient identification Early diagnosis Increased awareness Genotyping 2 7 AADC US efforts: No-cost testing via whole blood 3-OMD newborn screening test; reflex to AADC enzyme activity and DDC gene sequencing AADC Ex-US efforts: No-cost testing via dried blood spot cards 3-OMD screening;
Slide 28: Expertise in patient identification
Expertise in patient identification Early diagnosis Increased awareness Genotyping 2 8 AADC US efforts: No-cost testing via whole blood 3-OMD newborn screening test; reflex to AADC enzyme activity and DDC gene sequencing AADC Ex-US efforts: No-cost testing via dried blood spot cards 3-OMD screening;
Slide 29: Friedreich Ataxia is a severe neuromuscular disorder amenable to gene therapy
Friedreich Ataxia (FA) is a severe neuromuscular disorder amenable to gene therapy Larger repeat expansion Lower protein levels • Inherited, monogenic disease arising from triplet repeat expansion Mutation in frataxin gene limits protein production • • Most common hereditary ataxia (~25,000 patients
Slide 30: Frataxin expression seen in large animal models
Frataxin expression seen in large animal models PTC-FA Intracerebellar Dosing in Porcine Model* PTC-FA Intracerebellar Dosing in NHP Model* 3,000 1,000 Cerebellar Cortex Dentate Nucleus Cerebellar Cortex Dentate Nucleus 2,500 800 2,000 600 1,500 400 1,000 Target Frataxin Protein Healthy Human DN 200
Slide 31: Multiplatform approach builds diversified pipeline
Multiplatform approach builds diversified pipeline SCIENTIFIC PLATFORMS & RESEARCH * Investigator-initiated study with NYU 3 1 Potential registrational studies Commercial SMA AADC Clinical HD FA PTC857 GBA-PD Angelman Undisclosed IRDs Research Cog Disorders Undisclosed PTC299 AML PTC596 LMS PTC596 D
Slide 32: Redox platform targets inflammation and oxidative stress
Redox platform is a novel approach focusing on inflammation and oxidative stress O OH Arachidonic acid 15-Lipoxygenase PTC743 & PTC857 15-lipoxygenase Lipid signaling molecule that regulates fundamental disease processes OH O O OH 15-OOH-Arachidonic acid 15(S)-HpETE* Glial Cell Activation & Inflamma
Slide 33: Initiating three redox clinical trials in 2020
Initiating three redox clinical trials in 2020 PTC743 PTC743 PTC857 Trial Starting 2Q20 Proof-of-concept in dozens of patients Clinical trials demonstrated reduction in hospitalizations and mortality risk in MEDS patients To enroll patients with 4 most common sub-types of MEDS Trial Starting 3Q20 Me
Slide 34: Multiplatform approach builds diversified pipeline
Multiplatform approach builds diversified pipeline SCIENTIFIC PLATFORMS & RESEARCH * Investigator-initiated study with NYU 3 4 Potential registrational studies Commercial SMA AADC Clinical HD FA PTC857 GBA-PD Angelman Undisclosed IRDs Research Cog Disorders Undisclosed PTC299 AML PTC596 LMS PTC596 D
Slide 35: Leveraging LatAm infrastructure to commercialize Tegsedi & Waylivra
Leveraging our existing LatAm infrastructure to commercialize Tegsedi & Waylivra Waylivra to utilize our patient Best fit for Latin American hATTR market ANVISA approval granted in 2019 Only approved hATTR indication to include improvements in QoL hATTR most prevalent phenotype in Latin America with
Slide 36: 2020 Potential milestones to generate value
2020 Potential milestones to generate value Waylivra approval US Translarna NDA submission PTC857 GBA Parkinson’s PTC743 MEDS IND in Huntington Disease Aniridia data AADC gene therapy approval FA gene therapy to enter clinic PTC743 FA Risdiplam approval 3 6
Slide 37: A global, commercial, diversified biopharmaceutical company for rare genetic disorders
A global, commercial, diversified, biopharmaceutical company focused on innovative therapies for rare genetic disorders 3 7
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