Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — JP Morgan Healthcare Conference 2023
34 slides from Ptc Therapeutics's conference presentation, Jan 9, 2023
Slide 1: PKU Patient, Matthew Klein, M.D., COO – JP Morgan Healthcare Conference
PKU Patient Matthew Klein, M.D., COO JP Morgan Healthcare Conference January 9, 2023 PTC 2023
Slide 2: Forward-Looking Statements
Forward - qLooqking Statements This prqesqeqntatioqn coqntaiqns forward - lookiqng statqemqeqnts withiqn thqe mqeaqniqng of Thqe Privatqe Sqecuritiqes Litigatioqn Rqeform Act of 1995 .. All statqemqeqnts coqntaiqnqed iqn this prqesqeqntatioqn, othqer thaqn statqemqeqnts of historic fact, arqe forwar
Slide 3: Treating Rare Diseases by Modulating Gene and Protein Expression
Treating Rare Diseases by Modulating Gene and Protein Expression 3 3 DNA Protein RNA JP Morgan Presentation 2023
Slide 4: Treating Rare Diseases by Modulating Gene and Protein Expression
4 Treating Rare Diseases by Modulating Gene and Protein Expression JP Morgan Presentation 2023 Bio - e Splicing Nonsense Suppression Gene Therapy
Slide 5: Building a Pipeline to Produce a Therapy Every Two to Three Years
Building a Pipeline to Produce a Therapy Every Two to Three Years JP Morgan Presentation 2023 5 2 to 3 years New product every Commercialize Discover Develop
Slide 6: A Diverse and Robust Portfolio To Continually Create Value
Development Research Oncology Metabolism Neurology A Diverse and Robust Portfolio To Continually Create Value JP Morgan Presentation 2023 6 6 SCA - 3 MAP - Tau 3 Undisclosed GT - AS GT - FA PTC518 HD Utreloxastat ALS Unesbulin DIPG Unesbulin LMS Sepiapterin PKU V atiquinone MDAS V atiquinone FA Comm
Slide 7: Continuing Strong Financial Performance Expected in 2023
Continuing Strong Financial Performance Expected in 2023 JP Morgan Presentation 2023 7 *Non - GAAP measure wh:ich excludes est:imated non - cash, stock - based compensat:ion expense o:f approx:imately $120 m:ill:ion. GAAP R&D and SG&A expense :for the :full year 2023 :is ant:ic:ipated to be between
Slide 8: Continued Success Across Our Commercial Portfolio
JP Morgan Presentation 2023 Continued Success Across Our Commercial Portfolio Distributed in 50+ countries with continued growth from new patients and geographic expansion First and only corticosteroid for all US DMD patients with growth from new patient starts and favorable access E stablished mark
Slide 9: Building Foundation for Upstaza Success
JP Morgan Presentation 2023 9 Early Access Commercial Access Cross Border Building Foundation for Upstaza Success 2022
Slide 10: Substantial Growth in 2023 and Beyond with Additional Global Registrations
JP Morgan Presentation 2023 10 Early Access Commercial Access Cross Border 2022 2023 and Beyond Substantial Growth in 2023 and Beyond with Additional Global Registrations
Slide 11: Substantial Revenue Growth from 2021 to 2023
Substantial Revenue Growth from 2021 to 2023 JP Morgan Presentation 2023 11 Commercixaxl Sepiapterin | Vatiquinone | Unesbulin | PTC518 | Utreloxastat | Devexlopment $539 M 2021 Total revenue $116m Other $423m DMD Unaudited total revenue 2022 ~$710 M $ 203 m Other $507m DMD Potential future revenues
Slide 12: Strong R&D Execution and Value Creation in 2022
Strong R&D Execution and Value Creation in 2022 Clinical Achievements JP Morgan Presentation 2023 12 Regulatory Achievements Initiated SunriseLMS trial for unesbulin Initiated CardinALS study for utreloxastat Completed FITE - 19 study for emvododstat Waylivra approved in Brazil for treatment of FPL
Slide 13: Substantial Pipeline Progress Planned in 2023
JP Morgan Presentation 2023 13 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Substantial Pipeline Progress Planned in 2023
Slide 14: Substantial Pipeline Progress Planned in 2023
JP Morgan Presentation 2023 14 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Substantial Pipeline Progress Planned in 2023
Slide 15: Sepiapterin Can Potentially Treat Broad PKU Population Including Classical PKU
~ 58,000 Global Prevalence Sepiapterin Can Potentially Treat Broad PKU Population Including Classical PKU JP Morgan Presentation 2023 15 Disease Phenyxlketonurixa (PKU) is a metaboli(c (condition (caused by mutations to p(henylalanine (hydroxylase t(hat (can lead to (cognitive disabilities and seizu
Slide 16: Large Unmet Need Remains in PKU
qLarge Unmet Need Remains in PKU JP Morgan Presentation 2023 16 of pxatients xare wexlxl - controxlxled on Kuvxan Less thxan 10% 58,000 patients globally 30% therxapy nxaïve incxluding cxlxassicxaxl PKU 70% tried Kuvan 60% poorly controlled 40% well controlled Up to 70% fail ~30% respond 1. B:ioMar:
Slide 17: Established Pillars for Commercial Success
Newborn screening with ~58,000 patients worldwide 1,2,3 Well - known metabolic centers of excellence across the world Connected and coordinated patient advocacy community Disease pathology well understood and documented Established Pillars for Commercial Success JP Morgan Presentation 2023 17 1. NPK
Slide 18: Mechanistic Advantages of Sepiapterin Over Sapropterin
Mechanistic Advantages of Sepiapterin Over Sapropterin : Increased Bioavailability GI Trxact Pxlxasmxa Cell Membrane Intrxacexlxluxlxar (xliver, brxain, kidney) Synthetic Sepiapterin (PTC923) Rapid Cross - Membrane Active Transport Sepiapterin Sepiapterin reductase Oxidation Synthetic BH 4 (Kuvan ®
Slide 19: APHENITY is a Global Registration-Directed Trial of Sepiapterin for PKU
APHENITY is a Global Registration - Directed Trial of Sepiapterin for PKU Primary Endpoint Reduction in blood phenylalanine levels Primxary Endpoint R sepiapterin Placebo 42 Days Open - Label Extension Placebo - Controlled s epixapterin 12 Months s epixapterin 14 Days Run - in 19 JP Morgan Presentat
Slide 20: APHENITY Part 1 Preliminary Data (n=104)
JP Morgan Presentation 2023 20 APHENITY Part 1 Preliminary Data (n=104) Part 1 is an open - label run - in phase to identify subjects to be randomized The primary analysis population is those who have a ≥30% Phe reduction 0 10 20 30 40 50 60 70 - 65 % Sepiapterin - 29 % Kuvan 1 +122% 0 5 10 15 20 25
Slide 21: Substantial Pipeline Progress Planned in 2023
JP Morgan Presentation 2023 21 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Substantial Pipeline Progress Planned in 2023
Slide 22: Vatiquinone Has the Potential to Show Clinically Differentiated Improvement for MDAS Patients
JP Morgan Presentation 2023 22 Vatiqqk DTLuinone Has the Potential to Show Clinically Differentiated Improvement for MDAS Patients ~ 20,000 Global Prevalence Disease Mitochondrixaxl disexase xassocixated seizures (MDAS) is t(he (hig(hly morbid (condition of refra(ctory seizures in patients wit(h in(
Slide 23: MIT-E is a Global Registration-Directed Trial of Vatiquinone for MDAS
MIT - E is a Global Registration - Directed Trial of Vatiqqk DTLuinone for MDAS 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 ( vxatiquinone ) N = 60 Open - Label Extension Placebo - Controlled
Slide 24: Substantial Pipeline Progress Planned in 2023
JP Morgan Presentation 2023 24 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Substantial Pipeline Progress Planned in 2023
Slide 25: Vatiquinone Has the Potential to Provide Improvement in Neurological Function
JP Morgan Presentation 2023 25 Vatiqqk DTLuinone Has the Potential to Provide Improvement in Neurological Function ~ 25,000 Global Prevalence Disease Friedreich xatxaxxlnkaixa (FA) is a rare, in(herited, progressive disease resulting from mito(c(hondrial dysfun(ction Current Treatments No approved d
Slide 26: MOVE-FA is a Global Registration-Directed Trial of Vatiquinone for FA
MOVE - FA is a Global Registration - Directed Trial of Vatiqqk DTLuinone for FA Primary Endpoint Change in mFARS Key Secondary Endpoint Change in FA - ADL Open - Lxabexl Exxlnkatension Placebo - Controlled PTC743 ( vatiquinone ) Placebo N = ~ 60 72 weeks R N = ~ 60 24 weeks PTC743 ( vxatiquinone ) N
Slide 27: Substantial Pipeline Progress Planned in 2023
JP Morgan Presentation 2023 27 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Substantial Pipeline Progress Planned in 2023
Slide 28: PTC518 Reduces HTT mRNA and Protein to Target the Underlying Cause of HD
PTC518 Reduces HTT mRNA and Protein to Target the Underlying Cause of HD JP Morgan Presentation 2023 28 ~ 135,000 Global Prevalence Disease Huntington’s disexase (HD) is a progressive brain disorder t(hat (causes un(controlled movements and (cognitive loss Current Treatments No approved disease modi
Slide 29: PIVOT-HD is a Global Phase 2 Trial of PTC518 for HD
JP Morgan Presentation 2023 29 PIVOT - HD is a Global Phase 2 Trial of PTC518 for HD 3 mont(hs 9 mont(hs PTC518 5mg PTC518 10mg PTC518 20mg R Part 1 Part 2 Placebo PTC518 5mg PTC518 10mg PTC518 20mg Primary Endpoints • Safety and tolerability of PTC518 • Per(cent redu(ction in HTT mRNA and protein i
Slide 30: PIVOT-HD Expanding Trial Target Population
PIVOT - HD Expanding Trial Target Population JP Morgan Presentation 2023 30 • Ambulatory Huntington’s patients ages 25 and older • CAG repeats 40 - 50 inclusive • Motor and Cognitive Function: - UHDRS - IS score of 100 - UHDRS TFC score of 13 • PIN HD score 0.18 - 4.93 - Multivariate calculation inc
Slide 31: Transformational Commercial Revenue in 2023
Transformational Commercial Revenue in 2023 JP Morgan Presentation 2023 31 Commercixaxl $539 M 2021 Total revenue $116m Other $423m DMD Unaudited total revenue 2022 ~$710 M $ 203 m Other $ 507 m DMD Potential future revenues 2023 $940 M - $1bn $395 - $435m Other $545 - $565m DMD
Slide 32: Transformational Development Milestones in 2023
JP Morgan Presentation 2023 32 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Transformational Development Milestones in 2023
Slide 33: Transformational Development Milestones in 2023
JP Morgan Presentation 2023 33 Q1 2023 Q2 2023 Q3 2023 Q4 2023 DIPG BLA Subm:iss:ion Transformational Development Milestones in 2023
Slide 34: PKU Patient, Matthew Klein, M.D., COO – JP Morgan Healthcare Conference
PKU Patient Matthew Klein, M.D., COO JP Morgan Healthcare Conference January 9, 2023 PTC 2023
PTC Therapeutics — JP Morgan Healthcare Conference 2023 on 6ix