Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — William Blair Growth Stock Conference 2022
31 slides from Ptc Therapeutics's conference presentation, Jun 9, 2022
Slide 1: PTC Therapeutics William Blair Growth Stock Conference
PTC Therapeutics William Blair Growth Stock Conference Matthew Klein, M.D. Chief Operating Officer Kylie O’Keefe, SVP Global Commercial and Corporate Strategy June 9, 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 statemen
Slide 3: PTC Has a Growing Global Footprint
PTC Has a Growing Global Footprint 3 1,200+ employees 20 offices worldwide Footprint in >50 countries Founded in 1998
Slide 4: Diversified Platform Drives Strong Portfolio
Diversified Platform Drives Strong Portfolio 4 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 5: Strong Financial Performance Driven by Innovation
Strong Financial Performance Driven by Innovation 5 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 6: Strong DMD Franchise Growth Expected to Continue
192 236 47 79 139 187 44 49 2020 2021 1Q21 1Q22 DMD Franchise Revenue Translarna Emflaza • First and only corticosteroid approved for DMD; approved for all US DMD patients >2yrs • Data show clinical benefit over prednisone • New patients starts, continued high compliance and operational excellence •
Slide 7: Evrysdi Revenue Demonstrates Benefits of Safe and Effective Therapy for SMA
Evrysdi Revenue Demonstrates Benefits of Safe and Effective Therapy for SMA 7 • First at - home, oral treatment for SMA • Patients treated across all SMA types • Patients are treatment - naïve or previously treated with Spinraza , Zolgensma • Broadest range of ages treated Benefits of Evrysdi for SM
Slide 8: Leveraging Regulatory Expertise and Commercial Infrastructure in Latin America
• Innovative treatment for hATTR amyloidosis patients • Disease awareness and patient ID continuing • LATAM patients benefiting through early - access programs • Received Category 1 pricing, in final pricing negotiations • For treatment of familial chylomicronaemia syndrome (FCS) • LATAM patients be
Slide 9: Upstaza 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 No approved disease - modifying treatments Mechanism of Action Potential for AA
Slide 10: Upstaza-Treated Patients Make Significant and Sustainable Progress
Upstaza ™ - Treated Patients Make Significant and Sustainable Progress 10
Slide 11: Upstaza Gene Therapy Opportunity and Launch Preparation
Upstaza ™ Gene Therapy Opportunity and Launch Preparation 11 Potential over $1B in cumulative revenue Successful patient finding is ongoing Positive CHMP opinion received in May, ratification expected in July Upstaza ™ BLA submission expected in 4Q22 Development of virtual education: disease - speci
Slide 12: Pipeline Progress Planned in 2022
Pipeline Progress Planned in 2022 Study Results Study Initiations GT - FA Ph1 Initiation DIPG Ph2 Initiation Study 041 2022 Q4 2022 Q2 2022 Q3 2022 Q1
Slide 13: 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 14: 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 15: Vatiquinone MDAS Trial Design
Vatiquinone Has the Potential to Show Clinically Differentiated Improvement for MDAS Patients 15 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 16: 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 17: Vatiquinone FA Trial Design
Vatiquinone Has the Potential to Provide Improvement in Neurological Function 17 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 18: PTC857 Has the Potential to Slow Disease Progression in ALS
18 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 o
Slide 19: PTC857 ALS Trial Design
Primary Endpoints Change in ALSFRS - R Secondary Endpoints Safety and PK 19 Treatment Period PTC857 250 mg BID Placebo 24 weeks Primary Endpoint R Long - Term Treatment Extension 28 weeks PTC857 250 mg BID Trial Status • Enrolling • Trial initiated in Q1 2022 Screening 8 weeks PTC857 Has the Potenti
Slide 20: 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 21: PTC518 Reduces HTT mRNA and Protein to Target HD
21 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 22: 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 22 Results from Phase 1 Healthy Volunteer Study PTC518 Reduces HTT mRNA and Protein to Target the Proximal Cause of HD
Slide 23: PTC518 Has the Potential to Reduce HTT Protein in Huntington Patients
23 PTC518 Has the Potential to Reduce HTT Protein in Huntington Patients Trial Status • Enrolling • Trial initiated in Q1 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 Huntingt
Slide 24: PTC518 Endpoints and Inclusion Criteria
24 • 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 25: APHENITY Is a Global Registration-Directed Trial of PTC923 for PKU
25 Disease Phenylketonuria (PKU) is a metabolic condition caused by mutations to phenylalanine hydroxylase, which 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
Slide 26: APHENITY Trial Design
APHENITY I s a Global Registration - Directed Trial of PTC923 for PKU 26 Primary Endpoint Reduction in blood phenylalanine levels Primary Endpoint Trial Status • Enrolling • Data expected YE 2022 R PTC923 Placebo 42 Days Open - Label Extension Placebo - Controlled PTC923 12 Months PTC923 14 Days Run
Slide 27: Unesbulin Has the Opportunity to Provide Additional Progression Free Survival in LMS
27 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 28: Phase 1b Study Results
28 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 29: Unesbulin Phase 2/3 Trial Design in LMS
Unesbulin has 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 N ≈ 11
Slide 30: Multiple Platforms Provide Opportunity to Target Over 700,000 Patients by 2030
Multiple Platforms Provide Opportunity to Target Over 700,000 Patients by 2030 30 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 31: Enduring Innovation Drives Value Creation
31 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 ® , PTC
PTC Therapeutics — William Blair Growth Stock Conference 2022 on 6ix