Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — J.P. Morgan Healthcare Conference 2025
31 slides from Ptc Therapeutics's conference presentation, Jan 13, 2025
Slide 1: PTC Therapeutics – JP Morgan 2025
1 PTC Therapeutics – JP Morgan 2025 Matthew B. Klein, MD CEO PTC Therapeutics 2025 Pam and Kelsey living with PKU
Slide 2: PTC Therapeutics – JP Morgan 2025
PTC Therapeutics – JP Morgan 2025
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Slide 4: Forward-looking statements
PTC Therapeutics – JP Morgan 2025 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 stateme
Slide 5: Execution in 2024 Provides Foundation for Success
PTC Therapeutics – JP Morgan 2025 Execution in 2024 Provides Foundation for Success 5 Advanced Programs from Innovative Scientific Platforms Strong Cash Position Outstanding Revenue Performance All Development Milestones Achieved
Slide 6: Four U.S. Regulatory Approval Applications Submitted in 2024
PTC Therapeutics – JP Morgan 2025 Four U.S. Regulatory Approval Applications Submitted in 2024 6 AADC Gene Therapy BLA Approved Sepiapterin PKU NDA Accepted Translarna nmDMD NDA Accepted Vatiquinone Friedreich’s Ataxia NDA Submitted
Slide 7: Outstanding 2024 Revenue Performance Driven by Inline Products
PTC Therapeutics – JP Morgan 2025 Outstanding 2024 Revenue Performance Driven by Inline Products Unaudited Total Revenue ~$814M 2024 7
Slide 8: Strong Cash Position Enables Future Revenue Growth and R&D Innovation
PTC Therapeutics – JP Morgan 2025 Strong Cash Position Enables Future Revenue Growth and R&D Innovation 8 Reach Cashflow Breakeven Without Additional Capital Support Commercial Launches and Innovative R&D Programs Fund BD Activities to Complement Product Portfolio
Slide 9: Research Platforms Provide Continuous Source of Innovative Therapies
PTC Therapeutics – JP Morgan 2025 Research Platforms Provide Continuous Source of Innovative Therapies 9 Splicing Inflammation & Ferroptosis
Slide 10: Numerous Potential Value-Creating Milestones Expected in 2025
PTC Therapeutics – JP Morgan 2025 Translarna DMD FDA Approval Decision* Numerous Potential Value-Creating Milestones Expected in 2025 *No action date provided 10 **If priority review granted Vatiquinone FA FDA Approval Decision** Sepiapterin PKU CHMP Opinion Q1 Q2 Q3 Q4 Vatiquinone FA NDA Acceptance
Slide 11: 2025 Revenue and OPEX Guidance
PTC Therapeutics – JP Morgan 2025 2025 Revenue and OPEX Guidance *Includes in-line products, potential new product launches and royalty revenue from Evrysdi. **Non-GAAP measure which excludes estimated non-cash, stock-based compensation expense of approximately $75 million. GAAP R&D and SG&A expense
Slide 12: Sepiapterin PKU Program
12 PTC Therapeutics – JP Morgan 2025 Sepiapterin PKU Program Owen living with PKU
Slide 13: Sepiapterin Demonstrates Potential to Meet Unmet Need for Majority of PKU Patients
PTC Therapeutics – JP Morgan 2025 Sepiapterin Demonstrates Potential to Meet Unmet Need for Majority of PKU Patients 13 Evidence of diet liberalization with ~50% of patients exceeding Recommended Daily Allowance for protein Efficacy across all key subgroups in Phase 3 APHENITY trial including patien
Slide 14: Physician and Patient Statements Reflect Enthusiasm for Sepiapterin
PTC Therapeutics – JP Morgan 2025 Physician and Patient Statements Reflect Enthusiasm for Sepiapterin “I'm excited about the opportunity to offer sepiapterin to all my patients ” – Ania C. Muntau, MD, Professor of Pediatrics, Chair, University Children's Hospital, University Medical Center, Hamburg
Slide 15: APHENITY Results Support Potential to Address All Key PKU Population Segments
PTC Therapeutics – JP Morgan 2025 APHENITY Results Support Potential to Address All Key PKU Population Segments 15 Therapy-Naive Patients Including Classical PKU Patients Who Are Not Well Controlled by Current Therapies Patients Who Have Failed on Current Therapies Greater than $1 Billion Potential
Slide 16: Experienced U.S. Team Preparing for Planned 2025 Launch
PTC Therapeutics – JP Morgan 2025 Experienced U.S. Team Preparing for Planned 2025 Launch 16 HCPs engaged through multifaceted approach Centers of Excellence & prescribers mapped Payer & access discussions initiated Patient & community partnerships established
Slide 17: Vatiquinone FA Program
17 PTC Therapeutics – JP Morgan 2025 Vatiquinone FA Program Olivia living with FA
Slide 18: Vatiquinone Has Potential to be First Approved Treatment for Children with FA
PTC Therapeutics – JP Morgan 2025 Vatiquinone Has Potential to be First Approved Treatment for Children with Friedreich’s Ataxia Vatiquinone data support safety and efficacy for children and adults with FA NDA based on findings of efficacy from MOVE-FA placebo-controlled study and two long-term stud
Slide 19: Vatiquinone Demonstrated Meaningful Slowing of Disease Progression
PTC Therapeutics – JP Morgan 2025 Vatiquinone Demonstrated Meaningful Slowing of Short- and Long-term Disease Progression 19 Vatiquinone Placebo Mean Change from Baseline in mFARS Upright Stability score (+/- SE) ee 42% slowing p=0.021 4.8-point benefit (p<0.0001) on mFARS** over 2 years in ambulato
Slide 20: Vatiquinone Profile Supports Significant Commercial Opportunity
PTC Therapeutics – JP Morgan 2025 ~6,000 U.S. patients, ~1/3 are pediatric; global prevalence of ~25,000 Vatiquinone Profile Supports Significant Commercial Opportunity Including All Friedreich’s Ataxia Patients 20 Efficacy and safety data support opportunity for patients of all ages Strong 10+ year
Slide 21: PTC518 HD Program
21 PTC Therapeutics – JP Morgan 2025 PTC518 HD Program April living with HD
Slide 22: Key Attributes of PTC518 Drive Differentiation
PTC Therapeutics – JP Morgan 2025 Key Attributes of PTC518 Drive Differentiation Orally bioavailable Uniform mHTT lowering in all regions of the brain Highly selective and specific for HTT target Achieves excellent and broad CNS exposure Safe and well tolerated in clinical trials 22 Reduces HTT prot
Slide 23: 12-Month Interim Readout Met All Key Safety and Efficacy Objectives
PTC Therapeutics – JP Morgan 2025 Month 12 Interim Readout Met All Key Safety and Efficacy Objectives (June 2024) Dose-dependent and durable lowering of mHTT protein in blood PTC518 was well tolerated with no treatment-related NfL spikes Dose-dependent lowering of CSF mHTT protein levels Dose-depend
Slide 24: Planned Data Update in Q2 2025
PTC Therapeutics – JP Morgan 2025 Planned Data Update in Q2 2025 to Include 12-month Results for Additional ~100 Subjects Safety and tolerability of PTC518 Percent reduction in blood mHTT protein Percent reduction in CSF mHTT protein Changes in clinical scores (cUHDRS, TMS, TFC) Readout will include
Slide 25: Development and Commercialization Collaboration with Novartis
PTC Therapeutics – JP Morgan 2025 Development and Commercialization Collaboration Expected to close in Q1 2025, subject to customary closing conditions, including regulatory clearance. Refer to press release dated December 2, 2024 for additional details. Novartis to fund development activities follo
Slide 26: Validated Splicing Platform Provides Source of Innovative and Valuable Therapies
PTC Therapeutics – JP Morgan 2025 Validated Splicing Platform Provides Source of Innovative and Valuable Therapies PTC has pioneered discovery and development of oral splicing therapies Additional early-stage programs targeting non-CNS indications Platform innovations such as PTSeek accelerate disco
Slide 27: Inflammation & Ferroptosis Programs Targeting CNS and Non-CNS Disorders
PTC Therapeutics – JP Morgan 2025 Inflammation & Ferroptosis Programs Targeting CNS and Non-CNS Disorders Focused on novel targets key to inflammation and oxidative stress Active programs targeting CNS and non-CNS disorders Phase 2 ready DHODH inhibitor program for neuroinflammation indications NLRP
Slide 28: PTC Vision for Successful 2025 and Beyond
PTC Therapeutics – JP Morgan 2025 PTC Vision for Successful 2025 and Beyond Path to $2 Billion Topline Revenue Highly Differentiated and Innovative R&D Platforms Business Development to Accelerate Revenue Growth Cash Flow Break Even 28
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PTC Therapeutics – JP Morgan 2025
PTC Therapeutics — J.P. Morgan Healthcare Conference 2025 on 6ix