Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — 2019 JP Morgan Healthcare Conference
39 slides from Ptc Therapeutics's conference presentation, Jan 7, 2019
Slide 1: PTC 2019 JP Morgan Healthcare Conference
PTC 2019 JP Morgan Healthcare Conference Stuart Peltz, CEO
Slide 2: Forward looking statement
Forward looking statement All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements related to preliminary unaudited 2018 financial information with respect to 2018 net product revenue of Translarna for the treatment o
Slide 3: 2018: a transformational year
[Corporate Update] 2018: a transformational year $260-280M Expand Perform at least 1 revenue guidance TranslarnaTM Advance Enable SMA Advance 1 business for DMD availability to Oncology development internal research development franchise patients franchise success program to a D.C. deal Revenue 2-5
Slide 4: Started 2018 with a clear 3-year vision
Started 2018 with a clear 3-year vision PTC is a fully integrated, innovative rare disorder company leveraging research capabilities and core technology platforms, building out world- VISION class commercial capabilities, and being an ideal partner for late-stage, ultra-orphan disorders for which th
Slide 5: Building a diverse leading rare disorder biotech: Delivering on our vision
Building a diverse leading rare disorder biotech: Delivering on our vision Maximizing value with diverse portfolio Rare Disease Expansion Dual Product (DMD) Single Product (DMD) 5
Slide 6: Pipeline evolution: January 2017
[Corporate Update] Pipeline evolution: January 2017 *DMD Commercial Late develop. Aniridia Dravet / CDKL5 SMA Early develop. MPS1 BMI1 Preclinical HD Translarna™ NextGen Alternative Oncology (ataluren) Readthrough Splicing * MA requires annual renewal following reassessment by the European Medicines
Slide 7: Pipeline evolution: January 2018
[Corporate Update] Pipeline evolution: January 2018 DMD *DMD Commercial SMA Late develop. Aniridia Dravet / CDKL5 BMI1 Early develop. DHODH 1 Preclinical HD DHODH 2 Emflaza™ Translarna™ NextGen Alternative Oncology (deflazacort) (ataluren) Readthrough Splicing Key 2017 Additions * MA requires annual
Slide 8: Pipeline evolution: January 2019
[Corporate Update] Pipeline evolution: January 2019 DMD Commercial AADC Deficiency *DMD hATTR Amyloidosis Cardiometabolic SMA Aniridia FCS + FPL Late develop. Friedreich LGMD2i Ataxia PTC596 DIPG Dravet / CDKL5 PTC596 LMS PTC 299 AML Early develop. Angelman FD Preclinical Cognitive Disorder HD Gene
Slide 9: Delivering on our 3-year vision
Delivering on our 3-year vision PTC is a fully integrated, innovative rare disorder company leveraging research capabilities and core technology platforms, building out world- VISION class commercial capabilities, and being an ideal partner for late-stage, ultra-orphan disorders for which there is h
Slide 10: Looking forward: PTC growth vision for the next years
Looking forward: PTC growth vision for the next 5 years Now Future COMMERCIAL: 2 products (Translarna and (Translarna, Emflaza, Tegsedi, AADC, Emflaza) $262M* $~1.5B Risdiplam, FA) CLINICAL PROGRAMS: (AADC, SMA, Translarna, (AS, DIPG, AML, LMS, HD, FD, Emflaza, DIPG, AML) 6 10 +4) RESEARCH PROGRAMS:
Slide 11: ~$1.5B potential revenues to PTC by 2023
~$1.5B potential revenues to PTC by 2023 Risdiplam Emflaza (royalties to PTC) ~$300 ~$200 Tegsedi Translarna ex-US ~$150 ~$300 ~$400 ~$200 Emflaza Gene Therapy ~$90 Translarna US ~$170 Translarna ex-US ($ millions*) * Net of royalties 11 * Revenue based on PTC current assumptions and estimates
Slide 12: Building a Leading Rare Disorder Biotech: Global DMD Franchise
Building a Leading Rare Disorder Biotech Global DMD Franchise
Slide 13: PTC is the leader in DMD treatment of approved products
PTC is the leader in DMD treatment 2 of 3 approved products Translarna is the first-ever targeted therapeutic approved for DMD anywhere in the world (EMA, 2014) Translarna is now available in >40 countries worldwide ex-US and in trials for US potential approval in 2020 Emflaza is the first-and-only
Slide 14: Translarna: proven track record of performance
Translarna™: proven track record of performance • Unaudited 2018 net product revenue of $171M, an 18% increase over 2017 • Global sales outside of the U.S. • Pediatric expansion approved in 2018 • Label expansion for non-ambulatory patients under review • U.S. dystrophin study underway, completion Y
Slide 15: Emflaza: Establishing standard of care for all DMD patients in the US
Emflaza®: Establishing standard of care for all DMD patients in the US • 2018 Emflaza net product revenue of $91M (unaudited) • Revenue increase of >$60M over 2017 • Data from multiple publications demonstrate Emflaza’s clinical benefit over prednisone 15
Slide 16: Continuing to drive long-term growth of DMD franchise
Continuing to drive long-term growth of DMD franchise Label expansion under review for Translarna™ in non-ambulatory patients by the EMA sNDA for Emflaza® 2-5 year old U.S. patients submitted with potential approval in ‘19 16
Slide 17: An efficient, scalable business engine
An efficient, scalable business engine • 2018 unaudited product net revenue of $262M South Plainfield, Zug Switzerland, Marketing, • 2019 DMD franchise revenue New Jersey Medical and Regulatory Hub guidance of $285 - $305M • Established footprint in >40 countries worldwide • Experienced commercial a
Slide 18: Building a Leading Rare Disorder Biotech: Leveraging our Global Commercial Franchise
Building a Leading Rare Disorder Biotech Leveraging our Global Commercial Franchise
Slide 19: Preparing for successful launch
Preparing for successful launch All key hiring completed in Latam Tegsedi best fit for Latin American hATTR market Regulatory dossier filed hATTR polyneuropathy most prevalent Diversifies our rare with ANVISA and rare- phenotype in Latin America ~6,000 patients disease portfolio and revenues disease
Slide 20: Two potential assets in Latin America
Two potential assets in Latin America Tegsedi best fit for Waylivra: could utilize our Latin American hATTR market patient support in Latin America hATTR polyneuropathy most prevalent Diversifies our rare Similar economic opportunity to phenotype in Latin America Translarna in Latin America ~6,000 p
Slide 21: Building a Leading Rare Disorder Biotech: Leveraging our R&D platforms - Splicing platform
Building a Leading Rare Disorder Biotech Leveraging our R&D platforms to continue to grow our pipeline I. Splicing platform
Slide 22: Leaders in small molecule RNA-splicing technology
Leaders in small molecule RNA-splicing technology Development of 13 years of Cutting-edge 2nd Splicing Continue to SMA candidate discovering and tech platform Compound: A exploit Splicing as potential developing discovered and Development platform; ad- best-in-class drugs that target developed by Ca
Slide 23: Risdiplam in development for Spinal Muscular Atrophy (SMA)
Risdiplam in development for Spinal Muscular Atrophy (SMA) • Primary genetic cause of infant mortality • Small molecule promotes the correct splicing of the mutant RNA • Small molecule has potential for best in class therapy • Broad tissue distribution and protein restoration Risdiplam targets-splic
Slide 24: Risdiplam has potential to be a $2B product
Risdiplam has potential to be a $2B product • Revenue > $1B subject to mid-teens* Based on FDA feedback: royalty to PTC from Roche • Potential to PTC to exceed $200M/ Data from Sunfish year; including competitive assumptions for SMA gene therapy & Firefish part 1 should be • Firefish & Sunfish fully
Slide 25: The splicing technology is a proven platform to identify new therapeutics
The splicing technology is a proven platform to identify new therapeutics Development of 13 years of Cutting-edge 2nd splicing Continue to SMA candidate discovering and tech platform compound: A exploit splicing as potential developing discovered and development platform; best-in-class drugs that ta
Slide 26: Familial dysautonomia
Familial dysautonomia: • Genetic disorder primarily affecting the sensory and autonomic neurons • Caused by a splicing-altering mutation in the IKBKAP (ELP1) gene resulting in low levels of IKAP protein • Ashkenazi Jewish ancestry, carrier frequency is ~1:30 • No therapies are currently available fo
Slide 27: The splicing mutation in IKBKAP gene leads to IKAP protein deficiency resulting in FD
The splicing mutation in IKBKAP gene leads to IKAP protein deficiency resulting in FD c CAA gtaagt 27
Slide 28: PTC-258 splicing modifiers restore IKAP levels
PTC-258 splicing modifiers restore IKAP levels c CAA gtaagt Development candidate PTC-258 selected YE:18 Scheduled to enter the clinic in 2019 28 J a n - 19
Slide 29: Building a Leading Rare Disorder Biotech: A CNS gene therapy platform
Building a Leading Rare Disorder Biotech Leveraging our R&D platforms to continue to grow our pipeline II. A CNS gene therapy platform
Slide 30: Gene therapy development strategy
Gene therapy development strategy Execute on current Priority to secure in- Expand the programs house manufacturing pipeline with capabilities to support internal research Target dates: long-term capacity and external • AADC Launch 2020 collaboration • Friedreich Launch 2023 • Angelman IND 2020 • Re
Slide 31: AADC deficiency is a devastating disease with high unmet need
AADC deficiency is a devastating disease with high unmet need • Rare progressive childhood disease, affecting approximately 5,000 patients globally • Children with severe AADC deficiency never achieve motor development milestones Head Position Sitting Standing 3-4 months 6-9 months 10-12 months • Pr
Slide 32: Patient identification is our expertise
Patient identification is our expertise ? Earlier Increase disease diagnosis awareness ~100 AADC patients already identifiedStrategy into thebring US andEmflaza Europe® to naive • Genotypingand patients younger in seizure U.S. and cerebral palsypatients clinics Genotyping Improving • Plan to screen
Slide 33: Most advanced FA gene therapy program
Most advanced FA gene therapy program PTC plans to file IND in 2019 Targeted Micro Favorable Animal data Patient group dosing / direct to immunogenic supports engagement CNS profile appropriate dose 33
Slide 34: Moving toward IND filing in 2019
Moving toward IND filing in 2019 PTC-FA Intracerebellar Dosing in Porcine PTC-FA Intracerebellar Dosing in NHP Model* Model* 1000.0 Cerebellar 3000.0 Cerebellar Cortex Cortex 2500.0 800.0 Dentate Dentate Nucleus Nucleus 2000.0 600.0 1500.0 400.0 Target Frataxin Protein 1000.0 Healthy Human DN Target
Slide 35: 2019 goals: file an AADC BLA & FA IND
2019 goals: file an AADC BLA & FA IND IND IND Friedreich Angelman Ataxia Syndrome 2019 2020 FDA BLA + EMA IND MAA submissions Cognitive disorder / Reelin AADC Deficiency Immediate clinical manufacturing capabilities as well as the plan to expand to commercial scale 35
Slide 36: Potential addressable market in excess of $5B
Potential addressable market in excess of $5B AADC, FA, & AS: AADC: Aromatic L-amino acid decarboxylase FA: Friedreich ataxia ~100,000 patients AS: Angelman syndrome AADC & FA: ~30,000 patients Global Prevalence Global AADC deficiency: ~5,000 patients Launch Sequence 36
Slide 37: Niche oncology strategy prioritizes value creation
Niche oncology strategy prioritizes value creation Use of current platforms to add new targets Internal research to portfolio with focus on splicing PTC596 in pediatric brain tumor (DIPG) phase Solid tumors 1/2 trial and Leiomyosarcoma (LMS) Hematologic malignancies PTC299 AML dose escalation trial
Slide 38: Sustainable growth expected over next years
Sustainable growth expected over next 5 years Potential revenues to PTC from DMD franchise, Gene therapy programs, Tegsedi and Risdiplam 2023 2022 2021 Potential Emflaza Limb-Girdle 2i launch 2020 Potential Tegsedi, SMA, AADC and Translarna US launches 2019 Expected SMA milestones for regulatory fil
Slide 39: Everyone has a different definition of progress
Everyone has a different definition of progress. For the last 20 years, we’ve measured our progress researching rare disease in moments. Smiling ones and crying ones. Moments spent with our boys’ families and ones with their friends. We know that every step forward comes after several steps backward
PTC Therapeutics — 2019 JP Morgan Healthcare Conference on 6ix