Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — Conference Presentation (January 2018)
28 slides from Ptc Therapeutics's conference presentation, Jan 10, 2018
Slide 1: PTC Therapeutics: 20 years of commitment to bringing new treatments to patients with rare disorders
N PTC Therapeutics: 20 years of commitment to bringing new treatments to patients with rare disorders JANUARY 2018
Slide 2: Forward looking statements
Forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 A statment, ce than those of itr act, contained in Ws prosenation, are fwardocking preliminary 201 ot 2018; PTC hor iecton wih the FOA ragardng th Taran NDA, te ‘etint population gio fxEmaza voatnen
Slide 3: Our vision
Our vision To bring best in-class To leverage our global therapies with clinical commercial infrastructure benefit to patients to maximize value for affected by rare patients and all disorders stakeholders
Slide 4: Sustainable, growing DMD business enables continued innovation & growth
Sustainable, growing DMD business enables continued innovation & growth 1) Growing global DI iranchise with unaudited 2017 revenue of $174 i Translarna™ strong continued patient ‘s 0 Ilion) 2017 Unaudited Revenue 2018 Financial Guidance uptake reflected in annual growt! : Translarna $145, $170 - $18
Slide 5: Expanding pipeline through in-house innovation
Expanding pipeline through in-house innovation Commercial e oma @ pv Phase 3 @ su phase 2 @& priricia ase { } Dravet / CDKLS Phase 1 @ BMIi H ; Preclinical © vopH1 Discovery e © px00H2 Emflaza Trai rn NextGen Al ative (deflazacort) (ataluren) Readthrough Splicing a oan ony EMA) Gtote in UA a coming
Slide 6: Translarna & Emflaza: transforming standard of care for DMD patients
rna™ & Emfi Trans tandard of c: insforming for DMD patients PTC
Slide 7: DMD progression is sequential, non-linear and irreversible
‘ogression is sequential, non-linear and irreversible Age 15 Age 20 Age 25 Age 30 2 > Impaired ability to hop, run, jump, rise 1 Loss of stair climb Loss of stair ~—) physical manifestations Vv GE 6055 0/ ar tive of. Loss of ambulation LATE ——= physical oss of upper limb function manifestations ' ‘a
Slide 8: Translarna: Addressing the underlying cause of nonsense mutation DMD
Translarna™: Addressing the underlying cause of nonsense mutation Duchenne Muscular Dystrophy Birth e t 1 1 I 1 1 1:4,000 males DMD ~10-15% nonsense 1 1 1 L i 1 1 1 ' Age 2 Disease awareness, genetic testing and early treatment are ritical e 1 1 1 1 i 1 1 , i 1 1 | 1 i i i L i Translarna delays Dela
Slide 9: Establishing Emflaza as part of standard of care for U.S. patients
Establishing Emflaza™ : Establishing as part of standard of care for U.S patients Birth e (deflazacort) | i 1 1:4,000 males DMD i 1 1 1 1 L i 1 1 1 1 i Initiating treatment at | Emflaza delays loss Continued use beyond diagnosis i of ambulation by 4 loss of ambulation \ years \ i preserves pulmonary
Slide 10: Translarna: Proven track record of successful global sales
Translarna™: Proven track record of successful global sales Unaudited 2017 net revenue of $145 M Guidance for 2018 of $170 - $185 M Global sales outside of the U.S. since 2014 Pediatric expansion under EMA review ‘Teanslarna: HSER U.S. appeal process underway, expect update in Q1 eS
Slide 11: Emflaza: Establishing standard of care for all DMD patients in the U.S.
Emflaza™: Establis! of care for all D in the U.S. ., . Articles | Emflaza (deflazacort) ; jon qualtyot @%® © 2017 Emflaza unaudited net sales of $29 M life, and survival in patients with Duchenne muscular ¢ Guidance for 2018 of $90 - $110 M e Results from Lancet publication reinforce Emflaza efficac
Slide 12: Growing global DMD franchise
Growing global DMD franchise Translarna™ & Emflaza™ DMD Net Sales ($ in millions) $320 $280 4 a e 240 05 Geimaal $200 $160 $29 #120 e5) transtarna 2014 2015 2016 2017* 2018E + Unaudited
Slide 13: Improving patient outcome by delivering best in class therapy earlier
Improving patient outcome by delivering best in class therapy earlier to preserve function i Q Earlier diagnosis Increase disease awareness Genotyping Improving standards of care Pursuing label expansions to bring Emflaza™ & Translarna™ to younger patients to ultimately improve patient outcome &
Slide 14: An efficient, scalable commercialization engine
An efficient, scalable commercialization engine 2017 unaudited product net sales of $174 M Established footprint in 47 countries worldwide Experienced commercial team in orphan disease Fully integrated global commercial infrastructure af ee South Plainfield, Zug Switzerland, Dublin, Ireland Latam Re
Slide 15: Expanding pipeline through in-house innovation
Expanding pipeline through in-house innovation 1 I Commercial DMD *DMD f ew @ i Phase 3 i e SMA‘ ! Phase 2 Aniridia ase { } Dravet / CDKLS Phase 1 @ oun: 1 Preclinical © vopH1 Discovery e © px00H2 Emflaza™ Translarna’ NextGen Alternative (deflazacort) (ataluren) Readthrough Splicing : ox ose haere P
Slide 16: PTC
PTC
Slide 17: The SMA program validates PTC's small molecule splicing platform
The spinal muscular atrophy program validates PTC’s small molecule splicing platform Splicing Target splicing event to restore or reduce protein ‘SMA - SMN2 FD -IKBKAP HD-HTT Sinan modifiers to ton sed gery a mk ‘ith pina muscular atopy § Beefing up the right splice variant to treat spinal muscular
Slide 18: Spinal Muscular Atrophy: The leading genetic cause of infant death
Spinal Muscular Atrophy: The leading genetic cause of => Dorsal Root > Ventral Root Spinal muscular atrophy (SMA) is caused by the loss of SMN1 gene Low expression of SMN protein leads to the loss of motor neurons in the spinal cord One in every 10,000 children born is affected with the disorder of
Slide 19: RG7916 has the opportunity to be best in class
RG7916 has the opportunity to be best in class of SMA patients SMN protein level °D comple’ SMN RNA in SMA patients 200004 a i e RG7916 has shown 2.5 fold median > 150004 increase in protein in patients S = 100004 e Small molecule has potential for 2 L broad tissue distribution key to SMA = 50004 tr
Slide 20: Advancing towards registration of RG7916
Advancing towards registration of RG SUNFISH * Clinical study in SMA type 2/3 patients between 2 and 25 years old = Ongoing pivotal part will enroll 168 patients, placebo controlled 2:1, endpoint of total motor function measure (MFM-32) at 12 months FIREFISH © Clinical study in SMA type 1 patients b
Slide 21: Huntington's Disease is caused by a CAG repeat expansion in the HTT gene
Huntington’s Disease is caused the HTT gene a CAG repeat expansion in Gene with ‘mutated Healthy neuron. vamos ving rte at gy ¢ HD is caused by expression of mutant Huntingtin (HTT) protein due to the CAG repeat expansion e Neuron degeneration ___------- predominantly in the striatum and cerebral co
Slide 22: Treating HD by modifying HTT splicing
Treating HD by modifying HTT splicing CAG), Vv %° CAG), ( vy, 35 y Exon 1 {atmiy Exon X Exon Y Mutant toxic HTT protein
Slide 23: Opportunity to address HTT by leveraging PTC's splicing platform
Opportunity to address HTT by leveraging PTC’s splicing platform (CAS)-95 (CAG). 35 J | + Cpd_ Inclusion of alternative exon > E=--- Exon X Exon Y > aS) 2) Translation-linked mRNA decay
Slide 24: Lowering HTT remaining
e HTT remaining Venicte PTC compounds low i n a mou PTC compound ver human HTT in key regions of use model of HD HTT Remaining Venicle PTC compound % HTT remaining Vehicle PTC compound PTC
Slide 25: PTC's platform technologies target RNA biology
PTC’s platform technologies target RNA biology to modulate gene expression with small molecules Platform Mechanism Targeted Programs i ‘SMA - SMN2 Target splicing event to restore FD -IKBKAP or reduce protein HD -HTT Us 20 years of targeting RNA biology for drug discovery and development Splicing &
Slide 26: Developing a therapeutic pipeline
veloping a 1 th i PTC
Slide 27: PTC's future events and milestones
PTC’s future events and milestones SMA RG7916 data at EU SMA U.S. FDA Translarna™ decision Analyst Day highlighting pipeline Establish Emflaza™ as U.S. DMD standard of care 15% CAGR Translarna revenue through 2022
Slide 28: Sustainable, growing DMD business enables continued innovation & growth
Sustainable, growing DMD business enables continued innovation & growth led 2017 revenue of $174 2017 Unaudited Revenue —_2018 Financial Guidance Translamna $145 $170 - $185 Emflaza $29 $90 - $110 DMD Franchise $174 $260 - $295 2) Strong sustainable financial po: in 2018 GAAP R&D + SG&A $280-$290M 2
PTC Therapeutics — Conference Presentation (January 2018) on 6ix