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1 PTC Therapeutics, Inc. May 2014
2 Forward-looking statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of All statements, other than those of historical fact, contained in this presentation, including statements regarding the future expectations, plans and prospects for PTC, our Phase 3 clinical trials for ataluren in nmdmd and nmcf, our collaboration in SMA with Roche and the SMA Foundation, our current and planned regulatory filings with the EMA, the timing of the re-examination of the CHMP opinion, our earlier stage programs, including our initiation of a potential proof-of-concept study, our strategy, future operations, future financial position, future revenues or projected costs, the development of and potential market for PTC s product candidates, and objectives of management, are forward-looking statements. Other forward-looking statements may be identified by the words plan, guidance, anticipate, believe, estimate, expect, intend, may, predict, project, target, potential, will, would, could, should, continue, and similar expressions. Our actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements we make as a result of a variety of risks and uncertainties, including those related to the initiation and conduct of clinical trials, availability of data from clinical trials, expectations for regulatory approvals, our scientific approach and general development progress, the availability or commercial potential of our product candidates and the factors discussed in the Risk Factors section of our Quarterly Report on Form 10-Q for the quarter ended March 31, 2014, which is on file with the Securities and Exchange Commission. You are urged to carefully consider all such factors. The forward-looking statements contained herein represent PTC s views only as of the date of this presentation, and we do not undertake or plan to update or revise any such forwardlooking statements. May
3 Our mission To leverage our knowledge of RNA biology to bring novel therapeutics to patients affected by rare disorders May
4 Executing on PTC s strategy Focus on the late-stage development and commercialization of ataluren in nmdmd, nmcf and other indications Expand PTC s clinical stage pipeline using our scientific expertise to develop novel compounds for unmet medical needs Foster relationships with strong strategic partners to further leverage PTC's discovery and development capabilities May
5 PTC s RNA-focused small molecule technology platform DNA pre-mrna mrna Protein exon intron 5 cap ribosome poly(a) tail GLU VAL LYS trna PTC s Platform Technologies Nonsense Readthrough Alternative Splicing Nucleotide Repeat Protein Modification Transcript Regulators May
6 Pipeline summary Product / Platform Discovery Preclinical Phase 1 Phase 2 Phase 3 Status/Plans Ataluren nonsense readthrough Orphan genetic disorders nmdmd nmcf Other indications Enrollment ongoing Data expected mid-2015 Start P3 enrollment H Announce in 2014 SMA ( ) alternative splicing DMD Exon 51 alternative splicing SMA program DMD Entered P1 Jan 2014 Lead discovery PTC596 stem cell targeting PTC compound DNA synthesis Oncology BMI1 Antibacterial Gonorrhea IND-enabling studies Lead optimization May
7 Ataluren Nonsense mutation readthrough May
8 Ataluren binds to the ribosome and enables readthrough of nonsense mutation to produce functional protein Potentially treats any nonsense mutation across multiple orphan disorders High specificity for nonsense readthrough without affecting normal termination codons Nonsense mutations are routinely identified by genetic testing May
9 Ataluren: Late-stage asset with disease-modifying potential in multiple orphan disorders Characteristics Orally bioavailable Reliable manufacturing Pediatric-friendly formulation Generally well tolerated ~600 individuals dosed to date Market Potential ~11% of patients across all monogenic disorders have a nonsense mutation Potential disease modifying therapy Orphan designation EU & US PTC retains worldwide commercial rights Status Confirmatory Phase 3 trial in nmdmd ongoing Phase 3 nmcf trial initiation in H Additional indications in 2014 Patents until (composition & method of use) N O F N CO 2 H May
10 Ataluren is active in numerous indications Muscle disorders Duchenne muscular dystrophy (Welch et al. 2007, Kayali 2012, Arner 2013) Miyoshi Myopathy (Wang 2010) Ion channel disorders Cystic fibrosis (CF) (Du 2008, Kerem 2008, Sermet-Gaudelus 2010, Wilschanski 2011, Gonzalez-Hilarion 2012) Long QT syndrome (Yu 2014) Neurological disorders Infantile neuronal ceroid lipofuscinoses (INCL) (Sarkar 2011, Miller 2013) Late infantile ceroid lipofuscinoses (LINCL) (Miller 2013) Ataxia telangiectasia (Du 2013) Usher syndrome (USCH1C) (Goldmann 2011, Goldmann 2012) Skin disorders Pseudoxanthoma elasticum (Zhou 2013) Eye disorders Choroideremia (Moosajee unpublished) Aniridia (Gregory-Evans 2014) Pulmonary disorders Heritable pulmonary arterial hypertension (HPAH) (Drake 2013) Metabolic disorders Carnitine palmitoyltransferase 1A deficiency (Tan 2011) Methylmalonic aciduria (MMA) (Buck 2012) Propionic acidemia (PA) (Sanchez-Alcudia 2012) Maroteaux-Lamy syndrome (MPS VI) (Bartolomeo 2013) Hurler s syndrome (MPS I) (Keeling et al. unpublished) Reporters GFP and Luciferase (Welch et al. 2007, Lentini 2014) Xeroderma pigmentosum (Kuschal 2013) (independent investigators) May
11 Ataluren s activity in a Hurler nonsense mutation model 6 Brain Spleen Tissue GAG (ug GAG / mg tissue) WT ** Idua-W392X Tissue GAG (ug GAG / mg tissue) WT ** Idua-W392X untreated treated **p<0.01 (t-test, compared to Idua-W392X vehicle) Ataluren treatment reduces GAG levels in multiple tissues in a Hurler nonsense mutation mouse model Neufeld and Muenzer, The Mucopolysaccharidoses, in The Metabolic and Molecular Bases of Inherited May 2014 Disease, 2001 Keeling et al unpublished data 10
12 Ataluren s activity in an aniridia nonsense mutation model Ataluren increases PAX6 in Pax6 Sey mice 120 * Pax6 protein (% of wildtype) * WT = wild type Neu = splice-site mutation Sey = nonsense mutation Treated with ataluren 0 Wt Sey Sey Neu Neu Wt Sey Sey Neu Neu * p<0.001; n=6. Retina Corneal Epithelium Ataluren increases PAX6 protein in retina and cornea in the nonsense mutant (Sey), not in the splice-site mutant (Neu) May 2014 Gregory-Evans 2014 JCI 11
13 Ataluren treatment restores morphology and sight Wild type untreated Mutant + vehicle Mutant + ataluren P60 WT Mt Mt + ataluren Gregory-Evans 2014 JCI May
14 Efficacy of ataluren in nonsense mutation choroideremia X-linked recessive chorioretinal disease resulting in blindness in patients Caused by mutations in the REP1 gene Nonsense mutations account for over 33% of patients Wild type Mutant + vehicle Mutant + ataluren May 2014 Moosajee et al. unpublished data 13
15 Duchenne muscular dystrophy (DMD) is a progressive and fatal genetic disorder due to the loss of dystrophin protein Extracellular Matrix Muscles lacking dystrophin are more susceptible to muscle damage Dystrophin replacement is expected to prevent muscle damage Dystroglycans Sarcolemma Membrane Laminin-ɑ2 Sarcoglycan Complex Sarcospan ~34,000 boys in US/EU ~13% caused by nonsense mutations Dystrophin C-Term Syntrophins ɑ-dystrobrevin N-Term F-Actin Dystrophin stabilizes, but does not increase muscle strength May
16 DMD is a progressive and fatal genetic disorder Developmental delay Impaired standing Progressive ambulatory decline Transition to wheel chair skeletal deformity Very limited use of arms loss of self feeding Ventilation at night Ventilation 24 hours Death Ambulatory changes predict loss of function Age at loss of ambulation predicts loss of self feeding and need for ventilation May
17 Ataluren treats a distinct patient population within DMD Company Product candidate Target % DMD Population Phase Largest Trial ataluren Nonsense mutation 13% Ph drisapersen Exon 51 skip 13% Ph eteplirsen Exon 51 skip 13% Ph. 2b 12 PRO-044 Exon 44 skip 6% Ph. 1/2a 18 May
18 Ataluren slowed disease progression in nmdmd patients Based on Phase 2b post-hoc citt analysis May
19 6MWT outcomes in approved drugs that treat neuromuscular disorders are in the range of 30 m or less Aldurazyme (MPS I) 38 m Elaprase (MPS II) 35 m Treatment Ataluren (nmdmd) 31.3 m Lumizyme, Myozyme (Pompe Disease) 28 m Vimizim, Biomarin (MPS IVa) 23 m Mean Change in 6WMD Treatment vs Placebo May 2014 Measured in separate trials 18
20 Natural history from Phase 2b trial informs confirmatory Phase 3 clinical trial design Natural history and our Phase 2b data defined the best patient population to study McDonald et al., Goemans et al. and Mazzone et al. natural history 2013 studies using the 6MWT have shown similar results Maturational improvements Stabilization Decline May
21 Baseline 6MWD is a key factor in determining how DMD patients will perform over 48 weeks 350 m Change in 6MWD, mean (m) May
22 Baseline 6MWD is a key factor in determining how DMD patients will perform over 48 weeks <350 m Change in 6MWD, mean (m) Lessons learned The mean 6MWD in >350 meter population was relatively stable Difficult to demonstrate a drug effect over a 48-week study The mean 6MWD in <350 meter population show a large decline Best population to demonstrate an effect in ambulation in a 48-week study Confirmatory trial has inclusion criteria to enrich patients in the decline phase May
23 Ataluren demonstrated the greatest benefit over 48 weeks with patients in the ambulatory decline phase Change in 6MWD (m), mean (SEM) Baseline 40 mg/kg/day ataluren (n=32) Placebo (n=31) Based on Phase 2b post-hoc subgroup 1 analysis and citt Time (weeks) m (69) 49.9 m nominal p= m (85) May In patients on corticosteroids, between 7 and 16 years of age, with baseline 6MWD 150m and 80% of predicted 22
24 Ataluren treatment effect seen in all disease stages studied Severity Improving May
25 Ataluren demonstrated clinically meaningful differences in secondary endpoints 0 Stair Climb Stair Descend All pts Decline group All pts Decline group 10m Run/Walk All pts Decline group Improving s -2.8 s s -2.9 s s -2.9 s 40 mg/kg/day ataluren Placebo Secondary endpoints predict time to loss of ambulation 1.5 second threshold is a clinically meaningful difference in DMD 1 May Escolar
26 Ongoing confirmatory Phase 3 ACT DMD clinical trial targeting patients in the ambulatory decline phase Double-blind placebo-controlled study Length of Trial Ataluren (n) Placebo (n) 48 weeks Primary outcome measure: 6MWD (change from baseline) Secondary outcome measure: Timed-function tests North Star PODCI QoL Eligibility Criteria 7 years & 16 years Steroid use 6MWD 150 m 80% of predicted for age and height Stratification 350 m vs <350 m 9 years vs <9 On-track to complete enrollment in mid-2014 May
27 Ataluren: Status of Conditional Approval for DMD Application in Europe Past PTC applied for EU conditional approval in Q SAG-N meeting called by CHMP in December 2013 Oral Explanation held by CHMP in December 2013 CHMP notified PTC of negative opinion for conditional approval in January 2014 Next Steps PTC has filed for re-examination examination with the CHMP and a final decision is expected in Q ACT DMD confirmatory Ph 3 trial is enrolling and is on track for completion in mid-2014 May
28 Cystic fibrosis is a progressive and fatal genetic disease Life-threatening disease with average age of death in the mid-twenties Death typically due to respiratory failure Caused by defects in the CFTR gene ~70,000 in US/EU ~10% caused by nonsense mutations Nonsense mutations are the most severe form For patients with a nonsense mutation only palliative treatments are available CFTR May
29 Nonsense mutations cause the most severe form of CF CFTR channel through cell membrane CFTR CFTR CFTR CFTR CFTR protein CFTR CFTR CFTR CFTR CFTR CFTR CFTR CFTR CFTR cftr gene in cell nucleus Class Normal I No Synthesis II Processing Block III Gating IV Conductance V Reduced Synthesis % CF Patients 10% 70% 3-5% 2% 2% Compounds ataluren 1 VX809 VX661 Kalydeco Ataluren is only compound in development for the most severe form (Class 1) 1. VX809 and VX661 are only for homozygote, May % 28
30 Ataluren had a clinically meaningful benefit ( FEV %) in the 65% of nmcf patients not on inhaled TOBI Without Inhaled TOBI 1 With Inhaled TOBI 2 Week 48 = +5.7% (nominal p=0.008) Week 48 = -1.4% (nominal p=0.43) Ataluren decreased pulmonary exacerbation rates in Non-TOBI patients by 41% vs. placebo nominal p= No antibiotics or use of nonaminoglycoside antibiotics 2. Alone or with other antibiotics May Overall population decreased 23% (n=226, p=0.0992) 29
31 Our experience informed our Phase 3 trial design Initial Phase 3 Next Steps TOBI use interfered with ataluren s efficacy Non-TOBI patients had meaningful benefit Ataluren decreased pulmonary exacerbations Generally well tolerated Start confirmatory Phase 3 in H Primary endpoint: FEV 1 Restricted TOBI use Targeting complete enrollment mid-2015 Ataluren nmcf Phase 3 data expected mid-2016 May
32 Spinal Muscular Atrophy Discovery of a small molecule therapeutic
33 Spinal Muscular Atrophy: The leading genetic cause of mortality in infants Spinal muscular atrophy (SMA) is caused by the loss of a SMN-1 gene Low expression of SMN protein leads to the loss of motor neurons in the spinal cord Low levels of SMN lead to the death of motor neurons in the spinal cord and muscle atrophy One in every 10,000 children born is affected with the disorder No marketed therapies for SMA, only palliative treatments Dorsal Root Ventral Root May
34 Targeting alternative splicing in SMA SMA patients rely on a related SMN-2 gene which produces only low levels of SMN protein due to a splicing defect Small molecule targets splicing to correct defective splicing of exon 7 SMN-1 SMN-2 SMN-2 with Treatment DNA DNA DNA RNA 7 RNA RNA 7 SMN protein Unstable SMN protein Functional SMN protein May
35 Severity of SMA determined by % functional SMN Type I II III Disease Severity Most severe, infants have trouble breathing and don t usually live past 2 years of age Onset at 6-18 months, patient may walk briefly, death likely in early adulthood Patients may walk into early adulthood % Functional SMN % ~60% (+/- 5%) ~70% (+/- 5%) Incremental increases in SMN protein levels create meaningful clinical improvements 1. Protein levels measured in vitro fibroblast 1. May 2014 cells as a percent of heterozygous levels 34
36 PTC compounds have demonstrated dose-dependent increases in SMN protein Oral dosing for 10 days in SMA Type 3 mouse model Brain Peripheral Blood Mononuclear Cells SMN protein % increase SEM Heterozygous (no disease phenotype) ** *** *** SMN protein % increase SEM Heterozygous (no disease phenotype) *** *** *** 0 Vehicle Dose (mg/kg) 0 Vehicle Dose (mg/kg) SMN protein levels in peripheral blood cells correlate to those in brain Similar increases in SMN observed in spinal cord, muscle, heart, liver, skin Crosses blood brain barrier for pan tissue distribution May
37 Prolonged survival & body weight gain in Type I SMA mice Prolonged Survival Body Weight Gain Percent survival Age of mice (days) Heterozygous (n=10), Placebo (n=15), 1 mg/kg PO (n=16), 10 mg/kg PO (n=16) Pre-clinical data demonstrated improved phenotype and survival May
38 Prevention of muscle atrophy in severe Type I SMA mice Wild type SMA mouse Treated SMA mouse Mice were treated from 3-days old through 14-days old with Compound (3 mg/kg IP) May 2014 Courtesy of Dr. Ko (USC) 37
39 SMA Collaboration with Roche and SMAF SMAF/Roche Collaboration in SMA SMA Phase 1 Program (RG7800) Initial funding in part provided by SMA Foundation Partnered with Roche in late 2011 $30M upfront Up to $460M in milestones Healthy volunteer study n=48 Two arms: single ascending dose vs. placebo FPI Q Royalties Development Candidate declared in July 2013: $10M milestone Entered clinical development in Jan 2014: $7.5M milestone May
40 Observational studies in SMA program SMA Biomarker Study SMA Natural History Study Purpose: Single-center study with blood samples taken from SMA patients during a single visit for biomarker analysis Timeframe: Single visit Primary Outcome Measures: SMN1/SMN2 detection in blood by mrna assay Secondary Outcome measures: SMN protein level in blood / lymphocytes Purpose: Single-center study, 24-week longitudinal study in SMA patients matched to healthy volunteers by age and gender Timeframe: 24 weeks; testing at baseline, Week 12 and 24 Primary Outcome Measures: Quantitative muscle MRI and T2 values Disease progression, assessed through Motor Function Measure test Secondary Outcome measures: Disease progression using 6MWT Levels of SMN RNA Levels of SMN protein May
41 SMA program validates PTC s alternative splicing platform Focus on Alternative Splicing Include or exclude exons in mrna splicing to affect protein production DMD Exon51 Oncology Myostatin May
42 Financial snapshot March 31, 2014 cash balance: ~$247 million Market capitalization: ~$560 million (as of May 5, 2014) Approximately 30.1 million shares outstanding Sufficient capital to fund operations through 2016 May
43 PTC value drivers Additional SMA Data Expected Ataluren CF P3 Study Fully-Enrolled Submit for Ataluren Full Approval in DMD in 2016 Ataluren DMD P3 Study Fully Enrolled Initiate P3 Ataluren DMD Study Declare SMA Development Candidate Highlighted Pipeline at 1st R&D Day Initiate SMA P1 Study Initiate P3 Ataluren CF Study Ataluren Proofof-Concept Study in Other Indications Ataluren DMD P3 Top-Line Data Ataluren CF P3 Top-Line Data May
44 Questions & Answers Appendix slides May
45 Evaluating PTC s genetic disorder pipeline Ataluren and Nonsense readthrough program Additional potential indications Next generation readthrough Nonsense-mediated decay Leveraging PTC s RNA platform Nucleotide repeat sequences Exon skipping May
46 Ataluren targets a different sub-population in DMD vs. exon skipping therapies in development Nonsense Mutation Readthrough Exon Skipping VAL PHE TYR Normal Normal C A C A A A A U G G U G U U U U A C Nonsense VAL PHE C A C A A A No natural trna Singlepoint alteration Deletion G U G U U U U A G Nonfunctional Nonsense + Ataluren VAL PHE C A C A A A G U G U U U U A G TYR Exon Skipping May 2014 Truncated and 45 functional
47 Experience and natural history data informed our trial design What We Saw in Our Phase 2b trial Phase 3 Trial Ongoing 31 m delta in 6MWD for overall citt population 50 m delta in 6MWD in post hoc analysis of the decline subgroup Bell-shaped dose-response curve: demonstrated effective dose Generally well tolerated Inclusion criteria enriches for patients in decline 6MWD >150 m and <80% of predicted Boys 7-16 yrs 40 mg/kg/day dose Powered for 30 m delta in 6MWD Ataluren nmdmd Phase 3 data expected mid-2015 May
48 Extension cross-over trial demonstrates that patients FEV 1 is maintained or improves Relative Change in % predicted FEV 1, Mean Relative change in %-predicted FEV 1 through Week 96 May
49 Protection from neuromuscular junction denervation WT vehicle Blue = neuronal axon Green = NMJ, neuron Red = NMJ, muscle SMA treated SMA vehicle May 2014 Courtesy of Dr. Ko (USC) 48
50 Ataluren treatment was associated with improvements in dystrophin expression in Phase 2a nmdmd clinical trial Pretreatment End of Treatment (Day 28) Over 28 days, improvements were seen in ~60% of patients Phase 2a study; Bönnemann et al, May 2014 Neuromusc. Disord
51 Human myotube* data demonstrates a bell-shaped ataluren dose-response curve for dystrophin expression Mean Increase in Full-Length Dystrophin Expression Relative to Control * Samples from Phase 2a May 2014 nmdmd clinical trial patients 50
52 Time to persistent 10% worsening indicated a slower disease progression in the ataluren group mg/kg/day ataluren vs placebo Nominal p= % progressing 60 44% progressing mg/kg/day ataluren (n=57) Placebo (n=57) Weeks May
53 Individual change in 6MWD for patients meeting confirmatory Phase 3 nmdmd clinical trial entry criteria Placebo (n=30) Ataluren (n=30) Patients from Phase 2b study: on steroids, May 2014 age 7-16 y, baseline 6MWD 150 m, and baseline 6MWD <80%-predicted 52
54 All low-dose ataluren subjects had mean ataluren concentrations <19.3 g/ml 2 hours after the morning dose 25 Range <19.3 g/ml >19.3 g/ml 20 Percent of Patients mg/kg/day ataluren (n=60) 40 mg/kg/day ataluren (n=57) Concentration ( g/ml) May
55 6MWT results were better in the low concentration group of the 80 mg/kg/day group in the Phase 2b nmdmd trial 0-10 Mean change in 6MWD (m) m m 40 mg/kg/day ataluren (n=57) 80 mg/kg/day, low conc. ataluren (n=26) 80 mg/kg/day, high conc. ataluren (n=33) Placebo (n=57) m m May
56 Mean and significance levels for change in 6MWD by 2-hour plasma concentrations ranges confirmed better outcomes at lower exposures Low-Exposure Ataluren Minus Placebo (n=31) at Week 48 For patients on steroids, 7-16 y, 150 m 6MWD, <80% predicted May
57 Ataluren s activity in a Hurler nonsense mutation model 100 GAG reduction (% avg) genotype: (+/+) (-/-) Concentration (ug/ml) May
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