A Quick Guide to the G A. Mutation CFTR SCIENCE

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1 A Quick Guide to the G A Mutation FR SIENE 2016 Vertex Pharmaceuticals Incorporated VXR-HQ a(1) 03/2016

2 Loss of FR activity is the underlying cause of cystic fibrosis (F) 1 Spectrum of Phenotypes Associated With otal FR 1,2 otal FR % of Normal 100% No F Disease FR-related Disorders Some FR mutations result in residual or partial FR activity 3-5 0% 100% Some FR mutations result in little to no FR activity 3-5 0% 100% People with 2 FR mutations resulting in loss of FR activity generally have a F phenotype, which may include 1-3,6 Elevated sweat chloride (>60 mmol/l) Pancreatic insufficiency BAVD a Lung function decline over time Pseudomonas aeruginosa colonization 0% ystic Fibrosis otal FR otal FR a BAVD, congenital bilateral absence of the vas deferens. References: 1. Davis PB et al. Am J Respir rit are Med. 1996;154(5): Rowe SM et al. Proc Am horac Soc. 2007;4(4): Zielenski J. Respiration. 2000;67(2): Sheppard DN et al. Nature. 1993;362(6416): Welsh MJ, Smith AE. ell. 1993;73(7): astellani et al. J yst Fibros. 2008;7(3):

3 Levels of FR activity affect survival in F 1 Survival Probability Survival urves by FR During a 10-Year Follow-Up ( ) of Patients From the US FF Registry a Residual FR activity (lass IV, V): R117H, R334W, R347P, Kb, G A, A455E Severely reduced FR activity (lass I, II, III): G542X, R553X, W1282X, R1162X, 621-IG, G A, 1078del, 3659del, I507del, N1303K, S549N, G85E, F508del, G551D, R Risk ime (Age) n=1126 n=14, Adapted with permission from McKone EF et al. hest. 2006;130(5): his survival curve represents population-based outcomes. 1 Individual outcomes in cystic fibrosis are variable. a Data are from a retrospective study of patients enrolled in the ystic Fibrosis Foundation patient registry measuring risk of death over a 10-year observation period from 1993 to Patients were grouped as having a high-risk or low-risk genotype based on the functional effects of their class of FR mutation on phenotype and mortality. Patients having a lass I, II, or III mutation on both alleles were considered high-risk, while patients having at least 1 lass IV or V mutation were categorized as low-risk. A total of 15,651 patients had a FR genotype of a known functional class; 14,525 (93%) had a high-risk FR genotype and 1126 (7%) had a low-risk FR genotype.1 References: 1. McKone EF et al. hest. 2006;130(5): he World Bank. Accessed November 12, MacKenzie et al. Ann Int Med. 2014;161: Life expectancy in Western countries (general population born in 2000) is ~79 years 2 Between 1993 and 2002, median survival for US patients with genotypes associated with little to no FR activity was 36.3 years (95% I, 35.5 to 37.6 years), while median survival for those having genotypes associated with residual FR activity was 50 years (95% I, 47.1 to 55.9 years) 1 In this study, patients with the G A mutation (lass I) were part of the severely reduced FR activity group More recent US data ( ) suggest median survival across genotypes continues to improve 3 3

4 ountry registries listing the G A mutation report 0.1% to 3% prevalence among patients with F1-10 Prevalence of the G A Mutation in Patients With ystic Fibrosis (% of Patients With at Least 1 Allele) I n the FR2 global database, ~1% of patients with F have at least 1 copy of the G A mutation11 Additional sources report frequency of the G A mutation on F alleles US1: 2% % of Alleles ountry Brazil2: 0.1% Europe: Belgium3: 3% France4: 2% UK5: 1% Ireland6: 1% Netherlands7: 1% Germany8: 0.7% Aus : 2% 9 New Zealand : 0.5% 10 References: 1. ystic Fibrosis Foundation. ystic Fibrosis Foundation Patient Registry 2013 Annual Data Report. Bethesda, MD. 2014; ystic Fibrosis Foundation. 2. he Brazilian ystic Fibrosis Study Group. Brazilian ystic Fibrosis Patient Registry Annual Report. 3. Belgisch Mucoviscidose Register Registre Belge de la Mucoviscidose. he Belgian ystic Fibrosis Registry. Summary Report ; Institute of Public Health (WIV-ISP), Brussels, Belgium. 4. French ystic Fibrosis Registry. Annual Report Vaincre la Mucoviscidose and Ined, 2015; Paris, France. 5. ystic Fibrosis rust. UK ystic Fibrosis Registry Annual Data Report ; ystic Fibrosis rust; London, UK. 6. he ystic Fibrosis Registry of Ireland Annual Report. FRI, 2015; Dublin, Ireland. 7. Nederlandse ystic Fibrosis Stichting. Dutch ystic Fibrosis Patient Regristry. Report on the Year NFS. 8. Mukoviszidose e.v. und Mukoviszidose Institut gemeinnützige Gesselschaft für Forschung und herapienntwicklung mbh. Beriichtsband Qualitätssicherung Mukoviszidose , Bonn, Germany. 9. ystic Fibrosis Australia. Australian ystic Fibrosis Data Registry th Annual Report. 2015; ystic Fibrosis Australia; Baulkham Hills NSW, Australia. 10. POR FNZ National Data Registry, 2014 Registry Report, ystic Fibrosis New Zealand. hristchurch, New Zealand. 11. US F Foundation, Johns Hopkins University, he Hospital for Sick hildren. he linical and Functional Ranslation of FR (FR2). Accessed November 12, European ystic Fibrosis Society. EFS Patient Registry 2010 Annual Data Report Bobadilla JL et al. Hum Mutat. 2002;19(2): World Health Organization. he molecular genetic epidemiology of cystic fibrosis: report of a joint meeting of WHO/EFN/IF(M)A/EFS; June 19, 2002; Genoa, Italy. World Health Organization; Switzerland12 2% Italy13 2% Hungary 2% Poland13 2% anada 0.4%

5 he G A mutation results in defective biosynthesis of the FR protein 1-3 FR urnover FR Function FR rafficking FR Processing Illustrative Example of lass I Defect Golgi complex Endoplasmic reticulum FR gene (DNA) A DNA mrna Proteosome hromosome 7Rest of FR Endoplasmic FR gene gene Reticulum G A A Splicing defect Pre-mRNA RNA processing G A is a splice mutation, which produces a premature stop codon 1 he cell cannot synthesize a full-length FR protein, a lass I mutation 1-3 As a result, few to no FR proteins are present at the apical cell surface 3 mrna FR Synthesis ranscription DNA References: 1. Hull J et al. Hum Mol Genet. 1993;2(6): Green DM et al. Respir Res. 2010;11:140. doi: / Zielenski J. Respiration. 2000;67(2):

6 he G A allele results in little to no total FR activity otal FR activity can be defined as total ion transport mediated by FR protein channels at the cell surface, depending on FR protein quantity and function G A allele results in few to no FR channels at apical surface Defective Synthesis (lass I) FR Function FR hannel-open Quantity x x onductance = Probability hannel-open x onductance: N/A Probability: N/A x = 2 otal FR Little to No G A FR 3 1 A virtual absence of G A-FR protein quantity 2 regardless of function since few to no FR proteins reach the surface 3 results in little to no total FR activity N/A, not applicable. References: 1. Hull J et al. Hum Mol Genet. 1993;2(6): Green DM et al. Respir Res. 2010;11:14. doi: / Zielenski J. Respiration. 2000;67(2): Welsh MJ, Smith AE. ell. 1993;73(7): Sheppard DN et al. Nature. 1993;362(3):

7 Both FR alleles play a role in determining phenotype or disease severity % No F Disease Normal individuals and F carriers otal FR 0% FR-related Disorder linical entities associated with FR dysfunction that do not fulfill diagnostic criteria for F ystic Fibrosis A G A allele results in little to no FR activity. he phenotype of a particular patient is also influenced by the mutation on the other allele G A typically results in the indicated phenotypes Allele 1 otal FR Normal Normal Normal Residual Residual Little to None Allele 2 otal FR Normal Residual Little to None Residual Little to None Little to None Adapted from Zielenski J. Respiration. 2000;67(2): References: 1. US F Foundation, Johns Hopkins University, he Hospital for Sick hildren. he linical and Functional Ranslation of FR (FR2). Accessed November 12, degracia J et al. horax. 2005;60(7): ystic Fibrosis Genotype-Phenotype onsortium. N Engl J Med. 1993;329(18): Davis PB et al. Am J Respir rit are Med. 1996;154(5): astellani et al. J yst Fibros. 2008;7(3): Zielenski J. Respiration. 2000;67(2):

8 1717-1G A in combination with another allele that produces little to no FR activity usually results in a F phenotype 1-5 FR Genotype Allele #1: G A Little to No FR Protein Allele #2 Little to No FR Protein F Phenotype In patients registered in the FR2 database with a G A mutation on 1 allele and a pancreatic insufficient mutation on the second allele 1 Modifier Genes Little to No otal FR Environmental Factors Elevated sweat chloride (average):103 mmol/l Lung function decline over time Pseudomonas colonization: 54% of patients Pancreatic insufficiency: 97% of patients References: 1. US F Foundation, Johns Hopkins University, he Hospital for Sick hildren. he linical and Functional Ranslation of FR (FR2). Accessed Novemebr 12, degracia J et al. horax. 2005;60(7): ystic Fibrosis Genotype-Phenotype onsortium. N Engl J Med. 1993;329(18): Davis PB et al. Am J Respir rit are Med. 1996;154(5): astellani et al. J yst Fibros. 2008;7(3):

9 Summary Loss of FR activity is the underlying cause of F Levels of FR activity affect survival in F ountry registries listing the G A mutation report 0.1% to 3% prevalence among patients with F he G A mutation results in defective biosynthesis of the FR protein he G A allele results in little to no total FR activity Both FR alleles play a role in determining phenotype or disease severity G A in combination with another allele that produces little to no FR activity usually results in a F phenotype 9

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