Cystic Fibrosis: Progress in Treatment Management. Patrick A. Flume, M.D. Medical University of South Carolina

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1 Cystic Fibrosis: Progress in Treatment Management Patrick A. Flume, M.D. Medical University of South Carolina

2 Disclosures Grant support Mpex Pharmaceuticals, Inc Gilead Sciences, Inc Bayer Healthcare AG Novartis Vertex Pharmaceuticals, Inc Pharmaxis Limited Boehringer Ingelheim Pharmaceuticals Savara Pharma KaloBios Cystic Fibrosis Foundation National Institutes of Health Consultant Gilead Vertex Pharmaceuticals, Inc Novartis Pharmaxis Limited Speaker s Bureau Genentech

3 Pathogenesis of CF Lung Disease Gene mutations Abnormal CFTR

4 Pathogenesis of CF Lung Disease Gene mutations CFTR quantity Abnormal CFTR Reduced ASL CFTR function

5 Pathogenesis of CF Lung Disease Gene mutations CFTR quantity CFTR function Reduced ASL Impaired MCC Obstruction

6 Airway Mucous Plugging in Cystic Fibrosis Used with permission J. Wagener, 2004.

7 Pathogenesis of CF Lung Disease Gene mutations CFTR quantity CFTR function Reduced ASL Impaired MCC Obstruction Infection

8 Respiratory Infections in CF Patients Cystic Fibrosis Foundation Patient Registry Annual Data Report. Bethesda, MD.

9 Pathogenesis of CF Lung Disease Gene mutations CFTR quantity CFTR function Reduced ASL Impaired MCC Obstruction Infection Inflammation

10 Inflammation in Bronchioalveolor Lavage Fluid Control Cystic Fibrosis Used with permission - J. Wagener, 2004.

11 Pathogenesis of CF Lung Disease Gene mutations CFTR quantity CFTR function Reduced ASL Impaired MCC Obstruction Infection Inflammation

12 Pathogenesis of CF Lung Disease Gene mutations CFTR quantity CFTR function Reduced ASL Impaired MCC Obstruction Infection Inflammation Progressive, irreversible lung damage Respiratory failure

13 Chronic Therapies for Maintenance of Lung Health A Strong Recommendation for: B Recommendation for: D Recommendation against: Inhaled tobramycin Mod-severe dz Dornase alfa Mod-severe dz Inhaled tobramycin Mild-asx dz Dornase alfa Mild-asx dz Hypertonic saline Macrolides Ibuprofen Inhaled -agonists Oral steroids Age 6-18 Inhaled steroids Anti-Staph abx Insufficient Evidence to make a recommendation: Other aerosolized antibiotics N-acetyl cysteine Cromolyn Flume et al, Am J Respir Crit Care Med 2007; 176: Inhaled anticholinergics Leukotriene modifiers Oral steroids (age>18)

14 Chronic Therapies for Maintenance of Lung Health A Strong Recommendation for: Inhaled tobramycin Mod-severe dz Inhaled aztreonam Mod-severe dz Dornase alfa Mod-severe dz Ivacaftor (G551D) B Recommendation for: Inhaled tobramycin Mild-asx dz Inhaled aztreonam Mild-asx dz Dornase alfa Mild-asx dz Hypertonic saline Macrolides (with PA) Ibuprofen (<18 yrs) D Recommendation against: Oral steroids Age 6-18 Inhaled steroids Anti-Staph abx Insufficient Evidence to make a recommendation: Other aerosolized antibiotics Chronic oral anti-staph N-acetyl cysteine Cromolyn Flume et al, Am J Respir Crit Care Med 2007; 176: Mogayzel et al, Am J Respir Crit Care Med 2013; 187: Inhaled β-agonists (chronic) Inhaled anticholinergics Leukotriene modifiers Oral steroids (age>18) Macrolides (without PA)

15 Challenges in the Treatment of CF Lung Disease Gene mutations CFTR quantity CFTR function Reduced ASL Impaired MCC Dornase alfa Bronchodilators Obstruction Infection Inflammation Hydrators Antibiotics Ibuprofen, Macrolides Progressive, irreversible lung damage Respiratory failure

16 Challenges in the Treatment of CF Lung Disease Gene mutations CFTR quantity CFTR function Reduced ASL Impaired MCC Dornase alfa Bronchodilators Obstruction Infection Inflammation Hydrators Antibiotics Ibuprofen, Macrolides Progressive, irreversible lung damage Respiratory failure

17 Challenges in the Treatment Insert a normal gene of CF Lung Disease Gene mutations Fix the CFTR defect CFTR quantity CFTR function Bypass the function of CFTR Reduced ASL Impaired MCC Dornase alfa Bronchodilators Obstruction Infection Inflammation Hydrators Antibiotics Ibuprofen, Macrolides Progressive, irreversible lung damage Respiratory failure

18 Strategies to Treat Early Aspects of Pathogenesis of CF Lung Disease Replace the gene Gene Therapy

19 Gene Therapy for CF UK Cystic Fibrosis Gene Therapy Consortium Multi-dose clinical trial of gene therapy 130 patients age > 12 years First study to look at a clinical benefit Expected completion in

20 Strategies to Treat Early Aspects of Pathogenesis of CF Lung Disease Replace the gene Gene Therapy Bypass the effects of CFTR alternate channels Inhibit sodium absorption (ENaC) Stimulate chloride secretion (P 2 Y 2, CaCC)

21 Strategies to Treat Early Aspects of Pathogenesis of CF Lung Disease Replace the gene Gene Therapy Bypass the effects of CFTR alternate channels Correct the CFTR mutation CFTR modulators

22 Challenges in the Treatment of CF Lung Disease Gene mutations CFTR quantity CFTR function CFTR Potentiators (e.g. ivacaftor) Reduced ASL Impaired MCC Dornase alfa Bronchodilators Obstruction Infection Inflammation Hypertonic saline Antibiotics Ibuprofen, Macrolides Progressive, irreversible lung damage Respiratory failure

23 Ivacaftor in Patients with CF and G551D Sweat Chloride Normal: <40 mmol/l CF: >60 mmol/l Ramsey et al., NEJM 2011; 365:

24 Ivacaftor in Patients with CF and G551D Ramsey et al., NEJM 2011; 365:

25 Ivacaftor for G551D CFTR Modulation

26 Important Questions Would earlier treatment prevent CF lung disease?

27 What might we expect from earlier treatment with ivacaftor? Ramsey et al., NEJM 2011; 365: Variable Age at diagnosis (yrs) Sweat chloride (mean) FEV 1 (mean % pred) S aureus or P aeruginosa Clubbing CF-PI % 100% 71% Goubau et al., Thorax 2009; 64: CFTR-dysfunction % 65% 27%

28 Important Questions Would earlier treatment prevent CF lung disease? Will ivacaftor work for other CFTR gene mutations?

29 Ivacaftor Potentiation of CFTR Channels with Gating Mutations Yu H et al. J Cyst Fibr 2012; 11:

30 What about ivacaftor for other gene mutations? Trial of ivacaftor in subjects with CF who have a non-g551d CFTR gating mutation 1 G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D Clinicaltrials.gov: 1 NCT ;

31 Ivacaftor CFTR Modulation G551D plus age 2-5 years Other gating mutations

32 What about ivacaftor for other gene mutations? Trial of ivacaftor in subjects with CF who have a non-g551d CFTR gating mutation 1 G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D Trial of ivacaftor in subjects with R117H 2 Clinicaltrials.gov: 1 NCT ; 2 NCT

33 Ivacaftor CFTR Modulation G551D plus age 2-5 years Other gating mutations R117H

34 CFTR Gene mutations Cystic Fibrosis Foundation Patient Registry Annual Data Report. Bethesda, MD.

35 What about ivacaftor for other gene mutations? Trial of ivacaftor in subjects with CF who have a non-g551d CFTR gating mutation 1 G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D Trial of ivacaftor in subjects with R117H 2 Not effective for df508 3 Clinicaltrials.gov: 1 NCT ; 2 NCT Flume et al., Chest 2012; 142:

36 CFTR Mutations and Quantity of CFTR Yu H et al. J Cyst Fibr 2012; 11:

37 Challenges in the Treatment of CF Lung Disease Gene mutations CFTR Correctors (e.g. VX809, VX661) CFTR quantity Reduced ASL Impaired MCC CFTR function Hypertonic saline Dornase alfa Bronchodilators Obstruction Infection Inflammation Hypertonic saline Antibiotics Ibuprofen, Macrolides Progressive, irreversible lung damage Respiratory failure

38 F508del-CFTR Activity (% wild-type-cftr) F508-CFTR C-band (normalized to GAPDH) F508-CFTR C-band (normalized to GAPDH) F508-CFTR C-band (normalized to GAPDH) A Cellular Processing of F508del-CFTR B ER C- B- A C- B- B GAPDH wt A F508 C- B- B- C- B- GAPDH CFTR Corrector C- GAPDH wt F508 CFTR-mediated Cl Flux B B Normal wt Log F508 F508del- M [VX-809] Log M [VX-809] C 0-8CFTR CFTR C- C- B- Control VX-809 Log M [VRT ] C Control Corrector molecules appear to hit an efficacy ceiling at 10-20% correction Van Goor et al. Pediatr Pulmonol 2009; 44 (S32) 3 C * VX Log M 1[V 0-9 Lo

39 CFTR Corrector Monotherapy Clancy JP et al. Thorax 2012; 67: 12-18

40 F508del-CFTR Activity (% wt-cftr) CFTR Corrector + Potentiator F508del-CFTR CFTR corrector VX Ivacaftor CFTR potentiator VX-809 alone Log M [VX-809] Van Goor et al. Pediatr Pulmonol 2009; 44 (S32)

41 Clinical Evaluation of Lumacaftor Alone and in Combination with Ivacaftor: Cohort 2 Study Design Lumacaftor Monotherapy Lumacaftor and Ivacaftor Combination n=23 lumacaftor 200 mg qd lumacaftor 200 mg qd + ivacaftor 250 mg q12h F508del homozygotes n=21 n=21 lumacaftor 400 mg qd lumacaftor 600 mg qd lumacaftor 400 mg qd + ivacaftor 250 mg q12h lumacaftor 600 mg qd + ivacaftor 250 mg q12h F508del heterozygotes n=17 n=6 Placebo Placebo + Placebo n=21 lumacaftor 600 mg qd lumacaftor 600 mg qd + ivacaftor 250 mg q12h Day -21 Day 0 Day 14 Day 28 Day 42 Day 56 n=number of patients randomized and dosed 41

42 Boyle M, presented at NACFC 2012

43 Current Status for CFTR Modulators for df508 Phase 3 studies of lumicaftor (VX-809) + ivacaftor in subjects homozygous for df508 1 Phase 2 study of VX-661 +/- ivacaftor in subjects homozygous for df508 2 Press release April 18, 2013 Dose of VX-661 n Absolute change in FEV 1 (%) Relative change in FEV 1 (%) Placebo mg mg mg * 9.0* 150 mg * 7.5** Clinicaltrials.gov: 1 NCT and NCT Clinicaltrials.gov: 2 NCT *p=0.01; **p=0.02

44 Ivacaftor CFTR Modulation G551D plus age 2-5 years Other gating mutations R117H Homozygous df508

45 Ivacaftor CFTR Modulation G551D plus age 2-5 years Other gating mutations R117H Homozygous df508 Heterozygous df508

46 Strategic Initiative for Next Generation Discovery Vertex Combinatorial approach for synergy Genzyme Mechanistic CFTR-target based approach Pfizer Mechanistic and cell based, combinational therapy Proteostasis CFTR interactome and folding environment Reata Cell based NBD stabilization McGill/Glaxo Smith Kline CFTR surface expression, mechanistic target based

47 What else is in the pipeline?

48 CFTR Correctors (e.g. VX809, VX661) Challenges in the Treatment Dornase alfa of CF Lung Disease CFTR quantity Bronchodilators Gene Therapy Gene mutations Reduced ASL Impaired MCC CFTR function Obstruction Infection Inflammation Hypertonic saline Mannitol Antibiotics Hypertonic saline Mannitol Ibuprofen, Macrolides CFTR Potentiators (e.g. ivacaftor) Progressive, irreversible lung damage Respiratory failure

49 Inhaled Mannitol in CF Aitken ML, et al. Am J Respir Crit Care Med 2012; 185:

50 CFTR Correctors (e.g. VX809, VX661) Challenges in the Treatment Dornase alfa of CF Lung Disease CFTR quantity Bronchodilators Gene Therapy Gene mutations Reduced ASL Impaired MCC CFTR function Obstruction Infection Inflammation Hypertonic saline Mannitol More antibiotic choices Change treatment strategy Antibiotics Hypertonic saline Mannitol Ibuprofen, Macrolides CFTR Potentiators (e.g. ivacaftor) Progressive, irreversible lung damage Respiratory failure

51 Inhaled antibiotics targeting Pseudomonas Antibiotic Class Aminoglycosides FDA Approved TIS (TOBI ) Bethkis TIP (Powder) Used Off-Label Gentamicin Beta-lactams AIS (Cayston ) Ceftazidime Fluoroquinolones Polymyxins Colistimethate

52 What questions remain? Do we need more antibiotic choices for Pseudomonas? Do we have the optimal regimen? Should we exercise the same strategy for other pathogens?

53 Inhaled antibiotics targeting Pseudomonas Antibiotic Class FDA Approved In Development Used Off-Label Aminoglycosides TIS (TOBI ) Bethkis Bramitob TIP (Powder) Liposomal amikacin Gentamicin Beta-lactams AIS (Cayston ) Ceftazidime Fluoroquinolones Levofloxacin aerosol Liposomal ciprofloxacin Ciprofloxacin powder Polymyxins Colistimethate

54 standard of care Dosing regimens A A A A Time A (weeks) A B B B A A A A A A Time (weeks) intermittent monotherapy continuous combination therapy continuous monotherapy

55 Assessing combination inhaled antibiotic therapy by randomized controlled trial US standard of care TIS TIS TIS Placebo Placebo Placebo TIS AIS TIS AIS TIS AIS Time (weeks) clinicaltrials.gov identifier: NCT

56 Inhaled antibiotics in CF Pathogen Prophylaxis Eradication Suppression Pseudomonas No Yes Yes Staph (MRSA)??? Stenotrophomonas??? Burkholdheria?? No? NTM???

57 Respiratory Infections in CF Patients Cystic Fibrosis Foundation Patient Registry Annual Data Report. Bethesda, MD.

58 Inhaled antibiotics targeting Staphylococcus aureus Antibiotic Class FDA Approved In Development Used Off-Label Glycopeptides Vancomycin powder (Phase 2) Vancomycin IV solution Clinicaltrials.gov identifier: NCT

59 Inflammation in Bronchioalveolor Lavage Fluid Control Cystic Fibrosis Used with permission - J. Wagener, 2004.

60 Inflammation in CF Airways Inflammation in the airways is exaggerated Neutrophil is the predominant WBC Inflammation: Causes injury to the airways (neutrophil elastase) Recruits more inflammatory cells to the airways

61 Approach to Reducing Injury from Inflammation in CF Airways Inhibit neutrophil migration to the airways SB Inhibit neutrophil elastase Augmentation of alpha-1-antitrypsin 2 1 Clinicaltrials.gov: NCT ; J Cyst Fibr 2013; 12: Clinicaltrials.gov NCT

62 CFTR Correctors (e.g. VX809, VX661) Challenges in the Treatment Dornase alfa of CF Lung Disease CFTR quantity Bronchodilators Gene Therapy Gene mutations Reduced ASL Impaired MCC CFTR function Obstruction Infection Inflammation Hypertonic saline Mannitol More antibiotic choices Change treatment strategy Antibiotics Hypertonic saline Mannitol Ibuprofen, Macrolides CFTR Potentiators (e.g. ivacaftor) Alpha-1-antitrypsin Anti-Pseudomonas Ab Progressive, irreversible lung damage Respiratory failure

63 Pa Type III Secretion System and KB001-A Cell Membrane P. aeruginosa TTSS Anti-PcrV Antibody (KB001-A) PcrV Protein Membrane disruption and exotoxin injection into cells KB001-A renders PA non-toxic by binding to the PcrV protein on the tip of the TTSS on the surface of PA and therefore prevents PA from attacking host cells through its primary toxic mechanisms. Clinicaltrials.gov identifier: NCT

64 Conclusions We have made tremendous progress in the treatment of CF patients Survival has improved Yet, patients still die too young We need new medications Recent developments offer huge potential True example of personalized medicine

65 CFTR Modulation - this is the goal Ivacaftor G551D plus age 2-5 years Other gating mutations R117H Homozygous df508 Heterozygous df508 Nonsense mutations Others

66 Conclusions But until that goal is reached: We need to use current medications more effectively Comparative effectiveness Assess treatment regimens (e.g. strategy of inhaled antibiotics) Personalize patient s treatment regimen Focus on adherence Appreciate the treatment burden

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