Opening Doors to CF Clinical Research: Change is Coming

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1 Opening Doors to CF Clinical Research: Change is Coming

2 Disclosures Contracts with sponsors for clinical trials conducted at UNC-Chapel Hill: CFFT NIH/NHLBI AbbVie Inc. Aptalis Pharma US, Inc. Janssen Research & Development, LLC. Gilead Sciences Savara Pharmaceuticals Vertex Pharmaceuticals Materials for slides provided by: Nivalis Therapeutics, Inc., Savara Pharmaceuticals

3 NACFC GOAL Consensus Guidelines Mental Health Task Force PROSPECT LLC

4 National Cystic Fibrosis Research Foundation Founded By Parents

5 What We Learned from Plenary I The importance of therapies directed toward specific CF gene mutations Assessing individual responses to therapy The need for effective therapies regardless of the CF gene mutation How to begin thinking about the application of personalized therapy to CF care Using new measurements and new tools for improving adherence and engagement

6 CFTR gene defect CFTR Modulators Abnormal CFTR protein Defective ion transport Airway surface liquid depletion Delayed Mucociliary clearance Pathophysiology of Cystic Fibrosis CFTR Modulators Scarring Mucus Obstruction Cycle of Destruction Inflammation Infection End Stage Lung Disease

7 N91115: a GSNOR inhibitor (S-nitrosoglutathione reductase inhibitor) Orally bioavailable agent Increases S-nitrosoglutathione (GSNO) concentrations GSNO has a key role in modulating protein function May act as a new type of CFTR modulator Multiple potential sites of action

8 N91115 Affects CFTR Stability in F508del Addition of N91115 to potentiator + corrector approach could improve patient outcomes Slide courtesy of Nivalis

9 Phase 1b Study of N91115 Double-blind, randomized, placebo-controlled trial 51 total patients recruited at 19 TDN clinical sites Doses: 50, 100, 200 mg twice a day as monotherapy 28 days tx, followed by 2 week withdrawal & follow-up Primary endpoints safety, pharmacokinetics Exploratory endpoints Lung function, sweat chloride, inflammatory biomarkers

10 Phase 1b Study Conclusions Good safety profile N91115 was well-tolerated at all doses for 28 days Sweat chloride response consistent with a threshold dose effect S. Donaldson: W14 Early Studies of Novel Small Molecule Therapies: Phase 1b study- N91115, a Stabilizer that Modulates CFTR Activity: From Bench to Bedside. Fri. Oct. 9 th, 3:30 PM Poster #250: J. Taylor-Cousar: The Pharmacokinetics of N91115, an Inhibitor of S- Nitrosoglutathione Reductase, in Cystic Fibrosis Patients Poster #270: S Donaldson: Safety and Pharmacokinetics of N91115 In Patients with Cystic Fibrosis Homozygous for the F508del-CFTR Mutation.

11 Plans for N91115 Phase 2 Study N91115 added to Orkambi in adults homozygous for F508del Double-blind, randomized, placebo controlled, parallel group trial 3 treatment arms (45 patients each) Primary endpoint Absolute change in percent predicted FEV 1 Study Duration 12 weeks followed by 4-week follow-up period Study Start First Patient First Visit targeted for late 2015

12 Improving Pediatric Therapy As we bring approved therapies to younger age groups how do we show efficacy? How do you test the efficacy of a therapy in patients with normal lung function?

13 Multiple Breath Washout and Lung Clearance Index (LCI) LCI reflects the evenness or uniformity of ventilation in the lung Higher number means worse lung function Is it more sensitive than traditional lung function testing? Yes Can it be used in children? And used in clinical trials? Yes Subbarao, et al, Annals ATS, 215

14 Longitudinal Study of LCI Exciting results regarding MBW/LCI in children, from an NIH multicenter trial led by Dr. Felix Ratjen, Toronto Feasibility of testing young children Performance or stability over time Impact of Pulmonary Exacerbations Critical information for use in clinical trials Use clinically to assess lung function Results presented at: F. Ratjen: Symposium 10.3, Friday 11:30 Posters: R. Jensen: Poster 191: Progression and Variability of LCI in Preschool Children with CF H Webster: Poster 242: Utility of the LCI to Monitor Pulmonary Exacerbations in Young Children with CF Symposium 20 - Pro-Con Debate, Saturday 2:30, discuss use of MBW/LCI

15 Ivacaftor and LCI outcome Cross-over study (UK, US, Canada) Ivacaftor vs. placebo for 28 days FEV 1 >90% predicted G551D mutation 6 years age (range 8-43 yr.) Results: Treatment difference FEV 1 influenced by large airway flow resistance LCI measures evenness of distribution of ventilation changes in peripheral airways Davies, et al. Lancet Respir Med 2013; 1: VX : MBW/LCI selected as primary outcome for efficacy. Ivacaftor/lumacaftor study in younger children with CF, ages 6-11 years

16 Saline Hypertonic In Preschoolers (SHIP) Study Hypertonic saline improves mucociliary clearance in older patients with CF ISIS randomized, controlled trial (<6 years), HS did not affect rate of pulmonary exacerbations (primary endpoint) In small pilot ISIS sub-study in Toronto (N=27), treatment effect of HS seen in lung clearance index, driven by effect in preschool age HS also shown to improve LCI in school age children with CF SHIP Study (CFFT) 48 weeks of HS vs isotonic saline 3 to 6 years old Primary outcome is LCI by MBW PIs: S. Davis, F. Ratjen, M. Rosenfeld 16

17 CFTR gene defect Abnormal CFTR protein Defective ion transport Airway surface liquid depletion Delayed Mucociliary clearance Pathophysiology of Cystic Fibrosis MRSA Treatment Mucus Obstruction Scarring Cycle of Destruction Infection MRSA Treatment Inflammation End Stage Lung Disease

18 MRSA prevalence in CF Up to 50% positive in some centers 25% 2%

19 Limited Treatment Options for MRSA Exacerbations treated with IV vancomycin or linezolid Systemic toxicities and poor lung penetration of IV abx Growing use of suppressive oral anti-mrsa antibiotics Emerging use of off-label nebulized vancomycin Slide courtesy, Savara Pharmaceuticals

20 MRSA is a significant problem for some patients Can it be eradicated when first acquired? How to manage chronic infection? 20

21 STAR-Too Trial Eradication of newly acquired MRSA PIs: M. Muhlebach and C. Goss, CFFT funding Treatment vs. Observation arms Outcome: presence or absence of MRSA at day 28 Enrolled 45 patients, in 14 centers (86% <18 yrs) Treated group: 67% MRSA culture negative Observational group: 13% MRSA culture negative C. Goss STAR-Too Trial Results: Symposium 11.3, Friday 11:30

22 STAR-Too Trial

23 Inhaled Vancomycin Therapy Need for suppressive therapy for those chronically infected with MRSA Similar to suppressive therapy for Pseudomonas Inhaled tobramycin Inhaled aztreonam Inhaled colistin Illustrations courtesy, Savara Pharmaceuticals

24 AeroVanc Phase II Study in CF Screening 32 mg bid 64 mg bid 12 years old Persistent MRSA FEV 1 30% 100% 2 capsules bid 20 on AeroVanc 20 on placebo 4 weeks 4 capsules bid 24 on AeroVanc 23 on placebo 4 weeks Primary Endpoint MRSA CFU reduction Secondary Endpoints FEV 1 improvement Decrease of respiratory symptoms (CFRSD) Prolonged time to need of other antibiotics Preparing to start Phase III in 2016 E. Dasenbrook: Symposium 11.4, Friday 11:55 Slide courtesy, Savara Pharma.

25 CFTR gene defect Abnormal CFTR protein Defective ion transport Airway surface liquid depletion Delayed Mucociliary clearance Pathophysiology of Cystic Fibrosis Pulmonary Exacerbations Scarring Mucus Obstruction Cycle of Destruction Inflammation Infection End Stage Lung Disease

26 44% 6160 pts 27% 3752 pts CFF Patient Registry, 2013

27 Treating Pulmonary Exacerbations How do we treat pulmonary exacerbations? Which antibiotics? How long do we treat? What are outcomes and measurements of improvement? When do we start treatment? Early? With monitoring at home? When it s convenient? Do we use steroids? Do we treat at home or in hospital?

28 Standardized Treatment of Pulmonary Exacerbations (STOP) -pilot, feasibility Design: Prospective, observational study of patients admitted to hospital for treatment of a pulmonary exacerbation (PEx) with intravenous abx. Antibiotic, dose and tx duration determined by the treating clinician (not dictated). Treating clinician specified treatment goals at start of therapy. Inclusion: Patients 12 years of age, hospitalized for acute PEx, IV abx. Exclusion: Recent IV antibiotics; pneumotx.; hemoptysis; ABPA, NTM tx Collected: PFTs, PRO, type and duration of treatment, duration in hospital. Data into the CFF Pt. Registry, over 28 days from time of admit Primary endpoints: Feasibility and clinical equipoise for future pragmatic interventional studies on the treatment of PEx (modules for CFF Pt. Reg). PIs: C. Goss, P. Flume, S. Heltshe, D. VanDeVanter, DB Sanders, N. West

29 STOP Pilot Capture FEV 1 and symptoms at key time points during treatment of a PEx to help future trial endpoint selection Start Abx End of IV Tx End of Study Visit 1 2 Discharge 3 4 Day 1 7 Study Visit PFTs PFTs During Hospitalization (preferably 2-3 Days apart) Variable Symptom Diary Completed Daily 28 N=220 pts, over 1 year at 10 study sites

30 STOP Study Conclusions STOP-OB achieved its goals, necessary to plan first interventional study of PEx STOP 2 Provided data to establish equipoise for intervention studies in exacerbation Was a critical step for refining outcome measures and sample size estimates for next study However, broader community input was needed Feasibility and buy-in for study design Clinicians Patients and Caregivers CF Adult and Family Advisor (AFA) group

31 Physician s Rank of Key PEx Questions Number of respondents N= is most important and 5 least N=102

32 Physician's Rank of Key Outcomes Number of points N=88 FEV1 Symptoms Time to Next *Retreatment in 30 days By attributing a point value to the responses (1 through 4) and multiplying by the number of response (lower total suggests greater interest) N=88

33 Patient & Caregiver s Goals of Treatment Percent of respondents 77% of the patient/caregivers report symptom relief as the primary goal of treatment Recover lung function Relief of symptoms Other Don't know Percent of respondents Recover lung function Relief of symptoms to allow return to work or school Complete relief of symptoms Other However, respondents thought doctor s primary goal of antibiotic treatment was equally recovery of lost lung function (43%) and relief of symptoms (40%)

34 Patient and Caregiver Survey Highlights Willing to participate in such a study Complete symptom resolution is a high priority They trust their physicians Expressed legitimate concerns regarding tx duration, too short or too long NACFC 2015 Symposium 5, Thurs. Oct 8. Towards Rational Management of Pulmonary Exacerbations: Part III

35 eice Trial Multicenter, randomized, non-blinded trial Started by Johns Hopkins and Univ. of Washington Enrollment began October 2011 Expanded to 14 CF-TDN Centers October 2012 Home monitoring (symptoms and spirometry) vs. Standard Care Hypothesis: Earlier identification of acute pulmonary exacerbation will improve lung function in CF (FEV 1 )

36 Did the Home Monitoring Trigger Visits? Yes Total no. of alarms in intervention arm: 524 No. in intervention arm with at least 1 alarm: 97 (72%) Answer Response at Workshop to alarms 26, Session III % of subjects with at lease 1 alarm Abstract : Saturday 11: % of total alarms Acute visit Phone contact No patient contact 4 Distribution of Types of Alarms Did Home Monitoring affect the outcome? Decreased FEV1 Worsening symptoms Both decreased FEV1 and worsening symtpoms

37 What Studies are Underway or Starting Soon?

38 2015 Studies CFTR Modulators: 1. ProQR PQ Novartis QBW PTC GD-021 (Ataluren) 4. Bayer BAY /17020 (Riociguat) 5. Vertex (Homozygous) 6. Vertex (Heterozygous) 7. Vertex (Residual Function) 8. Vertex (Gating Mutation) 9. Vertex (Pediatric Efficacy) 10. Vertex (Advanced Lung Disease) 11. Nivalis SNO4 12. Nivalis SNO6

39 2015 Studies CFTR Modulators: QR-010, QBW251, SNO4, SNO 6, Riociguat, Ataluren, Vertex 661 (106, 107, 108, 109), Vertex 809 (106, 109) Anti-Inflammatories 1. Celtaxsys CTX-443) 2. Corbus JBT-101 (Resunab)

40 2015 Studies CFTR Modulators: QR-010, QBW251, SNO4, Riociguat, Ataluren, Vertex 661 (106, 107, 108, 109), Vertex 809 (106, 109) Anti-Inflammatories: Celtaxsys, JBT-101 Anti-Infectives: 1. OPTIMIZE-IP-12 (TOBI with/without Azithromycin) 2. IGNITE (Gallium) 3. Novoteris Inhaled NO

41 2015 Studies CFTR Modulators: QR-010, QBW251, SNO4, Riociguat, Ataluren, Vertex 661 (106, 107, 108, 109), Vertex 809 (106, 109) Anti-Inflammatories: Celtaxsys, JBT-101 Anti-Infectives: OPTIMIZE, IGNITE, Inhaled NO Restore Airway Surface Liquid: 1. Parion P-1037 CLEAN-CF (ENaC Inhibitor) 2. SHIP (Hypertonic Saline in Preschoolers)\ 3. Mannitol

42 2015 Studies CFTR Modulators: QR-010, QBW251, SNO4, Riociguat, Ataluren, Vertex 661 (106, 107, 108, 109), Vertex 809 (106, 109) Anti-Inflammatories: Celtaxsys, JBT-101 Anti-Infectives: OPTIMIZE, IGNITE, Inhaled NO Restore Airway Surface Liquid: Parion P-1037, SHIP, Mannitol Nutrition: 1. Anthera SOLUTION (Liprotomase) 2. Alcresta

43 2015 Studies CFTR Modulators: QR-010, QBW251, SNO4, Riociguat, Ataluren, Vertex 661 (106, 107, 108, 109), Vertex 809 (106, 109) Anti-Inflammatories: Celtaxsys, JBT-101 Anti-Infectives: OPTIMIZE, IGNITE, Inhaled NO Restore Airway Surface Liquid: Parion P-1037, SHIP, Mannitol Nutrition: Anthera SOLUTION, Alcresta Observational: 1. PROSPECT 2. TOBI Podhaler 3. GOAL e2 4. Validation of Predictive Sputum Biomarkers 5. Longitudinal MBW/LCI

44 2015 Studies CFTR Modulators: QR-010, QBW251, SNO4, Riociguat, Ataluren, Vertex 661 (106, 107, 108, 109), Vertex 809 (106, 109) Anti-Inflammatories: Celtaxsys, JBT-101 Anti-Infectives: Optimize, IGNITE, Inhaled NO Restore Airway Surface Liquid: Parion P-1037, SHIP, Mannitol Nutrition: Anthera SOLUTION, Alcresta Observational: PROSPECT, TOBI Podhaler, GOAL e2, Validation of Predictive Sputum Biomarkers, Longitudinal MBW/LCI

45 Number of Clinical Research Studies: Starting Up and Total

46 Growing Demand for Patient Participation

47 What doors need to be opened to improve access for everyone? Manage the steadily increasing needs of our clinical research programs, our care centers and our patients

48 The Role of a Research Network CF Therapeutics Development Network (TDN) est Established the same time as the CFF s Therapeutics Development Program to accelerate the pace of evaluating new therapies in CF Evolved to develop the highest standards for patient safety, best study design and optimum number of patients needed in a trial--enough for a conclusion, but not more than necessary Address the safety and optimization of trials with study sponsors Reflect the research priorities of the CF community

49 Increasing Capacity

50 We have a Worldwide Network CF Centers in many continents and countries North America: Canada and the United States Australia, New Zealand Europe: Clinical Trials Network ECFS CTN, est Expansion to 43 sites 15 countries, 17,500 pts

51 Given this large worldwide network do we have any worries?

52

53 Survey of Research Teams Barriers to Completing Research 0 = not a barrier = most significant barrier RC time 8.3% 8.3% 12.5% 20.8% 25.0% 25.0% Restrictive Protocols Willingness of patients to participate 2.1% 8.3% 12.5% 31.3% 29.2% 16.7% >50% 6.3% 14.6% 22.9% 27.1% 10.4% 18.8% Faculty time 17.0% 12.8% 31.9% 23.4% 6.4% 8.5% 48 TDN sites responded

54 Time to change the perception, end the myth

55 Listening to the CF Community Results of a Survey of 760 adults with CF and Parents of Children with CF

56 Results of Survey with Adults Interest in trials of therapies to treat the basic defect of CF, like ivacaftor combined with other potential drugs Somewhat Not Trials of therapies to treat the serious and chronic problems of CF, like studies of antibiotics and antiinflammatories as well as treatments to improve nutrition and digestion Somewhat Not 85% Very Interested 76% Very Interested N=760 56

57 Sources of Information Data from Patients and Parents: With what member of the CF Care team would you most prefer discussing participation in a clinical trial? RC, 16% 70% prefer discussing CT participation with physician Other, 14% Data from PIs and RCs: Who at your center talks to patients about clinical trials? (check all that apply) Study PI Research Coordinators 94% 96% Clinical Nurses 47% Non-research CF Physicians 37%

58 Participating in Clinical Trials How do you find out about clinical trials? Someone at my care center usually approaches me 23% 62% Have you/your child ever been asked to participate in a clinical trial? (Among those who have not participated, n=385) Don t Know, 7% Yes, 15% I ask my care team 30% 45% I actively research on my own 36% 42% No, 78% Participated in Clinical Trial(s) Hasn't Participated

59 Barriers to Participation What are some reasons that you/your child have not or would not participate in a CF clinical trial? (Adults with CF and Parent) Not qualifying Not being asked Not knowing about them Concern over possible negative side effects Travel distance to study site Scheduling conflicts Time commitment involved Not wanting to go off current medications Not wanting to potentially receive a placebo instead of an active drug Concern about study procedures Don t have enough information Concern over access to trial drug when the trial ends Concern that the study drug isn t going to work = Main reason you have not participated

60 Increasing Comfort with Participation What would make you most comfortable with participating in a CF clinical trial? (Adults with CF and Parents) Knowing closely monitored, pulled out immediately if negative health effects Knowing the risks and benefits of participation Knowing exactly what the trial entails 38% 23% 19% 63% 66% 70% Discussing trial with CF Care Center team 8% 27% Knowing that the cost of participation is covered by the study 5% 28% Hearing about the experiences of others 3% 18% Most Knowing it is okay to leave at any time 2% 16% 2nd Most Knowing family and friends support the decision to participate 1% 9% 3rd Most

61 Conversations about Clinical Research as Part of Clinic Visits What to discuss? What s the latest news in CF research Basics about participating in clinical research Discuss specific trials our patients may ask about Where s quick access to the information to answer the questions? Provide information about specific clinical trials How do you find out about the trials open or upcoming? Dual roles of physician talking about clinical care and research Addressing risk and safety is a HIGH PRIORITY Inform all physicians on team about research studies Physician can introduce the trial before the RC talks with them

62 Dealing with Physician Time Constraints Increasing demands on clinical productivity In future, a greater emphasis on outcomes Benefits of CF quality improvement programs for clinical care Apply the same approach to including discussions about CF research Address recent research accomplishments, put them into context Our patients want to know, What does the result mean? Answer questions about participating in research studies We need more tools to enhance our efficiency when talking about research

63 Improving Web Tools

64

65 Clinical Team Outreach Find on CFF.org

66 To order Clinical Trial Educational Material

67 Current and Future Resources for Centers Update and expand resources for CF care centers New educational and awareness material Resources on safety and DSMB role in clinical trials Send ongoing clinical trial spreadsheet to all centers Send out patient list for specific trials Plan system changes to improve access to information about trials Would be patient-specific Accessible through a secure link for clinicians

68 No CF Center can offer every research study to their patients How to Share

69 Referral Resources for Clinicians, Care Teams Research centers have tools to help structure the referral of patients to other centers for research studies. Seamless integration of clinical care and research Good communication and coordination Development of regional collaborations among Centers Support for Referrals Compensation for the extra work required to provide records, coordinate visits and communication Clinical Research Referral Support Program-CRRSP Will be discussed at Center Directors Meeting on Saturday Details to follow after NACFC

70

71 There s a cure, you know. They just haven t 2012 found 2013 it yet. -Wells 2014 Clark (2007) 2015

72

73 Change is happening now We don t have time to waste. - Parent of a child with CF

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