1. PFF Summit 2015 Highlights Washington, US references
The preparation of the slide kit and video recording was sponsored by Boehringer Ingelheim International GmbH and contains personal opinions from leading ILD experts. PFF was neither author nor reviewer of the content.
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2. PFF Summit 2015: From Bench to Bedside
Future Clinical Trials
Improving Care
Precision Medicine
The “PFF Summit 2015: From Bench to Bedside” was held in Washington, DC November and was organized by the Pulmonary Fibrosis Foundation
Largest pulmonary fibrosis specific conference in the world
More than 700 healthcare professionals, patients, caregivers and industry leaders attended the sessions
2015 PFF event Highlights Newsletter
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3. Precision Medicine for IPF

4. Precision Medicine for IPF
Over the course of the next years, treatment will become tailored to the individual patient2-7
Precision medicine combines1
Precision medicine for IPF2-7
Diagnose and classify IPF through genomic signatures
Predict clinical outcomes
Predict response to therapy, depending on the phenotype (pharmacogenomics)
Ultimately prevent pulmonary fibrosis by identifing the at-risk population
Genes
Lifestyle
Envi-ronment
1. NIH information brochure: Precision Medicine Initiative., 2. Spagnolo P, et al. Curr Opin Pulm Med 2015;21:470–478., 3. Rosas IO, Oral presentation, PFF Summit 2015.
4. Noth I, Oral presentation, PFF Summit 2015., 5. Richeldi L, Oral presentation, PFF Summit Garcia CK, Oral presentation, PFF Summit Martinez FJ, Oral presentation, PFF Summit 2015.
NIH information brochure: Precision Medicine Initiative
Spagnolo P., et al. Curr Opin Pulm Med 2015;21:470–478.
Rosas IO, Oral presentation, PFF Summit 2015
Noth I, Oral presentation, PFF Summit 2015
Richeldi L, Oral presentation, PFF Summit 2015
Garcia CK, Oral presentation, PFF Summit 2015
Martinez FJ, Oral presentation, PFF Summit 2015
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5. The Importance of Biomarkers for IPF
Biomarkers can1,2
Limit treatment heterogeneity
Enhance efficiency of future therapeutic developments
Identify new and highly promising therapeutic targets
Promising IPF biomarkers3
MMP7 (predicts mortality in IPF and is consistently increased in IPF and other ILDs), possibly best in combination with KL-6 and SP-D1
PBMC gene expression (52 gene signature predicts outcome in 5 cohorts)4
BAL gene expression (predicts outcome of IPF in 3 cohorts)5
For the future, it is important to routinely collect patient data and integrate biomarkers into clinical studies and registries
1. Spagnolo P, et al. Curr Opin Pulm Med 2015;21:470–478., 2. Martinez FJ, Oral presentation, PFF Summit 2015., 3. Kaminsky N, Oral presentation, PFF Summit 2015.
4. Herazo-Maya JD, et al. Sci Transl Med 2013;5:205ra Prasse A., et al. Abstract. ICLAF 2014.
Spagnolo P., et al. Curr Opin Pulm Med 2015;21:470–478.
Martinez FJ, Oral presentation, PFF Summit 2015.
Kaminsky N, Oral presentation, PFF Summit 2015.
Herazo-Maya JD., et al. Sci Transl Med 2013;5:205ra136.
Prasse A., et al. Abstract. ICLAF 2014.
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6. The Future of IPF Clinical Trials
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7. Future clinical trials
Nintedanib and pirfenidone combination trials are needed1
A trial investigating the long-term safety and tolerability of nintedanib given in addition to pirfenidone is ongoing (NCT )2,3
All new IPF treatments should be tested in combination or in head-to-head trials (with either nintedanib or pirfenidone at baseline)4
Testing the approved treatments for IPF in other ILDs is a priority5
1. Richeldi L, Oral presentation, PFF Summit Ogura T, et al, ERJ 2015;45:1382– ClinicalTrials.gov. NCT
4. Collard HR., et al. ERJ 2015;46:243– Brown K, Oral presentation, PFF Summit 2015.
Richeldi,L. Oral presentation, Session: Clinical care: New and evolving treatment strategies, PFF Summit 2015
Ogura T., et al. Safety and pharmacokinetics of nintedanib and pirfenidone in idiopathic pulmonary fibrosis. Eur Respir J 2015;45:1382–1392.
ClinicalTrials.gov,
Collard HR., et al. A new era in idiopathic pulmonary fibrosis: considerations for future clinical trials. Eur Respir J 2015;46:243–249.
Brown K, Oral presentation, Session: Clinical care: New and evolving treatment strategies, PFF Summit 2015
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8. Including patients’ priorities in clinical trials
Which symptoms matter most to patients with IPF?1,2
Cough
Shortness of breath
Fatigue
In addition, patients suffer from the inability to perform day-to-day activities
Patient-reported outcomes (PROs) are key4,5
HRQoL (as an overall measure)
Reporting key symptoms and activities of daily living
Further measures need to be developed together with patients and clinicians and by collecting patient data6,7
Monitoring the physical capacity rather than physio-logical measures: The continuous-scale physical function performance test (CS-PFP) was shown to be reliable and valid for IPF8-9
“A PRO is any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.” 3
1. FDA. Public Meeting on IPF Patient-Focused Drug Development (PDF). 2. Woodcock J, Oral presentation, PFF Summit FDA Guidance for Industry. Patient-reported outcome measures Russell A-M, et al. BMC Med 2015;13:240.
5. Wilson H, Oral presentation, PFF Summit Danoff SK, Oral presentation, PFF Summit Olson, AL. Oral presentation, PFF Summit Olson AL, et al. Expert Rev Respir Med 2015;9:361–367.
FDA. Public Meeting on IPF Patient-Focused Drug Development-The Voice of the Patient (PDF).
Woodcock J, Oral presentation, PFF Summit 2015.
FDA Guidance for Industry. Patient-reported outcome measures
Russell A-M, et al. BMC Med 2015;13:240.
Wilson H, Oral presentation, PFF Summit 2015.
Danoff SK, Oral presentation, PFF Summit 2015.
Olson, AL. Oral presentation, PFF Summit 2015.
Olson AL, et al. Expert Rev Respir Med 2015;9:361–367.
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9. Improving Care

10. Diagnosis of IPF
Diagnosing IPF is challenging and many patients do not meet the current diagnostic criteria1-3
Multidisciplinary discussion is the gold standard for diagnosing and treating IPF4,5 and ensures a better overall treatment of patients
1. Raghu G., et al. AJRCCM 2011;183:788– Brown K, Oral presentation, PFF Summit Wells AU, et al. Sarcoidosis Vasc Diffuse Lung Dis 2015;32:28–35.
4. Fell, CD. Oral presentation, PFF Summit Flaherty KR, et al. Am J Respir Crit Care Med 2004;170:904–910.
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11. Approved Treatments for IPF: New Insights
Nintedanib
Pirfenidone
Long-term
No head-to-head data: Data shown in this slide are from independent sources.
An interim analysis of data from INPULSIS®-ON indicated that the effect of nintedanib on slowing disease progression was maintained beyond 52 weeks.1
The safety and tolerability of nintedanib observed in the INPULSIS® trials were confirmed.1,2
Long-term nintedanib treatment (up to 40 months) had a manageable safety and tolerability profile, with no new safety signals identified.1
Consistent safety and tolerability profile demonstrated in an integrated analysis of cumulative data from 5 clinical trials3
1. Kaye M, et al. Poster. PFF Summit Richeldi L, et al. NEJM Lancaster L, et al. Poster. PFF Summit 2015.
Kaye M, et al. Poster. PFF Summit 2015.
Richeldi L, et al. NEJM 2014.
Lancaster L, et al. Poster. PFF Summit 2015.
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12. Approved Treatments for IPF: New Insights
Nintedanib
Pirfenidone
Real-world
No head-to-head data: Data shown in this slide are from independent sources.
Consistent safety and tolerability profile as described in the label (US, post-marketing surveillance)1
Treatment with nintedanib in the real-world clinical setting appeared to have a manageable safety and tolerability profile, with no new safety concerns identified1
Consistent safety and tolerability profile (EU), also in combination with NAC and/or corticosteroids (PASSPORT)2
Patient characteristics and adverse drug reactions were similar in the 3 largest enrolling countries (Germany, France and the United Kingdom).2
1. Noth I, et al. Poster. PFF Summit Cottin V, et al. Poster. PFF Summit 2015.
Noth I, et al. Poster. PFF Summit 2015.
Cottin V, et al. Poster. PFF Summit 2015.
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13. Approved Treatments for IPF: New Insights
Nintedanib
Pirfenidone
Subgroup analyses
No head-to-head data: Data shown in this slide are from independent sources.
Similar benefit of nintedanib on disease progression in patients with FVC ≤50% and >50% predicted at baseline1 (INPULSIS®-ON)
Decline in FVC was similar in patients treated with nintedanib irrespective of dose reductions, treatment interruptions, or dose intensity of ≤90% and >90%2
(pooled data: INPULSIS®)
Similar efficacy outcomes (FVC, 6MWD and dyspnea) for patients with baseline FVC below and above 80% (pooled data: CAPACITY/ASCEND)
Among patients who experienced a >=10% decline in %FVC during the first 6 months of treatment, continued treatment with pirfenidone resulted in a lower risk of %FVC decline or death during the subsequent 6 months (pooled data: CAPACITY/ASCEND)
Pirfenidone may reduce the risk of all-cause mortality5 (pooled data and meta analysis: CAPACITY/ASCEND)
1. Wuyts WA, et al. Poster. PFF Summit Richeldi L, et al. Poster. PFF Summit 2015.
3. Glassberg M, et al. Poster. PFF Summit Nathan SD, et al. Poster. PFF Summit Nathan SD, et al. Poster. PFF Summit 2015.
Wuyts WA, et al. Poster. PFF Summit 2015.
Richeldi L, et al. Poster. PFF Summit 2015.
Glassberg M, et al. Poster. PFF Summit 2015.
Nathan SD, et al. Poster. PFF Summit 2015.
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14. Non-Pharmacological Therapy for IPF
Pulmonary rehabilitation1-2
Can be used to improve physical and psychological aspects
Long-term data is missing
Needs to become more widely available and tailored to the needs of patients with IPF
Oxygen Therapy2-4
O2 therapy is very important to patients, but also has limitations
Direct data on long-term therapy is missing and clinical trials are needed
Palliative Care5-7
Few IPF patients are referred to palliative care, most of those who are, are referred late in the course of disease
Clinical research needed
Should become part of standard IPF management
1. Holland AE, et al. Respir Int Rev Thorac Dis 2015;89:89– Holland AE. Oral presentation, PFF Summit Raghu G, et al. Am J Respir Crit Care Med 2011;183:788–824.
4.FDA. Public Meeting IPF Patient-Focused Drug Development (PDF). 5. Lindell KO, Oral presentation, PFF Summit Lindell KO, et al. Chest 2015;147:423– Lindell KO, et al. Poster, PFF Summit 2015.
Holland AE, et al. Respir Int Rev Thorac Dis 2015;89:89–99.
Holland AE. Oral presentation, PFF Summit 2015.
Raghu G, et al. Am J Respir Crit Care Med 2011;183:788–824.
FDA. Public Meeting IPF Patient-Focused Drug Development (PDF).
Lindell KO, Oral presentation, PFF Summit 2015.
Lindell KO, et al. Chest 2015;147:423–429.
Lindell KO, et al. Poster, PFF Summit 2015.
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15. Abbreviations

16. Abbreviations
6MWD = 6-minute walk distance BAL = bronchoalveolar lavage BALF = bronchoalveolar lavage fluid EU = european union FVC = forced vital capacity HRCT = high resolution computed tomography HRQoL = health-related quality of life ILD = interstitial lung disease IPF = idiopathic pulmonary fibrosis KL = Krebs von den Lungen MDD = multidisciplinary discussion MDT = multidisciplinary team
MMP = matrix metalloproteinase MUC = Mucin NAC = N-acetylcysteine PBMC = peripheral blood mononuclear cell PF = pulmonary fibrosis PRO = patient-reported outcome SNP = single nucleotide polymorphism SP = surfactant protein TERT = telomerase reverse transcriptase TOLLIP = Toll-interacting protein UK = United Kingdom
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17. Acknowledgments

18. Acknowledgments
Boehringer Ingelheim would like to extend a special thank you to our two ILD experts, Prof. Vincent Cottin and Prof. Luca Richeldi for the guidance during this report and their valuable discussions in the PFF 2015 Congress Highlights TV filming.
Boehringer Ingelheim GmbH
Corporate TA Respiratory
Dr. Andrea Sambatti & Dr. Vasiliki Tsagkaraki
Imprint
Production: infill Kommunikation GmbH, Germany
Medical writing: Franziska Frey, Melissa Koch
© Boehringer Ingelheim International GmbH, 2016
Graphics: Sascha Tkacz
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