Transcript the PFF 2015 slide kit with the key

PFF Summit 2015
Highlights
Washington, US
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.
PFF Summit 2015: From Bench to Bedside
Precision
Medicine
Future
Clinical
Trials
Improving
Care
• The “PFF Summit 2015: From Bench to
Bedside” was held in Washington, DC
November 12-14 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
Precision Medicine for IPF
The preparation of the slide kit and video recording was sponsored by Boehringer Ingelheim and contains
personal opinions from leading ILD experts. PFF was neither author nor reviewer of the content.
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
Genes
• Predict clinical outcomes
• Predict response to therapy, depending on
the phenotype (pharmacogenomics)
Lifestyle
Environment
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.
• Ultimately prevent pulmonary fibrosis by
identifing the at-risk population
4. Noth I, Oral presentation, PFF Summit 2015., 5. Richeldi L, Oral presentation, PFF
Summit 2015. 6. Garcia CK, Oral presentation, PFF Summit 2015. 7. Martinez FJ, Oral
presentation, PFF Summit 2015.
The Importance of Biomarkers for IPF
Biomarkers can1,2
Promising IPF biomarkers3
• Limit treatment heterogeneity
• MMP7 (predicts mortality in IPF
and is consistently increased in
IPF and other ILDs), possibly
best in combination with KL-6
and SP-D1
• Enhance efficiency of future
therapeutic developments
• Identify new and highly
promising therapeutic targets
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.
• PBMC gene expression (52
gene signature predicts
outcome in 5 cohorts)4
• BAL gene expression (predicts
outcome of IPF in 3 cohorts)5
4. Herazo-Maya JD, et al. Sci Transl Med 2013;5:205ra136. 5. Prasse A., et al.
Abstract. ICLAF 2014.
The Future of IPF Clinical Trials
The preparation of the slide kit and video recording was sponsored by Boehringer Ingelheim and contains
personal opinions from leading ILD experts. PFF was neither author nor reviewer of the content.
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 (NCT01417156)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 2015. 2. Ogura T, et
al, ERJ 2015;45:1382–1392. 3. ClinicalTrials.gov. NCT01417156.
4. Collard HR., et al. ERJ 2015;46:243–249. 5. Brown K, Oral presentation, PFF
Summit 2015.
Including patients’ priorities in clinical trials
Which symptoms matter most to
patients with IPF?1,2
Patient-reported outcomes (PROs)
are key4,5
• Cough
• HRQoL (as an overall measure)
• Shortness of breath
• Reporting key symptoms and activities of
daily living
• Fatigue
In addition, patients suffer from the inability to
perform day-to-day activities
Monitoring the physical capacity rather than physiological measures: The continuous-scale physical
function performance test (CS-PFP) was shown to be
reliable and valid for IPF8-9
1. FDA. Public Meeting on IPF Patient-Focused Drug Development
(PDF). 2. Woodcock J, Oral presentation, PFF Summit 2015. 3.
FDA Guidance for Industry. Patient-reported outcome measures.
2009. 4. Russell A-M, et al. BMC Med 2015;13:240.
• Further measures need to be developed
together with patients and clinicians and by
collecting patient data6,7
“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
5. Wilson H, Oral presentation, PFF Summit 2015. 6. Danoff SK, Oral presentation, PFF
Summit 2015. 7. www.pcori.org 8. Olson, AL. Oral presentation, PFF Summit 2015. 9.
Olson AL, et al. Expert Rev Respir Med 2015;9:361–367.
Improving Care
The preparation of the slide kit and video recording was sponsored by Boehringer Ingelheim and contains
personal opinions from leading ILD experts. PFF was neither author nor reviewer of the content.
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;183:788–824 2. Brown K, Oral
presentation, PFF Summit 2015. 3. Wells AU, et al. Sarcoidosis
Vasc Diffuse Lung Dis 2015;32:28–35.
4. Fell, CD. Oral presentation, PFF Summit 2015. 5. Flaherty KR, et al. Am J Respir Crit
Care Med 2004;170:904–910.
Approved Treatments for IPF: New Insights
Nintedanib
Long-term
• 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
No head-to-head data: Data
shown in this slide are from
independent sources.
1. Kaye M, et al. Poster. PFF Summit 2015. 2. Richeldi L, et al.
NEJM 2014. 3. Lancaster L, et al. Poster. PFF Summit 2015.
Pirfenidone
• Consistent safety and tolerability profile
demonstrated in an integrated analysis of
cumulative data from 5 clinical trials3
Approved Treatments for IPF: New Insights
Nintedanib
Real-world
• 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
No head-to-head data: Data
shown in this slide are from
independent sources.
1. Noth I, et al. Poster. PFF Summit 2015. 2. Cottin V, et al. Poster.
PFF Summit 2015.
Pirfenidone
•
•
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
Approved Treatments for IPF: New Insights
Nintedanib
Subgroup analyses
Pirfenidone
• Similar benefit of nintedanib on disease
progression in patients with FVC ≤50%
and >50% predicted at baseline1
(INPULSIS®-ON)
(pooled data: CAPACITY/ASCEND)
• 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%3
• 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 months4
(pooled data: CAPACITY/ASCEND)
No head-to-head data: Data
shown in this slide are from
independent sources.
1. Wuyts WA, et al. Poster. PFF Summit 2015. 2. Richeldi L, et al.
Poster. PFF Summit 2015.
• Pirfenidone may reduce the risk of allcause mortality5
(pooled data and meta analysis: CAPACITY/ASCEND)
3. Glassberg M, et al. Poster. PFF Summit 2015. 4. Nathan SD, et al. Poster. PFF
Summit 2015. 5. Nathan SD, et al. Poster. PFF Summit 2015.
Non-Pharmacological Therapy for IPF
Pulmonary rehabilitation1-2
Oxygen Therapy2-4
Palliative Care5-7
• Can be used to improve physical
and psychological aspects
• O2 therapy is very important
to patients, but also has
limitations
• Few IPF patients are referred
to palliative care, most of
those who are, are referred
late in the course of disease
• Long-term data is missing
• Needs to become more widely
available and tailored to the needs
of patients with IPF
• Direct data on long-term
therapy is missing and
clinical trials are needed
1. Holland AE, et al. Respir Int Rev Thorac Dis 2015;89:89–99. 2.
Holland AE. Oral presentation, PFF Summit 2015. 3. Raghu G, et
al. Am J Respir Crit Care Med 2011;183:788–824.
• Clinical research needed
• Should become part of
standard IPF management
4.FDA. Public Meeting IPF Patient-Focused Drug Development (PDF). 5. Lindell KO,
Oral presentation, PFF Summit 2015. 6. Lindell KO, et al. Chest 2015;147:423–429.
7. Lindell KO, et al. Poster, PFF Summit 2015.
Abbreviations
The preparation of the slide kit and video recording was sponsored by Boehringer Ingelheim and contains
personal opinions from leading ILD experts. PFF was neither author nor reviewer of the content.
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
Acknowledgments
The preparation of the slide kit and video recording was sponsored by Boehringer Ingelheim and contains
personal opinions from leading ILD experts. PFF was neither author nor reviewer of the content.
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
Graphics: Sascha Tkacz
© Boehringer Ingelheim International GmbH, 2016