7:00 am Check-In & Breakfast
7:55 am Chair’s Opening Remarks
Exploring the Current State of Play in the Progressive Pulmonary Fibrosis Field
8:00 am Examining the Underlying Mechanism & Patients Subject to the Therapeutic Potential of NK T-Cell Targeting
Synopsis
• Exploring the scientific rationale behind NK T-cell targeting for the treatment of Idiopathic Pulmonary Fibrosis and supporting preclinical evidence
• Exploring clinical trial design, optimal patient populations and dosing strategies for clinical success
• Positioning NKT-cells on the landscape of currently available and developing therapeutics and what opportunity does targeting of the innate pathways present
8:30 am ADC-Mediated Depletion of Pathogenic Fibroblasts Ameliorates Pulmonary Fibrosis
Synopsis
• Discussing the therapeutic potential of depleting pro-fibrotic fibroblasts
• Using a single-cell-based target discovery engine to target very specific pathogenic sub-populations of cells
• Developing biologics, including antibody-drug conjugates, to deplete target-positive cells
• Evaluating biologics in preclinical models of pulmonary fibrosis to quantify the sensitivity and specificty of cell depletion and the effect on disease-relevant endpoints.
9:00 am Targeting Pro-Fibrotic Macrophages to Reverse Pulmonary Fibrosis with Small RNA Inhibitors
Synopsis
• Exploring the emerging understanding of pro-fibrotic macrophages as key drivers of disease progression
• The concept of small RNA inhibitors and their potential in regulating macrophage activity
• Examining the preclinical evidence for reversing IPF with macrophage reprogramming
9:30 am Exploring Nanotechnology for Myofibroblast-Specific Inhibition of Rho Kinase-MRTF-SRF Pathway
Synopsis
• Exploring the role Rho Kinase-MRTF-SRF pathway in myofibroblast activity and fibrotic lung diseases
• Defining nanotechnology-based myofibroblast inhibition as a novel and promising approach
• Exploring the new possibilities of nanotechnology and the promise it holds for targeted drug delivery in the lungs
Looking Beyond Monotherapy & The Current Standard of Care to Combining Existing & Emerging Therapeutics for a Combination Approach
10:00 am Panel Discussion: Unravelling the Rationale Behind Combination Therapy & Critical Design & Development Considerations for Emerging Therapeutic Candidates
Synopsis
• Exploring the current limitations of single agent therapeutics and the scientific rationale behind targeting multiple pathways and processes for enhanced efficacy in halting fibrosis
• Critical considerations into clinical trial design in combination therapy including patient population selection based on specific signatures predicting a better response to combination
• Defining appropriate endpoints for combination therapy trials beyond traditional single-agent measures moving from slowing the rate of decline to faster and more apparent measures of improvement
10:30 am Morning Break
Track 1: Emerging Biology & Early Translation
Chair: Marilyn Glassberg, Professor & Chair, Department of Medicine, Loyola University
Removing the Idiopathic from IPF: Exploring the Underlying Etiology of Pulmonary Fibrosis to Uncover Novel & Effective Therapeutic Targets
11:00 am Leveraging Novel Approaches Including Spatial Transcriptomics & Spatial Proteomics to Uncover Novel & Meaningful Therapeutic Targets
Synopsis
• Examining the numerous cell classes underrepresented from the isolation and separation process of single cell sequencing
• Moving past expression levels to enhanced insights into cellto-cell interactions not possible with scRNA-seq
• Utilizing spatial transcriptomics and spatial proteomics to validate target concepts increasing confidence and transforming the drug discovery process
11:30 am Progressive Subclinical Familial IPF: Moving Toward Secondary Prevention
Synopsis
• Integrating demographic, radiologic, and genetic data to identify high-risk ILA profiles and determine a risk score of acquiring a progressive phenotype
• Unravelling the cellular and biochemical processes driving ILA-to-fibrosis transition to identify targets for preventative therapies
• Designing of therapies aimed at halting or reversing ILA progression before fibrosis development
12:00 pm Gauging the Molecular Signatures of Progressive Pulmonary Fibrosis Patients Across Disease Progression
Synopsis
• Examining the physiological lung heterogeneity before disease and its implications on disease biology
• Enhancing understanding of molecular changes including temporal progression and spatial progression to identify the critical players driving the transition
• Exploring metabolomic and epi-proteomic signatures for a deeper understanding of environmental impact
Track 2: Late Translation & Clinical
Fernando Martinez, Chief of Pulmonary & Critical Care, Weill Cornell University
Assessing Clinically Relevant Patient Cohorts to Influence Chosen Patient Populations in Clinical Trials
11:00 am Unveiling Genetic & Proteomic Signatures for Targeted ILD/ILA Patient Selection
Synopsis
• Examining the ILD/ILA heterogeneity causing overlapping clinical features and complex differential diagnoses
• Identifying genetic and proteomic signatures to stratify patients based on their specific disease subtype
• Examining the implications on clinical trial design and endpoints of specific subgroups of ILD patients
• Balancing inclusivity to capture sufficient patient numbers with selecting the most relevant population for the targeted therapy
11:30 am Pioneering PRECISION with the First Pharmacogenetic Trial in IPF to Move Beyond “One-SizeFits-All” & Optimize Treatment Effectiveness
Synopsis
• Exploring the first-ever pharmacogenetic trial for IPF investigating how a specific genetic variant influences response to treatment
• Examining trial design to facilitate patient participation and reflect a real-world clinical setting
• Reviewing the potential to establish a new paradigm for personalized treatment in IPF by tailoring therapy based on individual genetic profile
12:00 pm Exploring the Interface Between Idiopathic Exploring the Regulatory Considerations for Clinical Trial Design when Evaluating Treatments for Idiopathic Pulmonary Fibrosis & Progressive Pulmonary Fibrosis
Synopsis
• With the evolution of the field and new combinatory approach should we be looking at IPF and PPF in the same light?
• What are the regulatory distinctions for IPF and PPF trial design? Should these be pursued as separate indications and run in parallel?
• Is clinical design a cut and paste entity from IPF trials or are there special considerations to navigate potential pitfalls?
12:30 pm Lunch Break
Track 1: Emerging Biology & Early Translation
Emerging Biology & Early Translation
1:30 pm From Genetic to Epigenetic, Proteomic, Environmental & Beyond: How Heterogenous Really is IPF?
Synopsis
• Exploring the varied cellular pathways, genetic and epigenetics influences, and environmental factors contributing to diverse disease manifestations
• Examining the different cell types and proteomics involved in disease progression
• Deciphering how many clinically relevant patient segmentations there are to distil patient populations informing personalized medicine
2:00 pm Blocking the Matrix Stiffness Signal: From Patient to Clinical Candidate
Synopsis
• Interrogation of progressive patients biopsies, a unique approach to fibrosis
• Exploring the matrix stiffness and its untapped promise as an alternative approach in IPF
• Leveraging tissue biomarkers to guide dose selection
Track 2: Late Translation & Clinical
Clinical Development Strategies for Translating Positive Proof of Concept Results into Effective Therapeutics
1:30 pm Exploring the Evolution of Clinical Trial Designs from The Early Nintedanib & Pirfenidone Days to Present to Avoid Underpowered Studies
Synopsis
• Examining the evolution of clinical trial design from early and successful design of trials
• Exploring the new ‘placebo population’ and the implications of incorporating current standards of care to baseline
• Considerations of current therapeutics on trial design and analysis to work with the new naive
• Exploring the growing challenge of predicting disease behaviour in IPF patients
2:00 pm Unveiling Progress: Clinical Trial Design & Strategic Insights from Phase 2 Clinical Program in IPF
Synopsis
• Exploring the intricacies of the clinical trial design for AK3280 in IPF, including patient selection criteria, endpoint selection, and the overall study methodology
• How the trial design addresses the unique challenges posed by IPF in the Chinese patient population, showcasing adaptations made to enhance efficacy and relevance
• Insights into the regulatory landscape in China for IPF therapeutics, and innovative strategies employed to streamline regulatory processes
2:30 pm Afternoon Break
3:00 pm Chairs Recap
Synopsis
• This afternoon session brings together the chairs of two dedicated tracks to offer a comprehensive overview of the latest advancements in pulmonary fibrosis research, spanning discovery to application.
Examining the Future Directions of the Pulmonary Fibrosis Landscape to Bring Better Drugs to Patients Faster
3:15 pm Examining OATD-01- A First-in-Class Chitnase Inhibitor’s MOA in Lung Sarcoidosis to Unveil Potential in Other ILDs
Synopsis
• Dive into the mechanism of action a first-in-class chitnase inhibitor
• Explore the potential of OATD-01, currently in Phase 2, to address a wider range of ILDs
3:45 pm Unveiling TTI-101 as a Novel Approach in Pulmonary Fibrosis & Interstitial Lung Disease Treatment
Synopsis
• Exploring TTI-101, an orally bioavailable, small-molecule inhibitor of signal transducer and activator of transcription 3 (STAT3) and its implications in IPF and ILD
• Examining the comprehensive Phase 2 multicenter study of TTI-101, explore the study’s scope, objectives, patient selection criteria, endpoint selection, and the study methodology
4:15 pm DAMPening Innate Immunity-Mediated Inflammatory Injury & Fibrosis via the eNAMPT-Neutralizing ALT-100 mAb
Synopsis
• Explore the MOA behind ALT-100 mAb, a monoclonal antibody targeting eNAMPT, to dampen innate immunity-mediated inflammatory injury and fibrosis
• Delve into the interplay between DAMP signaling and innate immunity, highlighting how the inhibition of eNAMPT holds promise in mitigating inflammatory responses implicated in pulmonary fibrosis progression.