7:00 am Check-In & Breakfast

7:55 am Chair’s Opening Remarks

  • Fernando Martinez Chief of Pulmonary and Critical Care, Weill Cornell Medical College

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

  • Adam Freund Founder and Chief Executive Officer, Arda Therapeutics

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

  • Jason McCarthy Scientific Operations Manager, Masonic Medical Research Laboratory

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

  • Imran Alibhai Chief Executive Officer, Tvardi Therapeutics
  • David Rowlands Global Project Leader, early Respiratory and Immunology Research and development, AstraZeneca

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

  • Matthew Thomas Head of Respiratory Exploratory Innovation, Boehringer Ingelheim

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

  • Kjetil Ask Director, Translational Research & Data Sciences, Novartis AG

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

  • Bhavika Kaul Assistant Professor, Medicine, University of California at San Francisco School of Medicine

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

  • Imre Noth Professor - Medicine, University of Virginia School of Medicine

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?

  • Carmel Nanthakumar Director - Clinical Development & Scientific Fellow, GlaxoSmithKline Plc

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

  • Jim Wu Founder & Chief Executive Officer, Ark Biosciences

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

  • Joe Garcia Founder & Chief Executive Officer, Aqualung Therapeutics

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.

4:45 pm Chair’s Closing Remarks

  • Fernando Martinez Chief of Pulmonary and Critical Care, Weill Cornell Medical College

5:00 pm End of Day Two – Goodbye’s & ‘Until Next Year’s’