Explore the Agenda
7:00 am Check in & Light Breakfast
Track 1: Emerging Biology & Early Translation
Workshop A
8:00 am Decoding the Disruptions in Natural Biological Processes to Advance Early Intervention in IPF – Unravelling the Molecular Biology of Immune Cells & Inflammation
Understanding the complex interplay between immune responses, epithelial dysfunction, and regeneration failures is crucial for advancing early intervention strategies in IPF. This workshop will explore the molecular underpinnings of immune cell activity, inflammation, and epithelial repair in disease progression. Through expert insights, we will dissect how disrupted biological processes drive fibrosis and discuss emerging therapeutic approaches targeting immune and epithelial pathways.
Get ready to explore:
Epithelial Dysfunction & Regeneration Failure in IPF
- The molecular biology of epithelial injury and its role in disease progression
- Why failed regeneration is central to fibrosis development
- Emerging biomarkers linking epithelial signatures to disease severity
The Enigmatic Role of Inflammation in IPF
- Understanding the paradox: why anti-inflammatory therapies worsen outcomes
- The potential of immune-targeting therapies and companies exploring this space
Bridging Immunology & Fibrosis in IPF Treatment
- Key immune players: which inflammatory cells are present, and what role do they play?
- How immune modulation could be leveraged for therapeutic intervention
- The next steps in developing immune-based therapies for IPF
Track 2: Late Translation & Clinical
Workshop C
8:00 am Innovating Endpoints in IPF: Including & Beyond FVC to Determine Clinical Efficacy
FVC remains the standard endpoint for IPF clinical trials, yet its variability poses challenges for trial reliability, cost, and regulatory approval. As the field advances, there is a growing need to refine clinical trial endpoints by integrating additional, more predictive measures. This workshop will explore innovative approaches to endpoint selection, incorporating lessons from other respiratory diseases and data-driven strategies to enhance trial sensitivity and efficiency.
Get ready to explore:
Addressing the Limitations of FVC
- Understanding the variability of FVC, its impact on trial design, and the need for complementary or alternative measures to improve accuracy and trial success
Learning from Composite Endpoints in Pulmonary Arterial Hypertension
- Evaluating how composite endpoints, including six minute walk distance, respiratory hospitalizations, and imaging biomarkers, can provide a more robust efficacy assessment
Advancing Clinical Trial Design
- Leveraging quantitative, data-driven approaches to define novel composite endpoints that enhance sensitivity, support regulatory approval, and transform the future of IPF clinical trials
10:30 am Morning Break
Workshop B
11:00 am Genetic & Familial IPF: Uncovering Susceptibility & Resilience to Improve Early Detection & Identify Novel Therapeutic Targets
While genetic predisposition plays a role in IPF, not all individuals with risk factors develop the disease. Understanding both susceptibility and resilience can provide critical insights into early detection and potential therapeutic strategies. This workshop will explore the latest advancements in genetics, familial risk factors, and emerging research on protective mechanisms that could inform new treatment approaches.
Get ready to explore:
Understanding Resiliency: Why Do Some Individuals Avoid IPF Despite Risk Factors?
- Exploring why some high-risk individuals never develop lung disease
- Identifying protective biological mechanisms that could inform new therapies
- Reviewing emerging research on cellular and molecular markers of resilience
Genetics & Familial Risk Factors: Improving Early Detection
- Investigating hereditary risk factors and genetic polymorphisms in IPF
- Leveraging familial studies and at-risk cohort screenings for early diagnosis
- Exploring predictive modeling and polygenic risk scores Beyond Known Genetics: Novel Research & Therapeutic Targets
- Examining new genetic discoveries beyond well-known mutations
- Understanding gene-environment interactions and epigenetics in IPF
- Exploring industry efforts in gene-targeted therapies and prevention
Workshop D
11:00 am Statistical Innovations & Considerations – Optimizing Sample Size, Trial Design & Data Integrity to Streamline Clinical Development
Effective clinical trial design in IPF is constrained by patient scarcity, high costs, and the need for robust statistical methodology to drive confident decisionmaking. This workshop will bring together statistical experts, clinical researchers, and industry professionals to explore how innovative statistical approaches can optimize sample sizes, improve trial efficiency, and mitigate the risks of underpowered studies.
Key discussion points:
Balancing Statistical Rigor with Feasibility in Trial Design
- How can we determine the optimal sample size that maintains statistical power while reducing costs and recruitment timelines?
- What alternative statistical methodologies can be applied to small patient populations to maximize data reliability?
Mitigating the Risks of Small, Underpowered Studies
- Explore historical IPF trial failures due to inadequate proof-of-concept data and lessons learned
- How to design early-phase trials to better de-risk phase three studies while working within financial constraints
Addressing Data Integrity & Outlier Impact
- The role of extreme outliers in phase two datasets— when should they be excluded or accounted for?
- Statistical innovations for improving disease modelling, endpoint selection, and predictive power of early-phase studies
This workshop will provide practical insights into statistical frameworks that enhance the success of clinical trials in IPF, ensuring more efficient study designs that improve the likelihood of regulatory success and commercial viability.
1:30 pm Lunch Break
Setbacks to Solutions Seminar Afternoon
Looking Back at Phase 2/3 Clinical Trial Failures to Pave the Path Forward
2:30 pm Breaking Down the Science: Did the Biology Fall Short?
Scientific missteps in target selection, mechanism validation, and translational predictability often set trials up for failure before they begin. This session will examine the biological assumptions that contributed to past setbacks and how we an improve early-stage decision-making.
- Target Selection & Validation: Were we confident in the mechanism of action before advancing to the clinic? Did preclinical models confirm target engagement and therapeutic relevance?
- Translational Gaps & Dosing Challenges: Did preclinical studies establish that effective doses were achievable in humans? Were PK/PD models predictive of clinical exposure and response?
- Fibrotic Pathways & Patient Variability: How did inter-patient heterogeneity impact response? Did we overlook key immune or regenerative mechanisms that may have influenced outcomes?
3:00 pm Preclinical & Translational Decisions: Did We Build a Strong Enough Case for Success?
Many failed trials reveal weaknesses in preclinical rigor and translational assumptions, including inadequate target engagement studies, incomplete biomarker strategies, or insufficiently powered phase 2 trials. Preclinical Strategy & Target Confidence: Did we truly establish a direct link between the target and IPF pathogenesis? Were preclinical efficacy signals robust, or were they driven by artificial conditions?
- Dosing, PK/PD & Translation to Humans: Was there a clear therapeutic window? Were effective concentrations realistically achievable in patients, or did preclinical studies rely on unrealistic drug levels?
- Early-Stage Trial Design & Biomarkers: How can we optimize early clinical studies to ensure meaningful phase 2 readouts? Are small, underpowered trials leading to misleading efficacy signals that don’t hold up in phase 3?
3:30 pm Clinical Trial Design: Were We Set Up for Success?
A well-designed clinical trial can make or break a promising therapy. This session will explore how trial design choices, endpoints, patient selection, and statistical power, have influenced past outcomes.
- Endpoints & Study Duration: Were phase 2 trials too short or underpowered to detect meaningful effects? Did reliance on FVC as the primary endpoint contribute to misleading signals?
- Patient Selection & Inclusion Criteria: Did we include the right patient populations, or was disease heterogeneity overlooked? Were exclusion criteria too broad or too restrictive?
- Statistical Pitfalls & Misleading Readouts: How often have positive phase 2 results been driven by outliers? Could alternative trial designs, such as adaptive approaches, improve decision-making?
4:00 pm Afternoon Break
4:30 pm Audience Discussion: Learning from Failure – What Should We Do Differently?
This interactive discussion invites all attendees to reflect on the key insights from the seminar and explore practical ways to refine IPF drug development strategies.
- Which preclinical and translational gaps have been most problematic, and how do we address them?
- What changes are needed in early clinical development to improve the likelihood of success in phase 3?
- How can industry and academia collaborate more effectively to de-risk future programs?
This session will provide a platform for open discussion, allowing attendees to share perspectives and generate actionable next steps for improving IPF clinical development.