Katie McCauley
Director & Head of Portfolio Strategy & Fibrosis Research Novartis AG
Katie McCauley, PhD, is Head of Portfolio Strategy and Discovery Biology in Disease Area X (DAx) at Novartis, where she leads early research at the intersection of exploratory biology, in vitro pharmacology, and portfolio strategy. She has extensive expertise in fibrosis and respiratory diseases, including IPF, integrating human biology, mechanistic insight, and translational models to drive target prioritization and project progression. In her role, she guides early portfolio decision making and resource allocation, with oversight over assay development across DAx and accountability for identifying and advancing emerging scientific opportunities. Katie earned her PhD at Boston University and completed postdoctoral training at Harvard Medical School, specializing in lung epithelial biology and fibrosis.
Seminars
Defining Value, Differentiation & De-Risking in a Competitive Landscape
As the IPF field advances beyond proof-of-concept and into an era of increasing mechanistic diversity, the question is no longer whether IPF drug development is viable, but what makes an asset truly investable.
With new mechanisms emerging and the bar for differentiation rising, clarity on what constitutes a compelling therapeutic story has never been more critical. This panel brings together investors, strategic pharma, and M&A leaders to define what “good” looks like, and what it doesn’t, in today’s IPF landscape.
Key Discussion Points:
- What Builds Conviction Early? What efficacy signals, durability data, mechanistic rationale, safety profile, and translational evidence are required at preclinical and early clinical stages to generate real investor confidence?
- Differentiation vs. Incrementalism? In a field moving beyond simply slowing FVC decline, what level of clinical impact, biomarker validation, or combination rationale is needed to stand out from the noise?
- De-Risking, Timing & Strategic Positioning? How do investors evaluate first-in-class versus best-in-class strategies? What role do trial design, regulatory strategy, management credibility, and partnership readiness play in investment and acquisition decisions?
For decades, the bleomycin model has served as the primary tool for evaluating antifibrotic candidates in pulmonary fibrosis research. Yet as the field has matured, it has become increasingly clear that reliance on a single preclinical model is insufficient to capture the biological complexity, chronic progression, and therapeutic responses observed in human disease.
Key Discussion Points:
- The strengths and limitations of traditional models, including bleomycin, and how they fit within a broader preclinical evaluation strategy
- How emerging platforms such as human lung tissue models, organoids, and PCLS are being used to complement in vivo studies and improve translational relevance
- Detecting safety signals and drug–drug interactions early in development to better inform clinical trial design and regulatory discussions
Despite decades of research, therapeutic development for IPF and progressive pulmonary fibrosis remains constrained by limited target diversity and high clinical attrition. With advances in single-cell technologies, imaging, and human tissue models, the field is rethinking how to discover and validate the next generation of fibrosis targets.
Discussion Topics Include:
- Reassessing Historical Pathways: Revisiting canonical mechanisms such as TGF-β signaling, integrins, and inflammatory mediators, what have we learned, and do these pathways still hold untapped therapeutic potential?
- Emerging Target Opportunities: Exploring epithelial repair, fibroblast heterogeneity, immune modulation, and regenerative biology as potential avenues for next-generation interventions.
- Bridging Preclinical Insights to Clinical Readouts: How preclinical findings and cellular-level insights can guide trial design, biomarker selection, and patient stratification; challenges of relying solely on lung function endpoints (FEV/FVC); and leveraging multi-omics, spatial biology, and imaging technologies to accelerate target validation and predict meaningful clinical outcomes.
- Forward-Looking Questions: Which cellular or molecular readouts best predict clinical efficacy? Can we design trials focused on fibrosis biology rather than aggregated lung function metrics? How can new technologies inform both target discovery and endpoint selection for more translatable therapies?
