Curis (CRIS) Conference Call Summary Company Overview - Curis is at a pivotal stage with a focus on a single drug, an IRAK4 and FLT3 inhibitor, primarily targeting Non-Hodgkin Lymphoma (NHL) and Acute Myeloid Leukemia (AML) [2][3] - The company has a promising pipeline and is conducting studies funded by the NIH in solid tumors [2] Key Drug Insights - The drug is being studied as an add-on therapy to BTK inhibitors, which block the BCR pathway, while Curis's drug targets the TLR pathway, potentially leading to higher response rates and complete remission [3] - Current proof-of-concept data in Primary CNS Lymphoma (PCNSL) shows an overall response rate (ORR) of 63% in a small patient population, compared to a historical ORR of 39% for BTK inhibitors [4][5] Clinical Development and Regulatory Strategy - Curis is pursuing accelerated approval based on a single-arm study, with pivotal status accepted by both the FDA and EMA [6][8] - The company aims to enroll between 45 to 60 patients, targeting an ORR of 20% to 22% to meet regulatory requirements [7][8] - Enrollment is expected to take 12 to 18 months, with plans for a confirmatory randomized study against ibrutinib [9][10] Challenges and Considerations - The ultra-rare nature of PCNSL presents challenges in patient recruitment, with a goal of one patient per site per year across 30 sites globally [9] - There is uncertainty regarding regulatory expectations, particularly concerning overall survival (OS) benefits in single-arm trials [11][12] Expansion into CLL - Curis is initiating a new trial in Chronic Lymphocytic Leukemia (CLL), which represents a larger market opportunity compared to PCNSL [25] - The company aims to combine its drug with BTK inhibitors to enhance efficacy and address unmet needs in CLL treatment [26][27] - The focus will be on patients who are on BTK inhibitors but have not achieved complete remission (CR) [30] Market Dynamics and Competitive Landscape - The CLL market is valued at approximately $11 billion, with BTK inhibitors being the standard of care [25] - Curis's approach aims to provide a time-limited treatment option, addressing the side effects associated with BTK inhibitors and offering a potentially better treatment paradigm [28][44] Clinical Endpoints and Future Directions - The company is considering minimal residual disease (MRD) negativity as a potential endpoint for regulatory approval, reflecting a shift in clinical trial design [32] - Curis plans to present interim data on its CLL trial by mid-year, with expectations of seeing early signs of efficacy [38][39] AML Development - Curis is also looking to combine its drug with azacitidine in AML, aiming for higher response rates and MRD negativity [46][47] - The focus will be on patient tolerability and regimen adjustments to ensure effective long-term treatment [47] Conclusion - Curis is strategically positioned to leverage its unique drug mechanism in both NHL and CLL, with a clear focus on regulatory pathways and market opportunities. The company is actively engaging with regulatory agencies to ensure alignment on clinical endpoints and trial designs, while also addressing the challenges of patient recruitment in rare diseases.