Release Date: February 19, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Q: For the myelofibrosis phase 3 trial, what reduction in Total Symptom Score (TSS) would be statistically significant, and how does this compare to previous trials? A: Reshma Rangwala, Chief Medical Officer & Head of Research, explained that they expect a meaningful improvement in absolute TSS, with a potential delta of 3-4+ points compared to ruxolitinib alone. Previous phase 3 trials have shown statistical significance with a narrower margin, suggesting a 2-point difference could be significant. This guides their powering assumptions for the ongoing phase 3 century trial.
Q: Can you provide more details on the discussions with the FDA regarding the endometrial cancer study and the focus on the pMMR population? A: Reshma Rangwala noted that discussions with the FDA were prompted by the evolving treatment landscape, including new therapies like checkpoint inhibitors. The FDA recommended focusing on the pMMR population, which represents 80% of endometrial cancer patients, as they benefit less from checkpoint inhibitors. The FDA is aware of the promising data from the [Sciendo] subgroup, which supports this focus.
Q: How are patient baseline profiles tracking in the Century study, and what are the expectations for discontinuation rates and dose reductions? A: Reshma Rangwala stated that patient profiles are consistent with previous trials, including phase 1 and other contemporary phase 3 trials. The study is blinded, so specific data on discontinuations and dose modifications are not available, but they are tracking as expected.
Q: Regarding the absolute TSS endpoint for the Century study, why is fatigue excluded, and how will the FDA assess this? A: Reshma Rangwala explained that fatigue is excluded from the primary analysis of absolute TSS, consistent with previous trials like the original comfort trials for Ruxolitinib. The FDA is aligned with this approach, and fatigue can be included in future analyses if needed.
Q: What are the characteristics of the dMMR population not eligible for checkpoint inhibitors, and how did they perform in the [Sciendo] study? A: Reshma Rangwala clarified that the dMMR population's ineligibility is due to comorbidities, not tumor characteristics. The [Sciendo] study suggested that p53 status is the key factor for efficacy with Selinexor, with robust improvements observed regardless of MMR status.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
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