Question · Q4 2025
Ashleigh Acker questioned the strategic rationale for evaluating R289 in an earlier line, post-ESA or treatment-naive MDS population now, rather than waiting for a registrational trial, and what specific benefit (response rates, durability) Rigel aims to demonstrate.
Answer
Lisa Rojkjaer, EVP and Chief Medical Officer, explained that the current study is in heavily pretreated patients, and Rigel is optimistic that R289's activity may be enhanced in an earlier line of therapy, similar to how other agents like luspatercept and imetelstat generated their data. Raul Rodriguez, President and CEO, added that R289's novel mechanism offers a significant opportunity to improve upon the 18-40% response rates seen with existing therapies, with preliminary data in refractory patients already showing a promising 33% red cell transfusion independence rate at higher doses.
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