Question · Q4 2025
Ashley Aloupis, on behalf of Alexandre Brasil, asked about the strategic rationale for exploring R289 in a less heavily pretreated, post-ESA or treatment-naive lower-risk MDS population now, rather than waiting for a registrational trial, and what specific benefit (response rates, durability) Rigel aims to show in this earlier-line population.
Answer
Lisa Rojkjaer (EVP and Chief Medical Officer, Rigel Pharmaceuticals) explained that the current study population is heavily pretreated, and moving to an earlier line (post-ESA or ESA-ineligible) aligns with how other agents like luspatercept and imetelstat generated their data, with an expectation of even better activity. Raul Rodriguez (President and CEO, Rigel Pharmaceuticals) added that current approved products have limitations (38-40% response rates for luspatercept/imetelstat, 18-20% for HMAs), and R289's different mechanism, showing 33% response in very refractory patients at doses >=500mg, suggests potential for broader and improved benefit in earlier lines.
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