Reports Q3 revenue $12.9M, consensus $10.61M. Cash, cash equivalents and marketable securities as of September 30, were $1.3B vs. $1.5B as of December 31, 2024. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND commercial launches in thalassemia and sickle cell disease, advance existing clinical programs, and opportunistically expand its pipeline through both internally and externally discovered assets. “As we approach year-end, we remain focused on our two key PYRUKYND milestones – the potential U.S. approval in thalassemia and the topline results from the RISE UP Phase 3 trial in sickle cell disease. Our recent engagements with these communities have underscored the urgent need for innovation and PYRUKYND’s potential to address critical gaps in care for these serious and life-threatening diseases,” said CEO Brian Goff. “We continued strong execution across our rare disease portfolio in the third quarter, including the completion of enrollment in our Phase 2b tebapivat trial for lower-risk MDS.”
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