Reports Q3 revenue $9.0M, consensus $9.46M. Cash, cash equivalents and marketable securities as of September 30, 2024, were $1.7B compared to $806.4M as of December 31, 2023. 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 launches in thalassemia and sickle cell disease, advance existing programs, and to opportunistically expand its pipeline through both internally and externally discovered assets. “We had a strong quarter, marked by several important advancements across our pipeline. We completed enrollment of our Phase 3 RISE UP study of mitapivat in sickle cell disease, on our way to sharing topline results in late 2025. Our Phase 2b study of tebapivat in lower-risk MDS was initiated, and we received orphan drug designation from the FDA to support the development of tebapivat in this indication,” said CEO Brian Goff. “Our cash position was further strengthened by the receipt of $1.1B in payments. This will allow us to maintain this great momentum and fuel our next phase of growth, building towards a franchise with multi-billion-dollar potential. We remain focused on progressing our promising clinical programs to address the critical needs of rare disease patients and look forward to sharing our progress in the coming months.”
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