Agios Pharmaceuticals: Mitapivat’s Accelerated Approval Path and De-Risked Sickle Cell Opportunity Support Buy Rating

H.C. Wainwright analyst Emily Bodnar maintained a Buy rating on Agios Pharma today and set a price target of $65.00.

Emily Bodnar has given his Buy rating due to a combination of factors related to Agios Pharma’s lead asset, mitapivat, in sickle cell disease. The company secured FDA alignment to pursue an accelerated approval filing after a positive pre-sNDA meeting, with the agency accepting a plan that leverages the drug’s hemoglobin benefit despite the prior trial not showing a statistically significant reduction in pain crises. This path is especially meaningful in light of the significant treatment gap created by Oxbryta’s withdrawal, and Bodnar views mitapivat’s established safety record in pyruvate kinase deficiency and thalassemia as a key de-risking element for regulatory review.

In addition, Agios has already submitted a confirmatory trial proposal that is expected to use a new, more clinically relevant primary endpoint, likely centered on organ function or hospitalization metrics rather than vaso-occlusive crisis rates. Management indicates that this incremental study will not materially increase operating expenses versus 2025 levels, which supports the financial attractiveness of the program. Bodnar also sees upcoming milestones—finalizing the confirmatory study, filing the sNDA, and future tebapivat Phase 2 data in sickle cell disease—as important value drivers that reinforce her positive stance on the stock.