Agios Pharmaceuticals (AGIO) investor relations material

Key milestones and regulatory updates

• Approval and launch of mitapivat (PYRUKYND) for thalassemia marked a major milestone, with ongoing expansion into sickle cell disease and other indications over the next 12–18 months.

• Phase 3 RISE UP study in sickle cell disease showed strong hemoglobin improvement but did not meet statistical significance for vaso-occlusive crisis reduction; regulatory discussions with the FDA are ongoing, with updates expected after receipt of meeting minutes.

• Additional pipeline progress includes Phase 2 studies for tebapivat in sickle cell disease and myelodysplastic syndrome (MDS), with data readouts anticipated in the first and second half of the year.

• Early-stage programs include a Phase 1 study in polycythemia vera and development for phenylketonuria (PKU).

• Plans are in place to expand approvals into pediatric populations for both thalassemia and sickle cell disease.

Commercial strategy and market dynamics

• Initial launch of mitapivat in thalassemia has seen strong physician and patient enthusiasm, with 44 prescriptions in the first 4–5 weeks and positive payer feedback.

• The commercial opportunity is driven by both transfusion-dependent and non-transfusion-dependent thalassemia patients, with a gradual launch trajectory expected as non-transfusion-dependent patients scale up over time.

• U.S. market is expected to be the primary revenue driver, with global opportunity estimated at $1 billion, though ex-U.S. penetration will be slower due to access and reimbursement dynamics.

• The product’s oral administration and favorable tolerability profile are seen as key differentiators, especially in MDS and thalassemia.

• REMS certification for prescribers is streamlined and not expected to be a barrier to adoption.

Competitive landscape and pipeline outlook

• Competitor data from a similar molecule (etavopivat) is expected soon but will not alter current regulatory or commercial plans.

• Tebapivat’s future in sickle cell disease will depend on Phase 2 data, regulatory outcomes for mitapivat, and the evolving competitive environment.

• For MDS, an attractive drug profile would combine efficacy, tolerability, and oral administration to keep patients out of clinics.

• AG-236 (TMPRSS6 siRNA) targets polycythemia vera, aiming for durable hematocrit control and long dosing intervals; Phase 1 data in healthy volunteers is expected in 2026.

• The company is leveraging its rare disease commercial platform to establish new standards of care in targeted indications.