Jun. 13 at 2:35 PM
$AGIO EHA 2026 data dump — the numbers that mattered:
Thalassemia (AQVESME, already approved)
• ENERGIZE OLE: Hb response climbed 42.3% → ~60% (72/121) with continued treatment
• ~1/3 of prior non-responders converted to responders over time
• Duration of response more than doubled: 17.9 → 43.6 wks
• High-baseline subgroup (≥9.5 g/dL): 38.9% (7/18) response vs 0% placebo; FACIT-Fatigue +5.1 vs +0.8
• SATISFY (membranopathies): 48% (10/21) Hb response, +1.1 g/dL, hemolysis + iron burden down
SCD (mitapivat, RISE UP Ph3 plenary — sNDA filed)
• Hb response 40.6% vs 2.9% placebo, p<0.0001 — maintained through Wk52
• SCPC pain-crisis co-primary MISSED: 14% RR, p=0.12
• ITT PROMIS-Fatigue also missed (p=0.71)
• Responders saw 26% fewer crises / 34% fewer hospitalizations / 53% fewer ER visits
• Post-hoc transfusion: 41% fewer pts transfused, 56% fewer units
• Safety clean — no treatment-related deaths, >1,300 patient-yrs across 3 anemias
Other
• AG-236 (PV): Ph1 HV done, advancing to late-stage — every-6-mo dosing potential with best in class potential in a
$1 billion TAM.
• AQVESME launch: 242 scripts as of 3/31 with new data presented that more than doubles their persciber bass with NTD patients.
Hb endpoints carried both programs; the clinical co-primary in SCD didn’t, and a case was presented to seek approval on endpoints presented today that showed statistical significance and improved biomarker with longer duration . NFAD / DYOR.
