Key Quotes/Transcript from our KOL expert call on thalassemia and AQVESME

研报英文原文证据摘录

Key Quotes/Transcript from our KOL expert call on thalassemia and AQVESME

Accessible version

US Biopharmaceuticals

Key Quotes/Transcript from our KOL

expert call on thalassemia and AQVESME

Industry Overview

We hosted a high-volume thalassemia KOL on AQVESME 12 June 2026

We hosted Dr. Sujit Sheth, Chief of Pediatric Hematology-Oncology and Vice Chair of Equity

Clinical Research at Weill Cornell Medicine, for an expert call on Agios’ AQVESME launch United States

in thalassemia. Dr. Sheth runs what he described as likely the largest single-center Biopharmaceuticals

thalassemia program in the US, with ~250 patients split roughly evenly between Alec W. Stranahan

transfusion-dependent and non-transfusion-dependent disease; he also participated in Research Analyst

Agios’ ENERGIZE studies. We discussed early AQVESME uptake, patient selection, BofAS+1 646 743 2109

REMS and reimbursement friction, durability expectations, and how mitapivat may fit alec.stranahan@bofa.com

alongside transfusions, luspatercept, and gene therapy. Maintain Buy on AGIO, $40 PO.

Non-transfusion patients clearest launch opportunity Abbreviations:

KOL: key opinion leader “The non-transfusion dependent patients really until AQVESME was approved had really

MRI: magnetic resonance imagingno good options... So now we have AQVESME and that's really the only thing that we

PK: pyruvate kinasehave that is disease modifying to offer them.”

PKD: pyruvate kinase deficiency

“I think it's going to have a major impact on my management of non-transfusion PRO: patient reported outcome

dependent patients… At max, I would say 30% to 40% of my non-transfusion dependent REMS: Risk Evaluation and

patients would be on AQVESME.” Mitigation Strategy

SCD: sickle cell disease