Agios Pharmaceuticals’ stock $AGIO jumped more than 19% on Monday morning after the company announced its oral candidate for the blood disorder thalassemia reduced patients’ need for transfusions in a Phase 3 trial.
Executives spelled out plans to file for US approval by the end of 2024. The drug, mitapivat, is already approved as Pyrukynd for another blood disorder called pyruvate kinase deficiency (PKD). Earlier this year, Agios announced a win in a separate Phase 3 trial for thalassemia patients who don’t require regular blood transfusions. Its marketing application will include both patients who depend on transfusions and those who don’t.
“Our commercial organization is laser-focused on building upon the infrastructure established through our current launch in PK deficiency to prepare for a potential US launch of mitapivat in thalassemia in 2025,” CEO Brian Goff said on a call with investors Monday morning.
The latest readout in transfusion-dependent patients measured transfusion reduction response, or whether patients achieved a 50% or greater reduction in transfused red blood cell units and a reduction of two or more transfused red blood cell units over any consecutive 12-week period. Of the patients who received mitapivat, 30% achieved a transfusion reduction response, compared to 13% of patients on placebo (p=0.0003).
Pyrukynd generated $26.8 million in 2023 sales, up from $11.7 million in 2022. If approved in thalassemia, it would compete with Bristol Myers Squibb’s subcutaneous treatment Reblozyl, bluebird bio’s gene therapy Zynteglo and Vertex and CRISPR Therapeutics’ gene therapy Casgevy. However, those drugs are only approved to treat transfusion-dependent beta-thalassemia. Agios tested its candidate in patients with alpha- or beta-thalassemia, and in both transfusion-dependent and transfusion-independent patients. Agios’ medical director Jeremie Estepp also touted its convenient oral dosing.
“With mitapivat being an oral administered therapy, this means true freedom from the clinic,” Estepp said on the investor call.