Agios’ Thalassemia Drug Mitapivat Faces Liver Injury Concerns Despite Second Phase III Success

Positive Phase III Results:
Agios Pharmaceuticals announced positive results from the Phase III ENERGIZE-T study of mitapivat in adult patients with transfusion-dependent alpha- or beta-thalassemia, achieving primary and all key secondary endpoints13.

Liver Injury Concerns:
Despite the success, concerns over liver injury associated with mitapivat have emerged, leading to a significant drop in Agios' stock price4.

Regulatory Applications:
Agios has filed regulatory applications for mitapivat in the U.S., European Union, Kingdom of Saudi Arabia, and United Arab Emirates, incorporating data from both the ENERGIZE and ENERGIZE-T studies1.

Liver Monitoring:
The company has proposed monthly monitoring of liver tests for the first six months of treatment with mitapivat due to the risk of hepatocellular injury1.

Clinical Significance:
Mitapivat is the first oral, disease-modifying treatment to demonstrate efficacy in transfusion-dependent alpha- and beta-thalassemia, offering a potential breakthrough for patients with limited treatment options13.

Regulatory Timeline:
Agios expects to receive regulatory approval for mitapivat in thalassemia by the end of 2024 or early 20255.

Sources:

1. https://investor.agios.com/news-releases/news-release-details/agios-presents-positive-results-phase-3-energize-t-study

3. https://www.thalassemia.org/agios-pharmaceuticals-announces-phase-3-energize-treatment-for-thalassemia-patients/

4. https://endpts.com/agios-stock-drops-after-disclosing-new-liver-related-safety-warning-in-filing-to-fda/

5. https://investor.agios.com/news-releases/news-release-details/agios-announces-key-anticipated-2024-milestones-across-rare