Agios Achieves Success in Phase III Thalassemia Trial

Agios Pharmaceuticals, a company specializing in rare diseases, has announced that it has achieved a significant milestone in its Phase III clinical trial for a drug called pyrukynd, also known as mitapivat. The trial focused on adults suffering from non-transfusion-dependent alpha- or beta-thalassemia and successfully met its primary endpoint.

The primary endpoint was defined as a hemoglobin response, which is an increase of 1g/dl or more in average hemoglobin levels from weeks 12 to 24. The drug demonstrated a substantial improvement over a placebo, with 42.3% of the patients on mitapivat showing a response, compared to only 1.6% in the placebo group. This resulted in a highly significant p-value of less than 0.0001.

While the secondary endpoints included changes in the fatigue subscale score and average hemoglobin concentration from baseline, specific figures for these were not disclosed. Nevertheless, the positive results of the late-stage study led to a noticeable increase in Agios Pharmaceuticals' stock price, rising by over 9% in the morning of the announcement.

Agios' Chief Medical Officer, Sarah Gheuens, highlighted the importance of these results, stating that they bring the company closer to providing a meaningful treatment option for adults with non-transfusion dependent alpha- or beta-thalassemia. The company is also planning to release results from its ENERGIZE-T trial, which focuses on thalassemia patients who regularly undergo blood transfusions, in the middle of 2024. Following this, Agios intends to seek regulatory approval for mitapivat as a treatment for thalassemia by the end of the year.

It's worth noting that there are currently no approved oral treatments for non-transfusion-dependent thalassemia patients globally. This condition is associated with significant health risks if untreated, and it represents over half of the clinically significant forms of thalassemia, indicating a considerable need for effective treatments.

Additionally, mitapivat has already been approved by the FDA in February 2022 for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. The drug has also been a significant contributor to Agios Pharmaceuticals' revenue, generating $7.4 million in the third quarter of 2023.

The successful trial and potential approval of mitapivat could mark a significant advancement in the treatment of non-transfusion-dependent thalassemia, offering hope to patients who currently have limited treatment options.