(RTTNews) - Agios Pharmaceuticals Inc. (AGIO) a commercial-stage biopharmaceutical company, on Tuesday announced the discontinuation of the Phase 2 trial evaluating Tebapivat for Sickle Cell Disease, as the drug failed to improve on currently available therapies with respect to safety and tolerability.
Sickle cell disease is a rare, inherited blood disorder caused by the production of abnormal hemoglobin that disrupts the ability of red blood cells to carry oxygen throughout the body. As a result, red blood cells become rigid and sickle-shaped, leading to chronic hemolytic anemia, vaso-occlusion, and a cascade of severe and life-threatening complications, including long-term damage to the lungs, kidneys, and cardiovascular system.
Tebapivat is an oral pyruvate kinase (PK) activator designed to enhance energy produced by red blood cells by increasing ATP levels required for sugar metabolism. The Phase 2 randomized, double-blind, placebo-controlled study conducted on 59 participants attempted to established that the drug demonstrated a meaningfully differentiated profile compared to available PK activators.
While the trial met the primary endpoint of exhibiting a hemoglobin response in 43.8% of patients in 12 weeks, the safety and tolerability of the drug was consistent with that of previously approved sickle cell disease treatments.
Considering the lack of meaningful differentiation to justify further development of tebapivat, the company chose to discontinue the trial yet highlighted the positive results validating PK activation as a viable option to treat sickle cell disease.
On July 7, 2026, the FDA accepted Agios' supplemental new drug application (sNDA) for Mitapivat in treating sickle cell disease. The prescription drug user fee act (PDUFA) date has been set at November 1, 2026, at which point the FDA may release its verdict on the sNDA.
AGIO is currently trading at $38.28, down 4.54%.
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