Agios Pharmaceuticals

Agios Pharmaceuticals has halted development of Tebapivat for the treatment of sickle cell disease after a phase‑2 study showed insufficient differentiation. The 12‑week study with 59 patients produced response rates of 43.8% (2.5 mg), 47.1% (5.0 mg) and 29.4% (7.5 mg) versus 33.3% in the placebo arm; hemoglobin improvements and tolerability were consistent with the expected mechanism of action. As a result, the stock fell by around 14%. Agios is now focusing on mitapivat, which is under priority review at the FDA; the PDUFA date is 1 November 2026. » More on de.investing.com

The FDA has accepted the supplemental new drug application (sNDA) for Mitapivat to treat sickle cell disease for accelerated review and granted Priority Review; the PDUFA date is November 1, 2026. The designation shortens the review period to six months, potentially speeding a market launch. BofA and Truist simultaneously raised their price targets, after which the stock rose about 3.9% in premarket trading. Agios holds more than $1 billion in cash and cash equivalents and is moving closer to its 52-week high. » More on de.investing.com

In the 52‑week double‑blind period of the phase‑3 RISE UP study, 40.6% of mitapivat patients achieved a hemoglobin response versus 2.9% on placebo; the mean Hb increase among responders was 1.6 g/dl. Mitapivat reduced the proportion of transfusion‑dependent patients by 41.1% (relative) and the average number of red blood cell units transfused per patient by 55.9%. Responders also experienced fewer annualized pain crises and hospitalizations and clinically meaningful improvements in fatigue, while the study met its primary endpoint (reduction in annualized pain crises versus placebo) but showed no overall difference in patient‑reported fatigue score. Mitapivat demonstrated a favorable tolerability profile; Agios has submitted a supplemental application to the FDA and the stock is being discussed in the market. » More on de.investing.com