Early results of trial testing tebapivat in SCD expected later this year
Enrollment now complete at 31 study sites in North America and Europe
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A small clinical trial testing Agios Pharmaceuticals’ oral therapy candidate tebapivat against a placebo for easing symptoms in people with sickle cell disease (SCD) has completed enrollment, the company announced.
Agios expects to report early results from the Phase 2 trial later this year, according to a company press release announcing its latest financial results and providing business updates. Among the highlights: “Phase 2 tebapivat trial in sickle cell disease fully enrolled; topline results expected in second half of 2026.”
Taking place at sites in North America and Europe, the trial (NCT06924970) is testing three doses of tebapivat — 2.5, 5, and 7.5 mg — versus the placebo for 12 weeks, or about three months. The trial now involves an estimated 56 SCD patients, ages 16 and older.
A genetic disorder, SCD is caused by mutations that lead to the production of an irregular form of hemoglobin, the protein in red blood cells that carries oxygen through the bloodstream. This mutated protein is prone to forming clumps in red blood cells, especially under low-oxygen conditions, which deform red blood cells into the sickle shape that gives the disease its name.
The deformed blood cells are more likely to be destroyed, which can cause anemia, and they are also prone to getting stuck in blood vessels, which can lead to painful vaso-occlusive crises among patients.
Tebapivat aims to reduce blood cell sickling, reduce symptoms
Tebapivat is designed to activate an enzyme called pyruvate kinase (PK) in red blood cells. The PK enzyme plays key roles in cellular energy production, and by boosting its activity, the investigational therapy aims to increase energy stores in red blood cells.
This would help stabilize these cells’ membranes and prevent mutated hemoglobin from clumping up, with the ultimate goal of reducing red blood cell sickling and easing SCD symptoms.
To be eligible for the trial, participants had to have anemia, or low levels of hemoglobin. Anyone with more than 10 pain crises in the year prior to the study was excluded.
The study’s main goal is to assess the proportion of participants who experience an increase in hemoglobin levels of at least 1 g/dL from study week 10 through 12, compared with values at the study’s start.
Secondary goals include changes in blood levels of hemoglobin, markers of red blood cell destruction, and energy-related molecules, as well as changes in patient-reported measures of pain, fatigue, and quality of life. The therapy’s safety and pharmacological properties will also be evaluated.
Agios’ first-generation PK activator, mitapivat, has so far shown positive results in SCD trials. Data from the Phase 3 RISE UP clinical trial (NCT05031780) demonstrated that the study met its main goal, with mitapivat proving superior to a placebo at boosting hemoglobin levels in adolescents and adults with SCD.
Based on those data, Agios plans to meet with the U.S. Food and Drug Administration in the coming months to discuss trial results, after which it plans to submit an application seeking the therapy’s approval for SCD in the country. The company said it will provide an update once the meeting has taken place.
Mitapivat is already approved in the U.S. for treating people with two other genetic disorders marked by problems with red blood cells. Specifically, it is sold under the name Pyrukynd for pyruvate kinase deficiency, and was recently approved under the name Aqvesme for thalassemia.
“In 2026, we are focused on driving a high-impact U.S. launch of Aqvesme, expanding our PK activation franchise into additional high-value indications such as sickle cell disease … and advancing our promising early-stage pipeline to further diversify across [blood-related diseases] and other rare diseases,” said Brian Goff, CEO of Agios.
