Early results of trial testing tebapivat in SCD expected later this year

A small clinical trial testing Agios Pharmaceuticalsexternal link, opens in a new tab’ oral therapy candidate tebapivat against a placebo for easing symptoms in people with sickle cell diseaseexternal link, opens in a new tab (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 releaseexternal link, opens in a new tab 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)external link, opens in a new tab 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 causedexternal link, opens in a new tab 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 crisesexternal link, opens in a new tab among patients.