Acceleron, Celgene Drug Shines in Phase 3 Test in Rare Blood Disorder

Xconomy Boston — 

A drug from Celgene and Acceleron Pharma has met the main goals of a late-stage test as a treatment for a rare blood disorder, the second set of positive results that the partners have released for the drug in as many weeks.

The latest announcement covered use of the drug, luspatercept, as a treatment for beta-thalassemia, which is an inherited disease that causes patients to have low levels of oxygen-carrying red blood cells. The disease is currently treated with monthly transfusions to prevent anemia, a shortage of red blood cells. Patients also need iron chelation therapy to flush out the excess iron from the transfusion treatments.

Celgene (NASDAQ: CELG) and Acceleron (NASDAQ: XLRN) said late Monday that patients treated with the drug in a Phase 3 study showed a “highly statistically significant” improvement meeting the main goal of reducing the need for red blood cell transfusions by at least 33 percent compared to a placebo. The companies say full data from the study will be presented at medical meeting later this year.

Luspatercept, developed by Cambridge, MA-based Acceleron, is believed to regulate the late-stage maturation of red blood cells. The protein-based drug, which is administered by injection, is meant to boost red blood cells production in patients who have rare types of anemia.

Acceleron and its Summit, NJ-based partner tested luspatercept in two beta-thalassemia Phase 3 studies that Xconomy listed among this year’s clinical trials to watch. The companies also tested the drug in a separate Phase 3 study for myelodysplastic syndrome (MDS), a disorder that leads to abnormally formed or improperly functioning red blood cells. In late June, Celgene and Acceleron announced that the drug met the goals of the late-stage MDS study. With positive results in hand for both MDS and beta-thalassemia, the partners now say they plan to file for approval in the U.S. and Europe in the first half of next year.

Here’s more on the discovery and development of luspatercept and Acceleron’s partnership with Celgene.

Image of red blood cell by Wikimedia user Rogeriopfm via a Creative Commons license