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Acceleron’s Blood Drug Hits Phase 3 Goals, Paves Way for FDA Filing

Xconomy Boston — 

An experimental Acceleron drug for a blood disorder has hit the main goals of a pivotal study, and the company and partner Celgene say they now plan to file for FDA approval next year.

The Acceleron (NASDAQ: XLRN) drug, luspatercept, is being tested as a treatment for myelodysplastic syndrome (MDS), a type of cancer characterized by abnormally formed or improperly functioning blood cells. According to the American Cancer Society, the disorder typically causes anemia—a shortage of red blood cells.

Luspatercept is an injectable, protein-based drug that is believed to regulate the late-stage maturation of red blood cells. The drug is part of a broad partnership between Acceleron, based in Cambridge, MA, and Summit, NJ-based Celgene (NASDAQ: CELG). The partners said Thursday after the market close that the placebo-controlled, Phase 3 study in MDS met the main goal of reducing patient dependence on red blood cell transfusions, posting a “highly statistically significant improvement.” The companies also said the study hit a secondary goal of improving blood transfusion independence of at least 12 weeks during the first 24 weeks of the study. No figures elaborating on those results were released.

On the surface, the preliminary results appear to be a win for Celgene, which has been battered by weaker than expected drug sales and setbacks to compounds in its pipeline. In a research note, Evercore ISI analyst Umer Raffat wrote that the MDS clinical trial had the probability that 30 percent of patients in the treatment group would achieve transfusion independence. Noting the press release worded the results as “highly statistically significant,” Raffat wrote that the drug likely performed better than the 30 percent mark. But he cautioned that it’s important to see the full data, including details about how well the drug works after 12 weeks.

The companies said that data from the clinical trial will be submitted to an unspecified medical meeting later this year. They added that they expect to file for regulatory approval in the U.S. and Europe in the first half of 2019.

The study of luspatercept in MDS is one of three Phase 3 studies for the drug, all of which Xconomy noted as among the top clinical trials to watch for 2018. Two of the luspatercept studies are for beta-thalassemia, an inherited disorder that leads to low levels of hemoglobin in the blood. Data for those studies have yet to be reported.

Here’s more on Acceleron and the origins of its blood drug.

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