Acceleron Pharma plans to seek regulatory approval next year for its investigational drug luspatercept. The treatment hit its Phase III endpoints.
Acceleron Pharma plans to seek regulatory approval next year for its investigational drug luspatercept. The treatment hit its Phase III endpoints as a treatment for patients with myelodysplastic syndrome (MDS), a disorder where the bone marrow does not produce enough healthy blood cells.
Luspatercept, which is being co-developed between Acceleron and Celgene Corporation, achieved a highly statistically significant improvement in the primary endpoint of red blood cell (RBC) transfusion independence of at least eight consecutive weeks of treatment during the first 24 weeks of treatment compared to placebo.
It’s needless to say that the news excited Acceleron investors. Shares of the company’s stock shot up more than 17 percent in premarket trading to $40. Shares of Celgene also rose slightly in premarket trading to $78.01. It was good news for Celgene, particularly following the stunning Refusal to File letter issued by the U.S. Food and Drug Administration (FDA) in February for multiple sclerosis treatment ozanimod.
The Phase III MEDALIST trial evaluated the safety and efficacy of luspatercept versus placebo in patients with IPSS-R very low, low or intermediate risk MDS with chronic anemia and refractory to, intolerant of, or ineligible for treatment with an erythropoietin-stimulating agent (ESA), ring sideroblast-positive and require frequent RBC transfusions. Luspatercept is a first-in-class erythroid maturation agent (EMA) that is believed to regulate late-stage red blood cell maturation
Not only did luspatercept hit its primary endpoint in the Phase III trial, Acceleron said luspatercept also met the key secondary endpoint of “demonstrating a highly statistically significant improvement in RBC transfusion independence of at least 12 consecutive weeks during the first 24 weeks.” Modified hematologic improvement-erythroid (IWG mHI-E), a meaningful secondary endpoint, was also achieved, Acceleron said.
Acceleron Chief Executive Officer Habib Dable said the company was encouraged by the top-line results from the Phase III MEDALIST trial. He added that luspatercept has the potential to “benefit the tens of thousands of patients suffering from low-to-intermediate risk MDS worldwide.”
Data from MEDALIST will be submitted to a future medical meeting in 2018, Acceleron said. The two companies plan to submit regulatory applications in the United States and Europe in the first half of 2019. Adverse events observed in the study were generally consistent with previously published data.
“This result from the Phase III MEDALIST trial demonstrates the potential clinical benefit of luspatercept as an erythroid maturation agent for the treatment of chronic anemia in patients with low-to-intermediate risk MDS,” Jay Backstrom, Celgene’s chief medical officer said in a statement. “Based on these results, we look forward to preparing the dossier for global regulatory submissions and also investigating the clinical potential of luspatercept in ESA-naïve, low-to-intermediate risk MDS patients through the initiation of our Phase III COMMANDS study.”
Dable said Acceleron remains committed to the study of luspatercept in “a range of anemia-related diseases.” In addition to MEDALIST and the planned COMMANDs trial, luspatercept is also being studied in patients with beta-thalassemia (BELIEVE trial). Luspatercept is being studied in two Phase II trials as well, in non-transfusion-dependent beta-thalassemia and in myelofibrosis.
