By Mark Terry
Celgene Corporation and Acceleron Pharma indicated their Phase III clinical trial of luspatercept in beta-thalassemia hit its primary endpoint of erythroid response. Acceleron’s shares rose about 11 percent in after-market trading at the news. Celgene stock climbed a little less than two percent.
Luspatercept is a first-in-class erythroid maturation agent (EMA). The companies believe it regulates late-stage red blood cell maturation. The two companies have developed the drug jointly. Their collaboration began seven years ago when the drug was called ACE-536. At that time, Celgene paid Acceleron $25 million to work together, and milestone payments totaled about $217 million.
The BELIEVE phase III trial showed a highly statistically significant improvement in the primary endpoint of erythroid response, defined as at least a 33 percent cut from baseline in red blood cell (RBC) transfusion burden with a reduction of at least two units during a defined period of 12 consecutive weeks, from weeks 13 to 24, compared to placebo. The drug also met all key secondary endpoints of statistically significant improvements in RBC transfusion burden from baseline of at least 33 percent decrease during the weeks 37 to 48 period, and at least a 50 percent reduction from week 13 to week 24, at least a 50 percent decrease from weeks 37 to 48, and a mean change in transfusion burden from weeks 13 to 24.
“For decades, the management of beta-thalassemia in adults has been limited to transfusions and iron chelation,” said Jay Backstrom, Celgene’s chief medical officer, in a statement. “Reduction of transfusion burden represents an important step forward for patients with this rare and debilitating blood disease. We thank the patients, as well as their families and physicians, for their participation in the BELIEVE study.”
At the same time, Celgene and Acceleron announced that luspatercept met the primary and key secondary endpoints in the MEDALIST clinical trial. MEDALIST is a Phase III, randomized, double-blind, multi-center trial that looked at the efficacy and safety of the drug compared to placebo in patients with IPSS-R very low, low or intermediate risk myelodysplastic syndromes (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.
The companies plan to present data at a future medical meeting his year as well as regulatory applications in the U.S. and Europe in the first half of 2019.
This news comes at a time when Celgene would benefit from a win. The last year has been marred with a number of missteps. In late 2017, it dropped a late-stage Crohn’s disease drug and two clinical trials associated with it. Much more troublesome in many ways, was when the U.S. Food and Drug Administration (FDA) issued a Refusal to File letter regarding its New Drug Application (NDA) for its multiple sclerosis drug ozanimod. For a company of Celgene’s size and experience, that was unexpected.
Celgene threw its California subsidiary, Receptos, under the bus on that one, saying that executives should have done a better job of integrating its operations after it bought it in 2015 for $7.2 billion. Nadim Ahmed, Celgene’s president of hematology and oncology, after saying it was Celgene’s responsibility, went on to say, “I think that 99 percent of folks at Celgene wouldn’t have submitted, but we had Receptos out on the West Coast and, for whatever reason, the decision was made to submit. We learned a lesson of humility and that when you do an acquisition it’s better to be more integrated rather than be completely away from the mothership.”