Bristol Myers Squibb, Acceleron rack up second OK for Reblozyl, onto blockbuster status
Reblozyl, the anemia drug Celgene paid a modest $25 million upfront to partner on 9 years ago, brought Bristol Myers Squibb one of only two new drug approvals in 2019. Merely five months later, it’s taking home another supplemental OK expected to catapult sales into blockbuster territory.
The FDA has approved Reblozyl (luspatercept) as a treatment for anemia in a subset of adults with lower-risk myelodysplastic syndromes. Specifically, it’s limited to patients who have failed an erythropoiesis stimulating agent and require 2 or more red blood cell units over 8 weeks. These MDS patients are further characterized by ring sideroblasts, where iron accumulates in the mitochondria of red blood cells, rendering them dysfunctional.
Bristol Myers and partner Acceleron emphasized the historic nature of the drug — an erythroid maturation agent — being “the first new treatment option in over a decade for patients with MDS who require red blood cell (RBC) transfusions and have failed an erythropoiesis stimulating agent.”
A fusion protein, Reblozyl boosts red blood cell growth by targeting TGF-beta proteins.
Transfusions can cause complications such as iron overload, site reactions and infections, said Guillermo Garcia-Manero, chief of the MDS section at MD Anderson, in a statement.
“In our current environment, we are reminded of the significant burden frequent blood transfusions can have on individuals and the healthcare system,” he added.
That’s the same proposition emphasized by the FDA’s oncology chief Richard Pazdur upon sanctioning Reblozyl for transfusion-dependent beta-thalassemia. Analysts have long anticipated follow-up OKs in MDS in order to reach the $2 billion consensus peak sales estimate.
In second-line MDS, late-stage data showed 38% of patients went 8 weeks without needing a blood transfusion and 28% managed 12 weeks without one.
At eight times the market size, “MDS is a much bigger opportunity for Reblozyl in the US, and we model peak US sales of $537M in 2L MDS and $1B in 1L MDS (positive data expected in late ’21 or early ’22),” Cowen’s Yaron Werber previously wrote in a note.
Dubbed COMMANDS, the frontline trial would pit Reblozyl head to head against erythropoiesis stimulating agents and measure red blood cell transfusions throughout 24 weeks.
If the numbers pan out, which he is optimistic about, Werber projects peak global sales of $3.3 billion based on “very conservative assumptions.”