Luspatercept hits primary and key secondary goals in Phase III study in MDS

Results from a Phase III, randomized, double-blind, multi-center clinical study (MEDALIST) showed that luspatercept achieved a highly statistically-significant improvement in the primary endpoint of red blood cell (RBC) transfusion independence of at least 8 consecutive weeks during the first 24 weeks compared to placebo, according to the drug’s developers. Celgene (Nasdaq: CELG) and Acceleron Pharma (Nasdaq: XLRN).

Following the announcement last Thursday, Acceleron’s shares leapt 26.55% to $43.00 in pre-market trading this morning, while Celgene was up a modest 1.75% at $78.00. Analysts have suggested that luspatercept could achieve peak sales of $2 billion a year.

For Celgene, luspatercept represents the first bit of good news for the beleaguered large-cap biotech damaged by a string of clinical and commercial setbacks. Luspatercept on its own is not enough to right the listing Celgene, but it’s a start, commented Adam Feuerstein on STAT.