- Celgene reported more positive data for its investigational blood disorder drug luspatercept on Monday, announcing the treatment met primary and key secondary endpoints of a Phase 3 study evaluating it in patients with transfusion-dependent beta-thalassemia.
- Overall, patients treated with Celgene's drug needed fewer red blood cell (RBC) transfusions. Compared to placebo, significantly more patients in the experimental group demonstrated at least a 33% reduction from baseline in RBC transfusion burden and a minimum reduction of two units during the 12 consecutive weeks between week 13 and week 24 of the study.
- Previous investigations showed beta-thalassemia patients tolerated luspatercept fairly well, with most adverse events being mild to moderate. Celgene said adverse events seen in this latest study were generally consistent with those earlier results. The big biotech and partner Acceleron Pharma plan to file the drug for approval in the U.S. and Europe during the first half of 2019.
Wall Street expected luspatercept to pass its latest clinical test. The drug already scored in a Phase 2 trial that showed it had positive effects on both hemoglobin levels for beta-thalassemia patients who weren't transfusion-dependent as well as transfusion requirements for those who were. And last month, it notched a "highly statistically significant improvement" in red blood transfusion independence for patients with low-to-intermediate risk myelodysplastic syndromes (MDS).
Though not exactly surprising, the new beta-thalassemia data are nonetheless important for Celgene's future.
Investors have chided the big biotech for quite some time because of its narrow portfolio and over-reliance on Revlimid (lenalidomide), a blood cancer drug that accounts for about 60% of company revenue. While it doesn't look like Revlimid will face generic competition anytime soon, analysts have pegged an $11 billion to $12 billion erosion of the franchise over the long-term.
Luspatercept, as one of Celgene's few near-term growth drivers, could therefore be a potential salve. Investment bank Jefferies pegs a $1 billion to $2 billion market opportunity for the drug across the combined indications of beta-thalassemia and MDS. Leerink, meanwhile, anticipates peak risk-adjusted global sales of $2.6 billion in 2025.
Perhaps more importantly, the fresh data — along with the potential for another product to hit the market before 2020 — could restore some investor optimism. Over the last year, Celgene stock has fallen more than 36%.
"In our view, these results should help turnaround sentiment on [Celgene's business development and] pipeline and potentially lead to stock appreciation as current levels are close to [Celgene]'s no pipeline value," Jefferies analyst Michael Yee wrote in a July 9 investor note.
Shareholders, however, seem to be taking a wait-and-see approach. The company's stock was up less than 2% Tuesday morning, trading at $85.21 per share at market's open. Acceleron shares, conversely, were up about 5% to $49.70 apiece.
Celgene said it will unveil more complete data on luspatercept for both the beta-thalassemia and MDS indications later this year.