- Celgene and partner Acceleron Pharma on Friday announced positive results from the Phase 3 MEDALIST study of the anemia drug luspatercept in patients with low-to-intermediate risk myelodysplastic syndromes.
- Patients on luspatercept showed a highly statistically significant improvement in red blood transfusion independence of at least eight consecutive weeks during the first 24 weeks when compared with placebo. The study also hit key secondary endpoints.
- Further clinical data will be presented at an upcoming medical meeting. The partners intend to file the drug for approval in the U.S. and Europe in the first half of 2019.
Celgene has been hoping at least one of its near-term late-stage assets would pan out. But a refusal-to-file letter from the Food and Drug Administration for its multiple sclerosis drug ozanimod, and the failure of its pricey Crohn's disease drug mongersen left it with a shakier pipeline — headlined by luspatercept.
Celgene gained nearly 4% in trading on Friday after the luspatercept results were announced. The company has been struggling to diversify its portfolio. Nearly 60% of its revenue comes from blood cancer drug Revlimid (lenalidomide), while three other products make up a majority of the remaining revenues. Deals struck with generic drugmakers could mean copycat versions of the multiple myeloma drug could begin entering the market as early as 2022, putting more than $8 billion in sales in jeopardy.
Celgene made two major acquisitions in the first weeks of this year, picking up Impact Biomedicines for $7 billion and then Juno Therapeutics Inc. for $9 billion. While the high-profile — and high pricetag — deals added to its mid- to late-stage pipeline, investors were left looking for more.
Luspatercept could be the jolt the big biotech needs. Beyond the MEDALIST study, Celgene and Acceleron have plans to begin a Phase 3 study, dubbed COMMANDS, in first-line, lower-risk patients with myelodysplastic syndromes — this is the patient population that is most important for this drug and will be most closely watched. The pair also have a Phase 3 trial ongoing in beta-thalassemia, which is expected to report out in the next two to three weeks.
Evercore ISI analyst Umer Raffat pointed out in a note to clients that investors were really hoping to see an effect size greater than 30% in the MEDALIST trial. While Celgene didn't release any of the numbers at this time, the trial arm of the study was powered to show that effect and Raffat believes the company's statement language indicates the drug did even better.
"We continue to really like Celgene at these levels, and today's update checks the box on first of three key events for stock to recover. Next two: re-file ozanimod and settle on Revlimid," added the analyst.