Trust favourite Celgene claims win in Medalist trial
US biotech and specialist trust favourite Celgene (Nasdaq: CELG) has reported a positive result in a closely-watched Phase III trial that was being seen as a key step necessary in its rehabilitation with investors. The trial was of the drug candidate luspatercept for treatment of anaemia associated with a type of myelodysplastic syndrome (MDS). Although luspatercept is forecast to achieve blockbuster sales, the outcome of this study had taken on additional importance over and above its commercial impact after a series of trial setbacks and a surprise US regulatory pushback in recent months eroded investor confidence in company.
Celgene reported that the trial, known as Medalist, hit its primary endpoint of demonstrating an improvement in the rate of red blood cell (RBC) transfusion independence of at least 8 consecutive weeks during the first 24 weeks. Luspatercept also met the key secondary endpoint of demonstrating an improvement in RBC transfusion independence of at least 12 consecutive weeks during the first 24 weeks. In both cases, the difference was described as “highly statistically significant”, which would usually be taken to mean a p value of <0.01.
The absolute values of these endpoints and the statistics have not been disclosed, pending publication at a scientific meeting. Although this is normal practice, it is frustrating and particularly so in this case, as it is commercially important for the study to show a clinically significant effect- assumed to be a 8-week transfusion independence rate of >30% – in addition to a statistically significant difference. The wording that implies higher levels of statistical significance is presumably meant to infer that this is the case.
The Medalist study specifically recruited low-to-intermediate risk MDS patients whose bone marrow produces ringed sideroblasts, an abnormal type of red blood cell, where earlier studies suggested its efficacy could be greatest. A second Phase III trial, called “Believe”, is underway in a separate indication of beta thalassemia and is due to report imminently.
Although Celgene’s shares have fallen by almost halved from their peak last year, the company remains one of the four largest components of the Nasdaq biotechnology index and is a top holding for three of the sector specialist trusts. It is the second largest shareholding for International Biotechnology Trust (IBT, 4.8% of NAV) and third largest holding for both Biotech Growth Trust (BIOG, 7.9% of NAV) and BB Biotech (BBB.S, 7.7% of NAV).
Trust favourite Celgene claims win in Medalist trial