ASH 2020 | Extramedullary disease in myeloma: updates from ASH 2020

Extramedullary disease is certainly an unmet need in multiple myeloma and the incidence is increasing at later phases of the disease regardless of the success of the treatment, can be observed after cellular therapies, such as CAR-T therapy or allogenic stem cell transplantation. In this congress, we have presented our ICARIA study subgroup analysis, which aims to compare the addition of isatuximab to the backbone of pom-dex, and there were 34 patients within the 300 patient profile of the ICARIA study and 24 of those patients were in the ICARIA pom-dex arm, versus 10 patients in the control arm. And we were able to observe response rate, which is more than equal and more than VGPR in experimental arm, which is 24%, double the control arm. And the median PFS was 4.5 months in the experimental arm versus 1.5 months in the control arm.

So, patients in advanced stages with the median two lines of therapy, this is a good achievement. And during the ASH congress, there were similar studies without a monoclonal antibody and there were two studies for instance, one from the Mayo Clinic, focusing on extramedullary disease on 11 patients using ixazomib plus pom-dex, but the median PFS was only around the range of four months. And in the HORIZON data, which is another important study, earlier Paul Richardson had presented the melflufen-dex data, which has a median PFS of around 2.9 months. And the data was re-presented during ASH and during an oral presentation Dr. Enrique Ocio presented melflufen combinations either with bortezomib or with daratumumab. And during the Q&A session, he mentioned that there were four out of four responses in with the dara-melflufen combination. So I think add monoclonal antibody to one of the backbone, so relapsed treatment seems to favor.

And in as a final point during this ASH meeting, there were CAR-T studies presented again in the context of extramedullary disease. And one was from the CARTITUDE study, and Noopur Raje presented data from the extramedullary disease comparing with the non-EMD and their CR rates around 20%, which is higher than which is unfortunately less than the non-EMD patients about half, but the PFS is 7.9 months which is a good duration. And the Chinese CAR-T study in a similar fashion obtained similar 8 months of PFS, which is a signal for future that bridging therapy with CAR T-cells can be integrated in the treatment of extramedullary disease.