The CARTITUDE-1 study investigated the BCMA-targeting CAR T-cell product called cilta-cel. It’s a dual targeting molecule or cell product. It was evaluated earlier in this CARTITUDE-1 study in relapsed/refractory late-stage patients. In this study 97 patients were enrolled. Patients were very advanced disease, median lines of therapy were six. A very resistant disease population – 88% of patients were triple class refractory, 42% had penta-refractory disease, almost a quarter of the patients had high-risk disease. So a very advanced patient population were treated with a single treatment of cilta-cel.
The results were presented and published earlier which led to its approval for commercial purposes, both in the United States and in Europe and Asian countries. What I’m presenting now is the final results or update of this study.
So overall response rate is incredible, it’s 98% with 83% of patients achieving a complete remission or stringent complete remission. The critically new data is that now the median PFS has been reached, which is 35 months, and the median duration of response is 34 months, which is incredibly high for the type of patients we treated. With the standard of care treatment we would expect responses or the PFS in the range of, at the most, 9-12 months overall survival, the PFS would be actually 3-4 months. So a very effective treatment.
We find that patients who get deeper responses have a longer PFS. So if you look at 30-month PFS it’s 75% for patients who had a sustained MRD negativity, so they’re doing exceedingly well. Overall survival has not yet been reached but at 30 months the overall survival is 63%, so very, very promising results.
An important finding is that this outcome, PFS and also the responses, do not correlate with persistence of CAR T-cells and/or expansion, which is a little bit different than in general what we believe.
So, overall this study clearly highlights that a single infusion of CAR T-cell provides deep and durable responses.
Now, this is going to change how we treat myeloma; it already has because it’s commercially available. But there are studies being done to bring it forward, earlier. The CARTITUDE-4 study showed that compared to standard of care treatment, cilta-cell given as a single infusion provides much better outcome, even in earlier stage patients, patients between one and three lines of prior treatment. So it is going to come early and it might end up becoming a mainstay of a consolidation regimen for treatment for even newly diagnosed myeloma.