ASCO 2016

CPX-351 improves outcomes for refractory AML

Dr Jeffrey Lancet - H. Lee Moffitt Cancer Center, Tampa, USA

We conducted a phase III randomised study in older adults with higher risk AML comparing CPX-351 versus the traditional daunorubicin plus cytarabine induction regimen and studied overall survival as the primary endpoint along with several other secondary endpoints including response rates, event free survival and toxicity.

What kind of outcomes were you finding?

The primary efficacy results revealed that CPX-351 induced a statistically significant longer survival than patients who received 7 3 and there was about a 31% overall reduction in the risk of death throughout the course of the study. We also found that CPX treated patients had a higher complete response and overall response rate compared to patients who were treated with 7 3 so we felt that this represents a significant advance in the treatment of up front AML in older patients with high risk disease.

7 3 being the standard of care at the moment, this sounds like a significant advance on that.

I think it is. I think it represents an important step forward. It’s certainly not the final answer, I think we still have work to do to improve outcomes in these patients but beginning with this step it gives us a new platform to advance the overall standard of care to the point where we can launch future trials, hopefully with this compound for this disease type and others.

Speaking of future trials, this was, of course, the phase III results, are there any plans to move forward with this or follow up with any further studies?

At this point we’re doing some secondary studies to better identify patients who may have the best chance for response or patients who are likely to be resistant to this kind of therapy. So we have several correlative studies in the works right now. There are other studies that are being planned for the use of this agent in different subsets of acute myeloid leukaemia as well as other types of leukaemia.

You mentioned this was in older patients, could this be dependent on pre-treatment or is this working in naïve as well?

We’re in the process right now of evaluating those data. We feel that in general the efficacy is improved, based on the use of this drug, in patients with higher risk AML, that includes patients with myelodysplastic syndrome who had been through prior therapy for that disease as well. But we’re still ironing out the details as to which patients specifically benefitted and which risk factors may affect the outcomes.

This study highlighted the importance of optimising drug delivery in that we have a lot of new targeted, very novel therapies that are out there being tested. But this is a fairly simple concept where we took two very well-known drugs and combined them in an optimal ratio that would allow for better cell kill. So it’s really all about the drug delivery system with this compound and we feel that this type of technology and this type of approach improves drug delivery and that that could be applicable across many different treatment types, not just chemotherapy.