Mixed results for new leukaemia drugs
pharmafile | December 9, 2014 | News story | Research and Development, Sales and Marketing | AML, Bayer, Nexavar, Qinprenzo, Sunesis Pharmaceuticals, sorafenib
Two trials of new drugs for acute myeloid leukaemia (AML) have shown promising results in younger and older groups of patients with AML.
A Phase II trial of Bayer drug Nexavar (sorafenib) found for the first time, that tyrosine kinase inhibitors – which have been standard therapies for other forms of leukaemia for a number of years – could also be a potential treatment for people with AML.
Researchers led by Christopher Röllig from the University of Dresden in Germany enrolled 267 adult patients aged 18 to 60 years with AML in a Phase II study comparing Nexavar or placebo in addition to standard AML treatment – of two cycles of chemotherapy with daunorubicin, followed by three cycles of high-dose cytarabine chemotherapy.
After three years of follow-up, more people treated with Nexavar met the primary endpoint compared to people who had standard treatment only. Some 40% of people who took sorafenib had experienced three years of event-free survival at the end of the study, compared with 22% of people treated with standard therapy.
At the end of the study median event-free survival was 21 months in the sorafenib group compared with nine months in the placebo group.
There was no difference between the two groups in overall survival, a secondary endpoint, and Rollig says that while the analysis of side effects was not yet complete, there was a higher risk of fever, diarrhoea, liver toxicity, bleeding and rashes.
Rollig adds he was excited about the potential for sorafenib as a new treatment option for people with AML, saying “as far as I am aware, this is the first randomised controlled trial evidence that tyrosine kinase inhibitors work in AML, although we will need a comparator trial to establish this”.
The second trial, a phase three study, looked at the benefits of a new compound for patients whose AML has been treated and returned (relapsed) or is still present after initial treatment (refractory).
This is a difficult to treat group for which there is currently no standard of care of approved treatment regimen. The treatments that are available often cause significant toxicity, and so many companies are investigating new compounds that overcome these limitations.
Researchers looked at Qinprenzo (vosaroxin) by Sunesis Pharmaceuticals in a Phase III study of 711 adults with relapsed or refractory AML. Patients were randomised to either chemotherapy with cytarabine together with either Qinprenzo or placebo.
Those treated with Qinprenzo achieved longer overall survival compared to those treated with placebo (7.5 months compared with 6.1 months), although this was not statistically significant. An analysis of patient aged over 60 years – about two thirds of the study group – did find a significant difference of two months.
The failure to find a significant difference in its primary endpoint was met with disappointment by other researchers in the industry.
Brent Wood, medical director at SCCA Laboratories, says: “There’s clearly benefit for patients aged over 60. But one of the disappointing things was that the magnitude of benefit was not great. I would have loved to have seen a five or six month benefit. But sometimes the research us incremental. We need to keep working because we would want to see more positive results than that.”
But lead researcher Dr Farhad Ravandi, from the University of Texas MD Anderson Cancer Center, remained bullish about the drug’s potential.
“In AML we set the bar high because we do cure about 40% of patients with AML. Our expectations are higher than they are for solid cancers. But we should still be excited by one or two months increase in benefit. Huge leaps happen only every once in a while. We should not discount smaller increases in benefit for our patients.”
Both studies were presented at the American Society of Hematology annual conference in San Francisco.
Lilian Anekwe
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