Clavis Pharma: Phase II trial demonstrates Elacytarabine may increase survival threefold in patients with late-stage leukaemia
Clavis Pharma ASA announced positive final results from a Phase II trial of its novel investigational cancer drug, elacytarabine (CP-4055), in patients with late-stage acute myeloid leukaemia (AML). In the trial, elacytarabine showed statistically significant superior efficacy compared to published clinical data for late-stage AML. Based on these encouraging results an elacytarabine registration study is being planned.
Elacytarabine is a novel cytotoxic agent being developed by Clavis Pharma for the treatment of hematologic cancers and solid tumors, especially those resistant to existing agents. One particularly difficult-to-treat patient group is late-stage acute myeloid leukaemia (AML) patients who have failed two previous therapeutic regimens. There is currently no standard therapy available for these patients and life expectancy is very short. It is in this patient group that Clavis Pharma has evaluated the safety and efficacy of elacytarabine.
In the Phase II study, 61 patients with late stage AML who failed to respond or relapsed after two separate rounds of treatments received third-line therapy (also called second salvage) with intravenous elacytarabine. The response to treatment was compared with a detailed historical outcome analysis of 594 similar second salvage AML patients, who were treated at the MD Anderson Cancer Center (Houston, TX, USA) (published by Giles et al, Cancer 2005;104:547-54). Median overall survival in the elacytarabine study was an impressive three times that of the historical control patients (5.5 months vs. 1.5 months). Further follow up on survival is ongoing.
In addition, 9 patients responded to elacytarabine with a complete remission or complete remission without full recovery of platelet counts as assessed by the investigator (of which 6 have been confirmed by an independent review of bone marrow histology), representing an overall remission rate of 15 per cent. By contrast, the expected remission rate for similar group of patients, matched for prognostic factors as described by Giles et al. was only 2.5 per cent. Using a pre-defined statistical analysis method, the improvement in outcome was statistically highly significant (corresponding to p<0.0001). If only patients whose complete remission has been confirmed by histology are included in the analysis, the improvement in remission rate is still statistically highly significant (corresponding to p<0.01).
Elacytarabine was relatively well tolerated and 30 day all cause mortality following treatment was substantially lower than published data for existing therapies (13 per cent vs. 25 per cent). Out of the 61 patients treated with elacytarabine, 10 were referred for stem cell transplantation following treatment, including some patients in complete remission and others with a more modest level of clinical benefit. Stem cell transplantation represents a potential cure for life for these patients.
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