PII (NCT03338348) trial completed in Oct 19 after enrolling only 9 patients. As at Jan 20, PII/III AML18 (NCT02272478) trial was still recruiting, and aiming for 1,600 participants. It is now due to complete collection of primary outcome data in Feb 21. As at Oct 20, PII/III BIG-1 study (NCT02416388) trial was also recruiting, aiming for 3,100 participants, and still aiming to complete collection of primary outcome data by Jun 24 .
Three university-sponsored studies are currently recruiting: PII (NCT03338348) is comparing vosaroxin with azacitidine in 168 older patients with newly diagnosed AML and intermediate or adverse genetic risk or MDS-EB-2 (due to complete collection of primary outcome data in Dec 21). PII/III AML18 (NCT02272478), with collaboration from Cancer Research UK, is evaluating several relevant therapeutic questions in AML and high risk myelodysplastic syndrome. The trial is primarily designed for patients over 60 years considered fit for an intensive chemotherapeutic approach. Vosaroxin is being assessed in combination with decitabine. Collection of primary outcome data is due to complete Oct 19. PII/III BIG-1 study (NCT02416388) is an open label study with multiple randomization phases at differents stages of AML treatment (induction, consolidation and HSCT where applicable) is designed to improve OS in younger (18 to 60 year-old) patients, with AML risk-adapted patient strategies. Within the intermediate risk AML group, optimal GvHD prophylaxis following allogeneic SCT in first CR, after either myeloablative (MAC) or reduced intensity (RIC) conditioning, will also be evaluated. With an adaptative design, this clinical trial could test up to 3 novel AML agents of interest, including vosaroxin. Collection of primary outcome data is due to complete Jun 24 .
Results of VALOR (NCT01191801) PIII, double-blind, placebo-controlled trial published in the Lancet Oncology. RCT (n=n=711) found addition of vosaroxin to cytarabine therapy did not lead to improvement in overall survival (7.5 months vosaroxin vs. 6.1 months cytarabine group; p=0.061). More patients achieved complete remission in vosaroxin group .
VALOR reported at ASH. In patients with relapsed or refractory AML, the addition of vosaroxin to cytarabine improved overall survival (median of 7.5 months vs. 6.1 months with cytarabine alone; p=0.02 when accounting for the stratification factors) .
VALOR study results (n=711): vosaroxin failed to reach the study’s primary endpoint of a statistically significant improvement in overall survival for patients with AML. Secondary endpoint of a clinically significant benefit in the complete remission (CR) rate of AML in patients was reached. Despite this, a marketing authorisation application will be filed with the EMA. 
PIII VALOR trial estimated completion date: June 2014. 
Independent Data and Safety Monitoring Board has completed its latest periodic safety review and recommended that the trial continue as planned without changes to study conduct .
As of 12 March 2013, 563 patients enrolled into the VALOR trial. Planned interim safety analysis by the DSMB expected June 2013, completion of patient enrolment by end of 2013 and unblinding of trial data in the first half of 2014 .
Sunesis Pharmaceuticals, Inc. today announced that it will implement a one-time, 225-patient sample size increase to its Phase 3 VALOR trial bringing target enrollment to 675 patients. This is in response to the recommendation of the trial´s independent DSMB .
As of March 2012, 260 pts have already enrolled in the VALOR trial. In December 2011, the Data and Safety Monitoring Board (DSMB) recommended the trial continue as planned. An interim analysis is expected to take place in the third quarter of 2012, with unblinding anticipated in mid-2013 .
The PIII VALOR trial of vosaroxin in combination with cytarabine in patients with first relapsed or refractory acute myeloid leukemia (AML) has started. The multinational, randomized, double-blind, placebo-controlled trial will enroll 450 evaluable patients at sites in the US Canada, Europe, Australia and New Zealand. The primary endpoint is overall survival. The trial has an adaptive design that provides for a single interim analysis by an independent Data and Safety Monitoring Board which will decide whether to implement a one-time sample size adjustment of 225 additional evaluable patients to maintain adequate power across a range of outcomes. The interim analysis is expected to take place mid-2012 .
The company has received advice from the EMA and the FDA on its proposed PIII study in patients with first relapsed or primary refractory AML. Based on feedback from the two agencies, the company expects that if the study demonstrates a convincing increase in overall survival (primary endpoint) and a favourable benefit-risk ratio it will be sufficient for registration in US and EU. It plans to start the multi-national, double-blind trial in 2H 2010. The trial will enrol around 450 patients who will be randomised to either voreloxin (90mg/m2) on days 1 and 4 in combination with cytarabine (1g/m2) daily for 5 days, or placebo in combination with cytarabine .
Voreloxin is currently being evaluated in a fully enrolled single agent PII study (REVEAL-1) in previously untreated elderly AML patients and in a fully enrolled PIb/II study combining voreloxin with cytarabine for the treatment of patients with relapsed/refractory AML. (A PII single agent trial in platinum-resistant ovarian cancer has also completed enrollment.) 
Voreloxin is a first-in-class anticancer quinolone derivative (AQD). It intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis .