Pevonedistat/Azacitidine Combo Demonstrates Encouraging EFS Data in Phase 2 Trial in MDS

Justin M. Watts, MD, an assistant professor of medicine at the Sylvester Comprehensive Cancer Center, University of Miami Health System, discusses the key takeaway from the phase 2 Study P-2001 clinical trial (NCT02610777), a randomized study comparing the safety and efficacy of pevonedistat in combination with azacitidine compared with azacitidine alone in patients with high-risk myelodysplastic syndromes (MDS).

The 120-patient study provided fairly robust data with improved event-free survival (EFS) at 5 months, says Watts. There was also marked improvements in the complete remission rates and durations of response. Watts notes this is only 1 of the active agents under development in MDS at this time, which is a good problem to have.

The difference with pevonedistat and other active agents in development is the response rates are comparable, but these are randomized phase 2 data with an emerging EFS benefit that remains to be seen in the phase 3 study, where the primary end point is EFS. However, Watts notes this regimen is appealing due to its minimal toxicity, particularly when added to azacitidine.