The phase 3 VERONA trial did not meet its primary endpoint of overall survival in patients with high-risk myelodysplastic syndromes, according to results presented at the Thirteenth Annual Meeting of the Society of Hematologic Oncology (SOHO 2025).
The study, presented by Guillermo Garcia-Manero, MD, interim chair of the Department of Leukemia at the University of Texas MD Anderson Cancer Center, evaluated the efficacy and safety of venetoclax in combination with azacitidine in treatment-naive patients with high-risk MDS. The global, double-blind trial enrolled 509 patients randomized 1:1 to receive venetoclax plus azacitidine or azacitidine with placebo.
“The addition of venetoclax to azacitidine did not have an impact on overall survival in this population,” Dr. Garcia-Manero said. “However, response rates were higher with the combination.”
Median overall survival was approximately 22 months in both arms, but the venetoclax group achieved higher overall response rates, particularly among patients with more than 5% blasts. The regimen was well tolerated, with no new safety signals or increase in early mortality.
“This is a very important study,” Dr. Garcia-Manero added. “It shows that the combination can be delivered safely worldwide, but we need to better understand which patients benefit most.”
In an interview with Hagop Kantarjian, MD, Dr. Garcia-Manero said that even though the study was negative, there is still a role for venetoclax.
“Although the study may be negative, I think we’re making overall progress,” Dr. Garcia-Manero said. “I still believe that … venetoclax has a role for a subset of these patients.”
Dr. Garcia-Manero’s presentation of the VERONA trial was recognized with the Top Abstract Award at the Annual Meeting.
Reference
Garcia-Manero G, Platzbecker U, Fenaux P. The primary analysis of the randomized, phase 3 VERONA study of venetoclax with azacitidine in patients with treatment-naïve, intermediate and higher-risk myelodysplastic syndromes. Presented at the Society of Hematologic Oncology 2025 Annual Meeting; September 3-6, 2025; Houston, Texas.
