Venclexta, when combined with azacitidine, elicited an overall survival benefit below 10% in patients with myelodysplastic syndromes.
Roche and AbbVie’s Venclexta, when used in combination with chemotherapy, does not improve survival in high-risk patients with myelodysplastic syndrome, adding to a growing list of setbacks for the blood cancer drug.
According to a press announcement on Monday, Venclexta plus azacitidine improved overall survival by under 10%, an effect that failed to reach statistical significance. Venclexta’s survival miss will not affect any of its existing approvals, the companies insisted.
Monday’s data come from the Phase III VERONA trial, a randomized study that pitted Venclexta plus azacitidine against azacitidine alone. The study enrolled more than 530 patients deemed intermediate, high or very high risk as per international assessment standards. Full data from VERONA will be available at a future scientific meeting and publication, the companies said.
Venclexta is an oral small molecule blocker of BCL-2, a protein that prevents cell death. Cancer cells overexpress BCL-2, which promotes the survival of the tumor and has been linked to treatment resistance.
Venclexta first received FDA approval in 2016 for chronic lymphocytic leukemia, garnering a handful of other approvals since then, including for acute myeloid leukemia. But further efforts to expand the drug’s label have run into roadblocks. In September 2023, for instance, AbbVie and Roche revealed that the Phase III CANOVA trial of Venclexta in relapsed or refractory multiple myeloma failed to demonstrate a significant benefit in progression-free survival (PFS). CANOVA combined Venclexta with dexamethasone and compared it against pomalidomide plus dexamethasone.
Years before, in 2019, the FDA slapped a clinical hold on all multiple myeloma Venclexta trials at the time after detecting a higher rate of deaths in a Phase III trial versus placebo. The regulator lifted the pause a few months later and in 2021, the study that triggered the freeze, dubbed BELLINI, demonstrated a significant PFS benefit to Venclexta plus bortezomib—but also detected a higher risk of mortality.
Venclexta remains a strong market asset for AbbVie and Roche. It made $2.6 billion in 2024, growing nearly 16% year-on-year on an operational basis.
