Roche and AbbVie have come up short again in their effort to gain an additional indication for their blockbuster blood cancer treatment Venclexta.
A phase 3 trial of the therapy in previously untreated patients with higher-risk myelodysplastic syndromes (MDS) did not achieve its primary endpoint of overall survival, the companies said Monday. In the global study of approximately 500 patients, the combination of Venclexta plus the chemotherapy azacitidine was pitted against placebo plus azacitidine.
The companies said they will present full data from the trial at an upcoming medical meeting.
MDS are a group of bone marrow disorders, with higher-risk patients having increased abnormal blood cell production and a greater likelihood of progressing to acute myeloid leukemia (AML).
The FDA granted accelerated approval to Venclexta as a treatment for newly diagnosed patients with AML in 2018, with a full nod following in 2020. Venclexta first hit the market in 2016 with a U.S. approval to treat chronic lymphocytic leukemia.
This is the second trial failure for AbbVie and Roche with Venclexta, which also came up short in the Canova study in patients with relapsed or refractory multiple myeloma who tested positive for a specific biomarker and who had tried at least two prior treatments.
In that study, which was read out in 2023, Venclexta was combined with dexamethasone tested against a combination of Bristol Myers Squibb’s Pomalyst and dexamethasone. While the Venclexta regimen improved patients' progression-free survival, the difference failed to achieve statistical significance.
The first major setback for the therapy came in 2019, when there were more deaths in the Venclexta arm of a phase 3 multiple myeloma trial than in the control arm, triggering a clinical hold on all multiple myeloma trials of the treatment. The hold was eventually lifted after the companies agreed to use risk mitigation measures.
Two years later, the companies reported that the Bellini trial, in which the deaths occurred, achieved its primary endpoint of progression-free survival but resulted in increased mortality in the overall patient population compared to the control arm.
Venclexta is a first-in-class drug that works by selectively binding and inhibiting the B-cell lymphoma-2 protein, helping restore the process of apoptosis that’s hindered in some blood cancers.
The treatment achieved blockbuster status in 2020 and generated sales of $2.6 billion last year, an increase of 13% from 2023.