Novartis Cancer Treatment Fails in Phase 3 Clinical Trial

Novartis recently announced that the Phase 3 clinical trial investigating its aggressive B-cell-non-Hodgkin lymphoma (NHL) treatment, Kymriah, did not meet its primary endpoint of event-free survival compared to standard of care.

The BELINDA study was a pivotal Phase 3, randomized, open-label trial that enrolled patients from over 73 sites in 18 countries worldwide. The primary endpoint of the trial was event-free survival.

Researchers compared two treatment strategies assessing the safety, efficacy, and tolerability of Kymriah compared to salvage chemotherapy, followed by high-dose chemotherapy and hematopoietic stem cell transplant. 

Patients in the trial had aggressive B-cell non-Hodgkin lymphoma with primary refractory disease or a relapse within 12 months of first-line treatment.

Study investigators will work with Novartis in the near future to understand the factors that contributed to the negative trial outcomes. 

“Kymriah continues to demonstrate durable responses for patients with certain advanced blood cancers in the third-line setting. We remain committed to accelerating development of Kymriah and our next-generation CAR-Ts and anticipate sharing early clinical results for these therapies at an upcoming medical meeting,” Jeff Legos, executive vice president and global head of oncology & hematology development at Novartis, said in the announcement. 

Kymriah is a genetically modified autologous T-cell immunotherapy. Each treatment dose is customized using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. 

FDA approved Kymriah in 2017 for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia. 

The cell-based gene therapy is the first to be approved in the US to treat patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse. 

Novartis is known for developing breakthrough treatments to address society’s most pressing issues. 

In Q1 2020, the company’s total net sales equaled $12.2 billion, while Q2’s total revenue was $11.3 billion. Q2 core operating income grew six percent due to lower spending and improved gross margin, but the net income declined four percent due to higher impairments.

The key products driving overall growth included six of its most popular drugs, including Kymriah. 

“We were hopeful the BELINDA study would show that Kymriah could improve outcomes and the overall treatment experience for these patients in need,” Michael R. Bishop, MD, professor of medicine and director of the hematopoietic stem cell transplantation program at the University of Chicago Medicine and BELINDA steering committee chair, said in the recent announcement. 

And a Novartis spokesperson added that the company is grateful to the patients, families, and investigators who participated in this trial for their determination to advance the treatment of this aggressive blood cancer.