Opdivo Fails to Meet Primary Endpoint in Phase 3 SCLC Trial

Last week, Bristol-Myers Squibb announced results from the phase 3 CheckMate -331 trial that investigated nivolumab (Opdivo) versus the current standard of care, chemotherapy, in the treatment of patients with small cell lung cancer (SCLC) who relapsed following platinum-based chemotherapy. The trial found that nivolumab did not significantly increase overall survival compared with chemotherapy.

Lung cancer is the leading cause of cancer deaths globally, accounting for approximately 1.8 million deaths each year, according to the World Health Organization. SCLC makes up about 10% to 15% of all lung cancer cases. Although chemotherapy remains the standard of care in the front-line setting, many patients experience relapse within 1 year.

The primary endpoint of overall survival with nivolumab, a PD-1 immune checkpoint inhibitor, versus chemotherapy was notably not met in the end-stage trial. CheckMate-331 is an open-label, randomized phase 3 study in which patients were randomized into 2 treatment arms: 1 evaluating nivolumab; and an active comparator arm evaluating topotecan or amrubicin, investigators choice. Though the drug did not meet the primary endpoint in the study, the safety profile remained consistent with that observed in previously reported monotherapy studies.

This finding is significant however, as it comes nearly 2 months after the FDA approved the drug to treat patients whose SCLC had progressed despite undergoing 2 previous therapies.

“Small cell lung cancer is a highly aggressive disease in which significant unmet needs remains. We are focused on researching innovative oncology therapies to improve outcomes for patients with lung cancer,” said Sabine Maier, MD, development lead for thoracic cancers at Bristol-Myers Squibb.

Nivolumab competes with pembrolizumab, sold as Keytruda. In the second quarter of 2018, nivolumab generated sales of $1.63 billion, while pembrolizumab earned $1.67 billion. Nivolumab is already approved for numerous indications across various disease states including melanoma, renal cell carcinoma, and classical Hodgkin lymphoma, among others.