Bristol-Myers Squibb Co.’s blockbuster cancer drug Opdivo prolonged survival in cases of recurrent head and neck cancer, a first for patients with the harshest form of the disease who often face a bleak prognosis.
Doctors studied Opdivo, already approved to treat melanoma and lung tumors, among patients whose cancer had returned and worsened even after treatment with chemotherapy or Eli Lilly and Co.’s Erbitux, which was originally developed with Bristol-Myers.
Indianapolis-based Lilly reported sales of $176 million from Erbitux in the fourth quarter of 2015. Erbitux was originally approved to treat colon cancer, but was later approved for head and neck cancer.
About 36 percent of patients on Opdivo were alive after one year, compared with just 17 percent of those getting the standard treatments, said lead researcher Maura Gillison, chair of cancer research at Ohio State University in Columbus.
Opdivo lengthened median survival to 7.5 months, compared with 5.1 months for those getting existing therapy, researchers reported Tuesday at the American Association for Cancer Research meeting in New Orleans, a finding that should make the drug a new standard for patients with recurring, metastatic disease. The trial was stopped in January after the therapy showed its effectiveness.
“I’ve been treating head and neck cancer for 20 years, and this is the first time I can reach for something that will be effective in this patient population,” Gillison said. “Average survival is typically less than six months for this group.”
Bristol-Myers and Merck & Co. are racing each other to get their competing treatments approved in new types of cancer. The U.S. Food and Drug Administration accepted Merck’s application for Keytruda for the same patient group earlier this month. The agency is expected to rule by August on that drug.
Bristol-Myers hasn’t yet announced its regulatory plans for Opdivo in head and neck cancer. However, its impact on overall survival may give it a leg up on competitors, Steve Scala, an analyst with Cowen and Co., said April 13 in a note to clients.
About 600,000 people worldwide and 48,000 in the U.S. are diagnosed annually with head and neck cancer, which can cause difficulties speaking, eating, swallowing and even breathing. While half of patients are cured by surgery, radiation or chemotherapy, the remainder have their tumors reappear within three to five years. The next step for those patients is chemotherapy. The Opdivo study included patients whose cancer returned within six months of chemotherapy, a group considered incurable.
The high one-year survival rate among patients treated with Opdivo raises the tantalizing possibility that some may eventually be cured, though it’s too soon to know, Gillison said. The drug also had fewer treatment-related side effects than the comparison therapies, a key issue for patients concerned about quality of life. Two patients taking Opdivo and one getting standard therapy suffered treatment-related deaths.
Doctors are excited about what the drug might do if it’s used earlier in the disease, given the response in the hardest-to-treat patients, said Lillian Siu, professor of medicine and oncologist at Princess Margaret Cancer Center in Toronto and co-chair of AACR’s clinical trial committee. It may allow lower-risk patients to avoid the more toxic chemotherapy, while higher-risk patients may benefit from adding it to their current treatment, she said.
Bristol-Myers plans to study Opdivo earlier in the disease and in combination with other drugs, said Nick Botwood, vice president and development lead for lung/head and neck cancers.
“This is the tip of the iceberg,” he said. “Our mind is very much on tomorrow, and what we can do in the future to bring treatment options to a wider range of patients.”
Seeking a biomarker
Opdivo appeared effective in patients regardless of whether they tested positive for human papillomavirus, a common sexually transmitted virus that can cause cancer, or whether their tumors expressed PD-L1, a protein that suppresses the body’s anti-tumor activity and is a main target of the drug. While Opdivo was more effective in those who did express PD-L1, two of the six patients who had a complete response to the drug didn’t fall into that group, Gillison said.
It will be important to find a biomarker to identify which patients are most likely to benefit from the treatment, particularly because drugs like Opdivo that help the body’s immune system fight cancer are so expensive, Siu said. Both Opdivo and Keytruda cost about $150,000 a year.
Even conventional indicators like disease progression didn’t help identify who would respond, with no difference in progression-free survival in the study. Opdivo didn’t seem to slow the growth of tumors, even though patients getting the drug had better survival, she said.
“You can split out the responders, but it doesn’t tell you who will get the long-term benefits,” she said. “There are people that look like they are progressing that remain stable for a long period of time. The tumor may not immediately shrink on a CT scan, but they still derive a benefit.”