Bristol-Myers' immunotherapy pioneer charts 'striking' long-term responses in melanoma

Stephen Hodi--Courtesy of Dana-Farber

A top-tier scientific team that includes investigators from Dana-Farber Cancer Institute and Johns Hopkins has tallied up some impressive long-term survival stats on a group of melanoma patients taking Bristol-Myers Squibb's ($BMY) nivolumab. According to the new research, which was published online Monday at the Journal of Clinical Oncology, 62% of the patients taking the immunotherapy were alive after one year and 43% were still alive after two years on the drug. And they say that the durable response was also reflected in patients who had stopped taking the drug.

Altogether 107 advanced melanoma patients, who shared an average life expectancy of one year, were given the drug intravenously every two weeks over a period of up to 96 weeks in the Phase I trial.

"These are striking results for patients with metastatic melanoma," said the study's senior author, Stephen Hodi, the director of the Melanoma Treatment Center at Dana-Farber. "This study provides the first demonstration of the long-term benefits this treatment approach can produce."

Two key observations caused some added excitement, says Hodi. Overall survival times for patients were longer than periods of progression-free survival, indicating that the drug was helping manage the disease even for patients who experienced new tumor growths--which is generally seen as a herald of a new assault by the cancer. In addition, tumors tended to remain the same size or shrink after the end of therapy.

"This suggests that blockade PD-1 may reset the equilibrium between the immune system and the tumor, keeping tumor growth in check," remarked Hodi.

Nivolumab is in a hectic race against rival therapies from Merck (MK-3475) and Roche ($RHHBY) which are designed to blockade PD-1 or the complementary PD-L1, helping unleash a T cell attack on cancer. And behind the leaders, practically every other major pharma company is assembling some sort of follow-up attempt to do the pioneers one better. Bristol-Myers, though, has been hit with some flak after analysts recently noted that the company has been slow to advance a combination of nivolumab with Yervoy into registrational studies, preferring to linger awhile in Phase II while Merck ($MRK) has been barreling ahead as fast as it can.

Merck--in bad need of a major new drug approval--says it plans to finish a rolling submission to the FDA in the first half of this year.

- here's the release on nivolumab
- get the research abstract

CORRECTION: This story was corrected to note that Bristol-Myers has been criticized for lingering in Phase II with its combination of nivolumab and Yervoy.

Special Reports: 10 top drugs in biopharma's late-stage pipeline - Nivolumab - MK-3475 | Top 10 experimental cancer drugs - 2013