Pfizer trial shows experimental drug doubles time until breast cancer tumours grow worse

US Food and Drug Administration grants “breakthrough” status for the drug, palbociclib, but overall survival still not statistically significant, say researchers

PUBLISHED : Monday, 07 April, 2014, 4:52pm
UPDATED : Monday, 07 April, 2014, 4:52pm

A clinical trial of an experimental Pfizer breast cancer drug nearly doubled the amount of time patients lived without their disease getting worse.

However, overall survival has not yet been shown to be statistically significant, researchers said.

Pharmaceutical company Pfizer’s Phase 2 study, involving women with the most common form of breast cancer, found that those treated with hormone drug letrozole, together with its experimental drug palbociclib, lived for an average of 20.2 months before their cancer progressed, compared with 10.2 months for patients given only letrozole.

The United States Food and Drug Administration has granted “breakthrough” status for palbociclib.

Pfizer is still discussing a regulatory pathway for the drug, but has not decided whether to seek accelerated approval based on the Phase 2 trial results, said Mace Rothenberg, chief medical officer of Pfizer’s oncology unit.

Palbociclib is viewed as one of the company’s most important experimental drugs, which some analysts believe could eventually claim annual sales of more than US$5 billion, if approved by regulators.

The trial tested the pill, which targets proteins involved in cell division, in post-menopausal women with locally advanced or newly diagnosed breast cancer that had spread to other parts of the body.

The women had cancer that was both oestrogen receptor positive – meaning that tumours grow in response to oestrogen – and HER2-negative, meaning that the HER2 protein is not causing the cancer. Such patients make up about 60 per cent of advanced breast cancer cases.

The initial data showed overall survival of 37.5 months for the combination treatment, compared with 33.3 months for patients given only letrozole, an oestrogen blocker sold by Novartis AG under the brand name Femara.

Researchers said that because only about 30 patients in each arm of the 165-patient trial had died it was still too early to define the drug’s impact on survival.

“The curves are starting to separate ... It hasn’t reached statistical significance, but patients are still being followed,” said Dr Richard Finn, associate professor of medicine at the University of California, Los Angeles, and a lead author of the study.

Side effects seen in the trial, including low blood cell counts and fatigue, were manageable, he said before the study was presented to the American Association for Cancer Research, in San Diego.

Hormonal agents, like Femara, have extended survival for women with oestrogen-positive, HER2 negative breast cancer, but there have been no big advances in treatment for nearly two decades, said Dr Judy Garber, a breast cancer specialist at Boston’s Dana-Farber Cancer Institute, who was not involved in the palbociclib trial.

“This is garden variety breast cancer,” she said. “When it recurs, all we really have are other hormonal agents ... This is the first new drug to really show promise.”

Pfizer is conducting Phase 3 trials in breast cancer patients as well as earlier-stage trials in other types of cancer.

Companies trying to develop treatments similar to palbociclib include Novartis and Eli Lilly.

Data from a small Phase 1 trial, carried out by Lilly, found that its drug, LY2835219, used on its own, shrank tumours in 25 per cent of women with metastatic oestrogen-positive breast cancer, and stabilised the cancer in 55 per cent of the same group of women.