CHICAGO— Pfizer On Friday it said its drug for an advanced form of lung cancer showed promising long-term results in a late-stage trial, which may help establish it as the new standard treatment for the condition.
The company's drug helped patients live longer without seeing cancer progress, and most people experienced that benefit for more than five years. The drug, called Lorbrena, also reduces the risk of cancer progressing in patients' brains.
Lorbrena is already approved in the US for the treatment of adults with advanced non-small cell lung cancer who have a mutation in a gene called ALK. Only about 5% of all non-small cell lung cancer patients have the mutation, which causes cancer cells to grow and spread abnormally.
But that translates to 72,000 people being diagnosed with that specific form of lung cancer each year worldwide, according to a statement from Pfizer. That cancer is typically aggressive and often affects younger people, the company added.
More generally, non-small cell lung cancer is a common form of the disease.
Lorbrena is specifically approved as a first-line treatment for that form of lung cancer, meaning patients taking it have not received any other therapy. But Pfizer's drug is not currently considered the standard (nor the most appropriate and widely used) treatment for this condition.
The company believes new five-year data on the drug will change that.
“In cancer medicine in general, you always want to offer the best medicine first. So we believe that this data… will lead to [Lorbrena] becoming a “standard” first-line treatment in this specific form of lung cancer, Chris Boshoff, Pfizer's head of oncology, told CNBC in an interview.
The new five-year data comes from the same phase three trial that led to Lorbreña's approval in the United States. Pfizer will present the results Friday at the American Society of Clinical Oncology's annual meeting in Chicago, the world's largest cancer research conference. The data was also published in the Journal of Clinical Oncology.
Nearly 300 people in the trial received Lorbrena or Xalkori, Pfizer's old lung cancer drug. After five years, 50% of patients in the trial were still receiving Lorbrena compared to 5% of people receiving Xalkori.
In the trial, Lorbrena reduced the risk of cancer progression or death by 81% compared to Xalkori, after five years.
Around 60% of the patients treated with Lorbrena were still alive without seeing their cancer progress after that same period. That compares with 8% among those taking Xalkori.
Dr. David Spigel, scientific director of the Sarah Cannon Research Institute, called those results “the best we've ever seen” during a briefing with reporters before the ASCO conference.
“We haven't seen anything like this. Other big drugs that are available… haven't reported the kind of durable, progression-free survival events of this magnitude,” Spigel said, referring to the rate of people who stayed alive. without seeing your cancer progress.
He noted that there are no head-to-head trials comparing Pfizer's Lorbrena with competing lung cancer drugs, including one called alectinib and another called brigatinib.
All three are called ALK inhibitors and are designed to block mutations in the ALK gene associated with abnormal cancer cell growth. Lorbrena is considered a newer third-generation ALK inhibitor, while the two competitors are second-generation.
But Spigel added that “it's hard to believe” that Lorbreña performed worse against those drugs.
Pfizer's other drug, Xalkori, is also an ALK inhibitor, but it is no longer used in the US.
Lung cancers with the “ALK-positive” mutation are also especially likely to spread to the brain. About a quarter or more of patients may develop brain metastases (when cancer cells spread from their original part of the body to the brain) within the first two years of being diagnosed.
Lorbreña reduced the risk of cancer progressing in the brain by 94% compared to the old Pfizer drug. Only four of 114 patients taking Lorbrena developed brain metastases in about 16 months, compared with 39 of 109 taking Xalkori.
Lorbrena is effective in preventing and treating brain metastases because it can cross a membrane called the blood-brain barrier and enter the brain, something that not all drugs can do.
Spigel called this finding another “stunning finding,” as the progression in the brain is “pretty scary for patients and something we're desperately trying to prevent or treat.”
No new security problems were reported for Lorrena. The most common side effects included bloating, weight gain, cognitive and mood changes, and high blood cholesterol, among others.
But Spigel called the cognitive problems associated with Lorbreña “unusual” as it is not seen with his competitors.
In a note published Thursday ahead of the data's release, Dr. Andrew Berens, an analyst at Leerink Partners, said he believes Lorbrena's side effects on the central nervous system are part of the reason why it is often used as a second-line rather than first-line treatment for this advanced form of lung cancer. . These changes in cognition and mood result in “a lower quality of life for the patient,” he said.
But Pfizer's Boshoff said that once doctors use Lorbrena for the first time, they feel comfortable managing any specific side effects associated with the drug.
He noted that educating doctors on how to manage adverse effects will be an important part of Lorbreña's “relaunch” following the publication of the new data.
