- A newly approved cancer cell therapy from Bristol Myers Squibb outperformed standard-of-care treatment in a late-stage study of patients with relapsed or resistant lymphoma, the pharmaceutical company said in a Thursday statement.
- The results mark the first time a so-called CAR-T therapy has been shown to improve upon chemotherapy followed by a stem cell transplant in previously treated patients with this type of lymphoma, according to Bristol Myers. In February, the company won U.S. approval of its therapy, called Breyanzi, for later-stage treatment.
- Bristol-Myers did not detail specific data in its announcement. But the drugmaker said Breyanzi results in a "clinically meaningful and highly statistically significant" improvement in event-free survival over chemotherapy and a transplant. Breyanzi also met secondary goals measuring tumor response and progression-free survival.
Breyanzi was the third CAR-T therapy approved to treat a type of lymphoma known as diffuse large B-cell, following earlier clearances for Gilead's Yescarta and Novartis' Kymriah. All three approvals were based on results from single-arm studies that showed the cellular treatments could spur powerful responses, shrinking tumors and, in about a third to a half of participants, inducing complete remission.
The studies tested the CAR-T therapies in very sick patients who had received multiple prior treatments for their lymphoma and who had few other options. (The median number of previous treatments was three in each of the trials.)
But moving into earlier lines of treatment has always been the drugmakers' aim, and an important step in realizing the potential of CAR-T therapy.
Bristol Myers, while the last of the three to win approval for its drug, is now a step closer to that goal. Noah Berkowitz, head of hematology and cell therapy development at Bristol Myers, said in a statement that the positive data announced Thursday could help Breyanzi supplant current treatment regimens in the second-line setting.
Bristol Myers' study primarily measured a metric known as event-free survival, which assesses the time until death, disease progression, an inadequate treatment response or the start of a new cancer treatment. Breyanzi proved superior to "salvage" therapy followed by high-dose chemotherapy and a stem cell transplant, which is the current standard of care.
According the company, the CAR-T therapy met the study's secondary goals assessing the rate of complete response as well as a narrower measure called progression-free survival. The results were from an interim readout; follow-up to measure whether Breyanzi helps patients live longer continues.
Bristol Myers plans to present detailed results from the study, dubbed TRANSFORM, at an upcoming medical meeting. Data on safety as well as the magnitude of benefit over standard treatment will be important to gauge Breyanzi's benefit.
High-dose chemo and transplants are intensive, have many side effects and don't cure most patients, so there is a significant opportunity for a better option. But CAR-T treatments are also complicated, requiring a logistically daunting process to extract patient immune cells, engineer them in a laboratory and then reinfuse them back into the patient. CAR-T therapies also have notable side effects, including a dangerous immune overreaction known as cytokine release syndrome and neurotoxicity.
Bristol Myers said the safety profile of Breyanzi in the second-line setting was "consistent" with past results in later lines of treatment.
Rivals Gilead and Novartis are also testing their respective CAR-T therapies as second-line treatments for diffuse large B-cell lymphoma. Results from a Phase 3 study of Gilead's Yescarta are due by the end of June and, if positive, the company expects to ask the Food and Drug Administration for approval in the second half of this year.
"If the study is positive, we're looking at replacing stem cell transplant as a standard-of-care in second-line for curative potential," Christi Shaw, head of Gilead's cell therapy division Kite Pharma, told investors on an April conference call.
Bristol Myers' TRANSFORM study enrolled about 180 patients, according to a federal database of clinical trials. Gilead's as well as Novartis' trials are about twice as large.