- An experimental cell therapy developed by Johnson & Johnson reduced or eliminated signs of multiple myeloma in nearly all of 94 patients treated in a mid-stage study, according to results presented Saturday at a virtual meeting of the American Society of Hematology.
- After one year, 89% of patients were still alive and roughly three quarters were still responding to treatment, the data showed. The study presentation at ASH strengthens earlier findings, which were similarly encouraging.
- The new data are from patients enrolled into an expansion portion of J&J's trial, called CARTITUDE-1. Participants were very sick, with their cancer having progressed following a median of six different treatments. While J&J's therapy appears strongly effective, side effects were common and one patient died from an adverse reaction typical of cell therapy treatment.
J&J's cell therapy is one of a broad slate of new medicines being developed for multiple myeloma that target a protein called BCMA, which is commonly found on diseased blood cells.
Blenrep, an antibody drug from GlaxoSmithKline, became the first such drug approved when the Food and Drug Administration cleared it in August.
Cell therapies like J&J's and another from Bristol Myers Squibb called ide-cel appear much more potent, inducing strong response rates. Both are CAR-T cell therapies, which involve the re-engineering of patients' own immune cells to seek out BCMA-expressing cancers.
Three CAR-T therapies are approved to treat leukemia and lymphoma, but ide-cel would be the first in multiple myeloma if approved as expected by March of next year.
The catch with CAR-T therapies is the process by which they are made is complicated, expensive and can sometimes fail. Both ide-cel and J&J's cell therapy, which the pharma licensed from China-based Legend Biotech, could face competition from other BCMA-targeting antibody drugs, a handful of which are in early- to mid-stage testing.
In CARTITUDE-1, treatment with J&J's therapy, cilta-cel, led to responses in 94 of 97 enrolled patients. Sixty-seven percent met the criteria for a "stringent complete response," meaning no signs of diseases cells were observed in the bone marrow.
At 12 months, 77% of patients showed no sign of their cancer progressing, and 89% were still alive.
J&J plans to submit the data from its trial to the Food and Drug Administration in the next weeks. But CARTITUDE-1 is only a first step in the drugmaker's planned work with cilta-cel in multiple myeloma, said Mark Wildgust, vice president of oncology global medical affairs with J&J.
"I think the CARTITUDE-1 data gives us confidence in [cilta-cel as a] single agent," he said in an interview. "Now we have to see if we can do it earlier in the disease."
One significant concern with CAR-T therapy is its side effect profile. Treatment is commonly associated with an immune system response known as "cytokine release syndrome," or CRS, and neurotoxicity.
Nearly all patients in J&J's trial experienced CRS, and one patient died from the condition. Ninety-five percent of those affected had symptoms mild enough to be managed with fever-reducing medications or, if hospital care was necessary, responded quickly to treatment, J&J said.
The company hopes cilta-cel could potentially be given as an outpatient treatment, a goal of many CAR-T drug developers. Whether J&J's data supports that approach will be up to Food and Drug Administration officials, who will look closely at the risks of allowing patients to go home after treatment.
A neurological side effect associated with CRS occurred in 16 patients, but subsided, at the median, four days following onset. Twelve other cases of neurotoxicity were also observed, with one being fatal, J&J said.
Also at ASH, Bristol Myers disclosed updated data from Phase 1 and 2 trials of ide-cel. In the early study, which included four different doses tested, results showed three-quarters of patients responded to treatment.
Median progression-free survival in that study stretched to nine months, and participants lived a median of 34 months post treatment.
An analysis of the later, pivotal KarMMa study showed more than two thirds of patients responded to treatment in most high-risk groups, Bristol Myers said, while median progression-free survival was longer than seven months.
Results presented earlier this year showed, across the entire KarMMa study, an overall response rate of 73%, which rose to 82% among those given the highest dose.