ASH: Bristol-Myers' CAR-T poised for FDA filing after banishing lymphoma in 53% of patients

A newly unveiled study tested liso-cel in 269 patients with relapsed or refractory diffuse large B-cell lymphomas who had undergone a median of three previous therapies. (Bristol-Myers Squibb)

In a newly unveiled study, Bristol-Myers Squibb’s CAR-T therapy banished tumors in more than half of relapsed blood cancer patients and shrank tumors in nearly three-quarters of them. The treatment, picked up in the company’s $74 billion Celgene buyout, could help patients with large B-cell lymphomas whose disease has worsened despite trying other treatments. 

Roche's Rituxan, in combination with chemotherapy drugs, is the first-line treatment for patients with diffuse large B-cell lymphomas (DLBCL). It works for many patients, but some patients must move on to other treatments, including stem cell transplants.

For patients who relapse, treatment options are virtually nonexistent, Bristol-Myers' chief medical officer Samit Hirawat, M.D., told FierceBiotech.

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Novartis’ Kymriah and Gilead’s Yescarta are both approved to treat DLBCL, so Bristol-Myers' treatment, lisocabtagene maraleucel—or liso-cel—would be third to market. But Hirawat said the “game-changer” data, presented Saturday at the annual meeting of the American Society of Hematology, could give it an edge.

For starters, the phase 1 study tested liso-cel in more than 250 patients, while the pivotal trial for Yescarta involved 77 patients with DLBCL and the one for Kymriah had 51 DLBCL patients.

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The study tested liso-cel in 269 patients with relapsed or refractory DLBCL who had undergone a median of three previous therapies. Chemotherapy had not worked in 67% of them. The patients received one of three doses of the treatment, with the majority of patients, 177, receiving the middle dose: 100 million CAR-T cells per kilogram of body weight.

In addition to quashing tumors in 53% of patients and shrinking tumors in 73% of patients, the treatment kept cancer at bay for a median of 6.8 months and kept patients alive for a median of 21.1 months. At a median of 12 months after treatment, the median duration of response had not yet been reached, suggesting the treatment’s effect on preventing cancer from growing or spreading could be long-lasting.

“It’s very encouraging and exciting to see 53% achieve a complete response. Most of them stay in response for more than 12 months,” Hirawat said.

“If you get into a complete response at the time of first evaluation at one month, we now know that 60% of them will continue to stay in that response at the six-month mark. Almost 55% stay in that response at 12 months, so we will continue to follow up to see if we can maintain that 55% number or if it will go down a little bit,” he said.

Of the patients who achieved a complete response, 86% of them were still alive one year after treatment and 65% of them did not see their cancer worsen.

More than half of the patients—58%—did not experience cytokine release syndrome (CRS), a dangerous side effect that happens when the CAR-T cells work too well and activate the immune system too strongly. Only six patients—2%—suffered severe CRS, and 10% of patients had neurological side effects.

Eight patients died from side effects that happened after treatment, with four of the deaths deemed treatment-related.

“One thing we have to remember is this is a study of 269 patients and this is a very late-stage treatment. These are very sick patients who are heavily pretreated and adverse events do occur … In a large study, we pay attention very closely to the safety of patients. This is the reality of the disease, as well as the treatment state,” Hirawat said.

The company plans to file liso-cel for FDA approval by the end of the year.

“These pivotal longer-term results from TRANSCEND NHL 001 continue to give us confidence in the clinical profile of liso-cel. Importantly, these results were demonstrated in a study with more than 250 patients in a broad population reflective of clinical practice, including those with poor prognoses and a range of histologies,” Stanley Frankel, M.D., senior vice president of cellular therapy development at Bristol-Myers Squibb, said in a statement. “We look forward to providing these data to support the regulatory approval for this treatment option for these patients with large B-cell lymphomas.”

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