- Treatment with Bristol Myers Squibb’s CAR-T cell therapy Breyanzi led to remission in adults with chronic lymphoyctic leukemia, meeting the goal of a yearslong clinical trial and suggesting the complex drug’s potential for use in patients with the slow-growing blood cancer.
- Bristol Myers did not disclose any specific data in its Thursday announcement of the positive study results, noting only that Breyanzi achieved the study’s primary endpoint comparing treatment response rates to historical controls among certain patients with relapsed or refractory chronic lymphocytic leukemia.
- The company, which in early 2021 won U.S. approval of Breyanzi for lymphoma, said it will present detailed results at an upcoming medical conference as well as discuss with them with regulators.
CAR-T cell therapy is now an established treatment option for certain types of lymphoma and for acute lymphoblastic leukemia that has returned or become resistant to other drugs. The approvals of Breyanzi and therapies from Gilead Sciences and Novartis followed years of clinical testing by academic centers like the University of Pennsylvania, smaller biotechnology companies and, most recently, the large drugmakers that now own them.
While none of those therapies are approved for chronic lymphocytic leukemia, or CLL, the blood cancer was the setting for one of the earliest demonstration’s of CAR-T therapy’s powerful potential. More than a decade ago, researchers at UPenn used CAR-T therapy to treat CLL patients, leading to remissions that, in two individuals, have lasted more than ten years.
The study of Breyanzi in CLL began in 2017, started by the treatment’s original biotech owner Juno Therapeutics. Initial results, presented at the American Society of Hematology’s annual meeting the next year, showed the treatment led to responses in 13 of 16 patients with CLL or small lymphocytic leukemia who had previously been given the drug Imbruvica.
By that time, however, Juno had been acquired by Celgene, which was primarily interested in Breyanzi’s potential in lymphoma. Celgene was then absorbed by Bristol Myers Squibb in a 2019 acquisition worth $74 billion.
Now, more than five years after the Phase 1/2 study began, Bristol Myers is reporting Breyanzi met the primary goal of the Phase 2 portion of the trial. The study was open-label, meaning patients knew the treatment they received, and didn’t include a control arm of patients who received placebo or another comparator drug.
Bristol Myers noted complete responses to Breyanzi in a prespecified subset of CLL patients who were refractory to drugs like Imbruvica and previously treated with another kind of therapy known as a BCL-2 inhibitor.
The company said there were no new safety signals reported for Breyanzi, which can cause dangerous immune reactions and neurological toxicity.
“In a population that has limited options, the ... study represents the first multicenter trial evaluating a CAR-T cell therapy in heavily pre-treated patients with relapsed or refractory CLL or SLL, with results showing the potential of Breyanzi as a personalized one-time treatment approach for patients with this difficult-to-treat disease,” said Anne Kerber, head of cell therapy development at Bristol Myers, in the company’s statement.