Janssen and Legend Biotech reported updated data from a phase 1/2 study of their BCMA-targeted CAR-T therapy in patients with relapsed or refractory multiple myeloma. In the 57 patients who received the treatment, the overall response rate was 88%, with 74% having a complete response.
Janssen announced the data on Monday afternoon during an oral presentation at the annual meeting of the American Society of Hematology. The study involved patients who had already undergone a median of three prior treatments for their cancer, with some patients having received as many as nine treatments. Nearly three-quarters of the patients—74%—had stage 3 multiple myeloma, the most advanced stage of the disease.
Of the 42 patients who had a complete response, 39 of them (68%) tested negative for minimal residual disease (MRD) in their bone marrow. Recent studies suggest that patients with this low level of disease “may be less likely to experience a relapse of their condition than patients with higher levels,” according to the Dana Farber Cancer Institute.
The investigators followed patients for a median of 12 months, finding the median duration of response to be 16 months. The median progression-free survival was 15 months, but for those patients who were MRD negative, this period was even longer at 24 months.
But the study wasn’t without adverse events, the most common of which were pyrexia—fever—and the dreaded cytokine release syndrome (CRS), a dangerous side effect stemming from the CAR-T activating the immune system too strongly. Of those patients who experienced CRS (90%), most of them had low-grade, or mild to moderate reactions, but four patients had severe, or grade 3, CRS.
All but one of the CRS cases were resolved, Janssen said. Seventeen patients died during the study and follow-up period; 14 from disease progression and one from suicide after their disease progressed. The remaining two died of esophagitis and pulmonary embolism and acute coronary syndrome.
The new data build on Legend’s presentation at ASCO 2017, which came six months before Johnson and Johnson forked over $350 million up front to team up on its CAR-T program. J&J inked a deal with the Nanjing, China-based biotech’s U.S. and European subsidiaries to develop, manufacture and sell LCAR-B38M in December last year.
The treatment is known as JNJ-68284528 in the U.S. and EU. At ASCO, researchers reported that 33 out of 35 patients (94%) with relapsed or refractory multiple myeloma were in clinical remission within two months of receiving LCAR-B38M.
“These updated data show the potential of this investigational CAR-T therapy in the treatment of patients with advanced multiple myeloma,” said Sen Zhuang, M.D., Ph.D., VP of oncology clinical development, at Janssen Research & Development.
“Through the ongoing global development program, we aim to further define and characterize the safety and efficacy profile of this BCMA-targeted immunotherapy in the hope of bringing forward a new treatment option for patients with multiple myeloma in the future,” he added.