Investors Sigh With Relief as bluebird bio, Celgene's CAR-T Candidate Keeps Multiple Myeloma Patients Relapse Free
June 5, 2017
By Mark Terry, BioSpace.com Breaking News Staff
Although a lot of attention went to unexpected data by China’s Nanjing Legend Biotech, Bluebird Bio and Celgene had CAR-T data of their own to brag about.
At the American Society of Clinical Oncology (ASCO) Annual Meeting held in Chicago this weekend, a number of companies presented data from studies of CAR-T in several different oncology indications. Bluebird and Celgene presented updated results from the ongoing CRB-401 Phase I trial of bb2121. The compound, which focuses on BCMA antigens, is being evaluated in 18 patients with relapsed/refractory multiple myeloma.
“The heavily pretreated, relapsed/refractory patients in this study have few effective treatment options, highlighting the importance of this interim data,” said Michael Pehl, Celgene’s president for Hematology and Oncology, in a statement. “All patients previously underwent autologous HSCT, and received a median of 7 lines of prior therapy. The consistency, depth and durability of these patients’ responses coupled with a manageable safety profile is very exciting, and we believe will provide hope for patients in this setting. Efforts are underway to advance the development of bb2121 for patients with relapsed/refractory multiple myeloma.”
The response rate in the trial was 100 percent, with zero patients relapsing. There were, as is often the case with CAR-T therapies, issues with cytokine release syndrome (CRs); however, in Bluebird’s trial, only two patients experienced grade 3 CRs, and were resolved within 24 hours.
The trial enrolled 21 MM patients that were no longer responding to a prior stem cell transplant and had a median of seven prior therapies. In other words, these patients were very sick and not responding to multiple therapies.
Eighteen of the patients were treated with one of three escalating “active” doses of the bb2121. Of them, 15 could be evaluated as of May 4, which was the cutoff date. The remaining three patients were treated with a fourth dose, which was the lowest dose.
Of the 15 patients, four (27%) had a complete response. Seven (47%) had a very good partial response. The remaining four were in a partial response.
On Day 14 or beyond, all 15 patients with bone marrow involvement at baseline did not have detectable MM cells. Four patients that could be evaluated for Minimal Residual Disease (MRD) were all MRD-negative.
There are quite a number of companies developing their own CAR-T products, including Novartis , Kite Pharma and Juno Therapeutics . However, all of them are developing CAR-T therapies for different indications than multiple myeloma, and that focus on different antigens.
Which is why the news about the little-known Nanjing Legend Biotech was so significant for Bluebird. Both companies are focused on multiple myeloma patients and both are developing CAR-T products that focus on BCMA. The actual technology is a little different, however.
One difference in the studies, as well, is that apparently the Bluebird Bio study was in far sicker patients than those in the Legend trial. They both, however, had stellar results with similar experienced with cytokine release syndrome.
“It is impressive to see objective responses in all patients treated at dose levels of 150 X 106 CAR+ T cells or higher in such a heavily pretreated population, including those with high tumor burden,” said David Davidson, chief medical officer of Bluebird, in a statement. “We are encouraged by the duration and depth of responses, and pleased that the safety profile remains readily manageable. Although these data are still early, it is encouraging that no patient in the active dose cohorts has had myeloma progression. In light of these results, we look forward to initiating the expansion phase of the CRB-401 study in the coming months.”
Investors were also apparently happy with the news. Bluebird Bio umped to a current trading price of $91.80, up from Friday’s $82.68.