June 7, 2017
By Mark Terry, BioSpace.com Breaking News Staff
There was a lot of big news at the recent ASCO meeting held in Chicago this weekend, especially in the area of CAR-T therapies for cancer. One of the winners was Bluebird Bio , whose stock has climbed to a 20-month high on data it presented on Monday.
On Monday at ASCO, Bluebird and Celgene announced updated results from their CRB-401 Phase I trial of bb2121. It is an investigational anti-BCMA CAR-T cell therapy. It was evaluated in 18 patients with relapsed/refractory multiple myeloma. All 15 of the patients who could be evaluated responded to treatment. And 89 percent had their tumors shrink, with 27 percent showing no continued presence of tumors.
“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, Bluebird’s chief medical officer, 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.”
Chimeric antigen receptors (CAR) are engineered to target any cancer antigen desired. The engineered T-cells are then reintroduced into the patient programmed to attack specific cancer cells.
Bluebird’s product is targeted at BCMA. What might be concerning to Bluebird and Celgene was a presentation by China’s Nanjing Legend Biotech at ASCO—a company few people had heard of before—of its own CAR-T product that targets BCMA.
Nanjing Legend Biotech’s data was in 35 relapsed, drug-resistant patients with multiple myeloma. Thirty-three, or 94 percent, showed clinical remission that ranged from complete to partial response. According to the presentation, there was a 100 percent objective response rate.
Despite that new competition, a J.P. Morgan analyst Cory Kasimov called Bluebird “the clear winner this weekend” at the ASCO meeting.
He wrote in a note to investors, “To generate efficacy data on this level with an overall very tolerable safety profile is highly impressive, in our view. With more key updates to come in 2017, we would continue to add to positions in BLUE.”
Bluebird Bio is currently trading at $104.40. Shares traded on November 3, 2016 for $41.30, rose to $97.85 on March 15, 2017, and were trading on May 3 for $75.35 before the most recent jump.
One thing that appears attractive in the Bluebird product is its safety profile. CAR-T therapies have been plagued with an adverse side effect known as cytokine release syndrome (CRs), or a transfusion reaction. Essentially it causes the immune system to overreact and results in severe inflammation that can be life-threatening. This doesn’t appear to be an issue with bb2121.
Brian Orelli, writing for The Motley Fool, said, “Theoretically the data should have been priced in since the press release came out before the market closed—and shares were up 8.5 percent Monday—but the company held a webcast Monday evening that seems to have bolstered interest in the data and convinced at least one analyst to upgrade the stock, which are presumably contributing to Tuesday’s additional double-digit gain.”
