bluebird bio (BLUE) Reports Interim Phase 1 Dose Escalation Data for its Anti-BCMA CAR T Product Candidate

November 30, 2016 5:28 PM EST

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bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced that interim data from its ongoing Phase 1 clinical study of bb2121, the company’s investigational anti-BCMA CAR T cell product candidate in patients with relapsed/refractory multiple myeloma, will be presented on Thursday, December 1, 2016 at the 28th EORTC-NCI-AACR Molecular Targets and Cancer Therapeutics Symposium in Munich, Germany. bluebird bio is developing bb2121 in collaboration with Celgene Corporation.

“We are pleased that these early data from our ongoing Phase 1 study of bb2121 demonstrate objective anti-tumor responses in heavily pre-treated patients with multiple myeloma, with all patients in the 15.0 x 107 and 45.0 x 107 CAR+ T cell dose cohorts achieving responses, including among them, patients with stringent complete responses and elimination of minimal residual disease,” said David Davidson, M.D., chief medical officer, bluebird bio. “We are also encouraged by the safety profile to date, particularly the lack of severe cytokine release syndrome or neurotoxicity. In light of these positive data, and thanks to the multiple participating clinical sites and centralized manufacturing infrastructure we and our partner Celgene have built for this program, we anticipate efficiently completing the dose escalation stage of the trial and initiating the expansion cohort.”

Clinical remissions and limited toxicity in a first-in-human multicenter study of bb2121, a novel anti-BCMA CAR T cell therapy for relapsed/refractory multiple myeloma (Abstract #14, LBA)

Presenter: Yi Lin, M.D., Ph.D., Assistant Professor of Medicine and Oncology, Mayo Clinic Division of Hematology, Rochester, MNDate: Thursday, December 1, 2016, 18:00 CET (12:00 pm ET)Session: Plenary Session 7

The open-label Phase 1 CRB-401 study (NCT02658929) is investigating the administration of bb2121 anti-BCMA CAR T cells in patients with relapsed and/or refractory multiple myeloma. The primary endpoint of the study is incidence of adverse events (AEs) and abnormal laboratory test results, including dose-limiting toxicities (DLTs). The study also seeks to assess disease-specific response criteria including: complete response (CR), very good partial response (VGPR), and partial response (PR) according to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma. The study also seeks to determine the recommended dose for further clinical trials. Patients on study were heavily pre-treated, with a median of six prior therapies (range: 5 - 13). As of the November 18th, 2016 data cut-off, 11 patients had been enrolled and dosed in four dose cohorts: 5.0 x 107, 15.0 x 107, 45.0 x 107 and 80 x 107 CAR+ T cells. All 11 dosed patients were evaluable for safety, and the first nine patients (5.0 x 107, 15.0 x 107, 45.0 x 107 dose cohorts) have undergone their first multiple myeloma tumor restaging and were evaluable for efficacy. This study is currently enrolling patients at seven sites in the U.S., with an anticipated total enrollment of 50 patients.

Patients received a conditioning regimen of cyclophosphamide and fludarabine, followed by an infusion of bb2121 anti-BCMA CAR T cells. The CAR T cells were produced from each patient’s own blood cells, which were modified using a lentiviral vector encoding the anti-BCMA CAR.

Results, as of November 18th, 2016 Data Cut-off:

Cohort 1 2 3
CAR+ T Cell dose 5.0 x 107 15.0 x 107 45.0 x 107

Overall ResponseRate in cohort

33% 100% 100%
Best Response PD

SD

PR

sCR (time to response: 2 months)

sCR* (time to response: 4 months)

VGPR*

*Both patients witha minimal residualdisease (MRD)assessment atMonth 1 were MRDnegative

PR

PR

PR

All patients in cohorts 2 and 3 with bonemarrow involvement at baseline had nodetectable multiple myeloma cells in theirbone marrow on Day 14 or beyond

Median PriorLines of Therapy

6 (range: 5-13); all patients had a prior autologous stem celltransplant, as well as prior exposure to a proteasome inhibitor andan immunomodulatory agent; 64 percent of patients hadpreviously received daratumumab or CD38 antibody

Safety

No dose-limiting toxicities and no Grade 3 or higherneurotoxicities or Grade 3 or higher cytokine release syndrome(CRS) have been observed. No patients received tocilizumab orsteroids.

Investor Webcast Information

bluebird bio will host a conference call to discuss these data at 8:00 a.m. ET tomorrow, December 1, 2016. The live webcast can be accessed under "Calendar of Events" in the Investors and Media section of the company's website at www.bluebirdbio.com. The webcast will be available for replay for 30 days on the company website. Alternatively, investors may listen to the call by dialing (844) 825-4408 from locations in the United States or (315) 625-3227 from outside the United States. Please refer to conference ID number 27009736.



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