The FDA has granted fast track designation to the investigational allogenic CAR T-cell therapy product CB-011 for the treatment of patients with relapsed or refractory multiple myeloma, according to a press release from Caribou Biosciences, Inc.1

"Fast track designation for CB-011 allows us instrumental interactions with the FDA as we progress our clinical development and regulatory plans for CB-011, according to the manufacturers of CB-011.

The FDA originally cleared an investigational new drug application for CB-011 in this patient population in November 2022, permitting investigators to advance with their evaluation of the agent in the phase 1 CaMMouflage trial (NCT05722418).2

According to findings from a poster session presented at the 2023 Tandem Meeting, CB-011 demonstrated potent anti-tumor activity in vitro and enhanced survival in multiple myeloma xenograft models.3 Additionally, investigators observed no adverse safety signals associated with treatment in vitro.

Fast track designation for CB-011 allows us instrumental interactions with the FDA as we progress our clinical development and regulatory plans for CB-011, Syed Rizvi, MD, chief medical officer of Caribou, said in the press release. Our goal is to develop CB-011 as a readily available off-the-shelf treatment option for patients with relapsed or refractory multiple myeloma to overcome the need for apheresis or bridging therapy, variable quality and long manufacturing timelines, manufacturing failures, or the inability to bear the burden of treatments that require frequent dosing over several months.

CB-011 is an allogeneic CAR T-cell therapy targeting BCMA that was engineered with Cas12a chRDNA technology. Investigators believe its design allows it to enable anti-tumor activity through an immune cloaking strategy that removes the B2M protein and inserts a B2MHLA-E fusion protein.

Investigators of the open-label, multi-center phase 1 CaMMouflage trial are assessing CB-011 as treatment for adult patients with relapsed or refractory multiple myeloma. Part A will include a dose escalation of CB-011 in ascending doses based on a traditional 3+3 design. In part B, up to 30 patients will receive CB-011 at the maximum tolerated dose or recommended phase 2 dose determined in part A.

The primary end point of part A is the number of patients who experience dose limiting toxicities. In part B, the primary end point is the overall response rate based on International Myeloma Working Group (IMWG) criteria.

Patients 18 years and older with a documented diagnosis of relapsed or refractory multiple myeloma and measurable disease per IMWG criteria are eligible to enroll on the trial. Additional inclusion criteria include receipt of at least 3 prior lines of therapy including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody; having an ECOG performance status of 0 or 1; and adequate hematologic, hepatic, renal, pulmonary, and cardiac function.

Patients who received prior treatment with a CAR T-cell therapy or autologous stem cell transplant within 6 weeks prior to undergoing lymphodepletion are not eligible for enrollment. Patients are also unsuitable for enrollment if they have received allogeneic stem cell transplant within 6 months prior to lymphodepletion, known active or prior central nervous system involvement, stroke or seizure within 6 months of study entry, or are seropositive or have a history of human immunodeficiency virus.

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FDA Grants FTD to Off-the-Shelf CAR T in R/R Multiple Myeloma - Cancer Network