FDA clears IND application for CAR-T targeting B-cell-activating factor receptor in ALL
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The FDA cleared an investigational new drug application for PMB-101, a chimeric antigen receptor T-cell therapy, for the treatment of refractory or relapsed B-cell acute lymphoblastic leukemia.
PMB-101 (PeproMene Bio) is an investigational allogeneic CAR T-cell therapy that targets the B-cell-activating factor receptor (BAFF-R), a soluble cytokine expressed on the surface of cancer cells. BAFF-R was shown to be expressed on human B-cell tumors in preclinical studies, including tumor models that were susceptible to CD19 antigen loss.
The CD19 antigen is the target used by the FDA-approved CAR T-cell therapies indicated for advanced B-cell malignancies and by blinatumomab (Blincyto, Amgen), a bispecific CD19-directed CD3 T-cell engager. Most patients who experience disease progression after CD19-targeted immunotherapy do so because of CD19 antigen loss on the surface of cancer cells.
Researchers at City of Hope developed the BAFF-R CAR technology and have licensed intellectual property relating to PMB-101 under an agreement with PeproMene.
"City of Hope is excited to leverage the numerous and very promising preclinical model outcomes of our investigational CAR-T project and to explore its effect in patients [with cancer] for whom few treatments are currently available,” Larry W. Kwak, MD, PhD, vice president and deputy director of City of Hope's comprehensive cancer center, said in a PeproMene-issued press release.
Kwak is also a co-inventor of the technology and member of PeproMene’s scientific advisory board.
The FDA based the clearance on results of two preclinical studies that demonstrated the ability of BAFF-R CAR T cells to target models of B-cell malignancies that were susceptible to CD19 antigen loss, in addition to the investigators’ ability to successfully manufacture the CAR T cells for clinic-ready settings using current good manufacturing practices.
The IND clearance allows PeproMene to begin a phase 1, first-in-human clinical trial for PMB-101. City of Hope will conduct the single-center, open-label trial for the investigational therapy. The study will determine the safety and preliminary efficacy of BAFF-R CAR T cells for patients with relapsed or refractory B-cell ALL who experienced disease progression after receiving previous CD19-targeted immunotherapy.
"We are very pleased to announce this important milestone after only 3 years of the company's existence. We are grateful to the scientific founders for advancing our project and to our shareholders for their support,” Bryan H.W. Kim, PhD, CEO of PeproMene, said in the release. “We look forward to collaborating closely with leading clinical investigators at City of Hope and the FDA to validate the safety of PMB-101 in humans in a controlled clinical setting.”
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