PeproMene Bio submits IND for BAFF-R CAR T...'first-in-human' trials
입력 2020-10-29 12:19 수정 2020-10-29 12:19
by Sungmin Kim
PeproMene Bio announced on the 28th, it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to study a novel chimeric antigen receptor (CAR) T cell therapy, PMB-101, the first CAR T cell therapy targeting the B cell-activating factor receptor (BAFF-R) on cancerous cells, for the potential treatment of refractory or relapsed B cell acute lymphoblastic leukemia (r/r B-ALL).
City of Hope has licensed intellectual property relating to PMB-101 under a Licensing and Collaboration agreement with PeproMene. City of Hope will also conduct the proposed first in-human, Phase 1 clinical trial.
The proposed trial is a single-center, open-label trial and will examine the safety and preliminary efficacy of BAFF-R-CAR T cells in cancer patients with r/r/ B-ALL who are ineligible for or have failed CD19-targeted immunotherapy in the U.S.
BAFF (B cell activating factor), a soluble cytokine expressed by B cells, is known for its proliferation and differentiation. In particular, BAFF-R (B cell activating factor receptor) is an excellent potential target for B-cell cancers not only because of its specificity to BAFF, but also for its expression level by lympho-proliferative as well as leukemia-proliferative disorders.
PMB-101 is a novel CAR T cell therapy which has shown synergistic activity in CD-19 resistant or relapsed animal models in preclinical trials. Animal models with CD19 therapy-resistant human-tumors (including Burkitt, mantel cell, and other non-Hodgkin's lymphoma subtypes and acute lymphoblastic leukemia) received BAFF-R CAR T therapy. Remarkable tumor regression and prolonged survival were observed after treatment with these CAR T cells. In animal models with human Burkitt lymphoma, BAFF-R CAR T therapy achieved complete tumor regression with 100% long-term survival. The research was published in Science Translational Medicine.
"City of Hope is excited to leverage the numerous and very promising pre-clinical model outcomes of our investigational CAR-T project and to explore its effect in cancer patients for whom few treatments are currently available," said Larry W. Kwak, M.D., Ph.D., vice president and deputy director of City of Hope's comprehensive cancer center, who along with Hong Qin, Ph.D., are inventors on the licensed intellectual property and Scientific Advisory Board members of PeproMene.
"We are very pleased to announce this important milestone after only three years of the company's existence; we are grateful to the scientific founders for advancing our project and to our shareholders for their support. 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," said Bryan HW Kim, CEO of PeproMene.