Jasper Therapeutics announces follow-up data from briquilimab study

Jasper Therapeutics announced that new follow-up data from Jasper’s investigator-sponsored study of briquilimab as a conditioning agent in the treatment of Fanconi Anemia were presented in a poster presentation at the 2023 Tandem Meetings: Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR taking place in Orlando, Florida. The study is a Phase 1/2 clinical trial utilizing briquilimab to treat FA patients in bone marrow failure requiring allogeneic transplant with non-sibling donors. The objective of the study is to develop cell therapy, which enables blood and immune reconstitution in FA patients with decreased toxicity, by using briquilimab as a part of conditioning which eliminates the need for busulfan chemotherapy or total body irradiation. In the follow-up data series presented, 100% total donor chimerism was achieved through six months for the first patient and at three months for the second patient. Neutrophil engraftment was reached on day 11 for both patients and platelet engraftment was achieved on days 14 and 9 for the first and second patient, respectively. Briquilimab was cleared by day 9 after dosing in both patients and no treatment-related adverse events or toxicities were observed. No veno-occlusive disease or graft-versus-host disease has been observed. Initial data from this study were previously presented at the annual conference of the Inborn Errors Working Party, a research group of the European Society of Blood and Marrow Transplantation, in September 2022. Allogeneic hematopoietic stem cell transplantation offers a potential cure for many diseases, including FA. It is also the only proven treatment for the bone marrow failure that occurs in a majority of patients with FA. However, current allo-HSCT protocols require conditioning with genotoxic chemotherapy and/or irradiation leading to toxicities, including mucositis, end organ damage, infertility and secondary malignancies. Furthermore, allo-HSCT can also be complicated by graft-versus-host disease and serious infections. Patients with FA are particularly vulnerable to such toxicities due to their inherent inability to DNA repair defects.