First Approach for the Prevention of Acute Immune Diseases
NJA-730, NapaJen’s lead program, is designed for the prevention of acute immune diseases associated with bone marrow transplants, such as acute graft-versus-host disease (GvHD). The novel oligonucleotide drug candidate targets CD40, a protein that is found on antigen-presenting immune cells and triggers their activation. By selectively and efficiently silencing the activity of CD40, NJA-730 is expected to prevent the body’s immune response to organ transplantation.
NJA-730 Development Program
NapaJen has completed dosing of all subjects in its Phase 1 study in Australia, with no reporting of dose-limiting toxicities or other noteworthy findings at any evaluated doses. The company intends to initiate a proof-of-concept Phase 2 clinical trial of NJA-730 in acute GvHD in 2021 in both United States and Australia.
Results from a preclinical study in an animal model of hematopoietic stem cell transplantation demonstrated statistically significant improvements in mouse survival rate up to 45 days following administration of a single injection of NJA-312, a murine orthologue of NJA-730 as compared to the vehicle control, suggesting the treatment’s potentially preventative nature in GvHD.
While NJA-730 is initially being developed as a prophylaxis for acute GvHD, evaluation of the compound has the potential to be expanded to several other inflammatory immunoreactive conditions.
GvHD is an immune reaction by which donor lymphocytes originating from organ transplantation attack patients as heterogenous, potentially resulting in a range of damaging health impacts. It can take the form or acute GvHD which occurs in the weeks immediately following transplantation, or chronic GvHD, which occurs several months post-transplantation and is typically less serious than acute GvHD.
Current preventative therapies for acute GvHD sometimes induce relapse of malignancies or infections in patients. As such, there is a critical unmet need for novel GvHD therapeutics capable of safely preventing the disease.