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.


About GvHD:
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.