Bitterroot Bio announced positive Phase 1 results for BRB-002, a first-in-class immune-modulating protein therapy targeting CD47 for atherosclerotic cardiovascular disease. The study in healthy volunteers showed BRB-002 was safe at all tested doses, with no serious adverse events, and demonstrated dose-dependent target engagement. A Phase 2 proof-of-concept study in patients with established atherosclerosis is anticipated to begin in the first half of 2025.
This positive Phase 1 data is a crucial step towards addressing the significant residual risk of cardiovascular events faced by atherosclerosis patients, even with existing treatments like lipid-lowering therapy. The demonstration of safety and target engagement in humans de-risks further development and supports the rationale for investigating BRB-002’s potential to reduce this risk. This successful first-in-human study provides valuable early clinical validation for Bitterroot Bio’s cardio-immunology approach, which aims to modulate the immune system’s role in cardiovascular disease.
The Phase 1 study demonstrated a dose-dependent increase in CD47 receptor occupancy, reaching up to 100% at the highest doses. Importantly, preclinical models have indicated that even lower occupancy levels (6-26%) correlated with meaningful plaque reduction. The absence of serious adverse events and concerning hematological effects, like anemia or thrombocytopenia, further strengthens the drug’s profile.
This positive Phase 1 data positions BRB-002 for advancement into Phase 2 trials in patients with atherosclerosis. Successful results in the upcoming Phase 2 trial would not only validate the therapeutic potential of BRB-002, but also represent a significant advance in the field of cardio-immunology, potentially offering a new treatment avenue for atherosclerotic cardiovascular disease. The results of this study will be presented at the American College of Cardiology Scientific Session in March 2025, offering a more detailed insight into the findings.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
