Bitterroot Bio Releases Phase 1 BRB-002 Safety and Target Data

Bitterroot Bio, a prominent company in cardio-immunology, has announced promising outcomes from a Phase 1 clinical trial of its innovative therapy, BRB-002. This therapy is designed to address atherosclerotic cardiovascular disease by targeting the CD47 receptor. The study was conducted with healthy participants to examine the safety, tolerability, and engagement of the drug.

The trial was structured as a randomized, placebo-controlled, double-blinded study involving thirty-six healthy individuals. It examined five groups, each receiving a single subcutaneous injection of BRB-002 at varying doses, from 0.1 mg/kg to 5.0 mg/kg. The primary goal was to assess the safety and pharmacokinetic characteristics of BRB-002, alongside its effectiveness in engaging its target.

The results revealed that BRB-002 was safe across all tested doses, with no serious side effects reported. Participants experienced similar rates of adverse events, whether they received the active drug or a placebo. The most frequently observed side effects were headaches and reactions at the injection site. Importantly, there were no significant impacts on blood parameters, and conditions such as anemia, thrombocytopenia, or febrile neutropenia were not observed.

In terms of target engagement, BRB-002 showed a dose-dependent increase in CD47 receptor occupancy, achieving up to 100% occupancy at the highest doses. Preclinical studies had shown that a receptor occupancy of 6-26% was associated with significant reductions in aortic plaque in comparison to control groups.

Craig T. Basson, MD, PhD, Chief Medical Officer at Bitterroot Bio, expressed enthusiasm about the findings, highlighting the drug's safety profile and effective receptor engagement. This data supports the identification of potentially therapeutic doses for an upcoming Phase 2 study, which is set to begin in the first half of the year.

Pavan Cheruvu, MD, Chief Executive Officer at Bitterroot Bio, emphasized the significance of this milestone for the company's efforts to advance BRB-002. Despite existing treatments like lipid-lowering therapies, atherosclerosis patients still face a considerable risk of severe cardiovascular events. The Phase 2 development program will explore BRB-002's potential in reducing this risk by modulating the immune response within inflamed atherosclerotic plaques.

Comprehensive data from this initial human study will be shared at the American College of Cardiology Scientific Session in March 2025. A Phase 2 trial focused on patients with established atherosclerosis is anticipated to start in early 2025.

BRB-002 represents an innovative approach as a protein therapy being tested for atherosclerotic cardiovascular disease. It functions as an immune modulator by blocking the CD47 receptor, known as the "don't eat me" signal, to mitigate atherosclerosis's root causes and reduce inflammatory plaque buildup. The Phase 1 study was executed by Bitterroot Australia Pty Ltd, a subsidiary of Bitterroot Bio, Inc.

Bitterroot Bio is at the forefront of cardio-immunology, examining the connection between the immune system and heart health. The company's research aims to identify crucial immune modulators involved in cardiovascular disease progression, striving to transform patient outcomes through these novel therapeutic strategies.