Bitterroot Bio Reports Data on Safety and Target Engagement from Phase 1 First-in-Human Study of BRB-002 in Healthy Volunteers

• BRB-002 was safe at all doses tested, with no SAEs reported
• Phase 2 proof-of-concept study in patients with established atherosclerosis is expected to initiate in 1H 2025

PALO ALTO, Calif. and NEEDHAM, Mass. and MELBOURNE, Australia, Jan. 08, 2025 (GLOBE NEWSWIRE) — Bitterroot Bio, a leader in developing innovative medicines in the field of cardio-immunology, today announced positive results from a recently completed Phase 1 study of BRB-002 in healthy volunteers. BRB-002 is a first-in-class, immune modulating protein therapy that targets CD47 for treatment of atherosclerotic cardiovascular disease.

This randomized, placebo-controlled, double-blinded, single ascending dose study evaluated the safety, tolerability and target engagement of BRB-002 in thirty-six (36) healthy volunteers. The trial studied five cohorts, with single subcutaneous doses of BRB-002 ranging from 0.1 mg/kg up to 5.0 mg/kg. The primary objective of the study was to evaluate the safety and tolerability of BRB-002, as well as its pharmacokinetic properties and target engagement profile.

Key Results

Safety: BRB-002 was safe at all doses tested with no serious adverse events described. Rates of treatment emergent adverse events were similar between active and placebo-treated participants, with the most common adverse events being headaches and injection site reactions. Notably, there was no clinically significant impact on hematologic parameters and no observed anemia, thrombocytopenia or febrile neutropenia.

Target engagement: CD47 receptor occupancy increased in a dose-dependent manner, ranging up to 100% receptor occupancy at the highest doses tested. In preclinical models of atherosclerosis, CD47 receptor occupancy of 6-26% correlated with meaningful reductions in aortic plaque burden relative to controls.

“We are thrilled to share these results from our Phase 1 study that demonstrated the remarkable safety of BRB-002,” said Craig T. Basson, MD, PhD, Chief Medical Officer at Bitterroot Bio. “We are also pleased that this profile is accompanied by robust and long-lasting receptor occupancy to allow us to identify potentially therapeutic doses for our Phase 2 proof-of-concept study that is planned to start in the first half of this year.”

“This is an important milestone for Bitterroot Bio in our journey to advance BRB-002, a first-in-class, immune-modulating molecule that targets the biology of atherosclerotic plaque,” said Pavan Cheruvu, MD, Chief Executive Officer at Bitterroot Bio. “Despite current treatment options such as lipid-lowering therapy, patients with atherosclerosis remain at significant residual risk for life-threatening cardiovascular events. We look forward to executing our Phase 2 development program and assessing how BRB-002 can potentially decrease this residual cardiovascular risk by modulating the macrophage response within inflamed atherosclerotic plaque.”

Detailed data from the Phase 1 first-in-human study of BRB-002 will be presented at the American College of Cardiology Scientific Session in March 2025.

A Phase 2 proof-of-concept trial of BRB-002 in patients with established atherosclerosis is expected to initiate in the first half of 2025.

About BRB-002
BRB-002 is a novel protein therapy that is being evaluated as an investigational agent in atherosclerotic cardiovascular disease. BRB-002 is an immuno-modulatory agent that inhibits the CD47 cell surface receptor (“don’t eat me” signal) to ameliorate the underlying causes of atherosclerosis and reduce inflammatory plaque burden. A Phase 1 clinical study of BRB-002 was completed by Bitterroot Australia Pty Ltd, a wholly owned subsidiary of Bitterroot Bio, Inc.

About Bitterroot Bio
Bitterroot Bio, Inc. is a pioneer in the field of cardio-immunology, which investigates the interplay between the immune system and cardiovascular health. Bitterroot Bio’s research seeks to uncover critical roles that immune modulators play in the progression of cardiovascular disease. By targeting these diseases in this novel way, Bitterroot Bio’s mission is to transform the lives of patients suffering from cardiovascular diseases.

For more information, please visit https://www.brbio.com

Media Contact: 
Emily Dale (Bitterroot Bio), media@brbio.com