Be Biopharma Launches BeCoMe-9 Trial and Showcases B Cell Medicines at ASH 2024

Be Biopharma, Inc. (Be Bio), a clinical-stage company at the forefront of developing Engineered B Cell Medicines (BCMs), recently shared significant advancements at the 66th ASH Annual Meeting held in San Diego, California. The updates included a detailed poster on the ongoing BeCoMe-9 clinical trial and an oral presentation showcasing the capabilities of Be Bio's BCM platform for creating innovative treatments.

The poster on the BeCoMe-9 clinical trial provided insights into the study's design, which focuses on evaluating BE-101 in individuals with Hemophilia B. BE-101 is Be Bio’s leading clinical program and is an autologous BCM therapy engineered to express human factor IX (hFIX). This pioneering therapy aims to deliver sustained levels of hFIX, offering a new therapeutic option for patients with Hemophilia B. Despite the progress in treatment, many patients continue to face regular bleeding episodes, leading to chronic pain and irreversible joint damage. BE-101 is designed to provide long-lasting protection using the patient's own B cells, which can be adjusted and redosed as required. The BeCoMe-9 trial, which is currently enrolling patients, aims to establish clinical proof of concept for both BE-101 and the BCM platform. The U.S. FDA has granted BE-101 Orphan Drug and Fast Track Designations, highlighting its potential to meet significant unmet medical needs.

Joanne Smith-Farrell, PhD, the CEO of Be Bio, stated that 2025 is expected to be a crucial year, as the BeCoMe-9 trial will likely provide clinical proof of concept for BE-101, further advancing the program and demonstrating the transformative potential of their B Cell Medicine platform. The BeCoMe-9 study is designed to assess the safety and clinical efficacy of a single intravenous dose of BE-101 in adults with moderately severe or severe Hemophilia B. The study is divided into two parts: the first part uses an ascending-dose design to identify a safe and effective dose, and the second part will confirm and further characterize the safety and efficacy of BE-101 at the selected dose, including a cohort of adolescents.

Additionally, Be Bio’s oral presentation on December 7 illustrated the versatility of BCMs in providing constant levels of therapeutic proteins for a wide range of diseases without the need for preconditioning. The presentation featured data from the company’s lead candidates: BE-101 for Hemophilia B and BE-102, which is designed to express tissue non-specific alkaline phosphatase (ALP) as a potential treatment for hypophosphatasia (HPP). The presentation also included data from a BCM prototype engineered to express an anti-CD3:CD19 bispecific T cell engager, which showed significant tumor reduction in a patient-derived xenograft model of acute lymphoblastic leukemia.

Rick Morgan, Ph.D., Chief Scientific Officer at Be Biopharma, emphasized that the data validated their platform's capability to develop novel BCMs that express therapeutically relevant proteins, offering transformative potential for treating various genetic diseases, cancers, and other conditions.

Engineered B Cell Medicines represent a new class of cellular therapies, utilizing precision genome editing to create B cells that produce therapeutic proteins. These BCMs have the potential to be durable, redosable, and administered without preconditioning, with broad applications across different protein classes, patient groups, and therapeutic areas.

BE-101 is designed to treat Hemophilia B by enabling continuous expression of active FIX through the insertion of the human Factor IX gene into primary human B cells. This allows for sustained therapeutic FIX activity levels with a single infusion, reducing the need for frequent dosing and potentially lowering bleeding rates and FIX usage.

Be Biopharma, founded in October 2020, is dedicated to revolutionizing treatment for patients with Hemophilia B, other genetic disorders, cancer, and other serious conditions through the development of Engineered B Cell Medicines. Their team comprises scientists, technologists, manufacturing experts, and business innovators, all working towards creating a new class of cell therapies.