Be Biopharma announced key updates on its engineered B Cell Medicines (BCMs) platform and the BeCoMe-9 clinical trial at the 66th ASH Annual Meeting. The BeCoMe-9 trial is a Phase 1/2 study evaluating BE-101, a first-in-class BCM designed to sustain Factor IX (FIX) levels in adults with Hemophilia B. This innovative therapy aims to address the unmet needs of approximately 40,000 people globally affected by Hemophilia B, many of whom continue to experience bleeding events despite current treatments.
BE-101: A Novel Approach to Hemophilia B Treatment
BE-101 is an autologous BCM therapy engineered to express human factor IX (hFIX). The therapy is designed to provide long-lasting FIX protection using the patient’s own B cells, with the flexibility to be titratable and redosable as needed. The BeCoMe-9 trial (NCT06611436) is a first-in-human Phase 1/2 trial actively enrolling patients to achieve clinical proof of concept for both BE-101 and the BCM platform. The FDA has granted BE-101 Orphan Drug and Fast Track Designations, underscoring its potential to address important unmet needs.
"This year marks a pivotal milestone for Be Bio as we have transitioned into a clinical stage company, advancing our groundbreaking science into lead programs that aim to deliver new therapeutic options for patients in need," said Joanne Smith-Farrell, PhD, Chief Executive Officer of Be Bio. "In 2025, the BeCoMe-9 trial is poised to generate clinical proof of concept for BE-101, advancing this critical program for patients while demonstrating the transformative potential of our B Cell Medicine platform."
BeCoMe-9 Trial Design
The BeCoMe-9 study is designed to evaluate the safety and clinical activity of a single IV dose of BE-101 in adults with moderately severe or severe Hemophilia B. Part 1 of the study employs an ascending-dose design to determine a safe and effective dose. Part 2 will add a cohort to confirm and further characterize the safety and activity of BE-101 at the selected dose, along with a cohort to include adolescents.
Versatility of B Cell Medicines Platform
Be Bio’s oral presentation highlighted the versatility of BCMs in delivering therapeutic biologics for diverse patient populations and diseases. BCMs are designed to produce constant levels of therapeutic proteins in a durable, redosable, and titratable manner, all without the need for preconditioning. The presentation highlighted data from BE-102, a development candidate engineered to express tissue nonspecific alkaline phosphatase (ALP) as a potential treatment option for hypophosphatasia (HPP). Data from a BCM prototype engineered to express an anti-CD3:CD19 scFv bispecific T cell engager, which demonstrated a significant reduction in tumor burden in a patient-derived xenograft model of acute lymphoblastic leukemia, was also presented.
"These data reinforce the capabilities of our versatile B cell engineering platform to develop novel BCMs that express therapeutically relevant proteins," said Rick Morgan, Ph.D., Chief Scientific Officer, Be Biopharma. "Our BCM platform offers transformative potential to improve the delivery of therapeutic biologics across a wide spectrum of genetic diseases, cancers and other therapeutic areas."
