On December 08, 2024 Be Biopharma, Inc. ("Be Bio"), a clinical-stage company pioneering engineered B Cell Medicines (BCMs), reported two key updates at the 66th ASH (Free ASH Whitepaper) Annual Meeting in San Diego, CA (Press release, Be Biopharma, DEC 8, 2024, View Source [SID1234648885]). These updates include a Trial in Progress poster detailing the BeCoMe-9 clinical trial and an oral presentation highlighting the potential of Be Bio’s BCM platform to create innovative programs with best-in-class profiles.
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"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."
The Trial in Progress poster highlights the design of Be Bio’s BeCoMe-9 clinical trial, evaluating BE-101 in patients with Hemophilia B. BE-101, the Company’s lead clinical program, is an autologous BCM therapy engineered to express human factor IX (hFIX). This innovative therapy aims to offer a transformative new therapeutic option by delivering sustained hFIX levels, addressing the needs in patients with hemophilia B, a bleeding disorder affecting approximately 40,000 people globally. Despite advancements, many people living with hemophilia B continue to experience regular bleeding events that can lead to chronic pain and irreversible joint damage. BE-101 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 first-in-human Phase 1/2 trial is actively enrolling patients and aims to achieve clinical proof of concept for both BE-101 and the BCM platform. BE-101 has been granted Orphan Drug and Fast Track Designations by the FDA, 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."
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. The study consists of two parts. Part 1 employs an ascending-dose design, enabling the stepwise evaluation of increasing dose levels to determine a safe and effective dose. Once this dose is identified, 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. Details of the poster presentation are as follows:
Abstract Title: BeCoMe–9: A Phase 1/2 Dose Escalation and Expansion Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B
Session Name: 322. Hemophilia A and B: Clinical and Epidemiological: Poster II
Publication Number: 2593.1
Date: Sunday, December 8, 2024
Time: 6:00 PM – 8:00 PM PT
Session Room: San Diego Convention Center, Halls G-H
Be Bio’s oral presentation on December 7 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 the Company’s two lead candidates, BE-101, a clinical-stage BCM engineered to express hFIX as a potential new treatment for Hemophilia B, and BE-102, a development candidate engineered to express tissue nonspecific alkaline phosphatase (ALP) as a potential treatment option for hypophosphatasia (HPP). The presentation also included 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.
"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."
About Engineered B Cell Medicines – A New Class of Cellular Medicines
The B cell is a powerful cell that produces thousands of proteins per cell per second at constant levels, over decades. Precision genome editing can now be used to engineer B Cells that produce therapeutic proteins of interest, driving a new class of cellular medicines – Engineered B Cell Medicines (BCMs) – with the potential to be durable, allogeneic, redosable, titratable and administered without pre-conditioning. The promise of BCMs could transform therapeutic biologics with broad application — across diverse protein classes, patient populations and therapeutic areas.
About BE-101
BE-101 is a first-in-class engineered B Cell Medicine (BCM) designed to treat Hemophilia B by inserting the human Factor IX (FIX) gene into primary human B cells, enabling continuous expression of active FIX. BE-101 offers the potential for sustained therapeutic FIX activity levels with a single infusion, with the flexibility to be titrated and/or re-dosed, and without the need for preconditioning. By maintaining sustained and constant therapeutic FIX activity levels while reducing dosing frequency required by current FIX replacement regimens, BE-101 could alleviate the considerable infusion burden faced by people with Hemophilia B while potentially lowering annualized bleeding rates and FIX usage. The US FDA cleared the BE-101 IND in May of 2024, and has granted the program Fast Track and Orphan Designations. The Phase 1/2 BeCoMe-9 Trial is open for enrollment and further details of the trial can be found at www.clinicaltrials.gov under NCT identifier: NCT06611436.