Phase 1/2 clinical trial of BE-101 now enrolling hemophilia B patients
First-in-human study testing Be Biopharma's B-cell therapy at 2 US sites
The first-in-human Phase 1/2 clinical trial testing BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B, is now enrolling patients at two sites in the U.S.
BE-101, a first-in-class therapy, aims to address the persistent unmet needs of hemophilia B patients — particularly the burden of ongoing treatment and disease management for the “many people living with hemophilia B [who] continue to experience regular bleeding events,” Be Biopharma stated in a company press release.
The two-part Phase 1/2 trial — dubbed BeCoMe-9 (NCT06611436) — was designed to assess the safety and clinical activity of a single intravenous, or into-the-vein, infusion of BE-101 in adults with moderately severe to severe hemophilia B.
Overall, BeCoMe-9 aims to evaluate how well BE-101 can lead to the production of factor IX (FIX), the clotting protein that’s defective or missing in people with hemophilia B. The therapy’s goal is to reduce the need for ongoing treatment, and improve patient quality of life, according to the company.
“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, CEO of Be Biopharma, said. “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.”
Phase 1/2 clinical trial now recruiting in Michigan, Minnesota
This clinical trial will also serve as a key step in establishing Be Biopharma’s B-cell medicine platform, which the company says could be applied to a range of genetic disorders beyond hemophilia B.
According to Rick Morgan, PhD, Be Biopharma’s chief scientific officer, the “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’s design was detailed in a poster, “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,” presented at the 66th American Society of Hematology Annual Meeting, held Dec. 7-10 in San Diego and virtually.
There are two study sites, both in the Midwest: one at the University of Michigan in Ann Arbor, and the other at the University of Minnesota in Minneapolis. Eligible participants will be adult men receiving prophylaxis, or preventive, treatment.
Hemophilia B is a genetic disorder caused by mutations in the F9 gene that impair the production or activity of FIX. As a result, people with hemophilia B face a heightened risk of prolonged and excessive bleeding.
The traditional approach used for managing the disease is factor replacement therapy, in which patients receive regular infusions of FIX to maintain adequate clotting. While effective, these treatments require lifelong commitment and can be burdensome for patients.
First part of trial will determine safest, most effective dose of Be-101
BE-101, a cutting-edge autologous B-cell therapy, may offer a transformative alternative.
The treatment candidate uses a patient’s own B-cells, which are collected and then genetically modified outside the body. A functional version of the F9 gene is inserted into the genome of these B-cells using advanced gene-editing techniques. Once modified, the cells are expanded and differentiated into plasma cells — specialized B-cells that can rapidly produce large amounts of antibodies. When these engineered plasma cells are infused back into the patient, they are expected to migrate to the bone marrow and begin producing and releasing FIX at stable, therapeutic levels, potentially eliminating the need for regular FIX infusions.
This approach, according to Be Biopharma, represents a leap forward in treating hemophilia B — offering the potential for a long-lasting, personalized solution that could reduce the frequency of bleeding episodes and improve patients’ quality of life.
Following a screening period to assess eligibility, in the first part of BeCoMe-9, up to 18 patients will receive single ascending doses of BE-101 to determine the safest and most effective dose. That’s defined as the dose required for FIX activity to be at or above 15% of normal 28 days following infusion. The selected dose should also minimize the number of patients potentially exposed to subtherapeutic or unsafe doses, per Be Biopharma.
Once this optimal dose is identified, as many as six adult patients will enroll in the trial’s second part, which will confirm and further assess the safety and activity of BE-101’s selected dose.
Additional treatment groups comprising adolescent patients and participants receiving multiple doses of the therapy also are planned.
Trial participants will be followed for 52 weeks, or one year, following BE-101 infusion.
The therapy has been awarded orphan drug and fast track designations by the U.S. Food and Drug Administration. These statuses come with incentives that are expected to help speed a treatment’s development.
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