Be Biopharma, Inc., a pioneer in developing Engineered B Cell Medicines (BCMs), has received IND clearance from the FDA for BE-101, a revolutionary treatment for hemophilia B. This Phase 1/2 clinical trial, BeCoMe-9, will evaluate BE-101’s safety and efficacy in adults with hemophilia B. The trial is expected to begin in late 2024.
BE-101 is an autologous BCM designed to insert the human FIX gene into patients’ B cells, enabling them to produce FIX. This one-time infusion has the potential to provide sustained FIX activity, reducing the frequency of dosing compared to current therapies. The study will assess the safety and effectiveness of BE-101 in improving bleeding control and joint damage.
Joanne Smith-Farrell, CEO of Be Bio, indicated that the IND clearance marks a significant milestone in our mission to provide an innovative treatment for individuals with hemophilia B and that BE-101 has the potential to transform patient care by offering long-lasting FIX protection and reducing the burden of infusions.
Hemophilia B is a bleeding disorder that can lead to severe consequences, including chronic pain and joint damage. Current treatments require frequent infusions to maintain FIX levels, but BE-101 aims to provide a more durable solution.
Preclinical studies have shown that a single dose of BE-101 can deliver sustained FIX levels and stably engraft in bone marrow tissue. Additionally, BE-101’s redosability allows for further adjustments in FIX levels as needed.
If proven safe and effective in adults, BE-101 could also revolutionize treatment for children, potentially mitigating the long-term consequences of hemophilia B and improving their quality of life.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
