Be Biopharma, Inc. has received clearance from the U.S. Food and Drug Administration for its Investigational New Drug application for BE-101, a novel treatment for hemophilia B. This milestone marks a significant step forward in the company's efforts to develop Engineered B Cell Medicines (BCMs). The Phase 1/2 clinical trial, named BeCoMe-9, will evaluate the safety and efficacy of BE-101 in adults with moderately severe to severe hemophilia B. Dosing of participants is expected to begin in the second half of 2024.

Be Bio, emphasized the patient-focused approach of the company, stating that BE-101 aims to provide a durable Factor IX replacement therapy in a single dose, offering flexibility without the need for preconditioning. She sees the FDA clearance as a significant milestone for Be Bio's transition into a clinical-stage company.

Dr. Steven Pipe, Medical Director at the University of Michigan, highlighted the potential of BE-101 to address unmet needs in hemophilia B treatment. He noted its potential to provide long-lasting FIX protection derived from the patient's own B cells, potentially transforming care by offering disease modification and early intervention.

Preclinical studies have shown promising results, with BE-101 demonstrating sustained FIX levels and stable engraftment in bone marrow tissue. Additionally, BE-101 has shown redosability, leading to predictable increases in plasma FIX levels.

BE-101 is an autologous first-in-class BCM engineered to incorporate the human FIX gene into primary human B cells, enabling the expression of active FIX for hemophilia B treatment. It offers the potential for sustained therapeutic FIX activity levels with a single infusion, along with the flexibility for redosing if necessary. This could significantly reduce the infusion burden associated with current therapies and potentially decrease bleeding rates and FIX usage.

Hemophilia B affects approximately 1 in 20,000 males and is caused by mutations in the gene encoding the FIX protein. Current treatments require frequent infusions of exogenous FIX, posing challenges for patients. BE-101 offers a promising alternative to address these challenges.

Engineered B Cell Medicines represent a new frontier in cellular therapy, offering the potential for durable, redosable treatments administered without pre-conditioning. This new class of medicines holds promise for various therapeutic areas and patient populations.

 

Source: businesswire.com

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