Be Biopharma Receives FDA Clearance for BE-101 IND Application in Hemophilia B

Be Biopharma Announces FDA Clearance for BE-101 IND Application in Hemophilia B

Be Biopharma, Inc. (“Be Bio”), a pioneer in Engineered B Cell Medicines (BCMs), has announced the U.S. Food and Drug Administration (FDA) clearance of its Investigational New Drug (IND) application for BE-101, a first-in-class BCM aimed at treating hemophilia B. The upcoming Phase 1/2 clinical trial, BeCoMe-9, will be a multi-center, first-in-human dose escalation study to evaluate the safety and preliminary efficacy of BE-101 in adults with moderately severe to severe hemophilia B. Be Bio plans to begin participant dosing in the second half of 2024.

Joanne Smith-Farrell, Chief Executive Officer, stated, “BE-101 aspires to offer a durable Factor IX replacement therapy with flexible dosing, using a simple infusion of a patient’s own B cells without preconditioning. The IND clearance marks a significant milestone in our mission to establish a transformative new standard of care for hemophilia B and propels Be Bio into the clinical-stage arena.”

Dr. Steven Pipe, Medical Director of the Pediatric Hemophilia and Coagulation Disorders Program at the University of Michigan, highlighted the potential impact of BE-101, noting its capacity to provide long-lasting FIX protection through the patient’s own B cells, with the flexibility of being titratable and redosable. He emphasized the transformative potential of BE-101, especially if it proves safe and effective in adults, potentially enabling early intervention in children.

Preclinical studies of BE-101 have shown that a single dose can achieve active and sustained FIX levels, confirming the expected biodistribution of FIX-expressing BCMs in bone marrow tissue. Additionally, BE-101 has demonstrated predictable increases in plasma FIX levels upon redosing.

About BE-101

BE-101 is an autologous B Cell Medicine (BCM) designed to insert the human FIX gene into primary human B cells, facilitating the expression of active FIX for hemophilia B treatment. BE-101 aims to provide sustained therapeutic FIX activity with a single infusion and the flexibility for redosing. This could potentially reduce the frequency of infusions required with current FIX replacement therapies, significantly lowering the annualized bleeding rates and FIX usage.

About Hemophilia B

Hemophilia B is an X-linked recessive bleeding disorder affecting approximately 1 in 20,000 males. It results from mutations in the gene encoding the FIX protein, a crucial enzyme in the coagulation cascade, leading to prolonged bleeding episodes. Hemophilia B can cause spontaneous bleeding or bleeding following injuries or surgery, with bleeds occurring internally or externally. Current treatments involve frequent prophylactic infusions of exogenous FIX, which have a short biological half-life.

About Engineered B Cell Medicines

Engineered B Cell Medicines (BCMs) leverage the B cell’s natural ability to produce proteins at high rates over long periods. Precision genome editing enables the engineering of B cells to produce therapeutic proteins, creating a new class of durable, allogeneic, redosable cellular medicines that can be administered without pre-conditioning. BCMs hold the potential to revolutionize therapeutic biologics across various protein classes, patient populations, and therapeutic areas.

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