News | May 28, 2024

Be Biopharma Announces FDA Clearance Of IND Application For BE-101 In Hemophilia B

Cambridge, MA (BUSINESS WIRE) - Be Biopharma, Inc. (“Be Bio”), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs), today announced the clearance of its Investigational New Drug application (IND) from the U.S. Food and Drug Administration (FDA) for BE-101, a first-in-class BCM in development for the potential treatment of hemophilia B. The Phase 1/2 clinical trial, BeCoMe-9, is a multi-center, first-in-human dose escalation study designed to assess the safety and preliminary efficacy of BE-101 in adult participants with moderately severe to severe hemophilia B. Be Bio expects to begin dosing participants in the second half of 2024.

“Be Bio was founded to develop transformative medicines, with eyes locked on the patient. Adults and children with hemophilia B have long sought a single dose Factor IX replacement therapy with extreme durability while retaining the dosing flexibility of the current standard of care. BE-101 seeks to deliver active and sustained levels of Factor IX using a patient’s own B cells in a simple infusion that requires no preconditioning,” said Joanne Smith-Farrell, Chief Executive Officer. “The IND clearance is a major milestone in our journey to offer a new, transformative standard of care for people with hemophilia B, and drives Be Bio’s transition to becoming a clinical-stage company.”

“Hemophilia B is not yet solved as bleeding events can create long-term consequences, such as significant chronic pain and irreversible joint damage,” said Dr. Steven Pipe, Medical Director of the Pediatric Hemophilia and Coagulation Disorders Program and Medical Director of the Special Coagulation Laboratory at the University of Michigan. “BE-101 has the potential to be disease modifying by providing long-lasting FIX protection using the patient’s own B cells while providing the option to be titratable and redosable as necessary. If proven safe and effective in adults, the ability to treat children and transform the phenotype as early as possible would be a game changer.”

In preclinical studies, a single dose of BE-101 has demonstrated the ability to deliver active and sustained FIX levels. The data confirmed the expected biodistribution of FIX-expressing BCMs in bone marrow tissue, where they engraft stably over time. Additionally, the redosability of BE-101 has been demonstrated, resulting in a predictable increase in plasma FIX levels.

About BE-101
BE-101 is an autologous first-in-class B Cell Medicine (BCM) that is engineered to insert the human FIX gene into primary human B cells, allowing for expression of active FIX for the treatment of hemophilia B. BE-101 has the potential to express sustained therapeutic FIX activity levels with a single infusion with the flexibility to be re-dosed, if needed. The potential to maintain therapeutic FIX activity levels while reducing dosing frequency associated with current FIX replacement regimens would address the considerable infusion burden associated with current therapies and potentially drive significant reductions in the annualized bleeding rates and FIX usage.

About Hemophilia B
Hemophilia B is an X-linked recessive bleeding disorder that affects approximately 1:20,000 males. It is caused by mutations in the gene that encodes for the FIX protein, an essential enzyme in the coagulation cascade. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery.1 People with hemophilia B bleed longer than other people. Bleeds can occur internally, into joints and muscles, or externally, from minor cuts, dental procedures or trauma.2 While an adeno-associated virus (AAV) vector-based gene therapy has been approved for some adults as a potential new option, the current standard of care and only treatment for children remains prophylactic administration of exogenous FIX derived from recombinant protein. The short biological half-life of FIX requires frequent infusions to maintain therapeutic levels.

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, and administered without pre-conditioning. The promise of BCMs could transform therapeutic biologics with broad application — across protein classes, patient populations and therapeutic areas.

About Be Biopharma
Be Biopharma (“Be Bio”) is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies. Be Bio was founded in October 2020 by B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D., from Seattle Children’s Research Institute. Be Bio is backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children’s Research Institute and others. Since our founding, Be Bio’s investors have committed over $180 million to enable the Company to re-imagine medicine based on the power of B cell therapy. For more information, please visit us at Be.Bio and our LinkedIn page.


1What is Hemophilia? U.S. Centers for Disease Control and Prevention. Accessed November 9, 2023

2Hemophilia B. National Bleeding Disorders Foundation. Accessed November 9, 2023

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