“Despite recent advances, patients with hemophilia B still suffer from bleeding events, joint damage and chronic pain,” said Joanne Smith-Farrell, Ph.D., Chief Executive Officer of Be Bio. “BE-101 has the potential to be the first and only Factor IX replacement therapy that is extremely durable, re-dosable and titratable, representing an opportunity to dramatically improve the treatment paradigm in hemophilia B. The Orphan Drug Designation reinforces the potential of this therapy and underscores the need for improved therapeutic options for these patients.”

The FDA grants Orphan Drug Designation to drugs or biologics designed to treat rare diseases or conditions affecting fewer than 200,000 people in the United States. This designation offers several significant advantages, including a seven-year period of exclusive marketing rights following approval, exemption from user fees, and eligibility for tax credits on qualified clinical trials.

About BE-101

BE-101 is a 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. Be Bio expects to initiate a phase 1/2 study, BeCoMe-9, evaluating BE-101 in patients with severe or moderately severe Hemophilia B in the second half of 2024.

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.

References

¹What is Hemophilia? U.S. Centers for Disease Control and Prevention. Accessed November 9, 2023
https://www.cdc.gov/ncbddd/hemophilia/facts.html
²Hemophilia B. National Bleeding Disorders Foundation. Accessed November 9, 2023
https://www.hemophilia.org/bleeding-disorders-a-z/types/hemophilia-b

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