FDA Awards Orphan Drug Status to BE-101 for Hemophilia B Treatment

Be Biopharma Inc. ("Be Bio"), based in Cambridge, Massachusetts, has secured Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for BE-101, an innovative B Cell Medicine (BCM) targeting Hemophilia B. This milestone sets the stage for Be Bio to commence its Phase 1/2 clinical trial, BeCoMe-9, in the latter half of 2024, focusing on adults with severe or moderately severe Hemophilia B.

Joanne Smith-Farrell, Ph.D., CEO of Be Bio, highlighted the ongoing challenges faced by Hemophilia B patients, including bleeding episodes, joint damage, and chronic pain. BE-101 could potentially revolutionize treatment as the first durable, re-dosable, and titratable Factor IX therapy, promising significant improvements for patients. The Orphan Drug Designation emphasizes the necessity for enhanced therapeutic options in this field.

The FDA grants Orphan Drug Designation to treatments for rare conditions affecting fewer than 200,000 individuals in the United States. This designation includes several benefits, such as seven years of exclusive marketing rights post-approval, exemption from user fees, and eligibility for tax credits on qualified clinical trials.

BE-101 represents a novel class of BCMs, designed to insert the human FIX gene into primary human B cells, enabling the expression of an active FIX protein for Hemophilia B treatment. This approach could sustain therapeutic FIX activity levels with a single infusion and offers the possibility of re-dosing when required. By potentially reducing the frequency of infusions necessary to maintain therapeutic FIX levels, BE-101 aims to alleviate the considerable burden associated with current FIX replacement therapies, potentially lowering annual bleeding rates and FIX usage. Be Bio is gearing up to launch the BeCoMe-9 study in the second half of 2024.

Hemophilia B is an X-linked recessive bleeding disorder impacting about 1 in 20,000 males, attributed to mutations in the gene responsible for the FIX protein, a crucial enzyme in the coagulation cascade. This deficiency results in prolonged bleeding, which can occur spontaneously or after injuries or surgeries. Bleeds may be internal, affecting joints and muscles, or external, from minor cuts or trauma. Although an adeno-associated virus (AAV) vector-based gene therapy is available for some adults, the current standard of care for children remains prophylactic administration of recombinant FIX protein, which necessitates frequent infusions due to its short biological half-life.

Engineered B Cell Medicines (BCMs) represent a groundbreaking class of cellular therapies. B cells are inherently capable of producing vast quantities of proteins consistently over long periods. With precision genome editing, these cells can be engineered to produce therapeutic proteins, creating BCMs that are durable, allogeneic, re-dosable, and can be administered without pre-conditioning. This new class of medicines holds the promise to revolutionize therapeutic biologics across various protein classes, patient populations, and therapeutic areas.

Founded in October 2020 by David Rawlings, M.D., and Richard James, Ph.D., Be Biopharma is at the forefront of developing BCMs to significantly enhance the lives of people with genetic diseases, cancer, and other serious conditions. Be Bio's dedicated team of scientists, technologists, manufacturing experts, and business professionals are committed to creating transformative cell therapies. The company has garnered substantial support from investors, including ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb, and others, accumulating over $180 million in funding to advance its vision of B cell therapy-based medicine.