Be Biopharma Announces New Preclinical Data for BE-102, a B Cell Medicine for the Potential Treatment of Hypophosphatasia

Be Biopharma, Inc. (“Be Bio”), a clinical-stage company pioneering the discovery and development of engineered B Cell Medicines (BCMs), today presented results from new preclinical research demonstrating a single administration of BE-102, a BCM for the potential treatment for Hypophosphatasia (HPP), produces continuous levels of active ALP long-term in vivo. The findings will be presented during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 28^th Annual Meeting on Saturday, May 17, at 10:45 AM CT.

HPP is a genetic disease caused by deficient ALP activity, resulting from pathogenic mutations in the ALPL gene, which leads to multi-systemic clinical complications including deficient bone mineralization. Enzyme replacement therapy (ERT) is the only approved treatment for HPP which requires frequent lifelong injections, and is only available for perinatal/infantile- and juvenile-onset forms of HPP. BE-102 was developed to address these limitations by providing continuous secretion of active ALP from a single infusion, with the flexibility to be titratable and re-dosable as needed. BE-102 is manufactured from primary human B cells by isolating, activating, and precision engineering with CRISPR/Cas9 followed by AAV-mediated delivery of a DNA donor template for the insertion of human ALPL gene into the CCR5 locus (a safe harbor locus) followed by expansion and differentiation in culture into ALP-secreting B lymphocyte lineage cells.

“These studies demonstrate the potential of our B cell medicine platform to deliver B cell derived active ALP with durability out to six months,” said Rick Morgan, Chief Scientific Officer of Be Biopharma. “BE-102 offers a novel and durable approach that may overcome the limitations of current enzyme therapy and does not require pre-conditioning, offering flexibility for re-dosing.”

The presentation highlights both in vivo and in vitro data supporting the target product profile for BE-102. In vivo studies were conducted in immune-deficient NOG-hIL6 mice, confirming long-term engraftment and continuous production of B cell derived active ALP (>175 days) following a single IV administration of BE-102. No BE-102 related adverse events have been observed across multiple in vivo studies. In vitro pharmacology data presented today demonstrates that BE-102 secretes active ALP, which is capable of rescuing calcium deposit inhibited by inorganic pyrophosphate (PPi), a potent inhibitor of bone mineralization and an ALP substrate which accumulates in people with HPP. Be Bio’s in vitro and in vivo pharmacology and safety data established preclinical proof-of-concept that BE-102 has the potential to be a disease-modifying therapy for people with HPP by providing continuous secretion of ALP, with the flexibility to be titratable and re-dosable as needed. A robust package of preclinical studies is planned in anticipation of submission of an IND for a first-in-human clinical trial for people with HPP.

About BE-102

BE-102 is a first-in-class BCM that has been engineered using artificial intelligence-guided protein design to modify primary human B cells to produce ALP, an enzyme deficient in people living with HPP. A single infusion of BE-102 has the potential to provide continuous secretion of therapeutic ALP with the flexibility to be titrated and/or re-dosed, if needed, and without the need for pre-conditioning. BE-102 has been selected as a Development Candidate and has the potential to transform the standard of care for people living with HPP.

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, and 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, re-dosable, 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, and is backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Nextech, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children’s Research Institute, Pathway to Cures (the venture philanthropy fund for the National Bleeding Disorders Foundation) and others to re-imagine medicine based on the power of Engineered B cells. For more information, please visit us at Be.Bio and our LinkedIn page.

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