Be Biopharma reveals promising preclinical results for BE-102 therapy

In the United States, specifically in Cambridge, Massachusetts, Be Biopharma, Inc. announced promising results from preclinical research, demonstrating the potential of BE-102 as a groundbreaking treatment for Hypophosphatasia (HPP). This presentation took place on May 17, 2025, at the American Society of Gene & Cell Therapy's 28th Annual Meeting. HPP is a serious genetic disorder resulting from mutations in the ALPL gene, leading to decreased alkaline phosphatase (ALP) activity and various complications like inadequate bone mineralization. Engagement with the scientific community has been significant, considering current treatment options predominantly involve enzyme replacement therapy (ERT). Although ERT is the only approved method to treat HPP, it requires lifelong, frequent injections and is limited to certain forms of the disease. This gap in care necessitated the development of BE-102, an Engineered B Cell Medicine (BCM), which could provide long-term benefits through continuous ALP secretion from a single infusion. Preclinical studies have shown that a single intravenous dose of BE-102 resulted in sustained production of active ALP for over six months. No adverse effects have been linked to BE-102 in multiple studies, showcasing its potential safety and efficacy. Furthermore, in vitro pharmacological data revealed that BE-102 effectively secretes active ALP capable of overcoming the inhibition of calcium deposits by inorganic pyrophosphate, which is an important consideration in the pathology of HPP. The findings indicate that BE-102 could represent a significant advancement in treating HPP, possibly altering the standard of care. Be Biopharma is preparing for a robust series of additional preclinical studies aimed at gathering the necessary data for an Investigational New Drug (IND) application to initiate first-in-human clinical trials for BE-102. The leadership at Be Biopharma expressed confidence in their ability to revolutionize the treatment landscape for HPP through this innovative approach, highlighting the platform's potential to offer flexible dosing without the need for pre-conditioning. The company expects that the success of BE-102 could lead to its selection as a preferred option for patients suffering from HPP, allowing for both high efficacy and patient-centered treatment.