On October 22, 2024 Be Biopharma, Inc. ("Be Bio" or "the Company"), a leader in the discovery and development of engineered B Cell Medicines (BCMs), reported key milestones alongside a new round of funding as its lead program, BE-101 for Hemophilia B, enters the clinic, and its second development candidate for Hypophosphatasia is unveiled (Press release, Be Biopharma, OCT 22, 2024, View Source [SID1234647301]). Both programs are built on Be Bio’s powerful and efficient BCM platform, which utilizes gene editing to engineer B cells to produce sustained levels of therapeutic proteins, resulting in durable, titratable, and redosable candidates that require no preconditioning and have the potential to become best-in-class genetic medicines.
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Key recent milestones include:
BE-101: Phase 1/2 BeCoMe-9 Trial Open for Patient Enrollment; Granted Fast Track Designation. In May 2024, Be Bio announced that BE-101, a first-in-class BCM, received clearance of its Investigational New Drug application. BE-101 is designed to produce constant levels of Factor IX in a durable, redosable, and titratable manner, without the need for preconditioning. BeCoMe-9 is a multi-center, first-in-human dose escalation study aimed at evaluating the safety and preliminary efficacy of BE-101 in adults with moderately severe to severe Hemophilia B. FDA has recently granted BE-101 Fast Track Designation.
BE-102 Nominated as a Development Candidate for Hypophosphatasia (HPP): BE-102 has been selected as a development candidate for the treatment of Hypophosphatasia (HPP), a severe genetic disease with very high unmet medical need, affecting approximately 50,000 patients. HPP is characterized by loss of function mutations in the ALPL gene which lead to deficient alkaline phosphatase (ALP) activity, resulting in weakened and underdeveloped bones and teeth. The only approved therapy for HPP requires multiple injections per week and is limited to pediatric-onset forms of the disease. Data presented at the 2024 American Society for Bone and Mineral Research (ASBMR) Meeting showed that BE-102 has the potential to generate active and sustained levels of ALP in vivo.
$82 Million Financing to Advance BE-101 to Clinical Proof of Concept and Progress BE-102: The Company has closed an $82 million financing, with backing from top venture capital firms and pharmaceutical companies, including ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb, and Takeda Ventures. The funds will be used to achieve clinical proof of concept for BE-101 and to advance the development of BE-102.
Key Hires in Clinical Development and Commercial: The Company is excited to welcome Suha Patel as Senior Vice President of Commercial & Franchise Strategy and Kiran Patki, MD as Senior Vice President of Clinical Development. Ms. Patel joins from Roche/Genentech, where she led the successful launch of Hemlibra and held senior roles across marketing, medical marketing and sales. Dr. Patki joins from Rally Bio, where he served as Senior Vice President and Global Team Leader for asset development projects, with prior leadership experience at Alexion Pharmaceuticals. Concurrent with building out these later-stage functions, the Company will streamline its early research organization and related functions to focus resources on product development.
"Advancing two BCMs that harness the potential of this new modality represents an exciting step in transforming the treatment landscape for their respective indications with potentially best-in-class genetic medicines. With support from a top-tier syndicate of investors and pharmaceutical companies, we are eager to clinically demonstrate BE-101’s potential to provide a highly durable FIX replacement therapy for Hemophilia B patients. Additionally, we are excited to advance BE-102 to address the needs of a large patient population with few to no therapeutic options," said Joanne Smith-Farrell, Ph.D., Chief Executive Officer of Be Bio. "As we build out the team to support product development, we are delighted to welcome Suha and Kiran, experienced leaders who bring deep development and commercial expertise to our team, further strengthening our leadership as we transition into a clinical-stage company."
Dr. Smith-Farrell added: "Our innovative and highly effective research team is the driving force behind the creation of BE-101 and BE-102. They quickly brought the BCM platform to life, achieving IND clearance for BE-101 in just 2.5 years from the program’s inception, and advancing BE-102 to DC in only 15 months, showcasing the platform’s versatility and modularity. As we shift resources toward product development, some valued colleagues will be moving on. We are incredibly proud of their achievements and deeply grateful for their tireless and impactful work on behalf of Be Bio and the patients whose lives we hope these therapies will one day transform."
About BE-101
BE-101 is a first-in-class BCM that is engineered to insert the human Factor IX (FIX) gene into primary human B cells, allowing for continuous 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 while having the flexibility to be titrated and/or re-dosed, and without the need for preconditioning. The potential to maintain therapeutic FIX activity levels while the reducing dosing frequency associated with current FIX replacement regimens could address the considerable infusion burden associated with current therapies and potentially drive reductions in the annualized bleeding rates and FIX usage. The US FDA cleared the BE-101 IND in May of 2024, and granted Fast Track designation in September of 2024. The Phase 1/2 BeCoMe-9 Trial has been initiated and further details of the trial can be found at www.clinicaltrials.gov under NCT identifier: NCT06611436.
About Hemophilia B
Hemophilia B is an X-linked recessive bleeding disorder that affects approximately 40,000 people globally. 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. 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. The current standard of care remains prophylactic administration FIX replacement therapy with a dosing frequency that ranges from every week to every 2 weeks. The short biological half-life of FIX requires lifelong frequent infusions to maintain therapeutic levels.
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 express sustained 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 Hypophosphatasia
HPP is a genetic disease caused by loss of function mutations in the ALPL gene, leading to a deficiency in ALP activity that is required for healthy mineralization of bones and teeth. Insufficient levels of ALP result in the inability of calcium and phosphate to mobilize from the blood, resulting in weakened and underdeveloped bones and teeth. HPP is estimated to affect up to 50,000 people. There is only one approved therapy which requires three to six times a week administration.
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, redosable, and administered without pre-conditioning. The promise of BCMs could transform therapeutic biologics across protein classes, patient populations and therapeutic areas.