Be Biopharma – a leader in the discovery and development of engineered B Cell Medicines (BCMs) – announced 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 revealed.
These 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. This leads to durable, titratable, and revocable candidates requiring no preconditioning and potentially becoming best-in-class genetic medicines.
The key recent milestones include:
1.) 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 preconditioning. BeCoMe-9 is a multi-center, first-in-human dose escalation study to evaluate 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.
2.) 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 leads to deficient alkaline phosphatase (ALP) activity and results 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.
3.) $82 million funding 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.
4.) 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. 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.
KEY QUOTES:
“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. 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.”
-Joanne Smith-Farrell, Ph.D., Chief Executive Officer of Be Bio
“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.”
-Dr. Smith-Farrell
