Be Bio has secured $82 million in funding to advance its gene therapy candidate, BE-101, for hemophilia B into a Phase I/II clinical trial. This investment will support the development of a potentially transformative treatment that aims to provide a durable, single-infusion solution for patients with this X-linked recessive bleeding disorder.
BE-101: A Novel Approach to Hemophilia B Treatment
BE-101 is a genetically engineered B cell therapeutic designed to insert the human Factor IX (FIX) gene into primary human B cells. This approach allows for continuous expression of active FIX, addressing the deficiency that characterizes hemophilia B. Unlike traditional treatments that require regular infusions of FIX protein, BE-101 aims to provide sustained therapeutic levels with a single administration.
Clinical Trial Details: BeCoMe-9
The Phase I/II trial, named BeCoMe-9 (NCT06611436), is a multi-center, first-in-human, dose-escalation study. It will evaluate the safety and preliminary efficacy of intravenously administered BE-101 in adults with moderately severe to severe hemophilia B. The trial aims to enroll 24 patients, with the primary endpoint being the assessment of adverse events and serious adverse events one year post-dose.
Addressing an Unmet Need in Hemophilia B
Hemophilia B affects approximately 40,000 individuals globally and is caused by a deficiency in clotting factor IX. Current treatments often involve frequent infusions of FIX protein to prevent bleeding episodes. While gene therapies like CSL Behring’s Hemgenix (etranacogene dezaparvovec) and Pfizer’s Beqvez (fidanacogene elaparvovec) have been approved, there remains a need for more treatment options with potentially improved durability and safety profiles. Be Bio’s BE-101 has received FDA orphan drug designation and fast track status, potentially granting seven years of market exclusivity upon approval.
Strategic Investments and Leadership Expansion
The financing round saw participation from major pharmaceutical companies such as Bristol Myers Squibb and Takeda Ventures, along with venture capital firms including ARCH Venture Partners, Atlas Venture, and RA Capital Management. In addition to the funding, Be Bio has strengthened its leadership team with the appointment of Suha Patel as Senior Vice President of Commercial & Franchise Strategy and Dr. Kiran Patki as Senior Vice President of Clinical Development. Patel was previously involved in the launch of Hemlibra (emicizumab-kxwh) at Roche’s Genentech.
According to Be Bio’s CEO, Dr. Joanne Smith-Farrell, the company is eager to clinically demonstrate BE-101’s potential to provide a highly durable FIX replacement therapy for Hemophilia B patients.
