Be Bio secures $82m for haemophilia B gene therapy Phase I/II trial

1 November 2024
Be Biopharma has successfully secured $82 million in funding to propel its gene therapy project for hemophilia B into Phase I/II clinical trials. This significant financial backing will also support a new program focusing on hypophosphatasia, designated BE-102. The investment round saw participation from major pharmaceutical companies Bristol Myers Squibb and Takeda Ventures, along with contributions from venture capital entities ARCH Venture Partners, Atlas Venture, and RA Capital Management. Additional funding came from Alta Partners and the Longwood Fund.

In conjunction with securing the funds, Be Biopharma has expanded its leadership team by appointing Suha Patel as the senior vice president of commercial & franchise strategy, and Dr. Kiran Patki as the senior vice president of clinical development. Patel brings valuable experience from her tenure at Roche’s Genentech, where she played a key role in the launch of Hemlibra (emicizumab-kxwh), a treatment for hemophilia A. Dr. Patki, with a background at rare disease biotechnology company Rally Bio, will contribute his expertise to the clinical development efforts at Be Biopharma.

Be Bio’s lead candidate, BE-101, represents an innovative approach to treating hemophilia B. This genetically engineered B cell therapeutic introduces the human Factor IX (FIX) gene into primary human B cells, facilitating continuous expression of active FIX levels through a single infusion. Hemophilia B, a condition impacting approximately 40,000 individuals worldwide, is characterized by a deficiency in clotting factor IX, leading to a bleeding disorder. Traditional treatments have relied on regular protein infusions to manage the condition.

The advancement of gene therapies is revolutionizing the treatment landscape for hemophilia B, offering the potential for single infusion treatments that alleviate the burden of repeated infusions for patients. Currently, there are two FDA-approved gene therapies for hemophilia B: CSL Behring’s Hemgenix (etranacogene dezaparvovec) and Pfizer’s Beqvez (fidanacogene elaparvovec). Be Bio's BE-101 has received the FDA's orphan drug designation and fast track status, providing benefits like the potential for seven years of market exclusivity.

Earlier this year, the FDA approved an investigational new drug application (IND) for BE-101, allowing Be Biopharma to commence a clinical trial. This multi-center, first-in-human dose escalation study, named BeCoMe-9 (NCT06611436), will assess the safety and preliminary efficacy of intravenously administered BE-101 in adult patients with moderately severe to severe hemophilia B. The trial aims to enroll 24 participants, with the primary endpoint focusing on adverse events and serious adverse events one year post-dose.

Dr. Joanne Smith-Farrell, CEO of Be Biopharma, expressed enthusiasm about the clinical potential of BE-101, highlighting its promise to provide a durable FIX replacement therapy for hemophilia B patients. Additionally, she voiced excitement about advancing BE-102 to address the unmet therapeutic needs of a broader patient population.

Be Biopharma's efforts are part of a broader movement in the cell and gene therapy industry, which is gaining momentum with a new wave of treatments poised to transform the management and potential cure of various diseases.

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