Be Bio raises $92M to advance hemophilia B treatment BE-101
Funding will support data generation from BeCoMe-9 study
Be Biopharma has raised $92 million in series C financing to support the first-in-human Phase 1/2 trial of BE-101, the company’s B-cell therapy candidate for hemophilia B.
Proceeds from the funding round will be partly used to generate proof-of concept data from the BeCoMe-9 trial (NCT06611436), which is enrolling patients at two sites in the U.S. The study will assess BE-101’s safety and clinical activity, when given once by intravenous (into-the-vein) infusion to adults with moderately severe to severe hemophilia B.
“We are on track to demonstrate clinical proof-of-concept for BE-101 in hemophilia B,” Joanne Smith-Farrell, PhD, Be Biopharma’s CEO, said in a company press release. “I am grateful to our existing and new investors, who share our vision that [B-cell medicines] can transform the treatment landscape for a wide range of diseases with potentially best-in-class medicines.”
Hemophilia B is caused by mutations in the F9 gene that impair the production or activity of factor IX (FIX), a clotting protein. As a result, people with the disease are prone to prolonged and excessive bleeding, the hallmark symptom of hemophilia, which can lead to other complications.
Treatment usually includes factor replacement therapy, wherein patients receive regular FIX infusions to maintain adequate factor levels and allow for proper blood clotting. This can be burdensome, however, and the treatment’s effectiveness may be reduced if the patient’s immune system recognizes the clotting factor as a threat and develops antibodies, called inhibitors, against it.
What is BE-101?
BE-101 is an autologous B-cell therapy, meaning it uses a patient’s own immune B-cells. After they’re collected, the cells are genetically modified to have a functional F9 version inserted into their genome, that is, the entire set of genes in a cell.
Once modified, the cells are expanded and differentiated into plasma cells, which are specialized B-cells that can produce large amounts of antibodies. They are then infused back into the patient, where they should migrate to the bone marrow and constantly produce FIX.
The therapy is expected to lower the frequency of bleeding episodes, while reducing the burden of frequent dosing in current FIX replacement therapies, according to the company.
The U.S. Food and Drug Administration granted BE-101 orphan drug and fast track designations, which offer certain incentives to help accelerate a treatment’s development and regulatory review.
Financial proceeds will also be used to advance the development of BE-102, the company’s B-cell medicine candidate for hypophosphatasia, a rare disorder that features defects in bone and teeth mineralization.
“With this funding in hand, we are well equipped to advance our two lead programs and solidify our position as a multi-program, clinical-stage company,” Smith-Farrell said.
The financing round featured participation from new investor Nextech, as well as existing investors, including ARCH Venture Partners, Atlas Venture, RA Capital Management, and others.
“Be Bio has established itself as a leader in this field with their lead programs having best-in-class potential,” said Melissa McCracken, PhD, partner at Nextech. “We believe BCMs, as a class, could revolutionize treatment paradigms across indications.”
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