Be Biopharma’s terminated trial is the latest setback in the hemophilia space, where companies like Pfizer and BioMarin have opted to pull their respective products from the market after weak traction.
Be Biopharma has terminated an early-stage study of its B cell therapy in hemophilia B, continuing the industry’s losing streak in the indication.
The decision to discontinue the Phase 1/2 BeCoMe-9 study was driven by a “strategic business decision,” according to a federal clinical trials database, and was not linked to any safety concerns. Be Bio, based in Cambridge, Massachusetts, had been running the study for its lead asset BE-101, an autologous cell therapy.
Be Bio confirmed the termination of BeCoMe-9 to Endpoints News on Tuesday. Before it was shuttered, the trial had enrolled five of 24 expected patients.
Hemophilia is a rare and genetic disorder characterized by the body’s inability to clot blood, resulting in prolonged and severe bleeding episodes that can be life-threatening, particularly when bleeding is internal. Be Bio’s BE-101 is specifically tailored to address the disease subtype called hemophilia B, which is caused by deficiencies in the clotting factor IX (FIX).
BE-101 makes use of a patient’s own cells, which are modified ex vivo to enable them to predictably and consistently produce FIX, according to Be Bio’s website. The treatment can be re-dosed if needed, the biotech added. The company has no other listed trials for BE-101, though it has a preclinical asset called BE-102 that it plans to study for hypophosphatasia.
With its early-stage study scrapped, BE-101’s fate follows in the unfortunate footsteps of Pfizer’s Beqvez, a gene therapy that was approved for hemophilia B in April 2024. In February last year, the pharma cut off all funding for Beqvez worldwide, ending the product’s R&D and commercialization activities.
The pullback, a Pfizer spokesperson told Nikkei Asia at the time, was driven by “the limited interest patients and their doctors have demonstrated in hemophilia gene therapies.”
The most common type of hemophilia is hemophilia A, which arises from deficiencies to the clotting factor VIII (FVIII). But here, too, biopharma has struggled. In October last year, BioMarin announced that it would let go of its hemophilia A gene therapy Roctavian and “remove it from our portfolio as we focus on the business units aligned with our strategic priorities,” CEO Alexander Hardy said in an investor call at the time.
Approved in June 2023, Roctavian delivers a shorter but functioning copy of the FVIII gene, restoring patients’ ability to form blood clots. But uptake has been slow, with the therapy making $26 million in 2024 and ticking up only slightly to $36 million last year.
Last month, alongside its Q1 earnings announcement, BioMarin disclosed that it had pulled Roctavian from the market.
