Be Biopharma has terminated its early-stage study of a B cell therapy for hemophilia B, according to information posted on Jun. 17. The decision to discontinue the Phase 1/2 BeCoMe-9 study was described as a “strategic business decision” in a federal clinical trials database and was not related to safety concerns. The Cambridge, Massachusetts-based company had been running the trial for its lead asset BE-101, an autologous cell therapy.
Be Bio confirmed the termination of BeCoMe-9 to EndpointsNews. Before being halted, the trial had enrolled five out of an expected 24 patients.
Hemophilia is a rare genetic disorder that impairs blood clotting and can cause severe or life-threatening bleeding episodes, especially when bleeding occurs internally. BE-101 was designed specifically for hemophilia B, which results from deficiencies in clotting factor IX (FIX). According to Be Bio’s website, BE-101 uses a patient’s own cells modified ex vivo so they can produce FIX predictably and consistently; the treatment can be re-dosed if necessary. The company does not have other listed trials for BE-101 but plans preclinical studies with another asset called BE-102 targeting hypophosphatasia.
The discontinuation of this study comes after similar setbacks in gene therapies for hemophilia by other companies. Pfizer recently ended funding worldwide for its approved gene therapy Beqvez due to what it described as “the limited interest patients and their doctors have demonstrated in hemophilia gene therapies.”
Other companies have faced challenges as well. In October last year, BioMarin announced it would remove its hemophilia A gene therapy Roctavian from its portfolio as part of shifting strategic priorities. Approved in June 2023, Roctavian generated $26 million in sales during 2024 and increased only slightly the following year before being pulled from the market.
