Phase 1 trial of oral BCX10013 enrolling PNH patients in S. Africa

Dose escalating study focusing on adults without access to approved therapies

Marisa Wexler MS avatar

by Marisa Wexler MS |

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A proof-of-concept clinical trial of the experimental oral therapy BCX10013 is enrolling people with paroxysmal nocturnal hemoglobinuria (PNH) in Cape Town, South Africa, with plans to expand to other sites where approved PNH treatments are not available.

The Phase 1 study (NCT06100900) is open to adults with PNH who either never have been treated for the condition or were not recently treated with any approved PNH therapy. It’s sponsored by BioCryst Pharmaceuticals, the company developing BCX10013.

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PNH is caused by genetic mutations in the stem cells that are responsible for giving rise to new blood cells. As a consequence, new blood cells are made without certain proteins that normally function like molecular identification badges, informing the immune system that these cells are a healthy part of the body.

Without the identifying proteins, blood cells can be mistaken for an infectious threat that the immune system then attacks. A specific group of immune proteins known as the complement cascade is activated to destroy blood cells in PNH as part of this attack.

BCX10013 is designed to dampen this immune attack by blocking the activity of Factor D — a protein that’s part of the alternative complement pathway. Other approved PNH treatments also are designed to block complement activation, but none target Factor D specifically, and none can be taken orally.

“An oral Factor D inhibitor with once-daily dosing that has an excellent safety profile and strong efficacy would be differentiated and address significant unmet needs of patients living with complement-mediated diseases,” Ryan Arnold, BioCryst’s chief medical officer, said in a company press release.

The open-label Phase 1 trial aims to enroll up to 15 adults with PNH at Cape Town and other locations where patients lack access to current complement-blocking therapies. Two sites in South Africa, one in Pretoria and one in Bloemfontein, are expected to open soon for enrollment.

All trial participants will be treated daily with BCX10013 for up to 24 weeks (about six months). The study plans to test up to four escalating doses, given in higher increments about once each month.

Its main goal is to evaluate the safety of the experimental therapy. Researchers also will look for early signs of efficacy through markers of blood cell destruction and cell health, such as lactate dehydrogenase (LDH) and hemoglobin, and assess patients’ need for blood transfusions.

“By studying BCX10013 in patients living with PNH, we plan to efficiently evaluate alternative pathway activity with well-defined biomarkers, such as LDH and hemoglobin, to determine if we have a safe, effective once-daily dose that meets our criteria to move forward into a pivotal program,” Arnold said.