WILMINGTON, DE — Incyte (Nasdaq: INCY) reported that its experimental drug latarcibart reduced the median annualized bleeding rate by 81% in a 16-patient Phase 1/2 study, supporting the company’s decision to advance the once-monthly treatment into a pivotal trial for von Willebrand disease.
The results provide early evidence that the subcutaneous antibody could reduce both bleeding and treatment burden for patients who may otherwise require intravenous prophylactic infusions several times a week. The findings remain preliminary because the study was small, lacked a disclosed control group and was not designed to establish approval-level efficacy.
All 16 patients completed six doses of latarcibart, with maintenance injections given every four weeks. The analysis included patients with different forms of von Willebrand disease and multiple bleeding types, including gastrointestinal, joint and muscle bleeds.
Among patients who switched from prophylaxis containing von Willebrand factor, bleeding reductions ranged from 75% to 100%, according to Incyte. For seven patients who had not previously received intravenous prophylaxis and had historical annualized bleeding rates above 12, reductions ranged from 46% to 100%.
Six of those seven patients experienced reductions exceeding 73%.
Treatment also produced an approximately 86% reduction in breakthrough bleeds requiring von Willebrand factor, the company reported. Seventy percent of patients with a history of such treated bleeds did not experience one while receiving latarcibart.
Latarcibart targets Protein S, a regulator of blood coagulation, to improve hemostasis rather than replace deficient clotting factors. Incyte is evaluating whether that mechanism could provide protection across the major forms of von Willebrand disease, the most common inherited bleeding disorder.
“These results continue to build a highly consistent body of evidence supporting latarcibart as a significant potential treatment advancement for patients with VWD,” Pablo Cagnoni, Incyte’s president and global head of research and development, stated.
The treatment was generally well tolerated during the multidose study. Drug-related adverse events consisted of two moderate headaches in one patient and one mild injection-site reaction.
One patient experienced severe gastrointestinal bleeding that investigators deemed unrelated to latarcibart. The patient had a history of frequent and severe gastrointestinal bleeding.
All participants who entered the trial with a substantial bleeding burden continued into an open-label extension study, according to Incyte.
The company is enrolling patients in its Phase 3 VIVID-6 trial, which will test latarcibart as a prophylactic treatment across von Willebrand disease populations. If approved, the drug could become the first targeted, once-monthly subcutaneous prophylactic therapy for the disorder.
The complete Phase 1/2 results were presented this month at the International Society on Thrombosis and Haemostasis congress in Paris.
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