AB2 Bio has concluded subject enrolment in its ongoing, pivotal Phase III clinical trial evaluating Tadekinig alfa (r-hIL-18BP) to treat primary monogenic IL-18 driven Haemophagocytic lymphohistiocytosis (HLH).
HLH is an ultra-rare and life-threatening condition, with no approved therapies.
Conducted in the US, Canada, and Europe, the multicentre, double-blind, placebo-controlled, randomised withdrawal trial has been designed for assessing Tadekinig alfa’s efficacy and safety in 15 primary monogenic IL-18 driven HLH patients.
These patients have verified NLRC4 or XIAP mutations and are suffering from severe, life-threatening hyperinflammation, even after receiving symptomatic treatment with current care.
The trial has an overall duration of nearly 34 weeks.
During the first single-arm, open-label (SAOL) phase for 18 weeks, Tadekinig alfa is being administered in addition to treatments that were previously used for controlling inflammation and related sequelae with limited success.
After completing the SAOL phase, patients will be enrolled in a randomised withdrawal (1:1) phase and receive Tadekinig alfa or a placebo for up to 16 weeks.
The trial’s design, size, and endpoints were validated by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
Topline results from the trial are expected during the second half of this year.
AB2 Bio CEO Dr Djordje Filipovic said: “We are pleased to have completed the targeted enrollment in this pivotal study, which represents a significant milestone for AB2 Bio and the patient community.
“This randomised, placebo-controlled study will provide the dataset required by regulatory authorities to evaluate the efficacy and safety of Tadekinig alfa in patients suffering from primary monogenic IL-18 driven HLH, which will be the basis to support marketing authorisation submissions.”
Tadekinig alfa is a new, recombinant human interleukin-18 binding protein, which inhibits the major proinflammatory cytokine IL18.