AB2 Bio expands Phase 3 trial of Tadekinig alfa in monogenic HLH

AB2 Bio Ltd, a Swiss advanced clinical-stage biotech company, specialized in developing innovative therapies for the treatment of severe systemic autoinflammatory diseases including rare diseases with high unmet medical needs, announces the expansion of its Phase 3 trial of Tadekinig alfa in the orphan disease IL-18 driven monogenic Hemophagocytic Lymphohistiocytosis (HLH).

Recruitment to the pivotal Phase 3 clinical trial, which aims to assess the efficacy and safety of Tadekinig alfa in patients with monogenic, interleukin-18 driven autoinflammation caused by NLRC4-MAS mutation or XIAP deficiency, has been opened up to adults in addition to children and is currently recruiting in the U.S., Canada and Germany.

Monogenic HLH is a rare, life-threatening disease characterized by hyperinflammation due to an overactivated immune system. There are currently no drugs specifically approved to treat IL-18 driven monogenic HLH and severe inflammatory conditions; disability and death are common outcomes.

Dr. Michael Soldan, CEO of AB2 Bio commented: “We are pleased to expand this Phase 3 trial of Tadekinig alfa to adults and thus offering participation in this trial to a wider patient population. With a manufacturing agreement in place with WuXi Biologics, we are now focused on completing enrolment in the Phase 3 trial and expect results in second half 2022. In parallel, we are preparing to file for marketing authorization in the U.S. and the European Union to bring this product as soon as possible to patients suffering from monogenic HLH associated with elevated IL-18 levels, a devastating condition for which there are currently no approved treatment options.”

Previous clinical research indicates that Tadekinig alfa binds free IL-18 and may help manage severe systemic disease manifestation. Eligible patients with either NLRC4-MAS mutation or XIAP deficiency can be identified by a genetic diagnosis. Patients with a XIAP deficiency and persistent disease manifestations after an unsuccessful stem-cell transplantation are also allowed for recruitment. Once enrolled, patients will be administered Tadekinig alfa for 18 weeks (single-arm open-label phase) in addition to the standard of care, followed by 16 weeks of randomized withdrawal.