AB2 Bio announces readiness for recruitment in pivotal Phase 3 trial of Tadekinig alfa, a novel human recombinant interleukin-18 binding protein, in children with genetic diagnosis of NLRC4-MAS mutation or XIAP deficiency, with amended design in the U.S.

AB2 Bio Ltd, a Swiss advanced clinical-stage biotech company, specialized in developing innovative therapies for the treatment of severe systemic autoinflammatory diseases, announced today that a single-arm open label design with a randomized withdrawal phase for its ongoing pivotal Phase 3 trial under an Investigational New Drug (IND) Application for its product candidate Tadekinig alfa, has been accepted by the U.S. Food and Drug Administration (FDA). Tadekinig alfa is a recombinant human interleukin-18 binding protein targeting the treatment of dysregulated and persistent IL-18-mediated inflammatory processes which are the basis of autoinflammatory and autoimmune diseases, such as NLRC4-MAS, XIAP deficiency, systemic Juvenile Idiopathic Arthritis, and Adult onset Still`s Disease.

“We are delighted about the FDA’s acceptance and look forward to continuing our pivotal Phase 3 clinical trial under the amended protocol, which will now give all patients the chance to be initially treated with Tadekinig alfa rather than having only a 50% chance as in the previous version of the protocol,” says Eduardo Schiffrin, M.D., AB2 Bio’s Medical Director. “So far there is no approved therapy for autoinflammatory diseases associated with high free IL-18 levels. As a result, there is a high unmet medical need among patients suffering from these severe and life-threatening conditions. By testing a potentially effective new therapy, we hope to improve their prospects and quality of life.”

AB2 Bio is currently enrolling patients in the U.S. and Canada. The clinical trial details including trial locations can be found under ClinicalTrials.gov Identifier: NCT03113760.

About the Pivotal Phase 3 Trial in Patients with Monogenic, Interleukin-18 Driven Autoinflammation due to NLRC4-MAS Mutation or XIAP Deficiency

Recently, single point mutations in the NLRC4 gene have been identified. These genetic, gain of function mutations give rise to severe, life-threatening systemic inflammation associated with extremely high levels of IL-18, the therapeutic target of Tadekinig alfa. AB2 Bio is conducting a pivotal Phase 3 clinical trial with its experimental drug Tadekinig alfa in patients with NLRC4 mutations and patients carrying mutations of the X-linked inhibitor of apoptosis protein (XIAP). NLRC4 mutations and XIAP deficiency are part of primary Hemophagocytic Lymphohistiocytosis (HLH).