AB2 Bio Completes Enrolment for Phase 3 Study of Tadekinig Alfa, a Novel Therapy for Primary Monogenic IL-18 Driven HLH

AB2 Bio Ltd. has completed enrolling participants in its Phase 3 study of Tadekinig alfa, a treatment for primary monogenic IL-18 driven HLH, an ultra-rare and life-threatening condition affecting children. The study aims to demonstrate the efficacy and safety of Tadekinig alfa (r-hIL-18BP). AB2 Bio expects to release topline results in the second half of 2023.

Publish Date: 07-03-2023 Source: AB2 Bio Ltd.

Hemophagocytic lymphohistiocytosis (HLH), also known as hemophagocytic syndrome (HPS), is a rare, life-threatening hematologic disorder manifested by clinical findings of extreme inflammation and unregulated immune activation. In both its congenital (primary) and adult (secondary) forms, an underdiagnosed syndrome is caused by the overwhelming dysregulation of the immune system, resulting in end organ damage or even death if untreated. It is described as a hyperinflammatory reaction with uncontrolled, persistent activation of lymphocytes and macrophages engulfing hematopoietic cells, creating a vicious cycle of a cytokine storm. The diagnostic criteria of HLH involve a molecular diagnosis or meeting five of the eight criteria, including fever, splenomegaly, bicytopenia, hypertriglyceridemia and hypofibrinogenemia, hemophagocytosis in the bone marrow, low or absent NK cell activity, elevated ferritin levels and elevated soluble CD25 levels. Hyperferritinemia and elevated soluble CD25 are excellent predictors of cytokinemia as elevated ferritin levels reflect activated macrophages and soluble CD25 is the gold standard of T-cell activation.

In the USA, about 25% of pediatric cases are primary Hemophagocytic lymphohistiocytosis (PHLH), whereas nearly all adult cases are secondary HLH; the annual incidence rate of PHLH is 1 to 2 cases per million children population.

However, the current Hemophagocytic lymphohistiocytosis (HLH) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (Emapalumab, NI-0501, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the HLH treatment. The key companies in the advanced development stage are Swedish Orphan Biovitrum, Light Chain Bioscience – Novimmune SA, etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Hemophagocytic lymphohistiocytosis (HLH) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2021 to 2032.

About Thelansis:

Thelansis specializes in pharmaceutical market outlook and market forecast reports. We published reports across the therapeutic area, including rare / ultra-rare and mainstream indications. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.