argenx reports positive topline results from Phase 2 proof-of-concept trial of efgartigimod in primary immune thrombocytopenia

argenx (Nasdaq:ARGX) announced positive topline results from its Phase 2 proof-of-concept clinical trial of efgartigimod (ARGX-113) in adult primary immune thrombocytopenia (ITP) patients.

As quoted in the press release:

“These data highlight the differentiating features of efgartigimod in this difficult-to-treat disease. Efgartigimod has the potential to address ITP in a novel way across patient types, targeting disease at the source by eliminating IgGs and restoring platelet numbers. Also, efgartigimod continues to be well-tolerated, which we attribute to the unique binding characteristics of our Fc fragment and view as an important advantage in this new therapeutic class. We intend to leverage our leadership position in the FcRn antagonist space and advance efgartigimod in ITP to reach patients as quickly as possible,” commented Nicolas Leupin, Chief Medical Officer of argenx.

“The need for new modalities in ITP is an urgent one as patients remain insufficiently managed despite several approved therapies. The variability seen across patients makes a one-size-fits-all treatment approach very difficult and current therapies come with significant side effects. Efgartigimod has been well-tolerated in clinical trials to date with a distinct mechanism of action, and has shown the potential to induce robust disease score improvements across a broad range of patients. We look forward to watching the progress of this program closely in hopes of offering this novel therapeutic option to patients,” commented Dr. Adrian Newland, M.D., Professor of Hematology, The Royal London Hospital and Principal Investigator on the trial.

Click here to read the full press release.