Amgen has announced positive topline results from a late-stage trial evaluating Uplizna (inebilizumab-cdon) in immunoglobulin G4-related disease (IgG4-RD), a rare and progressive fibroinflammatory disease estimated to affect one to five per 100,000 people.
IgG4-RD impacts almost any organ in the body and is characterised by periods of remission and unpredictable disease flares.
The phase 3 MITIGATE trial has been comparing the CD19-targeted drug, which already holds approvals in several regions to treat the rare autoimmune disease neuromyelitis optica spectrum disorder, to placebo in adults with IgG4-RD.
The study met its primary endpoint, demonstrating a statistically significant 87% reduction in the risk of IgG4-RD flare compared to placebo during the 52-week placebo-controlled period.
All key secondary endpoints were also met, including annualised flare rate, and the overall safety results during the placebo-controlled period of the trial were consistent with the known safety profile of Uplizna.
Full data from the trial will be presented at a future medical meeting, Amgen said, adding that it is planning to file for approval of the drug in IgG4-RD in the US and “other key markets” based on the MITIGATE primary analysis results.
Jay Bradner, executive vice president, research and development, and chief scientific officer at Amgen, said: “MITIGATE is a landmark study with results that demonstrate an important advance in the treatment of patients with IgG4-RD, a devastating and rare disease that currently has no approved therapy.”
The trial also includes an optional three-year open-label treatment period, as well as a safety follow-up period after Uplizna discontinuation of up to two years.
Principal investigator, John Stone, Harvard Medical School and the Massachusetts General Hospital, described the data as a “major milestone for the IgG4-RD community”, adding that it provides “substantial insight into not only how [Uplizna] can help manage IgG4-RD, but also key insights into the nature of [the] condition”.
The readout comes just over a week after Amgen’s Bkemv (eculizumab-aeeb) was approved by the US Food and Drug Administration as the first interchangeable biosimilar to AstraZeneca’s Soliris (eculizumab) for two rare diseases characterised by the breakdown of red blood cells.
