RARE Daily

Amgen Reports Positive Results from Registrational Study in Rare Immune Disorder

June 5, 2024

Rare Daily Staff

Amgen reported positive topline results from its phase 3 clinical trial evaluating the efficacy and safety of Uplizna for the treatment of the rare, immune condition Immunoglobulin G4-related disease.

Immunoglobulin G4-related disease (IgG4-RD) is a chronic, systemic, immune-mediated, fibroinflammatory disease that can affect numerous and multiple organs of the body. It is a progressive disease affecting new organs over time either consecutively or simultaneously and is characterized by periods of remission and unpredictable disease flares. IgG4-RD can cause irreversible organ damage with or without the presence of symptoms. B cells are central to the pathogenesis of IgG4-RD. In IgG4-RD, CD19-expressing (CD19+) B cells are thought to drive inflammatory and fibrotic processes and interact with other immune cells that contribute to disease activity.

Uplinza is a targeted CD19+ B-cell-depleting therapy. It is currently approved for Neuromyelitis Optica Spectrum Disorder (NMOSD) by several regulatory bodies, including the U.S. Food and Drug Administration, the European Medicines Agency, Health Canada and the Brazilian Health Regulatory Agency (ANVISA), among others.

The trial met its primary endpoint, showing a statistically significant 87 percent 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, which were annualized flare rate; flare-free, treatment-free complete remission; and flare-free, corticosteroid-free complete remission.

There were no new safety signals identified. The overall safety results during the placebo-controlled period of the trial were consistent with the known safety profile of Uplizna.

Based on the primary analysis results, Amgen is planning to file for approval in the U.S. followed by other key markets.

“IgG4-RD is a devastating, chronic, immune-mediated disease that has just begun to be fully understood over the last few decades,” said John Stone, principal investigator, and a professor of medicine at Harvard Medical School and the Edward A. Fox Chair in Medicine at the Massachusetts General Hospital. “These data mark a major milestone for the IgG4-RD community and provide substantial insight into not only how inebilizumab can help manage IgG4-RD, but also key insights into the nature of this condition.”

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