Amgen's Uplizna promising for rare IgG4-RD

Amgen recently reported encouraging topline findings from its phase 3 MITIGATE trial, which evaluated Uplizna (inebilizumab-cdon) for treating immunoglobulin G4-related disease (IgG4-RD). IgG4-RD is a rare, progressive fibroinflammatory condition that can affect nearly any organ in the body. It is estimated to impact between one and five individuals per 100,000 people and is marked by periods of remission and sudden flare-ups.

In the MITIGATE trial, Uplizna, a CD19-targeted therapy already approved for neuromyelitis optica spectrum disorder in various regions, was compared to a placebo in adult patients with IgG4-RD. The study achieved its primary goal, showing an impressive 87% reduction in the risk of disease flare-ups compared to the placebo over a 52-week period. Additionally, all key secondary endpoints were met, including the annualized flare rate. Safety data from the trial was consistent with the previously known safety profile of Uplizna.

Amgen announced that full results of the trial will be presented at an upcoming medical conference. The company also intends to seek regulatory approval for Uplizna in the US and other significant markets based on the primary analysis results from the MITIGATE trial. Jay Bradner, Amgen's executive vice president of research and development and chief scientific officer, highlighted the study as a landmark in the treatment of IgG4-RD, emphasizing that the results represent a significant advancement for patients suffering from this debilitating and rare disease, which currently lacks approved treatments.

The MITIGATE trial also features an optional three-year open-label treatment period and a safety follow-up period of up to two years after discontinuation of Uplizna. John Stone, principal investigator from Harvard Medical School and Massachusetts General Hospital, described the data as a critical milestone for the IgG4-RD community. He noted that the findings provide not only substantial insights into how Uplizna can manage IgG4-RD, but also valuable information about the nature of this condition.

These positive results follow closely on the heels of another significant achievement for Amgen. Just over a week prior, the US Food and Drug Administration (FDA) approved Amgen’s Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to AstraZeneca’s Soliris (eculizumab). This approval covers two rare diseases characterized by the destruction of red blood cells, further highlighting Amgen's ongoing commitment to addressing rare and difficult-to-treat conditions.

In summary, Amgen's phase 3 MITIGATE trial has demonstrated that Uplizna can significantly reduce the risk of IgG4-RD flare-ups, meeting both primary and secondary endpoints. This development marks a considerable step forward in the treatment of IgG4-RD, offering hope to patients who have had no approved therapeutic options. Amgen plans to seek regulatory approvals based on these positive results, aiming to make Uplizna available to patients in need.