Amgen's Phase III Success Paves Way for Uplizna Label Expansion in Rare Autoimmune Disease

Amgen announced significant results on Wednesday from its Phase III MITIGATE trial, which evaluated the anti-CD19 antibody Uplizna (inebilizumab-cdon) in patients with IgG4-related disease (IgG4-RD). The treatment successfully met its primary and key secondary efficacy endpoints, significantly reducing flares in patients.

The pivotal study's topline data revealed that Uplizna reduced the risk of flares by 87% compared to placebo, a highly statistically significant outcome with a p-value of less than 0.0001. Uplizna also excelled in all key secondary endpoints, which included annualized flare rate, flare-free and corticosteroid-free complete remission, as well as flare-free and treatment-free complete remission.

Safety data from the MITIGATE trial indicated no new concerns, with Uplizna’s adverse event profile consistent with previous studies. Amgen plans to present the full data and analyses at an upcoming medical congress.

Jay Bradner, Amgen's Chief Scientific Officer, described the trial's results as a significant advancement in treating patients with IgG4-RD. He highlighted Uplizna's potential as a promising treatment option for this rare disease, which currently lacks approved therapies. Amgen aims to file for regulatory approval for Uplizna in IgG4-RD later this year.

IgG4-RD is a chronic, systemic, progressive, and immune-mediated disease marked by tissue fibrosis and lesion formation in affected organs. It can lead to irreversible organ damage without visible symptoms and is characterized by periods of remission and unpredictable flares. The disease's pathology involves CD19-positive B cells driving inflammatory and fibrotic processes and activating other cells involved in the disease's activity. Uplizna works by binding to the CD19 surface protein and triggering B cell destruction, addressing the underlying disease process.

This mechanism of action also underpins Uplizna’s efficacy in treating neuromyelitis optica spectrum disorder (NMOSD), a rare neuroinflammatory disease for which the FDA approved the antibody in June 2020.

William Blair analyst Matt Phipps noted that the positive results from the MITIGATE trial could represent a significant expansion opportunity for Amgen. He emphasized the potential market for IgG4-RD, which is estimated to be roughly twice the size of the NMOSD market, and highlighted the straightforward registrational path due to the high unmet need and first-mover advantage for Uplizna.

Beyond IgG4-RD, Amgen is also evaluating Uplizna in a Phase III trial for myasthenia gravis, with results expected later this year.

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