Amgen Reports Positive Phase 3 Trial Results for Uplizna® in Treating IgG4-RD

Amgen , a leading biotechnology company, has revealed groundbreaking results from its Phase 3 clinical trial evaluating UPLIZNA® (inebilizumab-cdon) for treating Immunoglobulin G4-related disease (IgG4-RD). This study marks the first randomized, placebo-controlled trial demonstrating significant benefits for IgG4-RD patients, a rare and chronic condition with no currently approved therapies.

Key Findings from the Clinical Trial

The trial, identified as NCT04540497, was a double-blind, multicenter study comparing UPLIZNA to a placebo over a 52-week period. The primary endpoint of the study was a reduction in the risk of IgG4-RD flares. The data showed an impressive 87% decrease in flare risk with UPLIZNA compared to placebo, achieving a hazard ratio of 0.13 and a p-value of less than 0.0001.

Additionally, all key secondary endpoints were successfully met. These included the annualized flare rate, flare-free, treatment-free complete remission, and flare-free, corticosteroid-free complete remission. The safety profile of UPLIZNA remained consistent with previous data, and no new safety concerns were identified. The full results will be presented at an upcoming medical conference.

Significance of the MITIGATE Study

Dr. Jay Bradner, Amgen's Executive Vice President of Research and Development, emphasized the importance of the MITIGATE study. "This landmark study offers a significant advancement in treating IgG4-RD, a debilitating disease affecting multiple organs. We are thankful for the support from patients, clinicians, and advocacy groups that made this study possible," he remarked. The study was held at 80 sites across 22 countries and is the first to offer class 1 evidence for treating IgG4-RD.

Dr. John Stone, the principal investigator and a professor at Harvard Medical School, highlighted the broader implications of the study. "These results offer crucial insights into managing IgG4-RD and deepen our understanding of the disease itself," he said.

Future Steps and Regulatory Plans

UPLIZNA is already approved for treating Neuromyelitis Optica Spectrum Disorder (NMOSD) by several regulatory bodies, including the FDA and the European Medicines Agency. Based on the MITIGATE study results, Amgen plans to seek regulatory approval for UPLIZNA in the U.S. and other key markets.

The trial was conducted with support from Mitsubishi Tanabe Pharma and Hansoh Pharma. Mitsubishi Tanabe Pharma holds marketing authorization in several Asian countries, while Hansoh Pharma manages the drug's regulatory and commercial aspects in China, Hong Kong, and Macau.

Understanding IgG4-Related Disease

IgG4-RD is a chronic, systemic, immune-mediated disease that causes fibroinflammatory damage to various organs. The disease is characterized by periods of remission and unpredictable flares, potentially leading to irreversible organ damage. B cells, particularly those expressing CD19, play a significant role in the disease's pathology by promoting inflammation and fibrosis.

Although the incidence of IgG4-RD is estimated to be between 1 and 5 per 100,000 individuals, the actual number is difficult to determine due to limited epidemiological data. The disease primarily affects individuals aged 50 to 70 and is more common in men than in women.

About UPLIZNA

UPLIZNA is already indicated for treating NMOSD in adults who are anti-aquaporin-4 (AQP4) antibody-positive. The drug is generally well-tolerated, although it carries some risks, including infusion reactions, infections, and potential reactivation of Hepatitis B. Patients should be screened for these conditions before treatment.

Conclusion

Amgen's recent announcement marks a significant milestone in treating IgG4-RD, offering hope for patients suffering from this chronic and debilitating disease. With plans for regulatory approval underway, UPLIZNA could soon provide a much-needed therapeutic option for IgG4-RD patients worldwide.

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