Recent results from the RAISE study (NCT04115293) support the effectiveness and safety of the investigational treatment zilucoplan, a self-administered, subcutaneous peptide inhibitor of complement component 5, in adults with generalized myasthenia gravis (gMG).

“These exciting results give us additional reason to believe that zilucoplan can offer an important step forward in addressing the unmet needs of people living with gMG,” James F. Howard, MD, lead investigator of the RAISE trial, said in a press release from UCB, the biopharmaceutical company developing zilucoplan.

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Zilucoplan improved the MG-Activities of Daily Living score (primary endpoint), as well as the Quantitative MG, MG Composite, and MG Quality of Life 15 revised scores (secondary endpoints), from baseline to week 12.

Treatment with zilucoplan was well tolerated, with a similar incidence of serious treatment-emergent adverse events between the treatment and placebo arms, according to the company press release. In addition, no major unexpected safety findings were reported.

UCB is planning to progress with zilucoplan regulatory submissions in the United States, Europe, and Japan later this year.

The company is also developing rozanolixizumab, a subcutaneous humanized monoclonal antibody, as a treatment for gMG, among other autoimmune diseases. The results from phase 3 trials for both potential treatments will be presented this year at medical meetings.

References

UCB announces positive data in myasthenia gravis with zilucoplan phase 3 study results. News release. UCB, Inc.; February 4, 2022.

Safety, tolerability, and efficacy of zilucoplan in subjects with generalized myasthenia gravis (RAISE). ClinicalTrials.gov. October 4, 2019. Updated January 28, 2022. Accessed February 24, 2022.