New clinical trial results show promising potential for new lupus treatment
New results from the global WILLOW phase 2 clinical trials published in The Lancet and Lancet Rheumatology indicate that enpatoran, a first-of-its-kind oral pill, is highly effective for treating lupus patients with skin involvement and shows promising signs of managing the disease’s impact on the entire body.
Systemic lupus erythematosus (SLE) is a complex condition where the immune system’s ‘intruder alarms’, proteins called TLR7 and TLR8, are switched to the ‘on’ position, causing the body to attack itself. Enpatoran is designed to flip those switches back to ‘off’, blocking the trigger for inflammation that causes lupus flares.
The two-part study, led by Professor Eric Morand, a Sir John Monash Distinguished Professor and Head of the Rheumatology Group at the School of Clinical Sciences at Monash Health and Director of Rheumatology at Monash Health, suggests that enpatoran could offer a targeted, well-tolerated daily option for those living with both the skin-focused (cutaneous) and whole-body (systemic) forms of the disease
The trial was divided into two main patient cohorts - cohort A (skin focus) and cohort B (systemic focus). In cohort A, enpatoran significantly reduced disease activity, with some patients showing improvement as early as 2 weeks after starting treatment. By the end of the study, more than half of the patients taking the drug achieved clear or nearly clear skin. Patients on the highest dose saw a 72 percent improvement in their skin symptoms compared to 44 percent for those on the placebo.
In cohort B, the trial did not meet its specific statistical target for a ‘dose-response’ relationship, yet it still provided strong clinical signals that Enpatoran is effective. About 50–58 percent of patients improved compared to only 39% on the placebo. The treatment was especially effective for patients who had elevated blood inflammation markers or those who required high doses of steroids to manage their symptoms.
Professor Morand said that the trial results were very promising. “Biological data from the trial confirmed that enpatoran rapidly and consistently lowered interferon levels, a key protein that drives the disease’s progression,” he said. “Importantly, when patients stopped taking the medication, these levels returned to their previous state, proving that the drug was directly controlling the biological switch inside the body.
“Throughout the study, enpatoran was also well-tolerated across all dose levels, with most side effects being mild, such as common colds, headaches or mild diarrhea. There were no new or unexpected safety concerns, and the rates of serious side effects were very low and similar to those seen in patients taking a placebo,” he said.
“This medicine is now moving forward with Phase 3 clinical trials to fully define how enpatoran can be used as a new daily treatment to help people living with lupus lead healthier lives.”
Note: The WILLOW trial was funded by Merck Healthcare (Darmstadt, Germany).
About Monash University
Monash University is Australia’s largest university with more than 80,000 students. In the 60 years since its foundation, it has developed a reputation for world-leading high-impact research, quality teaching, and inspiring innovation.
With four campuses in Australia and a presence in Malaysia, China, India, Indonesia and Italy, it is one of the most internationalised Australian universities.
As a leading international medical research university with the largest medical faculty in Australia and integration with leading Australian teaching hospitals, we consistently rank in the top 50 universities worldwide for clinical, pre-clinical and health sciences.
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