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In a 31-patient phase II trial, brepocitinib was associated with substantially lower cutaneous sarcoidosis activity scores after 16 weeks than placebo. The findings are promising but preliminary; a phase III study is underway, and results are expected at the end of 2028.
A 31-patient phase II trial found that brepocitinib was associated with a marked reduction in skin disease activity among people with cutaneous sarcoidosis after 16 weeks, while scores did not fall in the placebo group. The results, published in JAMA Dermatology and presented at the American College of Rheumatology annual meeting, support further testing of the drug, but do not establish it as an effective or approved treatment for sarcoidosis.
Participants were randomly assigned to receive 45 mg or 15 mg of brepocitinib daily, or placebo. The primary measure was change in the Cutaneous Sarcoidosis Activity and Morphology Instrument-Activity score, known as CSAMI-A. After 16 weeks, both brepocitinib groups had an average reduction of about 22 points, exceeding the study’s 5-point threshold for clinical relevance. The placebo group had no average reduction.
Some secondary results favored the higher dose. Every participant assigned to 45 mg had at least a 10-point CSAMI-A reduction, compared with 73% in the 15-mg group and 14% in the placebo group. A CSAMI-A score below 5, which the researchers defined as functional remission, was reached by 62% on the higher dose, 46% on the lower dose and none on placebo. All five participants in the higher-dose group who had lupus pernio, a form affecting the face and head, had reductions of at least 10 points.
The researchers also reported improved skin-related quality of life among patients receiving brepocitinib. One participant in each study arm stopped treatment because of an adverse event, but no serious adverse events were recorded. Reported issues included a creatine kinase elevation in one person receiving 45 mg and anemia in another. The small trial cannot establish how frequently side effects may occur in a larger population.
A Potential Option for Difficult Skin Disease
Cutaneous sarcoidosis can cause persistent skin lesions and has limited reliably effective treatment options. Corticosteroids, often used first, do not work consistently, and patients who improve may relapse. Other commonly used medicines have also shown uneven results, while evidence for later-line treatments such as tumor necrosis factor inhibitors has been limited.
The trial’s size and placebo results make the findings a reason for further study, not proof that brepocitinib will work for most patients in routine care. If larger studies confirm the benefit and clarify risks, the drug could add an option for people whose skin disease remains difficult to control. The study focused on skin outcomes, so it does not show that brepocitinib improves sarcoidosis affecting the lungs, joints or other organs.
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From Early JAK Studies to Phase III
Clinicians have explored Janus kinase pathway medicines for cutaneous sarcoidosis after mixed results with existing therapies. Before this trial, evidence for drugs such as tofacitinib and upadacitinib came mainly from individual cases and small patient series. A report of improvement with the TYK2 inhibitor deucravacitinib also involved a single patient, according to the source report.
Brepocitinib inhibits both TYK2 and JAK1. The U.S. Food and Drug Administration approved it in August 2026 for dermatomyositis, but that approval does not cover sarcoidosis. In the phase II study, participants could continue conventional non-targeted treatments; oral steroids were tapered during the first eight weeks and topical steroids were stopped when participants were randomized. The groups also differed in average disease duration, a factor to bear in mind when interpreting a small trial.
“I imagine many patients would get it as first line.”
— Misha Rosenbach, MD, of the University of Pennsylvania, as quoted in MedPage Today
Questions Beyond the 31-Person Trial
The phase II trial was small and lasted 16 weeks, so it cannot show whether the improvements persist, how the treatment compares with established therapies, or what uncommon or longer-term harms might emerge. The reported score changes are encouraging, but results in a larger and more varied group of patients could differ.
It is also unclear whether brepocitinib helps sarcoidosis outside the skin, including lung and joint disease, or which patients are most likely to benefit. The study’s participants could continue some conventional treatments, and the groups had different average disease durations. The findings therefore do not settle how the drug should be used or whether regulators will approve it for cutaneous sarcoidosis.
Phase III Results Expected in 2028
A larger phase III trial is underway, with Priovant Therapeutics expecting primary results at the end of 2028. Rosenbach said the 45-mg dose will be tested for potential approval. That study should provide a more substantial assessment of benefit and safety and is expected to examine symptoms beyond skin disease.
Until those data are available, brepocitinib remains investigational for sarcoidosis, and the phase II findings alone do not justify treating it as a proven option for the condition. Decisions about current treatment should be made with a clinician familiar with the patient’s disease and available therapies.
Key Questions
Did brepocitinib improve cutaneous sarcoidosis in the trial?
In the 31-person phase II trial, both daily doses were associated with an average 22-point reduction in CSAMI-A skin activity scores after 16 weeks. The placebo group had no average reduction. These are preliminary results, not confirmation that the drug will work broadly in clinical practice.
Is brepocitinib approved for sarcoidosis?
No. The source report says brepocitinib was approved in the United States for dermatomyositis in August 2026. Its use for sarcoidosis is still being studied.
Were serious side effects reported?
No serious adverse events were recorded in this small trial. One participant in each group stopped treatment because of an adverse event; reported issues included a creatine kinase elevation and anemia. A larger study is needed to better characterize safety.
When will researchers know more?
Priovant Therapeutics expects primary results from the ongoing phase III study at the end of 2028. The larger study is intended to provide further evidence on safety and effectiveness.
Source: medpagetoday.com
