Global trial now underway to test oral therapy for sarcoidosis in the skin
No treatments approved for disease disproportionately affecting Black Americans
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The first participants have been enrolled in a global clinical trial that will test Priovant Therapeutics’ oral therapy candidate brepocitinib against a placebo in people with cutaneous sarcoidosis (CS) — a type of the autoimmune disease, with no approved treatments, in which clumps of immune cells form in the skin.
“Our vision is to establish brepocitinib as a leading treatment option across multiple rare diseases with high patient burden and few or no alternative therapies,” Ben Zimmer, Priovant CEO, said in a press release from Roivant, Priovant’s parent company. “The initiation of our CS Phase 3 study reflects that commitment.”
The Phase 3 trial will be conducted as the second part of an earlier U.S.-based Phase 2 clinical trial called BEACON (NCT06978725). Data from that study showed that brepocitinib was superior to a placebo at reducing disease activity in 31 people with sarcoidosis affecting the skin.
The new study — dubbed BEACON plus — is expected to enroll an estimated 140 people with CS at about 70 sites across the globe. No information about the specific locations of these sites has been provided at this time. Top-line data, or the study’s key early results, are anticipated in 2028.
In the U.S., African American people are more likely to develop the skin manifestations of sarcoidosis than those of other races, studies have shown.
A rare disease, sarcoidosis is marked by abnormal clumps of inflammatory immune cells that form in various tissues throughout the body. In CS, these immune clumps mainly affect the skin; this can lead to bumps or lesions, and may progress to damage to bone, cartilage, and hair follicles.
According to Roivant, CS affects approximately 40,000 adults in the U.S., disproportionately affecting Black Americans. To date, no treatments have been specifically approved for this form of sarcoidosis.
Trial testing brepocitinib’s effectiveness over 4 months
Brepocitinib is an investigational oral therapy designed to ease CS severity by blocking the activity of TYK2 and JAK1, two proteins that play key roles in the activation of disease-driving immune cells.
The Phase 2 BEACON study tested two doses of brepocitinib (15 or 45 mg) against a placebo, with each taken once daily for 16 weeks, or about four months. The main goal was to assess the therapy’s safety, and results were overall positive: No serious adverse events were reported.
A key secondary goal was to assess changes in a standard measure of disease severity called the Cutaneous Sarcoidosis Activity and Morphology Instrument – Activity Score (CSAMI-A). Higher scores reflect worse disease.
The data showed that brepocitinib, particularly its high dose, worked significantly better than the placebo at easing disease severity. Specifically, more than three-quarters of participants given 45 mg brepocitinib achieved a 50% or more reduction in CSAMI-A scores after 16 weeks, compared with none of those on the placebo.
A clinically meaningful CSAMI-A score drop, of at least 10 points, was achieved by all participants treated with the high dose of brepocitinib versus 14% of participants on the placebo.
Also, most (62%) participants on high-dose brepocitinib, but none of those given the placebo, achieved a CSAMI-A score lower than five points, representing functional remission.
Skin sarcoidosis med named breakthrough therapy by FDA
According to Roivant, this Phase 2 result represented the first placebo-controlled study in CS with positive results. These data led the U.S. Food and Drug Administration (FDA) to grant brepocitinib breakthrough therapy designation.
This is a regulatory tool that the FDA uses to speed the development and regulatory review of experimental medicines that have shown promise in early clinical testing and are designed to fill unmet medical needs in the treatment of serious conditions.
In the newly launched Phase 3 BEACON plus trial, participants will be randomly assigned to take either 45 mg brepocitinib or a placebo once daily. The main goal will be to determine how many participants experience at least a 50% reduction in CSAMI-A score after 16 weeks.
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