Priovant announces positive Phase 3 results for targeted dermatomyositis treatment

Durham-based Priovant Therapeutics, with Roivant Sciences, has shared encouraging results from its Phase 3 VALOR study evaluating brepocitinib, a potential new treatment for the autoimmune disease dermatomyositis. 

Dermatomyositis affects around 50,000 adults in the United States. It can cause progressive muscle weakness and skin rashes, sometimes leading to serious complications such as difficulty swallowing, lung issues or skin damage. 

Although corticosteroids and immunosuppressants can help manage symptoms, they often come with significant side effects and don’t fully address the disease.

Brepocitinib is an oral therapy taken once daily that targets key proteins involved in immune system activity, aiming to reduce the underlying autoimmune response.

“We are thrilled with the results of the VALOR study, and I would like to thank all of the patients, investigators and study site staff who contributed to this important research achievement,” said Ben Zimmer, Priovant CEO. “We are excited to continue working towards the rapid approval of brepocitinib in dermatomyositis and our broader goal of developing brepocitinib as a transformational therapy for multiple highly morbid autoimmune diseases where the need for novel efficacious therapies is greatest.”

Priovant Therapeutics was founded in 2021 through a partnership between Roivant Sciences and Pfizer. The company was created around brepocitinib, a drug previously in development at Pfizer and now Priovant’s lead molecule. 

VALOR study highlights

The Phase 3 VALOR study evaluated symptom changes in dermatomyositis patients taking brepocitinib or placebo. 

For the study’s primary measure of effectiveness, patients taking 30 mg of brepocitinib showed an average total improvement score (TIS) of 46.5 at week 52, compared to 31.2 for those on placebo. TIS measures improvement in dermatomyositis, combining changes in muscle strength, skin symptoms and lab results into a single score from 0 to 100, with higher scores showing greater improvement. 

The difference between TIS scores for the treatment and placebo groups was statistically significant at all study time points, starting as early as week 4. According to the companies, this is the first time a targeted therapy has shown positive results in a yearlong placebo-controlled trial for dermatomyositis.

Around two-thirds of the patients in the VALOR study were already on background steroids. For those taking steroids, 62% of brepocitinib 30 mg patients reduced were able to reduce their steroid dose to ≤2.5 mg/day by the end of the study, compared to 34% for placebo. Additionally, 42% of the brepocitinib 30 mg patients were able to stop steroids altogether, compared to 23% for placebo. 

The study also found that brepocitinib 30 mg had a safety profile consistent with previous studies, with no increase in adverse events of special interest compared to placebo.

Immune therapy pipeline

Priovant plans to file an NDA for brepocitinib in dermatomyositis in the first half of 2026 and is also evaluating brepocitinib in a Phase 3 study for non-infectious uveitis — an immune-related inflammation of the eye — and in a Phase 2 study for cutaneous sarcoidosis, an inflammatory skin condition.

According to the Triangle Business Journal, Priovant has about 80 employees, fewer than half of whom are based in the Triangle. Over the next year, the company could double its headcount to prepare for the commercial launch and run additional clinical trials on other programs in its pipeline.