Clinical Trial Win Bolsters Blockbuster Prospects for Roivant Rare Disease Drug Licensed From Pfizer

For the approximately 40,000 Americans living with cutaneous sarcoidosis, a rare inflammatory skin disease that can cause disfiguring rashes and lesions, treatment options have been severely limited. Until recently, no industry-sponsored clinical trials had successfully yielded an FDA-approved therapy. Now, preliminary results from a Phase 2 study suggest a potential breakthrough.

A New Hope for Cutaneous Sarcoidosis Patients

Priovant Therapeutics, a subsidiary of Roivant Sciences, has announced promising early data from a clinical trial evaluating brepocitinib, a once-daily pill. The study, involving 31 adults, demonstrated that patients receiving the high dose of the drug experienced significant improvements in their skin condition, with many achieving clear or almost clear skin.

Significant Improvements Observed

Participants in the high-dose group showed an average 22.3-point improvement in disease activity at week 16, compared to just 0.7 points for those receiving a placebo. Statistically significant differences were observed as early as week four and continued throughout the 16-week study period. All patients receiving the high dose experienced at least a 10-point improvement, while 62% achieved functional remission – a result not seen in the placebo group.

Did You Know? Roivant Sciences has a unique business model of acquiring and developing assets that have been shelved or deemed non-priority by larger pharmaceutical companies.

Dr. Misha Rosenbach, director of the Cutaneous Sarcoidosis programme at the Hospital of the University of Pennsylvania and principal investigator in the study, described the outcomes as “the sort of data you dream of seeing when you look at trial results — and I would call this a transformational moment for sarcoidosis.”

Navigating the Landscape of JAK Inhibitors

Brepocitinib works by inhibiting TYK2 and JAK1, proteins involved in inflammatory pathways. While JAK inhibitors have shown promise in immunology, concerns about potential cardiovascular risks and cancer—leading to FDA black box warnings for the class in 2021 following a post-marketing study of Pfizer’s Xeljanz—have cast a shadow over their development.

Roivant CEO Matt Gline believes these concerns are overblown, particularly when considering the unmet needs of patients with rare diseases. He stated that these drugs are “some of the best anti-inflammatory drugs ever discovered” and that focusing on severe orphan diseases, where treatment options are limited, is a viable path forward.

Expert Insight: The willingness to pursue JAK inhibitors in rare diseases reflects a different risk-benefit calculation. When patients have few or no alternatives, a higher tolerance for potential side effects may be acceptable, especially if the disease itself carries significant health risks.

In the cutaneous sarcoidosis trial, brepocitinib was well-tolerated, with no serious adverse events reported. The drug has been evaluated in over 1,500 patients across multiple indications, and its safety profile appears consistent with other approved JAK1 and TYK2 inhibitors.

Looking Ahead

Priovant plans to discuss the design of a Phase 3 clinical trial for brepocitinib in cutaneous sarcoidosis with the FDA, with a goal of initiating the study by the end of the year. The company has already submitted a new drug application to the FDA for brepocitinib in dermatomyositis, a separate rare inflammatory skin disease, based on positive Phase 3 results from a previous study. Preliminary data from a Phase 3 trial evaluating brepocitinib in non-infectious uveitis, a rare eye disease, is expected in the second half of the year.

Analysts at Leerink Partners anticipate blockbuster potential for brepocitinib, not only in dermatomyositis and non-infectious uveitis but now also in cutaneous sarcoidosis. Pfizer owns 25% of Priovant and holds rights to brepocitinib outside of the U.S. And Japan.

Frequently Asked Questions

What is cutaneous sarcoidosis?

Cutaneous sarcoidosis is a rare inflammatory skin disease that causes rashes and lesions, which can sometimes be disfiguring. The cause of the disease is unknown, but it involves an inflammatory response that attacks bodily tissues.

What is brepocitinib?

Brepocitinib is a small molecule drug designed to inhibit TYK2 and JAK1 proteins, which play key roles in inflammatory pathways. It’s taken as a once-daily pill.

What are the next steps in the development of brepocitinib for cutaneous sarcoidosis?

Priovant Therapeutics plans to meet with the FDA to discuss the design of a Phase 3 clinical trial for brepocitinib in cutaneous sarcoidosis, with a goal of starting that study by the end of the year.

Given the potential for a new treatment option for a disease that has long lacked effective therapies, what impact do you think this could have on the lives of those affected by cutaneous sarcoidosis?