Roivant, continuing ascent, claims another victory for rare disease drug

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Dive Brief:

• A Roivant subsidiary will ask regulators to approve a new indication for its anti-inflammatory medicine brepocitinib after posting successful results in two Phase 3 studies in an eye condition called non-infectious uveitis that can lead to blindness.
• In the trials, known as Clarity-1 and Clarity-2, patients were able to last for a median of more than 48 weeks before “treatment failure,” a measure of eye inflammation and other markers, compared with 12 weeks for those on a placebo. The brepocitinib patients also went 40 weeks without needing immunosuppressive steroids, setting a new bar in treating the disease. And the drug succeeded on every secondary endpoint, including resolving macular edema, the company said Wednesday.
• The Roivant unit, Priovant Therapeutics, already has Food and Drug Administration approval to sell brepocitinib under the brand name Lisraya to treat adults with the skin disease dermatomyositis. Priovant now plans to submit a supplemental application to expand the medicine’s use in the first half of next year.

Dive Insight:

The new results offer another win for Roivant, a “hub-and-spoke” biotechnology company whose subsidiaries specialize in different therapeutic areas. Last month, the company released positive research on the medicine mosliciguat for a high blood pressure condition associated with lung inflammation. Analysts say that drug may bank more than $10 billion in yearly sales.

Originally founded by Vivek Ramaswamy before he became a politician, Roivant looks for later-stage medicines that no longer fit in pharmaceutical companies’ pipelines and tucks them into “Vant” units that can explore new development strategies. CEO Matt Gline has said the company’s model allows for the recruitment of talented executives who want to be in charge, rather than serve as mid-level officials at a larger pharma.

Not all of the company’s efforts have paid off. Failed research in Alzheimer’s disease in 2017 took down one of the first high fliers to emerge from Roivant, Axovant Sciences. But Roivant has steadily built more programs and more than tripled its value over the past six years.

In 2022, Roivant joined forces with Pfizer to create Priovant. Pfizer got a 25% ownership stake in the new company and handed over two drugs, including brepocitinib. Successful Phase 2 testing in non-infectious uveitis followed in 2024. Its first clearance in dermatomyositis came in late August. 

Priovant’s trials suggest that brepocitinib may work better than AbbVie’s blockbuster Humira for non-infectious uveitis, though it’s hard to compare drugs that weren’t formally tested against one another. In Priovant’s statement, its CEO Ben Zimmer said the results suggest brepocitinib could set a new treatment standard for the eye condition. An expert the company quoted agreed.

“This is the kind of outcome that can redefine expectations for an entire field,” said Quan Dong Nguyen, a professor at Stanford who is also president of the International Ocular Inflammation Society and executive vice president of the Foster Ocular Immunology Society, in the statement.

With the latest results, Roivant and Priovant have a potential “best-in-disease” medicine with plenty of room to grow, Stifel analyst Alex Thompson said in a note to clients. “Ultimately, we see this as further validation of the potential for brepocitinib to carve out a broader multi-indication franchise across rare/specialty markets,” he wrote.

Non-infectious uveitis could be a $5 billion-in-peak-sales indication for brepocitinib, added Cantor Fitzgerald analyst Prakhar Agrawal in a separate note.