Dive Brief:
- Roivant and subsidiary company Priovant said Wednesday their inflammatory disease pill succeeded in a Phase 3 trial in the rare condition dermatomyositis, significantly reducing signs and symptoms of the disorder by more than placebo when used for one year.
- Company executives said they plan to ask the Food and Drug Adminsitration to approve the pill, called brepocitinib, in early 2026, based on trial results they called “the first ever positive registrational trial for a targeted therapy” in dermatomyositis.
- Brepocitinib is being developed by Priovant Therapeutics, which is part owned by Pfizer. The big drugmaker licensed the pill to Roivant in 2022 as part of a pipeline cull.
Dive Insight:
Brepocitinib inhibits TYK2 and JAK1, two members of a family of proteins called Janus kinases. Drugs in this class, such as Pfizer’s Xeljanz, Bristol Myers Squibb’s Sotyktu and AbbVie’s Rinvoq, have gained FDA approval in autoimmune conditions like rheumatoid arthritis, psoriasis and eczema.
Some also carry the risk of cardiovascular harm due to blot clots, although that doesn’t appear to have slowed sales of Rinvoq, which recorded nearly $6 billion in sales in 2024.
Pfizer, in outlicensing the drug to Priovant, elected to try a rare disease strategy with brepocitinib, which could support FDA approval on smaller and less-costly clinical trials. Besides dermatomyositis, a condition that causes muscle weakness and skin lesions and affects around 34,000 people in the U.S., Priovant is testing the pill in inflammatory eye and skin disorders.
In the dermatomyositis study, Priovant recruited 241 people and randomized one-third each to take 30 milligrams of brepocitinib daily, 15 milligrams daily or a placebo. People taking the 30 milligram dose had a significantly better report on a scale measuring biological markers, clinical signs and activity measures than those who got the placebo at the end of the 52-week treatment period, Roivant said.
More people taking the 30 milligram dose were able to cut their steroid use to less than 2.5 milligrams a day or eliminate steroid use altogether than in the group who received a placebo, Roivant said.
Side effects to brepocitinib treatment were similar to what was reported in past clinical trials, which included more frequent infections. Trial investigators said there were no cardiovascular events resulting from blood clots, though.
“The VALOR study’s success represents a groundbreaking moment for the dermatomyositis field, and the results reinforce brepocitinib’s potential to serve as a deeply impactful treatment option for a substantial number of dermatomyositis patients once approved,” said Ruth Ann Vleugels, director of the autoimmune skin disease center at Brigham and Women’s Hospital, in a statement provided by the companies.
Roivant shares climbed 10% in Wednesday trading.
