and will enroll about 120 patients with moderate to severe disease, according to Principia Biopharma, which is developing the drug.
In a press release, the company said that the randomized, double-blind PEGASYSwill compare PRN1008 with placebo, in about 120 patients with newly diagnosed or relapsing moderate to severe pemphigus.
The company also reported the results of an open label phase 2 study of patients with newly diagnosed or relapsing mild or moderate pemphigus, including pemphigus vulgaris and pemphigus foliaceus, which found that control of disease activity within 4 weeks of starting treatment – the primary efficacy endpoint – was achieved by more than 50% of patients taking PRN1008. Principia has extended the trial’s active treatment period from 12 to 24 weeks. The results also led the company to initiate the phase 3 trial.
PRN1008 is an inhibitor of BTK, an enzyme that “is present in the signaling pathways of most types of white blood cells except for T cells and plasma cells,” according to the company’s.