FDA/CDC

Phase 3 study of novel pemphigus treatment is initiated


 

A phase 3 pivotal study of an oral Bruton’s tyrosine kinase (BTK) inhibitor known as PRN1008 for treating pemphigus has been started 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 PEGASYS study will 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 press release.

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