Positive topline results from PEMPHIX, a phase 3 trial evaluating rituximab compared to mycophenolate mofetil (MMF), demonstrated rituximab’s superiority in patients with pemphigus vulgaris (PV).
Pemphigus vulgaris is a rare autoimmune disease that causes blistering on the skin and mucous membranes. Treatment is usually with corticosteroids that suppress the immune system. Left untreated, PV can be fatal. The disease can affect all races, genders, and ages, but is common in people of Mediterranean descent and middle-aged to older adults.
In PEMPHIX, a randomized, double-blind, double-dummy, active-comparator, parallel-arm, multicenter study, patients with moderate to severe, active PV requiring 60–120 mg/day oral prednisone or equivalent received either rituximab plus MMF placebo or rituximab placebo plus MMF. The primary endpoint was the proportion of patients who had sustained complete remission for at least 16 consecutive weeks at Week 52.
The secondary endpoints, cumulative corticosteroid dose, number of flares, time to sustained remission, and time to disease flare, were also met by rituximab.
Study data also indicated that rituximab may provide complete remission and a successful tapering of corticosteroid therapy that is superior to MMF in adults with PV.
Source: BioSpace, June 13, 2019