Genentech on the Verge of First Biologic Treatment for Pemphigus Vulgaris

September 3, 2015

Genentech has announced the initiation of a Phase III clinical trial investigating the use of Rituxan (rituximab) in patients with pemphigus vulgaris (PV), a rare, chronic and potentially fatal, blistering skin disease.

The aim of this study, called PEMPHIX, is to determine the efficacy and safety of Rituxan for the treatment of PV. The current standard of care includes high doses of corticosteroids taken for several weeks and corticosteroids in combination with the off-label use of corticosteroid-sparing immunosuppressive drugs for many months, which can cause significant, long-term side effects.

Results of independent studies have shown that Rituxan may provide substantial improvement in PV remission rates and successful tapering and/or cessation of corticosteroid (CS) therapy. If approved, Rituxan could be the first FDA-approved biologic treatment for this autoimmune disorder that impacts approximately 30,000 people in the U.S. Rituxan for PV has also been granted orphan drug designation by the FDA.

“We are taking the knowledge gained from the efficacy demonstrated by Rituxan in B cell-mediated autoimmune diseases such as rheumatoid arthritis and ANCA-associated vasculitis and applying it to research for pemphigus vulgaris,” said Paul Brunetta, M.D., associate group medical director, Genentech Immunology. “The ongoing investigation of Rituxan for the treatment of pemphigus vulgaris demonstrates Genentech’s commitment to follow the science and address the unmet medical needs of patients.”

Facts about Pemphigus Vulgaris:
· Pemphigus vulgaris is the most common type of a group of autoimmune disorders collectively called pemphigus.

· It is a painful, disfiguring and potentially fatal disease characterized by progressive blistering of the skin and/or the mucous membranes.

· It affects women and men equally.

· It occurs primarily in adults ages 30-60

· PV has a mortality rate between 5 and 10 percent, mostly due to complications of therapy.

“Current treatments for pemphigus vulgaris can cause significant, long-term side effects,” said Victoria P. Werth, M.D., Professor of Dermatology and Medicine at the University of Pennsylvania and lead investigator for the PEMPHIX trial. “It is our hope that the PEMPHIX trial will demonstrate the efficacy and safety of Rituxan for this disease and provide patients with a much-needed, FDA-approved treatment option.”

About The Study
PEMPHIX (NCT02383589) is a Phase III, randomized, double-blind, double-dummy, active-comparator, multicenter, international study to evaluate the efficacy and safety of Rituxan compared to mycophenolate mofetil in patients with moderate-to-severely active pemphigus vulgaris (PV). Patients in the Rituxan arm of the study will receive 1000mg rituximab given by intravenous infusion on Day 1 and 15, with repeat rituximab administration on Day 168 and 182 provided specific safety criteria have been met.

Rituxan is currently approved for these autoimmune disorders: rheumatoid arthritis, granulomatosis with polyangiitis (GPA), and microscopic polyangiitis (MPA).

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