Rare Disease Report

Phase 2 Study Targeting the Autoimmune Response in Pemphigus Vulgaris Is Underway

SEPTEMBER 27, 2017
James Radke
Yesterday, biotech company argenx announced they have begun their Phase 2 proof-of-concept clinical trial of ARGX-113 in patients with pemphigus vulgaris (PV).

argenx is currently developing therapies for a variety of cancerous and auto-immune condition, including PV.

PV is rare auto-immune disease of the skin for which very limited treatment options are available. Patients with the condition have autoantibodies against desmogleins, or proteins that normally help to connect squamous cells of the epidermis. The disruption of these connections lead to bullae developing, first in the mouth and then later on the skin. Current treatment involves the use of immunosuppressive agents but prognosis is variable among the population.

In a news release, Nicolas Leupin, Chief Medical Officer of argenx stated: “Disease severity is directly correlated to pathogenic antibodies of the immunoglobulin G (IgG) type targeting desmoglein-1 and -3 in the skin, leading to painful blister formation and skin damage."

"Current treatment options are limited to high-dose steroids and chronic immunosuppression. We are thrilled about the therapeutic potential of ARGX-113 in PV, based on its mode of action of clearing IgGs. PV represents a clean and rapid proof-of-concept indication and, we believe, is therefore our ideal beachhead into the field of severe auto-immune blistering diseases of the skin."

The Phase 2 study is an open-label, non-controlled clinical trial enrolling up to 12 patients with mild-to-moderate PV. Both newly-diagnosed or relapsing patients can participate. The primary endpoints are safety and tolerability, but secondary endpoints include efficacy and an assessment of pharmacokinetics (PK) and pharmacodynamic (PD) markers.

ARGX-113 blocks antibody recycling to deplete autoimmune disease-causing IgG autoantibodies, such as those being produced in PV against desmogleins.

The drug is also being tested in 2 other auto-immune diseases, myasthenia gravis (MG) and immune thrombocytopenia (ITP).

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