Enpatoran targets active skin manifestations in lupus
Merck announced that the US Food and Drug Administration granted Breakthrough Therapy designation to enpatoran for lupus with active cutaneous manifestations.
From Darmstadt, Germany, the company described the decision as a step toward a treatment area with no approved therapies for lupus skin manifestations.
Enpatoran is an oral selective toll-like receptor 7/8 inhibitor that aims to modulate pathways central to lupus-related inflammation.
FDA backed the designation with results from the Phase 2 WILLOW study, a multicenter, randomized, double-blind, placebo-controlled, dose-finding trial.
WILLOW used a basket and adaptive design and included patients with cutaneous lupus erythematosus, or CLE, and systemic lupus erythematosus, or SLE.
According to Merck, the Phase 2 trial showed clinically meaningful improvement in lupus symptoms, especially in patients with active cutaneous manifestations.
Because Breakthrough Therapy status is meant to speed development and review, the designation could shorten the path through the FDA process.
David Weinreich, MD, MBA, Merck’s Global Head of R&D and Chief Medical Officer for Healthcare, said: “For the 85% of lupus patients whose disease includes skin manifestations, often associated with substantial physical and psychosocial burden, the lack of targeted treatment makes the disease hard to control.”
Lupus is a chronic autoimmune disease that can affect the skin, joints, kidneys and central nervous system.
In 29% of lupus cases, skin manifestations are the first sign of disease.
Merck said those skin symptoms can appear as inflamed, photosensitive lesions on the face, scalp and other areas.
Some lesions may be life-altering, and some can become irreversible on their own.
Many patients still do not achieve adequate disease control, even though 85% of lupus patients have disease that includes skin manifestations.
Phase 3 ELOWEN studies
Following the WILLOW results, Merck recently started the global Phase 3 ELOWEN 1 and ELOWEN 2 studies.
Both trials are evaluating enpatoran’s effect on cutaneous and systemic symptoms in patients with lupus.
Researchers are also studying possible links between skin disease activity and systemic disease activity in ELOWEN 1 and ELOWEN 2.
Merck said the designation reflects enpatoran’s potential to move beyond current treatment standards by addressing visible rash and possibly helping disease beyond the skin.
Enpatoran remains investigational and has not been approved for any use anywhere in the world.
