Drug intelligence / Profile preview

Leramistat

Development stage
Phase 2
Lead developer
Istesso
Modality
Small Molecules
Administration
Oral
01

Overview

Leramistat is a first-in-class, oral small molecule drug in phase 2 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and moderate to severe rheumatoid arthritis (RA). It is designed as a programmed disease-resolving agent that augments the body's inherent repair response to restore damaged tissue and build resilience without suppressing the immune system. Leramistat selectively modulates inflammation at sites of tissue damage by promoting a pro-repair environment rather than broadly inhibiting inflammatory cytokine cascades. This unique mechanism aims to resolve chronic cycles of inflammation and tissue destruction seen in autoimmune diseases like RA, while also offering potential benefits in other conditions characterized by auto-inflammation, autoimmunity, fibrosis, or bone loss. Clinical studies have shown that leramistat can reduce progression of structural damage and improve bone dynamics, disability, and fatigue in RA patients with an inadequate response to DMARDs. The drug has received FDA Fast Track and Orphan Drug Designation for IPF.

02

Targets

ND1 (Mitochondrial electron transport chain complex I)TNFA (Tumor necrosis factor alpha (soluble))

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