Drug intelligence / Profile preview

SM04755

Development stage
Phase 1
Lead developer
Biosplice Therapeutics
Modality
Small Molecules
Administration
Topical
01

Overview

SM04755 is a small-molecule inhibitor of the Wnt signaling pathway developed as a topical treatment for tendinopathy and other inflammatory or fibrotic conditions. Its mechanism of action involves inhibition of CDC-like kinase 2 (CLK2) and dual-specificity tyrosine phosphorylation-regulated kinase 1A (DYRK1A), leading to downregulation of the Wnt pathway, reduced inflammation, decreased catabolic enzyme expression, and promotion of tenocyte differentiation. Preclinical studies demonstrated that topical application resulted in therapeutically relevant tendon exposure with minimal systemic absorption or toxicity. The drug has also been investigated for psoriasis and several cancers but development in oncology indications was discontinued[1][2][3][5][7].

02

Targets

CLK2 (CDC-like kinase 2)DYRK1A (Dual-specificity tyrosine-phosphorylation-regulated kinase 1A)

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