Drug intelligence / Profile preview

edasalonexent (Astria Therapeutics)

Development stage
Discontinued
Lead developer
Astria Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

Edasalonexent (CAT-1004) is an orally bioavailable small molecule conjugate of salsalate and docosahexaenoic acid (DHA) developed by Catabasis Pharmaceuticals (now Astria Therapeutics). It was designed using the company's SMART (Salsalate-Macromolecule-Activated-Release-Technology) platform to synergistically inhibit the NF-κB pathway. NF-κB is a key driver of skeletal and cardiac muscle degeneration in Duchenne muscular dystrophy (DMD). By delivering both salsalate (an IKKβ inhibitor) and DHA (an omega-3 fatty acid) in a single molecule, edasalonexent aimed to reduce inflammation and promote muscle regeneration. Despite promising early-phase results, the drug failed to meet its primary and secondary endpoints in the Phase 3 PolarisDMD trial, leading to the discontinuation of its development for DMD in 2020.

Other names
salsalate + docosahexaenoic acidsalsalate-DHA conjugate
02

Targets

FPR2 (Formyl peptide receptor type 2)CMKLR1 (Chemokine-like receptor 1)LTB4R (Leukotriene B4 Receptor 1)GPR32 (G protein-coupled receptor 32)PGHS-1 (Prostaglandin G/H Synthase 1)ELIC (Erwinia ligand-gated ion channel)CBP/p300 (CREB-binding protein / E1A-associated protein p300)

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