Drug intelligence / Profile preview

sonlicromanol

Development stage
Phase 3
Lead developer
Khondrion
Modality
Small Molecules
Administration
Oral
01

Overview

Sonlicromanol is an orally bioavailable small molecule developed by Khondrion for the treatment of primary mitochondrial diseases. It acts as a redox modulator and antioxidant, directly scavenging reactive oxygen species (ROS) and boosting the peroxiredoxin-thioredoxin system. Its active metabolite, KH176m (also known as KH183), selectively inhibits microsomal prostaglandin E synthase 1 (mPGES‑1), providing anti-inflammatory effects in addition to its antioxidant activity. Sonlicromanol is being investigated primarily for inherited mitochondrial disorders such as MELAS syndrome (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes), Leigh disease, maternally inherited diabetes and deafness (MIDD), and Leber's hereditary optic neuropathy (LHON). The drug has orphan drug designations in both Europe and the US for these indications[1][3][5][7].

Other names
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide
02

Targets

PTGES (Microsomal prostaglandin E synthase-1)

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