Drug intelligence / Profile preview

SMDG-MD22

Development stage
Preclinical
Lead developer
SMDG
Modality
Small Molecules
Administration
Oral
01

Overview

SMDG-MD22 is a small molecule therapeutic candidate being developed by the **Small Molecule Discovery Group (SMDG)** for the treatment of **Amyotrophic Lateral Sclerosis (ALS)**, also known as Motor Neuron Disease (MND). The compound functions as an activator of the **Nuclear factor erythroid 2-related factor 2 (Nrf2)** pathway, a master regulator of the cellular antioxidant response. SMDG-MD22 works by inhibiting the protein-protein interaction between Nrf2 and its negative regulator, **Kelch-like ECH-associated protein 1 (Keap1)**, thereby preventing Nrf2 degradation and promoting its nuclear translocation. This activation induces the expression of cytoprotective and antioxidant genes, aiming to mitigate the oxidative stress and neuroinflammation that drive motor neuron degeneration. As of 2024, the program is in the lead optimization stage of preclinical development.

02

Targets

KEAP1 (Kelch-like ECH-associated protein 1)NFE2L2 (Nuclear factor (erythroid-derived 2)-like 2)

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