Drug intelligence / Profile preview

MIDD0301

Development stage
Preclinical
Lead developer
Milwaukee Institute for Drug Discovery
Modality
Small Molecules
Administration
Oral, Inhalation
01

Overview

MIDD0301 is a **first-in-class small molecule** drug designed to relax airway smooth muscle (ASM) and reduce inflammation for the **treatment of asthma**. It acts as a **positive allosteric modulator of gamma-aminobutyric acid type A receptors (GABA_A receptors)**, particularly targeting α4- and α5-containing subtypes that are selectively expressed in airway smooth muscle, airway epithelium, and lung-infiltrating inflammatory cells. MIDD0301 is unique for its limited distribution to the brain, thereby avoiding central nervous system adverse effects, a common drawback of other GABA_A receptor modulators. Both oral and inhaled (nebulized) formulations have shown efficacy in preclinical asthma models, including those resistant to standard glucocorticoid therapy, with little to no impact on systemic immune function or sensorimotor performance. Development and patents are associated with the University of Wisconsin–Milwaukee and affiliated researchers. The drug has demonstrated a favorable safety profile, lacks significant systemic immunosuppression compared to corticosteroids, and does not inhibit the hERG channel or cause overt cardiotoxicity[1][2][4][5][10][11].

Other names
MIDD0301MIDD-0301MIDD 0301GL-II-93GL-II93GL-II 93
02

Targets

GABAA (Gamma-aminobutyric acid receptor)

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