Drug intelligence / Profile preview

AQU-118

Development stage
Unknown
Lead developer
Aquilus Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

AQU-118 is an orally bioavailable, non-hydroxamic acid small molecule inhibitor of matrix metalloproteinases, specifically targeting MMP-9 and MMP-2. Developed by Aquilus Pharmaceuticals, the compound is designed to address neuroinflammation and nerve damage by inhibiting inflammatory processes that are elevated in patients with neurodegenerative conditions. Preclinical evaluation in rodent models of neuropathic pain and Amyotrophic Lateral Sclerosis (ALS), including the TDP-43 over-expressed mouse model, demonstrated that AQU-118 can mitigate nerve injury, weight loss, and the reduction of grip strength. As of early 2026, Aquilus Pharmaceuticals has completed IND-enabling studies and is initiating Phase I clinical trials, with plans to advance into Phase II trials by the end of 2026. While ALS is the primary indication, the company holds intellectual property for its use in several other neurodegenerative and neurological conditions.

02

Targets

MMP9 (Matrix metalloproteinase-9)MMP2 (Matrix metalloproteinase-2)

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