Drug intelligence / Profile preview

AQX-1125

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

Overview

AQX-1125, also known as rosiptor, is a small molecule drug developed by Aquinox Pharmaceuticals. It acts as a first-in-class oral activator of SH2-containing inositol-5'-phosphatase 1 (SHIP1), an endogenous inhibitor of the phosphoinositide 3-kinase (PI3K) pathway involved in immune cell activation and chemotaxis. By activating SHIP1, AQX-1125 modulates PI3K/Akt signaling to reduce inflammatory mediator production and leukocyte migration, thereby exerting anti-inflammatory effects[7][8]. The drug was investigated for several indications including interstitial cystitis/bladder pain syndrome (IC/BPS), chronic obstructive pulmonary disease (COPD), atopic dermatitis, allergic asthma, and bone loss disorders such as osteoporosis[2][4][6]. Despite early promise in clinical trials for IC/BPS and other inflammatory conditions, development was discontinued after failing to meet primary endpoints in late-stage studies[1][3].

Brand names
Rosiptor
Other names
rosiptor
02

Targets

SHIP1 (SH2-containing inositol 5-phosphatase 1)

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