Matrix Metalloproteinase-2/-9 Inhibitor For The Treatment Of ALS

Aquilus Pharmaceuticals, Inc., Winchester MA

Investigators

Abstract

Project Summary/Abstract. Aquilus Pharmaceuticals, Inc. proposes to evaluate the effects of a selective, dual active, matrix metalloproteinase-2 (MMP-2) and MMP-9 inhibitor, AQU-118, in the superoxide dismutase-1 (SOD-1) mouse model of amyotrophic lateral sclerosis (ALS). The goals for Phase I are to chemically synthesize AQU-118, determine its oral pharmacokinetics (PK) in SOD-1 mice and then determine its efficacy in the SOD-1 mouse model of ALS with dose response. Lastly, a tissue morphology & immunohistochemical study will then be performed to determine whether or not oral dosing with AQU-118 results in any biochemical changes within spinal tissues as compared to controls. The ultimate goal of the proposed work is to evaluate whether a dual active MMP-2/-9 inhibitor can be used to treat ALS.