Matrix Metalloproteinase Inhibitor for the Treatment of ALS

Abstract

The overall goals of the proposed project are to complete all of the remaining in-vitro and in-vivo preclinical toxicity and formulation/stability studies on AQU-118, a first-in-class, experimental treatment for ALS, and then submit the completed investigational new drug (IND) application with the FDA. All of the IND enabling in-vitro toxicity studies on AQU-118 were successfully performed and completed. An Ames test of AQU-118 performed under good laboratory practice (GLP) was found to be negative. A hERG potassium channels assay was performed on AQU-118 under GLP conditions and itsIC50 value was determined to be greater than 8.5 microMolar. A pre-IND meeting was held with representatives from the FDA/Division of Neurology I which provided important feedback and suggestions toward completing the IND application of AQU-118. A bone marrow micronucleus test of AQU-118 in the rat was completed under GLP which showed no evidence of genotoxicity.

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Document Details

Document Type
Technical Report
Publication Date
Jul 01, 2023
Accession Number
AD1206362

Entities

People

  • Irving Sucholeiki

Organizations

  • Aquilus Pharmaceuticals (United States)

Tags

DTIC Thesaurus Topics

  • Accuracy
  • Alcohols
  • Biomedical Research
  • Blood
  • Body Weight
  • Bone Marrow
  • Cardiac Arrhythmias
  • Cells
  • Chemical Synthesis
  • Chemistry
  • Electrocardiography
  • Health Services
  • Inhibitors
  • Liquid Chromatography
  • Medical Personnel
  • Moisture Content
  • Quality Control
  • Standards
  • Test Methods
  • Toxicity

Readers

  • Neurotrauma and Rehabilitation Medicine.
  • Occupational Health and Safety.
  • Oncology (Cancer Research).