Indolcarboxamide is a preclinical candidate for treating multidrug-resistant tuberculosis

Sci Transl Med. 2013 Dec 4;5(214):214ra168. doi: 10.1126/scitranslmed.3007355.

Abstract

New chemotherapeutic compounds against multidrug-resistant Mycobacterium tuberculosis (Mtb) are urgently needed to combat drug resistance in tuberculosis (TB). We have identified and characterized the indolcarboxamides as a new class of antitubercular bactericidal agent. Genetic and lipid profiling studies identified the likely molecular target of indolcarboxamides as MmpL3, a transporter of trehalose monomycolate that is essential for mycobacterial cell wall biosynthesis. Two lead candidates, NITD-304 and NITD-349, showed potent activity against both drug-sensitive and multidrug-resistant clinical isolates of Mtb. Promising pharmacokinetic profiles of both compounds after oral dosing in several species enabled further evaluation for efficacy and safety. NITD-304 and NITD-349 were efficacious in treating both acute and chronic Mtb infections in mouse efficacy models. Furthermore, dosing of NITD-304 and NITD-349 for 2 weeks in exploratory rat toxicology studies revealed a promising safety margin. Finally, neither compound inhibited the activity of major cytochrome P-450 enzymes or the hERG (human ether-a-go-go related gene) channel. These results suggest that NITD-304 and NITD-349 should undergo further development as a potential treatment for multidrug-resistant TB.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Animals
  • Antitubercular Agents / administration & dosage
  • Antitubercular Agents / pharmacokinetics
  • Antitubercular Agents / pharmacology*
  • Antitubercular Agents / toxicity
  • Bacterial Proteins / antagonists & inhibitors
  • Bacterial Proteins / metabolism
  • Biological Availability
  • Disease Models, Animal
  • Dogs
  • Dose-Response Relationship, Drug
  • Drug Resistance, Multiple, Bacterial / genetics
  • Humans
  • Indoles / administration & dosage
  • Indoles / pharmacokinetics
  • Indoles / pharmacology*
  • Indoles / toxicity
  • Injections, Intravenous
  • Membrane Transport Proteins / drug effects
  • Membrane Transport Proteins / metabolism
  • Mice
  • Mice, Inbred BALB C
  • Microbial Sensitivity Tests
  • Mycobacterium tuberculosis / drug effects*
  • Mycobacterium tuberculosis / genetics
  • Mycobacterium tuberculosis / growth & development
  • Mycobacterium tuberculosis / metabolism
  • Rats
  • Rats, Wistar
  • Tuberculosis, Multidrug-Resistant / diagnosis
  • Tuberculosis, Multidrug-Resistant / drug therapy*
  • Tuberculosis, Multidrug-Resistant / microbiology

Substances

  • Antitubercular Agents
  • Bacterial Proteins
  • Indoles
  • Membrane Transport Proteins