# Aerosol spectinamide-1599 therapy against tuberculosis

> **NIH NIH R01** · COLORADO STATE UNIVERSITY · 2020 · $710,674

## Abstract

Summary
The lengthy treatment for tuberculosis (TB) is the primary cause of the emergence of multidrug resistant
tuberculosis (MDR-TB), as it frequently results in non-compliance. Current chemotherapy for MDR-TB can last
up to two years with multidrug regimens some of which are painful injectable drugs with serious associated
toxicity. Eradication and control of TB depends on the development of shorter and more effective treatment
regimens with minimal drug associated toxicity. One approach under study in this application is to develop an
inhalational TB therapy [to replace injectable drugs] that when administered with oral TB drugs eases and
shortens treatment. Aerosolized drug delivery unlike injectable agents is easy to administer and provides high
pulmonary concentrations of antibiotics to the local site of infection thereby reducing the systemic level of
exposure to the drug. A recently published article in Nature Medicine by the “spectinamide consortium” showed
that novel spectinamide analogs have excellent activity against Mycobacterium tuberculosis (Mtb) including
MDR and XDR Mtb strains in vitro, as well as in vivo when administered by subcutaneous injection. In the
same study, the lead compound spectinamide-1599 demonstrated strong efficacy against pulmonary TB when
administered as a liquid formulation directly to the lungs of mice via intrapulmonary aerosol (IPA) delivery.
By expanding on our preliminary data in this application Aim 1 will determine the pharmacokinetics (PK)
and tissue distribution of spectinamide-1599 after IPA. Aim 2 will determine optimal dose, duration and
frequency for treatment with spectinamide-1599 delivered by IPA and Aim3 will optimize a dry powder
formulation of spectinamide-1599 to be used as inhalational TB therapy. These studies will be developed
as a consortium between experts in the field of TB, inhalational animal models of TB and preclinical studies,
pharmacokinetics and pharmacodynamics of TB chemotherapy and aerosol formulations and formulation of
dry powders located at Colorado State University, University of Tennessee, Research Triangle Institute and St.
Jude Children's Research Hospital. Working all together this consortium of researchers aims to advance
research for this drug to the level that inhalational therapy via spectinamide-1599 can be considered a new
drug/therapy candidate for TB. Thus, these studies will provide a formulation and inhalational therapy regimen
of the spectinamide-1599 with well defined aerodynamic and PK properties and of well characterized in vivo
efficacy for future testing in multidrug combination studies and in larger inhalational animal TB models and
ultimately in TB patients.

## Key facts

- **NIH application ID:** 9949367
- **Project number:** 5R01AI120670-05
- **Recipient organization:** COLORADO STATE UNIVERSITY
- **Principal Investigator:** Miriam S. Braunstein
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $710,674
- **Award type:** 5
- **Project period:** 2016-06-10 → 2022-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9949367

## Citation

> US National Institutes of Health, RePORTER application 9949367, Aerosol spectinamide-1599 therapy against tuberculosis (5R01AI120670-05). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9949367. Licensed CC0.

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