# Development of Inhaled CPZEN-45 For Tuberculosis Therapy

> **NIH NIH R01** · RESEARCH TRIANGLE INSTITUTE · 2022 · $1,210,721

## Abstract

PROJECT SUMMARY/ABSTRACT
Tuberculosis (TB) is an insidious disease that kills between 1.5–2.0 million people annually. The increase in
multiple and extensively drug-resistant TB globally presents an urgent unmet medical need that is
underserved by the pace of new drug and vaccine development. Following a decade of research activity,
CPZEN-45, a caprazamycin derivative, has been shown to be safe and efficacious when administered via
both the subcutaneous and pulmonary routes in animals. The pulmonary route of administration is more
desirable than subcutaneous delivery because it is non-invasive and more acceptable to the severely
compromised target patient population. CPZEN-45 is poised for development in formal preclinical and initial
clinical studies. It is proposed that in supporting scale up and technical transfer to a current Good
Manufacturing Practice (cGMP) manufacturing facility, the performance of current Good Laboratory Practice
(cGLP) preclinical toxicology studies and preparation of Investigational New Drug (IND) Application
documents to support a Phase I single-dose escalating tolerability study would meet U.S. Food and Drug
Administration (FDA) requirements for product approval. The experiments required to complete the IND
materials are as follows: Specific Aim 1—Scale up and technical transfer of spray dried CPZEN-45 (scale up,
technical transfer to a cGMP environment, development of validated analytical methods); Specific Aim 2—
cGMP rat and dog 14-day toxicology studies (aerosol characterization, rat and dog studies, validated
bioanalytical methods); Specific Aim 3—Document preparation and project management to support pre-IND
meeting with the FDA (chemistry, manufacturing, and controls; data management; preclinical toxicology
studies; data management, Phase I—Protocol development; single-dose escalation study of tolerability and
pharmacokinetics).

## Key facts

- **NIH application ID:** 10348204
- **Project number:** 5R01AI141082-04
- **Recipient organization:** RESEARCH TRIANGLE INSTITUTE
- **Principal Investigator:** Diana M Severynse-Stevens
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $1,210,721
- **Award type:** 5
- **Project period:** 2019-03-01 → 2025-02-28

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10348204

## Citation

> US National Institutes of Health, RePORTER application 10348204, Development of Inhaled CPZEN-45 For Tuberculosis Therapy (5R01AI141082-04). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10348204. Licensed CC0.

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