# Phase 2 Clinical Trial Evaluating a Novel Therapy in Patients who are Post-lung Transplant

> **NIH NIH R44** · RENOVION, INC. · 2020 · $920,791

## Abstract

Chronic lung allograft dysfunction (CLAD) is the primary cause of mortality in lung transplant recipients beyond
one year of transplant and includes bronchiolitis obliterans syndrome (BOS). Currently, there are no therapeutic
agents available to prevent CLAD or BOS. Treatments are limited to supportive strategies with low and variable
success rates once CLAD develops. ARINA-1, a patented formulation for a nebulized therapy of ascorbic acid
(ASC), is designed to use when patients develop precursors to BOS, known as BOS-0p, rather than wait until it
progresses to potentially unrecoverable pathology. As a patient develops BOS-0p, the physical and chemical
barriers that typically maintain pulmonary health are compromised, making the lung more susceptible to irritants
and bacteria in the environment. This initiates a cycle of inflammation and infection that leads to decreased
pulmonary function and, ultimately, graft rejection and failure. ASC is critical to first-line airway defenses and is
significantly depleted in the airway of patients who are post lung transplant. Thus, the mechanism of ARINA-1
therapy is simple: ARINA-1 delivers ASC directly to the lung, which restores the first lines of defense against
inflammation and infection. In preclinical studies, we demonstrate that ARINA-1 both activates CFTR-mediated
ion transport and mucus transport, decreases mucus viscosity and inflammation, and inhibits bacterial growth.
Pilot studies in three patients with severe pulmonary disease demonstrate ARINA-1 safety and preliminary
efficacy. A successful Phase 1 study in stable lung transplant patients confirmed ARINA-1 safety and prompted
the FDA to permit an NDA-enabling Phase 2 clinical trial in patients with BOS-0p. The Aims of this proposal are
designed to advance ARINA-1 to FDA approval for use in lung transplant patients with BOS-0p, for whom there
are no current therapies. In this Direct-to-Phase II submission, we will conduct the NDA-enabling multi-center
clinical trial to rigorously evaluate ARINA-1 efficacy in BOS-0p. The successful completion of these Aims will
result in NDA filing for the first FDA-approved therapy for lung transplant patients with BOS-0p to prevent the
progression to CLAD and graft failure. ARINA-1 approval will improve clinical practice for lung transplant care,
by offering physicians a therapeutic option for treating BOS-0p before it progresses to CLAD, increasing not only
survival, but also quality of life during the prolonged survival.

## Key facts

- **NIH application ID:** 10008908
- **Project number:** 1R44HL151144-01A1
- **Recipient organization:** RENOVION, INC.
- **Principal Investigator:** Daniel W. Copeland
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $920,791
- **Award type:** 1
- **Project period:** 2020-07-01 → 2022-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10008908

## Citation

> US National Institutes of Health, RePORTER application 10008908, Phase 2 Clinical Trial Evaluating a Novel Therapy in Patients who are Post-lung Transplant (1R44HL151144-01A1). Retrieved via AI Analytics 2026-05-21 from https://api.ai-analytics.org/grant/nih/10008908. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*