# Advancing the commercialization readiness of nanoparticle-based immunotherapy for cancer treatment

> **NIH NIH SB1** · PDX PHARMACEUTICALS, INC. · 2024 · $160,652

## Abstract

Treatment of non-small cell lung cancer (NSCLC) requires a combination of many highly toxic drugs and
radiation therapy, yet the 5-year survival rate remains about 18%. Immune checkpoint inhibitors (ICIs) targeting
PD-L1/PD-1 has caused a paradigm shift in NSCLC treatment, but the response rate is still only 15-20%.
PDX Pharmaceuticals in partnership with Oregon Health and Science University is developing a next
generation immunotherapy called ARAC-02 that may lead to curative outcomes in a large number of NSCLC
patients. ARAC-02 (Antigen Release Agent and Checkpoint Inhibitor) is built upon our core patented
nanoparticle platform (Pdx-NPTM) capable of co-delivering multiple therapeutic agents, while keeping a small
size in saline, suitable for systemic administration and tumor accumulation. ARAC-02 co-delivers a polo-like
kinase 1 (PLK1) siRNA to kill cancer and release antigens as well as modulate immunosuppression in the
tumor microenvironment, a PD-L1 antibody to home to cancer cells and serve as immune checkpoint blockade,
and immune-stimulant CpG to enhance antigen presentation, leading to strong anti-tumor immune response.
The parent fast-track SBIR application was deemed highly impactful and novel that it received a rare perfect
score from the NIH scientific review panel. The data was published in Nature Communications and a US patent
of ARAC-02 compositions and uses was issued in 2022. We have met the Phase I milestones (formulation
optimization) and progressed to Phase II project (efficacy and safety evaluation in multiple animal models).
To increase commercialization readiness of ARAC-02, we request the CRP fund to support two activities.
Aim 1: To protect intellectual properties (IPs) of the ARAC pipeline. PDX Pharma has been developing
next-generation nanoparticle delivery platform (Pdx-NP) and first-in-class combination immunotherapies
utilizing our expertise in nanotechnology along with cancer systems biology to select synergistic drug
combinations that maximize efficacy and minimize toxicity. Our effort and strong data have led to five US
patents issued in the past 2 years. Specific to ARAC-02, we request CRP funds to (1) pursue IP protection of
Pdx-NPTM and ARAC-02 abroad (in 10 countries and the EU) and (2) expand the IP coverage to other
therapeutic classes beyond PLK1 inhibitors through continuation patent applications.
Aim 2: To initiate GMP-compliant manufacturing of ARAC-02. A key ARAC-02’s component is PD-L1
antibody conjugated nanoparticle (A-NP), which will be mixed with PLK1 siRNA and CpG in the clinic. The core
nanoparticle (NP) has already been manufactured at large-scale by the CRO. In this Aim, we will perform tech-
transfer the synthesis and characterization of A-NP to the same CRO for process familiarization. This aim will
be followed by a full GMP-compliant synthesis campaign, which will be funded through private investment.
Strong IP portfolio and GMP readiness will enhance our success rate in the upcoming priva...

## Key facts

- **NIH application ID:** 10922377
- **Project number:** 1SB1CA287735-01A1
- **Recipient organization:** PDX PHARMACEUTICALS, INC.
- **Principal Investigator:** Worapol Ngamcherdtrakul
- **Activity code:** SB1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $160,652
- **Award type:** 1
- **Project period:** 2024-09-05 → 2026-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10922377

## Citation

> US National Institutes of Health, RePORTER application 10922377, Advancing the commercialization readiness of nanoparticle-based immunotherapy for cancer treatment (1SB1CA287735-01A1). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10922377. Licensed CC0.

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