# Phase I Clinical Testing of a First-in-Class Therapy against Metastatic Cancer

> **NIH NIH R44** · TRANSCODE THERAPEUTICS, INC. · 2024 · $1,011,207

## Abstract

SUMMARY
Metastatic disease is responsible for ~90% of cancer deaths and is the primary determinant in the life-limiting
aspect of cancer. One validated driver of metastasis is miRNA-10b, a non-coding RNA associated with
metastatic progression in numerous preclinical and > 100 clinical studies. Transcode has developed a novel
therapeutic agent (termed MN-anti-miR10b and commercially developed as TTX-MC138) that relies on specific
eradication of metastatic tumor cells. MN-anti-miR10b consists of antagomirs against miRNA-10b conjugated
to a unique delivery platform, called TTX, which is optimized for the targeting of primary and metastatic tumor
cells. Transcode’s proprietary and patented technology allows for the selective targeting of microRNA-10b in
metastatic cells independent of their type or primary tumor origin. Numerous preclinical studies conducted by
Transcode have shown that MN-anti-miR10b mediates significant miR-10b inhibition in vivo eliciting a marked
and durable regression of lymph node and distant metastases in mouse models of breast cancer with no
evidence of systemic toxicity. Specifically, as few as four to six weekly treatments with MN-anti-miR10b in
combination with low dose chemotherapy led to complete regressions of detectable metastases. Of critical
importance, following elimination of metastases and following discontinuation of therapy, no evidence was
found to suggest recurrence over the remaining natural life span of the animals. In addition, similar studies in
mouse models of pancreatic cancer were conducted with complete responses, defined as complete regression
with no disease recurrence. Finally, initial evidence of safety and therapeutic activity was confirmed in
companion cats with spontaneous metastatic breast cancer. TransCode has completed critical exploratory IND
enabling studies in rats, dogs, and non-human primates with radiolabeled MN-anti-miR10b. Specifically, the
information generated resulted in FDA authorization in support of an ongoing microdosing Phase 0 clinical trial
in patients with advanced metastatic cancer. At this point, TransCode has conducted initial studies in patients,
demonstrating delivery of the drug to metastatic lesions. The Phase 0 trial involves a single injection of a
microdose of Cu-64 labeled MN-anti-miR10b which allows for direct visualization in vivo via PET-MR imaging,
with a primary endpoint of determining the %ID/cc of the therapeutic that is delivered to the metastatic lesions.
Based on the outcome of formal pIND communications with the FDA Office of Oncologic Diseases, the current
application will support full IND-enabling studies, generation and filing of an IND application (Aim 1), as well as
the Phase Ia dose-escalation trial in patients with advanced solid cancers (Aim 2). These funds will accelerate
the program towards commercialization by enabling safety studies and potentially obtaining efficacy signals
that could enable FDA approval of a first-in-class therapeutic aga...

## Key facts

- **NIH application ID:** 11005913
- **Project number:** 1R44CA295173-01
- **Recipient organization:** TRANSCODE THERAPEUTICS, INC.
- **Principal Investigator:** Zdravka O Medarova
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $1,011,207
- **Award type:** 1
- **Project period:** 2024-09-04 → 2026-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11005913

## Citation

> US National Institutes of Health, RePORTER application 11005913, Phase I Clinical Testing of a First-in-Class Therapy against Metastatic Cancer (1R44CA295173-01). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/11005913. Licensed CC0.

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