# Repurposing of Pyrvinium Pamoate for Familial Adenomatous Polyposis (FAP)

> **NIH NIH R44** · STEMSYNERGY THERAPEUTICS, INC. · 2020 · $621,402

## Abstract

ABSTRACT
Individuals with the rare, inherited disorder Familial Adenomatous Polyposis (FAP) develop thousands of
precancerous polyps at an early age and ultimately develop colorectal cancer (CRC). The current standard of
care for FAP is colectomy. However, unavoidable side-effects of colectomy are debilitating. Furthermore,
colectomy does not prevent subsequent development of extra-colonic intestinal tumors, the main cause of death
post-colectomy. There are currently no drugs FDA approved for the treatment or chemoprevention of FAP
patients. We have demonstrated that pyrvinium can be repurposed as a potent inhibitor of WNT signaling and
is efficacious in an animal model of FAP. Oral dosing of pyrvinium pamoate produces negligible systemic
exposure and limits the exposure to the gut which reduces the likelihood of toxicity. Submitting these data to the
FDA, we were awarded an Orphan Drug Designation to market pyrvinium exclusively for FAP patients. To clarify
the steps required to take pyrvinium back into the clinic as a repurposed agent for FAP patients, using a
505(b)(2) regulatory mechanism, we recently had a pre-IND meeting with the FDA. This Phase I/II Fast-Track
application is driven by the FDA’s recommendations for a successful Investigational New Drug (IND) application
for pyrvinium in the treatment of FAP. The repurposing of pyrvinium for FAP patients, if successful, would
represent 1) A major improvement in therapy and in the quality of life, and 2) The successful bench to bedside
application of a rationally selected targeted therapy.

## Key facts

- **NIH application ID:** 10016217
- **Project number:** 5R44CA228840-03
- **Recipient organization:** STEMSYNERGY THERAPEUTICS, INC.
- **Principal Investigator:** Darren Orton
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $621,402
- **Award type:** 5
- **Project period:** 2018-03-15 → 2021-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10016217

## Citation

> US National Institutes of Health, RePORTER application 10016217, Repurposing of Pyrvinium Pamoate for Familial Adenomatous Polyposis (FAP) (5R44CA228840-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10016217. Licensed CC0.

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