# Small molecule inhibitors of oncogenic miR-21

> **NIH NIH R41** · ITHAX PHARMACEUTICALS, INC. · 2020 · $249,109

## Abstract

Project Summary:
Company development - Ithax Pharmaceuticals is a new spin-off biotechnology company located in Seattle and
focused on developing small drug-like molecules that target non-coding RNAs in cancer and other chronic
diseases. The experience of its founders’ spans drug design, RNA structure, cancer biology and biotech
management, and includes the successful spin offs of several biotech companies. It gives the company an
unmatched experience in the technological and commercial development of new companies in the RNA-
oncology space.
 This Phase I STTR proposal lays out a series of experiments, beyond the scope of academic discovery,
critical to advance and de-risk the commercialization plans of Ithax in the eyes of investors and pharmaceutical
partners. Completion of this project will help the company fill gaps left in the academic discoveries of the founders
by providing the data required to initiate medicinal chemistry optimization under a subsequent already planned
phase II SBIR, and to successfully raise private capital needed for pre-clinical and clinical development.
Technology - The proto-oncogenic non-coding RNA miR-21 is over-expressed in nearly every human tumor. Its
over-expression is linked to unfavorable patient prognosis in multiple cancers and resistance to
chemotherapeutic and immunotherapeutic treatment. Accumulating evidence in many cellular and multiple small
animal models of cancers demonstrates that pharmacological inhibition of miR-21 would stop the progression of
even late stage cancers, which are ‘addicted’ to this oncomiR, reduce chemo-resistance and potentiate
checkpoint immunotherapy.
 Ithax’s first lead small molecule emerges from a collaboration between the University of Washington and
the MD Anderson Cancer Center to identify new inhibitors of miRNA biogenesis. Preliminary data demonstrate
that ITX-0052, a ‘Lipinski’ drug-like molecule with very safe in vitro pharmacological profile, inhibits miR-21
accumulation and reduces proliferation of gastric and pancreatic cancer cells with low M IC50 by binding directly
to pre-miR-21. This project will establish the structural, biochemical and cellular mechanism of inhibition; and
evaluate safety of administration, pharmacokinetics, pharmacodynamics and efficacy in murine models of gastric
cancer.

## Key facts

- **NIH application ID:** 10081975
- **Project number:** 1R41CA254746-01
- **Recipient organization:** ITHAX PHARMACEUTICALS, INC.
- **Principal Investigator:** George A. Calin
- **Activity code:** R41 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $249,109
- **Award type:** 1
- **Project period:** 2020-08-14 → 2023-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10081975

## Citation

> US National Institutes of Health, RePORTER application 10081975, Small molecule inhibitors of oncogenic miR-21 (1R41CA254746-01). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10081975. Licensed CC0.

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