Inspirna’s novel target discovery platform, miRNA-DRIVEr, reveals new cancer drivers through microRNA profiling.

Inspirna has developed the unique expertise to discover novel and actionable cancer drivers that affect large patient populations. We conduct comprehensive studies including genomic, transciptomic, proteomic and metabolomic approaches for the identification and validation of our targets as well as the characterization of the underlying biology. The team has extensive expertise across all areas of drug development to move programs quickly from lab-to-clinic. Our approach has generated extensive scientific and preclinical data, which have led to multiple publications in high profile journals.

Our proprietary and clinically-validated miRNA-DRIVEr platform, coupled with our expertise in miRNA mapping, allows us to reveal new cancer drivers and actionable targets of miRNA dysregulation.

miRNA-DRIVEr = miRNA Dysregulated Drug Target In Vivo Elucidation

Why target miRNA regulated pathways?

Micro RNAs (miRNAs)* have important biological functions:

  • Regulate gene expression at the post-transcriptional level;
  • Modulate cell growth and differentiation;
  • Regulate pathways and networks via coordinated activities;
  • Act co-operatively with other miRNAs and with transcription factors, which are frequent targets of miRNAs.

miRNA dysregulation is a frequent contributor to cancer growth and progression.

  • Overexpression or depletion of distinct sets of miRNAs is frequently found in cancer.
  • Delineating miRNA functional effects requires elucidation of their upstream regulators and downstream targets.

*short, endogenous noncoding RNAs 19–22 nucleotides long

Our miRNA-DRIVEr platform reveals actionable miRNA-dysregulated cancer targets

We are dedicated to stopping metastatic progression and providing long-term survival benefits for patients by providing more practical treatment options that prolong survival without decreasing quality of life. Our innovative medicines possess improved dosing schedules and safety profiles that will become seamless and simple additions to the existing standard of care (i.e., oral BID) to treat indications with large patient populations and high unmet need.

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We discover critical targets that drive the growth of metastatic disease.

We start by purifying the extremely rare, aggressive cancer cells that are highly effective in driving the growth of metastatic disease. We then identify miRNAs that are dysregulated in these metastatic cells and search for the protein that is regulated by them.

microRNA Profiling

Generic Parental Cell
Poorly metastatic
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Inspirna’s Proprietary
Highly metastatic
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We test and validate the role of the discovered gene in driving metastatic disease.

We perform a series of extensive molecular and genetic studies to determine the role of the identified gene on driving metastatic disease in mice, utilizing state-of-the-art models including cell line and patient derived xenografts in immunocompetent and immunocompromised mouse models. These mouse studies are corroborated by clinical association studies where the expression levels of the gene are tested for association with cancer patients’ survival outcomes in multiple clinical datasets.

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Our experienced team develops new drug candidates suitable for clinical evaluation.

Having validated our targets through robust in vitro and in vivo phenotypic assays as well as genetically modified cells and models, we design and screen lead small-molecules or monoclonal antibodies that specifically alter the activity of the critical protein. Such leads are optimized to yield first in class therapeutics that exhibit strong activity against both primary tumor growth and metastatic progression in vivo.

SLC6A8 = Solute Carrier family 6 member 8
LXR = Liver X Receptor

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We transform cancer medicine by leveraging RNA biology driven insights.

Inspirna’s deeply mechanistic studies that start with the identification of miRNA regulators in metastatic disease to the in vitro and in vivo validation of the downstream protein targets, coupled with our human clinicopathologic correlative data allow to design novel and innovative therapies. Our highly experienced clinical team uniquely positions Inspirna to advance these first-in-class drug candidates from IND to clinical proof of concept studies and beyond. Our extensive mechanistic pre-clinical studies guide our biomarker enriched patient selection, which is validated and refined by phase 1b expansion trial findings.

Presentations & Publications

Science underlying Inspirna’s pipeline has been published in leading scientific journals and presented at renowned medical conferences.

Ompenaclid (RGX-202)

  • ESMO GI 2022 Poster

    Phase 1b study of ompenaclid (RGX-202-01), a first-in-class oral inhibitor of the SLC6a8/CKB pathway, in combination with FOLFIRI and bevacizumab (BEV) in second-line advanced colorectal cancer (CRC)

  • ASCO 2022 Poster

    Phase 1b study of ompenaclid (RGX-202-01), a first-in-class oral inhibitor of the SLC6a8/CKB pathway, in combination with FOLFIRI and bevacizumab (BEV) in second-line advanced colorectal cancer (CRC)

  • Science Advances - Oct, 2021

    Therapeutic targeting of SLC6A8 creatine transporter suppresses colon cancer progression and modulates human creatine levels.

  • ASCO 2020 Presentation

    Phase 1 monotherapy dose-escalation of ompenaclid (RGX-202), a first-in-class oral inhibitor of the SLC6a8/CKB pathway, in patients with advanced gastrointestinal (GI) solid tumors

  • AACR 2018 Presentation

    Ompenaclid (RGX-202), a first-in-class small-molecule inhibitor of the creatine transporter SLC6a8, is a robust suppressor of cancer growth and metastatic progression

  • Cell - Jan, 2015

    Extracellular Metabolic Energetics Can Promote Cancer Progression (Cell160, 393–406, January 29, 2015)

Abequolixron (RGX-104)

  • AACR 2020 Poster

    Correlative analysis of pharmacokinetics and pharmacodynamics of RGX-104, a first-in-class Liver-X-Receptor (LXR) agonist, and clinical outcomes in patients with advanced solid tumors

  • AACR 2020 Presentation

    RGX-104, a first-in-class immunotherapy targeting the Liver-X Receptor (LXR); Initial results from the Phase 1b RGX-104 plus Docetaxel combination dose escalation cohorts

  • ASCO 2018 Poster

    Pharmacodynamic and clinical activity of RGX-104, a first-in-class immunotherapy targeting the liver-X nuclear hormone receptor (LXR), in patients with refractory malignancies

  • Cell - Feb, 2018

    LXR/ApoE Activation Restricts Innate Immune Suppression in Cancer (Cell172, 1–16, February 8, 2018)

  • Cell - Feb, 2014

    Broad-Spectrum Therapeutic Suppression of Metastatic Melanoma through Nuclear Hormone Receptor Activation (Cell156, 1–16, February 27, 2014)

  • Cell - Nov, 2012

    Convergent Multi-miRNA Targeting of ApoE Drives LRP1/LRP8-Dependent Melanoma Metastasis and Angiogenesis (Cell151, 1068–1082, November 21, 2012)


  • AACR 2020 Poster

    In vivo efficacy and safety of RGX-019, a MerTK targeting monoclonal antibody

  • AACR 2019 Presentation

    Characterization of the anti-cancer and immunologic activity of RGX-019, a novel pre-clinical stage humanized monoclonal antibody targeting the MERTK receptor

Discovery Platform

  • Nature Cell Biology - Jun, 2013

    Control of Metastatic Progression by microRNA Regulatory Networks (Nat Cell Biol. 2013 Jun; 15(6): 546–554)

  • Nature - Oct, 2012

    A microRNA regulon that mediates endothelial recruitment and metastasis by cancer cells (Nature 481, 190–194 (2012))

  • Nature - Jan, 2008

    Endogenous human microRNAs that suppress breast cancer metastasis (Nature volume 451, pages 147–152 (2008))