Research & Development

  • Our Pipeline – Potential breakthrough targets
  • Unique RNA platform
  • Targeting unexplored proteins
  • Publications & posters
  • Grants

Our Pipeline – Potential breakthrough targets

Molecure discovers, develops and commercializes breakthrough small molecule drugs of the future which interact with novel RNA and protein targets to treat cancer, fibrotic and inflammatory diseases.
Molecure’s exceptional in-house medicinal chemistry and discovery capabilities combined with in-licensing from leading universities has created a broad pipeline of drug candidates targeting unique and unexplored protein targets, as well as a small molecule mRNA targeting discovery platform.


Targeting RNA – a new strategic direction

Human RNA offers access to thousands of new therapeutic targets with the ability to generate multiple small molecule medicines that have disease-modifying potential.

Molecure is developing a unique RNA platform to discover small molecule compounds which interact directly with mRNA to prevent downstream RNA translation and protein expression. The platform is focused on the mRNA of proteins involved in fibrotic disease and cancer. These proteins do not allow direct modulation of their function and are therefore considered undruggable using traditional small molecules.

Molecure has an exclusive partnership & research collaboration agreement with the International Institute of Molecular and Cell Biology (IIMCB) in Warsaw. This provides the company with access to world-leading mRNA science including a unique bioinformatics platform developed by the Laboratory of Bioinformatics and Protein Engineering (LBIB), headed by prof. Janusz Bujnicki. This platform gives Molecure the potential to outperform standard methods used in discovery of small molecule drugs targeting RNA.

Protein Targets

Molecure is discovering and developing first-in-class small molecules in oncology, inflammation and fibrosis that target/interact with selected, unexploited proteins.

Molecure has already validated the strength of its medicinal chemistry/translational capabilities by generating a diverse pipeline consisting of ten distinct programs

– 1 in the clinic and another molecule due to enter the clinic in the second half of 2022

– other programs are in the discovery phase, spanning and targeting 4 unchartered novel target families (arginases, chitinases, deubiquitinases and an undisclosed target).

Arginase Inhibitors

Arginase 1 (ARG1) and Arginase 2 (ARG2) are validated targets that have been found on a variety of tumor types where their increased activity correlates with more advanced disease and worse clinical prognosis due to diminished arginine levels.

Molecure’s lead proprietary candidate, OATD-02 is potentially the first and only dual acting, highly potent arginase inhibitor in cancer development, involved in both tumor immunity and metabolism. It has been selected as a clinical candidate for the potential treatment of a broad range of tumors in combination with other anti-cancer therapeutics. OATD-02 is expected to enter Phase I/II clinical trials in the second half of 2022.

Molecure, in collaboration with its partner SyVento, is also developing proprietary liposomal formulations of arginase inhibitors with the potential to increase their bioavailability and targeted exposure in the tumor.

Chitinase Inhibitors and chitinase-like-protein modulators

Chitotriosidase (CHIT1) and acidic mammalian chitinase (AMCase) are the enzymatically active chitinases that have been implicated in the pathology of chronic lung diseases such as asthma and interstitial lung diseases (ILDs), including idiopathic pulmonary fibrosis (IPF) and sarcoidosis.

Elevated levels of chitotriosidase (CHIT1) are associated with inflammatory and fibrotic diseases, driven by excessive activation of macrophages.

Molecure has developed a unique array of dual and selective chitinase inhibitors which modulate the function of macrophages, offering a new therapeutic approach to inflammatory and fibrotic disease.

Galapagos Deal – OATD-01 (GLPG4716)

OATD-01 is a first-in-class chitinase inhibitor for the treatment of fibrotic diseases, including idiopathic pulmonary fibrosis (IPF) and other interstitial lung diseases such as sarcoidosis.

OATD-01 is a dual inhibitor of CHIT1 and AMCase and has demonstrated in preclinical studies to have potent anti-inflammatory and anti-fibrotic effects in fibrous disease models, including lung fibrosis.

OATD-01 has been out-licensed to Galapagos NV for the global development and commercialization of the product, now named GLPG4716. GLPG4716 is due to enter a Phase II clinical trial for the treatment of IPF in 2022.

YKL-40 Program

YKL-40 is a chitinase-like protein (CLP), belonging to the chitinase family, however, has no enzymatic activity. YKL-40 has demonstrated growth factor activity and a role in cancer cell proliferation and survival by activation of key signaling pathways.

Molecure’s program has led to the discovery of OAT-3912 which strongly and selectively binds YKL-40 without blocking the activity of other chitinases. Preclinical colorectal cancer models demonstrate that OAT-3912 slows tumor growth by reactivating the immune response offering potential therapeutic benefit in various types of cancer.

Selective CHIT1 inhibitor

Molecure is developing selective CHIT1 inhibitors, structurally different from OATD-01, for the treatment of non-alcoholic steatohepatitis (NASH) and potentially a broad spectrum of neurological diseases that are characterized by excessive activation of inflammatory cells in the brain (neuroinflammation).

Deubiquitinase Inhibitors Program (DUBs)

Deubiquitinases (DUBs) are enzymes which modulate the ubiquitination and the subsequent degradation of proteins. Excessive activity of DUBs and resulting impaired ubiquitination is associated with many cancers and neurodegenerative diseases.

Molecure is developing inhibitors of DUBs, including a selective inhibitor of ubiquitin specific protease 7 (USP7), whose aberrant activation has been shown to promote oncogenesis, presenting as a promising therapeutic target for the treatment of cancer.