Molecure’s success in development of the mRNA discovery platform

Molecure confirms that small molecules designed to target mRNA inhibit translation of the encoded protein relevant in cancer

Proof-of-Concept has been demonstrated in a cell assay

Reaching the PoC stage for the first mRNA target provides evidence of the effectiveness of Molecure’s strategy in identifying mRNA-binding compounds with therapeutic potential

The success in advancing the mRNA platform increases the likelihood of signing a collaboration agreement with partners operating in this field


Warsaw, December 12, 2023 – Molecure S.A. (“Molecure”: WSE: MOC) a clinical stage biotechnology company developing first-in-class small molecule drug candidates that directly modulate unexplored protein and mRNA targets to treat multiple incurable diseases, has confirmed in vitro that small molecule binding to the selected mRNA fragment inhibit the translation of the protein encoded by that mRNA. Confirmation of the mechanism of stopping the pathological proteins translation in a dose dependent manner, represents a significant milestone in the development of the mRNA platform and has been one of the strategic goals of the Company during 2023-2025.

'The initial stage of scientific research in the mRNA platform involved identifying the structure of the mRNA region with high potential for binding small molecules (druggable regions). After confirming the functionality of this region, we conducted modeling of its tertiary structure, a necessary step to proceed to virtual screening. The binding of the best compounds to the mRNA fragment was confirmed using biophysical methods. The identified molecules, directly interacting with the mRNA fragment, demonstrated inhibition of the protein translation encoded by the given mRNA in a dose dependent manner. These results mark our anticipated Proof-of-Concept (PoC) for the first mRNA target in the platform. This big success achieved by our scientists validated of our ambitious approach for platform development. The next stage will involve evaluating the possibilities of continued preclinical and potentially clinical development of lead molecules optimized in this program. The company is also expanding its in-house expertise in identifying new mRNA regions that may serve as attractive therapeutic targets’ says Dr. Zbigniew Zasłona, Chief Scientific Officer, and Member of the Board at Molecure S.A.


'We are proud to have achieved the in vitro Proof-of-Concept (PoC) for the first hit molecules developed in the mRNA platform. As the result of combining creativity, innovative approach, and the determination of our team in discovery of new molecules, leveraging strong in-house expertise in drug design using advanced digital methods. It confirms that we are among the global leaders in the field of mRNA-targeting small molecule drugs, a breakthrough technology with the potential to change the paradigm of treating many diseases, where the protein structure itself prevents direct interaction with small molecules (so-called undruggable targets). The development of the mRNA discovery platform, alongside our two clinical programs, is one of Molecure’s strategic priorities. Achieving the in vitro proof-of-concept this year, as outlined in our 2023-2025 strategy, is a significant milestone that we have reached and confirmed as planned. This will enable us to intensify partnering discussions and increase the likelihood of establishing commercially attractive collaborations with industry partners’ says Dr. Marcin Szumowski, CEO, and President of the Board at Molecure S.A.

The business model of the Company in the mRNA platform involves a hybrid approach. Molecure aims to develop its own proprietary projects targeting internally selected mRNA structures as well as provide services to external companies in the biopharmaceutical sector. These services involve validating mRNA fragments chosen by the client as therapeutic targets. This approach enables partnering and the generation of revenue as early as the optimization stage of active binders, i.e., after achieving in vitro Proof-of-Concept (PoC) for the relevant mRNA target.

Molecure is one of the few biotechnology companies globally developing small molecule drugs that directly interact with mRNA targets.


About Molecure S.A.

Molecure is a clinical stage biotechnology company that uses its world leading medicinal chemistry and biology capabilities to discover and develop first-in-class small molecule drug candidates that directly modulate underexplored protein targets and the function of RNA to treat multiple incurable diseases.

Molecure has generated a diverse pipeline of seven distinct programs with the support of leading academic life science institutions globally, including Yale University, Rutgers University, the Flemish Institute for Biotechnology (VIB) in Ghent, the University of Michigan and the International Institute of Molecular and Cell Biology in Warsaw (IIMCB).

Molecure’s most advanced in-house drug candidate is OATD-01, a first-in-class dual chitinase inhibitor for the treatment of interstitial lung diseases, such as sarcoidosis and idiopathic pulmonary fibrosis, that is Phase II ready. A Phase II trial in patients with sarcoidosis is expected to start in 4Q 2023.

OATD-02 is the second candidate drug, an oral, selective, first-in-class, dual inhibitor of arginase (ARG1 and ARG2), intended for the treatment of cancers. The Phase I clinical trial commenced with the first patient dosing in 1Q 2023.

The headquarters and laboratories of Molecure are located in Warsaw and Łódź. The company is listed on the Warsaw Stock Exchange (ticker: MOC).

Further details can be found on the following pages:

LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA

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