Molecure has obtained confirmation of its scientific assumptions (so-called proof of concept) in its ongoing program to discover small molecules targeting mRNA, the company announced.
The company confirmed in in vitro tests that the small-molecule compounds it develops as part of the mRNA platform bind to a selected mRNA fragment and inhibit the translation of the protein encoded by this mRNA. Confirmation of the mechanism of stopping the translation of a pathological protein, in which the degree of inhibition of protein synthesis depends on the concentration of the compound, is an important milestone in the development of the mRNA platform and is one of the company’s strategic goals for 2023-2025, it was emphasized.
“The initial stage of scientific research in the mRNA platform was the identification of the structure of the mRNA region with a high potential for binding small-molecule compounds (druggable regions). After confirming the functionality of this region, we performed modeling of its tertiary structure, a necessary step to move to virtual screening. We confirmed the binding of the best compounds to the mRNA fragment using biophysical methods. The identified compounds, interacting directly with the mRNA fragment, showed inhibition of the translation of the protein encoded by the given mRNA – which is our PoC for the first mRNA target in the developed platform. This is a huge success for our scientists involved in such an innovative project and at the same time confirmation of our ambitious assumptions for the development of the platform. The next stage will be to assess the possibility of further preclinical and potentially clinical development of the program. The company is also expanding its internal expertise in identifying new mRNA regions that may constitute attractive therapeutic targets,” said Zbigniew Zasłona, chief scientific officer and member of the management board of Molecure, quoted in the release.
“The development of the mRNA platform, next to our two clinical programs, is one of Molecure’s strategic priorities, and achieving the in vitro proof-of-concept stage this year is an important milestone that we achieved within the assumed deadline. This will allow us to intensify partnering talks and increases the likelihood of establishing commercially attractive cooperation with a large industry partner,” added CEO and president of the management board Marcin Szumowski.
The company’s business model for the mRNA platform assumes a hybrid approach. Molecure’s goal is both to develop its own projects targeting internally selected attractive mRNA targets, as well as to provide services to external companies from the biopharmaceutical sector, consisting in the validation of mRNA fragments selected by the client as a therapeutic target. This approach enables partnering and reaching the revenue generation phase already at an early stage of optimization of active compounds, i.e. after achieving in vitro PoC, it was recalled.
Molecure (formerly OncoArendi Therapeutics) is a biotechnology company specializing in the search, development and commercialization of new drugs used in the treatment of respiratory diseases and cancer. The company has been listed on the main market of the WSE since 2018; is included in the sWIG80 index.