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Research: Healthcare
Molecure has announced that it has generated the in vitro proof-of-concept (PoC) data for its mRNA platform, marking a step toward one of its long-term strategic objectives. Based on an in vitro study, management has confirmed the small molecule inhibition of the translation of a protein implicated in cancer via direct binding to mRNA encoding this protein. While the field of RNA-based biopharmaceuticals has gained traction during the pandemic with the use of mRNA vaccines, Molecure is one of the few biotechs focused on advancing small molecules designed to directly interact with mRNA structures. This approach has potential advantages compared to biologics, most notably patient compliance, given that most of the small-molecule candidates will likely be designed to be dosed orally (versus the more invasive injection/intravenous approaches that biologics generally require). While Molecure is in the early stages of developing this platform approach, we believe the announcement of PoC data marks an important milestone. Management will now evaluate continued preclinical and potentially clinical development of lead molecules optimised in this programme.
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Molecure |
Progressing the validation of mRNA platform
Pharma and biotech |
Spotlight – Flash
19 December 2023 |
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Molecure is a research client of Edison Investment Research Limited |
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Molecure has announced that it has generated the in vitro proof-of-concept (PoC) data for its mRNA platform, marking a step toward one of its long-term strategic objectives. Based on an in vitro study, management has confirmed the small molecule inhibition of the translation of a protein implicated in cancer via direct binding to mRNA encoding this protein. While the field of RNA-based biopharmaceuticals has gained traction during the pandemic with the use of mRNA vaccines, Molecure is one of the few biotechs focused on advancing small molecules designed to directly interact with mRNA structures. This approach has potential advantages compared to biologics, most notably patient compliance, given that most of the small-molecule candidates will likely be designed to be dosed orally (versus the more invasive injection/intravenous approaches that biologics generally require). While Molecure is in the early stages of developing this platform approach, we believe the announcement of PoC data marks an important milestone. Management will now evaluate continued preclinical and potentially clinical development of lead molecules optimised in this programme.
Molecure announced that based on its in vitro cell-based assay (ie typical for early-stage preclinical research) it was able to confirm that its small molecule directly interacted with mRNA to inhibit the translation of an encoded protein implicated in cancer. The company also stated that the extent of protein synthesis inhibition correlated with the concentration of small molecules used, suggesting a dose-dependence, which we believe helps validate Molecure’s approach. Its mRNA platform first involves an assessment of an mRNA structure, including the identification of possible binding regions. The functionality of the identified region(s) is then confirmed, followed by modelling of its tertiary structure and virtual screening. The binding of the most promising small molecule compounds is then assessed using biophysical methods, before evaluating the inhibition of the protein translation encoded by the mRNA.
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The generation of in vitro PoC data marks an important milestone for Molecure, as the field of small molecule mRNA inhibition may still be considered in its infancy. The company will now evaluate the possibilities of continued preclinical and potentially clinical development of lead molecules optimised in this programme.
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Research: Healthcare
Nicox has reported that an in vivo study carried out in beagles found that NCX-470 (0.1%) treatment led to a greater degree of intraocular pressure (IOP) reduction compared to AbbVie’s Lumigan (bimatoprost 0.01%), the best-selling branded prostaglandin F2α analogue (PGA) drug, in both single and repeated (five-day) dosing. The newly reported data from the open-label cross-over study assessing NCX-470, Lumigan and vehicle were published in the peer-reviewed Journal of Ocular Pharmacology and Therapeutics. While we acknowledge the limitations in read-across from animal data and note the Mont Blanc study did not show statistical superiority against PGA drug latanoprost (although it did meet the non-inferiority primary efficacy endpoint), we believe the new findings add to the body of collective data suggesting a competitive commercial profile for NCX-470 versus standalone PGA drugs such as bimatoprost.