MMR Inhibition

 

MMR-d tumours commonly display high TMB and MSI-H status due to progressive accumulation of mutations that, once transcribed and translated, engender novel neoantigens and a subsequent CPI-responsiveness. Unfortunately, most human cancers, including most colorectal tumours, are MMR-p and unresponsive to CPI therapy. ​

​Thus, there is a strong rationale to develop a therapeutic intervention against key components of the MMR pathway. Treatment with MMR inhibitors in combination with CPI immunotherapies could provide clinical benefit by reawakening the patients’ anti-tumour immune response by effectively turning immunologically ‘cold’ tumours ‘hot’. ​

 
 

MMR inhibition with small molecules ​

We have focused our drug discovery efforts on the MutLα heterodimer partners MLH1 and PMS2 recognising their requirement for initiating excision of the errant DNA daughter strand – the first irreversible step of DNA repair. Of this pair, PMS2 harbours the critical exonuclease domain and has emerged as our priority target ahead of MLH1. ​