Small molecule activators of cAMP PDE4 long isoforms for the treatment of ADPKD

Autosomal dominant polycystic kidney disease (ADPKD) is a life-threatening and currently incurable genetic disorder of the kidneys characterised by the development of pathological cysts. It affects some 12.5 million people worldwide, with around 50% of sufferers requiring treatment for kidney failure by the age of 60. Mironid is applying its unique understanding of cellular signalling to develop a pioneering approach to ADPKD therapy, reversing the genetically driven chemical imbalance in the kidneys that drives cyst formation. The project will deliver novel active molecules for rapid optimisation into therapeutics that offer, for the first time, the ability to halt and reverse disease progression in ADPKD. Mironid’s new approach to modulating cellular signalling also has potential for treating a number of other diseases with a high unmet medical need, including both rare and more prevalent diseases. This innovative programme therefore provides an excellent opportunity to transform the treatment options available to patients, improve the cost-effectiveness of the NHS, whilst also maximising sustainable economic impact by further establishing Mironid as a leading UK pharmaceutical company with long term growth prospects and employment benefits.