New molecules for the treatment of inflammatory diseases

The NLRP3 inflammasome is a multiprotein complex that plays a central role in the activation of the immune system and the consequent onset and progression of many different diseases. The activation of NLRP3 brings to the release of powerful inflammatory and triggers a pro-inflammatory cell death process known as “pyroptosis”. A series of proof of concept studies demonstrated that the block of NLRP3 activation is an efficient strategy to treat chronic inflammatory, autoinflammatory, autoimmune and neurodegenerative diseases. New molecules able to inhibit NLRP3 for the treatment of various pathological conditions have therefore been developed. Specifically, the object of the patent consists of new chemical entities belonging to the "small molecules”, which have been validated in different animal models. Compared to conventional therapies currently used for the treatment of chronic inflammatory, autoimmune and neurodegenerative diseases, the use of NLRP3-blocking molecules offers a more practical, more effective and safer therapy. According to biopharma market outlook reports “Inflammasome Science” is one of the top five ranked events in 2020. To date, no NLRP3 inhibitors have reached the market yet.

• Do not block immune response from other biological pathways (i.e, other inflammasomes) thus preserving the body’s ability to respond to infections by invading pathogens
• Can be delivered orally, thus improving patient’s compliance
• Blocks inflammatory response and cell death by pyroptosis
• The pharmacokinetics can be tailored through the development of molecules endowed with selectivity for different body districts
• Is less expensive compared to the use of biological IL-1β blockers

Filing date: 3/5/2021

Application number: 102021000011237