NLRP3 activation plays a role in many diseases. At Inflazome we have narrowed our focus to develop our suite of therapies for the following diseases where is validated involvement of NLRP3 and the potential to significantly benefit patients:
Cryopyrin-associated periodic syndromes (CAPS), also called cryopyrin-associated autoinflammatory syndromes, are three diseases related to a defect in the NLRP3 gene. CAPS encompasses neonatal onset multisystem inflammatory disease (NOMID), Muckle-Wells syndrome (MWS) and familial cold autoinflammatory syndrome (FCAS). The differences in these diseases lie in their severity and the organs involved.
Parkinson’s Disease (PD) is a chronic progressive neurodegenerative condition caused by the death of key cells in the brain, leading to the loss of dopamine, a chemical used to control the movements a person makes as well as emotional responses. While symptoms can be controlled by Levodopa therapy over a few years, the disease is still progressing and no disease-modifying treatments are currently available. Targeting neuroinflammation through inhibiting NLRP3 could address this major unmet medical need.
Approximately 30 million people globally have Alzheimer’s Disease (AD), for which there is no known cure. The pathogenesis of AD is widely believed to be driven by the production and deposition of the β-amyloid peptide (Aβ). Aβ drives neuroinflammation leading to neuronal death and disease progression involving NLRP3 activation. As such, inhibition of NLRP3 may slow or stop the progression of AD.
Gout is a relatively common disease, with a prevalence of 1-2 % in Europe, and 3-4% in the USA. Current drug therapies focus on anti-inflammatory/analgesic and the lowering of the serum urate concentration. In the chronic treatment phase, there is a sub-population of patients that do not respond to treatment. This can be due to hypersensitivity to the drug or adverse events. These patients, called ‘refractory gout patients’, have a significant unmet need and are the focus for Inflazome.
three areas of cardiovascular disease - Heart Failure, Atherosclerosis and Myocardial Infarctions - have shown potential for treatment with NLRP3 inhibitors. Inflazome is exploring clinical proof of concept indications for Somalix, a non-brain-penetrant oral NLRP3 inhibitor, in cardiology. This strategy is initiated by the observation that inflammation reduction has a strong impact on patients with atherosclerosis as indicated by results derived from various clinical trials.
Osteoarthritis (OA) is the most common form of arthritis in the world affecting approximately 3.5% of the population. OA is caused when inflammatory proteins and proteases cause joint destruction. NLRP3 activation has been shown to drive the inflammatory component and its inhibition may arrest disease progression.