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Inzomelid completes Phase I studies and shows positive results in the treatment of Cryopyrin-Associated Periodic Syndrome (CAPS)

  • The Phase I study demonstrated Inzomelid has an excellent safety, tolerability and pharmacokinetic profile in healthy subjects, with dose-dependent target engagement
  • Inzomelid also delivered positive preliminary results from a patient with Cryopyrin-Associated Periodic Syndrome (CAPS)
  • Inzomelid is a potent, selective, brain-penetrant NLRP3 inflammasome inhibitor intended for treatment of debilitating inflammatory diseases of the brain and is expected to move into Phase II trials this year

Dublin (IE), Cambridge (UK), Brisbane (AU) | 26 March 2020: Inflazome (, the pioneering inflammasome biotech company developing multiple drugs that stop harmful inflammation, today announces the successful completion of a Phase I study of Inzomelid, alongside positive results from a Cryopyrin-Associated Periodic Syndrome (CAPS) patient dosed with Inzomelid. CAPS is an autoinflammatory orphan disease driven by mutated NLRP3. The results support the progression of Inzomelid into Phase II clinical trials.

Inzomelid is a selective, orally available small molecule inhibitor of the NLRP3 inflammasome. Activation of the NLRP3 inflammasome is implicated in many diseases caused by chronic, harmful inflammation.

Drug exposures in the double blinded and randomised Phase I trial in healthy subjects increased linearly with dose, and correlated with markers of target engagement. The drug also demonstrated an excellent safety and tolerability profile.

Preliminary analysis from the CAPS part of this study showed that a patient with a confirmed NLRP3 mutation suffering from a CAPS-related flare showed rapid clinical improvement within hours and remission within days. A follow-on Phase II trial is planned to identify the best dose for CAPS patients.

Inzomelid follows Inflazome’s other NLRP3 inflammasome inhibitor, Somalix, in successfully completing Phase I trials. Somalix, an orally available, peripherally-restricted drug for inflammatory diseases, also demonstrated excellent safety, tolerability and pharmacokinetics in healthy subjects and is expected to enter Phase II trials in 2020.

Dr. Thomas Jung, Chief Medical Officer of Inflazome, commented, “The positive Phase I results from our two lead candidates, Inzomelid and Somalix, further validate our technology platform as we head with real momentum towards multiple Phase II trials later this year. The Inzomelid CAPS data also represents a very exciting step for the field in orphan indications. We believe this is the first example of positive data in a human disease setting with a small-molecule NLRP3 inhibitor. Our primary motivation is to develop drugs for which a high unmet medical need exists, so we are pleased to see that Inzomelid has demonstrated clear clinical potential.” 

About Inflazome      

Inflazome is a biotech company leading the development of orally available drugs to address clinical unmet needs in inflammatory diseases by targeting inflammasomes.  Inflammasomes are understood to drive many chronic inflammatory conditions, from Parkinson’s and Alzheimer’s to asthma, inflammatory bowel disease, chronic kidney disease, cardiovascular disease, arthritis and NASH. Inflazome has two clinical-stage investigational drugs. Inzomelid is under development as an orally available, brain-penetrant drug and Somalix is under development as an orally available, peripherally-restricted drug. Both will address clinical unmet needs in inflammatory conditions. Inflazome is headquartered in Dublin, Ireland, with offices in Cambridge, UK and Brisbane, Australia.

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About the NLPR3 Inflammasome

Activated NLRP3 acts as a ‘danger sensor’ in the body to release the pro-inflammatory cytokines IL-1β, IL-18 and induce uncontrolled, lytic cell death (pyroptosis). These processes lead to chronic inflammation, and as such, NLRP3 has been implicated in a large number of diseases.

About Cryopyrin-associated periodic syndromes

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.

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Last Updated: 26-Mar-2020