Inflazome: Validation of a New Target in Parkinson’s Disease

Oct. 31, 2018 18:24 UTC

Innovative research was funded by The Michael J. Fox Foundation for Parkinson’s Research and the Shake It Up Australia Foundation

DUBLIN & CAMBRIDGE, England--(BUSINESS WIRE)-- Inflazome Ltd., (“Inflazome”) is a pioneering biotech company developing small molecule drugs that stop harmful inflammation by targeting inflammasomes. Research leading towards a potential approach to treat Parkinson’s patients was published today in Science Translational Medicine. The study was co-authored by Inflazome Co-founder and Chief Executive Officer, Prof. Matt Cooper with research teams at The University of Queensland, Australia, led by A/Prof. Trent Woodruff. Funding for these studies was provided by The Michael J. Fox Foundation for Parkinson’s Research and the Shake It Up Australia Foundation.

Parkinson’s disease is the second most common neurodegenerative disorder worldwide, affecting more than 10 million people. It is characterised by the loss of dopamine-producing neurons, accompanied by chronic inflammation in the brain. Inflazome has identified drugs to stop the chronic cycle of inflammation in the brain. The researchers found that the tool compound MCC950, a potent inhibitor of the NLRP3 inflammasome, given orally once a day could stop neuroinflammation. MCC950 arrested the effects of Parkinson’s in several animal models of the disease, leading to reduced brain neuron loss and higher levels of dopamine.

NLRP3 inflammasome activation drives chronic inflammation and is implicated in many diseases - from Parkinson’s and Alzheimer’s to Asthma, Inflammatory Bowel Disease, Chronic Kidney Disease, Cardiovascular Disease, Arthritis and the liver disease, NASH. Inflazome is developing orally available drugs to address clinical unmet needs in these inflammatory diseases by blocking the activation of inflammasomes in our immune system.

Prof. Matt Cooper, Co-founder and CEO of Inflazome, commented: “We are indebted to The Michael J. Fox and Shake It Up Australia Foundations for supporting this work. We also thank those generous individuals affected by Parkinson’s who donated samples to aid medical research. Drug companies have tried for decades to develop medicines to combat neurodegenerative diseases by blocking the many different neurotoxins and amyloids that build up in the brain as we age.”

“The problem is that if one toxin is blocked by a drug, another may still build up and cause disease. In contrast, our line of research focuses not on individual toxins, but instead on the immune cells in our brains that clear amyloid and other neurotoxins. These cells, called microglia, normally protect us from infections that can lead to encephalitis and meningitis. However, as we age our immune system can become over-activated, which leads to neuroinflammation. Over-active microglia can no longer function as efficient ‘cleaners’ of neurotoxins, but instead contribute to long-term damage in the brain.”

The progression of Parkinson’s disease was studied using brain and blood samples donated by patients for medical research. The study demonstrated a key target in microglia, called the NLRP3 inflammasome, is highly activated in Parkinson’s. The same signals produced by inflammasome activation in human brains were seen in different pre-clinical models of Parkinson’s disease. A small molecule, MCC950, prevented the progression of Parkinson’s in these animal models, leading to reduced brain cell loss, and higher levels of dopamine and motor function to effectively ‘cool the brain on fire’.

Prof. Cooper added “We look forward to progressing this research through to clinical trials using our proprietary, improved drug candidates”.

Kuldip Dave, PhD, Director of Research Programs at The Michael J. Fox Foundation said, “Inflazome has validated a promising new target for Parkinson’s therapeutics and translated that finding into a potential drug to treat Parkinson’s disease. This is a key aspect of The Michael J. Fox Foundation’s research strategy, and we look forward to their candidate drug’s continued development.”

“Shake It Up is proud and excited to be a part of the collaboration’s funding team, allowing highly-talented researchers an opportunity to create world leading breakthroughs that have the opportunity to be a game changer for people with Parkinson’s,” said Clyde Campbell, Founder and CEO of the Shake It Up Foundation.

The Science Translational Medicine paper can be found here

A link to an explainer video can be found here


About Inflazome

Utilizing the scientific expertise of our founders and advisors, Inflazome is leading the way in developing orally available drugs to address clinical unmet needs in inflammatory diseases by targeting the NLRP3 inflammasome, which is now understood to drive many chronic and acute inflammatory conditions from Parkinson’s and Alzheimer’s to Cardiovascular Diseases.

Headquartered in Dublin, Ireland, Inflazome was founded in 2016 by leading academics Prof. Matt Cooper, The University of Queensland (Australia) and Prof. Luke O’Neill, Trinity College Dublin (Ireland), following a highly productive joint collaboration.

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Source: Inflazome

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