Inflammasome Therapeutics Completes Enrollment for Oral Dual Inhibitor Study

Inflammasome Therapeutics, a private firm based in NEWTON, Mass., has successfully concluded enrollment for a Phase I pharmacokinetics and safety study of its innovative drug K9. This drug, capable of penetrating the brain and retina, shows promise in treating a variety of neuroinflammatory and degenerative diseases, including Alzheimer's, multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), Parkinson's, and geographic atrophy, as well as autoimmune diseases like lupus and thyroid eye disease.

The CEO and co-founder of Inflammasome Therapeutics, Dr. Paul Ashton, explains that these conditions are associated with a specific type of inflammation known as inflammasome activation. Notably, the activation of two particular inflammasomes accelerates the progression of these diseases. K9, administered in tablet form, successfully penetrates the brain and retina, effectively blocking both inflammasomes. Dr. Ashton highlights that drugs targeting a single inflammasome have shown limited or no efficacy in MS and macular degeneration models. In contrast, their Kamuvudine molecules, which inhibit multiple inflammasomes, have demonstrated significantly better results.

Overcoming the barriers of blood-brain and blood-retina to deliver therapies to disease sites driving persistent neuroinflammation in the central nervous system remains a formidable challenge. According to Dr. Jayakrishna Ambati, co-founder of Inflammasome Therapeutics, their data indicates that Kamuvudines possess disease-modifying potential by directly targeting inflammation within the central nervous system and peripheral regions.

The successful conclusion of the Phase I study sets the stage for additional clinical trials anticipated to commence later this year. This advancement paves the way for potentially groundbreaking treatments for debilitating conditions.

Moreover, Inflammasome Therapeutics recently initiated clinical trials for another dual inflammasome inhibitor, K8, targeting geographic atrophy, a severe stage of macular degeneration. This condition is a leading cause of blindness among the elderly and currently lacks an effective treatment. K8 is administered through a sustained delivery implant injected directly into the eye, releasing small but effective doses of the drug over three months.

Dr. Ambati has devoted over a decade to researching the role of inflammasomes in triggering inflammation, which is believed to underpin numerous neurodegenerative, autoimmune, and ophthalmic disorders for which no cures currently exist. His earlier research identified that individuals taking a class of anti-HIV drugs known as NRTIs had a significantly reduced risk of developing Alzheimer's disease and geographic atrophy. However, the substantial toxicity associated with NRTIs necessitated the development of new, safer molecules. This led to the creation of Kamuvudines, which retain the inflammasome-inhibiting effects of NRTIs but eliminate their toxicity.

Inflammasome Therapeutics, co-founded by Jayakrishna Ambati, M.D., and Paul Ashton, Ph.D., is committed to advancing therapies for prevalent degenerative diseases and pioneering novel delivery technologies for sustained release of therapeutic agents. The company merges scientific excellence with proven development expertise, striving to create groundbreaking products through a combination of licensing agreements and internal development.

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