On March 21, 2023 EpicentRx Inc. ("EpicentRx"), a clinical stage biopharmaceutical company, reported the publication of a review article on its small molecule, RRx-001, a direct NLRP3 inflammasome inhibitor with activity in cancer, neurodegenerative diseases and other inflammatory conditions (Press release, EpicentRx, MAR 21, 2023, View Source [SID1234629107]). The article is entitled, A Review of RRx-001: A Late-Stage Multi-Indication Inhibitor of NLRP3 Activation and Chronic Inflammation.

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This is the first publication from the collaboration between EpicentRx and Associate Professor Richard Gordon’s team based at the Queensland University of Technology’s Centre for Microbiome Research (CMR) and the Translational Research Institute (TRI). Dr. Gordon is an internationally recognized expert on inflammasome signaling in neurodegenerative diseases. EpicentRx and Dr. Gordon’s team were awarded research grants from the Michael J. Fox Foundation (MJFF), Shake It Up Australia Foundation, and Fight MND to study the neuroprotective effects of RRx-001 in Parkinson’s Disease and Amyotrophic Lateral Sclerosis (ALS).

RRx-001 is one of the most advanced direct NLRP3 inflammasome inhibitors in clinical development and has been evaluated in over 300 multimorbid cancer patients both alone and in combination with other therapies. Currently in a Phase 3 trial for the treatment of small cell lung cancer, RRx-001 is also in late-stage development as an anti-mucositis agent in head and neck cancer and as a medical countermeasure against acute radiation syndrome (ARS) in the event of nuclear fallout. RRx-001 has been evaluated in multiple independent studies for conditions where immune and inflammasome activation contribute to disease pathology. These include cancer, myocardial infarction, pulmonary hypertension, acute kidney injury, acute radiation syndrome (ARS), malaria, multiple sclerosis, Parkinson’s disease, and Alzheimer’s disease.

Lead author Dr. Nanthini Jayabalan, a senior researcher working on the CNS RRx-001 program in Dr. Gordon’s lab said, "Our review article synthesizes the wealth of data on RRx-001 to date, which supports its disease-modifying potential in a range of conditions."

Added Dr. Gordon: "Emerging evidence suggests, chronic, unresolving inflammation is present in most if not all neurological diseases, and this makes RRx-001 an attractive therapeutic agent for neuroprotection given its track record in human studies."