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Positive Phase II Results for Potential Alzheimer’s Treatment
Positive clinical results have been reported from a phase II study of CHF 5074 (CereSpir Incorporated/Chiesi Farmaceutici), a first-in-class small-molecule microglia modulator, in patients with mild cognitive impairment (MCI).
The 90-week study, which included a 14-week, double-blind, placebo-controlled trial (n = 96) and an ongoing 76-week, open-label extension study, tested three titrated doses of oral CHF 5074 (200 mg, 400 mg, and 600 mg once daily) in MCI patients. Upon completion of the 14-week study, 74 patients entered the open-label extension phase and continued receiving CHF 5074 at a dose equal to that of their originally assigned double-blind study cohort.
An interim analysis of 30 patients at week 88 showed statistically significant improvements compared with baseline on several cognitive tests, including the Digit Symbol Substitution Test (P P
In the double-blind, placebo-controlled phase of the study, treatment with CHF 5074 resulted in a dose-dependent reduction in inflammatory biomarkers (TNF-alpha and sCD40L) in cerebrospinal fluid collected after 12 weeks of dosing.
CHF 5074 is a small molecule with a microglial-modulating mechanism of action capable of selectively reducing pro-inflammatory activities of microglial cells while increasing their ability to remove neurotoxic amyloid beta aggregates in the brain by phagocytosis. Microglia are small cells that migrate through the brain to remove waste products, such as amyloid aggregates that cause inflammation and irreversible damage to nerve cells. Chronic dysfunction of microglia is increasingly believed to play an important role in early Alzheimer’s disease.
Source: CereSpir Incorporated; July 26, 2013.