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New study begins for the treatment of cognitive defects in patients with Huntington's Disease

In a recent press release, EIP Pharma announced they have initiated a new Phase 2 proof-of-concept study evaluating neflamapimod as a treatment for the cognitive dysfunction associated with Huntington's disease.

According to the press release, the primary objective of the study is to determine whether neflamapimod, a brain-penetrant, oral small molecule that inhibits the enzyme p38 alpha, can reverse the hippocampal synaptic dysfunction associated with early Huntington's disease. Hippocampal synaptic dysfunction (i.e. impaired synaptic plasticity) is linked directly to memory deficits

John Alam, MD, founder and CEO of EIP Pharma said in the release, "We believe that neflamapimod has the potential to treat cognitive impairments associated with Huntington's disease and can therefore benefit patients early in the course of their disease. The initiation of this trial represents an important milestone for EIP as we expand our development pipeline and seek to bring new treatment options to patients affected by CNS (Central Nervous System) diseases."

EIP's press release states: Though Huntington's disease is often defined as a movement disorder, cognitive deficits generally precede the motor deficits. Recently, impairment in hippocampus-dependent cognitive function was defined as one of the earliest clinical manifestations of Huntington's disease. Previous studies in multiple animal models have demonstrated dysregulation of hippocampal synaptic dysfunction. 
This Phase 2 proof-of-concept study is a double-blind, placebo-controlled, two-period, 10-week treatment, within-subject, crossover study of neflamapimod in early-stage Huntington's disease (HD), with a washout period of eight to 12 weeks between the two treatment periods. The primary objective is to determine whether neflamapimod can reverse hippocampal dysfunction in patients with early-stage HD, as assessed by the virtual water maze test for evaluating spatial learning and selected tests on the Cambridge Neuropsychological Test Automated Battery (CANTAB). The study will be conducted at one clinical site in Cambridge, UK and will evaluate 16 patients in the cross-over design. Data from the study are expected to be reported in the second half of 2020.

About Neflamapimod Neflamapimod is a brain-penetrant, oral small molecule that inhibits the intra-cellular enzyme p38 MAP kinase alpha (p38α). P38α, which is expressed in neurons under conditions of stress and disease, plays a major role in inflammation-induced synaptic toxicity, leading to impairment of synaptic function. Synaptic dysfunction is known to be a major drive of the deficits in cognitive function that are defining characteristics of many CNS diseases. Neflamapimod is currently being studied in a Phase 2b trial for early Alzheimer's disease.


1 комментарий

Herwig Walter Lange
Herwig Walter Lange
09 июл. 2019 г.

Link to the press release: Let's hope that 10 weeks treatment show an effect. If too short, then a potentially helpful drug will not enter clinical evaluation.

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