EIP Pharma has started a proof-of-concept Phase II clinical trial to assess its investigational drug neflamapimod for treating cognitive dysfunction caused by Huntington's disease.
Huntington's is a rare, genetic disorder characterised by progressive degeneration of nerve cells of the brain. Recent findings revealed hippocampus-dependent cognitive function impairment as one of the earliest signs of the disease.
Neflamapimod is an oral, brain-penetrant, small molecule designed to inhibit the intra-cellular enzyme p38 MAP kinase alpha (p38α), which is known to be associated with impairment of synaptic function.
Synaptic dysfunction is considered to be responsible for the cognitive function deficits related to several CNS diseases.
The Phase II study is being conducted to assess neflamapimod over ten weeks in 16 early-stage Huntington's disease patients at a single site in Cambridge, UK.
This double-blind, placebo-controlled, two-period crossover trial will involve an eight to 12-week washout period between the two treatment periods.
The primary objective of the trial is to establish whether neflamapimod will be able to reverse the hippocampal synaptic dysfunction associated with early Huntington's.
The outcome will be evaluated using the virtual water maze test for spatial learning and certain Cambridge Neuropsychological Test Automated Battery (CANTAB) tests.
Trials results are expected to be available in the second half of next year.
EIP Pharma founder and CEO John Alam said: “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 diseases.”
Neflamapimod is also being evaluated in a Phase IIb study involving early Alzheimer's disease patients.