New Clinical Trial Testing Drug for Huntington’s Disease

3-D brain wiring illustration (NIH)

19 February 2014. Omeros Corporation in Seattle began an intermediate-stage clinical trial testing its compound code-named OMS824 to treat symptoms of Huntington’s disease. The company says it’s administering the first doses in the trial, which is expected to enroll 120 patients.

Huntington disease is an inherited disorder in which nerve cells in certain parts of the brain degenerate. It is caused by a defect in a chromosome where a portion of the DNA repeats many more times than normal, and because the disease starts in the DNA, it is passed along from parents to children. Huntington’s disease is marked by hallucinations, restlessness, moodiness, paranoia, or psychosis, and in some cases progressive dementia.

About 30,000 people in the U.S., about 1 in 10,000,  have Huntington’s disease. There is no cure for the disorder, and the only approved drugs address symptoms involving involuntary movements.

OMS824, says the company, is a compound that binds to and regulates the phosphodiesterase 10 or PDE10 enzyme released in the brain. PDE10 is associated with disorders affecting cognition and psychomotor functions, including schizophrenia and Huntington’s disease. The U.S. Food and Drug Administration granted OMS824 an orphan drug designation and a fast-track review earlier this month.

In early-stage clinical trials, Omeros reported healthy volunteers were able to take single and multiple doses of OMS824 with at most mild adverse reactions, and without involuntary movements or tremors. The earlier trial also tested the binding effect of OMS824 with PDE10, as seen in positron emission tomography scans of the brains of the volunteers, and found OMS824 bound to an average of about two-thirds (66%) of PDE10 in the volunteers.

In the new clinical trial, some 120 patients with Huntington’s disease will be randomly assigned to receive one of three doses of OMS824 based on results from the early-stage safety trials, or a placebo. The study will assess the effects of the various drug doses against measures of motor, cognitive, and behavior factors. The company also plans to test a higher dose of OMS824 for increased binding with PDE10.

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