CHDI and Edison Pharmaceuticals have entered into alliance that looks to develop effective therapeutics in targeting Huntingdon's disease - a disorder that currently has no way to stop or reverse the its progression.
The deal's focus centres on a genetic disorder in which symptoms become apparent at midlife but in some people onset occurs in infancy or old age. The average survival time after onset is approximately fifteen to twenty years. It is estimated that about one in every 10,000 persons has the HD gene.
The CHDI and Edison Pharmaceuticals', partnership will develop analogs of CoQ(10) selectively targeted to reach the brain and address the mitochondrial component of Huntington's disease.
It is anticipated that a 2nd generation CoQ(10) molecule for Huntington's disease will eventually be developed.
The terms of the agreement will see Edison contribute expertise in redox pharmacology and translational biology relating to bioenergetics and disease. CHDI will contribute its competencies in Huntington's disease and drug development.
Additional terms of the agreement will see Edison retain rights to the development of therapeutics derived from this program. Financial terms of the agreements were not disclosed.
"As Edison has a demonstrated competency in the design and synthesis of CoQ(10) analogs, and more broadly in redox medicinal chemistry, they were a logical partner to pursue this line of therapeutic exploration," said Robert Pacifici, Chief Scientific Officer, Drug Discovery & Development and Chief Scientific Advisor to CHDI.
"The program centres on the identification of a next-gen CoQ(10) with improved blood brain barrier penetration properties that is also tailored to the discrete biochemical energy defect reasoned to be associated with Huntington's disease."
Recent laboratory and clinical investigations suggest that alterations in energy metabolism may contribute to Huntington's disease, and that administration of CoQ(10), a component of the mitochondrial respiratory chain and an endogenous antioxidant, may have beneficial effects in combating the progression of the disease.
"The CHDI partnership will allow us to leverage our skills and contribute more broadly to orphan indications such as Huntington's disease where emerging data suggests mitochondrial involvement in the disease mechanism," said Guy Miller, Chairman and CEO of Edison.
"CHDI's commitment to expediting a cure for Huntington's disease is evident in its organisational structure and the skill sets they have assembled. In combining forces, we will be able to rapidly derive data about the pharmacology and efficacy of redox analogs of CoQ(10) bioisosteres - which are of mutual interest to Edison and CHDI."
HD is a familial disease, passed from parent to child through a mutation in a gene. Each child of an HD parent has a 50-50 chance of inheriting the HD gene which causes programmed degeneration of brain cells and results in emotional disturbance, loss of intellectual faculties and uncontrolled movements.
Although there is no treatment to fully stop the progression of the disease, there are treatments available to help control the chorea, although these may have the side effect of aggravating bradykinesia or dystonia.
Other standard treatments to alleviate emotional symptoms include the use of antidepressants and sedatives, with antipsychotics (in low doses) for psychotic symptoms.
In the June 16, 2006 issue of Cell, scientists at the University of British Columbia (UBC) and Merck Labs presented findings that the neurodegeneration caused by mutant huntingtin (htt) is related to caspase-6 cleaving the enzyme. Transgenic mice that have caspase-6 resistant huntingtin did not show effects of the huntingtin enzyme.
The researchers found substantial support for the hypothesis that cleavage at the caspase-6 site in mhtt [mutant huntingtin] represents a crucial rate-limiting event in the pathogenesis of HD.