Dystonia is a movement disorder that causes the muscles to contract and spasm involuntarily, according to the Dystonia Medical Research Foundation.
The involuntary muscle contractions force the body into repetitive and often twisting movements as well as awkward, irregular postures. There are approximately 13 forms of dystonia, and dozens of diseases and conditions include dystonia as a major symptom.
Dystonia may affect a single body area or be generalized throughout multiple muscle groups. Dystonia affects men, women, and children of all ages and backgrounds. Estimates suggest that no less than 300,000 people in North America are affected. Dystonia causes varying degrees of disability and pain, from mild to severe. There is presently no cure, and, although many drugs are utilized to try to treat dystonia, the leading treatment is botox injections and many patients are left with inadequate efficacy.
Preclinical data demonstrated that dipraglurant (ADX48621), a metabotropic glutamate receptor 5 (mGluR5) negative allosteric modulator (NAM), effectively reduced dystonia in a well-known preclinical model of Parkinson’s disease levodopa-induced dyskinesia (PD-LID). The neurophysiology of different forms of dystonia is thought to be similar - suggesting that dipraglurant may also work for non-parkinsonian forms of dystonia. Addex has completed preclinical development of dipraglurant-ER, extended release, which has an appropriate pharmacokinetic profile to be studied as a potential treatment for dystonia.
We are currently collaborating with the Dystonia Medical Research Foundation to design and prepare the development of dipraglurant in dystonia as well as initiating an investigator led Phase II POC trial to explore dipraglurant’s therapeutic potential in dystonic patients in collaboration with Professor Dirk Dressler of the Hanover medical school.