Date: 2015-07-21
Type of information: Initiation of the trial
phase: 1-2a
Announcement: initiation of the trial
Company: Isis Pharmaceuticals (USA - CA) Roche (Switzerland)
Product: ISIS-HTTRx
Action
mechanism: antisense oligonucleotide. ISIS-HTTRx is a Gen. 2.0+ antisense drug in development for the treatment of Huntington\'s disease. ISIS-HTTRx is designed to reduce the production of all forms of the huntingtin protein, which is the protein responsible for Huntington\'s disease. Huntington\'s disease is a rare genetic, progressive neurological disease resulting in deterioration in mental abilities and physical control. HD is referred to as a triplet repeat disorder, and is one of a large family of genetic diseases in which certain gene sequences are mistakenly repeated. In HD, the gene that encodes for the HTT protein contains a trinucleotide sequence that is repeated in the gene more than 36 times. The resulting HTT protein is toxic and gradually damages neurons in the brain. Symptoms of HD usually appear between the ages of 30 to 50 years, and continually worsen over a 10 to 25 year period. Ultimately, the weakened individual succumbs to pneumonia, heart failure or other complications. Presently, there is no effective disease modifying treatment, and current approaches only focus on managing the severity of some disease symptoms. ISIS-HTTRx has been granted orphan drug designation by the European Medicines Agency for the treatment of patients with HD. The drug is developed with Roche.
Disease: Huntington\'s disease
Therapeutic area: Genetic diseases - Neurodegenerative diseases - Rare diseases
Country:
Trial details:
Latest
news: * On July 21, 2015, Isis Pharmaceuticals announced that it has initiated a Phase 1/2a clinical study of ISIS-HTTRx in patients with Huntington\'s disease (HD). ISIS-HTTRx is the first therapy to enter clinical development that is designed to directly target the cause of the disease by reducing the production of the protein responsible for HD. Presently, there are no disease-modifying treatments for HD, with current therapies focused only on treating disease symptoms. The randomized, placebo-controlled, dose escalation Phase 1/2a clinical study will evaluate the safety and activity of ISIS-HTTRx in patients with early stage HD. In this study, ISIS-HTTRx will be administered intrathecally as an injection directly into the cerebral spinal fluid. Intrathecal administration of antisense drugs has been shown to be well tolerated in multiple clinical studies in patients.
