Earlier today, Ionis Pharmaceuticals announced the enrollment completion in its Phase 1/2a randomized, placebo-controlled, dose escalation study of IONIS-HTTRs in Huntington’s Disease (HD) patients.
IONIS-HTTRs is an antisense drug, and the first therapy designed to directly target the cause of the disease by reducing production of the responsible huntingtin protein to enter clinical development. It was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) in January 2016.
HD is an often fatal, rare genetic neurological disease in which patients experience depreciation of both physical control and mental abilities. Symptoms typically start to show between the ages of 30 and 50, and worsen over a 10-to-25-year period until eventual death.
Dosing in the final patient cohort continues, and Ionis intends to report top-line results from the study in the final quarter of 2017.
"We are encouraged by the safety profile of IONIS-HTTRx we have observed to date in the completed dosing cohorts in the Phase 1/2a study,” said C. Frank Bennett, Ph.D., senior vice president of research at Ionis Pharmaceuticals. “Upon completion and full analysis of this study, the next step for this program will be to conduct a study to investigate whether decreasing mutant huntingtin protein with IONIS-HTTRx can slow the progression of this terrible disease.”
Per a press release
, the safety and tolerability profile of IONIS-HTTRx in the concluded cohorts of the Phase 1/2a study backs its continued progression. Patients who finish the Phase 1/2a study will be eligible to participate in an open-label extension study that Ionis intends to start within the next year.
Roche, Ionis' partner for this drug, continues to advance and support the program, and has the option to license IONIS-HTT through the completion of the Phase 1/2a study.
"We believe that IONIS-HTTRx, which is designed to reduce the production of all forms of the huntingtin (HTT) protein – the known cause of HD, represents the most promising opportunity to address this significant unmet medical need,” said Bennett.
“Together with our partners at Roche, we are committed to developing IONIS-HTTRx, which has the potential to transform the treatment of HD."
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