Rare Disease Report

IONIS-HTTrx Proves Effectiveness in Treating Cause of HD

DECEMBER 12, 2017
Mathew Shanley
Yesterday, it was announced that the first study of IONIS-HTTRx was completed. The Phase 1/2a evaluated the first therapy in clinical development intended to target the underlying cause of Huntington’s Disease (HD).

In the dose-escalation study, it was proven that the drug exhibited the ability to reduce levels of a mutation in the huntingtin protein (HTT). Participants enrolled were capable of continuing on the drug through an open-label extension, demonstrating the safety of the potential therapy.

HD, a neurodegenerative disease, is caused by an unstable cytosine-adenine-guanine (CAG) repeat expansion in the huntingtin gene, which can lead to the production of a mutant form of HTT. It is characterized by a combination of motor, cognitive and psychiatric problems, and current standard-of-care is limited to a management of symptoms. IONIS-HTTRx, if approved, would be the first treatment to prevent or attenuate disease progression.

“As the next phase of clinical study of IONIS-HTTRx gets underway, we look forward to continuing to partner with Roche and the many dedicated clinicians and scientists who work tirelessly to bring greater help and hope to Huntington’s families everywhere,” said Louise Vetter, President and CEO of the Huntington’s Disease Society of America (HDSA) in the organization’s official statement. “We know families will have many questions about what happens next. We always recommend you speak with your doctor about any medical questions you may have. HDTrialFinder.org and HDSA.org will continue to provide the timeliest information regarding development of meaningful treatments for HD.”

IONIS-HTTRx is an antisense drug intended to reduce the amount of huntingtin RNA in the brain. With less RNA “message” available, less huntingtin protein is made.

Following conclusion of the study, Roche has exercised its option to license IONIS-HTTRx and intends to continue development of the drug because of the safety and tolerability profile that was observed. Results from the study will be presented at medical conferences in the first half of 2018, and will be submitted for publication in a future issue of a peer-reviewed medical journal. Roche will now become independently responsible for the further clinical development of IONIS-HTTRx, including the next step for the program, which will be conducting global trials to demonstrate safety and efficacy.

In the video below, Vetter discusses the “clock on the wall” that is ever-present for HD patients, and why she thinks the science and research being discovered could bring hope to the HD community.

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