Ionis Completes Patient Enrollment in Phase 1/2a Trial of Ionis-HTTRx for Huntington’s Disease

Ionis Completes Patient Enrollment in Phase 1/2a Trial of Ionis-HTTRx for Huntington’s Disease

Ionis Pharmaceuticals has completed enrollment of the Phase 1/2a study evaluating the safety, tolerability, properties and movement within the body of Ionis-HTTRx in patients with Huntington’s disease (HD).

Patients who complete the randomized, placebo-controlled, dose escalation study (NCT02519036) will be eligible to enroll in an open-label extension trial that should begin in the next few months. Top-line results are expected by year’s end.

Ionis-HTTRx targets the root cause of HD by reducing production of the toxic mutant huntingtin protein (mHTT) from the mutated huntingtin gene. This antisense drug offers the possibility to treat all patients with HD, regardless of their individual HTT mutation.

The candidate therapy results from a strategic collaboration between Ionis and Roche to develop antisense drugs to treat HD. The alliance combines Ionis’ antisense expertise with Roche’s knowledge in clinical development of anti-neurodegenerative therapeutics.

“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,” C. Frank Bennett, senior vice president of research at Ionis, said in a press release. “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.”

He added that “Ionis-HTTRx represents the most promising opportunity to address this significant unmet medical need.”

Ionis-HTTRx received orphan drug designation from the U.S. Food and Drug Administration (FDA) in January 2016. That same month, it also won similar status from the European Medicines Agency.

The development of Ionis-HTTRx has been followed with anticipation. The drug candidate was one of the highlights at the Hereditary Disease Foundation‘s 10th biennial “Milton Wexler Celebration of Life” Symposium last year in Cambridge, Massachusetts.

HD is a rare genetic, progressive, neurodegenerative disease that results in deteriorating mental abilities and physical control. Some 30,000 Americans are symptomatic HD patients, and more than 200,000 are at risk of inheriting HD.



  1. Sylvia Cordis says:

    My name is Sylvia Cordis & I was wondering if there’s a trail for Ionis – Httx in the U.K. as my husband Chris suffers with HD himself & he’ll be extremely interested in putting him self forward for any upcoming trails in the future.

    I remain yours sincerely

    Mrs S M Cordis

  2. Bowen Briles says:

    My son was diagnosed with huntingtons August a 2015. Is there any way that I can get him enrolled in this study. If so please send some contact information. Thank you and have a blessed day

  3. Lacritia Duhart says:

    My niece(24)and nephew(18) are taking care of there mom, who has HD. How can we signed up my sister up for the trail or how can we get the medicine?

  4. David GIDET says:

    My French business partner’s wife has HD (her ailing mum also). They have 6 children so having more information about a possible treatment is extremely relevant to them all. I’m very close to the family & a day to day witness to the far reaching side effects of this awful ilness. I was therefore wondering if there’s the prospect of any trials here in France? Please could you send me any possible contacts that might be able to help or give advice/information so that I can pass them on, thanks!

  5. Melissa says:

    What is the process for getting enrolled in the clinical trials? Could you please provide information on the roche website? Families are desperate for this information. Kind regards melissa

  6. Erin Timoch says:

    My husband and 4 of his siblings suffer from Huntington’s. All are in different stages of the disease. Would love to get my husband in a trial study. He is 43 years old and is having more of mental and psychological symptoms of the disease more than the motor problems.

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