Roche Licenses Promising Huntington’s Therapy IONIS-HTTRx, Begins Extension Study

Roche Licenses Promising Huntington’s Therapy IONIS-HTTRx, Begins Extension Study

Roche will now be responsible for the development and marketing of Ionis PharmaceuticalsIONIS-HTTRx after exercising its option to license the investigational drug, designed to treat Huntington’s disease (HD).

This move follows highly promising results from a Phase 1/2a clinical trial.

The randomized, double-blind, placebo-controlled trial (NCT02519036) tested the safety and tolerability of several increasing doses of IONIS-HTTRx in Huntington’s disease patients. Pharmacokinetics (the movement of a drug within the body) and pharmacodynamics (the effect and mode of action of a drug) of IONIS-HTTRx were also evaluated.

The drug was found to have an acceptable safety and tolerability profile. It also reduced the amounts of the mutant huntingtin protein (mHTT) that causes Huntington’s disease in the patients tested. A trial with a larger number of participants is needed to assess the effect of IONIS-HTTRx on disease progression.

Ionis and Roche were just initiating an open-label extension of the study (NCT03342053) for participants who completed the Phase 1/2a trial. The two companies will transition the extension study to Roche, which will manage this trial and future studies of IONIS-HTTRx.

The results of the trial will be presented at medical conferences in 2018. There are also plans to publish a scientific article about the trial results.

“We are encouraged by the performance of IONIS-HTTRx in the Phase 1/2a clinical study. The dose-dependent reductions of mHTT we observed in the study substantially exceeded our expectations and we were equally encouraged by the safety profile of the drug,” Dr. C. Frank Bennett, senior vice president of research at Ionis Pharmaceuticals, said in a press release.

“We are grateful to the patients and investigators participating in this study. We could not have reached this important milestone without their commitment and that of the broader HD community.”

B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals, said the company is “extremely pleased that to have reached this important milestone in our collaboration with Roche, to discover and develop a therapy for people with Huntington’s disease.”

“This is our second antisense drug targeting a neurodegenerative disease to demonstrate a positive impact on a disease target in the CNS,” Parshall added.

Dr. Sarah Tabrizi, professor of clinical neurology, director of the University College London’s Huntington Centre, and the global lead investigator of the Phase 1/2a study, called the results of the trial “groundbreaking” for HD patients and families.

“For the first time, a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well tolerated,” she said. “The key now is to move quickly to a larger trial to test whether IONIS-HTTRx slows disease progression.”

IONIS-HTTRx has been awarded orphan drug status, meaning that incentives for developing the drug are in place, by the U.S. Food and Drug Administration and the European Medicines Agency as a treatment for Huntington’s patients.

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13 comments

  1. Nick Mauro says:

    This news made me cry tears of joy.

    Please please please inform me how to get my niece into the next clinical trial. She is approaching advanced stages of the disease and needs help.

    Please please please.

    Thank you so much.

    • It was 1983 that my late husband was diagnosed. He died in 1961. My eldest son is in stage 4 and my youngest is still in early stages. My eldest is 57 and the youngest is 43. I don’t hold out any hope for the eldest but wonder if the new trials will be ready in the near future.

  2. Dr Akila Ramanathan says:

    We are in India my husband is in mid stage how he can be participated in the clinical trials will anyoneed guide me thank you
    Dr Akila

  3. Sylvia says:

    This is such good news. We live in New York State and my brother in law was diagosed a year ago, but we suspected he has the disease for at least a few years. Time is of the essence, how can we get him on the next larger clinical trial. We were told the medication would probably be available at the end of 2018. We can’t wait that long. He is quickly deteriorating. A father of 5 young children. The youngest is only one years old, the older children are boys ages 11 and 12. They know what is happening with their dad. They know they have a chance of getting the disease. Everytime I see my brother in law or those beautiful babies, tears come to my eyes. The pain they are going through and the thought of losing their dad to such a devastating disease breaks my heart. I would give my life to take their pain away. Someone please, please help we are at a stand still and dont know what else to do.my sister has been running in circles, crying herself to sleep every night. If anyone can direct us or have more information,it would be greatly and sincerely appreciated.

  4. Ronald &Pamela Aldsworth says:

    This great news,WE would very much like to get my wife in a trial with this medication it sounds very promising. She was diagnosed about 9 months ago her brother has had the disease for 8-10 years and and not doing all that well with it so this would be very important to us thank you

  5. Maxine says:

    While we await news of how to get my husband and daughter into the trial, I wonder does the statement ‘option to license’ mean that it will be available for purchase soon???

  6. Ken Exsterstein says:

    This is absolutely promising. My father of 77 years of age is battling this disease for years. With the limited amount time left for him, please guide us on how he can also partake on any additional clinical studies. We eagerly look forward to your positive reply.

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