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.

Janet Stewart is a life sciences writer and editor, who completed both PhD course work and oral examinations in the Department of Microbiology and Immunology at McGill University, and holds an M.Sc. in Virology and Immunology.
Janet Stewart is a life sciences writer and editor, who completed both PhD course work and oral examinations in the Department of Microbiology and Immunology at McGill University, and holds an M.Sc. in Virology and Immunology.


  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.

      • Helle Højfeldt says:

        Fantastic news, Are there anyone who can help tell how my husband can come in next try, his HD must be stopped as soon as possible, as long as he still works pretty much. We are willing to provide all efforts and opportunities.
        Contact: [email protected]

  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 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.

  7. I am right there with everyone else. I need to know
    where and when I can get the treatment for my daughter.
    Time is of the escense. I still have a fighting chance
    for my daughter to survive this horrifying disease but I need the treatment sooner than later. It’s been over 20 yrs. when I first learned about it and now my daughter is diagnosed with it. We are so close to a treatment. The HD community needs the treatment now as we live everyday as if it was a time bomb. We don’t know what tomorrow will bring caring for a love one with this disease. It’s hard being a caretaker and watching this disease take their life away. We need to move faster for these patients. Please contact me as soon as possible. (Time is not on our side!)

  8. Janet Sutton says:

    Oh please, please let me know how get both my son, 42 and daughter 41 into the next trials…..we are desperate for a slower progression….Thank you!!!

  9. Ghada Zayed says:

    Highly appreciate if you can advice me how to let my niece participate in the trial. Whom should i contact, ready to travel anywhere.

  10. Robert Ferraro says:

    This is great news! Please forward info for the next trial, my wife has been diagnosed, she is 30 and we have to small children.

  11. Rudy Avila (303-809-6472) says:

    I have personally witnessed HD destroy my family starting with my mother that lost her battle at age 65, a brother that took his life at 49, another brother that was diagnosed 2 years ago that I would pray that he be allowed to participate in this clinical trial. He seems to be battling HD much better than my mom and my two other brothers. My other baby brother who will be 56 in April is already in a nursing home, and losing his battle to HD at a much faster pace than my mom did. His twin brother, is showing signs of the on-set, but has not been tested, and not sure he wants to be? I’m the oldest of us 6 siblings, and then my sister. Both of us are HD negative, but out other siblings are HD positive. I was the president of the Rocky Mountain HDSA Chapter in Denver, CO for a few years and donated much of my time to help HD families. I’m so excited that our prayers have been answered with hope that my family and other HD families have a chance to live a normal happy life with the discovery of this drug. I am pleading that my siblings are placed in this clinical trial to see if this drug can truly reverse the progression of this disease, and if so, that we can extend the trial to my nieces and nephews that have been tested and are HD positive. This disease needs to be eradicated for ever from this world so no one has to experience what I have experienced in seeing my family succumb to this disease.

    I would love to offer my time assisting in coordinating this effort with my siblings and other families in Colorado, Texas, and anywhere I could help remotely. I plan to retire soon, and would have more time to donate to this effort.

    Rudy Avila

  12. Alecia says:

    My older brother has had the disease for about 10 years now. He is honestly one of the most kind and charismatic guys I have ever met and sacrificed so much to look after me as a child in our single parent home. He graduated with honors from High School in Texas and by his early twenties, was already a leader in Sales and Marketing. He is known for his sense of humor and love for music and art, which he still displays even in his altered physical state. He loves everyone and has no problem giving big hugs (and kisses haha), compliments and showing kindness even to the most reserved or aloof individuals. It doesn’t matter who you are, he will bring a smile to your face and lighten your day with his light-hearted nature. He is only in his early thirties and has never had the chance to visit any beaches or see his favorite basketball team play. He is now living in a nursing home full- time and constantly dealing with symptoms on a day to day basis. He recently has started to lose a great deal of weight.

    I would love to learn more about how he can get involved in this trial.

  13. Johann Kruger says:

    Good morning,
    I have a friend who has been diagnosed with Huntington’s disease. Is there any indication of when Ionis-HTT will be available for use , we live in South Africa.
    Can one participate in the trials?
    Kind regards
    Johann Kruger.

  14. Daniel Cazares says:

    Hello my name is Daniel Cazares, I have 2 brothers who suffer from this disease, how could I contact you and participate in the testing of your new IONIS-HTTRx drug?

  15. Gecko Gal says:

    Is anyone answering all of these replies? Has anyone learned the parameters and how to become involved in the trial?

  16. Laura Guerrero says:

    Hello, My whole family is at risk! My grandmother died at 64 due to Huntington’s disease. My uncle who is 47 is in an advanced stage. My aunt, 53y, found out she has gene, but has not developed the symptoms. Finally, my father, 52y, started to developed the symptoms 1 year ago. He lives Mexico and information on these new developments is unknown. How can He get involved in the larger trial? . My brothers and cousins, we are all in our 20’s – 30’s and we are quite worried for our parents and our future. Can my family participate in the trials??

  17. Helidiane Francisco de Almeida says:

    Greetings. I am 33 years old, lawyer, mother of 3 children. On 02/04/2018 I discovered that two of my children are carriers. They are currently 5 and 3 years old. The father discovered in 2017 that he is a bearer. The maternal grandmother discovered in 2016. I need help. I am willing to do anything and also to help in whatever it takes. I’m in search of the cure !!! BRAZIL – Itajaí – Santa Catarina – [email protected]

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