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Old 03-08-18, 01:41 PM   #1
Ellie
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Default Ionis HTT Rx Update

Hi

This has been released this morning -

http://ir.ionispharma.cpom/news-rele...pean-medicines

Roche are clearly keen to get this drug on the market asap.

Ellie
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Old 06-08-18, 04:57 PM   #2
Allan
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Default Re: Ionis HTT Rx Update

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Hi Ellie

Your link doesn't work for me. However, I think these links give the same content for this report: IONIS-HTT Rx (RG6042) Granted PRIME Designation by the European Medicines Agency for the Treatment of People with Huntington's Disease

http://ir.ionispharma.com/news-releases/news-release-details/ionis-htt-rx-rg6042-granted-prime-designation-european-medicines

https://www.prnewswire.com/news-releases/ionis-htt-rx-rg6042-granted-prime-designation-by-the-european-medicines-agency-for-the-treatment-of-people-with-huntingtons-disease-300691421.html

http://ir.ionispharma.com/node/23971/pdf

ABOUT IONIS-HTTRx (RG6042)

IONIS-HTTRx (RG6042) is an antisense drug designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT, which is the driver of HD. IONIS-HTTRx (RG6042) offers a unique approach to treat all patients with HD, irrespective of their individual HTT mutation. Roche and Ionis are collaborating to develop antisense drugs to treat HD.

In a Phase 1/2 study, IONIS-HTTRx (RG6042) demonstrated a significant reduction in mHTT, which breaks down the nerve cells in the brain. The study demonstrated a mean 40% (up to 60%) reduction of the specific HD protein in the cerebrospinal fluid (CSF) of adult patients treated with IONIS-HTTRx (RG6042) for three months at the two highest doses. Furthermore, levels of mHTT measured in the CSF were still declining in the majority of treated patients (~70%) as of the last measurement in the study. IONIS-HTTRx (RG6042) was well tolerated in this study.

Roche plans to initiate a pivotal study to evaluate IONIS-HTTRx (RG6042) in a larger patient population to further characterize its safety and efficacy profile in adults with HD.

IONIS-HTTRx (RG6042) has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) and by the European Medicines Agency (EMA) for the treatment of patients with HD.


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Old 11-08-18, 09:26 AM   #3
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Default Re: Ionis HTT Rx Update

Thanks for that Allan
Having worked most of my life in the pharmaceutical industry I have watched potential HD treatments come and go but there seems to be a lot more going on behind the scenes of this one.
I believe that Phase 3 will begin enrolling towards the end of this year and will be hundreds rather than thousands of participants because they are keen to get this trial under way. I think it will run for around 18 months / 2 years and, if - a big if - it is meeting the end points, I think they will start rolling it out “off license” to HD sufferers. They do this a lot with new cancer drugs allowing patients to have the chance of a treatment where there are no other options.
Don’t forget that they are still monitoring those participants in the phase 1/2a trial who continued to receive this drug so that will be providing valuable information on the longer term use.
Once licensed then it will have to be authorised by NICE for provision by the NHS as they are obliged to fund any new drug for indications that currently have no effective treatment.
Obviously the license will need to specify the indications and it will be interesting to see how far down the HD progression line they feel it is going to be effective.

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Old 11-08-18, 04:31 PM   #4
Allan
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Default Re: Ionis HTT Rx Update

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This long awaited drug, when it eventually becomes available, won’t be offered to everyone with HD - not even all pre-symptomatic and early symptomatic hd-individuals. It looks like those in the middle to late stages are going to miss out. Also, it wouldn’t get into the brain if swallowed in pill form.

So the drug will be administered (possibly monthly at a regional hospital) by injection into the lower back, known as an intrathecal injection - a form of lumbar puncture - to deliver the drug into the cerebrospinal fluid. CSF is a clear liquid that surrounds the brain and spinal cord. Treatment by this method means that the drug is most likely to reach the cells of the brain that are affected by the mutant huntingtin protein.

It can’t, however, be certain that the mutant huntingtin will be suppressed, defeated or destroyed sufficiently in other important parts of the brain. HD, as we know, spreads through many brain circuits that connect to deeper brain structures that cause havoc to health and the eventual dysfunction of life support systems.

We can but hope …

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Old 13-08-18, 05:28 PM   #5
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Default Re: Ionis HTT Rx Update

We shall have to wait and see!
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Old 17-09-18, 04:40 PM   #6
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Default Ionis HTT Rx Update = RG6042

.
Hot off the press:

Update on RG6042 (formerly known as IONIS-HTTRx) Huntington’s disease global development programme: Two clinical studies to begin by end of 2018

... and twitterfeed:

Dateline: 16 September 2018
EHDN Vienna update via
HDBuzz‏ @HDBuzzFeed
  1. Asked about treating young people and juvenile HD patients, Scott Schobel says they are enthusiastic but urges caution because of the risk that the drug might act differently in developing brains. We don't want to make things worse for these kids and young people.

  2. Schobel: Roche has plans to address the challenge of delivering the drug (if it works). Devices are being considered to avoid the need for frequent lumbar punctures and we may need to think laterally e.g. infusion centres

  3. Schobel asked about pricing. Responds that Roche has a history of fair pricing and "balances head and heart". Also announces that Roche is planning sites in 15 countries including one in Latin America

    Other relevant research:

  4. Mike Panzara from WAVE says they and others are working hard on a measurement system to help figure out to what extent the wild-type versus mutant proteins are reduced. Obviously important for the WAVE program which aims to reduce mutant only.

  5. But we have every reason to assume that the wild-type protein was reduced by the same amount (40-60%)

  6. Question about lowering of the wild-type or normal protein. Sarah Tabrizi points out that there isn't a way to measure that protein on its own because it's just like the mutant protein but a little smaller.

  7. Mark Guttman from Toronto rightly points out that participants shouldn't expect to feel *better* after treatment - the aim is to slow progression compared to where they would otherwise have been 2 years later.

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Old 18-09-18, 06:31 PM   #7
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Default Re: Ionis HTT Rx Update

More news of the trial out today -

https://en.hdbuzz.net/263

660 patients over 2 years. 1 in 3 will receive the placebo. 1 in 3 will have monthly injections. 1 in 3 will have bi-monthly injections starting early 2019.
I still predict that - if the results are positive - this will be rolled out to more of the HD community at the end of the 2 years as an unlicensed drug.

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