The 12th Invest in ME Research Conference June, 2017, Part 2
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Hemispherx and Emerge Health to Commence Named Patient Sales While Seeking Ampligen Approval

Discussion in 'General ME/CFS News' started by JohnnyD, Mar 9, 2015.

  1. JohnnyD

    JohnnyD Senior Member

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    Hemispherx Enters a Collaboration with Emerge Health for the Commercialization of Ampligen for Chronic Fatigue Syndrome (CFS) in Australia and New Zealand


    …Hemispherx and Emerge Health to Commence Named Patient Sales While Seeking Formal Regulatory Approval

    Philadelphia, PA, March 9, 2015: Hemispherx Biopharma, Inc. (NYSE MKT: HEB) (the "Company” or "Hemispherx”), reported today that it has executed an agreement with Emerge Health Pty Ltd. ("Emerge”) to seek approval of Ampligen, an experimental immunotherapeutic, for Chronic Fatigue Syndrome in Australia and New Zealand and to commence distribution of Ampligen in both countries on a named-patient basis, where deemed appropriate.

    Hemispherx and Emerge will collaborate on seeking regulatory approval from Australia’s Therapeutic Goods Administration ("TGA”) and New Zealand’s Medicines and Medical Devices Safety Authority ("Medsafe”).
    Under an exclusive license to sell, market, and distribute Ampligen in Australia and New Zealand to treat Chronic Fatigue Syndrome, Emerge will implement regulatory-compliant programs to educate physicians about Ampligen for CFS and seek orphan drug designation and approval of Ampligen to treat CFS. Hemispherx will support these efforts and will supply Ampligen at a predetermined transfer price.

    Chris Rossidis, Co-Founder and COO of Emerge, said "This agreement with Hemispherx represents an opportunity which is exactly why Emerge was formed, to provide Australian and New Zealand patients with products that their physicians believe would be helpful. Given that there is no product approved specifically for the treatment of Chronic Fatigue Syndrome anywhere in the world, and at estimated prevalence rates of CFS in Australia and New Zealand, there could be 50,000 to 150,000 CFS patients with no dedicated treatment of this debilitating disease. We hope to be able to start providing Ampligen to the most severe of these patients during this year.”

    Recently, a blue-ribbon committee of the Institute of Medicine (IOM) of the National Academy of Sciences (USA) issued a comprehensive report on CFS reaffirming the seriousness of the disease and the need to accelerate research on potential treatments.

    Thomas K. Equels, Executive Vice Chairman and CFO of Hemispherx said "We are very pleased to be collaborating with Emerge to provide Ampligen under these unique access programs and to work with them to gain formal regulatory approval. While continuing our efforts to gain approval in the US, we are seeking similar named-patient programs and approvals elsewhere.”
     
    Justin30, zzz, heapsreal and 6 others like this.
  2. Iquitos

    Iquitos Senior Member

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    Thanks Johnny. Do you know what a "named patient" is?

    It will be interesting to find out what the "predetermined transfer" price is.
     
    oceiv likes this.
  3. Jonathan Edwards

    Jonathan Edwards "Gibberish"

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    'Named patient basis' is a standard set up for limited supply of a drug without general approval. It simply means that the drug can only be supplied to patients whose names are recorded so that any subsequent investigation into safety or other issues can track down all those who received the drug. In different situations there tend to be different stipulations about which patients qualify for inclusion - on grounds of severe disease or unusual circumstances etc.
     
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  4. JohnnyD

    JohnnyD Senior Member

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    NK17 and Iquitos like this.
  5. Jonathan Edwards

    Jonathan Edwards "Gibberish"

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    It is not absolutely clear from the quote posted whether or not they actually have approval to sell on a named patient basis. I do not know the antipodean regulatory laws on this sort of thing. New Zealand has a reputation for being even more conservative than the UK so I will be interested to see what happens.
     
    aimossy and NK17 like this.
  6. JohnnyD

    JohnnyD Senior Member

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    Also, HEB filed a 'Prospectus Supplemental' on Friday - which is a SEC document describing an overview of their operation. They describe how they get proceeds and what those proceeds are used for. Here is the Used for part and I'll bold a very interesting and new part of it.

    USE OF PROCEEDS

    We plan to use the net proceeds from the offering towards research and development, operations and general and administrative purposes related to the commercialization of Ampligen® and Alferon® related products, including, but not limited to, the following: (1) Costs to finalize the upgrade of the Alferon N Injection® manufacturing facility and to prepare for the FDA pre-approval inspections of the Ampligen® facility, (2) Manufacture of commercial product, (3) Potential new preclinical and/or clinical studies in order to gain commercial approval for Ampligen® and broader approvals for Alferon® and Alferon LDO®, (4) Working capital to build and maintain sufficient inventory by procuring raw materials, supplies and other items for the New Brunswick manufacturing facility, as well as to remunerate outside contractors for necessary services, such as, final filling and finishing operations in order to meet any anticipated demand from normal operations as well as through the possible pursuit of other disease areas and/or geographic regions that may present themselves, (5) Pursuit of potential partnering opportunities for Ampligen®, (6) Potential establishment of sales and marketing capabilities, as well as consideration towards the expansion of our manufacturing capacity, and (7) working capital for general and administrative expenses.

    Hard to imagine this meaning anything else but that the new revised protocol for the AMP511 trial and NK study is going very well. to put it mildly.
     
    NK17 likes this.
  7. Iquitos

    Iquitos Senior Member

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    Thanks Johnny & Dr. Edwards.

    Another question: Dr. Mikovits said that Ampligen responders have antibodies to SFFV (spleen focus-forming virus.) Any info or thoughts about that? Would it benefit patients to have that antibody test, IF there is one available outside research situations?
     
  8. JohnnyD

    JohnnyD Senior Member

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    The deal Hemispherx is working with the FDA now involves NK cell function. They are trying to establish a surrogate endpoint or biomarker. You can read the protocol change (In November) in the AMP511 trial on clinicaltrials.gov. And other recent Hemispherx PR's concern CFS/ME. Basically this is an open label trial with 3 arms, of 100 each. 100 ME patients that are receiving ampligen, 100 ME patients that are not (controls that are sick) and 100 healthy controls. They can then correlate ampligen->NK cell function improvement->Physical improvement. This is a basis for establishing a sub-group. I imagine that if FDA approves the drug, it will be a conditional approval under FDASIA, probably a phase 4.

    The FDA's original requirement was a double blind placebo controlled trial of 600. this is now an open label trial of 100. The FDA is obviously interested in this drug for ME as that protocol change along with the consideration of a surrogate bio-marker will save years of another phase 3 trial and 10's of millions of dollars. This kind of change really highlights the changes that are happening in government with regard to the FDA - that of accelerating drug development for 'rare' diseases that have no approved treatments.
     
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  9. CFS_for_19_years

    CFS_for_19_years Hoarder of biscuits

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    I was looking to see if someone here on PR was going to post this story. I think the topic would get more attention if the word "Ampligen" could fit into the long title. It is possible to edit the title, but if you're unsure of how to do it perhaps a moderator can help.

    In any case, thanks for posting the story. Ampligen has been in the works for at least 25 years and I'm glad more patients will have access.
     
    Sasha likes this.
  10. JohnnyD

    JohnnyD Senior Member

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    Good idea, and done.

    The PR made Yahoo's home page news feed for awhile as well.
     
    CFS_for_19_years likes this.
  11. medfeb

    medfeb Senior Member

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    JohnnyD

    Thank you for posting. This is great

    Can I ask a question - I looked for the info on NK Cell in the clinical trials record but didn't see it. Did I miss it or is that somewhere else?
     
  12. heapsreal

    heapsreal iherb 10% discount code OPA989,

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    Is the cost payed by the patient or is it subsidized by the pharmaceutical benefits scheme in Australia as well as NZ equivalent?
     
  13. aimossy

    aimossy Senior Member

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    I have only just come across this as the article was posted by the NZ org. It really will be interesting to see what happens with this here. We can be more conservative in some ways, and other times we just go for it. Hope they have the balance right. I'm personally pleased it's for certain cases and involved with looking into the drugs effects. Do you think this is careful enough Jonathan?!
     
  14. JohnnyD

    JohnnyD Senior Member

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    The company is being quite low key about this (I've got a couple of opinions why).. so you have to put 2 + 2 together. The amp511 protocol, the only CFS/ME clinical that Hemispherx has going, was changed in November. The only other 'announcement' of that change was in a February 23rd company PR about NK cell function. It is a long PR, worth reading. I'll quote the relevant sentence which appears at the end:

    "Clinical testing is presently underway to determine whether or not NK augmentation by Ampligen(R), to the extent it may occur in vivo, is associated with decreased CFS disease severity and, specifically, increased physical performance."

    http://finance.yahoo.com/news/hemispherx-biopharma-institute-medicine-health-133000450.html

    If you know any patients under Peterson, you can cross verify, NK function is Peterson's baby. He is the clinician with the most experience with ampligen, by far:

    "Dr. Peterson distinguishes what he believes to be an archetypal ME/CFS patient from the others primarily by using one test – the natural killer (NK) cell functioning test. The International Consensus Criteria for ME considers post-exertional neuroimmune exhaustion or PENE to be the hallmark of ME and Dr. Hyde focuses on SPECT scans, but Peterson has found the NK cell functioning test to be the most definitive."

    http://phoenixrising.me/archives/6103
     
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  15. JohnnyD

    JohnnyD Senior Member

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    I don't know how Australia and NZ government regulations work. Still early to ask that question, IMO. Treat this new information as hopeful and promising and we'll see how it shakes out.
     
    aimossy likes this.
  16. aimossy

    aimossy Senior Member

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    Less chance of getting sued in NZ and Aus if things dont go well. They tried eftpos here first too in case anything dodgy happened I think. Experiment on the antipodean's first maybe.
     
  17. heapsreal

    heapsreal iherb 10% discount code OPA989,

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    I cant see the australian PBS paying for a treatment so expensive like this unless one was going to die.

    I think it will be used by the rich who can afford it, but thats a foot in the door for potentially getting it approved wider and maybe bringing costs down.

    Also i think it will be good to stimulate interest in developing other treatments for cfs/me, i hope.

    It would be nice if the PBS would atleast cover my antivirals which would be a nice saving.
     
  18. medfeb

    medfeb Senior Member

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    Thanks for this, JohnnyD. This angle of NK Cell as a biomarker for disease severity was a conclusion in the IOM report also.

    Has the paper referenced in the above link been released yet? I don't think I have seen it.
     
    Bob likes this.
  19. JohnnyD

    JohnnyD Senior Member

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    No, the paper has not been published yet.
     
  20. M Paine

    M Paine Senior Member

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    Anyone in NZ been prescribed Ampligen yet? I'm booked in to see an immunologist in a couple of weeks, I intended asking about Ampligen and came across this. I'll be sure to report back on what I learn.
     
    daisybell likes this.

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