Ampligen - Hemispherx Biopharma re ME/CFS

"PHILADELPHIA, Nov. 15, 2017 (GLOBE NEWSWIRE) -- Hemispherx Biopharma (NYSE American:HEB) announced today its financial results for the third quarter ended September 30, 2017."

"We have secured our first country approval for Ampligen® in ME/CFS and we expect several more in the coming years."

"Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the clinical development of new drug entities for treatment of seriously debilitating disorders. Hemispherx's flagship products include Alferon N Injection® and the experimental therapeutic rintatolimod (tradenames Ampligen® or Rintamod®). Rintatolimod is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system, including Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS)."

http://ir.hemispherx.net/profiles/i...ryID=86477&BzID=2265&g=980&Nav=0&LangID=1&s=0

eta: see also http://scr.zacks.com/News/Press-Rel...ause-A-Turnaround-For-Hemispherx/default.aspx

 

"The new management team has achieved several recent milestones including:

1. The first shipment of Ampligen delivered for sale in Europe utilizing the Early Access Program (EAP)

2. EAP in Europe extended to pancreatic cancer patients beginning in the Netherlands

3. FDA approval to increase price for US-based Ampligen cost recovery program

4. Initiated a commercial scale production plan with our primary contract manufacturer to meet anticipated future demands in both international programs as well as domestic programs

5. Collaboration with Millions Missing Canada to bring medication to Canadians for ME/CFS

6. Completed an intranasal human safety study of Ampligen plus FluMist® and has commenced full data analysis."

http://livetrades.tv/hemispherx-heb...-multi-billion-dollar-immuno-oncology-market/

 

Update:
"
Some of the key developments in our Ampligen program have included regulatory approval in one of South America's largest markets, a stepped-up collaboration to support a regulatory submission for ME/CFS in Canada, positive results from an intranasal safety study, and several new initiatives to leverage the immunotherapeutic properties of Ampligen in state-of-the-art cancer treatment strategies.

Key Advancements For Ampligen

Ampligen has been approved in Argentina for severely debilitated Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) patients and we are pursuing similar approvals in Chile and Peru. We are collaborating with Millions Missing Canada to bring Ampligen to Canadians suffering from ME/CFS. In addition, we are in continuing discussions with the FDA regarding our New Drug Application in ME/CFS. Ampligen is the only drug to have completed a Phase 3 clinical trial in the U.S. in ME/CFS, a condition which according to the Center for Disease Control (CDC) afflicts more than one million people in this country. Taking into account that trial's results, and the FDA's indication at our last meeting that they would be open and receptive to review a proposal by the Sponsor (Hemispherx), we plan to seek approval tied to a protocol we are developing for conducting a study, limiting enrollment to patients with high responder potential."

http://www.nasdaq.com/press-release...ogress-outlines-key-objectives-20171219-00452

 

It seems like good news. Real world patients experience.

 

From your resident Hemispherx grouch:

The new CEO and his team have been in place for 18 months. In the linked video, at 12 1/2 minutes in, the CEO talks about the amazing changes that have occurred in that time. Along with those listed above, note the one near the bottom of the list:

"Continuing discussions with the FDA to identify path toward approval for ME/CFS"

I'm sorry, but what a load of crap. They KNOW what that path is. The FDA made it very clear five years ago when they asked for one additional clinical trial. That's the path. It's been identified, and they've done nothing about it.

Also note about thirty seconds later, as he continues to brag about Hemispherx's accomplishments, at 13:06 minutes in he says:

"We've got the FDA, uh, who has allowed for many years, Ampligen to be used in clinics, under a compassionate care-type program, to increase the price, uh, for Ampligen, from $75 dollars to $200 dollars a vial."

Great. That's compassionate...for the stockholders.

But even that hasn't worked, as during the last 18 months, the stock price has plummeted from $1.46/share to 35 cents.

Sorry to be a danny-downer.

 

Of course they know. However, Hemispherx is a tiny biotech. They don't have the money (it will take 20+ million) for another trial and have been unsuccessful in finding a partner. What would you propose they do?

The stockholders have not made money. Hemispherx has subsidized compassionate care for over 20 years, and if approval is not on the horizon, they need to charge what the drug costs to make and then some, in order to stay afloat - which they are barely doing. MyTomorrows has signed up/approved 50 pancreatic cancer patients in Europe to receive ampligen. Paid entirely by the government. Imagine that? They are currently working on expanding the program to other countries (other than Norway). If they don't have the money for an additional trial, cannot find a partner, cannot get any help from the FDA for ME (it really should have been approved in 2012). As a business, they are obligated to shareholders to find those who want to do business with them.

I think you've got the wrong bogey-man Danny.

 

Hemispherx has been around for 30+ years. If their drug(s) are so amazing and have so much potential, why in the world are they still a 'tiny biotech'. Could it be because the former CEO manipulated the stock price for his own gain over and over and over again (which is why he was fired), or is it just that the drug isn't that effective. If the drug was more effective they'd have no problem raising $20 million or finding a partner. In fact, they probably would've been bought out long ago.

And of course stockholders have made money. Not long term holders, but short term, they have. Long term it's been a disaster, for years and years. Why is it that after all these years the stock has never done well for long term shareholders (including the 'amazing' last 18 months, and how would you propose they change that?

And do you work for Hemispherx Johnny?

 

Because 30 years ago they decided not to pursue cancer, but to put all their resources in ME/CFS. I don't have to remind you that until recently no one believed that ME/CFS was a real disease.

If the disease had a biomarker and a well defined cohort they'd have no problem raising $20 million or finding a partner. In fact, they probably would've been bought out long ago. Fixed that for you. Know of ANY other company developing a new drug for ME? The FDA had an entire workshop devoted to attracting drug companies. They got zip.

I'm an investor who got interested in the company because the drug helped my sister.

And you? Do you work for the Hedge Fund that has been shorting the stock and spreading lies about the company?

 

With all due respect, I'm not sure what you're trying to suggest -- that if only they had had biomarkers their results would've been better and that would've attracted a partner? They've been (almost) exaggerating the drug's effectiveness for years without bringing up this biomarker issue...

Anyway, I tried googling to see what would turn up using the search terms 'Hemispherx', 'Ampligen' and 'cancer', and besides the usually press releases, I found this quote (but wasn't able to access the full article as I'm not a 'bizjournal' subscriber):

"Jun 15, 2017 - Dr. David R. Strayer, Hemispherx's chief scientific and medical officer, said about 25 years ago the company conducted some preliminary work testing Ampligen in renal cell carcinoma and melanoma, which were then considered rare forms of cancer. “What we found was a lot of the cancers stabilized..."

The cancers stabilized and they decided not to pursue that avenue of potentially huge profits to instead focus on a disease that hardly anyone took seriously? It seems like common sense suggests it would've been wise to continue that work, as it could've helped finance their ME/CFS trials, doesn't it?

That's funny. Truly. Not only do I not work for a hedge fund, I haven't worked in 19 years as I've been disabled w/ME/CFS and am currently housebound living on $750/month in a section 8 apartment.

If anyone has spread lies about the company it's the former CEO, William Carter. I'm not the only patient who feels this way, and no doubt there are stockholders who felt this way as well, since he was forced out of the company he founded.

I'm sincerely glad to hear the drug benefited your sister. She's one of the lucky few, including Mary Schweitzer and what seems to be a handful of others, perhaps more.
I have a friend who traveled from Canada to North Carolina to give it a try and is getting mixed results -- it's worsened some things and stabilized others.

My main beef is with the company, their incredible mismanagement, especially that of the former owner who clearly manipulated the stock price for more than a decade while doing nothing for patients. And now, knowing that they just dropped any research that 'stabilized' patients with certain types of cancer...well, that doesn't exactly make me want to send 'em a Valentine.

 

Anyone know what the results of their earlier phase-3 trial were?

 

The FDA Advisory vote is here: https://www.drugs.com/nda/ampligen_121227.html

Complete response letter here: https://www.drugs.com/nda/ampligen_130205.html

All advisory material is online somewhere and is extensive, including video of the advisory committee, where 30 people, including doctors and clinicians, lined up to beg for approval... many patients traveled to Washington D.C. in wintry snowy conditions. It should have been approved.

 

It's pretty simple Danny, the FDA held a workshop trying to attract ANY biotech that would develop a drug for this disease. No takers. Here we have a devastating disease, the largest unmet medical need in the world and no company is interested, why would that be? Actually one company took a very big gamble, Hemispherx, and it's astounding to me that patients want to throw the company under the bus, when it has unquestionable helped a sub-group of ME patients. Astounding. BTW, it took them 4 years to get Argentina's equivalent of the FDA (AMAT) to approve amplígen for severe ME using essentially the same data the FDA rejected.

It now costs 2.5 billion to develop a new drug. It was closer to a billion 20 years ago. https://www.scientificamerican.com/article/cost-to-develop-new-pharmaceutical-drug-now-exceeds-2-5b/

LaMattina says, “The value of the Tufts study is that it helps the public understand that this is a high-risk, expensive, and long-term endeavor.” Imagine that.

Pawel Kalinski of the Roswell Cancer Institute in Buffalo NY has been doing cancer trials for colon, ovaries, peritoneal mesothelioma cancers since 2013 using amplígen. You can find the trials on clinicaltrials.gov. He plans on starting trials in breast, prostate and melanoma using amplígen. Kalinski is using amplígen as an adjuvant, the MyTomorrows pancreas treatment is mono therapy. The approval that MyTomorrows got to treat pancreatic cancer are from earlier cancer trials and is based on the Argentina approval of amplígen, which is similar approval process to the Europeans. The irony is, ME patients have paved the way for cancer trials. ME patients proved the drug was safe and well tolerated. You can read about MyTomorrows pancreatic treatment in the companies SEC filings.

That was a sarcastic response to you thinking I worked for Hemispherx. Which TiredSam got after us about.

The company estimates 25% on their data. Some clinicians like Dan Peterson estimate 70%. This is for severely affected. Logically it would also help those less severely affected.

That is your opinion, it is not based in fact. The company has certainly had problems and Carter was difficult and not a popular guy. I refer you back to the LaMattina quote above about the Tufts study.

I beg to differ, it is the FDA, CDC and NIH who have done nothing (until very recently) for patients.

 

i can't follow to well atm. how is it going with ireland and the drug? i thought that was being applied for.

 

now how can we get some case studies on argentinian patients for drs to look at. this drug def helps some. i remember reading the research when it was up a few years ago.

there was some test trial some inc mary perhaps were involved in, what happeed to that?

 

Excellent letter by Hemispherx CEO. Filed with the SEC

https://www.sec.gov/Archives/edgar/data/946644/000149315218000820/ex99-1.htm

January 22, 2018



Thomas K. Equels, chief executive officer of Hemispherx Biopharma would like to draw your attention to a compelling new film that aired January 8 on PBS and documents the failure of the medical establishment to treat Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) as a serious biological disorder, as compared to a mental disorder, despite overwhelming new evidence the disease is a severe and disabling immune system disorder. According to The Wall Street Journal, the film has made the Oscar Awards short list for best documentary. Unrest can also be viewed on Netflix or on PBS.


To Our Employees, Contractors, Partners and Stockholders:


In our efforts to share important new information about one of the primary disease targets for our drug Ampligen, we are providing a link (http://www.pbs.org/independentlens/videos/unrest/) to the recently released movie “Unrest”, a PBS broadcast by filmmaker Jennifer Brea, who turned the lens on herself and others suffering from severe ME/CFS to document the symptoms associated with ME/CFS, a debilitating disease suffered by more than a million Americans, according to the CDC. I am hereby instructing every Hemispherx employee to watch this film and I urge our affiliates and stockholders to do the same.

...
The company recently met with the agency and is attempting to negotiate terms and a protocol for the FDA required confirmatory trial in patients with severe ME/CFS. The recent failure of the Norwegian Rituximab Phase 3 study in ME/CFS underscores the challenge that this severely debilitating disease presents. The Rituximab randomized controlled trial enrolled 152 ME/CFS patients and involved a collaboration between five hospitals. No positive treatment effect was reported. This disheartening result follows the inability to show a convincing treatment effect of psychology based Cognitive Therapy during reanalysis of the PACE study. These setbacks of other potential therapies now leave Ampligen as the only approved therapy worldwide and the only late-stage candidate drug with a pending NDA for ME/CFS in the U.S.A.

 

Do you have a link for this? It would be very helpful if I could pass this information along.

 

The statistics used by Pharma to cite research and development costs of new drugs is rather muddied as a fair amount of research is government funded, yet the patents are held by private companies. Seems a bad return on taxpayer funds.

The next tiny violin goes to the fact that they spent ten times as much on advertising than research. The US and New Zealand are the only two countries that allow it and I don't think they are selling a lot of ads for Lyrica to Kiwis.

So the reason drug prices are high in the US is not only a law against Medicare from negotiating bulk pricing or re-importation from Canada who does, not only absurd profit margins demanded by executives and shareholders, but that some of the most expensive advertising campaigns are for you 'to ask your doctor about _____,' the evening news is basically paid for by the drugs you buy.

 

Here is a workshop announcement Link:
http://solvecfs.org/fda-drug-development-workshop-dates-announced/ (The Federal Register link no longer works)

And here is the industry guidance draft that the FDA came up with about a year later: (There are some transcripts as well
https://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm388568.pdf

FDA public Patient Focused Drug Development seminar, December 18, 2017. Includes some talk of MECFS
https://www.fda.gov/downloads/drugs/newsevents/ucm591861.pdf

From which:
Defining research objectives and designing studies for CFS, now called myalgic encephalomyelitis, or chronic disease syndrome, MECFS is still stifled by the multiple case definitions that have not been operationalized, lack of funding, absence of validated objective markers and lack of interest from the pharmaceutical industry.

 

Hemispherx Comments on $500+ Million Market Opportunity for Ampligen®, the Only Late Stage ME/CFS Candidate in the U.S.
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Dr. Daniel Peterson, a world renowned independent expert in the treatment of ME/CFS and in the clinical use of Ampligen® (or rintatolimod), states that it “… is a potentially useful drug for a subset of severely ill ME/CFS patients to improve quality of life and functional status. Based on extensive clinical experience and testing, rintatolimod has an excellent safety profile. In addition, there are clear outcome measures that can be used to assess the drug’s efficacy in planned confirmatory trials.”

https://globenewswire.com/news-rele...y-Late-Stage-ME-CFS-Candidate-in-the-U-S.html