Ampligen approval granted extension by FDA

[Nielk]

FDA Grants Hemispherx Biopharma Extension in Its Pending New Drug Application (NDA) for the Treatment of Chronic Fatigue Syndrome (CFS)



PHILADELPHIA, Jan. 11, 2012 (GLOBE NEWSWIRE) -- Hemispherx Biopharma, Inc. (NYSE Amex:HEB) (the "Company" or "Hemispherx") announced today that the Food and Drug Administration ("FDA") granted an extension for the Company to modify its New Drug Application ("NDA") in response to a Complete Response Letter ("CRL") received November 25, 2009 for the Chronic Fatigue Syndrome ("CFS") therapeutic indication. The extension will remain open while Hemispherx submits an amended NDA.

In year 2011, a team of researchers at the Centers for Disease Control ("CDC") and Harvard University reported new data on the magnitude of medical and economic impact of untreated CFS (Cost Effectiveness and Resource Allocation, vol. 9, 2011). According to the study, "CFS patients, their families, employers and society bear significant costs associated with the illness. The symptoms characterizing CFS are common to many illnesses, hence diagnosis is complex... and requiring extensive diagnostic testing and clinical assessment."

In the request for extension, the Company advised the FDA of the findings presented at the IACFS/ME Biennial Scientific Conference held September 22-25, 2011 in Ottawa, Ontario, Canada of a new potential companion diagnostic tests for CFS. The proof of principle study presented in Ottawa compared sequences from massively parallel sequenced serum DNA (Deoxyribonucleic Acid) extracted from either well vetted Hemispherx CFS sera or matching controls. Signature DNA sequences were identified that showed statistically significant separation of CFS serum from matching control serum. "We continue to move forward and are working diligently to pursue approval for Ampligen(R) as the first treatment for Chronic Fatigue Syndrome in the U.S. and other countries," stated Dr. David Strayer, Medical Director for Hemispherx.

About Hemispherx Biopharma

Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the manufacture and clinical development of new drug entities for treatment of seriously debilitating disorders. Hemispherx's flagship products include Alferon N Injection(R) (FDA approved for a category of sexually transmitted diseases) and the experimental therapeutics Ampligen(R) and Alferon(R) LDO. Ampligen(R) is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system. Hemispherx's platform technology includes large and small agent components for potential treatment of various severely debilitating and life threatening diseases. Hemispherx has an extensive number of patents comprising its core intellectual property estate and a fully commercialized product (Alferon N Injection(R)). The Company wholly owns and exclusively operates a GMP certified manufacturing facility in the United States for commercial products. For more information please visit www.hemispherx.net.

Information contained in this news release, other than historical information, should be considered forward-looking and is subject to various risk factors and uncertainties. For instance, the strategies and operations of Hemispherx involve risk of competition, changing market conditions, change in laws and regulations affecting these industries and numerous other factors discussed in this release and in the Company's filings with the Securities and Exchange Commission. Any specifically referenced investigational drugs and associated technologies of the Company (including Ampligen(R) and Alferon(R) LDO) are experimental in nature and as such are not designated safe and effective by a regulatory authority for general use and are legally available only through clinical trials with the referenced disorders. The forward-looking statements represent the Company's judgment as of the date of this release. The Company disclaims, however, any intent or obligation to update these forward-looking statements. The planning, completion, results or submission of clinical trials do not imply that any study product will ever be approved commercially for the studied or other treatment indications.

 

[Firestormm]

Thanks Neilk. You know I haven't followed this Ampligen development story particularly closely - but what is going on? Is this another extension? You'd think it would be relatively 'easy' to put a drug through clinical trials. I mean the whole saga has certainly knocked it's share price for six. But there's probably more to it like I said - shall have to investigate methinks...

 

[Nielk]

What I find interesting is their claim that they have a test that can determine if one has CFS. Does anyone know what they mean?

 

[LaurelW]

Check out this thread where it is explained:

http://forums.phoenixrising.me/show...nt-Data-on-a-Novel-Diagnostic-Finance-Article

 

[undcvr]

What does this mean ?

 

[Tony Mach]

What I find interesting is their claim that they have a test that can determine if one has CFS. Does anyone know what they mean?

I think it is something they got from the company "Chronix". Last year, I think it was spring, I read something about a sort of "next generation sequencing" or some such. Much marketing talk, difficult to discern what they were up to. They worked together with the university of Gttingen. There was talk that they were looking for XMRV. Maybe they beat Lipkin and found something? But I don't think so.

As far as I know the company is in "economic difficult times" and I think they need to make publicity from time to time. I wait until they publish a proper research paper and we had time to pick it apart.

 

[Nielk]

Check out this thread where it is explained:

http://forums.phoenixrising.me/show...nt-Data-on-a-Novel-Diagnostic-Finance-Article

It's weird that we don't hear more about this. It sounds like they have isolated 4 genes that seperate CFS patients from controls?

A total of about 10,000 high quality sequence reads were generated from each serum sample and four genes were identified by Multivariate Regression that separated CFS patients from the normal control group with a c-value of 0.95. These results support additional studies with a larger CFS cohort using more powerful Massively Parallel Sequencing platforms with the aim of reduction to validated clinical assays for the diagnosis and evaluation of CFS and to explore whether the technology can be used to identify how different persons with CFS will respond to Hemispherx's experimental drug Ampligen(R).

If this test was user worthy, I'm sure we would know about it. Right?

 

[Tony Mach]

Thanks Neilk. You know I haven't followed this Ampligen development story particularly closely - but what is going on? Is this another extension? You'd think it would be relatively 'easy' to put a drug through clinical trials. I mean the whole saga has certainly knocked it's share price for six. But there's probably more to it like I said - shall have to investigate methinks...

In Osler's Web there is some info about how they started in the eighties. As far as I know, back then they had a chance to get Ampligen licensed by the FDA under some relaxed rules, but they choose not to for unknown reasons. They are definitely not a big pharm-company, so they can't put Millions into drug trials, but they managed to get the drug approval along in Canada so I don't really no what the hold up is. Maybe they are afraid that a Phase III? trial will not be as good as it should be? This whole thing is not satisfying. I wish them luck, as Ampligen seems to get some patients on their feet again.

 

[Tony Mach]

If this test was user worthy, I'm sure we would know about it. Right?

They had a small test with few patients and it looks like they are checking their results in a larger cohort. This can take some months.

Usually then you write up your results, submit them to a journal, get them reviewed, maybe add or change something and then it is published. Under normal circumstances this could be a couple of months. With CFS and after the Mikovits train wreck, I guess the journals will conduct their reviews very thorough and rather refuse something they find fishy. So you can add a couple of months if the researchers want to publish in a paper with impact

 

[Nielk]

They had a small test with few patients and it looks like they are checking their results in a larger cohort. This can take some months.

Usually then you write up your results, submit them to a journal, get them reviewed, maybe add or change something and then it is published. Under normal circumstances this could be a couple of months. With CFS and after the Mikovits train wreck, I guess the journals will conduct their reviews very thorough and rather refuse something they find fishy. So you can add a couple of months if the researchers want to publish in a paper with impact

Right - we have to wait and see but, if they do come up with it and it's viable - it will be HUGE!!!!

 

[heapsreal]

There was talk last year about an oral version of ampligen, not the interferon med they mention but an actual oral ampligen. Has anyone heard anything of this??

cheers!!!

 

[Firestormm]

11 January 2012: http://www.bizjournals.com/philadel.../01/hemispherx-granted-more-time-to-work.html

Those waiting and waiting and waiting for action on Ampligen, an experimental treatment for chronic fatigue syndrome under development by Hemispherx Biopharma since the 1970s are going to have to wait a little longer.

The Food and Drug Administration Wednesday granted the Philadelphia biotechnology company an extension to modify its new drug application for Ampligen.

In November 2009, the FDA rejected the application submitted by Hemispherx (NYSE Amex:HEB), stating the two primary clinical studies submitted by the company did not provide credible evidence of efficacy for Ampligen. The agency recommended the company conduct at least one additional large clinical study.

Hemispherx requested an extension on filing an amended new drug application noting researchers are working on potential companion diagnostic tests that could have a bearing on the companys application. Details on the tests were presented at a conference in Canada four months ago.

We continue to move forward and are working diligently to pursue approval for Ampligen as the first treatment for chronic fatigue syndrome in the U.S. and other countries, said David Strayer, the companys medical director.

The extension the FDA granted Hemispherx is open-ended, according to the company.

In 2010, Hemispherx received a $244,479 grant for research costs associated with its Ampligen development program through the federal governments Qualifying Therapeutic Discovery Project program, created to help support innovative drug development by smaller life sciences companies.

See more here: http://www.bizjournals.com/philadel...6/controversial-chronic-fatigue-drug-put.html

 

[Nielk]

Firestorm,

This last link is dated Nov. 2010.

The newer data which you high-lite first is the one that I started the thread with.
With akll the mysteries following Hemispherx, it's a wonder that they are still around and that the FDA gave av extension for Ampligen must mean that they are happy with some of the data otherwise this thing would be dead.

My husband, who is in Finanance and follows many biotech companies says in all his years, he has never seen a drug take so long to be approved by the FDA. It just doesn't happen. If they are not convinced that it has some value, they kill it and at a much earlier stage. The fact that Ampligen is still being studied is strange. It's definitely not because of money because the Hemispherx has no money so what's left is the fact that the FDA can't ignore the very positive results they see with some patients. If they can really come up with this testing (of the four isolated genes), they will be able to direct their efforts to the patients who do have these genes and show much more positive results. I really hope it happens because it's not like we have a line up of treatments for us just waiting to be approved.

 

[Firestormm]

I know I was simply reading previous reports and this one from November 2010 carried more information/speculation about the possible ramifications for Ampligen if the link to XMRV had been proven. You are correct of course it is the first bit - the one I pasted - that is most relevant to your thread.

Thanks for additional opinion though. This is my concern too. Whilst many biotech companies are speculative and attract speculative investors who 'hype' the claims - this one actually received FDA funding which gave the product at least some credibility in the eyes of investors.

My research - when I can get around to it - will try and find what their 'blood test' is all about in relation to CFS and why they are still taking so long to amass a large enough patient cohort to satisfy the FDA (I believe the FDA wanted 200 patients at least to take part or something?).

The main problem seems to be - and I have read very little about this to be honest - trying to prove that Ampligen actually does anything in CFS-affected people. The product - as you can see from that link - has been touted as a treatment for many other diseases but even they have not been proven.

You might recall but in the late 80s and 90s there was much interest in speculative investments such as this. I used to work in the 'industry' so to speak and several of our more err... gambler-type clients would insist on us taking a punt on just such ambitious projects.

I recall one actually - as an aside if you will permit me - that claimed it was able to invest a machine that could 'sniff' infections in human urine. I can't recall the specifics of course but at the time and amid all the other biotech excitement it actually won my boss over and he invested quite a lot of his own money. Indeed I seem to recall he became the major shareholder! Always wondered what happened to that company and his investment...

Sorry. That's not to say Hemispherex is anything like that of course (if indeed the 'sniffer' actually failed). And like you said, the main point is that Hemispherex are still in the running and receiving cash from the FDA. So one could say that there is indeed something to their claims - hence my search I guess for reasons behind this massive delay.

Though how many other companies are in a similar position - receiving paltry funding from the FDA - I couldn't possibly say really. Anyway, that wonderful resource Wikipedia has this to say:

'Hemispherx reports that it completed a Phase III clinical trial for CFS in 2004 and filed a new drug application (NDA) with the U.S. Food and Drug Administration (FDA) to market and sell rintatolimod for the treatment of CFS,[1] but this was rejected in December 2009 because the FDA concluded that the two RCTs "did not provide credible evidence of efficacy."[2][3]'

http://en.wikipedia.org/wiki/Ampligen

I mean how hard can it be? Seriously. Anyway, my research continues I suppose...

 

[Nielk]

The FDA did approve though a present ongoing open study. A few CFS doctors are conducting it. I know two people who suffer from CFS personally who are "in remission" while taking Ampligen. When they are off for a while, it comes back. I'm sure that there are many more beside these two.

 

[justy]

Chances of getting Ampligen in Europe?

 

[Tony Mach]

I took a look at Howard B. Urnovitz, PHD, senior manager of Chronix, as I did not have him on my radar before.

Oh my.

I will just say my quack alarm went off quite noticeable and I will leave it at that. I going not to take statements from Chronix at face value, not until they present evidence and it has been thoroughly examined a position I should have adopted anyway

 

[Tony Mach]

I recall one actually - as an aside if you will permit me - that claimed it was able to invest a machine that could 'sniff' infections in human urine.

Supposedly Howard B. Urnovitz has developed an test that can find HIV-antibodies in urine. Maybe he is just an unconventional mind, and I do him injustice. Still, I will not bet the farm (one way or the other), even if I had a farm

 

[Mark]

I took a look at Howard B. Urnovitz, PHD, senior manager of Chronix, as I did not have him on my radar before.

Oh my.

I will just say my quack alarm went off quite noticeable and I will leave it at that. I going not to take statements from Chronix at face value, not until they present evidence and it has been thoroughly examined a position I should have adopted anyway

You don't present any evidence yourself for this conclusion Tony, just an insult which you don't even attempt to justify. Would you care to present evidence for us to examine yourself, or do you expect us to take your own statements at face value?


Here's the profile I found through Google (with my emphasis):

Dr. Howard B. Urnovitz, PhD Co-Founded Chronix Biomedical Inc., in 1997 and serves as its Chief Executive Officer and Chief Science Officer. Dr. Urnovitz serves as Science Director of both the Chronic Illness Research Foundation and Pepgen Corporation. He founded Calypte Biomedical Corp. in 1988 and served as its Chief Science Officer from 1988 to 2001. He served as a Director of Molecular and Cellular Engineering at Xoma Corporation, a biotechnology corporation, from 1983 to 1985. He worked in biotechnology for over 2 decades, in the field of genomics and chronic disease. He was the first scientist to show that nucleic acids circulating in plasma and serum originate from specific host genomic sequences. Dr. Urnovitz served as Senior Scientist at the Institute of Cancer Research in San Francisco from 1985 to 1987. He served as Chairman of Chronix Biomedical Inc. and serves as its Director. He served as a Director of Calypte Biomedical Corp. from 1988 to 2001. He served as a Director of the Hybridoma Laboratory at the University of Iowa. Dr. Urnovitz received a B.S. in Microbiology and a Ph.D. in Microbiology and Immunology from the University of Michigan, and completed a post-doctoral fellowship in the Departments of Pathology at both Washington University (St. Louis) and University of Iowa.

I also found through Google a link where it seems that Urnovitz has supported (at least in the past) the theory that the first oral polio vaccines 'may have contained live simian immunodeficiency virus (SIV) or they were present environmentally as an opportunistic infection.', that 'in a certain number of African vaccine recipients, SIV recombined with their own normal genes to create a monkey-human hybrid now known as HIV-1' and that 'early "inactivated" Salk vaccines given to some 98 million Americans were also contaminated with monkey viruses and may be one reason why there has been an explosion of cancer, new infectious agents and other new immune and neurological disorders among the baby boomers born between 1941 and 1961'
http://ffitz.com/nyvic/health/monkey.htm

Perhaps it's his voicing of this theory that set off your quack alarm? Or do you have any other evidence against Dr Urnovitz to share with us?

 

[Evangelina]

Hemispherx is just keeping up with its paperwork. They needed an extension granted by the FDA to keep their AMP 511 going. As far as Chronix is concerned it is just my opinion that they are tryong to boost their investment capital. Hemispherx needs the AMP 511 patients so it can fund another double blind study. Without another double blind study Ampligen will never be approved. Which is too bad, because it really works.
This from an AMP 511 patient (Sophie Church) in her 7th month of Ampligen:

"On the other hand Ive waited a long time to announce with confidence that I can exercise again. Not a lot, but some. I can do 20 minutes of aerobics or a brisk 30 minute walk every day. Best of all I still have leftover energy to shop, to cook dinner, play with the dogs and even have friends over. After a full day I can expect to do less the next day, but I can still exercise and leave the house. This is truly a remarkable thing for me. It has been a long, hard road so far, but a road very much worth taking."


http://theglassmountain.wordpress.com/2012/01/12/ampligen-weeks-30-31-and-32/

I hope they eventually get it approved.

Evangelina