Ampligen approval granted extension by FDA

[Firestormm]

There was a further article posted by The Street on 2 October 2012 that I don't think I posted:

How to Trade Hemispherx Even If You Hate the Stock

Unlike Feuerstein, I'm a trader of biotech stocks. Hemispherx is a bad investment but the stock remains a good trading opportunity, particularly with an FDA advisory panel for Ampligen scheduled for Dec. 20.

Once upon a time, in a galaxy, now far far away, alas and alack, when little old me was engaged in an albeit 'small', but nonetheless professional way with the various stock markets of the world. This was exactly how 'we' (royal we there) treated such companies.

Of course there are those who think that in a small way and as part of one of the 'eggs in your basket' it's worth taking a longer term punt and locking such stock away somewhere, in the religious hope that something might come from their discoveries (though perhaps you are far more likely to end up with stock in a major Pharma purchaser should anything that looks really good turn up!).

Anyway, this article is looking at the stock in terms of trading and not investment per se but it does include some rather interesting (I think) summations of what exactly Hemispherx as a viable company with viable products should be seen as e.g.

1. Hemispherx is a cult stock, and like all cult stocks, there exists a group of retail investor fanatics who will defend the stock to their death. No counter-argument, no matter how reasonable or factually supported, will be considered. Negative stories about Hemispherx are reasons to buy more Hemispherx. Positive stories about Hemispherx are reasons to buy more Hemispherex. If a cult investor can't afford to buy more Hemispherx, he will work to convince others to buy more Hemispherx. As catalysts like FDA panels or approval decisions dates near, the Hemispherx cultists will become more bullish, his price targets more outlandish. For these reasons, the run-up in cult stocks is often exaggerated.

Of course the above is GREAT NEWS for traders who will exploit such things to the hilt. Also seems to be perhaps what Hemispherx themselves are now guilty of....

2. The same Hemispherx trade worked great in 2009, so no reason to expect history won't repeat itself this time around. In the middle of 2009, Hemispherx shares went on a run from 50 cents to more than $4.50 fueled by speculation and hype surrounding the FDA's original review of Ampligen. Good traders remember stocks that trade well -- Hemispherx included. I expect traders to be drooling over another chance at Hemispherx this time around.

And what's driving any 'faith' in Hemispherx? Obvious answer really:

3. There are no drugs approved specifically treat chronic fatigue syndrome. Whether or not you believe Ampligen works, Hemispherx will own the market if the drug is approved. That's a big opportunity for a company with a $100 million market value and another reason why bulls are so excited.

Traders take advantage of such optimism. [Bastards]

http://www.thestreet.com/story/11725280/1/how-to-trade-hemispherx-even-if-you-hate-the-stock.html

While I personally don't believe Ampligen will receive FDA approval and I'm not a fan of the company, I do see an excellent trading opportunity as we get closer to the FDA advisory panel.

Make note that Hemispherx's share price has already increased over 100% since late July, which could lessen the run-up trade opportunity.

And remember, Hemispherx is a trade, not an investment. Never fall in love with a small-cap biotech stock.

 

[Firestormm]

See what I mean? From $1.00 to 0.92 (when company bailed) to 72 cents now. Tsk tsk tsk

 

[perovyscus]

Why not do a retrospective analysis of an objective cognitive endpoint instead?

The only ethical explanation is the FDA would not prefer this.

 

[Firestormm]

RTT News: 26 November 2012: http://www.rttnews.com/2011750/can-...py-ending-to-long-fatiguing-wait.aspx?type=ts

Can Hemispherx's Ampligen See Happy Ending To Long Fatiguing Wait?

In less than a month, Hemispherx Biopharma Inc. (HEB: Quote) will face an FDA advisory panel that will review its lead compound Ampligen for the proposed treatment of chronic fatigue syndrome or CFS...

...Pacing the sidelines for nearly four decades, Ampligen has been a lightning rod for controversy. Will Ampligen be successful in passing regulatory muster at least this time? Stay tuned...

Shares of Hemispherx have thus far hit a 52-week low of $0.17 and a 52-week high of $1.10. The stock closed Friday's trading at $0.75, up 1.52% on a volume of over 551 thousand shares.

 

[Nielk]

Hi Firestormm,

I guess some who know how to play this short term trades can be making a lot of money with this volatile stock.

 

[Firestormm]

Here's some comment ahead of the imminent decision from someone claiming to be uniquely qualified:

...I am a professionally trained scientist with a formal background in multivariate statistics, clinical trials, and have worked closely with the FDA in the past. So I feel my view on Ampligen is unique in this regard, and should help to clarify some of the outstanding issues. Before launching into my view of Ampligen's prospects, however, I believe it is important to give brief introductions to the drug's biochemical mechanism, some insightful comments from patients, and its recent history with the FDA....

Am reading it now: 3 December 2012:http://seekingalpha.com/article/1040611-hemispherx-biopharma-a-voice-of-reason-amid-the-din

 

[maryb]

Be grateful for a brief summary once you've read it - cheeky I know

 

[Firestormm]

Will do. Not even sure of this chap's credentials yet. I'll get round to it

 

[Firestormm]

Be grateful for a brief summary once you've read it - cheeky I know

I have read it and it's worth persevering with - especially if you a statistical leaning as I know some of us here do

However, I don't really have the time to do more than post his conclusion - which is probably all is needed. Needless to say it is positive:

Reading of the Ampligen Tea Leaves
The FDA is under pressure to approve drugs for unmet medical needs, and chronic fatigue syndrome certainly meets that criteria.

Ampligen has been profoundly mismanaged by Hemispherx over the course of 30 years, leading to the mess we have today. That mismanagement, however, should not be held against the drug itself, because it has the potential to help so many victims of CFS.

So for the patients and investors alike that desperately want to see the FDA approve Ampligen, I have some good news. Having worked closely with the FDA and similar governmental agencies during my career, I can assure you of one thing: the FDA did not do an about-face if it didn't seriously intend to consider approving the application. Why it did so is still a mystery, but this event did happen.

Even though the re-analysis of old clinical trial data is certainly not optimal, I see no mathematical or logical reason to disregard the analysis wholesale. The patient testimonials, combined with the new analysis, tell my lying eyes that Ampligen is indeed a "safe and effective" treatment for CFS.

In conclusion, I believe the FDA will agree with this view for the most part, and finally approve Ampligen as a treatment for CFS. For investors, I believe this will mean that the share price of HEB will rise several fold in short order. How high is anyone's guess, but it's going to be a "memorable investment" in my opinion.

Good luck to all those personally or financially invested in an FDA approval of Ampligen. It's been a long time coming.

I'll perhaps write and comment more after I've had a decent rest

 

[maryb]

Thanks F

 

[Firestormm]

Thanks F

No worries. His is only an opinion don't forget. Unfortunately

 

[Marlène]

Market traders manipulate the news with this kind of news. They are in a business and they want to make money just like any stockholder.

So what they will do before D-day is accumulating buy and sell orders (the market goes up and down a bit) and when the time is right, they will release all their orders (buy or sell).

That's how they make money. They don't care if Ampligen makes it or not.

 

[Firestormm]

More comment before D-Day. If you can tolerate the volume:

9 December 2012

Uncertainty in near term regulatory outcome

Typically, to approve a drug, the FDA requires that an NDA include two large well designed randomized control trials, each achieving a level of significance of p<0.05 on the primary endpoint (treatment vs. control).

There have been exceptions to that rule, particularly in the field of oncology, with very rare diseases or with dramatically effective treatments in areas of therapeutic need.

The FDA's preference, if only one study is the basis for an NDA, is that the study be well designed and very compelling (e.g., p<0.01 favoring the treatment arm, to reduce the risk of Type I error, i.e., false positive outcome).

In this regard, the p value reported in the Phase 3 publication is only marginally significant at p=0.047. It should be noted that since CFS is relatively common, the only limitation to Hemispherx performing a second larger well-controlled study, which the Agency originally requested, is time and money, not a dearth of patients.

Also, from the Agency's perspective, there may be a greater risk of Type I error in the absence of a compelling biologic rationale for a specific treatment. Furthermore, the large size of the addressable population may encourage conservativism in the approval process.

Finally, there may also be a question of whether a gain of less than one minute on average in exercise tolerance in the rintatolimod compared to the placebo groups has clinical significance or makes for a meaningful improvement in the quality of life.

On the other hand, there are currently no approved treatments for CFS, and patient advocacy groups are understandably desirous to have available to patients safe and effective treatments.

FDA's approach to CFS has also undergone some evolution since the previous NDA submission, though how much impact this will have on the approval process remains unknown.

http://seekingalpha.com/article/105...ronic-fatigue-syndrome-a-regulatory-conundrum

 

[Firestormm]

Am trying to decide if I am more excited about Amligen (8 days and counting) or Christmas (13 days and counting). Must say Xmas wins by a head in my case What about you guys?

I seem to recall that last year we were awaiting an end of XMRV. There must be something about this time of year. Not a nice time for bad news is it? Still it will be good to get this FDA hearing out of the way I suppose - are we expecting a decision on 20th there and then or will it take a while? I think the meeting is a webinair event isn't it?

Should try and post a link I suppose at some point...

 

[Firestormm]

Llewellyn King: 15 December 2012:

FDA to rule on 'Lazarus' drug

http://www.stamfordadvocate.com/local/article/FDA-to-rule-on-Lazarus-drug-4121250.php

Extract:

"...Dr. Andreas Kogelnik, who runs the Open Medicine Institute in Mountain View, Calif., puts the chances of FDA approval for Ampligen at just 50 percent. Although he is rooting for the drug to be approved, he says the FDA may require more data on collateral effects. This has happened in the past and the FDA has not been satisfied with previous applications.

Dr. Daniel Peterson, who has been treating CFS since 1984 in Incline Village, Nev., said recently that he thinks fewer than 100 people at any one time, either through trials or compassionate waivers, are on Ampligen. The drug is only available in a few states, most prominently New York and Nevada. It is very expensive (about $25,000 for a course of treatment) and has to be administered through intravenous infusion -- a long, slow process, at regular intervals.

According to Kogelnik, some patients react poorly right off the bat, while others show substantial improvement almost immediately..."

 

[Firestormm]

Is this an old one from Cort or a new one? The date is not evident but it does show web links to the conference I believe:

http://simmaronresearch.com/simmaro...s-ampligens-fate-live-on-webcast-on-dec-20th/

Simmaron:
Dec 20th: High Noon for Ampligen and Chronic Fatigue Syndrome


n.b. Is Cort not posting here on PR any more then or what? Thanks.

 

[Firestormm]

The FDA's final decision on Ampligen is set for February 2, 2013. The FDA usually follows the recommendations of its advisory panels, although it is not required to do so.

HEB has thus far hit a 52-week low of $0.19 and a 52-week high of $1.10. The stock closed Friday's trading at $0.68, down 4.85% on a volume of 3.18 million shares.

17 December 2012: http://www.rttnews.com/story.aspx?Id=2024336

 

[Firestormm]

FDA: Doubts Raised by Chronic Fatigue Studies

WASHINGTON -- FDA reviewers are skeptical of the results of separate Phase III trials for the chronic fatigue syndrome drug rintatolimod (Ampligen) and question the way the studies were conducted, according to an agency document released Tuesday.

While the mechanism of action of the drug is unknown, rintatolimod's manufacturer, Philadelphia-based Hemispherx Biopharma, believes the double-stranded RNA functions like an immune modulator.

One double-blinded, placebo controlled, randomized trial of 92 patients demonstrated a slight benefit in the quality-of-life measure, "but multiple conduct issues with the trial suggest that results should be interpreted with caution," the FDA noted in briefing documents released ahead of a Thursday advisory committee meeting on the injectable drug. "The confirmatory trial failed to replicate these results."

"In trial AMP-516 neither the primary endpoint of [exercise tolerance testing] duration nor any of the secondary endpoints, including KPS, demonstrated a statistically significant effect of Ampligen using the protocol-specified analyses," the FDA reviewers noted.

The FDA's review of rintatolimod's safety data also caused concern for the agency. Serious adverse events were not counted, adverse events were miscoded, and others weren't properly defined.
"These discrepancies raise significant questions about data from all of the trials and whether or not the data are sufficient to determine the safety profile of Ampligen. This will be an important question at the advisory committee meeting," the FDA reviewers said.

18 December 2012: http://www.medpagetoday.com/Psychiatry/sleepdisorders/36514

 

[dannybex]

Finally, there may also be a question of whether a gain of less than one minute on average in exercise tolerance in the rintatolimod compared to the placebo groups has clinical significance or makes for a meaningful improvement in the quality of life.

There are other reasons, but that 'less than one minute' improvement, is one of the main reasons why I believe it won't be approved. What drug, especially one that costs $2,000 a month, would get approved based on that evidence? And what insurance company would pay for the treatment?

Krill oil, nattokinase, b12, etc., can -- in many, but of course not all cases -- improve one's 'exercise' tolerance by a minute or ten+ minutes -- at a fraction of the cost.

I'd be stunned if it is approved, but happy for those who have found it to be beneficial, like Mary Schweitzer.

 

[dannybex]

Okay, I'm really shocked that the stock price has tumbled so quickly. That can't be good...

http://www.fool.com/investing/general/2012/12/18/hemispherxs-unsurprising-45-plunge.aspx

"Apparently, the agency disagreed with that approach as ​

negative briefing documents​

ahead of Thursday's advisory committee meeting sent shares down 45%..." ​