Ampligen: It's a NO

From the GlobeNewswire:

Hemispherx Biopharma, Inc.(NYSE MKT:HEB) (the "Company" or "Hemispherx"), announced that it received a Complete Response Letter from the US Food and Drug Administration ("FDA") declining to approve its new drug application ("NDA") for Ampligen® for Chronic Fatigue Syndrome ("CFS"). The FDA said Hemispherx should conduct at least one additional clinical trial, complete various nonclinical studies and perform a number of data analyses.

In its Complete Response Letter ("CRL"), the FDA set forth the reasons for this action and provided recommendations to address certain of the outstanding issues. The Agency stated that the submitted data do not provide substantial evidence of efficacy of Ampligen® for the treatment of CFS and that the data do not provide sufficient information to determine whether the product is safe for use in CFS due to the limited size of the safety database and multiple discrepancies within the submitted data. 

In the two pivotal clinical studies that form the basis of approval for Ampligen®, Hemispherx believes that the primary efficacy endpoints were met and that they showed a statistically significant improvement (i.e., with a p-value of 0.05 or less). The FDA and Hemispherx do agree that in clinical study AMP-502, the primary endpoint was met (p=0.02). In clinical study AMP-516, the FDA's analysis resulted in a p-value of 0.10, while Hemispherx's calculation resulted in a p-value of <0 .="" nbsp="" p="">

Hemispherx plans to request an end-of-review conference with the FDA as a precursor to submitting a formal appeal to the Office of New Drugs in the FDA'sCenter for Drug Evaluation and Research regarding the Agency's decision. The purpose of the conference is to review all of the issues raised in the Agency's CRL as well as to discuss the corroborating data and experiences of clinicians and patients who have seen the benefits of Ampligen® therapy. 

Hemispherx has become aware that a prominent CFS advocate and long-time CFS sufferer, who has been on Ampligen® since 1999 through a treatment IND, began a hunger strike on January 30, 2013 to seek FDA approval of Ampligen®.  Hemispherx understands the frustration that there is still no FDA-approved treatment for CFS and the concern that patients may lose access to Ampligen® therapy. Out of concern for the health of the CFS community, Hemispherx has asked any hunger strikes be discontinued and that patients join in a collaborative effort between the FDA, Hemispherx, CFS clinicians and patient advocates to find a solution to this significant unmet medical need. 

In the past, the FDA has shown great willingness to work with stakeholders to find solutions for serious and life-threatening illnesses. Dr. Margaret Hamburg, Commissioner of the FDA has previously stated that, "FDA has an important role to play in shaping the future of medical breakthroughs by bringing stakeholders together to identify and overcome challenges." Hemispherx hopes that the FDA will view the Ampligen® end-of-review conference as an opportunity to involve patient advocacy, clinicians and researchers in a concentrated effort to do something for these patients over the near-term, including further evaluation of how new legislation, such as the recently enacted "FDASIA" statute, may have a role in finding a solution. The views of one internationally recognized researcher/clinician, Dr. Nancy Klimas, who has over 20 years' experience evaluating and treating CFS patients, can be found athttp://www.sciencedaily.com/releases/2013/01/130124183448.htm

DISCLOSURE NOTICE: The information in this press release and the article referenced therein includes certain "forward-looking" statements (explained below), including statements about the remaining steps, including the aforementioned end-of-review conference and appeals process, which the FDA may require and Hemispherx may take in further seeking FDA approval of the Ampligen® NDA for the treatment of Chronic Fatigue Syndrome. The final results of these and other ongoing activities could vary materially from Hemispherx's expectations and could adversely affect the chances for approval of the Ampligen® NDA.  Any failure to satisfy the FDA's requirements could significantly delay, or preclude outright, approval of the Ampligen® NDA. 

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® (FDA approved for a category of sexually transmitted diseases) and the experimental therapeutics Ampligen® and Alferon® LDO. Because both Ampligen® and Alferon® LDO are experimental in nature, they 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. Ampligen® is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system including Chronic Fatigue Syndrome. Hemispherx's platform technology includes components for potential treatment of various severely debilitating and life threatening diseases. Hemispherx has patents comprising its core intellectual property estate and a fully commercialized product (Alferon N Injection®). The Company wholly owns and exclusively operates a GMP certified manufacturing facility in the United States for commercial products. For more information please visitwww.hemispherx.net.

Forward-Looking Statements

To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as "believes," "plans," "anticipates," and similar expressions are intended to identify forward-looking statements. These statements are based on the company's current beliefs and expectations and represent the Company's judgment as of the date of this release. The inclusion of forward-looking statements should not be regarded as a representation by Hemispherx that any of its plans will be achieved, including its intent to pursue the end-of-review conference and appeals process. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond Hemispherx's control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. Examples of such risks and uncertainties include those set forth in the Disclosure Notice, above, as well as the risks described in Hemispherx's filings with the Securities and Exchange Commission, including the most recent reports on Forms 10-K, 10-Q and 8-K and Hemispherx's beliefs that the Ampligen® NDA may be covered by the new provisions of the FDASIA statute, which are subject to FDA interpretation and implementation, or that such provisions, if applicable, will be helpful with regard to obtaining FDA approval of the Ampligen® NDA. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Hemispherx undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise revise or update this release to reflect events or circumstances after the date hereof.

Hamlet Without Hamlet

Patient advocate Bob Miller, who's on the experimental drug Ampligen, has been urging FDA to approve the medication for ME by going on a hunger strike.  He's been fasting since January 29th: six days.  Healthy people fasting for six days would be weak. But someone with ME?

Last month another patient advocate suggested to me that ME patients or their loved ones go on such a strike.  I shook my head and gave a gallows-humor kind of laugh.  I thought it cruel to ask such sick patients to stop eating, more unthinkable than doing Hamlet without Hamlet.  And as for loved ones who'd lend that kind of support, well, I don't know about you, but I don't know too many people who believe in ME, let alone be willing to fast for someone with the disease.

But maybe it doesn't always take a lot of people to effect real change.

Ironically, perhaps his being on Ampligen has given Bob Miller enough strength for a hunger strike.  Or maybe Bob Miller's just so fed up with the pathetic state of affairs with ME that he brought out the big guns.  With no drugs except tottering Ampligen and little useful research, it's drier than the Sahara out there.  Miller's one-man hunger strike is, thankfully, garnering a lot of attention, including that of a well-known early AIDS activist, Cleve Jones.

Jones's character appeared in Gus Van Sant's fantastic movie Milk, about the slain politician and gay activist Harvey Milk. In this clip, Milk (played by Sean Penn), enlists Jones (Emile Hirsch) to join the revolution for gay rights. For a grim laugh, try substituting "CDC" for "Franco" and "ME people" for "gay people."


Here's the press release about Bob Miller's hunger strike:

FOR IMMEDIATE RELEASE

Contact: Bob Miller and Courtney Miller
Tel: 703-554-5575 
Email: bobmiller42@msn.com

PATIENT ON HUNGER STRIKE GAINING ATTENTION 

Well-known Journalist and Long-time AIDS Activist Offer Support 

Calling attention to the plight of one million disabled Americans suffering with ME/CFS (myalgic encephalomyelitis/chronic fatigue syndrome), patient Robert Miller continues his hunger strike -- and it is gaining attention from a well-known journalist and a long-time HIV/AIDS activist, as well as an NBC affiliate in Nevada.

These new developments come as we wait for the Food and Drug Administration's decision, due Monday, Feb 4, 2013, on whether or not it will approve Ampligen, the first medication ever considered by the FDA for ME/CFS. This illness disables one million Americans, leaving many bedridden, homebound and suffering for decades on end with little hope for recovery and little help from the U.S. government, which dedicates a mere $6 million dollars each year to ME/CFS research.

First, well-known HIV/AIDS activist, Cleve Jones, founder of the AIDS quilt project, came out in support of ME/CFS patient Robert Miller's hunger strike. Mr. Jones writes: “My friend is on a hunger strike to get the first medicine approved for his severe case of chronic fatigue syndrome. FDA is likely to deny the drug this weekend. We know we wouldn’t be where we are today if some of us hadn’t protested inaction by federal agencies on AIDS treatments. Email: kathleen.sebelius@hhs.gov  Urge FDA to approve Ampligen, so all CFS patients can have just one treatment option. CFS patients suffer without any treatments, and I can remember those days in my life. I urge Secretary Sebelius to approve Ampligen for CFS and apply what we learned with HIV about getting treatments to patients like every minute counts.”

Secondly, long-time Washington DC journalist, Llewellyn King, commented on ME/CFS sufferer Robert Miller's hunger strike on his PBS television program, "White House Chronicle." It is airing this weekend on PBS stations in Washington --Sunday, Feb. 3 at 9 a.m. on WETA, Channel 26; Sunday at 11:30 a.m. and 6:30 p.m. on WHUT, Channel 32. It will air throughout the week on some 200 PBS and public, educational and governmental cable TV stations and worldwide on Voice of America Television. An audio version of the episode will air Saturday, Feb. 2 at 9:30 a.m., 3:30 p.m. and 9:30 p.m. on Sirius XM Radio's POTUS (Politics of the United States) Channel 124. The episode will be posted on the "White House Chronicle" Web site, whchronicle.com, on Monday, Feb. 4.

Lastly, NBC's Reno, Nevada affiliate has covered the hunger strike: http://www.mynews4.com/news/local/story/Patients-fight-for-FDA-to-approve-drug-application/zuCxhkI0eUOiECmK_McvmA.cspx

Activist Rivka Solomon, a Boston-area playwright who has spent 23 years disabled by ME/CFS, much of it homebound and bedridden, states, "One million disabled Americans feel abandoned by their government for over two decades. They feel this can not continue and that they have no choice but to take extraordinary measures, such as a hunger strike, to get the help they need from the U.S. government. I support Robert Miller and other patients who are doing all they can to get us the medications we need to help us survive. We hope the FDA cares enough about its disabled citizens to approve Ampligen."

# # #

If you'd like more information about this topic, or to schedule an interview with Robert Miller, please call 703-554-5575 or email bobmiller42@msn.com

BOB MILLER'S HUNGER STRIKE

Advocate Bob Miller has been on a hunger strike to try to convince FDA to approve the experimental drug Ampligen for ME.  He and fellow patient Dr. Janet Smith got a terrific piece of local news coverage in Reno, Nevada. FDA is scheduled to make a decision on Ampligen's fate by tomorrow.  Given that the FDA committee voted no to Ampligen on December 20, odds are slim that the FDA will overturn its committee's decision, but it ain't over until it's over.

Bob Miller posted this on Facebook today:
"Patients who are able can call NBC News at 212-664-4444 and comment on the story [above]. Be sure to tell the person who answers why you are calling, they will put you through to a comment line where you can request for them to please cover this important news story nationally, be sure to tell them where you are calling from."

FDA'S DOUBLE STANDARD

A mad-as-hell Sid Wolfe of the watchdog group Public Citizen sued FDA about the agency's refusal to ban the highest dose of the Pfizer Alzheimer's drug Aricept--and lost. Studies don't show increased efficacy of the 23-milligram pill compared with the lower doses of 5 and 10 milligrams.  However, the higher dose does show increased toxicity, including, Wolfe said, increased mortality.

According to a comprehensive article by reporter Ed Silverman, the director of the FDA’s Division of Neurology Products, Rusty Katz, explained his reasoning for keeping the higher dose by posing a question and then answering it:  “Does the absence of a demonstration of any superiority of the 23 milligram dose to the 10 milligram dose on measures of overall functioning, coupled with the increased incidence of potentially significant adverse events, argue against the approval of this product?” The 23 milligram dose is “clearly superior to the 10 milligram dose” on a cognitive measure.* “In my view, this strongly argues for a conclusion that the 23 milligram dose is very likely to also have an effect on overall functioning, despite this not having been demonstrated directly in this study” [emphasis added].

Reporter Ed Silverman also wrote that given the few Alzheimer's treatments available, FDA believes that physicians may decide the added side effects justify the benefits. 

Following that logic, FDA, Ampligen's side effects are in most cases, minor or manageable, and given that there are no FDA-approved ME treatments, shouldn't Ampligen be approved?  Instead the FDA committee, while endorsing Ampligen's safety, voted that the drug didn't meet FDA's standards for efficacy--though it was "probably effective."  Probably effective sounds an awful lot like the "very likely" of two paragraphs up.

Why the double standard, FDA?  Could it be FDA is throwing the pharmaceutical giant a big bone because the patents on the lower doses are expiring?  Could it be that FDA believes Alzheimer's to be more important than ME?  Could it be, as Sid Wolfe suspects, that FDA doesn't want to admit it was wrong in approving the 23 milligram dose in the first place?

Sid Wolfe also testified at the FDA Ampligen hearing on December 20 that Ampligen shouldn't be approved either.  (And a patient I talked to at the meeting said she overheard him say that cognitive therapy works.) 

Here's Ed Silverman's story, if you want more info.

*In three of the four tests, there was no increase in cognitive function at the higher dose.