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CFS and Ampligen: A Creative Solution from the FDA?

Sasha

Fine, thank you
Messages
17,863
Location
UK
What's a CRL?
Oh - found it on google - it's 'Complete Response Letter' and is issued by the FDA when they're not approving a drug and tells them what changes need to be made before it can be approved.

Here's more detail on CRLs:

http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/LawsActsandRules/ucm084143.htm

I'm more than a bit baffled that Hemispherx would leave Bob Miller still on his hunger strike if they've known the FDA's decision since Friday.

Looking forward to getting a proper public statement one way or the other...
 

JohnnyD

Senior Member
Messages
206
The response is complicated - Hemispherx must comply with a host of SEC regulations. They said they will respond as soon as they are able to.
 

Sasha

Fine, thank you
Messages
17,863
Location
UK
The response is complicated - Hemispherx must comply with a host of SEC regulations. They said they will respond as soon as they are able to.

SEC? US Securities and Exchange Commission? Financial stuff, not medical/research?

Have I mis-googled???
 

JohnnyD

Senior Member
Messages
206
SEC? US Securities and Exchange Commission?

Yes, Hemispherx is a public company and must disclose, within certain time constraints, material information to the (investing) public. Parts of a CRL will be material, other parts will not.
 

Ember

Senior Member
Messages
2,115
Speculation on my part. Regarding identifying sub-groups and those severely afflicted. I think this is what the CDC has been doing with their multi-site clinical assessment. They started this assessment in July, posted to webiste in November, results are due this month or next I believe.
I think that you're being overly optimistic. Dr. Unger is committed to initiating a dialogue that will result in a broad umbrella definition with severity scales:
Dr. Unger: It’s a broad umbrella with various degrees of severity. There are lots of approaches that we could take. I do not think that any one approach is absolutely wrong, but we have got to start the dialog, we have got to make it data‐driven, and we have to be unified.
In November 2011, she described three phases in creating the new definition, i.e., data collection, the selection of severity scales and dialogue:
Dr. Unger: As far as change, we are going to start with our clinical contract and getting advice from our clinical colleagues who are directly involved.... We are going to start with data collection.... That is step number one.

Step number two is, if we have good measures of the domains...then we try to say how can we capture this the best way.... We have to use a dynamic range in all of those measures. It is not enough to have just fatigue, but how much fatigue? It is not enough to have any one measure.

Going by what has happened at CDC in the past, we will next convene a meeting of experts.... We can anticipate that it will be at least one year before we could start the dialog in the process.
However in October 2012, Dr. Belay predicted that the data wouldn't be evaluated until the fall of this year:
Dr. Belay: We are asking [the contractors] to use their best clinical judgment from their years and years of experience to select CFS patients and then collect as much data as possible from all those cases and provide it to us independently so that we will be able to evaluate the different case definitions and see where they stack up.

Mr. Krafchick: What’s your timing on that?

Dr. Belay: We’ve already completed the enrollment in 30 percent of the cases. I would say the study will be completed within a year.

Here is Dr. Belay's comment on the delay from June 2012:
Dr. Rose: When will those data be ready?

Dr. Belay: They have started collecting the data, but I can’t give you a date because it’s dependent on those physicians, not us at CDC. Some of the sites have already started enrolling patients and we’re shooting for about 400 patients eventually to be enrolled. If that happens within the next 16 to 18 months, that will be great.
It isn't clear from Dr. Belay's comments in October what phase of the study is expected to be completed within a year.
 

Ember

Senior Member
Messages
2,115
Regarding identifying sub-groups and those severely afflicted. I think this is what the CDC has been doing with their multi-site clinical assessment. They started this assessment in July, posted to webiste in November, results are due this month or next I believe.
I notice that Cort recently commented, “The study was just greatly expanded; its now a five year study with biological samples included….”
 

JohnnyD

Senior Member
Messages
206
https://www.bioworld.com/content/fda-adds-ampligen-experts-roster-cfs-workshop-0

FDA Adds Ampligen Experts to Roster for CFS Workshop

By Randy Osborne
Staff Writer

The FDA published a notice in the Federal Register about the public workshop called "Drug Development for Chronic Fatigue Syndrome [CFS] and Myalgic Encephalomyelitis [ME]," slated for April 25-26 in Bethesda, Md.


It's the first such workshop held as part of the Patient-Focused Drug Development initiative, mandated in the authorization of the Prescription Drug User Fee Act, and a prime topic, given the uproar over the agency's refusal to approve Ampligen (rintatolimod) after a negative advisory panel meeting in December. (See BioWorld Today, Dec. 21, 2012.)


Ampligen was the only potential therapy on the horizon for CFS, long derided as "not a real disease," and many patients protested the FDA's inaction, especially those who had been benefiting from Ampligen by way of clinical trials conducted by its sponsor, Philadelphia-based Hemispherx Bioscience Inc. One activist patient, Robert Miller, went on a hunger strike to call attention to the issue. (See BioWorld Today, Feb. 6, 2013.)


Over the weekend, Miller wrote to the Health and Human Services' (HHS) secretary, Kathleen Sebelius, and to Margaret Hamburg, commissioner of the FDA, as well as others, calling the advisory panel meeting that turned thumbs-down on Ampligen "a pony show," and asking that he, as well as Daniel Peterson, who headed Hemispherx's trials and collaborated on others, be invited to the workshop.


"Did we not learn anything from HIV/AIDS?" asked Miller, who has been a CFS patient for 30 years and gained results with Ampligen. "Yes, [patients] know you are doing more, but after 30 years of nearly nothing, a Band-Aid appears huge. Every year, there are excuses they [HHS and its agencies] cannot do more."


In the Federal Register posting Monday, the FDA requested that anyone who wants to register to attend and/or take part in discussions submit their requests to the agency by April 8. The workshop will take up two main topics: disease symptoms and daily impacts that matter most to patients, and patients' perspectives on current approaches to treating CFS and ME.


Meanwhile, although the full roster of those invited to the workshop has not been made public, Kimberly McCleary, president and CEO of the Chronic Fatigue and Immune Dysfunction Syndrome Association of America, said she has seen a partial list.


"It's an interesting blend of CFS experts, so I think that will make [Miller] and others happy to see that, including two people who have been involved in the Ampligen studies," she told BioWorld Today. "They're both very capable spokespeople for what it's like to live with this. One is a physician herself who had to retire after she became ill. The core group [of those who formally present at the workshop] will be very compelling."


Peterson is not on the roster McCleary has seen. "One person contacted me independently and told me that he was going to be on the list," she added.


That's Bernard Munos, a consultant formerly with Eli Lilly and Co., of Indianapolis.


A CFS expert "who has looked at other treatments and done some pilot studies using other drugs and drug combinations" is included in the lineup, too, McCleary said.


The post in the Federal Register said the FDA "will try to accommodate all participants who wish to speak on [the first day, which is an afternoon session only], either through the panel discussions, audience participation, or the open public comment period," but warned that "the duration of comments may be limited by time constraints," probably anticipating a flood.
McCleary said "the bulk of the time, I would imagine, is going to be spent with the people the FDA has asked to attend," and each of those has been asked to stay for both days of the workshop, "so that they can then have a dialogue about the meeting in its entirety."


Hemispherx, which got its complete response letter from the FDA in February, said its hoped-for end-of-review conference with the agency will serve a precursor to submitting a formal appeal to the Office of New Drugs in the FDA's Center for Drug Evaluation and Research.


Ampligen, a Toll-like receptor 3 modulator, once was slated to undergo a Phase III trial to follow the AMP516 study, but the strategy later became to submit a new analysis of existing data from the 516, which used saline as its placebo control. But the decision to use saline was made in the early 1990s, before researchers began to use saline as a therapy for orthostatic and blood-volume problems in CFS. This meant the results may have been confounded by using a comparator with clinical efficacy, McCleary noted. (See BioWorld Today, Feb. 11, 2013.)
 

Ember

Senior Member
Messages
2,115
Hemipherx Biopharma to meet with FDA regarding Ampligen decision
Tue 3:57 pm by Deborah Bacal
Hemispherx BioPharma (NYSE MKT:HEB) has said it plans to request a meeting with the FDA regarding the regulatory agency's decision to to reject approval for its Ampligen drug for chronic fatigue syndrome (CFS)....

Hemispherx said in its statement on Monday that it plans to request an end-of-review conference with the FDA to discuss the agency's decision....
 

dannybex

Senior Member
Messages
3,561
Location
Seattle
JohnnyD -- perhaps you could help us understand this transaction:

"In this regard, we have registered $150,000,000 of securities for public sale pursuant to a universal shelf registration statement and we have been selling shares under this shelf registration statement and the New Maxim EDA. Through December 31, 2012, we had sold an aggregate of approximately 29,500,000 shares under this EDA."
...

"On November 26, 2012, all of the members of the Compensation Committee authorized the payment of bonus for the Company stock sold through the Maxim ATM based on the contractual obligation and opinion of independent counsel. For the year ending 2012, compensation was granted or paid related to the Executive Performance Incentive Program, as set forth in Section 3(c)(ii) of their respective Employment Agreements, for approximately $1,159,000 to each Dr. Carter and Mr. Equels, respectively."

From this page:

http://finance.yahoo.com/mbview/thr...8fa5-a446-4e5f-a4fa-7a23a9e9479c&tls=la,d,7,3

???