DISCUSSION WITH DR. WILLIAM CARTER, DEVELOPER OF AMPLIGEN Biography William A. Carter, M.D. William A. Carter, M.D., is Chairman, CEO of HEMISPHERx Biopharma, Inc., (NASDAQ: HEMX). He is the co-inventor of Ampligen, and is a co-inventor of record on over 200 patents relating to the Company's technology. Dr. Carter was a leading innovator in the development of human interferon for a variety of treatment indications including various viral diseases and cancer. In this context, he received the first FDA approval to initiate clinical trials on a beta interferon product manufactured in the U.S. under his supervision. His interest in CFS/CFIDS goes back to 1987 when he and several board members became acquainted with PWC's and their severely debilitating disease. He regularly attends scientific meetings and presentations on research about the diagnosis and treatment of CFS/CFIDS, most recently the American Association for Chronic Fatigue Syndrome (AACFS) annual conference in San Francisco on October 13-16, 1996. Dr. Carter serves as Professor of Medicine at Allegheny University, PA., a position he has held since 1980. He is also director of Clinical Research for the University's Institute for Cancer and Blood Diseases. Dr. Carter has served as professor at Johns Hopkins School of Medicine, Hahnemann University, and the State University of New York at Buffalo. Dr. Carter received his degree from Duke University and underwent his post-doctoral training at the National Institutes of Health and Johns Hopkins University. He is currently a consultant to the NIH. He is the author of over 125 published sceintific articles including the Co-Editor, Handbook of Experimental Pharmacology on Interferon, Springer-Verlag, Publisher. In addition, Dr. Carter has authored or co-authored nearly 40 book sections. Contacts: William A. Carter, CEO, HEMISPHERx Biopharma, Inc. (215) 988-0080 ------------------------------------------------------------------- TRANSCRIPT HOST: OK, LET'S WELCOME DR. WILLIAM CARTER AS OUR GUEST SPEAKER HERE TONIGHT & WE WILL WASTE NOT IME.... E-MAIL FOR LOGS PLEASE... DR. CARTER, THANK YOU FOR BEING HERE.... PLEASE TAKE THE FLOOR IF YOU WILL..... DrWmCarter: Good evening Charla, thank you very much for inviting me to be with you and your HOST: PLEASE CONTINUE DR. CARTER.... DrWmCarter: forum tonight. I would like to give you and your group a quick synopsis of a few things that are happening at HemispheRx before we go to the question and answer period. Hemispherx biopharma is focused entirely on the development of double stranded RNA compounds, particularly Ampligen, for antiviral and anticancer applications. We have no other, competing priorities. Our current strategy is to pursue the applications of Ampligen in Chronic fatigue and HIV through appropriate clinical trials funded by the company and designed with the guidance of the FDA and key members of the medical community. Any other applications of Ampligen will only be pursued by large pharmaceutical company partners We are committed to utilizing the highest professional standards in the clinical development of Ampligen. We currently have a Phase II clinical trial in CFS under way in Belgium. This trial will be expanded in the near future. Other clinical trials in CFS are under discussion with the FDA and announcements will be made when the details of these trials are Our Canadian distributor, Rivex, has been authorized to distribute Ampligen to doctors in Canada under the emergency drug release regulations of that country. It is expected that one of the indications for which Ampligen will be used in Canada will be CFS. Discussions are under way with Canadian doctors regarding supply of Ampligen for their patients. We recognize that Ampligen is currently a relatively expensive drug. Some of the reasons for this cost included the relatively small scale in which the drug is currently made, the high cost of the raw materials required, and the physical form of the drug. We are addressing all these issues in order to reduce the cost as much as possible We are ad in the near future, while continuing to follow Good Manufacturing Practices as defined by the FDA Starting with our public offering in 1995, the company has been able to raise sufficient financing to continue its development program for Ampligen. While no Guarantees caN BE MADE we are confident that we will be able to obtain the additional financing which will be required to complete the clinical development of Ampligen. We recognize that our goal is to make Ampligen available to all patients who can benefit from its use at a reasonable price. The company and its staff are fully committed to that goal. HOST: DR. CARTER......WILL YOU TAKE SOME QUESTIONS THAT I HAVE FROM SOME OF THE PWC'S HERE NOW????? DrWmCarter: Yes of course HOST: Why is the cost of Ampligen so prohibitively high,& is there any way around that for people on disability, ETC? We are hearing that the cost may be as high as $20,000 IF it is approved by the FDA? DrWmCarter: HemispheRx has invested approximately $100 million in research on Ampligen. Current costs cover only a fraction of the real cost of the drug. As production increases, and the Company realizes economies of scale, it is expected that the cost of the drug will be reduced. Also there are programs of various sources available to patients who cannot afford certain drugs. It^Òs hard to speculate which specific programs will be available to PWCs, but it is conceivable that such programs will be made available once the drug is approved. Separately, the Company will look into implementing its own programs to help PWCs who don^Òt have the financial resources to obtain the drug. HOST: I WOULD LIKE TO SAY TO EVERYONE THAT I PREPARED THESE QUESTIONS FROM THE ONES SENT TO ME BY THE PWC'S ON MY MAILING LIST.... READY, DR. CARTER? DrWmCarter: yes HOST: Besides writing to the folks at the FDA, what else can PWC's do to assist in expediting the approval of Ampligen in the US? DrWmCarter: As many of you know, there are national and regional organizations organized to help patients with their needs, including advocacy support. You may want to contact these groups, including the CFIDS Association at 1-800-44-CFIDS or the National Chronic Fatigue Syndrome and Fibromyalgia Association at 816-313-2000. HOST: Because PWC's tend to respond positively for a few days to any type of slow drip IV infusion, including saline solution, how can double blind studies be done to avoid imprvmnt. in the placebo group that cancels improvements in the Ampligen group? DrWmCarter: Studies are designed to look at long-term response to Ampligen ^Ö six months or longer. How hard is Ampligen on the liver, and over what period have the effects been studied? than those seen in the placebo group. Overall, Ampligen has been injected now over DrWmCarter: 30,000 times in various clinical trials. HOST: Whay aren' t there more CFIDS/ Ampligen studies happening in the US? DrWmCarter: Since our public offering in 1995, the Company has been properly financed. Studies have been presented to the FDA, and we are going through the normal course of speaking about their design with the Agency, and getting the proper input. The design of studies in CFS is especially important because the natural history of the the diease is not as well understood as it is for Hepatitis B infection, HIV infection and the like HOST: END OF THE LAST QUESTION???? OK TO GO AHEAD NOW???? DrWmCarter: Charla, yes HOST: Part of HEM/Hemispherx's problems have been related to the lack of capital. Why not go into a partnership with a larger drug company? If the reason is that Hemispherx is undercapitalized, isn't it the fiscal & moral obligation of Hemispherx to join with a larger drug company? DrWmCarter: Typically in our industry it's the smaller biotech companies that bring the major technologi to makrket (eg Genetech, Amgen, and Biogen) The larger companies historically play a mjor r role on the marketing side of new techonologies as opposed to devleloping the clinical and r research data necessary to reach th e approval stage. Charla, another question? HOST: Why is Hemispherx slowing down the approval process by charging huge amounts of money to patients in Ampligen studies? Isn't this counterproductive to both parties? DrWmCarter: This is a very important question. We cannot and do not charge patients for drugs in trials that are initiated for the development of the drug. How would patients on a placebo feel if they had to pay for a drug they do not receive? However, when it comes to individual patients whose doctor is requesting the drug on a single treatment IND application, the Company would like permission to obtain cost recovery. There are two reasons for this. One is that the data obtained from these patients cannot be used in trials needed to obtain approval. The other is that the Company cannot financially afford to put a large number of patients on the drug for free. Our feeling is that the number of patients who are severely debilitated and desperately ill is growing every months ^Ö out of 400,000 or more PWCs in the U.S., many have been sick for a long time. The research tax exempt foundations tell us time maybe running out for some of these patients who are critically ill, and much as we want to, we can^Òt meet the needs of all these patients and provide the drug at no cost. HOST: What steps need to be taken to gain Ampligen access for US PWC's as a whole group rather than just a few? What can PWC's do to help put the drug on the FDA's accelerated approval path? DrWmCarter: The FDA provides several venues to gain access to drugs for patients who suffer from serious or life-threatening illnesses such as AIDS, cancer and Alzheimer^Òs disease. These venues are well described in the FDA's WEB site, for example http:www.fda.gov/opacom/backgrounders/aids.html, or http://www.fda.gov/oc/oha/charging.html, or http://www.fda.gov/fdac/special/newdrug/speeding.html, or http://www.mco.edu/research/emerguse.html, or http://www.critpath.org/treatment/expand.html Peer reviewed clinical studies published in recognized scientific journals have suggested that Ampligen therapy for CFS may have clinical activity and may have a favorable safety profile. However, as with any chronic disease with variable clinical outcomes more studies will be useful. The Company is working closely with the FDA to design these studies. For its part, the Company will do every thing in its power to satisfy the FDA^Òs requirement for studies deemed necessary to obtain approval and looks forward to working closely with the FDA toward that goal. HOST: How is Ampligen administered? Is the treatment IV every few days, meaning we can not travel far from home? DrWmCarter: The product is given twice weekly by intravenous infusion for approximately thirty minutes. Clinical investigators, working with the Company, have learned that the drug may be safely infused in various settings, including the home, and self administered under certain conditions -- patient safety is always paramount. Physicians and their patients would need to work closely together to determine the situation in which the patient would be able to receive the product in a setting other than in a certified clinic or by a fully qualified health professional. Ultimately, patients will learn to administer the drug themselves and with their care givers (much as hemophiliacs do), however, this requires careful supervision and complete educational programs for patients. HOST: How many people have been included in Ampligen trials.... 20, 200 or 2000? How long were trials conducted per person & in total? DrWmCarter: More than 300 patients have been treated in trials which typically cover a time frame of six to twelve months. HOST: How have CFIDS patients faired since they have been off Ampligen? How long off Ampligen before CFIDS symptoms return? DrWmCarter: Clinical data is still being collected to provide qualitative answers to these questions. HOST: Is Ampligen being used for other diseases? How safe is it? DrWmCarter: In addition to the studies being conducted by the company, its corporate partner Bioclones Proprietary (an affiliated company of the South African Breweries Limited), is actively developing the product for potential treatment of Hepatitis B and C. Regarding the safety profile of the product, a patient^Òs physician should form an independent opinion by evaluating the peer reviewed public information and technical booklets the company has prepared for physicians titled ^ÓInvestigators Brochure^Ô. Generally the product is well tolerated, but idiosyncratic reactions to investigational drugs can exist as with any pharmaceutical product HOST: Why hasn't Hemispherx gone through with Ampligen the regular FDA process & done Phase 1,2 & 3 studies? And what is their cure rate? DrWmCarter: In fact, Phase 1 and 2 studies have been peer-reviewed and published. We want to clarify the terminology. We don^Òt refer to Ampligen as a ^Ócure^Ô, but rather as a investigational treatment therapy. HOST: Is there information available about the safety of Ampligen, & if it works for CFIDS? Doctors are asking why there are no published papers ???? DrWmCarter: If your doctor has specific questions on previous Ampligen trials, or wants to obtain a copy of the published scientific papers, we suggest he or she writes to Dr. David Strayer at: Hemispherx Biopharma, Inc. 1617 JFK Blvd. Philadelphia, PA 19103 Ph: 215-988-0080, Fax: 215-988-1739 HOST: BEFORE I ASK THE LAST QUESTION ON MY LIST, I WANT TO MAKE SURE THAT EVERYONE REALIZES THEY MAY ASK Q'S.....BUT PLEASE TRY ONE AT A TIME....DO NOT JAM.... THEN WE WILL GO TO THE PWC ROOMS AT 10PM..... READY, DR. CARTER???? DrWmCarter: yes HOST: Has Hemispherx submitted a proposal to the FDA for expanded access to Ampligen under procedure IND & if Hemispherx succeeds in its petition, will insurance cover the cost? DrWmCarter: To facilitate building scientific and medical consensus on a multifaceted area of clinical research, discussions and meetings with the FDA are not publicly disclosed until full agreement has been reached. Once agreement is reached appropriate public announcements will be made. As far as cost is concerned, interested parties may wish to consult various Internet web sites which deal with these matters. Refer to: http://www.apla.org/apla/9509/serotism.html, or http://www.critpath.org/treatment/expand.html, or http://www.his.com/~cwealth/reform.html Any questions anyone? A: Who should a PHYSICIAN contact at HEM to apply for an individual investigator IND? B: since cfs shows up in so many ways, what specifically does ampligen target? C: Dr. Carter, In your opinion, how long do you think it will be before Ampligen is FDA approv? D: What percentage of PWCs treated with ampligen have improved? HOST: HOLD ALL QUESTIONS TILL THESE ARE ANSWERED.... DrWmCarter: Clinical and medical issues should go to Dr. Strayer at Hemispherx E: Dr. Carter, will fibromyalgia Pts. qualify for ampligen? Do u think FM could be helped? DrWmCarter: 80 % of PWCs in Belgium trials who has been bedridden from 3 to 7 years were able to go back ro scxhool or to work after a 6-month treatment F: Does ampligen seem to help people varying severity G: Is there a distincted lower molecular RNase L involved. and if so were we born with it or is it inserted into our genes DrWmCarter: We cannot speculate on the time for FDA approval but we will continue to work closely with the FDA to get Ampligen approved as quickly as possible H: Dr. Carter, what are common side effects of Ampligen, are they ill-tolerated as before? A: Can you discuss the CFIDS test that the HEM PR release spoke of? HOST: HOLD THE QUESTIONSPLEASE.... YOU MAY ASK ALL YOU WANT IN THE PRIVATE PWC ROOMS.... DrWmCarter: In the public papers on the different studies, you will note that in general Ampligen was well tolerated ------------------------------------------------------------------------- AT THIS POINT, THE CONFERENCE ENDED AS DR. CARTER WAS UNABLE TO CONTINUE DUE TO TECHNICAL DIFFICULTIES. ========================================================================