Saturday, November 25, 2017

Hemispherx Biopharma Announces Corporate Progress and Financial Results for the Nine Months Ended September 30, 2017

Source:  Hemispherx Biopharma, Inc.

Hemispherx Biopharma (NYSE American:HEB) announced today its financial results for the third quarter ended September 30, 2017.

The net loss was $1.3 million for the three months ended September 2017 compared to $2.9 million in this period one year ago. The more than fifty-percent decrease is attributed to a reduction of operating expenses of $0.5 million and an increase in revenue of $1.4 million resulting from the revaluation of the redeemable warrants as of September 30, 2017.  Sales for the nine months ending September 30, 2017 totaled $387,000 compared to $76,000 one year ago. Operating costs and expenses, inclusive of Research and Development, were $2.7 million in the quarter ended September 2017 vs. $3.2 million in the same period one year ago.  

Cash and cash equivalents at September 30, 2017 stood at $2.3 million.

Hemispherx believes that the stage is set to capitalize on significant new opportunities during the balance of this year and 2018. We have repaired our flood-damaged manufacturing facility.  It is now ready, pending FDA certification and additional funding needed to facilitate restarting manufacturing, to produce FDA-approved Alferon N Injection®. A Specialty Distribution and Services Agreement is already in place for Alferon®. We have secured our first country approval for Ampligen® in ME/CFS and we expect several more in the coming years. We are making progress in defining the next steps for FDA registration of Ampligen®. We are aggressively opening new opportunities for Ampligen® in the fast-growing field of immuno-oncology. All of this is being accomplished with cautious use of capital and our commitment to increasing shareholder value.

About Hemispherx Biopharma
Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the clinical development of new drug entities for treatment of seriously debilitating disorders. Hemispherx's flagship products include Alferon N Injection® and the experimental therapeutic rintatolimod (tradenames Ampligen® or Rintamod®). Rintatolimod is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system, including Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS). Hemispherx's platform technology includes components for potential treatment of various severely debilitating and life threatening diseases. Because rintatolimod is experimental in nature, it is not designated safe and effective by the FDA for general use and is legally available only through clinical trials.

Hemispherx Human Safety Study of Intranasal Ampligen® with Influenza Vaccine Shows Ampligen was Generally Well-Tolerated

Source:  Hemispherx Biopharma, Inc.

Hemispherx Biopharma (NYSE MKT:HEB) announced that it has commenced full data analysis of an intranasal human safety study of Ampligen® plus FluMist® known as AMP-600. The study was previously closed, but the initiation of full data analysis awaited the FDA’s evaluation of preliminary reports of blinded study findings. That evaluation was completed per formal notification from the FDA on August 9, 2017. Intranasal Ampligen was generally well-tolerated in the study.

Thomas K. Equels, President & CEO, Hemispherx Biopharma, Inc.

This study is the first to evaluate the human safety of nasally-administered Ampligen. Test subjects in other human studies of Ampligen, including the pancreatic cancer treatment program currently underway in The Netherlands, have been administered Ampligen intravenously (IV). All told, approximately 100,000 IV doses of Ampligen have been administered and found to be generally well-tolerated.

Hemispherx believes that the top line data from the intranasal safety study, coupled with extensive pre-clinical data, support further development of Ampligen as a broadly applicable highly selective Toll-like Receptor 3 (TLR-3) based new generation, immune enhancing adjuvant for intranasally-administered vaccines.

“We’re very encouraged by the outcome of this study, establishing a foundation for Ampligen as an intranasal adjuvant. This shows Ampligen’s potential to take us to the next step of developing Ampligen either as a strain-specific or more universal influenza intranasal vaccine immune enhancing adjuvant”, said Thomas Equels, Hemispherx’s Chief Executive Officer. “This safety study was the first milestone to our ultimate goal, which is to combine Ampligen with a non-live virus vaccine to produce stronger and broader coverage and to confer longer immunity than is possible with the vaccine alone. I firmly believe that Ampligen’s multi-faceted impact on the immune system redefines the term adjuvant.”

Ampligen is a highly selective TLR-3 agonist that induces the innate immune responses required for adaptive protective immunity. Monkeys immunized with H5N1 vaccine and Ampligen showed enhanced protection after being challenged with a homologous highly pathogenic H5N1 virus. Pre-clinical studies also showed cross-protection against H5N1 viruses using trivalent seasonal influenza vaccine in mouse models. In theory, Ampligen induced cross-protection may extend to any vaccine for viruses subject to a high mutation rate and genetic drift.

Ampligen is currently being developed as an immuno-oncology agent in solid cancer tumors and as a treatment in Chronic Fatigue Syndrome (CFS). Studies have shown that the two totally different indications both benefit from Ampligen’s foundational strength as an immuno-therapeutic. The company has completed a pivotal Phase 3 clinical trial in ME/CFS, where it is the only late-stage drug in the pipeline for this disease. The U.S. FDA has advised that potential approval for commercial sale in the U.S. for a ME/CFS indication will require the conduct and review of a follow up confirmatory clinical trial. Hemispherx is supplying the drug for use by pancreatic cancer patients in an Early Access Program sponsored by the Dutch government.

In AMP-600, Ampligen was nasally-administered as an adjuvant in conjunction with FluMist®, a seasonal live-virus flu vaccine, to healthy human volunteers at the University of Alabama at Birmingham under the auspices of Paul Goepfert, MD, Associate Professor of Medicine in the Division of Infectious Diseases and Director of the Alabama Vaccine Research Clinic. Twenty-five subjects were enrolled, twelve in Stage 1 and thirteen in Stage 2. The data from the AMP-600 safety study extends the pre-clinical results into humans. In the study, Ampligen was generally well-tolerated when administered intranasally with a live-virus influenza vaccine. Future studies with non-live virus vaccines will seek to replicate Ampligen’s efficacy as shown in the animal model results.

“Our next milestone for intranasal Ampligen is to collaborate with an established vaccine maker to administer Ampligen intranasally with a non-live virus vaccine,” said Equels, “this next milestone will allow us to determine Ampligen’s clinical efficacy as a viral vaccine adjuvant and to evaluate it as a mechanism for providing cross protection from virus mutations as well as other forms of the virus. We have every expectation of results similar to the prior animal studies.”

About Hemispherx Biopharma
Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the clinical development of new drug entities for treatment of seriously debilitating disorders. Hemispherx’s flagship products include Alferon N Injection® and the experimental therapeutics rintatolimod (tradenames Ampligen® or Rintamod®). Rintatolimod is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system, including Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS). Hemispherx’s platform technology includes components for potential treatment of various severely debilitating and life threatening diseases. Because rintatolimod is experimental in nature, it is not designated safe and effective by the FDA for general use and are legally available only through clinical trials. For more information about Hemispherx visit www.hemispherx.net.

Hemispherx Biopharma Announces Collaboration with Millions Missing Canada to Bring Medication to Canadians for Myalgic Encephalomyelitis/Chronic Fatigue Syndrome

Source:  Hemispherx Biopharma, Inc.

Advocacy Organization to Work with Health Canada with the Goal to Gain Approval

Hemispherx Biopharma (NYSE MKT:HEB) has announced that on Thursday, August 3, 2017 it participated in a meeting in Toronto, Canada with Canadian advocates for Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS). The meeting was  lead by Millions Missing Canada and a Canadian physician representing Canadian patients with ME/CFS, in support of a collaborative effort to advance ME/CFS research and potential treatments in Canada. http://www.millionsmissingcanada.ca/links-and-resources/ This joint effort, which includes Dr. Ian Hyams, a leading physician specializing in ME/CFS management is the next step  to bringing Ampligen® to Canadians suffering from the disease.

One year ago, Ampligen® became the first drug therapy approved in the world for ME/CFS, receiving government approval from the Republic of Argentina. Applications for Early Access Programs (EAP) in Europe and elsewhere are planned in the coming year. In the United States, Hemispherx has completed a pivotal Phase 3 clinical trial in ME/CFS, where it is the only late-stage drug in the pipeline for this disease. The U.S. FDA has advised that potential approval for commercial sale in the U.S. will require the conduct and review of a follow up confirmatory clinical trial. Ampligen® is also currently being used in patients with pancreatic cancer in an EAP in Europe.

Dr. Hyams is the medical director of the chronic fatigue and pain clinic in West Vancouver, British Columbia ("BC"), specializing in ME/CFS and fibromyalgia management as well as the management of similar disorders. Dr. Hyams is a consultant at the Complex Chronic Diseases Program at BC Woman's Hospital in Vancouver and is a clinical instructor in the Department of Family Medicine at the University of British Columbia with responsibilities in the training of medical students and residents.

Dr. Hyams, who has treated ME patients with Ampligen®  in England and United States said that, both in his practice and in the Phase 3 clinical trial data, he has "seen significant improvement in ME patients receiving Ampligen®." Treatment with Ampligen® is by twice weekly intravenous infusions.

"We are pleased to work with Millions Missing Canada on this very important issue," said Thomas K. Equels, CEO and President of Hemispherx. "By working together we hope to be better able to achieve the goal of providing an effective therapy for the hundreds of thousands of people who suffer from ME. This unmet medical need must be addressed. Hemispherx and Millions Missing Canada will follow the model that Hemispherx used to obtain approval in Argentina by seeking a Canadian Pharmaceutical Partner who will file for regulatory approval in Canada."

The plan is to use the existing Ampligen® new drug application database, to gain approval of Ampligen® in severely debilitated ME/CFS patients in Canada.  

"Canada has the highest ME rates in the world with 1.9% of the Canadian population suffering from this disease," said Scott Simpson, a ME patient and advocate and representative of Millions Missing Canada. Mr. Simpson further stated   "For Millions Missing Canada to collaborate with Hemispherx to bring Ampligen® to Canada brings real data-driven hope to more than half a million Canadians with ME where little existed before."

"The time has come for physicians in Canada to realize that Myalgic Encephalomyelitis (ME) is a severely disabling disease for many, causing an increase in patients going on disability. I have personally witnessed as a physician treating these disorders for the last 25 years the potential for severely afflicted patients to rebuild quality of life, often resulting in patients being able to go back to work. It is an honor for me to be involved in the process of potentially introducing this drug to Canada, which will hopefully lead to many patients in need receiving this treatment," said Dr. Hyams.

About Hemispherx Biopharma
Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the clinical development of new drug entities for treatment of seriously debilitating disorders. Hemispherx's flagship products include Alferon N Injection® and the experimental therapeutic rintatolimod (tradenames Ampligen® or Rintamod®). Rintatolimod is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system, including Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS). Hemispherx's platform technology includes components for potential treatment of various severely debilitating and life threatening diseases. Because rintatolimod is experimental in nature, it is not designated safe and effective by the FDA for general use and is legally available only through clinical trials. For more information about Hemispherx visit www.hemispherx.net.  


Hemispherx Encouraged by Cytokine Biomarker Discovery Which May Lead to a Diagnostic for Chronic Fatigue Syndrome


 Source:  Hemispherx Biopharma, Inc.

Cytokine Signatures Could Lead to the First Empirical Measure of Disease Severity in Chronic Fatigue Syndrome

Hemispherx Biopharma (NYSE MKT:HEB) said today that a new study led by a team of researchers from Stanford University may provide the basis for a long-sought diagnostic to test for Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS), which is estimated to afflict more than one million Americans, according to the U.S. Centers for Disease Control and Prevention.

The study, led by Jose Montoya, M.D., found that people with ME/CFS had blood levels of certain pro-inflammatory cytokines in that correlated with disease symptom severity.  These findings suggested a link between excess inflammation and the disease. As stated in the Stanford Medicine Press Release dated Monday, July 31, 2017, “When comparing patients versus control subjects, the researchers found that only two of the 51 cytokines they measured were different. Tumor growth factor beta was higher and resistin was lower in ME/CFS patients. However, the investigators found that the concentrations of 17 of the cytokines tracked disease severity. Thirteen of those 17 cytokines are pro-inflammatory.” (http://med.stanford.edu/news/all-news/2017/07/researchers-id-biomarkers-associated-with-chronic-fatigue-syndrome.html)  The research was published Monday July 31st, in The Proceedings of the National Academy of Sciences (http://www.pnas.org/content/early/2017/07/25/1710519114.full) and was also mentioned on ABC’s Good Morning America (https://www.yahoo.com/gma/study-sheds-light-chronic-fatigue-syndrome-131405106--abc-news-wellness.html).

One year ago Ampligen became the first therapy ever approved anywhere in the world for ME/CFS, receiving government approval from the Republic of Argentina in August 2016. Applications for Early Access Programs (EAP) in Europe and elsewhere are planned in the coming year. In the United States, Hemispherx has completed a pivotal Phase 3 clinical trial in ME/CFS, where it is the only late-stage drug in the pipeline for this disease. The U.S.  FDA has advised that potential approval for commercial sale in the U.S. will depend on the conduct and review of a follow up confirmatory clinical trial. Ampligen’s immuno-therapeutic properties are also currently being used in pancreatic cancer in a 50-patient EAP approved by the Netherlands.

“There’s still a good deal of work to be done before this important biomarker discovery can be turned into a readily available diagnostic test for ME/CFS, possibly available in a doctor’s office. It represents a seminal breakthrough in developing a diagnostic for ME/CFS in patients whose symptoms are often misunderstood, or even not believed,” said Thomas Equels, CEO of Hemispherx. “This is an important step towards a blood test that could benefit millions around the globe suffering from this severe debilitating disease.”

About Hemispherx Biopharma
Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the clinical development of new drug entities for treatment of seriously debilitating disorders. Hemispherx’s flagship products include Alferon N Injection® and the experimental therapeutics rintatolimod (tradenames Ampligen® or Rintamod®) and Alferon® LDO. Rintatolimod is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system, including Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS). Hemispherx’s platform technology includes components for potential treatment of various severely debilitating and life threatening diseases. Because both rintatolimod and Alferon® LDO are experimental in nature, they are not designated safe and effective by the FDA for general use and are legally available only through clinical trials.


Hemispherx Updates Status of Immuno-Oncology Program in Pancreatic Cancer





Source:  Hemispherx Biopharma, Inc.

12 Pancreatic Cancer Patients Receiving Ampligen® Immuno-Oncology Therapy in Early Access Program

Hemispherx Biopharma (NYSE MKT:HEB) announced that 12 pancreatic patients are currently undergoing treatment with single-agent Ampligen immuno-oncology therapy in an Early Access Program (EAP) managed by Amsterdam-based myTomorrows, an international leader in providing physician access to experimental medicines.

Eight of the 12 patients entered the EAP with metastatic disease (late stage) and have been on drug for 12 weeks or longer. The four other patients entered the program in the last several weeks with earlier stage disease.

The program is being conducted at Erasmus Medical Center in Rotterdam under the supervision of lead clinician C.H.J. (Casper) van Eijck, MD, a noted Dutch oncologist specializing in pancreatic cancer.

 "Ampligen has been shown in numerous earlier studies to possess both tumor-killing and immuno-modulating activity, the two characteristics that form the basis of immuno-oncology therapy. This is developing into one of the fastest growing and promising forms of cancer treatment," noted Thomas K. Equels, CEO of Hemispherx.

He continued, "The program at Erasmus is intended to see if Ampligen's immuno-modulating properties correlate to disease progression and increased survival of pancreatic cancer patients who have run out of conventional therapeutic options. We are very pleased with the progress so far.  As evidenced in our many prior clinical studies, Ampligen appears to be generally well-tolerated by the pancreatic patients in this program."

The EAP program in the Netherlands is designed to treat up to 50 pancreatic patients with government funding to compensate Hemispherx for the experimental use of Ampligen in the program. Top line data from the program is expected to be reported before year-end.

Hemispherx and myTomorrows are working together on plans to expand early access to Ampligen to other European countries with full government reimbursement. 

Separately, Hemispherx is laying plans for cancer trials of Ampligen in the U.S.

Mechanistically, Ampligen's Toll-like Receptor 3 mechanism activates the innate and adaptive immune systems and, as such, modulates the tumor microenvironment, potentially enhancing tumor-killing activity and patient survival rates.

About Hemispherx Biopharma
Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the clinical development of new drug entities for treatment of seriously debilitating disorders. Hemispherx's flagship products include Alferon N Injection® and the experimental therapeutics rintatolimod (tradenames Ampligen® or Rintamod®) and Alferon® LDO. Rintatolimod is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system, including Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS). Hemispherx's platform technology includes components for potential treatment of various severely debilitating and life threatening diseases. Because both rintatolimod and Alferon® LDO are experimental in nature, they are not designated safe and effective by the FDA for general use and are legally available only through clinical trials. 


Wednesday, November 1, 2017

Citius Pharmaceuticals, Inc. Receives "Fast Track" Designation By FDA For Mino-Lok™ Investigational Trial





Source:  Citius Pharmaceuticals, Inc.

Citius Pharmaceuticals, Inc. ("Citius") ("Company") (NASDAQ: CTXR), a specialty pharmaceutical company focused on adjunctive cancer care and critical care drug products, announced today it has received official notice from the U. S. Food and Drug Administration ("FDA") that the investigational program for Mino-Lok™ is designated "Fast Track".  Mino-Lok is a catheter lock solution that has entered phase 3 trials for an adjunctive treatment for catheter related blood stream infection ("CRBSI"). Fast Track is a designation by the FDA of an investigational drug to expedite review to facilitate development of drugs which treat a serious or life-threatening condition and fill an unmet medical need.


A drug that receives Fast Track designation is eligible for the following:
  • More frequent meetings with FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval;
  • More frequent written correspondence from FDA about the design of the clinical trials;
  • Priority review to shorten the FDA review process for a new drug from ten months to six months; and,
  • Rolling Review, which means Citius can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the application is completed before the entire application can be reviewed.
There are currently no approved therapies to salvage infected central venous catheters ("CVCs"), a potential $750 million sector in the US alone.  CRBSIs are responsible for mortality rates up to 25% in some patients, and contribute to significant morbidities. Citius is currently starting up sites for a phase 3 trial of Mino-Lok in the United States.
Mr. Myron Holubiak, Chief Executive Officer of Citius, commented "We are extremely pleased to receive Fast Track designation from the FDA. The agency has been very supportive and helpful in developing the trial design for Mino-Lok as salvage therapy for infected CVCs in CRBSI.  We look forward to our continuing collaboration in developing Mino-Lok."

About Citius Pharmaceuticals, Inc.
Citius is a specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives, cancer care and unique prescription products that use innovative, patented or proprietary formulations of previously-approved active pharmaceutical ingredients. We seek to achieve leading market positions by providing therapeutic products that address unmet medical needs; by using previously approved drugs with substantial safety and efficacy data, we seek to reduce the risks associated with pharmaceutical product development and regulatory requirements. Citius develops products that have intellectual property protection and competitive advantages to existing therapeutic approaches. For more information, please visit www.citiuspharma.com.

Citius Pharmaceuticals Issues Shareholder Letter





Source:  Citius Pharmaceuticals, Inc.

Company Highlights Recent Nasdaq Uplisting and Additional Operational Milestones

Citius Pharmaceuticals, Inc. ("Citius") ("Company") (NASDAQ: CTXR), a specialty pharmaceutical company focused on adjunctive cancer care and critical care drug products, announced today that the Company mailed a letter to its shareholders updating them on recent developments, financial results, and new business opportunities. Highlights include:

  • Successfully uplisted to Nasdaq stock exchange where the company will gain access to broader investor audience.
  • Received positive response from the U.S. Food and Drug Administration (FDA) for phase 3 trial amendments of Mino-Lok™, our proprietary product that address a potential $750 million sector.
  • Secured funding of more than $26.8 million, including a recent raise of $6.8 million, with significant insider participation.
  • Finalized the addition of South America to The Company's worldwide license for Mino-Lok™.
  • Completed Phase 2a studies for Hydro-Lido, which could become the first FDA-approved product to address the estimated $500 million U.S. hemorrhoid marketplace.
"We are extremely pleased with the significant advancements that we have made and the foundation for long-term growth that we have established," stated Mr. Myron Holubiak, President and Chief Executive Officer of Citius Pharmaceuticals.  "Having transitioned to a more senior exchange and completed key initiatives in advancing our unique technologies, we felt it necessary to provide a thorough update on the overall health and trajectory of the Company.  We are confident, now that we now well-funded, to ideally be positioned as potential market leaders in two greatly underserved therapeutic areas. Management is committed to advancing the commercialization efforts of our products, Mino-Lok™ and Hydro-Lido, while remaining focused on our goal of improving shareholder value."

About Citius Pharmaceuticals, Inc.Citius is a specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives, cancer care and unique prescription products that use innovative, patented or proprietary formulations of previously-approved active pharmaceutical ingredients. We seek to achieve leading market positions by providing therapeutic products that address unmet medical needs; by using previously approved drugs with substantial safety and efficacy data, we seek to reduce the risks associated with pharmaceutical product development and regulatory requirements. Citius develops products that have intellectual property protection and competitive advantages to existing therapeutic approaches. For more information, please visit www.citiuspharma.com.