• To reduce or prevent serious or life threatening conditions caused by exposure to lethal or permanently disabling toxic chemical, biological, radiological, or nuclear substances. • Expected to provide meaningful benefit over existing therapies. • Human efficacy trials not feasible or ethical. • Use of animal efficacy data scientifically appropriate. • Does not apply if approval can be based on efficacy standards elsewhere in FDA regulations.
Animal Rule (cont.) • Still need human clinical data: – PK/immunogenicity data, and – Safety in population(s) representative of use. • Civilian use often includes pregnancy, children. • Approval subject to post-marketing studies and/or restrictions on use. • Please work closely with FDA on planning animal studies before starting them. • Potential limitations: • Where there is no valid animal model of disease; • How to predictably bridge animal data to humans; and • Confidence may be an issue, even in valid models.
• Examples of current studies on threat pathogens: – Smallpox - assays for immune response and potency, vaccine safety (neurovirulence), risk assessment on vaccine strategies and blood safety.... ----------------------------------------------
Steve King:
"....tarvacin has shown activity in the treatment of animals infected with lethal doses of lassa fever which is a category-A bio-terrorism watchlist virus."
...."Our plans for developing the Tarvacin antiviral program are really going in two different directions. The first is really geared toward bio-defense applications, so this could be either bioterrorism applications or widespread viral outbreaks. Our priority is to generate preclinical data. As the previous speaker mentioned there's really no way to run clinical trials for many of these bioterrorism threats, so collecting preclinical data is really about the best you can do. We then want to combine that with human safety studies that we will be generating in the Hepatitis C clinical trial, as well as our anti-cancer clinical trial, and we believe- pending positive results of course- that this could be a fairly rapid route to regulatory approval for tarvacin for the treatment of- again- widespread viral outbreaks."
...."In April of this year, Peregrine and NIAID entered into an Agreement to screen tarvacin for activity against a broad spectrum of enveloped viruses, and these really fall into two categories- those of health concern, and those of bio-terrorism concern."...
"So to kind of summarize why we're excited about tarvacin in particular for the bio-defense applications:
Number one- we've received promising preclinincal results in the lassa fevr animal model. Those included both short-term treatment advantages, as well as long term immunity to the viral infection.
The second is we have current collaborations with NIAID that are underway to screen against viral bio-terrorism and health threats. This is quite an extensive list of viruses they're testing the drug against. In fact, their indication to us was that this is one of the first times they've ever tested such a broad spectrum of viruses with a single agent.
Thirdly is discussions are taking place to screen tarvacin for activity against additional bio-terrorism and health threats again these would fall under the marburg and ebola virus families.
Fourth, data from preclinical human phase one safety could be used to support expedited FDA approval as a therapy for bio-terrorism and health threat concerns, and this kind of falls under the animal rule which was recently enacted and implemented by the FDA, in which if you can combine good preclinical efficacy data along with good human phase one safety data, then you can effectively go directly for the approval process.
Fifthly, the mechanism of action and target indicates potential as a single agent that can be effective against a wide number of enveloped viruses, including those that are known about and those that are not known about,
and, lastly- human clinincal studies are not far away. They will be beginning over the short term. This will provide us with critical- number one- safety data for the program, which will be necessary for any future approval filings, but also, in those same clinical trials we'll be looking for the effectiveness of removing the hepatitis C virus by looking at viral load changes."....
Steve King, president/CEO --------------------------------------
National Institute of Allergies and Infectious Diseases to Test Tarvacin(TM) for Antiviral Potential Monday April 4, 8:30 am ET - Peregrine Pharmaceutical's Anti-Phospholipid Therapy to Be Screened Against Broad Spectrum of Viruses That Threaten Worldwide Health and Global Security
TUSTIN, Calif., April 4 /PRNewswire-FirstCall/ -- Peregrine Pharmaceuticals Inc. (Nasdaq: PPHM - News) and the National Institute of Allergy and Infectious Disease (NIAID) have agreed to a collaboration in which NIAID's testing laboratories will screen Peregrine's Anti-Phospholipid Therapy agents, including Tarvacin(TM), for activity against a broad spectrum of enveloped viral pathogens of health and bioterrorism concern. Virus types to be screened as part of the collaboration potentially include herpes viruses, respiratory viruses, pox viruses, Hepatitis B and C, Papillomavirus and viruses of biodefense concern including Pichinde, Yellow Fever, West Nile and Dengue.
"We are very pleased to be able to work with the NIAID to further explore the anti-viral potential of Tarvacin(TM) and the rest of our Anti-Phospholipid Therapy agents," said Steven King, president and CEO of Peregrine Pharmaceuticals. "The data that will be generated from this collaboration will be very helpful in guiding development of our Tarvacin(TM) anti-viral clinical program."
During the first year of an ongoing 3-year, $1.68 million grant from the NIAID, scientists at the University of Texas Southwestern Medical Center at Dallas determined that Tarvacin(TM) binds to virally infected cells and viral particles including Pichinde virus, which causes a fatal viral hemorrhagic fever and is used as an established model for Lassa fever. Pichinde virus is on the U.S. government's biodefense Category A watch list. The researchers further determined that Tarvacin(TM) also significantly protected animals challenged with a lethal dose of Pichinde virus. Results from these studies are being presented at the annual meeting of the American Association of Immunologists taking place this week in San Diego, California.
Pathogens to be screened in this collaboration belong to a class of viruses known as "enveloped viruses," which derive their outermost coating from their host cell membrane during viral replication. Enveloped viruses account for many of the most concerning viral health risks including HIV, Hepatitis B and C, cytomegalovirus, hemorrhagic fever, SARS and various types of influenza including Avian influenza.
About Anti-Phospholipid Therapy in the Treatment of Viral Diseases
Anti-Phospholipid Therapy is Peregrine Pharmaceuticals' novel approach to treating cancer, viral infections and certain ocular diseases. It is based on the finding that aminophospholipids, which are basic components of the inner surface of the cell membrane, become exposed as antigenic targets in certain disease states.
A large number of viruses that impact global health and security possess an "envelope" derived from their host cell membrane. Since viruses lack the means to maintain structural organization of the envelope, amino-phospholipids such as phosphatidylserine (PS) and phosphatidylethanolamine (PE) become exposed on the surface of these viruses, making them potential therapeutic targets. Peregrine Pharmaceuticals, together with its collaborators, has developed a series of monoclonal antibodies directed against aminophospholipids to take advantage of this property.
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