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Pluristem's Radiation Antidote Bests Blockbuster Drug Neupogen

|Includes: AMGN, Pluristem Therapeutics, Inc. (PSTI)

PLX-R18 heals 100% of population exposed to lethal radiation through nuclear accident or attack; Ready to supply U.S. government

A new cell therapy can heal radiation damage and save lives following a dirty bomb detonation, nuclear attack, or nuclear power plant accident. In preclinical studies PLX-R18 has shown a 100% recovery rate in animals exposed to radiation. Within an astonishing 48 hours of injecting PLX-R18, bone marrow blood cell production returned to normal. "We saw that injecting the placenta cells enabled nearly 100% of the population to recover, compared to 30% of the group that did not receive the injections," stated Yaky Yanay, President and COO of Pluristem Therapeutics (NASDAQ:PSTI), in a recent Jerusalem Post article.

The Jerusalem Post broke the story on February 21 that not only are PLX-R18 cells 100% effective in healing patients exposed to nuclear attack, it also revealed that Pluristem has agreed to "make available [a] supply of the radiation antidote to the U.S. if needed." This is significant.

Pluristem's ability to deliver doses from its factory in Israel positions PLX-R18 to go head-to-head with Amgen's (NASDAQ:AMGN) blockbuster cancer drug Neupogen®. First approved in 1991, Neupogen boosts white blood cell production to fight infections in cancer patients going through chemo and radiation therapy. It has since been approved for many other cancer-related indications. Neupogen (filgrastim) is on the World Health Organization's Model List of Essential Medicine, the most important medications needed in a basic health system. The drug produced $1.05 billion in revenues for Amgen in 2015.

Neupogen became the first FDA approved drug in March 2015 for the indication of radiation induced myelosuppression, following a nuclear incident, the hematopoietic syndrome of Acute Radiation Syndrome (ARS). Myelosuppression happens when radiation damages bone marrow, stopping the production of blood cells. According to the FDA, Neupogen is the first FDA approved medical countermeasure to increase survival of patients for this indication.

A $157.5 million contract was granted to Amgen for Neupogen by the U.S. Department of Health and Human Services' Biomedical Advanced Research and Development Authority's (BARDA) Project BioShield. With strategic goals including to "support advanced product development" and "stockpile acquisition", Project BioShield's contract dictates Amgen will manage the inventory of ARS treatments and will rotate inventory so that the prepared doses don't expire. This appears to describe an ongoing contract with recurring revenues to continually replenish the stockpile.

With the FDA's blessing, PLX-R18 is also heading towards approval for treatment of the hematologic components of ARS. The U.S. National Institutes of Health's National Institute of Allergy and Infectious Diseases (NIAID) is initiating studies in large animals to determine the proper dosage for PLX-R18. Once the dose is established, NIAID will move into the pivotal and final trial for full FDA approval of PLX-R18 for use in humans.

Approval could come fast for PLX-R18, with no need for Phase I, II, or III trials in humans. This is because the FDA can't require that humans be exposed to harmful levels of radiation for the purpose of a clinical trial. That would be unethical. Cases like this fall under the FDA's established Animal Rule, whereby the treatment is tested in large animals. Moreover, these studies are being paid for by the NIH, not Pluristem. Stockpiling PLX-R18 would be the most likely move by the U.S. government following FDA approval.

Similar to Pluristem's FDA approval track, Neuprogen was tested for ARS in large animals, was approved under the Animal Rule, and was developed in collaboration with NIAID . According to the FDA, "With the approval of Neupogen for the treatment of humans acutely exposed to myelosuppressive doses of radiation, the U.S. population is better prepared for a possible radiological or nuclear emergency." They might be stating the same, and more, about PLX-R18 soon.

With an expired patent, Neupogen was the first drug to be faced with competition from biosimilar knock offs. Before its approval for the treatment of ARS, Neupogen generated blockbuster revenues for Amgen because it's been widely used in cancer patients. PLX-R18, a new cell therapy with new patents issued and pending, has similar potential and a long time before facing patent expiration.

Potentially besting Neupogen, PLX-R18 has shown to boost production of platelets, red blood cells and white blood cells, while Neupogen only claims to boost white blood cell production. There is a huge distinction here that favors PLX-R18 because patients exposed to high levels of radiation need to boost production of all three blood components. Treating with PLX-R18 instead of Neupogen would eliminate the need to give two additional drugs which each increase red blood cells and platelets. In an emergency ARS situation, this could mean the difference between life and death.

Speeding up the process of blood production while bone marrow rebuilds itself, PLX-R18 works at a critical time when the patient could die from infections. PLX-R18 could go beyond blockbuster status with an ability to treat a broad range of indications related to bone marrow function damaged by either cancer treatments or nuclear catastrophes.

NIAID is funding studies for a few other ARS treatments for lung and gastrointestinal damage. BARDA has also purchased Leukine from Sanofi, which also increases white blood cell production, but is not specifically FDA approved to treat ARS. Although PLX-R18 has not yet been tested specifically in the treatment of lung or gastrointestinal indications, its mechanism of action suggests the cell therapy may have efficacy in these ARS indications beyond hematology.

The FDA has already cleared PLX-R18 to be used in humans for a different indication. Pluristem is about to enter a Phase I trial for PLX-R18 in humans for the treatment of hematopoietic recovery following bone marrow transplants, an indication to treat the side effects of radiation and chemotherapy. Clearly the FDA sees the radiation antidote as safe, based on a wealth of preclinical data, including 20 studies, from the world's leading research institutions.

Highlighting the dire and immediate need for a radiation antidote, the Fukushima Daiichi nuclear disaster on March 11, 2011 created a real and pressing case for a treatment like PLX-R18 to be readily available. Coming up on the 5th anniversary of the disaster, doctors at Fukishima are looking to Pluristem for help.

Fukushima Medical University's collaboration with Pluristem to use PLX-R18 to treat people who were exposed to radiation during the nuclear power plant meltdown is an important endorsement. In December Pluristem announced it signed a Memorandum of Understanding with the university's Fukushima Global Medical Science Center to develop PLX-R18 for the treatment of both ARS and morbidities following radiotherapy in cancer patients.

Japan is actively accelerating the practical usage of regenerative medicine, allowing the outsourcing of cell culturing and processing. Pluristem has been cited several times in official economic reports issued by Japan's Prime Minister Shinzo Abe, as an example of a cell therapy company active in Japan and part of making the Asian nation the leading country in the practical usage of regenerative medicine. They have enacted an adaptive licensing approval system with the goal of making Japan the leading country in the practical usage of regenerative medicine.

Pluristem's state-of-the-art cell manufacturing facility uses proprietary, industry leading 3D cell expansion technology that produces commercial quantities. Approved by the world's major health regulatory authorities including the FDA and European Medicines Agency (EMA), Pluristem is ready to supply PLX-R18 to the U.S. and Japan. The cells are an 'off the shelf' product that don't need tissue matching and have a relatively long shelf life, suitable for stockpiles.

Neupogen has been a blockbuster drug for Amgen since 1991. Racking up $7.42 billion in sales between 2010 and 2015 alone, it has been the wonder drug for boosting white blood cells in cancer patients having to undergo radiation and chemo. PLX-R18 could be the newer, better Neupogen, with a proven ability to produce red blood cells and platelets in addition to white blood cells. On the FDA approval track for both ARS and a cancer-related indication, Pluristem is well positioned to be the world leader in regenerative medicine.

Disclosure: I am/we are long PSTI.