OneMedRadio: How Pluristem’s PLX Cells Saved a Dying Child

Pluristem Therapeutics, Inc. (NASDAQCM:PSTI; TASE:PLTR) recently announced that a seven year-old girl suffering from an aplastic bone marrow whose condition was rapidly deteriorating is now experiencing a reversal of her condition with a significant increase in her red cells, white cells and platelets following the intramuscular injection of the company’s PLacental eXpanded (PLX) cells. Aplastic bone marrow is a disease where the patient has no blood-forming hematopoietic stem cells in the bone marrow.

Pluristem Therapeutics is a leading developer of placenta-based cell therapies. The company’s patented PLX (PLacental eXpanded) cells are a drug delivery platform that releases a cocktail of therapeutic proteins in response to a host of local and systemic inflammatory and ischemic diseases. PLX cells are grown using the company’s proprietary 3D micro-environmental technology and are an “off-the-shelf” product that requires no tissue matching prior to administration. Pluristem is focusing on the use of PLX cells administered locally to treat systemic diseases and potentially obviating the need to use the intravenous route.

Data from two phase I/II studies indicate that Pluristem’s first PLX product candidate, PLX-PAD, is safe and potentially effective for the treatment of end stage peripheral artery disease when given locally. Additionally, Pluristem is developing PLX-PAD for cardiac ischemia, PLX-BMP for Acute Radiation Exposure, Bone Marrow Transplant Failure and Chemotherapy induced Bone Marrow Aplasia, PLX-ORTHO for orthopedic indications and PLX-PAH for Pulmonary Hypertension in collaboration with United Therapeutics. Pluristem’s pre-clinical animal models have demonstrated PLX cells are also potentially effective in other inflammatory/ischemic indications, including diastolic heart failure, inflammatory bowel disease, neuropathic pain and pulmonary fibrosis.

Senior Vice-President of Corporate Development, Bill Prather is interviewed by OneMedRadio below.

Click to hear full audio interview and see transcript that follows.

Matt Margolis             Greetings from OneMedRadio, I’m Matt Margolis. Today, I’m with Dr. William Prather, senior vice-president of corporate development for Pluristem Therapeutics, an American developer of placenta-based therapies headquartered in Haifa. Pluristem trades in the NASDAQ under the symbol PSTI. Thank you for joining us Dr. Prather.

Dr. Bill Prather:           Thank you, appreciate it.

Matt Margolis:            So maybe we can start with a quick overview of the company.

Dr. Bill Prather:           Well, as you said, Pluristem is a public company. We’re a cell therapy company. We are an American company that is headquartered in Haifa with the other people or so, and what we do is we take adult stem cells, so called mesenchymal stromal cells from the body of the placenta, and then expand them in a very proprietary three-dimensional manner, and then the cells that come out of that process we call PLX cells, and those cells we believe are available for a variety of inflammatory ischemic tissue in the blood in those states, and those cells can be administered to patients without any matching necessary sorts of off-the-shelf type of product.

Matt Margolis:            And why would you say this process is unique?

Dr. Bill Prather:           It’s unique, I think, from a couple of perspectives. The company’s basis was really the three-dimensional expansion of cells, and we believe we have the monopoly actually on the ability to take the MSCs no matter where you get from what source, and if you grow them into three-dimensional environment that’s a space we own, and you can do a lot of things to cells. You grow them in a three-dimensional environment versus cooling them in a flat surface like a Petri dish. You can actually change the biology of the cell by changing its physical characteristics, its physical environment that it grows within, and being able to change this biology, we could change the secretome or the proteins that are expressed by the cell, permanently by the way, and that basically allows us to manufacture specific PLX cell products, kind of an indication-by-indication basis, and you can’t do that if you grow cells in a flat surface.

The other cool part about as far as the company is concerned is that we get our cells from the placenta. It’s a medical waste. Basically thrown away. And that’s where we’re getting the cells right now. We have relationships with possibilities in the Haifa area, and moms are asked if they’d be willing to donate their placenta to science.

Matt Margolis:            On May 9th, Pluristem announced that the company’s placental-expanded cells had reversed the condition of a 7-year-old girl suffering from aplastic bone marrow. The intramuscular injection of PLX has significantly increased her red blood cells, white cells, and platelets. In this case, PLX was approved for compassionate use. I was wondering if you could talk a little bit about this fantastic story. What’s the background of your relationship with this therapy onsite? What’s your relationship with the regulatory bodies and this whole process?

Dr. Bill Prather:           It’s an interesting story. First of all, I’d like to say that, you know, I was here in the United States during this entire time that this patient was treated, and really, it’s, I think, a tribute to how people can work together with Israeli FDA, regulatory authority. Certainly, the people at the hospital, the physician and the personnel at Pluristem that, you know, they all kind of combined together to, you know, get this little girl her cells. The way this evolved was that we have relationships with the German government, the Israeli army, the US as well in trying to development a product that is going to be potentially useful in radiation exposure. So you know, God forbid, a nuclear power plant explosion, or a dirty bomb, or something like that. Then providing a therapy or treatment. And what these people want, so they want to be able to deliver the medicine that will help the bone marrow, recover the bone marrow that’s heard were destroyed with radiation. And they want to be able to give the medicine after the exposure to radiation, those victims that were exposed, and they want to be able to give the medicine conveniently to the patient. Ideally, the patient themselves, and be able to store those units of medicines that are stockpiled around the country.

And so we believe we fulfill a lot of those criteria, and we’ve been working at the Haddasah Hospital in Jerusalem using the cells in animals that have been radiated so we’ve kind of zapped animals with lethal and sub-lethal doses of radiation, and then waited 48 hours before we’ve given our cells, our PLX cells, and found significant results in terms of being able to save the rodent’s, you know, life but even better than that, the bone marrow seems to come back almost normal, and all of the peripheral elements of the blood have come back.

And we did this by – We actually gave the cells intramuscularly, IM, locally. So this is great because patients would now be able to inject themselves.

Matt Margolis:            So what were the steps involved in saving this girl’s life?

Dr. Bill Prather:           So all of this was going on at the Hadassah Hospital on this little girl who had been hospitalized with aplastic bone marrow, I think it’s in August of 2011, and she had gotten a bone marrow, and that had failed, and you know, she had aplastic bone marrow. I think she might have a hereditary disorder that has caused this problem for her. I think she had a sib that died of something like this. This was historical. The patient and the patient’s mother were – I think they were from Bulgaria and so they did not speak Hebrew, but she has been hospitalized for a long time in Hadassah. Her first bone marrow didn’t work. Her second bone marrow, they were able to detect robust hematopoietic stem cells that, that’s the blood-forming stem cells that are the element of transplant. They were able to detect the robustness of that, and they were detecting that this girl was, her second transplant wasn’t going to work. Plus she was emaciated, and she wasn’t able to eat. I mean she was dying.

So the doctor, her attending physician, knew about the work that was being done with our cells at Haddasah, and I’m not exactly sure how it worked, but somehow, the Israeli FDA, if you will, the regulatory authority, gave permission to the compassionate use of our cells in this patient. So we gave our cells to her. About 180 million cells were given to her intramuscularly, and that was – and then about a week or 10 days later – I can’t remember now – but that timeframe she got another dose of about 180 million cells, and you know, she was discharged yesterday. I think it was like 6 or 7 weeks after her first dose of PLX cells. Her just really dramatic increase in her white counts and red counts, her platelets are back where she’s no longer, you know, bleeding, and she was discharged.

Matt Margolis:            So maybe you can talk about from a company growth perspective, how this particular wonderful instance can actually serve to further the clinical phase for PLX and actually get it to approval and market.

Dr. Bill Prather:           Well, I mean we believe and we were so happy the little girl got better of course, but we believe that she really kind of represents a data point – Yeah, those are data points. I mentioned the effects of our cells given intramuscularly in those animals that were given their therapy or radiation exposure. That’s another data point that the IM use of our cells has this effect.

We have other data points as well. So we think we’re building or getting compelling evidence to suggest that you may not give our cells anyway intravenously for a systemic vindication. So this is a process that I think everyone kind of thought, but if you wanted to treat a systemic disease, you had to give the cells systemically IV, and that may not be true at all, which in our opinion was the kind of a paradigm change in the paradigm of how cell therapy is given. That may not work for others as well, but certainly, we’ve proved them that we cover bone marrow patients and animals when we give ourselves IM.

Matt Margolis:            You’re touching on the concept of applying PLX for various different indications, and you’re currently in clinical stage for a peripheral artery disease, CNS, and muscle injuries as well. So I’m wondering if you could talk a little bit about the milestones for these.

Dr. Bill Prather:           Our lead product is actually PLX-PAD, and those are cells that are given again locally. They’re given in the afflicted limb of patients suffering from peripheral artery disease. That’s where you have cholesterol plaquing that goes into the arteries that primarily feeds your lower extremities, and this cholesterol plaquing is associated with things like diabetes and high blood pressure and obesity, et cetera, so it’s a growing problem. Patients will begin to have symptoms of the disorder when they start to walk. They’re not getting blood there through the calf muscles in their legs, and so, the muscles are talking to them, and you’re having pain.

As the disorder progresses, there’s pain at rest, and that’s followed by ulcers or wounds, and then gangrene. If you have significant problems in the lower extremities, that’s a systemic disorder. The same disease going on in your heart, in your brain, et cetera.

And there’s no good therapy for it. There’s angioplasty. There’s bypass, there are surgical procedures, but there is no good medicine. What our cells do is we’ll inject our cells into the muscle of the afflicted limb, and then that inflamed tissue that needs blood will then talk to our cells. Our cells then secrete a cocktail of therapeutic protein, to stimulate you to grow new vessels into the area that use blood, anti-inflammatory proteins, protective proteins, his cocktail of therapeutic proteins that induces you to heal yourself.

The cells were totally safe even if given repeatedly in patients. The endpoint has to be amputation-free survival after one year. After a year did you have an invitation or did you die? Those are the endpoints and our industry norm of endpoints is like 60% invitation for survival. We had an invitation free survival rate of 85%, so it was very encouraging.

Matt Margolis:            So what other intriguing trials and experiments have you recently conducted?

Dr. Bill Prather:          We’re, of course, pursuing all the area of peripheral artery disease in Phase II trials or beginning Phase II trials.

We just got approval to start these trials in the area that of a lesser form of peripheral artery disease called Intermittent Claudication. And then we’re also going to be embarking upon a study in patients that have what’s called Berger’s disease. That’s kind of like cholesterol plaquing. It’s a rare disorder, we have offer in drug approval for it. And if we’re successful with our strategies, we’ll be able to embark upon those trials in the beginning this 2013, and they will be the little trials, although it’s the last trial before commercialization.

Matt Margolis:            On April 30th, you received a $3.1 million grant from the Israeli government.

Dr. Bill Prather:           That is a grant that we get actually every year from the Israeli government, and it is derived from 40% of the research dollar that we spent in Israel. So you know, it goes up every year.

Matt Margolis:            Experts have actually picked you as a sleeping giant among companies that are similar in size as well as in therapeutic effort. So just curious if you could go into a little bit about what opportunities lie ahead for Pluristem. How close are you to meeting capital goals? What strategies you will venture into to build shareholder value?

Dr. Bill Prather:           We have about $35 million in cash in the bank right now. Assuming we’re going to embark upon the trials that I mentioned. We’re starting trial in Germany and muscle injury Phase II trial, and then first of all, we’re going to be doing in the area of cardiac ischemia, cardiac disease, and then this radiation work. So we have enough money to about 2014 starting all of those activities.

Matt Margolis:            That was a company snapshot of Pluristem Therapeutics with senior vice-president of corporate development, Dr. William Prather. Pluristem trades in the NASDAQ under the symbol PSTI. This is Matthew Margolis signing off.

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