Less than a month ago shares of Pluristem Therapeutics (NASDAQ: PSTI) were trading as low as 2.38 but with news released on Monday that the company’s placenta-derived stem cell therapy had helped restore bone-marrow function in a 54-year-old patient suffering from lymphoma who had failed to respond to bone-marrow transplant those shares have shot to a 52-week high, hitting the 4.23 mark and they are currently holding strong around the 4.06 level.
This news brought has brought considerable attention to PSTI and specifically their patented PLX (PLacental eXpanded) cells, their drug delivery platform that releases a cocktail of therapeutic proteins in response to a host of local and systemic inflammatory and ischemic diseases. Making this news all the more significant is that it builds off a similar success story involving a 7-year-old cancer patient who was treated with the same therapy after not responding to a bone-marrow transplant.
Just what kind of impact can this patented drug delivery platform make? In the words of Professor Reuven Or, Director of Bone Marrow Transplantation and Cancer Immunology at Hadassah Medical Center in Jerusalem, “This is a real breakthrough — the woman was in isolation due to low white blood cells and high susceptibility to infections and in addition her red blood cells and platelets were low, leading to a very dangerous and life-threatening situation. Further, autologous bone marrow transplantation that she received engrafted poorly, and as a last resort, we applied for a compassionate treatment using Pluristem’s PLX cells based on our previous experience with those cells. The treatment with PLX has saved her life and can certainly be classified as a medical miracle.”
Or went on to say, “The result of this unique case demonstrates that PLX cells could potentially be effective for use in cancer patients, who receive bone marrow transplantation following severe radiation and chemotherapy treatments, which severely damage their bone marrow.” What PSTI has is considered a “real breakthrough” given the fact that the bone marrow transplant procedure has been relatively unchanged for more than four decades with almost no answer for those in which the procedure fails, those like the recently treated 54-year-old patient as well as the 7-year-old cancer patient. Based on the results of these two cases PSTI could have a real alternative to blood marrow transplant in the form of intramuscular injections of their PLX cells.
PSTI has already applied for Orphan Drug status for bone marrow anemia and if granted by the FDA would give them closer guidance from the agency as well as enable them to sidestep the required 1,000 patients for a Phase III clinical trial. This could be a game-changer for PSTI as it would enable them to complete the clinical trial process in a fraction of the time while saving them considerable costs associated with clinical trials. According to PSTI the bone marrow transplant market is an estimated $1.3 billion per year in the U.S. alone, based on 30,000 bone marrow transplants in the U.S per annum.
What makes PSTI so attractive to investors is the fact that their PLX cells, grown using their proprietary 3-D micro-environmental technology and are an “off the shelf” product that requires no tissue matching prior to administration, can be used to treat a host of diseases and afflictions outside of Bone Marrow Transplant Failure and Chemotherapy induced Bone Marrow Aplasia.
Data from two Phase I studies have indicated that PSTI’s first PLX product candidate, PLX-PAD, is safe and potentially effective for the treatment of end stage peripheral artery disease when given locally. PSTI is also developing PLX-PAD for cardiac ischemia, PLX-BMP for Acute Radiation Exposure, PLX-ORTHO for orthopedic indications and PLX-PAH for Pulmonary Hypertension in collaboration with United Therapeutics (UTHR). This gives them an impressive pipeline and provides investors some protection, giving them the security of knowing that they aren’t putting all their eggs in one basket.
In addition to the above listed treatments PSTI has shown that in pre-clinical animal models that PLX cells are also potentially effective in other inflammatory and ischemic indications, including diastolic heart failure, inflammatory bowel disease, neuropathic pain, and pulmonary fibrosis.
Of course there is still a risk with PSTI considering they have only recently applied for Orphan Drug status and any negative response could do damage to their outlook. That being said, there is always a chance that the biotech could be the target of a bigger company given the fact that their pipeline has immense potential in a variety of markets.