“To be able to claim a paradigm change as significant as we saw with monoclonal antibodies 20 years ago, we need to bring something significant to the market. For something truly significant, we realized we would have to build it ourselves.”
Having said that, Zami Aberman, the CEO of Pluristem Therapeutics, sets the central tenets of his vision. First is a rock solid conviction that to control the quality of a cell therapy, Pluristem must tightly control the manufacturing processes. Today in Haifa, Israel there is a USD 30 million state-of-the-art, custom-built facility that will either stand as a monument to his passion, or as the starting point for what he believes will be one of the world’s leading pharmaceutical companies.
Zami Aberman, CEO, Pluristem.
Which leads to his second conviction, that Pluristem is not another biotech startup looking for an exit but a company that will grow to join Celgene, Amgen, Genentech and Genzyme in the pantheon of pharmaceutical industry successes.
“I realize it sounds very ambitious,” he admitted. “We are pioneering in this space and came to the conclusion that from Day One we would have to develop all the manufacturing tools that will enable us to control precisely the process. Since 2006 we have done a lot of work, step by step, to build the bioreactor, as well as all the downstream operations, including a dedicated thawing device to make sure when the cells are to be used at the patient bedside, the quality is exactly what is needed.”
Pluristem can claim an industry-first accomplishment for its ability to draw cells from two different donors and through its processes manufacture a final product where the cells are identical in their properties.
“It is only today through the outcome of clinical trials we are realizing we may have been right to do all of this,” he said.
In January of this year, Pluristem announced top-line results from its Phase I/II clinical trial in the treatment of muscle injury. The PLacental eXpanded (PLX) cells proved to be safe and a statistical significance (p=0.0067) was reached for the primary efficacy endpoint of the change in maximal voluntary isometric contraction force of the gluteal muscle at six months after total hip replacement.
“The improvement was 500% in muscle force after six months, which is an objective measure,” said Aberman, adding that “MRI of the muscle showed the volume of the treated patients was significantly increased in correlation with the outcome of the study.”
“We are taking a different angle in personal medicine,” he said. “Cells are adaptive creatures. By this I mean they send and receive signals, for example regarding the inflammatory situation of a specific patient in indications like peripheral artery disease (PAD) and ischemic stroke. Based on the signals they receive , they change the secretion profile. We have documented that the PLX cells can adapt themselves to a patient’s condition.”
“This is not to say the Pluristem cells can do everything, and the same cells cannot treat all conditions,” he added. Yet the potential indications PLX cells can address is expanding rapidly. Clinical trials include testing treatment for cardiovascular, orthopedic and pulmonary diseases. Preclinical programs are exploring applications in women’s health, hematology and sports medicine.
Also in January the Haifa facility, with its proprietary 3D cell expansion platform, received approval from the German health authority, the Paul-Ehrlich-Institute, enabling the company to support ongoing trials with its placental-derived mesenchymal-like adherent stromal cells (ASCs). The capacity of the scaled-up capabilities is estimated to be over 150,000 doses annually.
“The question now is how all this may influence the pharma business model,” Aberman said. “The only way to assure the supply of high quality, stable cells, and to protect our interests, is to keep the manufacturing rights. This is a completely different model.”
“We know we can reach agreements with pharma partners, as we did with United Therapeutics, or CHA Bio&Diostech for the South Korean market,” he said. “Yet the real proof will come when one of the very large companies will agree to this business model.”
Pluristem is not interested in building an infrastructure for marketing its cell therapies but providing pharma partners with a final product in a vial, ready to use.
“Today we have documented a one-year stability for our product and we believe we will get stability to four years. These products can be shipped to the pharma company who can store it until it is needed. They would handle the distribution and logistics needed to bring it to the hospital. If we were to try to build this ourselves, we would spend all our funding on marketing and distribution networks instead of developing products for new indications and new partners.”
Aberman said he has already booked a string of meetings at BIO-Europe Spring® in Turin.
“Our bread and butter is discovering new indications, so our first interest is a pharma company willing to explore the possibilities of cell therapy products for a specific indication, much like United did,” he said. “A second type of partner would be an organization seeking a reliable cell source for a new indication that could lead to clinical studies as soon as possible. In the cell therapy industry there is a tremendous difference between structuring cells for trials with 10 to 20 patients, and then scaling up to a few hundred patients. From our facility we can consistently provide the same cells for initial studies, and five years later when they are in Phase III, we can provide exactly the same cells.”
“We are less interested today to partner on new ideas,” said Aberman. “Many people have new therapies, new manufacturing processes, new cell types. At this point in our development we need to concentrate on bringing pharma partners to the table, educating them about our technology and our unique business model.”
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