United Therapeutics is paying Pluristem Therapeutics $7 million up front for an exclusive worldwide license to use the latter’s placental expanded (PLX) cells in the development and commercialization of a cell-based product for treating pulmonary hypertension. Pluristem could earn up to an additional $48 million in milestone and other payments, and reimbursement for development and clinical costs. The firm retains all manufacturing rights to the cells and will participate in preclinical and clinical trials activities. United will shoulder all costs associated with clinical trials for the pulmonary hypertension indication and will purchase the commercial and clinical trials supplies from Pluristem.
“This is an important milestone for our company, as it exemplifies our belief that Pluristem’s PLX cells are a platform technology that can be used for the treatment of numerous diseases,” remarks Zami Aberman, Pluristem chairman and CEO. “This agreement is in line with our strategy of being a state-of-the-art cell manufacturer while maintaining all the production and IP rights for future product candidates.”
Israel-based Pluristem is focused on the development of placenta-based cell therapies utilizing the PLX technology. PLX cells are mesenchymal-like adherent stromal cells (ASCs) derived from full-term placenta. The cells are expanded in a bioreactor system that provides a 3-D microenvironment enabling full control over the manufacturing process, large-scale growth of the cells, and batch to batch consistency, the firm claims. The resulting off-the-shelf products require no tissue matching or immune-suppression treatment prior to administration.
Pluristem has already completed Phase I studies in the U.S. and Germany with its lead product, PLX-PAD, for the treatment of peripheral artery disease (PAD) patients. In April the firm reported positive six-month follow up results from the two Phase I trials in critical limb ischemia patients. The data showed PLX-PAD was safe, improved quality of life, and demonstrated early indications that treatment reduced amputations. Among the 27 patients treated with PLX-PAD, only one amputation was recorded, representing a 3.7% amputation rate. Pluristem maintains that this represents a 75% reduction in amputation rate compared with historical data, which varies from 20–40%.
The firm says feedback from the EMA and FDA as a result of the Phase I studies means it is now preparing for a multinational Phase II/III study evaluating PLX-PAD against critical limb ischemia in the EU and U.S., and a Phase II study in patients with intermittent claudication in Germany, the U.S., and Israel. The PLX-PAD product is also in preclinical development for the treatment of diabetic foot ulcers. Pluristem’s earlier-stage pipeline includes preclinical-stage candidates for the treatment of skeletal injuries, inflammatory bowel disease, multiple sclerosis, neuropathic pain, ischemic stroke, and an adjuvant for umbilical cord blood transplantation.