Date: 2014-09-08
Type of information: Interim results
phase: 1
Announcement: completion of the dosing of the first cohort
Company: Pluristem Therapeutics (Israel) United Therapeutics (USA - MD)
Product: PLX-PAD Cells (Placental eXpanded cells)
Action
mechanism: PLX-PAD is a cell-based product made of allogeneic mesenchymal-like adherent stromal cells (ASCs), derived from human full-term placentas following an elective caesarean section.
Disease: pulmonary arterial hypertension (PAH)
Therapeutic area: Rare diseases
Country: Australia
Trial
details: This clinical study will assess the safety of PLX-PAD to treat pulmonary arterial hypertension. This year-long study will evaluate the safety of three different dose levels of PLX-PAD, each given as a single intravenous infusion. This study will also evaluate effects that PLX-PAD may have on PAH, such as changes in the ability to exercise and on other tests used to measure the disease severity. The study is designed to enroll 9 patients diagnosed with PAH. The first cohort of 3 patients has received 0.5 million PLX cells per kilogram body weight. An independent Data Safety Monitoring Board recommended advancement to the second cohort. The second cohort will receive 1 million cells per kilogram, while the third cohort is planned to be administered 2 million cells per kilogram. The primary endpoint of the study is the safety of PLX-PAD cells, which will be evaluated at 12 weeks and one year after dosing. Secondary efficacy endpoints are to be measured at six weeks post-treatment in order to assess changes in the ability to exercise, disease severity and cardio-respiratory function; measures include six-minute walk distance and cardio-pulmonary hemodynamic parameters evaluated via right heart catheterization and echocardiogram. (NCT01795950)
Latest
news: * On September 8, 2014, Pluristem Therapeutics, a developer of placenta-based cell therapy products, announced that its licensee, United Therapeutics Corporation, has completed the dosing of the first cohort of patients in its Phase I study using Pluristem’s PLacental eXpanded (PLX-PAD) cells in patients diagnosed with pulmonary arterial hypertension (PAH). The Phase I study, being conducted in Australia, is designed to enroll 9 patients diagnosed with PAH. The first cohort of 3 patients has received 0.5 million PLX cells per kilogram body weight. An independent Data Safety Monitoring Board recommended advancement to the second cohort. The second cohort will receive 1 million cells per kilogram, while the third cohort is planned to be administered 2 million cells per kilogram.
The Phase I study is being conducted as part of a 2011 licensing agreement between United Therapeutics and Pluristem. Pursuant to the agreement, United Therapeutics will develop, market and sell Pluristem’s PLX-PAD cells for PAH. Pluristem is eligible to receive up to $55 million based on successful achievement of clinical milestones and commercialization, and reimbursement of certain R&D costs. Following commercialization, United Therapeutics will purchase commercial supplies of PLX-PAD cells from Pluristem at a specified margin over Pluristem’s cost, and will pay royalties at a percentage of its gross profits.
* On April 29, 2013, Pluristem Therapeutics has announced that independent researchers from the Queensland Lung Transplant Service at the University of Queensland, Australia demonstrated that following infusion of Pluristem’s PLX (Placental eXpanded) cells in a human lung model of pulmonary arterial hypertension (PAH), blood flows were maintained and no adverse hemodynamic effects were noted. The findings were presented on April 25, 2013 at the 33rd Annual Meeting and Scientific Sessions of the International Society of Heart and Lung Transplantation (ISHLT) in Montreal, Canada. The presentation and poster entitled “Safety of a Mesenchymal-like Adherent Stromal Cell (PLX-PAD) in a Human Model of Pulmonary Arterial Hypertension (PAH)” are available at Pluristem’s website: http://www.pluristem.com/investors/ for the next 30 days.
Daniel C. Chambers MD et. al. from Prince Charles Hospital and the University of Queensland, Australia induced pulmonary hypertension in four human lungs that had been declined for transplantation. Supra-therapeutic doses of Pluristem’s PLX-PAD cells were then infused over 15 minutes directly into the pulmonary artery. Pulmonary vascular resistance stabilized during and for the hour post PLX-PAD infusion without adverse hemodynamic manifestations. The authors concluded they have demonstrated the acute hemodynamic safety of supra-therapeutic doses of PLX-PAD cells in an ex vivo model of pulmonary arterial hypertension.
* On February 26, 2013, Pluristem Therapeutics has announced that following favorable preclinical studies, its partner United Therapeutics intends to begin a human Phase I clinical trial in Australia using Pluristem’s PLacental eXpanded (PLX-PAD) cells in patients diagnosed with pulmonary arterial hypertension upon approval by the Australian regulatory authorities. In the proposed clinical trial, PLX-PAD cells will be injected intravenously (IV) into humans for the first time, opening potential new fields for PLX cells. On June 2011, United Therapeutics and Pluristem entered into a licensing agreement whereby United Therapeutics will develop, market and sell Pluristem’s PLX-PAD cells for PAH.
