Pluristem Phase I for Hypertension
Pluristem Therapeutics Inc. (Nasdaq:PSTI) (TASE:PLTR) announced today that United Therapeutics Corporation has received approval to perform a human Phase I study in Australia using Pluristem’s PLacental eXpanded (PLX-PAD) cells in patients diagnosed with Pulmonary Arterial Hypertension (PAH). United Therapeutics and Pluristem have a licensing agreement under which United Therapeutics is developing Pluristem’s PLX-PAD cells for PAH.
PAH is characterized by abnormally high blood pressure in the arteries of the lungs and leads to an increased workload on the right side of the heart.
United Therapeutics plans to conduct a single center, open-label, dose-escalation study in patients diagnosed with PAH. The study will enroll up to nine patients in three dosing levels with PLX-PAD cells administered intravenously (IV). The primary endpoint of the study is to evaluate the safety of PLX-PAD cells in a follow-up period of one year. Secondary efficacy endpoints at six weeks post-treatment are designed to assess cardio-respiratory function and include six-minute walk distance, cardiac hemodynamic parameters via a right heart catheterization and echocardiogram and pulmonary metabolism via a positron emission tomography (PET) scan.
Under the terms of Pluristem’s licensing agreement with United Therapeutics Corporation, United Therapeutics made an upfront payment of $7 million to Pluristem. Pluristem is also eligible to receive regulatory milestone and other payments totaling up to an additional $48 million with United Therapeutics bearing all the costs of conducting all of the clinical trials for the indication of PAH. The licensing agreement also provides that, following commercialization, United Therapeutics will purchase the PLX-PAD cell product from Pluristem at a specified margin over Pluristem’s cost. In addition, United Therapeutics will pay Pluristem specified royalties as a percentage of its gross profits generated from the sale of the PLX-PAD cell developed product.
Preclinical studies to evaluate the hemodynamic effects from the IV administration of PLX-PAD cells in the monocrotaline (MCT) rat model of PAH have been performed. These studies demonstrated that PLX-PAD cell treatment can lead to improvements in right ventricular systolic pressure (RVSP) compared to placebo-treated animals. Some treatment groups also demonstrated an improvement in right ventricular hypertrophy (RVH) as compared to placebo-treated animals.