Israel’s Pluristem Therapeutics’ PLX cells therapy has been cleared by the US Food and Drug Administration for a Phase II study in the treatment of severe COVID-19 cases complicated by Acute Respiratory Distress Syndrome.
The study, “A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Parallel-Group Phase II Study to Evaluate the Efficacy and Safety of Intramuscular Injections of PLX-PAD for the Treatment of severe COVID-19,” will treat 140 adult patients that are intubated and mechanically ventilated and are suffering from respiratory failure and ARDS due to COVID-19.
“We believe we can complete enrollment quickly and we expect to provide guidelines on the expected study duration a few weeks following the commencement of the study,” said Pluristem CEO and president Yaky Yanay. “In the last few weeks, we have received dozens of applications from physicians and families seeking to participate in the Expanded Access per patient program.”
He said that Pluristem believes its PLX cells will offer a key advantage in addressing the COVID-19 global pandemic.
The primary efficacy endpoint of the study is the number of ventilator free days during the 28 days from day 1 through day 28 of the study.
Until now, Pluristem has been treating patients suffering from severe complications caused by COVID-19 in the US and Israel through compassionate use programs.
On April 12, the company reported that six critically ill coronavirus patients at three different medical centers in Israel who were considered high-risk for mortality had been treated with Pluristem’s placenta-based cell-therapy product and survived.
The patients were suffering from acute respiratory failure and inflammatory complications associated with COVID-19. Four of the patients also demonstrated failure of other organ systems, including cardiovascular and kidney failure. Not only did all the patients survive, according to Pluristem, but four of them showed improvement in respiratory parameters and three of them are in the advanced stages of weaning from ventilators. Moreover, two of the patients with preexisting medical conditions are showing clinical recovery in addition to the respiratory improvement.
The company treated its first COVID-19 patient in the United States on April 14.
A Clinical Trial Authorization has also been filed in Europe for a Phase II COVID-19 trial, with the first European clinical sites planned in Germany and Italy. Moreover, in late April, Pluristem secured $54m. in financing from the European Union's European Investment Bank (EIB) to advance its regenerative cell therapy platform, with a focus on the clinical development of a placenta-based treatment for complications associated with COVID-19.
Yanay said that he believes the company’s off-the-shelf, easy to use PLX cell product candidate, “may potentially accelerate recovery time from life threatening conditions, and to improve survival, in the most severe COVID-19 cases.”
The objective of the study is to evaluate the efficacy and safety of one or two intramuscular injections, in three different dosages, of PLX-PAD for the treatment of ARDS resulting from COVID-19. The primary endpoint determination will be performed at the end of the 28 day main study period. Safety and survival follow-up will be conducted at week eight, 26 and 52.
Pluristem’s PLX cells are “allogeneic mesenchymal-like cells that have immunomodulatory properties,” meaning they induce the immune system’s natural regulatory T cells and M2 macrophages, the company explained in a previous release. The result could be the reversal of dangerous overactivation of the immune system. This would likely reduce the fatal symptoms of pneumonia and pneumonitis (general inflammation of lung tissue).
Previous preclinical findings regarding PLX cells, which are grown using the company’s proprietary three-dimensional expansion technology, revealed significant therapeutic effects in animal studies of pulmonary hypertension, lung fibrosis, acute kidney injury and gastrointestinal injury.