Two Israeli biomedicine firms received US Food and Drug Administration (FDA) approval for separate trials in the US with their respective solutions for COVID-19, the companies announced over the weekend.
Both RedHill BioPharma, a publicly-traded specialty biopharmaceutical company, and Pluristem Therapeutics, also a public company that specializes in placental cell therapy, reported on Friday that they were given the green light for their imminent separate studies.
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RedHill BioPharma said its Investigational New Drug (IND) application for a Phase IIa clinical study evaluating its investigational drug, opaganib, in patients with confirmed moderate-to-severe SARS-CoV-2 infection, has been given approval and expects to begin the randomized, double-blind, placebo-controlled trial with up to 40 patients.
Opaganib was used to treat some patients with COVID-19 in Israel as part of a compassionate use program, a treatment option that allows for the use of not-yet-authorized medicine for severely ill patients. Preliminary findings in April demonstrated clinical improvement in two trials with a total of six patients. The pharmaceutical was also tested previously in Phase I and Phase II clinical studies in oncology indications, in pharmacokinetic studies in healthy volunteers in the US, and under the existing FDA-approved expanded access requests from physicians for individual oncology patients.
The preliminary goal of the upcoming COVID-19 study will be to evaluate the reduction in total oxygen requirement for patients over the course of treatment for up to 14 days, RedHill said in a statement. Secondary goals include a 50 percent reduction in oxygen requirements, looking at the proportion of patients without fever after 14 days of treatment, and the proportion with negative nasal swabs at Day 14.
Dr. Mark L. Levitt, medical director at RedHill, said the company “was grateful to the FDA for the timely review of our IND and look forward to initiating the study. There is a strong scientific rationale for the potential efficacy of opaganib in the treatment of COVID-19, including pre-clinical data demonstrating that opaganib may inhibit viral replication and reduce levels of IL-6 and TNF-alpha, important mediators of inflammation that are elevated in moderate-to-severe COVID-19 patients.”
“This is coupled with encouraging preliminary data from the compassionate use program in Israel, which demonstrated objective measurable clinical improvement in all six patients analyzed, including a decrease in required supplemental oxygenation, higher lymphocyte counts, and decreased CRP levels,” he added.
Dr. Kevin Winthrop, professor of Infectious Diseases and Public Health at the OHSU-PSU School of Public Health in Oregon will lead the RedHill study.
“We are pleased to offer opaganib to hospitalized patients as part of a clinical study and are hopeful to meet the strong unmet need for treatments to decrease the severity and duration of respiratory symptoms due to COVID-19,” he said.
Pluristem Therapeutics, meanwhile, also received FDA Investigational New Drug (IND) clearance for a Phase II study of its PLX cells to treat severe COVID-19 cases complicated by Acute Respiratory Distress Syndrome (ARDS).
Pluristem develops placenta-derived cell therapy to treat various medical conditions such as infections, inflammation, ischemia (inadequate blood supply to organs), and muscle injury. The company recently said its PLX cell therapy may potentially be used to treat COVID-19 patients with pneumonia and pneumonitis.
The study, a “randomized, double-blind, placebo-controlled, multicenter, parallel-group” trial, will treat 140 adult patients who are intubated and mechanically ventilated and are suffering from respiratory failure and ARDS due to COVID-19. The objective is to evaluate the efficacy and safety of one or two intramuscular (IM) injections, in three different dosages, of PLX-PAD for the treatment of ARDS resulting from COVID-19, the company said in the announcement.
The goal is to see the effect on the number of ventilator-free patients over the 28 days of the study, Pluristem added.
In April, the first patient suffering from COVID-19 complications was treated with the therapy in the US under the FDA’s Single Patient Expanded Access Program (the compassionate use program). The patient was treated with PLX cell therapy at Holy Name Medical Center in New Jersey, an acute care facility that is currently an active site for Pluristem’s Phase III critical limb ischemia (CLI) study. Prior to treatment, the patient was critically ill with respiratory failure due to acute respiratory distress syndrome (ARDS) and was under mechanical ventilation in an intensive care unit for three weeks, according to the company. Pluristem previously treated seven COVID-19 patients in Israel with severe complications under the compassionate use program, with preliminary results showing a 100 percent survival rate, according to the company. In addition, four of the seven patients (66 percent) showed respiratory improvement, and three of the seven were able to start being weaned off ventilators.
A Clinical Trial Authorization (CTA) has also been filed in Europe for a Phase II COVID-19 trial, with the first European clinical sites planned in Germany and Italy, Pluristem said on Friday.
“We are very pleased to gain clearance to commence our Phase II COVID-19 study in the US. We are shifting gears now with a main focus on a rapid initiation of the clinical trial, leveraging our technological and logistical competitive advantages developed through our clinical trial experience in the US and Europe,” said Pluristem CEO and President Yaky Yanay.
Yanay said the company expects to complete enrollment quickly and to provide guidelines on the expected study duration a few weeks following the commencement of the study.
“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. We look forward to working with hospitals and physicians on a larger scale to deliver our PLX cells, through an off-the-shelf, easy to use PLX cell product candidate, which may potentially accelerate recovery time from life-threatening conditions, and to improve survival, in the most severe COVID-19 cases,” he said.
Pluristem is set to receive $54 million in funding as part of an agreement with the European Investment Bank in collaboration with the Israel Innovation Authority, signed last month