Stemming the threat of ARDS

CRANFORD, N.J. & CAMBRIDGE, Mass.—Citius Pharmaceuticals Inc., a specialty pharmaceutical company focused on developing and commercializing critical-care drug products, recently signed an exclusive six-month option agreement to in-license a stem cell therapy for acute respiratory distress syndrome (ARDS) from a subsidiary of preclinical-stage biotechnology company Novellus Inc.

 

Novellus’ patented process uses its exclusive non-immunogenic synthetic messenger ribonucleic acid (mRNA) molecules to create induced pluripotent stem cells (iPSCs) that, in turn, generate mesenchymal stem cells (MSCs) with what the companies say are “superior immunomodulatory properties.” They add that MSCs have been shown to be safe in more than 900 clinical trials and to be effective in treating a number of inflammatory diseases, including ARDS.

 

“ARDS is the most common cause of respiratory failure and mortality in COVID-19 patients. Currently, there is no proven treatment for ARDS. Literature supports the use of counter-inflammatory MSCs for ARDS, and papers published in China have shown that at least seven COVID-19 patients with ARDS responded to MSC therapy,” said Citius CEO Myron Holubiak. “Clearly this is an avenue that shows promise and should be pursued as a potential treatment for ARDS. We believe Novellus is at the forefront of creating allogeneic, iPSC-derived MSCs. These cells have the potential to overcome the limitations of MSCs derived from adult donors, which are telomere shortened and introduce variability into the manufacturing process.”

 

Added Novellus Chief Science Officer Dr. Matt Angel: “Using our mRNA-based cell-reprogramming technology, Novellus can provide a near-unlimited supply of MSCs for treating patients with ARDS, including those critically ill from COVID-19. These will be allogeneic (“off-the-shelf”) cells that in vitro have demonstrated much greater expansion potential and much higher immunomodulatory protein expression than donor-derived MSCs. We are excited to employ our technology to such an urgent medical crisis, and believe that our MSCs represent an ideal source of cells to be used in this extremely important development effort.”

 

ARDS is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs. This rapidly progressive disease occurs in critically ill patients, most notably now in those diagnosed with COVID-19. ARDS affects approximately 200,000 patients per year in the United States, exclusive of the current COVID-19 pandemic, and has a 30- to 50-percent mortality rate. It is sometimes initially diagnosed as pneumonia or pulmonary edema.

 

“No effective pharmacotherapy for ARDS exists, and ARDS-related morbidity and mortality are high,” Holubiak added. “MSCs have been studied in the treatment of lung injury, and we aim to build upon this work with Novellus’s iPSC-derived MSCs to improve the immunomodulatory response in humans. We have assembled a team of experts who are dedicated to advancing this project to an Investigational New Drug application as quickly as possible.”