Citius Pharmaceuticals has made a Pre-IND submission to the US Food and Drug Administration for a novel stem cell therapy for acute respiratory distress syndrome (ARDS) in COVID-19 patients.

In this regard, the late-stage specialty pharma company submitted a pre-IND meeting request and supporting briefing documents to the FDA’s Center for Biologics Evaluation and Research (CBER) under the Coronavirus Treatment Acceleration Program (CTAP).

Citius Pharmaceuticals is seeking the feedback of CBER to support the most expeditious pathway into the clinic to assess the stem cell therapy in patients with COVID-19-related ARDS.

The cells intended to be used in the clinical trial, which are called NoveCite Cells or NC-MSCs are made by Cambridge-based biotechnology company Novellus by using its mRNA-based cell-reprogramming process.

NoveCite Cells are mesenchymal stem cells extracted from the fibroblasts of single donors. These cells have been dedifferentiated into an induced pluripotent stem cell (iPSC) master cell bank, thereby avoiding the necessity to procure additional donor cells. The iPSCs are then differentiated further into a mesenchymal stem cell (MSC) therapy.

Citius Pharmaceuticals and Novellus are looking to develop NoveCite Cells for the treatment of ARDS, and last month the firms signed an exclusive option agreement in this regard.

The specialty pharma company is planning to launch a multi-center randomized placebo-controlled dose-finding trial followed by an expansion phase to evaluate the safety, tolerability, and efficacy of NoveCite Cells in patients having moderate to severe ARDS due to COVID-19.

Myron Holubiak – CEO of Citius Pharmaceuticals said: “We believe we have the key elements in place from a clinical design and manufacturing point of view to evaluate this novel cell therapy approach to deal with the current pandemic.

“ARDS is a very serious complication for many patients suffering from COVID-19, and is believed to account for about 80% of the deaths in ventilated patients. There is no proven or FDA-approved treatment for it, other than oxygen therapy, including use of mechanical ventilation, and fluid management.

“Literature from previous investigational studies with MSCs in the treatment of lung injuries support the idea that MSCs could prove effective in treating COVID-19-related ARDS. We look forward to our FDA discussions and are excited to be at the cusp of what could be a novel and effective therapy for ARDS.”

The proposed phase 1b/2 clinical trial, to be called “A Randomized Placebo-Controlled Dose-Finding Study Followed by a Dose Level Expansion to Assess the Safety and Efficacy of NoveCite MSCs in Subjects with Acute Respiratory Distress Syndrome (ARDS) Due to SARS-CoV-2 Disease (COVID-19),” or simply called “MARCO”.

The main objectives of the MARCO clinical trial are to assess the safety and efficacy of NoveCite cells as a treatment for patients having moderate-to-severe ARDS due to COVID-19 and also for identifying therapeutic doses.

Matt Angel – co-founder and Chief Science Officer at Novellus said: “MSCs have an established track-record of clinical safety, and have shown promise in the treatment of inflammatory lung disease.

“Our research has shown that the NoveCite cells, being derived from mRNA-reprogrammed iPSCs, secrete higher levels of immunomodulatory proteins than donor-derived MSCs, and have unique manufacturing advantages.”

  acute respiratory distress syndrome, Citius Pharmaceuticals, Coronavirus, COVID-19, Myron Holubiak, Stem cell, stem cell therapy, US Food and Drug Administration