26 June 2020 - - US-based specialty pharmaceutical company Citius Pharmaceuticals, Inc (NASDAQ: CTXR) has received a written response from the US Food and Drug Administration in regards to its pre-investigational new drug application for its induced mesenchymal stem cells (iMSCs) to treat and reduce the severity of acute respiratory distress syndrome in patients with COVID-19, the company said.

The FDA acknowledged that the company could apply for fast track designation and also provided Citius with the chemistry, manufacturing, and control requirements for the proposed trials.

The company plans to initiate actions on the FDA's recommendations and follow up with the FDA with an Investigational New Drug application under the Coronavirus Treatment Acceleration Program.

Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care.

Citius's mesenchymal stem cell therapy product is derived from a human induced pluripotent stem cell line generated using a proprietary mRNA-based (non-viral) reprogramming process.

The iMSCs produced from this clonal technique are differentiated from adult donor-derived MSCs (bone marrow, placenta, umbilical cord, adipose tissue, or dental pulp) by providing genetic homogeneity.

In in-vitro studies, iMSCs exhibit superior potency and high cell viability.

The iMSCs secrete immunomodulatory proteins that may reduce or prevent pulmonary symptoms associated with acute respiratory distress syndrome in patients with COVID-19. The Citius iMSC is an allogeneic (unrelated donor) mesenchymal stem-cell product manufactured by expanding material from a master cell bank.

Acute Respiratory Distress Syndrome is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs.

ARDS is a rapidly progressive disease that occurs in critically ill patients most notably now in those diagnosed with COVID-19.

ARDS affects approximately 200,000 patients per year in the US, exclusive of the current COVID-19 pandemic, and has a 30% to 50% mortality rate. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease).

Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

In response to the pandemic, the FDA has created an emergency program called the Coronavirus Treatment Acceleration Program to accelerate the development of treatments for COVID-19.

By redeploying staff, the FDA is responding to COVID-19-related requests and reviewing protocols within 24 hours of receipt. The FDA said CTAP "uses every available method to move new treatments to patients as quickly as possible, while at the same time finding out whether they are helpful or harmful."

In practice, that means developers of potential treatments for COVID-19 would benefit from an unusually faster track at the FDA to shorten wait times at multiple steps of the process.