EMD Serono announced the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug application (IND) for M5049 for the treatment of patients with Covid-19 pneumonia. The Company will initiate a Phase II randomized, controlled clinical study evaluating the safety and efficacy of M5049 in this patient population. M5049 is a potentially first-in-class small molecule that blocks the activation of Toll-like receptor (TLR)7 and TLR8, two innate immune sensors that detect single-stranded (ss) RNA from viruses such as SARS-CoV-2, the virus responsible for Covid-19. Activation of TLR7/8 leads to immune cell activation and inflammation, which when not properly controlled can cause severe immunopathology.

The aim of the study is to investigate if M5049 intervention at a critical point in the course of Covid-19 disease may prevent or ameliorate the hyper-inflammatory response in patients with Covid-19 pneumonia and prevent progression to 'cytokine storm'. Successful intervention with the investigational drug may reduce life-threatening complications of Covid-19, including severe respiratory symptoms that often necessitate further medical interventions such as mechanical ventilation.

"We have a clear priority to identify potential solutions across the full spectrum of the Covid-19 pandemic, including prevention, symptom management, severe infection and recovery," said Belén Garijo, CEO Healthcare and Member of the Executive Board of Merck KGaA, Darmstadt, Germany. "With M5049, we aim to study a novel approach to treating severe complications of Covid-19 that we hypothesize could translate to other single-stranded RNA viruses including other coronaviruses."

M5049 will be investigated in a randomized, placebo-controlled study at sites in the United States and Brazil. In addition to standard of care, which may include antiviral treatments, subjects will receive one of two doses of M5049 or placebo with the intent of comparing M5049 to placebo based on patient outcomes. Approximately 150 subjects will be randomized into one of the three treatment arms. In addition to the FDA, Merck KGaA, Darmstadt, Germany is working with other global health authorities and investigators to accelerate clinical development to meet the critical need presented by the current global pandemic and expects results from the Phase II study around the end of 2020.