eXIthera Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application to initiate a Phase 2 clinical trial for EP-7041, a parenteral, potent, and selective small-molecule inhibitor of coagulation Factor XIa. The IND application indicates EP-7041 is an investigational treatment for COVID-19, addressing the increased morbidity and mortality risk due to blood clot formation in patients who are critically ill with COVID-19.

"It has become apparent that severely ill patients with COVID-19 have increased risk of developing blood clots, which can be fatal. There is a notable medical need for safer anticoagulants to treat COVID-19 patients, which EP-7041 has the potential to meet," said Charles Pollack, Jr., MD, Medical Director eXIthera Pharmaceuticals. "We are grateful to the FDA for their quick action on our IND application, and for the opportunity to improve the lives of patients suffering from COVID-19 and its debilitating consequences."

The Phase 2 trial is a multicenter, prospective, open-label study that will first evaluate the safety and tolerability of two dose levels of EP-7041 in thirty COVID-19 patients admitted to an Intensive Care Unit (ICU). Once the appropriate dose level is determined, the second phase of the study will evaluate EP-7041's safety as reflected by the occurrence of major bleeding episodes (primary safety endpoint), as well as its efficacy in preventing major thrombotic or thromboembolic events, in sixty additional COVID-19 ICU patients randomized to receive the study drug or the institutional standard of care for thromboprophylaxis at a ratio of 2:1, respectively.

"We are enthusiastic about the opportunity to provide a new path of treatment for severe COVID-19 patients," said Neil Hayward, PhD, CEO of eXIthera. "We have all experienced the devastation of COVID-19 firsthand, and, while vaccines offer hope to curb viral spread, therapeutics will be essential to our long-term strategy in the fight against COVID-19 – especially considering the viral variants that may elude current vaccines."

EP-7041 is a small molecule inhibitor of coagulation Factor XIa, designed to reduce the risk of thrombosis while simultaneously mitigating the risk of bleeding associated with standard of care anticoagulants. This is accomplished through the unique mechanism of action of EP-7041, which selectively blocks clotting through the intrinsic (contact) pathway without impacting normal hemostasis (bleeding) through the extrinsic pathway. EP-7041 was found to be safe and well tolerated in sixty healthy volunteers following single or multiple ascending doses in a Phase 1 clinical study, and the pharmacokinetic and pharmacodynamic profile indicated desired efficacy with minimal potential for bleeding. In addition to COVID-19 patients, EP-7041 has potential to decrease the mortality risk associated with blood clotting in patients undergoing complex cardiac surgery or orthopedic surgery.