Harsco Pharmaceutical(002653) announced on the evening of March 8 that its subsidiary, Sichuan Haisco Pharmaceutical Co., Ltd., recently received an Acceptance Notice from the National Medical Products Administration (NMPA) for its independently developed HSK39297 tablets for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). This drug is classified as a Class 1 chemical drug under China's new chemical drug registration classification and is a leading domestic CFB inhibitor targeting PNH.

According to the announcement, HSK39297 is a highly potent and selective small molecule inhibitor of complement factor B. It inhibits FB activity, blocking the activation of the alternative pathway and complement amplification circulation, thereby suppressing the activity of the entire complement pathway. It is designed to treat various diseases mediated by abnormal complement activation. In PNH patients, this drug acts at the proximal end of the complement cascade alternative pathway, simultaneously controlling C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis.

Clinical research results show that HSK39297 tablets are well-tolerated and safe, with good efficacy observed in different subgroups, indicating that patients in different subgroups can significantly benefit. Compared with eculizumab, this drug has significant therapeutic advantages in improving anemia, reducing transfusion requirements, and relieving fatigue symptoms, and its efficacy can be maintained long-term. The announcement stated that there is a large unmet clinical need for PNH and a promising market prospect. In addition, HSK39297 tablets are currently undergoing a Phase II clinical trial for patients with lupus nephritis, and a Phase III clinical study for primary IgA nephropathy is also being initiated.

https://finance.eastmoney.com/a/202603093665534188.html