Trevor Baglin, MD, PhD, Vice President of Centessa Pharmaceuticals UK, discusses SerpinPC, an investigational drug candidate, for the potential treatment of severe hemophilia.
Hemophilia is a bleeding disorder that slows the blood clotting process. People with this disorder experience prolonged bleeding following an injury or surgery. In severe cases, heavy bleeding occurs after minor trauma or in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. The major types of this disorder are Hemophilia A and Hemophilia B. Although the two types have very similar signs and symptoms, they are caused by genetic changes in different genes.
SerpinPC is an investigational serine protease inhibitor. Designed to increase thrombin levels caused by tissue damage, it reduces circulating activated protein C (APC). This reduction prolongs prothrombinase. Data from Part 5 of the ongoing phase 2a study of SerpinPC for the treatment of hemophilia was presented at the European Association for Hemophilia and Allied Disorders 2024 meeting. AP-0101 is an adaptive first-in-human study investigating the safety, tolerability, efficacy, and pharmacokinetics of SerpinPC.
Results from the study showed a significant reduction in the annualized bleeding rates and target joints of participants. SerpinPC also exhibited a safe and well tolerated profile with no adverse events observed in Parts 2 through 5 caused by SerpinPC. Data also showed no unexplained, sustained D-dimer elevations and pharmacokinetic parameters were consistent and stable.
Two studies are currently recruiting, one of which is focusing on hemophilia B patients with the factor IX inhibitor, and the other designed around gaining regulatory approval for SerpinPC.
SerpinPC has not been approved by the FDA or any other regulatory authority for any use.
For more information on hemophilia and other rare hematologic disorders, visit https://checkrare.com/diseases/hematologic-disorders/