- Thinly traded Ablynx (OTCPK:ABLYF +23.5%) heads north on a healthy 29x surge in volume in response to its announcement of positive results from a Phase 3 clinical trial, HERCULES, assessing caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood disorder characterized by the formation of blood clots in small blood vessels.
- The study met its primary endpoint and key secondary endpoints. Patients in the treatment group experienced a 74% reduction in the risk of aTTP-related death, recurrence of aTTP or at least one major thromboembolic event during the study period (p<0.0001) compared to placebo. The proportion of patients whose aTTP recurred during the treatment period was 67% lower in the caplacizumab arm compared to the control arm (p<0.001).
- Caplacizumab's safety profile was consistent with earlier studies. One patient in the treatment arm died during the follow-up period, but the death was determined to be unrelated to the study drug.
- Orphan Drug-tagged caplacizumab is a bivalent anti-von Willebrand (vWF) factor Nanobody that blocks the interaction of ultra-large vWF proteins with platelets which inhibits the formation of microclots.
- The company's marketing application in Europe is currently under review. A U.S. application is on tap for next year.
- Previously: FDA Fast Tracks Ablynx's caplacizumab for rare blood disorder (July 26)
- Now read: Your Daily Pharma Scoop: Alexion's Canada Pricing, Zogenix, RedHill, Flexion
Original article