Scientists are exploring whether reducing plasminogen activity can help control bleeding in people with inherited bleeding disorders.
The findings showed no evidence of an increased risk for arterial thrombosis, addressing one of the key safety questions surrounding the novel treatment strategy.
These findings bode well for the safety of ALN-6400 in patients with bleeding disorders.”The encouraging safety data come as ALN-6400 is being tested in patients with two inherited bleeding disorders that can have a significant impact on quality of life.
Current treatment options for these conditions are limited, and many patients require repeated interventions or multiple medications to control bleeding.
The ongoing phase 2 trials will provide additional information about its effectiveness and long-term safety for patients with bleeding disorders.
A new experimental therapy designed to treat bleeding disorders may offer a safer approach by reducing bleeding without increasing the risk for dangerous blood clots, according to findings from the phase 2 AXIOMATIC-TKR study. Data from this study were presented by researchers at the International Society on Thrombosis and Haemostasis (ISTH) Congress.
The research focused on ALN-6400, an investigational therapy that lowers levels of plasminogen, a protein involved in breaking down blood clots. Scientists are exploring whether reducing plasminogen activity can help control bleeding in people with inherited bleeding disorders. However, because plasminogen also plays a role in the body’s clotting system, researchers have been concerned that lowering its level could increase the risk for harmful arterial or venous blood clots.
The new findings suggest that this may not be the case.
Researchers found that lowering plasma plasminogen levels did not increase the risk for arterial thrombosis, providing encouraging evidence that the treatment may be able to reduce bleeding while maintaining an acceptable safety profile.
The study examined the effects of reduced plasminogen levels on thrombosis, focusing on whether the therapy might inadvertently promote clot formation. The findings showed no evidence of an increased risk for arterial thrombosis, addressing one of the key safety questions surrounding the novel treatment strategy.
Lead Investigator Jeffrey Weitz, MD, professor of medicine and biochemistry and biomedical sciences at McMaster University, executive director of the Thrombosis and Atherosclerosis Research Institute, and president of Council for the International Society on Thrombosis and Haemostasis, told Heme Today that the results are reassuring as ALN-6400 continues to advance through clinical development.
“A concern with reducing the plasma level of plasminogen is that this could increase the risk of arterial and venous thrombosis. Our study suggests that low plasma levels do not increase the risk of arterial thrombosis. These findings bode well for the safety of ALN-6400 in patients with bleeding disorders.”
The encouraging safety data come as ALN-6400 is being tested in patients with two inherited bleeding disorders that can have a significant impact on quality of life.
“ALN-6400 is currently being evaluated in two phase 2 clinical trials in patients with bleeding disorders—one in patients with hereditary hemorrhagic telangiectasia and another in women with von Willebrand disease who experience heavy menstrual bleeding,” added Dr. Weitz. “The findings from this study support the safety of this approach.”
Hereditary hemorrhagic telangiectasia, or HHT, is a rare genetic disorder that causes fragile blood vessels, leading to frequent nosebleeds and bleeding in other parts of the body. Von Willebrand disease is the most common inherited bleeding disorder and, in many women, can cause heavy menstrual bleeding that affects daily life and may lead to anemia.
Current treatment options for these conditions are limited, and many patients require repeated interventions or multiple medications to control bleeding. A therapy that safely reduces bleeding without increasing the risk for dangerous blood clots would represent an important advance.
Although the results are promising, ALN-6400 remains an investigational therapy and has not yet been approved for clinical use. The ongoing phase 2 trials will provide additional information about its effectiveness and long-term safety for patients with bleeding disorders.
If larger clinical studies confirm these findings, ALN-6400 could become a novel treatment option for patients living with chronic bleeding disorders, offering a new approach that targets the underlying biology of bleeding while avoiding one of the greatest concerns associated with altering the body’s clotting system.
