Therapeutic Focus

Glanzmann thrombasthenia (GT) is a congenital severe, lifelong bleeding disorder characterized by deficient or dysfunctional glycoprotein IIb/IIIa expression on platelets, which impairs platelet-fibrinogen binding and platelet aggregation during primary hemostasis. Patients suffer from what can be a devastating combination of life-threatening acute hemorrhages and relentless chronic bleeding that profoundly impact patients’ physical health, mental health, and quality of life.

Von Willebrand Disease (VWD) is the most common inherited bleeding disorder. It is characterized by both life-threatening hemorrhagic events and relentless mucocutaneous bleeding episodes that combine to inflict significant harm to physical health, mental well-being, and quality of life. Women face a particularly severe burden from heavy menstrual bleeding, which can profoundly impact quality of life, reproductive choices, and iron status. Even patients with Type 1 VWD, which has traditionally been considered “mild”, experience a serious clinical burden. VWD results from reduced quantity or function of von Willebrand factor (VWF), a plasma protein essential for normal hemostasis through its dual role in platelet adhesion and as a carrier protein protecting coagulation Factor VIII from degradation.

Factor VII deficiency is a congenital severe bleeding disorder characterized by reduced levels of Factor VII, a naturally circulating blood coagulation protein. Patients with clinically severe Factor VII deficiency suffer from recurrent, unpredictable, life-threatening or potentially disabling bleeding at critical sites, such as in the central nervous system, gastrointestinal tract and intra-articular locations, as well as recurrent mucocutaneous bleeds of the nose and gums with additional risks for female patients, consisting of heavy menstrual bleeding and potentially life-threatening post-partum hemorrhage.

Heavy menstrual bleeding is a common condition characterized by excessive menstrual blood loss that disrupts a person's physical, emotional, social, and daily life, affecting an estimated one in three reproductive-age women, or more than 23 million women in the U.S. alone. The condition is frequently associated with pain, iron-deficiency anemia, and fatigue, and carries a significant social burden, including stigma and substantial lost days from work and school. Current treatment options are limited. Tranexamic acid (TXA), the primary non-hormonal therapy, has a short half-life that requires frequent dosing throughout menstruation, limiting adherence. Hormonal therapies are not suitable, or not preferred, for many women. To date, there remains no approved non-hormonal, customized hemostatic treatment option for heavy menstrual bleeding.