The 65th American Society of Hematology (ASH) Annual Meeting took place from December 10-13, 2022. During the meeting, stakeholders in the hematology field came together to discuss trends, research, and news related to hematology and hematological malignancies. According to a news release, clinical-stage biotechnology company Vega Therapeutics, Inc. (“Vega”) unveiled and launched VGA039, a therapy designed to treat von Willebrand disease (VWD), during the Meeting.
On December 12, 2022, Vega shared information on VGA039 in a presentation (Session 321, Abstact 691). VGA039 is a Protein S-modulating monoclonal antibody that has the potential to treat multiple bleeding disorders. During the presentation, Vega explained:
- How VGA039 fits into Vega’s pipeline of therapies designed to treat rare blood disorders
- What the preclinical data has shown so far
- VGA039 pharmacokinetics when given in different forms (ex: intravenously vs. subcutaneously)
- The potential for VGA039 to address a fundamental mechanism of blood clot formation
Additionally, Vega shared that the company had been approved to launch a Phase 1 clinical trial to begin evaluating VGA039 in a more clinical setting. Given that there is an urgent need to develop new therapies for VWD, VGA039 definitely has the potential to change the treatment landscape down the line.
An Overview of von Willebrand Disease (VWD)
Originally described in 1925 by Erik von Willebrand, von Willebrand disease (VWD) is an inheritable bleeding disorder in which the blood does not clot properly. Over 300 mutations in the VWF gene have been implicated in VWD development. These mutations cause people to have either poorly function VWF protein or VWF protein deficiency. This protein normally helps to hold platelets together as a clot to seal a hole in a blood vessel or to carry factor VIII in the bloodstream. People with VWD often bruise or bleed easily, as well as experience abnormal bleeding.
VWD may be broken into three categories. 75% of those affected have type 1, the mildest and most common form, which is characterized by low VWF levels. Next, type 2 is of medium severity and patients often have normal, but non-functioning, VWF. Both types are inherited in an autosomal dominant fashion. Alternately, VWD type 3 is the rarest and most severe form. Inherited in an autosomal recessive pattern, this form causes no VWF in the blood (or very little).
VWD is believed to affect 1 in every 100 to 10,000 people. Additionally, it is believed to be undiagnosed in up to 90% of those affected. Potential symptoms and characteristics can include:
- Easy bruising or lumpy bruising (bruises with a raised lump in the middle)
- Frequent, long-lasting, and heavy nosebleeds
- Abnormal bleeding from the gums
- Anemia (low red blood cell count)
- Bloody urine or stool
- Excessive bleeding following an injury, dental procedure, surgery, or other similar experience
- Joint pain (during joint or muscle bleeds)
- Limited movement (during joint or muscle bleeds)
- Inflammation and heat (during joint o muscle bleeds)
- Increased menstrual flow with blood clots more than 1cm in diameter
