Benjamin Kim, MD, MPhil

Vice President, Clinical Development

Today marks the official launch of Vega Therapeutics, spun out from Star Therapeutics, to bring novel, first-in-class therapies to patients with rare blood disorders, starting with von Willebrand disease (VWD).

While other bleeding disorders, such as hemophilia A, have benefited from a boom of new medicines – including antibody and gene therapies – drug innovation for VWD has lagged. At Vega, we see an equal need for VWD patients to benefit from a new era of effective therapies that reduce treatment burden for patients, and we have our sights set on fulfilling this need with our discovery and development of VGA039, the first ‘purpose-built’ antibody therapy for the treatment of VWD.

People living with VWD either have defective or low levels of von Willebrand factor, a protein that helps blood clot. The resulting bleeding can be severe and can cause organ damage and impact quality of life. Like hemophilia A, VWD is a genetic disorder in which the blood does not clot properly. Unlike hemophilia A, though, which predominantly impacts males, VWD is a disease with equal prevalence among males and females. As a result of heavy menstrual bleeding, girls and women with VWD often times develop anemia and may experience debilitating fatigue and pain, to the extent where they are unable to participate in school, work, or other routine pursuits of daily life. During pregnancy and following childbirth, women with VWD have a 10-fold higher rate of maternal mortality compared to other women.¹

Shifting the paradigm beyond conventional therapy

I’m a physician who has cared for patients with bleeding disorders for over 15 years, so I know and respect the innovative success of treating individuals with hemophilia and bleeding disorders with clotting factor replacement therapies for many decades now. I also know from my experience working on the clinical development of Hemlibra^® – an antibody for hemophilia A that was discovered by scientists at Chugai and is marketed by Chugai/Genentech/Roche – that a subcutaneous antibody therapy can be truly transformational for patients.

I have seen first-hand how hemophilia A patients’ lives have dramatically benefited from a less frequent and more convenient treatment. Instead of constantly thinking about their disease and next treatment, sometimes on a daily basis, such therapies allow for routine treatments every 1 to 4 weeks. As one of the many scientists and clinicians who worked to enable Hemlibra^® to become an approved and widely used product, it is gratifying to know that there are many people who can benefit from novel, differentiated, and effective therapies.

Seeing the reality of a therapeutic antibody for prophylaxis against bleeding associated with hemophilia A has inspired us at Vega to achieve the same for VWD. We have discovered and developed VGA039, a first-in-class antibody therapy that represents a novel approach to prevent and reduce bleeding in VWD. By promoting thrombin generation through targeting Protein S, VGA039 is mechanistically well suited to overcome the unstable clot formation that results in bleeding in VWD, and as a subcutaneously self-administered antibody therapy, has potential to transform VWD treatment.

Understanding the treatment needs of people living with VWD

With the launch of Vega Therapeutics and with VGA039 poised to move into clinical trials, we are bringing therapeutic innovation and advancing new possibilities for people living with VWD. Current VWD treatment options have significant limitations. They may have minimal effectiveness or are associated with high treatment burden. Factor replacement therapies, in particular, require frequent intravenous infusions. Many girls and women with VWD have resorted to taking hormonal contraception, which may resolve some symptoms of heavy menstrual bleeding but carries with it several side effects; for some, it simply may not be a viable treatment choice. Other treatments, such as anti-fibrinolytic agents or desmopressin, may not be suitable to prevent other types of bleeding, including spontaneous joint bleeds, in those who have high unmet need for prophylactic therapy.

With its novel mechanism of action, subcutaneous delivery, and long half-life, we believe VGA039 has the potential to be an effective, convenient therapy that dramatically reduces the treatment burden for people living with VWD. At Vega, we’re looking to chart a better course for patients with underserved bleeding disorders and are working with great urgency to advance VGA039 and bring innovation to the VWD community.

¹James AH and Jamison MG. J Thromb Haemost. 2007;5(6):1165-9.