New Hope for Von Willebrand Disease: Biotech Innovation Gains Momentum with Star Therapeutics Leading the Charge.

Von Willebrand disease (VWD), a commonly inherited bleeding disorder affecting approximately 1% of the global population, is stepping into the biotech spotlight thanks to advancements by companies like Star Therapeutics, Chugai, and Band Therapeutics. Earlier this week, Star Therapeutics received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its novel therapeutic candidate, VGA039, a development that could mark a turning point for patients who have long lacked innovation in care.

Despite being widespread, VWD has historically been underrepresented in drug development efforts. “While other bleeding disorders, such as hemophilia, have benefitted from new therapies, von Willebrand disease has seen relatively little progress,” said Adam Rosenthal, CEO of Star Therapeutics.

Von Willebrand disease stems from a deficiency or abnormality of von Willebrand factor (VWF), a critical protein that supports blood clotting by enabling platelet adhesion and stabilizing clotting factor VIII. Patients may present with symptoms ranging from nosebleeds and bruising to severe bleeding episodes, especially in cases of type 3, the most serious form. Types 1 and 2 are generally milder but still significantly impact daily life.

Current treatments vary based on disease severity. Desmopressin, a synthetic hormone that promotes VWF release, is often prescribed for milder cases. However, its effectiveness is inconsistent and limited, especially in type 3 VWD. For more severe or unresponsive cases, patients rely on plasma-derived or recombinant VWF concentrates. Vonvendi, the only recombinant VWF currently approved, provides both on-demand and preventive treatment. Adjunct therapies like antifibrinolytics and hormonal treatments can also support bleeding control, particularly for women.

Nevertheless, treatment remains burdensome. Most therapies require intravenous infusions several times a week, limiting patient adherence and quality of life. “VWD’s complexity, with its multiple types and subtypes, has made it a challenging target for drug developers,” Rosenthal noted.

Star Therapeutics hopes to change this with VGA039, a first-in-class antibody that targets protein S, a component involved in thrombin generation and clot formation. The drug’s unique mechanism seeks to restore balance in the coagulation process. Unlike existing intravenous treatments, VGA039 is administered subcutaneously, a potentially more convenient alternative.

Clinical data from the VIVID 2 study presented at the 2024 American Society of Hematology (ASH) meeting showed that a single subcutaneous dose of VGA039 sustained therapeutic drug levels for weeks. Patients with high baseline bleeding saw their annualized bleeding rates drop by 75% to 88%—comparable to current treatments but with fewer infusions.

Rosenthal also hinted at VGA039’s potential beyond von Willebrand disease, given its mechanism’s relevance to other bleeding disorders.

Meanwhile, other companies are also pushing forward. Chugai Pharmaceutical, part of the Roche Group, is exploring emicizumab—a bispecific monoclonal antibody initially developed for hemophilia A. Emicizumab facilitates clotting by linking activated factors IX and X, mimicking factor VIII. Early data suggests it could be effective in patients with type 3 VWD. A 2024 ASH report detailed two such patients who experienced fewer bleeding episodes during a six-month regimen, and broader clinical trials are now underway.

Band Therapeutics, a subsidiary of Guardian Therapeutics, is advancing BT200, a pegylated aptamer that binds to the A1 domain of VWF. This interaction reduces VWF clearance, boosting plasma levels of VWF and factor VIII. BT200 is furthest along in phase 3 trials for type 2B and is in phase 2 for types 1 and 3.

Other promising avenues include platelet-inspired nanoparticles, designed to mimic natural platelet function and improve clot formation with fewer side effects and less frequent dosing.

In December 2023, the FDA expanded its approval of Octapharma’s VWF concentrate, making it the first therapy cleared for prophylactic use across all VWD types.

With new therapies on the horizon, from subcutaneous antibodies to nanotech innovations, the outlook for von Willebrand disease patients is finally starting to brighten.

Source:https://www.labiotech.eu/trends-news/von-willebrand-disease-treatment-advancements/

This is non-financial/medical advice and made using AI so could be wrong.