Star injection therapy awarded FDA breakthrough status for VWD
Clinical trial testing VGA039 for bleeding prevention now recruiting in 4 countries
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Star Therapeutics‘ experimental therapy VGA039, developed to prevent bleeding in von Willebrand disease (VWD), has been granted both rare pediatric disease and breakthrough therapy status by U.S. regulators.
These designations, awarded by the U.S. Food and Drug Administration (FDA), incentivize development of medications for serious and rare conditions with unmet needs. Alongside a previous fast track designation for VGA039, they are intended to support or speed the therapy’s development. The rare pediatric disease designation particularly focuses on rare diseases that primarily affect children. If a drug is approved, the company is eligible to receive a prioity review voucher that can be redeemed for a subsequent application.
“The FDA’s decision to grant both [rare pediatric disease designation] and [breakthrough therapy designation] to VGA039 underscores the urgent need for new treatments for these patients, as well as Star’s potential to make a meaningful difference,” Gary Patou, MD, chief medical officer at Star, said in a company press release.
Star’s Vega Program designed VGA039 as a preventive, or prophylactic, treatment option for VWD. The antibody-based therapy may help people with different types of VWD and, potentially, other bleeding disorders, according to the developer.
“We are excited to continue to partner with physicians, patients and advocacy organizations to enroll people aged 12 and over with all types of VWD in our ongoing, pivotal Phase 3 study evaluating VGA039 prophylaxis,” Patou said. That trial, dubbed VIVID-6 (NCT07115004), is now recruiting particpants at 11 sites in the U.S., and one each in Georgia, Germany, and South Africa.
VWD is a bleeding disorder caused by lack of functional von Willebrand factor (VWF), a protein that helps blood form clots. Low levels of working VWF can cause episodes of abnormally heavy and prolonged bleeding.
”People living with VWD may face serious health complications, including frequent bleeding and hospitalizations that can significantly impact their quality of life,” Patou said.
VGA039 given by injection, not infusions
Factor replacement therapy, which aims to increase levels of working VWF, is the primary preventive treatment option for VWD. However, currently available treatments require regular intravenous, or into-the-vein, infusions, which can be burdensome for some patients.
VGA039, instead, is given by monthly subcutaneous, or under-the-skin, injections that target an anticlotting factor called protein S.
Protein S works with VWF and other proteins to restore balance to the blood clotting process. VGA039 binds to protein S, aiming to rebalance anti- and pro-clotting processes. This may help people with VWD form clots more normally.
“VGA039 could be transformative for patients, as it is designed to prevent or reduce bleeding across all types of VWD while reducing treatment burden via its once monthly, subcutaneous dosing regimen,” Patou said.
The ongoing Phase 1/2 VIVID trial (NCT05776069) is testing various doses of VGA039 in VWD. Interim results from 16 patients showed that monthly subcutaneous injections were well tolerated and substantially reduced bleeding rates.
Some participants in this group had previously been on intravenous prophylaxis for VWD. For these individuals, switching to VGA039 reduced bleeding rates by 75% to 100%.
Another subgroup of participants, who started the study with at least 12 bleeds per year — similar to the patients being recruited for the Phase 3 trial — and hadn’t previously been on prophylaxis, saw 73% to 87% reductions in bleeding rates.
VGA039 could be transformative for patients, as it is designed to prevent or reduce bleeding across all types of VWD while reducing treatment burden via its once monthly, [under-the-skin injection] dosing regimen.
VIVID-6 is building off these early results. The trial will enroll about 60 people, ages 12 to 75, with VWD. Eligible individuals cannot be routinely using preventive therapies containing VWF concentrates. Participants will undergo observation for 24 weeks, or nearly six months. Then, they will receive monthly subcutaneous doses of VGA039 for about 49 weeks, or just less than a year.
The main goal of VIVID-6 is to measure changes in bleeding events during the treatment period. Investigators will also assess safety and the development of antibodies against the drug, an immune process that could make VGA039 less effective.
Star hopes that these ongoing trials and the new FDA designations will help facilitate development of VGA039. ”We aim to bring this new treatment to people with VWD as efficiently as possible,” Patou said.
