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VGA039 · 2 trials · 2 indications
Incidence, nature and severity of adverse events (AEs) and serious adverse events (SAEs), including dose-limiting toxicities (DLTs).
| Arm | Type | Description |
|---|---|---|
| Multiple Doses of VGA039 after observational run-in | EXPERIMENTAL | Participants who meet eligibility criteria and complete a 24-week observational phase will receive VGA039 SC for approximately 49 weeks. |
| Part 1 | PLACEBO_COMPARATOR | Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined |
| Part 2 | EXPERIMENTAL | Cohorts A-H IV or SC VGA039 dose to be determined |
| Part 3 | EXPERIMENTAL | Cohorts MD-1 to MD-4, SC VGA039 multiple doses, dose to be determined |
| Part 4 | EXPERIMENTAL | Cohorts of VGA039 single dose for surgical prophylaxis |
| Part 5 | EXPERIMENTAL | Multiple doses of VGA039 in open label extension |
| Name | Type | Description |
|---|---|---|
| VGA039 | DRUG | VGA039 SC administered for 49-weeks during the interventional phase following a 24-week observational period. |
| Placebo | OTHER | Single doses of Placebo |
Key Inclusion Criteria * 12 to 75 years of age, inclusive * No clinically significant laboratory, ECG, or vital signs results * Documented diagnosis consistent with VWD of any type * Historical annualized bleeding rate (ABR; excluding menstrual bleeds and bleeds under the skin) of both untreated an...
VGA039 is an investigational small molecule being developed for the treatment of Von Willebrand Disease, a bleeding disorder. It is administered subcutaneously and is being studied in patients aged 12 years and older. Incyte Corporation is developing it as a potential new treatment option for this rare hematology condition.
VGA039 targets Protein S. By binding to this protein, VGA039 is designed to modulate the coagulation pathway in patients with Von Willebrand Disease. Protein S is a naturally occurring anticoagulant, and targeting it represents a novel approach to treating this bleeding disorder.
VGA039 is being developed by Incyte Corporation, which trades on the Nasdaq under the ticker INCY. Incyte is a biopharmaceutical company focused on oncology and hematology, and VGA039 is part of its pipeline for rare bleeding disorders.
VGA039 is in Phase 3 clinical development. The Phase 3 trial is currently recruiting participants, and a Phase 1 trial is also ongoing. VGA039 has not been approved by the FDA and remains an investigational therapy for Von Willebrand Disease.
VGA039 is being evaluated in two clinical trials. The Phase 3 study, NCT07115004, is recruiting 60 patients with Von Willebrand Disease across multiple countries. The Phase 1 study, NCT05776069 (VIVID), is recruiting 116 healthy volunteers and patients. Both trials are randomized, double-blind, and placebo-controlled.
Yes, VGA039 has received four FDA designations: Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease. These designations are intended to expedite the development and review of therapies for serious or rare conditions such as Von Willebrand Disease.