Approval Probability
TA Base Rate
Adjusted LOA
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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...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 3 | PHASE3 | Vonicog Alfa, ADVATE |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE2 | ALN-6400 |
| Hemab Therapeutics Holdings, Inc. | COAG | 2 | PHASE1 | HMB-002 |
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 1 | - | Undisclosed |
VGA039 is an investigational small molecule being developed for the treatment of Von Willebrand Disease (VWD), a bleeding disorder. It is currently in Phase 3 clinical development and has not been approved by the FDA. The drug is being studied in patients with VWD, including those aged 12 years and older.
VGA039 targets Protein S, a protein involved in the regulation of blood coagulation. By modulating Protein S activity, VGA039 aims to address the underlying bleeding tendency in Von Willebrand Disease. This mechanism is being evaluated in clinical trials to assess its safety and efficacy in patients.
VGA039 is being developed by Incyte Corporation, a biopharmaceutical company traded on the NASDAQ under the ticker symbol INCY. Incyte is conducting clinical trials to evaluate VGA039 for the treatment of Von Willebrand Disease, with studies currently in Phase 3 development.
VGA039 is currently in Phase 3 clinical development for Von Willebrand Disease. It is an investigational drug that has not received FDA approval. The drug has been granted Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations by the FDA, reflecting its potential in this indication.
VGA039 is being studied in two clinical trials. NCT05776069 is a Phase 1 study in healthy volunteers and patients with Von Willebrand Disease, with an enrollment of 116 participants. NCT07115004 is a Phase 3 study evaluating subcutaneous VGA039 in patients with VWD, with an enrollment of 60 participants.
VGA039 is the sole name provided for this investigational drug. It is not known by any alternative names in the available information. The drug is being developed by Incyte Corporation and is currently in Phase 3 trials for Von Willebrand Disease.