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ALN-6400 · 2 trials · 3 indications
| Arm | Type | Description |
|---|---|---|
| ALN-6400 (Treatment Group A) | EXPERIMENTAL | Participants will be administered multiple doses of ALN-6400. |
| ALN-6400 (Treatment Group B) | EXPERIMENTAL | Participants will be administered multiple doses of ALN-6400. |
| Part A: ALN-6400 | EXPERIMENTAL | Participants will be administered a single dose of ALN-6400. |
| Part A: Placebo | PLACEBO_COMPARATOR | Participants will be administered a single dose of placebo. |
| Part B: ALN-6400 | EXPERIMENTAL | Participants will be administered multiple doses of ALN-6400. |
| Part B: Placebo | PLACEBO_COMPARATOR | Participants will be administered multiple doses of placebo. |
| Name | Type | Description |
|---|---|---|
| ALN-6400 | DRUG | ALN-6400 will be administered subcutaneously (SC). |
| Placebo | DRUG | Placebo will be administered subcutaneously (SC) |
Inclusion Criteria - * Is an adolescent or adult patient with a diagnosis of VWD (including Type 1, Type 2, Type 3, and platelet-type VWD) and HMB for 2 cycles during screening Exclusion Criteria - * Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 2 times the u...
Top 4 of 5 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 4 | PHASE3 | Vonvendi, ADVATE |
| Incyte Corporation | INCY | 2 | PHASE3 | VGA039 |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE2 | ALN-6400 |
| Hemab Therapeutics Holdings, Inc. | COAG | 2 | PHASE1 | HMB-002 |
ALN-6400 is an investigational small molecule drug being developed by Alnylam Pharmaceuticals for rare bleeding disorders. It is being studied in hereditary hemorrhagic telangiectasia and von Willebrand disease, including heavy menstrual bleeding associated with VWD. It remains in clinical development and is not approved for any indication.
ALN-6400 targets plasminogen. By acting on plasminogen, the drug is designed to influence the fibrinolytic pathway involved in bleeding. This mechanism is being explored in conditions where abnormal blood vessel structure or clotting protein deficiency leads to excessive or recurrent bleeding.
ALN-6400 is being developed by Alnylam Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker ALNY. Alnylam is the sponsor of the clinical studies evaluating the drug in hereditary hemorrhagic telangiectasia and von Willebrand disease.
ALN-6400 is in clinical development. A Phase 1 study in healthy volunteers and patients with hereditary hemorrhagic telangiectasia has been conducted, and a Phase 2 study in von Willebrand disease with heavy menstrual bleeding is recruiting. The drug is investigational and has not been approved by the FDA.
ALN-6400 has been studied in NCT06659640, a Phase 1 trial in healthy volunteers and patients with hereditary hemorrhagic telangiectasia across the United States, Australia, Canada, France, Germany and Spain. The Phase 2 HMBeacon trial, NCT07575308, is recruiting adult and adolescent female patients with von Willebrand disease and heavy menstrual bleeding in the United States, Netherlands, Sweden and the United Kingdom.
ALN-6400 is being evaluated for von Willebrand disease, specifically in female patients who also experience heavy menstrual bleeding. The Phase 2 HMBeacon study, NCT07575308, is a randomized, double-blind, placebo-controlled trial enrolling adult and adolescent female patients aged 16 years and older.