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Also known as BAY2599023 (DTX201)
BAY2599023 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| BAY2599023 / (DTX201) | EXPERIMENTAL | Adult patients with severe hemophilia A, who have been previously treated with FVIII products |
| Name | Type | Description |
|---|---|---|
| BAY2599023 (DTX201) | DRUG | Single escalating doses with 4 dose steps; Single intravenous (IV) administration. |
Inclusion Criteria: * Males age 18 years or older. * Confirmed diagnosis of hemophilia A as evidenced by their medical history with plasma FVIII activity levels \< 1% of normal or at screening. * Have \>150 exposure days (EDs) to FVIII concentrates (recombinant or plasma-derived). If on prophylaxi...
BAY2599023 is an investigational small molecule being developed for the treatment of hemophilia A. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities. The drug is designed to deliver a healthy version of the defective factor VIII gene into liver cells using an altered, non-infectious virus as a shuttle.
BAY2599023 targets the factor VIII gene, delivering a healthy copy into the nucleus of liver cells using an adeno-associated virus (AAV) vector. This approach aims to address the underlying genetic defect in hemophilia A, a condition characterized by deficient or defective factor VIII protein.
BAY2599023 is being developed by Bayer AG, a multinational pharmaceutical company. Bayer's stock is traded over-the-counter under the ticker symbol BAYRY. The drug is currently in Phase 1 clinical trials for hemophilia A.
BAY2599023 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The ongoing Phase 1 trial is active but not recruiting participants, and the drug remains in early-stage clinical testing.
BAY2599023 is being studied in one Phase 1 clinical trial with the identifier NCT03588299. This trial is testing the safety and response to treatment in patients with severe hemophilia A. The study is active but not recruiting, with an enrollment of 11 male participants aged 18 years and older.
Yes, BAY2599023 is also known as DTX201. The drug is referred to by both names in clinical research, with the Phase 1 trial title using the designation BAY 2599023 (DTX 201). Both names refer to the same investigational therapy for hemophilia A.