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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...
Top 8 of 9 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 15 | PHASE3 | Concizumab, Mim8, Denecimig, NNC0442-0344 A, turoctocog alfa pegol |
| Sanofi SA Sponsored ADR | SNY | 5 | PHASE3 | Fitusiran, efanesoctocog alfa |
| Pfizer Inc. | PFE | 8 | PHASE3 | Fidanacogene elaparvovec, Marstacimab |
| BioMarin Pharmaceutical Inc. | BMRN | 2 | PHASE3 | Valoctocogene roxaparvovec |
| Hoffmann-La Roche | RHHBY | 8 | PHASE3 | Emicizumab, NXT007, SPK-8011QQ, SPK-8011, SPK-8016 |
| Regeneron Pharmaceuticals, Inc. | REGN | 2 | PHASE1 | REGV131, LNP1265 |
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 2 | PHASE1 | AskBio009 |
| Bayer | BAYRY | 6 | - | Damoctocog-alfa-pegol |
BAY2599023 is an investigational gene therapy being developed by Bayer AG for severe hemophilia A. It delivers a healthy version of the factor VIII gene into the nucleus of liver cells using an altered, non-infectious adeno-associated virus (AAV) as a shuttle. It is a Phase 1 asset and has not been approved.
BAY2599023 is being studied as a treatment for severe hemophilia A, a bleeding disorder caused by a defective factor VIII gene. The therapy is designed to deliver a healthy copy of that gene into liver cells so the body can produce factor VIII. It is investigational and has not been approved for any use.
BAY2599023 is developed by Bayer AG, which trades on the OTC market under the ticker BAYRY. Bayer sponsored the Phase 1 study of the therapy in severe hemophilia A. The asset is also referred to as BAY2599023 (DTX201).
BAY2599023 is in Phase 1 clinical development. Its single Phase 1 trial, NCT03588299, has been completed. The study enrolled 11 participants and was an uncontrolled, non-randomized, open-label study, so it did not include a placebo or comparator arm. The drug remains investigational.
BAY2599023 has been evaluated in one Phase 1 trial, NCT03588299, titled a study to test the safety and how well patients with severe hemophilia A respond to treatment with BAY 2599023 (DTX 201). The trial is completed and enrolled 11 male participants aged 18 years and older across the United States, Bulgaria, France, Germany, the Netherlands, and the United Kingdom.
Yes. BAY2599023 is also known as DTX201, and the two names refer to the same investigational gene therapy for severe hemophilia A. Trial records list the asset as BAY 2599023 (DTX 201). Bayer AG is the developer behind the program.