Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as ISIS 702843, IONIS-TMPRSS6-LRx
sapablursen · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Sapablursen Dose Level 1 | EXPERIMENTAL | Sapablursen will be administered by SC injection every 4 weeks. |
| Sapablursen Dose Level 2 | EXPERIMENTAL | Sapablursen will be administered by SC injection every 4 weeks |
| Name | Type | Description |
|---|---|---|
| sapablursen | DRUG | Sapablursen will be administered by SC injection. |
Inclusion Criteria 1. Meet modified World Health Organization (WHO) 2016 diagnostic criteria for polycythemia vera (PV) at the time of clinical diagnosis 2. Participant must be phlebotomy dependent. 3. Participants do not need to be on cytoreductive therapy and do not need to have been previously t...
Sapablursen is an investigational RNA therapy being developed by Ionis Pharmaceuticals, Inc. for phlebotomy dependent polycythemia vera, a blood disorder in the hematology therapeutic area. It is an antisense inhibitor designed to target TMPRSS6 and is currently in Phase 2 clinical development.
Sapablursen is being studied for the treatment of phlebotomy dependent polycythemia vera, a myeloproliferative blood condition in which patients require regular blood removal to control red blood cell counts. It is an investigational therapy and has not been approved for any indication.
Sapablursen targets TMPRSS6, also known as matriptase-2. It is an antisense inhibitor, an RNA therapy designed to reduce production of the TMPRSS6 protein. By targeting TMPRSS6, the drug is intended to influence iron regulation and red blood cell production in polycythemia vera.
Sapablursen is developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker IONS. Ionis is the sponsor of the clinical program evaluating the drug in phlebotomy dependent polycythemia vera.
Sapablursen is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The Phase 2 study in phlebotomy dependent polycythemia vera has been completed.
Sapablursen has been evaluated in the Phase 2 trial NCT05143957, titled A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera. The completed trial enrolled 50 patients across the United States, Australia, Canada, Poland, and the United Kingdom.
Yes. Sapablursen was formerly known as ISIS 702843 and IONIS-TMPRSS6-LRx. These names refer to the same investigational antisense RNA therapy developed by Ionis Pharmaceuticals for phlebotomy dependent polycythemia vera.