Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Open-label rusfertide, PTG-300
Rusfertide · 7 trials · 5 indications
Median hematocrit
Number of phlebotomies
Response was defined as absence of phlebotomy eligibility. Phlebotomy eligibility was defined as either: 1) A confirmed hematocrit (HCT) greater than or equal to (≥) 45% that was at least 3% higher than the baseline HCT, where confirmation was defined as 2 consecutive HCT assessments that were ≥ 45% and at least 3% higher than the baseline HCT, or 2) HCT ≥ 48%.
Proportion of subjects with hematocrit \<45%
Change from Baseline to Week 24 (or End of Treatment) in transferrin saturation (TSAT) as measured by blood laboratory tests.
Change from Baseline to Week 24 (or End of Treatment) in serum iron as measured by blood laboratory tests.
A subject will be considered a responder during the blinded randomized withdrawal phase if hematocrit control is maintained without phlebotomy eligibility. "Phlebotomy eligibility" is defined as any one of the following criteria being met: * hematocrit ≥45% that was ≥3% higher than Week 29 pre-randomization hematocrit value, or * hematocrit \>48%, or * an increase of ≥5% in hematocrit compared to Week 29 pre-randomization hematocrit value.
the long-term safety and tolerability of PTG-300 in Beta Thalassemia.
NTD subjects who achieve an increase in Hgb without transfusion
TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period
| Arm | Type | Description |
|---|---|---|
| Open-label rusfertide | EXPERIMENTAL | Open-label rusfertide |
| Part 1a: Randomized, Double-Blind Period: Placebo | PLACEBO_COMPARATOR | Participants received their ongoing therapy for polycythemia vera (PV) along with rusfertide-matching placebo, subcutaneous (SC) injection, once weekly, from baseline up to Week 32. |
| Part 1a: Randomized, Double-Blind Period: Rusfertide | EXPERIMENTAL | Participants received their ongoing therapy for PV along with rusfertide once weekly, SC injection, from baseline up to Week 32. Rusfertide doses were titrated to maintain hematocrit less than (\<) 45 percent (%). The starting dose was 19 milligrams (mg). The doses that were administered to Part 1a participants were 9.5, 19, 28, 41, 54, 69 and 82 mg. |
| Part 1b: Open-Label Treatment Period: Rusfertide | EXPERIMENTAL | Participants received rusfertide once weekly, SC injection, from Weeks 32 to 52. Rusfertide doses were titrated to maintain hematocrit \< 45%. Participants who received rusfertide in Part 1a continued at their current dose unless dose adjustment was required. Participants who received placebo in Part 1a started rusfertide at 19 mg. The doses that were administered to Part 1b participants were 9.5, 19, 28, 41, 54, 69 and 82 mg. |
| Part 2: Long Term Extension Period: Rusfertide | EXPERIMENTAL | Participants received rusfertide once weekly, SC injection, from Weeks 52 to 156. Rusfertide doses were titrated to maintain hematocrit \< 45%. The doses that were administered to Part 2 participants were 9.5, 19, 28, 41, 54, 69, 82, 95 and 108 mg. For weekly doses greater than (\>) 90 mg, the dose of rusfertide was and will be divided into 2 doses administered 3 to 4 days apart. |
| Part 3: Treatment Extension Period: Rusfertide | EXPERIMENTAL | Participants will receive rusfertide once weekly, SC injection, from Week 156 to the end of the study (approximately 5 years). Rusfertide doses will be titrated to maintain hematocrit \< 45%. The doses that will be administered to Part 3 participants are 9.5, 19, 28, 41, 54, 69, 82, 95 and 108 mg. For weekly doses \> 90 mg, the dose of rusfertide will be divided into 2 doses administered 3 to 4 days apart. |
| PTG-300 | EXPERIMENTAL | Evaluate PTG-300's efficacy and safety in subjects with PV and baseline elevated hematocrit. |
| Dose finding PTG-300 (Part 1); PTG-300 (Part 2); Open label extension PTG-300 (Part 3) | EXPERIMENTAL | - |
| Dose finding PTG-300 (Part 1); Placebo (Part 2); Open label extension PTG-300 (Part 3) | EXPERIMENTAL | - |
| Interventions | EXPERIMENTAL | PTG-300 |
| PTG-300 Active | EXPERIMENTAL | Drug: PTG-300 Subcutaneous |
| Name | Type | Description |
|---|---|---|
| Open-label rusfertide | DRUG | subcutaneous rusfertide |
| Rusfertide | DRUG | Rusfertide SC injection |
| Placebo | DRUG | Rusfertide-matching placebo SC injection |
| PTG-300 | DRUG | Hepcidin mimetic |
Inclusion Criteria: * Subject who has completed at least 12 months of dosing with rusfertide and successfully completed the end of treatment visit of a previous Phase 2 study of rusfertide. * Subject understands the study procedures, is willing and able to adhere to study requirements and agrees to...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Merck & Co., Inc. | MRK | 2 | PHASE3 | Bomedemstat |
| Protagonist Therapeutics, Inc. | PTGX | 2 | PHASE3 | Rusfertide |
| Disc Medicine, Inc. | IRON | 1 | PHASE2 | DISC-3405 |
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 3 | PHASE2 | Rusfertide |
| Agios Pharmaceuticals, Inc. | AGIO | 1 | PHASE2 | AG-236 |
| Prelude Therapeutics, Inc. | PRLD | 1 | PHASE1 | PRT12396 |
Rusfertide is an investigational small molecule being developed for polycythemia vera, a blood cancer in the hematology area. It is being studied in adults 18 years and older with PV, including a long-term safety study and a larger placebo-controlled Phase 3 trial. It is not yet approved.
Rusfertide targets hepcidin. It is a peptide, reflected in its -tide class, and is designed to act on hepcidin, the hormone that regulates iron availability. By targeting hepcidin, rusfertide is being developed as a small molecule therapy for polycythemia vera.
Rusfertide is being developed by Protagonist Therapeutics, Inc., which trades under the ticker PTGX. The company is running Phase 3 clinical development of rusfertide in polycythemia vera, with two active trials in adults with the condition.
Rusfertide is in Phase 3 clinical development for polycythemia vera. Both of its registered trials are Phase 3 studies, and both are currently active but not recruiting. Rusfertide remains investigational and is not approved for use.
Rusfertide is being studied in two Phase 3 trials in polycythemia vera: NCT05210790, a placebo-controlled study of rusfertide in adults with PV enrolling 293 participants across the United States, Europe, and other countries, and NCT06033586, a long-term safety study in the United States with 46 participants.
Yes, open-label rusfertide refers to the same investigational drug, rusfertide. Open-label describes the study design in which participants and investigators know the treatment being given, rather than a separate product. Rusfertide is also identified by the code PTG-300 in its trial listings.