Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Rusfertide
PTG-300 · 7 trials · 5 indications
Median hematocrit
Number of phlebotomies
Response is defined as absence of phlebotomy eligibility.
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 |
| Rusfertide | EXPERIMENTAL | Rusfertide (32 Weeks) - Rusfertide (124 Weeks Open-label) |
| Placebo | EXPERIMENTAL | Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label) |
| 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 |
| Placebo | DRUG | Placebo |
| Rusfertide | DRUG | Experimental drug |
| 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...
PTG-300 is an investigational small molecule being developed for rare blood disorders, specifically hereditary hemochromatosis, polycythemia vera, and beta-thalassemia. It is designed to address conditions involving iron overload and ineffective red blood cell production. The drug is currently in Phase 2 clinical development and has not been approved by any regulatory agency.
PTG-300 is a hepcidin mimetic. Hepcidin is a hormone that regulates iron absorption and distribution in the body. By mimicking hepcidin, PTG-300 aims to reduce iron overload and modulate erythropoiesis in conditions such as hereditary hemochromatosis, polycythemia vera, and beta-thalassemia.
PTG-300 is being developed by Protagonist Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol PTGX. The company focuses on discovering and developing peptide-based therapeutics for hematology and other diseases.
PTG-300 is in Phase 2 clinical development. It has completed two Phase 2 trials, and no active trials are currently listed. The drug remains investigational and has not been approved for any indication.
PTG-300 has been evaluated in completed Phase 2 trials, including NCT04767802 in polycythemia vera, NCT04202965 in hereditary hemochromatosis, NCT04057040 (REVIVE) in polycythemia vera, and NCT04054921 in beta-thalassemia. These trials enrolled a total of 97 participants across multiple countries.