Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PTG-300 · 6 trials · 6 indications
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
Bioavailability (area under the plasma-concentration time) of PTG-300 following subcutaneous and intramuscular administration in healthy volunteers
| Arm | Type | Description |
|---|---|---|
| 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 |
| Intravenous | EXPERIMENTAL | PTG-300 Intravenous |
| Subcutaneous Low Concentration | EXPERIMENTAL | PTG-300 Subcutaneous Low Concentration |
| Subcutaneous High Concentration | EXPERIMENTAL | PTG-300 Subcutaneous High Concentration |
| Intramuscular | EXPERIMENTAL | PTG-300 Intramuscular |
| Name | Type | Description |
|---|---|---|
| PTG-300 | DRUG | Hepcidin mimetic |
| Placebo | DRUG | Placebo |
Inclusion Criteria: 1. Known diagnosis of polycythemia vera. 2. Hematocrit \>48% before dosing. 3. Evidence of hematocrit \>48% three or more times in the 28 weeks before dosing or five or more times in 52 weeks before dosing (except for newly diagnosed patients). 4. Clinically reasonable alternati...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Agios Pharmaceuticals, Inc. | AGIO | 5 | PHASE3 | Mitapivat |
| Bristol-Myers Squibb Company | BMY | 6 | PHASE3 | Luspatercept |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 2 | PHASE3 | Etavopivat A, Etavopivat B, Etavopivat C |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE2 | REGN7999 |
| ICON Plc | ICLR | 1 | PHASE2 | SP-420 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
PTG-300 is an investigational small molecule being studied for the treatment of polycythemia vera, hereditary hemochromatosis, and beta-thalassemia. It has also been evaluated in healthy volunteers to assess its pharmacokinetics and pharmacodynamics. The drug is being developed by Protagonist Therapeutics, Inc. and is currently in clinical development.
PTG-300 is a hepcidin mimetic, meaning it mimics the action of the hormone hepcidin, which regulates iron absorption and distribution in the body. By acting like hepcidin, PTG-300 aims to reduce iron levels and manage conditions associated with iron overload, such as polycythemia vera and hereditary hemochromatosis.
PTG-300 is being developed by Protagonist Therapeutics, Inc., a biopharmaceutical company listed on the NASDAQ under the ticker symbol PTGX. The company is conducting clinical trials to evaluate the safety and efficacy of PTG-300 in various indications, including polycythemia vera and hereditary hemochromatosis.
PTG-300 is in Phase 2 clinical development. It has completed Phase 1 and Phase 2 trials, including studies in healthy volunteers, patients with polycythemia vera, and patients with hereditary hemochromatosis. The drug is investigational and has not been approved by regulatory authorities.
PTG-300 has been studied in several clinical trials, including NCT04057040 (REVIVE) in patients with polycythemia vera, NCT04202965 in subjects with hereditary hemochromatosis, NCT04516382 in healthy volunteers, and NCT04767802 in patients with polycythemia vera and elevated hematocrit. All trials have been completed.
Yes, PTG-300 is a hepcidin mimetic. It is designed to replicate the function of hepcidin, a hormone that controls iron levels in the body. By mimicking hepcidin, PTG-300 aims to reduce iron overload in conditions like polycythemia vera and hereditary hemochromatosis.