Protagonist Announces Updated Phase 2 Data Presented at ASH Annual Meeting Supporting Long Term Efficacy of Hepcidin Mimetic PTG-300 in the Treatment of Polycythemia Vera
"These results provide strong support for the potential for PTG-300 to improve on standards of care, not only for our patients receiving frequent phlebotomy but also for our patients who continue to receive phlebotomies in combination with other treatments," commented
Summary of Results
- Of the 18 PV patients treated with PTG-300, the vast majority were able to eliminate therapeutic phlebotomies and maintain a target hematocrit level of less than 45 percent.
- Treatment with PTG-300 was also shown to reverse iron deficiency, a serious side effect of regular therapeutic phlebotomies as a treatment for PV.
- Early observations suggest a decreased symptom burden over time, including overall burden (MPN-TSS), as well as measurements specific to mental function, fatigue and itching.
Administration of PTG-300was well tolerated, with injection site reactions and bruise as the only observed adverse events.
"We are very pleased with the study results obtained to date in controlling hematocrit with PTG-300, which mimics the mechanism of the natural hormone hepcidin in the body," commented Sam Saks, M.D., Chief Medical Officer of Protagonist. "We look forward to sharing these compelling data with
The ongoing Phase 2 PTG-300 polycythemia vera study is designed to monitor the safety profile and obtain evidence of efficacy in approximately 50 patients requiring frequent phlebotomies (at least three phlebotomies in prior six months). The study design consists of three stages: a 16-week open-label stage with dose escalation, reduction, or maintenance every four weeks from 10 mg to 80 mg by subcutaneous administration at weekly intervals, a 12-week maintenance period at doses that generate desired hematocrit levels, and then a randomized and blinded withdrawal stage (1:1 treatment vs. placebo) for up to 12 weeks. The study also has an open-label extension for up to one year to monitor long-term safety and other effects. The primary endpoint is the control of hematocrit below 45 percent during the blinded randomized withdrawal period. Other endpoints of this clinical proof-of-concept study include measurement of blood parameters (hematocrit and hemoglobin levels), reductions or delay in phlebotomy requirements, and symptoms related to quality of life.
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