Ronald Hoffman, MD, Director of the Myeloproliferative Diseases Program at The Icahn School of Medicine at Mount Sinai, describes the findings of an ongoing Phase II clinical trial assessing the safety and efficacy PTG-300 to treat individuals with polycythemia vera.
As Dr. Hoffman explains in this video, the data to date is impressive in providing persistent control of hemocrit levels in the patients with weekly administration of PTG-300, a mimetic of the endogenous iron regulator. The drug was safe, well tolerated, and found to eliminate the need for phlebotomy in all six patients that received the dosing as per the protocol.
Polycythemia vera is a rare blood disorder characterized by an increased concentration of blood cells. Most cases of polycythemia vera is acquired and mostly afflicts older persons, but it is associated with mutations in the JAK2 and TET2 genes.
Persons with polycythemia vera are at increased risk for deep vein thrombosis and one of the main goals of treating polycythemia is to reduce that risk, mainly by keeping hemocrit levels below 45%.
For more information about the trial, visit https://clinicaltrials.gov/ct2/show/NCT04057040