Dr. Hoffman on the Safety Profile of PTG-300 in Polycythemia Vera

Ronald Hoffman, MD, a professor of medicine, hematology, and medical oncology at Mount Sinai, discusses the safety profile of the first-in-class synthetic hepcidin mimetic agent PTG-300 in patients with polycythemia vera (PV).

PTG-300 is under examination in an ongoing phase 2 study (NCT04057040), says Hoffman. The primary objective of this research is to introduce a novel therapeutic agent to the limited PV treatment paradigm. To date, a limited number of patients have received the agent, says Hoffman. A total of 8 patients were included on the trial; of those, 7 have received the treatment and 6 were considered to be evaluable. Notably, these patients were heavily dependent on phlebotomy, Hoffman notes.

Results from the trial showed that PTG-300 was tolerable with no notable adverse effects (AEs) beyond those associated with the administration of a subcutaneous drug, such as local irritation, says Hoffman. That effect was reported in several patients, but was found to be easily managed. Additionally, no allergic reactions to the drug were observed, nor did patients experience progression to myelofibrosis, myeloid leukemia, or myeloproliferative neoplasm (MPN) blast phase. Patients also did not experience a hemorrhagic or thrombotic event during the administration phase of the trial, Hoffman concludes.