A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms
Key claim: A first-in-human Phase 1 dose-escalation and dose-expansion study is evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of PRT12396 in patients with high-risk polycythemia vera and myelofibrosis.
Abstract
This is a first-in-human, open-label, multi-center Phase 1 study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of PRT12396 in participants with high-risk polycythemia vera (PV) and myelofibrosis (MF), and to determine the maximum tolerated dose (MTD) and recommended dose(s) for expansion (RDE[s]). The study consists of a dose-escalation phase followed by a dose-expansion phase to further evaluate selected dose level(s).
Why this matters
PRT12396’s Phase 1 enrolls both high-risk PV and MF, positioning it as a cross-subtype investigational agent at the earliest stage of clinical testing. For the Disease Subtypes vault, this expands the already crowded pipeline of novel mechanisms being tested in advanced PV and MF populations where current therapies (cytoreduction in PV; ruxolitinib in MF) leave significant unmet need. Tracking will depend on disclosure of the agent’s mechanism and any subtype-specific efficacy signals.