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December 8, 2025BloodOpen Access

First-in-human randomized placebo-controlled Phase 1 study of RPT1G, a novel hyperbolic NAMPT inhibitor for use in acute leukemias: Safety, pharmacokinetics, and pharmacodynamics in healthy volunteers

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Authors

MSMichael W. SchelleTJTimothy S. JarvelaKJKelechi Joe-Nweke

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Overview

Phase 1 trial demonstrates RPT1G's safety and favorable pharmacokinetics in healthy adults, indicating a potential for treating acute leukemias.

Key Points

  • The study aimed to evaluate the safety and pharmacokinetics of RPT1G, a NAMPT inhibitor, in healthy volunteers.
  • Conducted a first-in-human, Phase 1, randomized, double-blind, placebo-controlled study.
  • 56 healthy adult participants were enrolled, receiving either RPT1G or placebo in ascending doses.
  • Evaluated safety, tolerability, pharmacokinetics, and NAMPT target engagement.
  • No significant adverse events reported, with common TEAEs being mild.
  • Demonstrated a dose-proportional pharmacokinetic profile with no drug accumulation.
  • Showed meaningful NAMPT engagement with up to 5-fold induction of NAM and 1-MNA compared to placebo.

Cite This Study

Schelle et al. (2025) studied this question.

synapsesocial.com/papers/693624d74fa91c937236d12ehttps://doi.org/10.1182/blood-2025-5044
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