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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

First-in-human trial shows RPT1G is safe and well-tolerated, demonstrating meaningful NAMPT inhibition in healthy adults.

Key Points

  • This trial aims to evaluate the safety, pharmacokinetics, and pharmacodynamics of RPT1G in healthy individuals.
  • Phase 1, single-center, randomized, double-blind, placebo-controlled trial
  • Administered RPT1G at single and multiple ascending doses to healthy volunteers
  • Primary objectives focused on safety, with secondary objectives on pharmacokinetics and pharmacodynamics.
  • No serious adverse events or treatment emergent adverse events above Grade 2 reported in participants
  • RPT1G demonstrated favorable pharmacokinetics with a half-life of approximately 6 hours
  • Significant NAMPT target engagement with increased levels of nicotinamide and 1-methylnicotinamide observed.

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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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Phase 1, open-label, multi-center trial of RPT1G in patients with relapsed/refractory acute myeloid leukemia and higher-risk myelodysplastic syndromes/neoplasms.2026
  2. 2Abstract CT267: Phase 1, open-label, multi-center trial of RPT1G in patients with relapsed/refractory acute myeloid leukemia and high-risk myelodysplastic syndromes/neoplasms2026
  3. 3Abstract 3050: RPT1G, a novel hyperbolic NAMPT inhibitor, effectively synergizes with PARP inhibitors in solid tumors <i>in vitro</i> and in a xenograft breast cancer model2026
  4. 4DDDR-73. Preclinical evaluation of blood brain permeable, novel nicotinamide phosphoribosyl-transferase (NAMPT) inhibitor in gliomas2025
  5. 5Abstract 419: Targeting NAMPT using Novel Inhibitor RPT-E-037 in Pancreatic Neuroendocrine Tumor.2026