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NewsA new class of NAMPT inhibitors is reigniting hope for patients with AML and MDS. The investigational drug RPT1G works differently from earlier agents — selectively depleting NAD in cancer cells while sparing healthy ones. Early data from a healthy volunteer study showed a clean safety profile, and a phase 1 trial in AML/MDS patients is now underway.
NAMPT, an enzyme that regulates NAD levels inside cells, has long been considered a promising but frustrating target in oncology. Earlier inhibitors like CHS 828 and FK866 showed preclinical promise but fell short in clinical trials — producing no tumor responses and causing serious side effects including thrombocytopenia and retinal toxicity. The core problem: blocking NAMPT too completely harms healthy cells alongside cancer cells.
Enter RPT1G, a first-in-class hyperbolic NAMPT inhibitor designed to partially — not fully — suppress the enzyme. This nuanced approach aims to deplete NAD enough to kill cancer cells, particularly those already vulnerable due to deletions on chromosome 7q (common in AML and MDS), without starving normal cells of a critical metabolic cofactor.
Key Takeaways:
Why it matters: Relapsed/refractory AML remains one of oncology's toughest challenges. If RPT1G's favorable safety signal holds in cancer patients, it could open a new therapeutic avenue — both as monotherapy and in combination with already-approved agents.
