Remedy Plan Therapeutics to Present Results from Phase 1 Healthy Volunteer Study of NAMPT Inhibitor RPT1G and Other Progress at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition
NAMPT inhibitor RPT1G demonstrated favorable safety and significant target engagement in first-in-human study
Company to present Trial in Progress design from ongoing Phase 1 study of RPT1G in patients with advanced myeloid cancers
RPT1G anti-tumor efficacy data in pre-clinical non-Hodgkin lymphoma models to be showcased
GAITHERSBURG, Md., Dec. 04, 2025 (GLOBE NEWSWIRE) -- Remedy Plan Therapeutics (“Remedy Plan”), a clinical-stage biotech company focused on developing NAMPT inhibitors for patients with hematological malignancies and solid tumors, today announced the results from the Company’s first-in-human Phase 1 healthy volunteer study of lead candidate RPT1G. The candidate was safe and well-tolerated with significant NAMPT target engagement in healthy adult participants. Full results will be presented at the upcoming 67th American Society of Hematology (ASH) Annual Meeting and Exposition taking place December 6-9, 2025, in Orlando, Florida.
RPT1G is a novel hyperbolic inhibitor of nicotinamide phosphoribosyltransferase (NAMPT), a key enzyme in cellular metabolism and a well-established oncology target. This Phase 1 randomized, placebo-controlled trial (NCT06667765) assessed the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of RPT1G. The study (n = 56) included both single-ascending dose (SAD) and multiple-ascending dose (MAD) cohorts to inform dose selection and target engagement. Key results are as follows (poster #5044):
- RPT1G was well tolerated by all SAD and MAD cohort participants with no Treatment Emergent Adverse Events (TEAEs) above Grade 2 and no serious adverse events.
- There was no discontinuation due to TEAE.
- Favorable PK profile established, without observed RPT1G accumulation over the 5-day dosing regimen.
- Dose-proportional NAMPT inhibition versus placebo observed.
- The magnitude of target engagement was greater than responses achieved in xenograft mouse models at efficacious doses, supporting a therapeutic level of NAMPT inhibition in human subjects.
- The magnitude of target engagement was greater than responses achieved in xenograft mouse models at efficacious doses, supporting a therapeutic level of NAMPT inhibition in human subjects.
"Establishing the safety, PK, and PD profile of RPT1G is a huge leap forward, scientifically and clinically," said Michael Schelle, PhD, Chief Scientific Officer of Remedy Plan. "The ability of RPT1G to safely inhibit NAMPT in people is compelling validation of our therapeutic approach and the years of research and development we've dedicated to NAMPT inhibition. Thanks to all of the study participants and investigators who helped us achieve this important milestone."
Remedy Plan will also present a Trial in Progress abstract (poster #5208) outlining the designs and objectives of its ongoing multi-center, open-label Phase 1 study of RPT1G in patients with relapsed/refractory acute myeloid leukemia (R/R AML) and higher-risk myelodysplastic syndrome (HR-MDS) (NCT07107126). In a third presentation (poster #5080), the Company will highlight new pre-clinical data demonstrating the preliminary anti-tumor efficacy of RPT1G in cellular and xenograft models of non-Hodgkin lymphoma (NHL), including 83% tumor growth inhibition (p = 0.0012) in a mouse xenograft model of NHL originating from germinal center B cells.
"Reaching this stage with our RPT1G lead program is a transformative moment for our company,” said Greg Crimmins, PhD, Founder and CEO of Remedy Plan. “Our momentum stems from a novel scientific approach and our commitment to bringing impactful therapies to patients with cancer."
Details for Remedy Plan poster presentations at the 2025 ASH Annual Meeting are as follows:
| Poster Number and Title |
Presentation Details |
|
Poster #5044 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 |
Session 604. Molecular Pharmacology and Drug Resistance: Myeloid Neoplasms Monday, December 8, 2025, 6 – 8 pm ET |
|
Poster #5208 Phase 1, open-label, multi-center trial of RPT1G in patients with Relapsed/Refractory Acute Myeloid Leukemia and high-risk Myelodysplastic Syndromes/neoplasms |
Session 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies Monday, December 8, 2025, 6 – 8 pm ET |
|
Poster #5080 RPT1G, a novel hyperbolic NAMPT inhibitor, is highly effective against B-cell non-Hodgkin's lymphoma with germinal center origin |
Session 605. Molecular Pharmacology and Drug Resistance: Lymphoid Neoplasms Monday, December 8, 2025, 6 – 8 pm ET |
About RPT1G
RPT1G inhibits nicotinamide phosphoribosyltransferase (NAMPT) using a unique mechanism—hyperbolic inhibition—to selectively reduce NAD in malignant cells. RPT1G is in Phase 1 trials for R/R AML (Relapsed or Refractory Acute Myeloid Leukemia) and HR-MDS (High-Risk Myelodysplastic Syndromes). A first-in-human study showed that RPT1G provides potent NAMPT inhibition that is well-tolerated, without the on-target toxicities that ended previous NAMPT programs. With the NAMPT enzyme dysregulated in many cancers, we believe RPT1G signals a new era of treatment.
About the Phase 1 Healthy Volunteer Study
RPT1G was determined to be safe and well-tolerated in a first-in-human, Phase 1, single-center, randomized, double-blind, placebo-controlled, single and multiple ascending dose study in healthy adult participants (NCT06667765). 56 participants were enrolled in the study with 42 receiving RPT1G. All participants completed treatment as planned. There were no reports of treatment emergent adverse events (TEAE) above Grade 2, no serious adverse events, and no TEAE leading to drug discontinuation. Target engagement data indicated that RPT1G inhibits NAMPT in humans at doses predicted to be clinically meaningful in hematologic malignancies.
About Remedy Plan Therapeutics
Remedy Plan Therapeutics is a clinical-stage biotech company focused on developing NAMPT inhibitors for patients with hematological malignancies and solid tumors. Our mission is to solve a decades-old medical-science puzzle: targeting NAD synthesis in cancer cells without damaging healthy cells. Our lead asset, RPT1G, is a novel, small-molecule NAMPT inhibitor designed for this purpose. Our team comprises experienced scientists, clinicians, and drug developers working together to bring hope to communities affected by these diseases. For more information on Remedy Plan, visit remedyplan.com and follow us on LinkedIn.
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