Remedy Plan Therapeutics Advances RPT1G for Myeloid Cancer Patients with Phase 1 Safety Data and IND Approval

• RPT1G is the first well-tolerated NAMPT inhibitor, demonstrating safety in healthy volunteers at single and multiple ascending dose levels.
• Target engagement data indicate that RPT1G inhibits NAMPT in humans at doses predicted to be therapeutically relevant for hematologic malignancies.
• FDA has cleared Remedy Plan's IND application for RPT1G in R/R AML and HR-MDS.
• Remedy Plan granted orphan drug designation for RPT1G in AML.

GAITHERSBURG, Md., Nov. 12, 2025 (GLOBE NEWSWIRE) -- RPT1G, a revolutionary new NAMPT inhibitor developed by Remedy Plan Therapeutics (RPT), was found to be safe and well-tolerated in a Phase 1 study in healthy volunteers at single and multiple ascending dose levels. NAMPT target engagement was seen at every dose level and predicted clinical efficacy against blood cancers.

“We have achieved something the field has been trying to do for decades,” says Greg Crimmins, PhD, Founder and CEO of Remedy Plan. “RPT1G is a new kind of drug, a new class of inhibitor, that targets NAMPT activity in cancer cells while avoiding toxicity in healthy cells. We think this drug has immense potential to help cancer patients.”

The FDA cleared an investigational new drug (IND) application to test RPT1G in a Phase 1 study in cancer patients with relapsed or refractory acute myeloid leukemia (R/R AML) and higher-risk myelodysplastic syndromes/neoplasms (HR-MDS). The FDA approved initial dosing of RPT1G at the highest dose tested in healthy volunteers.

“Data from the first-in-human healthy volunteer study confirmed the favorable preclinical data package for RPT1G and supports our newly opened study in myeloid cancers,” says Steve Abella, MD, Chief Medical Officer of Remedy Plan. “The upcoming study is being conducted at doses we believe are clinically relevant to target NAMPT, a key driver of these diseases.”

RPT will present results from the Phase 1 healthy volunteer study, upcoming R/R AML and HR-MDS study, and pre-clinical data on RPT1G efficacy in lymphoma at the American Society of Hematology general meeting on December 8th in Orlando, FL.

RPT also earned an orphan drug designation for AML from the FDA. Through the Orphan Drug Act, orphan drug status provides tax credits, grants and waivers of certain administrative fees for clinical trials, and the potential for seven years of market exclusivity following drug approval.

Drug companies have targeted the NAMPT enzyme for more than 20 years, but inhibiting it safely in people has eluded the scientific community until now. With NAMPT dysregulated in many different disorders, the scope of possibilities for a safe NAMPT drug is wide—blood cancers, solid tumors, and a range of other disorders and conditions.

“We’re in an exciting place, getting our drug to R/R AML and HR-MDS patients and then expanding to treat patients with solid tumors,” Crimmins said. “Looking ahead, we have a pipeline of novel hyperbolic NAMPT inhibitors for both oncology and non-oncology applications. This is just the beginning.”

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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