Remedy Plan Therapeutics Advances RPT1G Trial and Strengthens Financial Position

Remedy Plan Therapeutics, a clinical-stage biotechnology company, has announced significant progress in its development of RPT1G, a first-in-class hyperbolic NAMPT inhibitor. The company has advanced the Phase 1 dose escalation trial of RPT1G, which is being evaluated in relapsed/refractory acute myeloid leukemia (R/R AML) and higher-risk myelodysplastic syndromes (HR-MDS). Patients in Cohort 1 have successfully completed their first full 28-day treatment cycle, and the company has now initiated enrollment in Cohort 2.

In a related development, the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application for RPT1G for the treatment of solid tumors. This acceptance marks an important milestone for the company and patients, as it paves the way for further clinical trials.

Remedy Plan has also bolstered its clinical leadership team by appointing Oleg Zernovak, M.D., as Vice President, Clinical Development. Dr. Zernovak brings over 20 years of clinical development experience, particularly in hematologic malignancies, and will play a crucial role in advancing RPT1G and expanding the company’s pipeline of first-in-class NAMPT inhibitors.

The company’s financial position has been strengthened with the closing of a Series A extension financing, raising approximately $30 million. This funding will support the advancement of RPT1G, including expanding patient volume in the ongoing Phase 1 study in hematology, initiating clinical activities for expansion into solid tumors, and supporting the continued development of proprietary NAMPT inhibitors across additional indications.

RPT1G targets NAMPT, an enzyme involved in cellular metabolism and energy production. Unlike previous approaches to NAMPT inhibition, which have been limited by toxicity, RPT1G is designed to modulate NAMPT activity through a hyperbolic inhibition mechanism. This approach aims to enable therapeutic activity while maintaining tolerability.

Greg Crimmins, PhD, Founder and CEO of Remedy Plan, stated, "Our progress to date in this trial marks the first time that therapeutic NAMPT inhibition, without limiting toxicity, has been successfully achieved in patients with cancer for longer than just a few days. This represents a significant scientific advancement that had previously been considered impossible to accomplish."

The Phase 1 trial is an ongoing multi-center, open-label, dose escalation study in patients with R/R AML and HR-MDS. The study follows a traditional 3+3 dose escalation design, with the option to add additional patients to dosing cohorts after they clear their respective safety review.

Remedy Plan Therapeutics is focused on developing novel therapies that target fundamental metabolic vulnerabilities in disease. The company’s proprietary drug discovery platform has enabled the development of a new class of novel NAMPT inhibitors designed to overcome decades-long challenges associated with targeting this pathway. The lead candidate, RPT1G, is currently being evaluated in an ongoing Phase 1 clinical study in patients with R/R AML and HR-MDS.