Remedy Plan Advances First-in-Class Hyperbolic NAMPT Inhibitor RPT1G Into Cohort 2 in R/R AML and HR-MDS; FDA Accepts Solid Tumor IND
核心洞察
Remedy Plan Therapeutics (搜索) has initiated enrollment in Cohort 2 of its Phase 1 dose escalation trial of RPT1G in relapsed/refractory AML and higher-risk MDS.
The FDA has accepted an Investigational New Drug application for RPT1G in solid tumors (搜索), expanding the program beyond hematologic malignancies.
Cohort 1 patients completed the first full 28-day treatment cycle, which the company says marks the first sustained therapeutic NAMPT (搜索) inhibition without limiting toxicity in cancer patients.
Remedy Plan Therapeutics (搜索) reported that enrollment has advanced into Cohort 2 of its ongoing Phase 1 dose escalation trial of RPT1G, a first-in-class hyperbolic NAMPT (搜索) inhibitor, in patients with relapsed/refractory acute myeloid leukemia (搜索) (R/R AML) and higher-risk myelodysplastic syndromes (搜索) (HR-MDS). Patients enrolled in Cohort 1 completed the first full 28-day treatment cycle, according to the Gaithersburg, Maryland-based clinical-stage biotechnology company. In parallel, the U.S. Food and Drug Administration (搜索) has accepted the company's Investigational New Drug (IND) application for RPT1G for the treatment of solid tumors (搜索).
A Hyperbolic Mechanism Aimed at a Long-Standing Toxicity Barrier
RPT1G targets NAMPT (搜索), an enzyme involved in cellular metabolism and energy production. Prior approaches to NAMPT inhibition have been limited by toxicity. RPT1G is designed to modulate NAMPT activity through a hyperbolic inhibition mechanism, with the goal of enabling therapeutic activity while maintaining tolerability. Rather than complete enzyme shutdown, the drug is intended to allow controlled modulation of NAMPT activity, maintaining metabolic function in healthy cells while disrupting the metabolic vulnerabilities of cancer cells.
"Our progress to date in this trial marks the first time that therapeutic NAMPT (搜索) 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," said Greg Crimmins, PhD, Founder and CEO of Remedy Plan.
Crimmins framed the solid tumor IND acceptance as a broadening of the program's reach: "The FDA's acceptance of our IND application for RPT1G for the treatment of solid tumors (搜索) marks an important milestone for patients and the growth of Remedy Plan, as we believe our novel approach to NAMPT (搜索) inhibition unlocks value across the entire spectrum of oncology."
Trial Design and Endpoints
The Phase 1 study (NCT07107126) is an ongoing multi-center, open-label, dose escalation trial in patients with R/R AML and HR-MDS. It is assessing the safety, tolerability, pharmacokinetics, and pharmacologic activity of RPT1G. 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.
Financing and Clinical Leadership
Remedy Plan announced the closing of a Series A extension financing totaling approximately $30 million, with participation from Schooner Capital (搜索) and primarily existing investors. Proceeds will be used to accelerate advancement of RPT1G, including expanding patient volume in the ongoing Phase 1 study in hematology, initiating clinical activities around expansion into solid tumors (搜索), and supporting continued development of proprietary NAMPT (搜索) inhibitors across additional indications.
The company also appointed Oleg Zernovak, M.D. as Vice President, Clinical Development. Zernovak brings more than 20 years of drug development experience with expertise in hematologic malignancies and clinical trial execution. Most recently he served as Executive Director, Hematology Clinical Development at BeOne Medicines (搜索), where he led clinical and medical oversight in early development in AML and MDS and provided clinical leadership for regulatory engagements. Previously he served as Vice President, Clinical Development for Curis (搜索), contributing to clinical strategy for assets in AML, MDS, and solid tumors (搜索), and earlier held roles as Senior Medical Director, Early Development, Hematologic Malignancies at Cellectis and Janssen, and Medical Director at Daiichi-Sankyo (搜索), Celgene Corporation, and Novartis Oncology (搜索).
"Oleg's expertise in hematologic malignancies, with direct experience managing clinical trials in AML and MDS, will be deeply valuable as we advance RPT1G and expand our emerging pipeline of first-in-class NAMPT (搜索) inhibitors," Crimmins said. "Meanwhile, the closing of our Series A extension directly supports our ability to generate deeper and strategically actionable data from the ongoing Phase 1 trial during this upcoming year of growth for our company."
Broader Development Ambitions
Remedy Plan describes its proprietary drug discovery platform as having enabled development of a new class of NAMPT (搜索) inhibitors designed to overcome decades-long challenges associated with targeting this pathway. In parallel with the hematology study, the company is advancing clinical studies exploring NAMPT inhibition across additional oncology applications, as well as autoimmune and metabolic diseases.