Remedy Plan advances RPT1G to Cohort 2 in AML/MDS Phase 1 trial
Remedy Plan Therapeutics has moved its lead asset, RPT1G, into the second dosing cohort of a Phase 1 dose-escalation trial in relapsed/refractory acute myeloid leukaemia and higher-risk myelodysplastic syndromes, after patients in Cohort 1 completed a full 28-day treatment cycle without reported dose-limiting toxicities. The Gaithersburg, Maryland company simultaneously announced FDA acceptance of an Investigational New Drug application for RPT1G in solid tumours, the closing of a $30 million Series A extension, and the appointment of an experienced haematology clinician to lead clinical development.
RPT1G is a small-molecule inhibitor of NAMPT, an enzyme central to cellular NAD biosynthesis and a target that has attracted oncology drug hunters for more than two decades. Earlier NAMPT inhibitors, including daporinad and GMX1778, reached clinical testing but were abandoned after narrow therapeutic windows produced haematologic and ocular toxicities at doses required for anti-tumour activity. Remedy Plan says RPT1G sidesteps this problem through a "hyperbolic" inhibition mechanism that modulates rather than abolishes NAMPT activity, leaving healthy-cell metabolism largely intact while disrupting the heightened metabolic dependency of cancer cells.
Clinical and regulatory progress
Greg Crimmins, founder and chief executive, said the Cohort 1 data represent "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." The statement is a notable clinical claim: if sustained through dose escalation, it would mark a meaningful departure from the historical record of the target. The Phase 1 study follows a traditional 3+3 design and is evaluating safety, tolerability, pharmacokinetics, and pharmacological activity. No efficacy data have been released at this stage, and the trial remains in its early safety-finding phase.
The FDA's acceptance of the solid-tumour IND broadens the potential addressable population considerably, and Remedy Plan said it intends to initiate clinical activities in that setting in parallel with the ongoing haematology study. The company is also exploring NAMPT inhibition in autoimmune and metabolic diseases, though no timelines or candidate names were disclosed for those indications.
Corporate developments
The $30 million Series A extension was led by existing investors alongside participation from Schooner Capital. Proceeds are earmarked for expanding patient volume in the AML/MDS trial, initiating solid-tumour clinical work, and supporting the broader NAMPT pipeline. No post-money valuation was disclosed.
Oleg Zernovak, appointed as Vice President of Clinical Development, brings more than 20 years of drug development experience in haematologic malignancies. He joins from BeOne Medicines, where he led early-development clinical oversight in AML and MDS. Earlier roles include vice president positions at Curis, and medical director roles at Daiichi-Sankyo, Celgene, and Novartis Oncology, giving him direct experience with most of the major players in this space.
Market context
AML and MDS remain areas of intense commercial and clinical activity. Approvals of venetoclax-based combinations, IDH inhibitors such as enasidenib and ivosidenib, and FLT3 inhibitors have reshaped the treatment landscape, but relapsed/refractory disease retains a significant unmet need and carries a poor prognosis. A differentiated metabolic approach that avoids the toxicities of prior NAMPT inhibitors could find a niche, particularly in patients who have exhausted targeted options. That said, the field has a history of promising early readouts in AML that do not translate to meaningful efficacy at tolerated doses. Investors and clinicians will be watching for pharmacodynamic evidence of NAMPT pathway suppression and early signals of tumour response as the dose-escalation study progresses toward a recommended Phase 2 dose.