Remedy Plan Therapeutics Secures $18 Million to Advance Novel NAMPT Inhibitor for AML and MDS Treatment
核心洞察
Gaithersburg-based Remedy Plan Therapeutics (搜索) has raised $18 million in an oversubscribed insider financing round, bringing their total funding to $55 million to date.
The funding will accelerate the development of RPT1G, their first-in-class NAMPT (搜索) inhibitor, into Phase 1/2 clinical trials for acute myeloid leukemia (搜索) (AML (搜索)) and high-risk myelodysplastic syndromes (搜索) (MDS (搜索)).
Remedy Plan's innovative "hyperbolic" approach to NAMPT (搜索) inhibition avoids the on-target toxicities that have hindered previous development efforts, potentially addressing over 20 diseases spanning oncology, autoimmunity, and metabolic disorders.
Remedy Plan Therapeutics (搜索), a clinical-stage pharmaceutical company based in Gaithersburg, Maryland, has secured over $18 million in an oversubscribed insider financing round to advance its novel NAMPT (搜索) inhibitor program. The funding, which includes participation from Schooner Capital (搜索) and Alexandria Venture Investments (搜索), brings the company's total funding to $55 million.
The investment will primarily support the advancement of RPT1G, the company's first-in-class NAMPT (搜索) inhibitor, into a Phase 1/2 clinical study for patients with acute myeloid leukemia (搜索) (AML (搜索)) or high-risk myelodysplastic syndromes (搜索) (MDS (搜索)). This development follows the company's ongoing first-in-human study in healthy adults (NCT06667765), which is scheduled to conclude later this month.
"Pharma companies have been trying to unlock the NAMPT (搜索) target for over 25 years, and we have finally cracked it," said Greg Crimmins, Ph.D., Founder and CEO of Remedy Plan. "We are eager to advance RPT1G into Phase 1/2 clinical studies for patients with AML (搜索)/MDS (搜索), for which few treatment options exist. This funding not only allows us to sprint toward that goal, but also allows us to further develop our pipeline of novel NAMPT inhibitors for use in other diseases."
Breakthrough in NAMPT Inhibition
NAMPT (搜索) (Nicotinamide Phosphoribosyltransferase (搜索)) is a critical enzyme that controls cellular energy utilization and is fundamental to human biology. Its dysregulation has been linked to more than 20 diseases, making it a high-value therapeutic target for biopharmaceutical drug development.
Previous attempts to develop NAMPT (搜索) inhibitors have been hampered by severe on-target toxicities in healthy tissues, preventing meaningful clinical activity. Remedy Plan has developed what they call a "hyperbolic" approach to NAMPT inhibition that never fully turns off NAMPT activity, thereby avoiding on-target toxicity in healthy tissues while still meaningfully inhibiting NAMPT activity in diseased cells.
RPT1G is the first well-tolerated NAMPT (搜索) inhibitor and the first to be tested in healthy volunteers. This represents a significant breakthrough in the field, as previous NAMPT inhibitors developed by other companies failed due to toxicity issues.
Expanding Beyond AML and MDS
While the immediate focus is on advancing RPT1G into clinical trials for AML (搜索) and MDS (搜索), the company plans to use part of the funding to continue developing its pipeline of NAMPT (搜索) inhibitors for applications across solid tumors, autoimmune diseases, and obesity.
"We continue to be impressed by Remedy Plan's differentiated scientific approach and the potential of hyperbolic NAMPT (搜索) inhibition," said Alexandra Manick, Principal at Schooner Capital (搜索). "We believe the company is well-positioned to create significant value by addressing key unmet needs in oncology, autoimmunity, and obesity with their pipeline of unique NAMPT inhibitors."
Clinical Development Timeline
The Phase 1 clinical study of RPT1G in healthy volunteers is set to conclude in May 2025. Following this, Remedy Plan plans to initiate a Phase 1/2 study in patients with AML (搜索) or high-risk MDS (搜索) in the second half of 2025. The trial will evaluate RPT1G both as a monotherapy and in combination with venetoclax, a BCL-2 inhibitor commonly used in AML treatment.
For patients with AML (搜索) and high-risk MDS (搜索), treatment options remain limited, with poor outcomes for many patients. The development of RPT1G could potentially address a significant unmet medical need in these hematologic malignancies.
The Science Behind Hyperbolic NAMPT Inhibition
Remedy Plan's approach to NAMPT (搜索) inhibition represents a novel paradigm in drug development. Rather than completely blocking NAMPT activity, which leads to toxicity in healthy tissues, their hyperbolic inhibitors reduce NAMPT activity to levels that are still compatible with normal cellular function in healthy cells but are insufficient for cancer cells to thrive.
This selective approach exploits the differential dependency of cancer cells on NAMPT (搜索) activity compared to normal cells. Cancer cells, with their altered metabolism and increased energy demands, are more sensitive to partial NAMPT inhibition than normal cells, creating a therapeutic window that previous NAMPT inhibitors lacked.
The company's technology platform has potential applications beyond oncology. The same hyperbolic NAMPT (搜索) inhibition approach could be applied to autoimmune diseases, where immune cells show altered metabolism, and obesity, where NAMPT plays a role in energy homeostasis.
As Remedy Plan advances its lead candidate into clinical trials for AML (搜索) and MDS (搜索), the company is positioned to potentially deliver the first successful NAMPT (搜索) inhibitor to patients, addressing a target that has eluded drug developers for more than two decades.