Rapafusyn Pharmaceuticals to Present In Vivo Data on ENT1 Inhibitor for Acute Kidney Injury at the American Society for Nephrology (ASN Kidney Week 2024)


BALTIMORE, Oct. 17, 2024 (GLOBE NEWSWIRE) -- Rapafusyn Pharmaceuticals, a leader in the field of non-degrading molecular glues, is pleased to announce the upcoming presentation of preclinical in vivo data from its selective ENT1 inhibitor for Acute Kidney Injury (AKI) at the American Society for Nephrology (ASN) Kidney Week 2024 to be held from October 24-27, 2024, in San Diego, CA.

Rapafusyn’s lead program, RAP-0001, is advancing rapidly to address a critical unmet need: the prevention of Acute Kidney Injury (AKI) in patients undergoing cardiac surgery. AKI is a severe and common complication, leading to prolonged hospital stays, increased morbidity, and higher mortality rates. With no approved therapies currently available, novel solutions are urgently needed.

RAP-0001, a potent and selective inhibitor of the ENT1 transporter, has shown preclinical efficacy in preventing ischemia/reperfusion injury, a major driver of AKI. “This compound was discovered through the RapaGlue™ platform. As a molecular glue, RAP-0001 uniquely binds to the intracellular chaperone protein FKBP12 and ENT1 to form a ternary complex. To our knowledge, it's the only SLC inhibitor that targets the intracellular domain of a transporter,” said Dr. Matthew Olson, VP of Biological Sciences of Rapafusyn, who will present in vivo data demonstrating RAP-0001’s ability to significantly reduce kidney damage in this high-risk context on October 25th, 2024.

Dr. Sean Hu, CEO of Rapafusyn, stated, “We believe RAP-0001 could change the standard of care for AKI, providing a critical therapeutic option where none currently exist. This is yet another example of recent developments demonstrating how non-degrading molecular glues could potentially tackle challenging disease targets and address high unmet medical needs in ways more effective than other drug modalities.”

About Rapafusyn

At Rapafusyn, we’re creating non-degrading molecular glues to address hard-to-drug targets. Using our FKBP12-binding macrocyclic peptide platform, we've built large DNA encoded libraries (DELs) and arrayed libraries, yielding novel starting points. Our approach targets intracellular proteins and domains of transmembrane proteins like SLCs, ion channels, and GPCRs. With a modular design, we rapidly optimize key drug properties to accelerate drug-discovery.

For more information, please visit https://www.rapafusyn.com/ or contact Heather Lavin at hlavin@rapafusyn.com.