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news.Biopharmaceutical company, Endocyte has announced that its targeted Small Molecule Drug Conjugate (SMDC) approach to the folate receptor (FR) may offer a treatment for patients with autosomal-dominant polycystic kidney disease (ADPKD).
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In a mouse model of PKD, treatment with a folate-conjugated form of rapamycin, FC-rapa, (EC0371) attenuated proliferation and growth of renal cysts and preserved renal function.
Endocyte has chemically synthesized EC0371 by linking the folic acid targeting ligand with the rapamycin drug payload.
EC0371 is a folate-targeted, water soluble form of rapamycin that selectively inhibits mTOR activity in polycystic kidneys to reduce abnormal growth.
Endocyte research and development vice president Christopher Leamon said currently, there is no pharmaceutical treatment available for PKD patients, and patients with end stage renal failure get lifelong dialysis or receive a kidney transplant.
”Our results with EC0371 are very exciting, demonstrating that we may be able to reduce the side effects observed in the clinical trials with untargeted rapamycin by delivering the drug directly to the cystic cells,” Leamon added.
Rapamycin is a potent inhibitor of mTOR while the mTOR pathway is deregulated in ADPKD, and its inhibition reverses renal cystogenesis in animal models.