The Food and Drug Administration (FDA) has granted Orphan Drug designation to RGLS4326 (Regulus Therapeutics) for the treatment of patients with autosomal dominant polycystic kidney disease.

Autosomal dominant polycystic kidney disease (ADPKD) is caused by mutations of the PKD1 and PKD2 genes that lead to the development of numerous fluid filled cysts primarily in the kidney. RGLS4326 is a novel oligonucleotide that works by inhibiting miR-17, which has been found to be upregulated in kidney cysts. Inhibition is therefore expected to reduce cyst proliferation and improve kidney function. 

In preclinical studies, the investigational agent showed direct regulation of the PKD1 and PKD2 genes, as well as a reduction in kidney cyst formation in human in vitro ADPKD models. Additionally, RGLS4326 was observed to be well tolerated, with no serious adverse events, in a multiple ascending dose study among healthy volunteers.

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The Company is initiating a phase 1b study to evaluate short term treatment with RGLS4326 in patients with ADPKD. As for treatment of extended duration, the FDA has placed the Investigational New Drug Application on partial clinical hold until certain requirements have been met.

“We are pleased RGLS4326 has been granted Orphan Drug designation by FDA,” said Jay Hagan, CEO of Regulus. “This is an important milestone for our ADPKD program and our efforts to address the significant unmet medical needs with this disease.”  

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This article originally appeared on MPR