On November 18, 2019 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs, reported that Jay Hagan, President and Chief Executive Officer of Regulus, will present a company overview at the Stifel Healthcare Conference in New York City on Wednesday, November 20, 2019 (Press release, Regulus, NOV 18, 2019, View Source [SID1234551439]). The company overview will include an update on RGLS4326 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) and the Company’s recent submission to FDA in response to the Partial Clinical Hold.
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About ADPKD
ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.
About RGLS4326
RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of PKD1 and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold by the U.S. Food and Drug Administration.