Regulus Therapeutics Announces First Subject Dosed in Phase 1 Single-Ascending Dose (SAD) Clinical Trial of RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- Category: DNA RNA and Cells
- Published on Tuesday, 14 June 2022 10:09
- Hits: 1884
SAN DIEGO, CA, USA I June 13, 2022 I Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced the dosing of the first healthy volunteer in the Phase 1 SAD study of RGSL8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD).
"The initiation of the clinical trial of RGLS8429 with the dosing of the first subject marks another important milestone for our program targeting the treatment of ADPKD" said Jay Hagan, President and Chief Executive Officer of Regulus Therapeutics. "There is a significant unmet need for patients with this disease and we look forward to the continued advancement of this trial."
The Company is conducting a Phase 1 SAD study in healthy volunteers to assess safety, tolerability, and pharmacokinetics of RGLS8429. Following the SAD study, the Company plans to initiate a Phase 1b multiple ascending dose (MAD) study to assess safety, tolerability, and pharmacokinetics of RGLS8429 in adult patients with ADPKD, and to evaluate the efficacy of RGLS8429 treatment across three different dose levels including changes in polycystins, cystic kidney volume (htTKV), and overall kidney function.
Autosomal Dominant Polycystic Kidney Disease (ADPKD), caused by 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. Approximately 160,000 individuals are diagnosed with the disease in the United States alone, with an estimated global prevalence of 4 to 7 million.
RGLS8429 is a novel, next generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity have been demonstrated along with a superior pharmacologic profile in preclinical studies compared to Regulus' first-generation compound.
Regulus Therapeutics Inc. (Nasdaq: RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. Regulus maintains its corporate headquarters in San Diego, CA.
SOURCE: Regulus Therapeutics