Regulus Therapeutics Inc. (RGLS) announced Monday positive topline results from the third cohort of patients in its Phase 1b Multiple-Ascending Dose (MAD) clinical trial of RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease or ADPKD.
In pre-market activity on the Nasdaq, Regulus shares were gaining around 14 percent to trade at $2.86.
The Phase 1b MAD study is a double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of RGLS8429 in adult patients with ADPKD.
The study is evaluating RGLS8429 treatment across three different weight-based dose levels and one fixed dose level, including measuring changes in urinary polycystins 1 and 2 (PC1 and PC2), htTKV, cyst architecture, and overall kidney function.
The company noted that enrollment in the fourth cohort, which will include up to 30 patients, has been initiated and patients will receive an open label fixed dose of 300 mg of RGLS8429 every other week for three months. In addition to PC1 and PC2 and safety, imaging biomarkers will also be evaluated.
In the third cohort, RGLS8429 was well tolerated with no safety concerns, and continued evidence of a mechanistic dose response was observed following completion of 3 mg/kg dose level based on urinary biomarker analyses PC1 and PC2.
Jay Hagan, CEO of Regulus Therapeutics, said, "Building on the positive results from our first two cohorts, these cohort 3 data, particularly the reduction in htTKV seen in the majority of patients, further strengthen our conviction in RGLS8429's ability to potentially address the underlying, genetic cause of ADPKD. We anticipate requesting an End-of-Phase 1 meeting with the FDA in the fourth quarter of this year and look forward to providing a data update from the open-label fourth and final cohort of the Phase 1b MAD study by year-end."
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