Regulus Therapeutics (NASDAQ: RGLS) stock soared over 70% on Tuesday after the company reported positive phase 1b ADPKD trial results.
Regulus Therapeutics (RGLS) made waves on Monday with the release of promising preliminary results from the second cohort of its Phase 1b clinical trial for RGLS8429, a potential treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD).
The trial demonstrated a dose-dependent response based on urinary and exploratory imaging-based biomarkers, showing significant reductions in kidney volume among patients with increased levels of polycystins 1 and 2 (PC1 and PC2).
The Phase 1b trial investigates RGLS8429 through a double-blind, placebo-controlled approach to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics. This study evaluates different weight-based dose levels, with the second cohort comprising 14 patients administered either a 2 mg/kg dose of RGLS8429 or a placebo every other week for three months. The medication demonstrated good tolerance with no observed safety issues.
Urinary measurements of PC1 and PC2 revealed heightened biological activity at the 2 mg/kg dosage in contrast to the 1 mg/kg dosage and placebo, especially following three months of treatment. Three patients exhibiting the most significant elevations in PC1 and PC2 demonstrated reductions in height-adjusted total kidney volume (htTKV) greater than 4%, coupled with a decline in total kidney cyst volume (TKCV).
The company plans to increase the fourth cohort’s sample size to 30 patients to explore further the impact on cystic volume in ADPKD patients. This decision follows encouraging results from the second cohort, indicating a potential effect on htTKV and TKCV, which the company will investigate with higher doses in the upcoming cohorts. The topline data from the third cohort is expected by mid-2024 while screening for the fourth cohort is slated to commence in the second quarter of 2024.
Regulus FDA Discussion
Regulus also confirmed a productive discussion with the FDA concerning an expedited approval pathway based on a solitary Phase 2 study of RGLS8429 for treating ADPKD. The company reported having around $23.8 million in cash and cash equivalents as of December 31, 2023.
Regulus Therapeutics (NASDAQ: RGLS) Stock Reaction
RGLS stock surged 71.01% to close at $2.36 on Tuesday. The traders had exchanged hands with 167,365,421 (167.36 million) shares compared to the average daily trading volume of 39.42K.
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