Today, Regulus (NASDAQ:RGLS) Therapeutics Inc., a biopharmaceutical company focused on developing innovative treatments, announced encouraging top-line results from a key study of its investigational drug RGLS8429, designed to treat autosomal dominant polycystic kidney disease (ADPKD). The findings come from the third patient cohort in the Phase 1b multiple-ascending dose study, which is assessing the safety, tolerability, and pharmacokinetics of the drug.
In this double-blind, placebo-controlled trial, 16 participants were randomized to receive either 3 mg/kg of RGLS8429 or a placebo every other week for three months. The treatment was well-tolerated with no significant safety concerns reported. Notably, the study demonstrated a dose-response relationship, with the 3 mg/kg dose showing more consistent increases in urinary levels of polycystins 1 and 2 (PC1 and PC2), proteins that inversely correlate with disease severity.
Furthermore, a statistically significant change from baseline in PC1 and PC2 levels was observed at the 3 mg/kg dose compared to placebo. Preliminary imaging analysis suggested that RGLS8429 may reduce kidney volume, measured as height-adjusted total kidney volume (htTKV), after three months of dosing, with 70% of subjects showing reductions at the 3 mg/kg level.
The company has initiated enrollment for a fourth cohort, which will involve up to 30 patients receiving a fixed dose of 300 mg of RGLS8429 every other week for three months.
This news is based on a press release statement from Regulus Therapeutics Inc . filed with the SEC today.
InvestingPro Insights
In light of Regulus Therapeutics Inc.'s recent announcement regarding their promising study results, current metrics from InvestingPro offer a snapshot of the company's financial health and market performance. Regulus, with a market capitalization of $164.32 million, appears to be navigating a challenging financial landscape. The company's P/E ratio stands at -1.73, reflecting investor concerns about profitability, a sentiment echoed by the adjusted P/E ratio for the last twelve months as of Q1 2024, which is -5.24.
InvestingPro Tips suggest that while Regulus holds more cash than debt, indicating some financial stability, analysts are concerned about its cash burn rate and weak gross profit margins. Additionally, despite a high return over the last year and a strong return over the last month, analysts do not expect the company to be profitable this year. On the positive side, the company's liquid assets exceed short-term obligations, providing some cushion for its operations.
For those interested in a deeper dive into Regulus Therapeutics' financials and market potential, InvestingPro offers additional insights. There are more tips available on the platform, which can be accessed with the coupon code PRONEWS24 for an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
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