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Investors - Regulus Therapeutics Regulus Therapeutics Reports Third Quarter 2021 Financial Results and Recent Updates -Company advances RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease ( ADPKD ) - -Validating sections of IND - enabling toxicity studies for RGLS8429 completed- -IND submission and Phase 1 initiation expected in Q2 2022- -Data from first cohort of patients in Phase 1b clinical trial of first - generation compound , RGLS4326 , presented at ASN Kidney Week and Biomarkers for Rare Diseases Summit- SAN DIEGO , Nov. 10 , 2021 / PRNewswire / -- Regulus Therapeutics Inc. ( Nasdaq : RGLS ) , a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs ( the " Company " or " Regulus " ) , today reported financial results for the third quarter ended September 30 , 2021 and provided a corporate update . " We are pleased with the progress we have made in advancing our next generation candidate RGLS8429 as a potential treatment for ADPKD , as the compound has been shown to have the favorable properties of RGLS4326 without the limitations of the first - generation compound , " commented Jay Hagan , CEO of Regulus Therapeutics . " RGLS8429 has demonstrated comparable potency , as well as similar pharmacokinetic and pharmacodynamic profiles , without the off - target CNS effects seen in chronic preclinical toxicology studies with RGLS4326 at the top doses tested . We look forward to our pre - IND meeting in December with the FDA to help finalize our IND submission and reach another important milestone in our mission to improve the lives of ADPKD patients . " Program Updates RGLS8429 for ADPKD : In October 2021 , the Company discontinued development of its first - generation compound , RGLS4326 , to allocate resources and efforts towards the development of its more promising next - generation compound , RGLS8429 . The Company believes RGLS8429 has demonstrated a superior pharmacological profile , with the absence of the off - target central nervous system ( CNS ) effects that were seen with RGLS4326 at the top doses tested in chronic preclinical toxicology studies . RGLS8429 has also shown equal potency to RGLS4326 for its molecular target ( miR - 17 ) in both in - vitro and in - vivo efficacy studies . The Company expects to have a pre - IND meeting with the U.S. Food and Drug Administration ( FDA ) for RGLS8429 before year - end and is on track for an IND submission and initiation of clinical development in the second quarter of 2022 , subject to FDA clearance of the IND . The Company's Phase 1 plans include a single dose escalation study in healthy volunteers to enable a multi - dose escalation study in ADPKD patients around the dose levels where robust clinical biomarker effects were demonstrated with RGLS4326 . The Company anticipates reporting top - line biomarker data in the first cohort of RGLS8429 - treated patients in early 2023 . RGLS4326 for ADPKD : On November 4 and November 9 , data from the first cohort of patients in the Phase 1b clinical trial of RGLS4326 , the Company's first - generation compound , for the treatment of ADPKD , was presented at the American Society of Nephrology ( ASN ) Kidney Week , and at the Biomarkers for Rare Diseases Summit . In the first cohort , nine patients were enrolled and received 1 mg / kg of RGLS4326 subcutaneously every other week for four doses . The mean increase in polycystins 1 and 2 at the end of study compared to baseline levels for all nine patients in the first cohort were 58 % ( p = .0004 ) and 38 % ( p = .026 ) , respectively . These data demonstrate clinical evidence that treatment with RGLS4326 increased PC1 and PC2 , most likely through inhibition of miR - 17 in the kidney of patients with ADPKD . These results also imply that overexpressed miR - 17 in ADPKD patients represses Pkd1 and Pkd2 expression , further validating miR - 17 as a therapeutic target for ADPKD treatment . The details for each presentation are below : ASN Kidney Week ePoster : Poster Title : RGLS4326 Increases Urinary PC1 and PC2 Levels in Individuals with Autosomal Dominant Polycystic Kidney Disease ( ADPKD ) Poster Date and Time : Thursday , November 4 , 2021 , 10:00 AM PDT Poster Number : PO1244 Biomarkers for Rare Diseases Summit Presentation : Presentation Title : Results from the First Cohort of Phaselb Clinical Trial of RGLS4326 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease ( ADPKD ) Presenter : Edmund Lee , PhD , Executive Director , Biology , Regulus Therapeutics Presentation Date and Time : Tuesday , November 9 , 2021 , 9:30 AM PDT A copy of each presentation is available at www.regulusrx.com/publications/ Financial Results Cash Position : As of September 30 , 2021 , Regulus had $ 35.8 million in cash and cash equivalents .