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TM Dicerna Dicerna Announces Third Quarter 2021 Financial Results and Provides a Business Update November 9 , 2021 - Reported Positive Top - Line Data From Pivotal PHYOXTM2 Clinical Trial of Nedosiran Investigational GalXCTM RNAi Therapy for Treatment o Primary Hyperoxaluria ( PH ) — - Initiated Phase 1 Clinical Trial of DCR - AUD for the Treatment of Alcohol Use Disorder ( AUD ) - - Company Plans to Unveil First GalXC - PlusTM Extrahepatic Target in Early 2022 - - Cash Runway Extends Into 2025 - Company to Host Conference Call Today at 8:30 a.m. ET - LEXINGTON , Mass .-- ( BUSINESS WIRE ) -- Nov . 9 , 2021-- Dicerna Pharmaceuticals , Inc. ( Nasdaq : DRNA ) , a leading developer of investigational ribonucleic acid interference ( RNAi ) therapeutics , today reported its financial results for the third quarter ended September 30 , 2021 and provided a business update . " We had an exciting and productive third quarter as we continued to execute and advance key initiatives that we believe help position Dicerna for multiple value - creating milestones over the next 12 to 24 months , " said Douglas Fambrough , Ph.D. , President and Chief Executive Officer at Dicerna . " We look to build on this progress as we continue advancing core programs that include nedosiran for primary hyperoxaluria ( PH ) , RG6346 for chronic hepatitis B virus ( HBV ) infection with Roche , belcesiran for alpha - 1 antitrypsin deficiency - associated liver disease ( AATLD ) and DCR - AUD for alcohol use disorder ( AUD ) . We also look forward to unveiling the next wave of innovations from Dicerna in early 2022 with the first of several wholly owned extrahepatic programs harnessing our GalXC - Plus technology . " Dr. Fambrough continued , " Between Dicerna and our collaborative partners , we have 16 programs in preclinical or clinical development as well as more than 20 discovery - stage programs . Given the breadth of these activities , we expect new programs to be entering the clinic , on average , one per quarter over the next two years and potentially beyond that at a similar rate . We believe this creates the opportunity for Dicerna to dramatically expand in the future and meaningfully advance our mission to improve the lives of patients . " Corporate Highlights • Announced Positive Top - Line Data From Pivotal PHYOX2 Clinical Trial of Nedosiran for Primary Hyperoxaluria . In August 2021 , Dicerna announced positive top - line results from the pivotal PHYOX2 clinical trial of nedosiran , Dicerna's late - stage investigational GalXC RNAi therapeutic candidate in development for PH . Nedosiran achieved the primary endpoint in the PHYOX2 trial , demonstrating a statistically significant reduction from baseline in urinary oxalate ( Uox ) excretion compared to placebo ( p < 0.0001 ) . The study also achieved the key secondary endpoint , with a significantly higher proportion of patients given nedosiran achieving and sustaining normal or near - normal Uox at two or more consecutive visits after Day 90 compared to placebo ( p = 0.0025 ) . Uox reductions were significant in participants with PH1 while participants with PH type 2 ( PH2 ) ( 5 nedosiran and 1 placebo ) showed inconsistent results in this trial . Nedosiran was generally well tolerated in the study with an overall adverse event ( AE ) profile consistent with previously reported data from PHYOX trials . • Presented PHYOX2 Data as Late - Breaker Poster Presentation at American Society of Nephrology ( ASN ) Kidney Week 2021. The data from Dicerna's pivotal PHYOX2 study were presented at ASN on November 4 , 2021 . • Reported Top - Line Results for PHYOX4 Single - Dose Study of Nedosiran in Primary Hyperoxaluria Type 3 ( PH3 ) . In October 2021 , Dicerna announced that its PHYOX4 study designed to evaluate the safety and tolerability of a single subcutaneous dose of nedosiran compared to placebo in patients with PH3 met its primary safety endpoint . Patients administered nedosiran also showed a trend in Uox reduction ; however , these reductions did not meet prespecified secondary efficacy endpoint criteria . • Initiated Phase 1 Clinical Trial of DCR - AUD for the Treatment of Alcohol Use Disorder . In September 2021 , Dicerna announced that it dosed the first subjects in its Phase 1 clinical trial to assess DCR - AUD , Dicerna's investigational GalXC RNAi therapeutic candidate in development for the treatment of AUD . The randomized , double - blind study is evaluating the safety , tolerability , pharmacokinetics and pharmacodynamics of single ascending doses of DCR - AUD in up to 36 healthy volunteers over a 24 - week observation period . The trial is also assessing the interaction between DCR - AUD administration and alcohol consumption using standardized Ethanol Interaction Assessments ( EIA ) performed serially over the trial's duration . • Reported Interim Data From Phase 1 Trial of Belcesiran . In July 2021 , Dicerna reported interim data from the Company's Phase 1 trial of belcesiran , a GalXC RNAi therapeutic candidate in development for the treatment of AATLD . The trial is designed to evaluate the safety , tolerability , pharmacokinetics and pharmacodynamics of belcesiran in healthy volunteers . Data from this interim analysis of the four completed active - treatment dose cohorts ( 0.1 , 1.0 , 3.0 and 6.0 mg / kg ) demonstrated dose - dependent reductions in serum AAT with administration of a single dose of belcesiran . In this analysis , belcesiran was found to have an acceptable safety profile and was generally well tolerated . Dosing in the final