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TM Dicerna Dicerna Announces Second Quarter 2021 Financial Results and Provides a Business Update August 9 , 2021 TM - Reported Positive Top - Line Data From Pivotal PHYOX ™ M2 Clinical Trial of Nedosiran Investigational GalXC ™ M RNAi Therapy for Treatment of Primary Hyperoxaluria - - Announced U.S. Food and Drug Administration ( FDA ) Clearance of Investigational New Drug ( IND ) Application for DCR - AUD for Alcohol Use Disorder - - Announced Interim Phase 1 Results and Initiated Patient Dosing in ESTRELLA Phase 2 Study of Belcesiran for the Treatment of Alpha - 1 Antitrypsin Deficiency - Associated Liver Disease ( AATLD ) - - Received $ 10.0 Million Milestone Payment Following FDA Acceptance of Lilly's Second GalXC RNAi IND Application Under Companies ' Global Research Collaboration and Licensing Agreement - - Company Reported $ 709.6 Million in Cash , Cash Equivalents and Held - to - Maturity Investments as of June 30 , 2021 - - Management to Host Conference Call Today at 8:00 a.m. ET LEXINGTON , Mass .-- ( BUSINESS WIRE ) -- Aug . 9 , 2021-- Dicerna Pharmaceuticals , Inc. ( Nasdaq : DRNA ) ( the " Company " or " Dicerna " ) , a leading developer of investigational ribonucleic acid interference ( RNAi ) therapeutics , today reported its financial results for the quarter ended June 30 , 2021 and provided a corporate update . " We have seen significant progress across our core and collaborative pipelines over the past few ns , including the achievement of a major milestone in our maturation as a company , " said Douglas Fambrough , Ph.D. , President and Chief Executive Officer at Dicerna . " Specifically , our recently announced PHYOXTM2 results represent our first positive pivotal trial readout . PHYOX2 generated robust data , meeting the primary and key secondary efficacy endpoints , and nedosiran was generally well tolerated in the trial . Based on these results , we believe nedosiran has significant potential as a treatment for patients with PH1 , and we are on course to submit a New Drug Application to the FDA for the treatment of PH1 in the fourth quarter . Notably , these positive results reflect broadly on our GalXCTM RNAi platform and bode well for our additional proprietary and partnered programs . " As positive as these results are for PH1 , the inconsistent data seen specifically in participants with PH2 have led us to make the strategic decision not to move forward with our plan to build Dicerna into a fully integrated commercial enterprise to support nedosiran , " Dr. Fambrough continued . " Instead , we intend to pursue commercial out - licensing opportunities to help ensure global access to nedosiran , subject to necessary approvals . This approach will allow us to deploy our capital and talent on our discovery and development pipeline efforts with our GalXC and GalXC - PlusTM RNAi investigational therapeutics for ourselves and our partners . With these strategic adjustments focused on our core strengths , we can extend our cash runway into 2025. " Recent Updates • Positive Top - Line Data From Pivotal PHYOX2 Clinical Trial of Nedosiran for Primary Hyperoxaluria ( PH ) . Dicerna announced in August positive top - line results from the pivotal PHYOX2 clinical trial of nedosiran . 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 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 . The Company expects the results from the PHYOX2 trial to support marketing authorization applications for the treatment of PH1 in the U.S. and other major markets and intends to submit a New Drug Application ( NDA ) to the U.S. Food and Drug Administration ( FDA ) in the fourth quarter of 2021 . • Announced Clearance of Investigational New Drug ( IND ) Application for DCR - AUD for the Treatment of Alcohol Use Disorder ( AUD ) . In July 2021 , Dicerna announced U.S. Food and Drug Administration ( FDA ) clearance of the IND application for DCR - AUD , the Company's investigational GalXC RNAi candidate for the treatment of AUD . Dicerna plans to initiate a Phase 1 trial in the third quarter of 2021 to evaluate the safety , tolerability , pharmacokinetics and pharmacodynamics of single ascending doses of DCR - AUD in healthy volunteers . • 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 alpha - 1 antitrypsin ( AAT ) deficiency - associated liver disease ( 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 ) showed 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