Exhibit 99.2
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Dicerna Announces Positive Clinical Data for Investigational Treatments RG6346 for Chronic
Hepatitis B Virus and Nedosiran for Primary Hyperoxaluria, and Presents Preclinical Data Applying
RNAi Technology in New Tissues
– In the Ongoing Phase 1 Proof-of-Concept Trial Multidose Group, RG6346 1.5, 3.0 and 6.0 mg/kg Dosing
Cohorts Reached Mean HBsAg Reduction From Baseline of 1.39, 1.80 and 1.84 Log10 IU/mL, Respectively, at
Day 112 End-of-Treatment; Data for Earliest-Dose Cohort of 1.5 mg/kg Showed Durability of Response Through
Day 336 –
– Self-Resolving ALT Elevations With Preserved Liver Function in Several Participants Treated With RG6346
Suggest ALT Flares Potentially as a Result of Treatment-Induced Enhanced Immune Responses –
– In the Ongoing PHYOX™3 Multidose, Open-Label Extension Trial of Nedosiran, 82% (Nine of 11 Participants)
Achieved Normal or Near-Normal Levels of Key Primary Hyperoxaluria Measure at Day 120 –
– Company Presents for First Time Preclinical Data of Proprietary RNAi Technology in Extrahepatic Tissues –
– Company to Host Virtual R&D Event Today at 10:00 a.m. ET –
LEXINGTON, Mass., Aug. 6, 2020 – Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced positive data from its Phase 1 proof-of-concept trial of RG6346, an investigational candidate for the treatment of chronic hepatitis B virus (HBV) infection in development in collaboration with Roche, and from its PHYOX3 open-label trial of nedosiran, an investigational candidate for the treatment of primary hyperoxaluria (PH). These data will be highlighted today as part of the Company’s virtual R&D Day event taking place from 10:00 a.m. to noon ET. Today’s event will also feature the Company’s first public presentation of preclinical data highlighting Dicerna’s proprietary RNAi technology in multiple new tissue types.
“The data presented today represent a cross-section of early- to late-stage results that highlight the power and significant potential of Dicerna’s RNAi technology platform, the benefit of innovative and thoughtfully designed development strategies, and our ability to generate a pipeline of potentially best-in-class new therapies for rare and prevalent diseases,” said Douglas Fambrough, Ph.D., president and chief executive officer of Dicerna. “I am very excited by the results we are seeing across our portfolio, demonstrating the strength of our RNAi technology platform and clinical enterprise, which together form the strong foundation upon which we are building our business to evolve into a fully integrated, commercial-stage biopharmaceutical company.”
RG6346 Phase 1 Proof-of-Concept Trial for Treatment of Chronic Hepatitis B Virus Infection
RG6346 is an investigational GalXC™ RNAi therapeutic candidate in Phase 1 for the treatment of chronic HBV infection. The ongoing Phase 1 proof-of-concept trial in adults comprises three groups: Group A, a dose-ranging cohort with healthy volunteers, which was completed last year; Group B, composed of newly diagnosed participants not on any antiviral therapy who received a single dose of RG6346; and Group C, which includes participants concurrently receiving nucleoside analog (NUC) therapy and four monthly doses of one of three dose levels of RG6346. In the Phase 1 study, nine of 10 participants who received RG6346 and have