Benzinga | Jul 29, 2021 07:54AM EDT

Dicerna Reports FDA Clearance Of Its Investigational New Drug Application For DCR-AUD For Treatment Of Alcohol Use Disorder

-- Novel ALDH2-Targeting GalXC(tm) RNAi Candidate Designed to Address a Highly Prevalent and Undertreated Disorder1 --

-- Dicerna Expects to Initiate Phase 1 Clinical Trial of DCR-AUD in the Third Quarter of 2021 --

LEXINGTON, Mass.--(BUSINESS WIRE)-- Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) ("Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced the U.S. Food and Drug Administration ("FDA") clearance of its Investigational New Drug (IND) application for DCR-AUD, the Company's investigational RNAi candidate for the treatment of alcohol use disorder (AUD).

AUD is a medical condition characterized by the inability to stop or control alcohol use despite social, occupational or health consequences.2 Aldehyde dehydrogenase 2, or ALDH2, is an enzyme that plays a key role in metabolizing alcohol. DCR-AUD has been shown to induce long-lasting liver-specific ALDH2 messenger RNA (mRNA) knockdown in nonclinical studies. By silencing ALDH2 in the liver and interrupting the alcohol metabolic pathway, DCR-AUD has the potential to induce real-time physiological feedback to help individuals seeking treatment for AUD regain control over harmful levels of alcohol use.

"The key to developing a successful treatment for the more than 14 million people affected by AUD is finding a way to help individuals regain control over their alcohol use," said Bob D. Brown, Ph.D., Chief Scientific Officer and Executive Vice President of R&D at Dicerna. "We believe that RNAi may be ideally suited to do this. RNAi's long duration of effect and target specificity can achieve liver-specific reduction of ALDH2, which could be an effective strategy to reduce alcohol consumption. We've performed extensive screening and optimization of GalXC(tm) RNAi molecules targeting human ALDH2 to identify DCR-AUD and believe DCR-AUD has the potential to improve health outcomes for those with AUD."

Dr. Brown continued, "We would also like to acknowledge the financial support we received from the National Institutes of Health in the form of a cooperative Small Business Innovation Research grant that has contributed to the development of DCR-AUD. The grant reviewers and the agency's votes of confidence in Dicerna's drug development capabilities and the liver-specific RNAi mechanism of action of GalXC have helped make our DCR-AUD program possible."

Dicerna plans to initiate a 24-week, randomized, double-blind, placebo-controlled Phase 1 trial in the third quarter of 2021 to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics of single-ascending doses of DCR-AUD in healthy volunteers. The trial will also assess the interaction between DCR-AUD treatment and alcohol consumption using standardized Ethanol Interaction Assessments.