Dicerna Pharmaceuticals Launches Phase 1 Trial of DCR-AUD, an RNAi Therapeutic for Alcohol Use Disorder Affecting 283 Million Globally

Dicerna Pharmaceuticals today announced that it has completed dosing of the first cohort of subjects in a Phase 1 clinical trial evaluating its investigational RNAi therapy DCR-AUD in patients with alcohol use disorder (AUD).

Alcohol use disorder is a chronic disease characterized by an inability to stop or control alcohol use despite adverse social, occupational, or health consequences. Symptoms may include compulsive drinking, loss of control over alcohol consumption, and a negative emotional state when not drinking. AUD is one of the most common mental disorders; according to data from the World Health Organization, AUD affects approximately 283 million people worldwide.

DCR-AUD is a GalXC RNAi therapeutic candidate developed by Dicerna, designed to silence the expression of mRNA encoding aldehyde dehydrogenase 2 (ALDH2) in the liver. In nonclinical studies, DCR-AUD durably reduces hepatic mRNA encoding ALDH2. In individuals carrying ALDH2 gene mutations, uncomfortable physiological reactions, including flushing, headache, and nausea, occur shortly after alcohol consumption. This is considered the reason why patients with ALDH2 mutations are less susceptible to alcohol use disorder (AUD).

Based on human genetic data, Dicerna designed DCR-AUD, indicating that silencing ALDH2 mRNA in individuals with AUD may elicit similar physiological feedback, thereby preventing harmful levels of alcohol consumption.

Image source: Dicerna official website

This study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of DCR-AUD in healthy volunteers. The trial will also assess the interaction between DCR-AUD administration and alcohol consumption.

References:

[1] Dicerna Initiates Phase 1 Clinical Trial of DCR-AUD, a Novel Investigational GalXC™ RNAi Therapeutic Candidate for the Treatment of Alcohol Use Disorder. Retrieved September 28, 2021, from https://investors.dicerna.com/news-releases/news-release-details/dicerna-initiates-phase-1-clinical-trial-dcr-aud-novel

（Original text abridged）

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