Dicerna Initiates Phase 1 Clinical Trial of DCR-AUD, a Novel Investigational GalXC™ RNAi Therapeutic Candidate for the Treatment of Alcohol Use Disorder
Sep 28, 2021
– Trial to Evaluate Safety and Tolerability of DCR-AUD in
– Trial Includes Ethanol Interaction Assessments to Evaluate Participants’ Response to Alcohol After Receiving DCR-AUD –
AUD is characterized by the inability to stop or control alcohol use despite social, occupational or health consequences.1 Treatment approaches include psychosocial support and pharmacotherapy, but many individuals with AUD do not receive treatment, and currently available pharmacotherapies are not widely prescribed.2 Overall, of the estimated 14 million individuals in the
“We designed DCR-AUD leveraging our GalXC RNAi technology to serve as a potential therapeutic that offers real-time physiological feedback to complement the evidence-based psychosocial treatments used to help individuals with AUD,” said
The randomized, double-blind Phase 1 clinical trial (NCT05021640) will evaluate single doses of DCR-AUD compared to placebo in up to 36 healthy volunteers over a 24-week observation period.
About Alcohol Use Disorder (AUD)
Alcohol use disorder, or AUD, is a chronic disorder characterized by the inability to stop or control alcohol use despite social, occupational or health consequences. AUD presents as a problematic pattern of alcohol use leading to clinically significant impairment or distress. Symptoms can include compulsive drinking, loss of control over alcohol use and negative emotions when not drinking.1 AUD is one of the most common psychiatric disorders, affecting more than 14 million adults in the
DCR-AUD is Dicerna’s GalXC™ RNAi investigational candidate designed to silence ALDH2 (aldehyde dehydrogenase 2) messenger RNA (mRNA) expression in the liver. DCR-AUD has been shown to induce long-lasting liver-specific ALDH2 mRNA knockdown in nonclinical studies. Some individuals are born with naturally occurring mutations in one or both gene copies that encode the ALDH2 enzyme. In these people with ALDH2 mutations, alcohol consumption can result in uncomfortable physiological effects that occur soon after drinking. These effects are thought to be the reason people with ALDH2 mutations are much less likely to be affected by AUD. Dicerna designed DCR-AUD based on human genetic data that suggest knocking down ALDH2 mRNA in individuals with AUD may provide similar physiological feedback that is protective against harmful levels of alcohol consumption. Preclinical research for DCR-AUD was supported by a grant from the
About Dicerna’s GalXC RNAi Platform Technologies
Dicerna’s proprietary GalXC™ RNAi platform aims to advance the development of next-generation RNAi-based therapies. Investigational therapeutics developed using our flagship GalXC technology utilize a proprietary N-acetyl-D-galactosamine (GalNAc)-mediated structure of double-stranded RNA molecules that are designed to bind specifically to receptors on liver cells, leading to selective hepatocyte internalization and access to the RNAi machinery within the cells. Dicerna is continuously innovating and exploring new applications for RNAi technology beyond GalNAc-mediated delivery to the liver, including alternative RNA structures and fully synthetic ligands that target other tissues and enable new therapeutic applications, referred to as GalXC-Plus™.
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Examples of forward-looking statements include, among others, statements we make regarding our product candidates and the development thereof, including about the Company’s Phase 1 clinical trial of DCR-AUD for the treatment of alcohol use disorder, including our trial design; expected timing of results from the Company’s Phase 1 clinical trial of DCR-AUD; the Company’s clinical development programs, including for DCR-AUD; the design and therapeutic potential of our product candidates, including DCR-AUD; the estimated market size for AUD and other diseases for which we are developing potential therapies to address; our business and operations, including the discovery, development and commercialization of our product candidates; the development of our RNAi technology platform, and the therapeutic potential thereof; and our collaboration with partners and any potential future collaborations.
The process by which investigational therapies, such as DCR-AUD, could potentially lead to an approved product is long and subject to highly significant risks. Applicable risks and uncertainties include those relating to Dicerna’s clinical research and other risks identified under the heading "Risk Factors" included in the Company’s most recent filings on Forms 10-K and 10-Q and in other future filings with the
2. Witkiewitz, K. et al. Advances in the science and treatment of alcohol use disorder.
4.Grant et al., JAMA Psychiatry 2015.
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