Chicago, IL, September 30, 2025 – Sophrosyne Pharmaceuticals, a clinical stage biopharmaceutical company dedicated to developing novel therapies for patients suffering from Alcohol Use Disorder (AUD), related conditions, and other drug addictions, announced today that it has received clearance from the FDA to initiate a phase 1 study for its lead compound, SOPH-110S, in healthy volunteers. This first-in-human (FIH) study is designed to demonstrate the safety and efficacy of SOPH-110S as a potential treatment for Alcohol Use Disorder (AUD) via an adaptive design approach. The study will include the safety components of a single ascending dose (SAD) and multiple ascending dose (MAD, as well as an assessment of efficacy in a proof-of-concept alcohol challenge study.
“We are very excited to have received clearance to proceed into the first human study with our lead novel new chemical entity (NCE), SOPH-110S,” stated Herm Cukier, CEO of Sophrosyne Pharmaceuticals. “Our proprietary asset is highly active and selective agent targeting the inhibition of ALDH2, an enzyme required for the breakdown of ethanol in humans and holds the promise of bringing new clinical solutions for a disease state that has had little innovation over the last several decades. Alcoholism remains a leading cause of preventable morbidity and mortality in the U.S. and around the world with more than 200 thousand estimated deaths per year in the U.S. alone. Sophrosyne’s novel compounds hold the promise of transforming the treatment experience for the roughly 30 million patients of AUD in the U.S. and hundreds of millions more globally.”
The study is titled “A Phase I, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose, Multiple Ascending Dose, and Exploratory Alcohol Challenge Study to Evaluate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Profile of SOPH- 110S for Subcutaneous Injection in Healthy Adult Subjects.” The study is an adaptive design using an efficient methodology to identify and secure confirmatory evidence of the safety, tolerability, and efficacy for the ideal dose of SOPH-110S to proceed with in future patient studies. Analysis of the data will also inform on the durability of the efficacy effect for SOPH- 110S. The study will consist of up to 104 subjects and is expected to be completed within 12 months from the time of initiation. Sophrosyne expects to start the study by early 1Q26.
Alcohol Use Disorder (AUD), encompassing the conditions referred to as alcoholism, alcohol abuse, and alcohol addiction, is a chronic disease characterized by uncontrolled drinking and the preoccupation with alcohol despite adverse social, occupational, or health consequences. AUD is the inability to control drinking due to both a physical and emotional dependence on alcohol. Considered a brain disorder, AUD may create lasting changes in the brain, thus perpetuating the need and consumption of alcohol despite the resulting consequences. AUD affects approximately 30 million Americans and has a relapse rate of more than 90% with today’s treatments. It is a leading cause of preventable morbidity and mortality leading to more than 200 thousand deaths in the U.S. Hundreds of millions of people are estimated to suffer from AUD worldwide.
Sophrosyne Pharmaceuticals is a clinical stage biopharmaceutical company dedicated to developing and commercializing novel therapies for patients suffering from Alcohol Use Disorder (AUD), related conditions, and other drug addictions. The Company is planning to initiate clinical studies with its leading new chemical entity (NCE) in the United States in the coming months for AUD, as well as further advance its other novel development programs. The Company’s NCEs are patent protected globally through 2042.
This press release contains, or may contain, forward-looking statements that involve significant risks and uncertainties. Actual results may differ materially from those expressed or implied in such forward-looking statements due to various factors and circumstances, without limitation, many of which are outside of Sophrosyne Pharmaceuticals’ control. Readers are cautioned not to place undue reliance on these forward-looking statements which speak only as of the date of this press release. Sophrosyne Pharmaceuticals undertakes no obligation to update or revise its forward-looking statements to reflect future events, circumstances, or otherwise.
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