Chicago, IL, June 18, 2026 - Sophrosyne Pharmaceuticals, a clinical stage biopharmaceutical company dedicated to developing novel therapies for patients suffering from Alcohol Use Disorder (AUD), related conditions, and other brain health afflictions, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its lead product, SOPH-110S, a novel ALDH2 inhibitor new chemical entity (NCE) intended to be a long-acting treatment option for AUD. Sophrosyne is currently conducting a comprehensive phase I trial employing an adaptive design using an efficient methodology to identify and secure 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.
FDA's Fast Track program is designed to facilitate the development and expedite the review of therapies intended to treat serious conditions and address unmet medical needs. Companies receiving Fast Track designation may benefit from more frequent interactions with the FDA, eligibility for rolling review, and potential qualification for priority review if relevant criteria are met.
"Receiving FDA Fast Track Designation is a major milestone for our Company and an exciting development for patients suffering with AUD," stated Herm Cukier, CEO of Sophrosyne Pharmaceuticals. "This is further validation that SOPH-110S, a highly active and selective agent targeting the inhibition of ALDH2, an enzyme required for the breakdown of ethanol in humans, can potentially be a new long-acting clinical solution for a disease state that has had little innovation over the last several decades. Alcoholism remains a leading cause of preventable morbidity and mortality leading to an estimated 200,000 deaths per year in the U.S."
Preclinical studies demonstrated that SOPH-110S is a highly potent and dose-dependent inhibitor of ALDH2. Furthermore, studies across several species highlighted an acceptable safety profile and NOAEL, as well as no off-target effects in a panel of 84 common targets, no CYP interactions, and no meaningful inhibition of the hERG channel.
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 approximately 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 brain health afflictions. The Company has initiated a clinical study with its lead new chemical entity (NCE) in the United States for AUD and is planning to further advance its other novel development programs. The Company's NCEs are globally patent protected through the mid-2040s.
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 employs an adaptive design using an efficient methodology to identify and secure 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 approximately 12 months from the time of initiation.
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.
Neil Chapman, Investor Relations, at nchapman@sophrosynepharma.com or visit the company website at www.sophrosynepharma.com
