Newleos Therapeutics, a clinical-stage biotechnology company developing treatments for neuropsychiatric disorders, has received a grant of up to $15.5 million from the National Institute on Drug Abuse (NIDA), part of the National Institutes of Health (NIH).
The five-year, milestone-based grant will support the development of NTX-2001 for cocaine use disorder (CUD). The funding is expected to cover a Phase 1 drug-drug interaction study and a subsequent Phase 2 efficacy study. It is Newleos’ first federal grant.
NTX-2001 is an investigational partial agonist of trace amine-associated receptor 1 (TAAR1). The company is developing the drug as a potential treatment for substance use disorders, including cocaine use disorder and alcohol use disorder (AUD). Newleos said the grant was awarded based on preclinical evidence and previously established safety and tolerability data in humans.
FDA Grants Fast Track Designation for NTX-2001
Newleos also announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to NTX-2001 for the treatment of alcohol use disorder.
Fast Track is designed to support the development and review of therapies for serious conditions with unmet medical needs. The designation may allow more frequent communication with the FDA and could make the drug eligible for priority review or accelerated approval, if applicable requirements are met.
Newleos is currently conducting a Phase 1b proof-of-concept study in the United States to assess NTX-2001’s effects on alcohol consumption compared with a placebo. The study will also evaluate safety, tolerability and pharmacokinetics in people with AUD.
Developing Treatments for Substance Use Disorders
NTX-2001 is designed to act on TAAR1, a receptor involved in regulating dopamine signaling in brain circuits associated with reward and reinforcement. The company is investigating whether the drug can help reduce the reinforcing effects of alcohol and other substances.
Newleos said NTX-2001 has been evaluated in previous clinical trials involving different formulations and dosing regimens, with consistent safety and tolerability results.
Cocaine use disorder remains an area of significant unmet medical need, with no FDA-approved medications specifically for its treatment. Newleos is developing NTX-2001 as a potential pharmacological option.
Newleos Therapeutics was founded in 2025 and licensed its pipeline from Roche. Its small molecule programs include targets such as GABAA-γ1, V1a, TAAR1, and GABAA-α5 with potential indications in anxiety, substance use disorders, and cognitive impairment. The company first announced a $93.5 million Series A financing, co-led by Goldman Sachs Alternatives.
