Boston – September 17, 2026 -- Xylo Bio has dosed the first participants in a Phase 1 clinical trial of XYL-1001, a selective 5-HT2A receptor agonist targeting major depressive disorder (MDD), marking the company's transition to a clinical-stage biotechnology organization.
Phase 1 trial spans single and multiple ascending dose cohorts in Australia
The randomized, double-blind, placebo-controlled study is evaluating safety, tolerability, pharmacokinetics, and pharmacodynamic activity of XYL-1001 in healthy volunteers. The trial design incorporates translational biomarkers intended to characterize pharmacodynamic engagement and inform future clinical development decisions.
Compound designed to avoid hallucinogenic effects tied to psychedelic therapies
XYL-1001 is described by the company as the most selective 5-HT2A receptor agonist in its category and the most advanced such candidate in clinical development. Unlike psychedelic-based treatments, it is designed to activate the 5-HT2A receptor without producing hallucinogenic effects, removing the need for medical supervision and specialized sites of care associated with existing psychedelic therapies. In preclinical models, XYL-1001 demonstrated 5-HT2A-dependent neuroplastic effects and efficacy against anxiety and depressive-like behaviors without signs of hallucinogenic-related behavior.
Depression affects 332 million people globally, with one-third of patients failing current treatments
According to the World Health Organization, depressive disorders affect an estimated 332 million people worldwide and remain among the leading causes of disability globally. Up to one-third of patients fail to achieve adequate response after two or more treatment attempts with existing antidepressants, which also carry delayed onset and undesirable side effects.
Company showcases program at Boston neuropsychiatric drug summit
Xylo Bio is presenting the XYL-1001 program this week at the 9th Neuropsychiatric Drug Development Summit in Boston, running September 15-17, where Chief Scientific Officer and co-founder Dr. Samuel Banister is speaking. Dr. Gabriel Vargas, Chief Clinical Advisor at Xylo Bio, said the Phase 1 study is designed to rigorously characterize the compound's safety and pharmacology from the outset. XYL-1001 remains an investigational product and has not been approved by any regulatory authority.