Alto Neuroscience, Inc. (NYSE: ANRO), a clinical-stage biopharmaceutical company focused on innovative treatments for neuropsychiatric disorders, announced today that its Phase 2b trial of ALTO-100 for major depressive disorder (MDD) did not achieve its primary endpoint, as measured by the Montgomery-Åsberg Depression Rating Scale (MADRS) compared to placebo. However, the drug maintained a favorable safety and tolerability profile consistent with prior studies.
CEO Amit Etkin, M.D., Ph.D., expressed disappointment over the results, noting the significant unmet need in MDD. He praised the team’s efforts in conducting a pioneering precision biomarker-based study and emphasized their commitment to advancing precision medicine in psychiatry. Alto expects to analyze the complete data set to inform future steps and is confident in its financial position to support ongoing clinical milestones.
Study Overview
The randomized, double-blind, placebo-controlled Phase 2b study involved 301 adults with MDD and was conducted at 34 sites across the U.S. The study aimed to assess ALTO-100’s efficacy in a biomarker-defined patient group. Key findings include:
- No statistically significant improvement in depressive symptoms for ALTO-100 compared to placebo.
- Lack of benefit over placebo in key secondary analyses.
- ALTO-100’s safety profile remained consistent, with common side effects like headache and nausea reported at similar rates to placebo.
Chief Medical Officer Adam Savitz, M.D., Ph.D., acknowledged that while the Phase 2b results differed from earlier trials, the biomarker-focused approach represents a promising direction in neuropsychiatric drug development.
ALTO-100 is also being studied as an adjunctive treatment in bipolar depression.
Alto anticipates its current cash reserves will support operations through 2027, with several clinical readouts, including data on ALTO-203 and ALTO-300 in MDD expected in early 2025.
About Alto Neuroscience
Alto Neuroscience aims to redefine psychiatry by developing personalized treatment options through its Precision Psychiatry Platform™, which utilizes brain biomarkers from EEG, neurocognitive assessments, and wearable data to identify patients likely to respond to its drug candidates. The company’s pipeline includes candidates targeting depression, PTSD, schizophrenia, and other mental health disorders.
