Alto Neuroscience, Inc. announced that its novel drug candidate, ALTO-101, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of cognitive impairment associated with schizophrenia (CIAS). This designation underscores the critical nature of the unmet medical need, as CIAS is a core, debilitating feature of schizophrenia that severely impacts daily functioning for millions of patients worldwide, and for which there are currently no approved treatments.

The FDA’s Fast Track process is designed to facilitate the development and expedite the review of drugs targeting serious conditions that address a significant unmet need, offering benefits such as more frequent consultation with the agency and potential eligibility for accelerated approval and priority review.

ALTO-101 is a novel small molecule phosphodiesterase-4 (PDE4) inhibitor, whose mechanism of action involves increasing levels of cyclic adenosine monophosphate (cAMP) in the brain. Since cAMP is crucial for neuronal signaling and synaptic plasticity—the foundations of learning and memory—inhibiting PDE4 is believed to enhance neural circuits and, consequently, improve the impaired attention, memory, and executive function seen in schizophrenia.

This potential is supported by encouraging Phase 1 data, which demonstrated significant and clinically relevant effects on both EEG measures and cognitive performance in healthy subjects. Alto Neuroscience's founder and CEO, Amit Etkin, M.D., Ph.D., stated that the designation is a significant milestone that attests to the potential of ALTO-101’s novel treatment approach. The company is currently advancing the program, with enrollment ongoing in a Phase 2 proof-of-concept study for patients with CIAS, as part of its mission to redefine psychiatry through its Precision Psychiatry Platform™ that leverages neurobiological biomarkers for developing personalized and effective treatments.