Alto Neuroscience has received FDA Fast Track designation for its investigational therapy ALTO-101, aimed at treating cognitive impairment associated with schizophrenia (CIAS). CIAS is a core feature of schizophrenia that severely impacts memory, attention, executive function, and daily functioning, affecting millions of patients worldwide. Currently, there are no approved therapies specifically for this aspect of the disease.

ALTO-101 is a novel small molecule phosphodiesterase-4 (PDE4) inhibitor. By inhibiting PDE4, ALTO-101 increases levels of cyclic adenosine monophosphate (cAMP), enhancing neuronal signalling and synaptic plasticity, which are critical for learning and memory. Early Phase 1 data in healthy volunteers demonstrated significant pro-cognitive effects on EEG measures and cognitive performance, providing strong validation for the drug’s mechanism of action.

“Receiving Fast Track designation from the FDA underscores the critical need for new, effective treatments for patients suffering from cognitive impairment associated with schizophrenia,” said Amit Etkin, founder and chief executive officer of Alto Neuroscience.

“This designation is a significant milestone for the ALTO-101 program. We are committed to advancing this program expeditiously for the millions of patients who currently have no approved treatment options to address these debilitating cognitive deficits.”

The FDA’s Fast Track programme is designed to facilitate the development and expedite the review of therapies for serious conditions that address unmet medical needs. Drugs granted Fast Track designation may benefit from more frequent meetings with the FDA, rolling submissions of data, and eligibility for accelerated approval and priority review pathways, potentially speeding patient access.

ALTO-101 is currently being evaluated in an ongoing Phase 2 proof-of-concept study in patients with CIAS. The trial will provide further insight into the therapy’s safety, efficacy, and pro-cognitive effects in the target population. By directly targeting the cognitive deficits that drive functional impairment, ALTO-101 represents a potential first-in-class approach for an underserved aspect of schizophrenia.

Cognitive impairment in schizophrenia affects multiple domains including memory, attention, processing speed, and executive function. These deficits are a major determinant of poor functional outcomes, influencing patients’ ability to maintain employment, social relationships, and independent living. Addressing CIAS is considered a critical unmet need within neuropsychiatric care, highlighting the potential impact of therapies such as ALTO-101.