Alto Neuroscience’s ALTO-101 Gets FDA Fast Track for Schizophrenia-Related Cognitive Impairment

10/03/2025 – 07:34 AM

– ALTO-101, a novel PDE4 inhibitor, has demonstrated pro-cognitive effects in healthy volunteers –

– Designation highlights the significant unmet need for new treatments for cognitive impairment associated with schizophrenia (CIAS) –

– Enrollment remains ongoing in a Phase 2 proof-of-concept study of ALTO-101 in patients with CIAS –

MOUNTAIN VIEW, Calif. – Alto Neuroscience, Inc. (NYSE: ANRO), a clinical-stage biopharmaceutical firm concentrating on precision medicines for neuropsychiatric conditions, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ALTO-101 for the treatment of cognitive impairment associated with schizophrenia (CIAS). This regulatory decision arrives as CIAS continues to present a substantial challenge, with no currently approved treatments available, despite its significant impact on the daily lives and overall well-being of millions affected by schizophrenia.

The FDA’s Fast Track program is strategically designed to expedite the development and review processes for drugs exhibiting the potential to treat serious conditions and fulfill unmet medical needs. This designation opens the door for Alto Neuroscience to engage in more frequent dialogues with the FDA concerning ALTO-101’s development pathway. Furthermore, it potentially positions the drug for accelerated approval and priority review, contingent upon meeting the requisite criteria. This regulatory tailwind is critical as Alto Neuroscience navigates the complex path of bringing a novel therapy to market.

“The Fast Track designation from the FDA underscores the urgent demand for new and effective interventions for patients experiencing cognitive impairment linked to schizophrenia,” stated Amit Etkin, M.D., Ph.D., Founder and CEO of Alto Neuroscience. “We believe this milestone for the ALTO-101 program highlights its promise as a novel treatment strategy. The Phase 1 data, demonstrating considerable and clinically relevant effects of ALTO-101 on both EEG measures and cognitive performance among healthy volunteers, strongly validates its mechanism of action. Our commitment lies in rapidly advancing this program to serve the millions of patients currently lacking approved treatment options for these debilitating cognitive deficits.”

ALTO-101 is a novel small molecule inhibitor targeting phosphodiesterase-4 (PDE4). PDE4 plays a critical regulatory role in the brain, responsible for the degradation of cyclic adenosine monophosphate (cAMP), a key signaling molecule pivotal for neuronal communication and synaptic plasticity – processes foundational for learning and memory. By selectively inhibiting PDE4, ALTO-101 effectively boosts cAMP levels, theoretically strengthening neural circuits and subsequently enhancing cognitive functions. Schizophrenia often manifests in cognitive deficits characterized by impairments in attention, memory, and executive function, representing a severely under-addressed facet of the disease. Analysts view ALTO-101’s mechanism as a potentially significant advancement, contrasting with existing symptomatic treatments that often fail to address the core cognitive impairments associated with schizophrenia.

Understanding Cognitive Impairment in Schizophrenia (CIAS)

A core characteristic of schizophrenia is cognitive impairment, which manifests across various cognitive domains, including memory, attention, processing speed, and executive function. These cognitive deficits are primary drivers of diminished functional outcomes, affecting patients’ ability to maintain employment, forge and sustain social bonds, and live independently. Given the absence of FDA-approved treatments targeting CIAS directly, the condition constitutes a substantial unmet medical need for the global schizophrenia population.

Alto Neuroscience: A Precision Approach to Mental Health

Alto Neuroscience operates as a clinical-stage biopharmaceutical company whose mission is to transform the landscape of psychiatry through precision medicine. The company’s Precision Psychiatry Platform™ employs a comprehensive approach, analyzing brain biomarkers obtained through EEG activity, neurocognitive assessments, wearable sensor data, and other relevant factors. This rich dataset enables Alto to identify patients more likely to respond favorably to their pipeline of product candidates. The company’s clinical pipeline includes novel therapeutic agents aimed at addressing bipolar depression, major depressive disorder, treatment-resistant depression (TRD), schizophrenia, and related mental health disorders. Alto’s approach is predicated on the hypothesis that targeted therapies guided by individual patient biomarkers can lead to more effective and personalized treatment outcomes, a concept that is gaining traction within the psychiatric community as pharmaceutical companies seek to improve efficacy rates and reduce the trial-and-error nature of current treatment paradigms.

This press release contains forward-looking statements subject to the Private Securities Litigation Reform Act of 1995. These statements involve risks and uncertainties, including those related to the potential benefits, activity, effectiveness, and safety of Alto’s product candidates and Precision Psychiatry Platform; expectations for the design, progress, and results of clinical trials, including the Phase 2 POC trial of ALTO-101 in CIAS; and Alto’s ability to realize the benefits of the Fast Track designation. Actual results could differ materially due to various factors, including uncertainties inherent in clinical trials and other important factors detailed in Alto’s filings with the SEC. Alto disclaims any obligation to update these forward-looking statements except as required by law.

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