Company Announcements

– 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.--(BUSINESS WIRE)--Oct. 3, 2025-- Alto Neuroscience, Inc. (“Alto”) (NYSE: ANRO) a clinical-stage biopharmaceutical company focused on the development of novel precision medicines for neuropsychiatric disorders, today announced 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). There are currently no approved treatments for CIAS, a core feature of schizophrenia that severely impacts daily functioning and quality of life for millions of patients.

Fast Track designation is an FDA process designed to facilitate the development and expedite the review of drugs intended to treat serious conditions and fill an unmet medical need. A drug that receives Fast Track designation may be eligible for more frequent meetings with the FDA to discuss the drug’s development plan, as well as eligibility for accelerated approval and priority review, if relevant criteria are met.

“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, M.D., Ph.D., founder and chief executive officer of Alto Neuroscience. “This designation is a significant milestone for the ALTO-101 program and we believe it is a testament to its potential as a novel treatment approach. Our Phase 1 data, which demonstrated significant and clinically relevant effects of ALTO-101 on both EEG measures and cognitive performance in healthy subjects, provides strong validation for its mechanism. 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.”

ALTO-101 is a novel small molecule phosphodiesterase-4 (PDE4) inhibitor. The PDE4 enzyme plays a key role in the brain by breaking down cyclic adenosine monophosphate (cAMP), a molecule crucial for neuronal signaling and synaptic plasticity, which are fundamental to learning and memory. By inhibiting PDE4, ALTO-101 increases cAMP levels, which is believed to enhance neural circuits and improve cognitive function. The cognitive deficits in schizophrenia, which include impaired attention, memory, and executive function, represent a substantial and underserved aspect of the illness.

About Cognitive Impairment Associated with Schizophrenia (CIAS)

Cognitive impairment is a core and disabling feature of schizophrenia, affecting areas such as memory, attention, processing speed, and executive function. These deficits are a primary determinant of poor functional outcomes for patients, impacting their ability to work, maintain social relationships, and live independently. Currently, there are no approved treatments for CIAS, representing a significant unmet medical need for the millions of people living with schizophrenia worldwide.

About Alto Neuroscience

Alto Neuroscience is a clinical-stage biopharmaceutical company with a mission to redefine psychiatry by leveraging neurobiology to develop personalized and highly effective treatment options. Alto’s Precision Psychiatry Platform™ measures brain biomarkers by analyzing EEG activity, neurocognitive assessments, wearable data, and other factors to better identify which patients are more likely to respond to Alto product candidates. Alto’s clinical-stage pipeline includes novel drug candidates in bipolar depression, major depressive disorder, treatment resistant depression (TRD), and schizophrenia, and other mental health conditions. For more information, visit www.altoneuroscience.com or follow Alto on X.

Forward-Looking Statements

This press release may contain forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “expects,” “plans,” “will” and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding Alto’s expectations about the potential benefits, activity, effectiveness and safety of its product candidates and Precision Psychiatry Platform (“Platform”); statements regarding Alto’s expectations for the design, progress and results of its ongoing Phase 2 POC trial of ALTO-101 in CIAS; Alto’s ability to recognize the benefits of receiving Fast Track designation, Alto’s expectations with regard to the design and results of its clinical trials. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including uncertainties inherent in the initiation, progress and completion of clinical trials, including the Phase 2 POC trial of ALTO-101, and other important factors, any of which could cause Alto’s actual results to differ from those contained in the forward-looking statements, which are described in greater detail in the section titled “Risk Factors” in each of Alto’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and Alto’s Quarterly Report on Form 10-Q for the fiscal quarter ended June 30, 2025 filed with the Securities and Exchange Commission (“SEC”) as well as in other filings Alto may make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Alto expressly disclaims any obligation to update any forward-looking statements contained herein, whether because of any new information, future events, changed circumstances or otherwise, except as required by law.

Availability of Information on Alto’s Website

Alto routinely uses its investor relations website to post presentations to investors and other important information, including information that may be material. Accordingly, Alto encourages investors and others interested in Alto to review the information it makes public on its investor relations website.

View source version on businesswire.com: https://www.businesswire.com/news/home/20251003096029/en/

Investor and Media Contact:
Nick Smith
investors@altoneuroscience.com
media@altoneuroscience.com

Source: Alto Neuroscience, Inc.