Alto Neuroscience Initiates Phase 2 Study of ALTO-101, a Novel PDE4 Inhibitor, in Cognitive Impairment Associated with Schizophrenia
Alto Neuroscience announced the initiation of a Phase 2 trial for ALTO-101, a PDE4 inhibitor aimed at treating cognitive impairment in schizophrenia. The study, expected to conclude by the second half of 2025, will involve 70 adults and compare a transdermal formulation of ALTO-101 against a placebo. Previous Phase 1 trials showed positive cognitive effects and fewer adverse events compared to oral administration. CEO Amit Etkin highlighted the potential of ALTO-101 to improve cognitive function across various psychiatric disorders.
The primary outcome will measure the effect on theta band activity using EEG. This follows promising data presented at the SOBP Annual Meeting, affirming the relevance of EEG measures in assessing cognitive function in schizophrenia.
- Initiation of Phase 2 study for ALTO-101.
- Favorable results in Phase 1 study showing positive cognitive effects.
- Reduction in typical adverse events with transdermal formulation.
- Exploration of cognitive performance and EEG markers in new study.
- Potential to benefit patients with CIAS and other psychiatric disorders.
- Top-line data from the Phase 2 study will not be available until the second half of 2025.
The initiation of a Phase 2 study for ALTO-101 is a noteworthy milestone in the field of psychiatric treatment, particularly for Cognitive Impairment Associated with Schizophrenia (CIAS). The transdermal formulation of ALTO-101 could be groundbreaking, as it potentially offers greater systemic drug exposure and fewer adverse events compared to its oral counterpart. Transdermal delivery can improve patient compliance, reduce gastrointestinal side effects and provide more stable drug levels in the bloodstream.
In terms of cognitive improvement, the focus on theta band activity in EEG measurements is critical. Theta rhythms have been closely linked with cognitive processes, particularly in the domains of memory and attention. It's promising that ALTO-101 has shown positive effects on these EEG markers in initial trials.
Given the complexity of treating CIAS, this study could pave the way for more personalized mental health treatments, aligning with Alto Neuroscience's broader goals of precision psychiatry. However, the results will not be available until the second half of 2025, so investors need to keep in mind the extended timeline and inherent risks of clinical trials.
From a market perspective, the initiation of this Phase 2 study positions Alto Neuroscience as a key player in the niche but critical area of cognitive impairment in psychiatric disorders. The potential market for effective CIAS treatments is substantial, given the high unmet needs in conditions like schizophrenia, depression and neurodegenerative diseases.
The reduced side effects and increased drug exposure reported for the transdermal formulation of ALTO-101 are significant selling points. If successful, this could differentiate ALTO-101 from other PDE4 inhibitors, which often struggle with tolerability issues. However, the market will closely watch the outcome data due in 2025 to gauge the true value and potential market penetration of this treatment.
Investors should be prepared for a long-term investment horizon, as the biotech sector often involves high volatility tied to clinical trial results and regulatory approvals. Nevertheless, the initial data and strategic focus on precision psychiatry provide a solid foundation for optimism.
Alto Neuroscience's initiation of this Phase 2 study for ALTO-101 is financially significant as it marks continued progress in their clinical pipeline. The company's ability to advance multiple Phase 2 studies simultaneously demonstrates strong financial health and effective resource allocation. The announcement also adds credibility, potentially enhancing investor confidence and attracting further funding opportunities.
It is important to consider the burn rate associated with clinical trials and the timeline for potential revenue generation. The data release scheduled for the second half of 2025 suggests that any revenue generation from ALTO-101 is still a few years away. Hence, short-term financial benefits are limited, but the long-term potential could be substantial if the drug successfully passes through the clinical trials and gains regulatory approval.
Given the risks inherent in clinical trials, particularly in the psychiatry space, investors should diversify their portfolios accordingly and stay updated on interim results and milestones.
– ALTO-101 has been shown to positively impact cognition and key cognition-related EEG markers in humans when delivered orally –
– Novel transdermal formulation of ALTO-101 exhibited favorable pharmacokinetics and tolerability, greater drug exposure, and fewer PDE4 associated adverse events compared to ALTO-101 administered orally –
“We are excited to initiate our sixth Phase 2 study as a company, across our pipeline programs, as we continue to redefine mental healthcare with Alto’s Precision Psychiatry Platform,” said Amit Etkin, M.D., Ph.D., founder and CEO of Alto Neuroscience. “Cognitive impairment is disabling in many psychiatric disorders including schizophrenia, depression, and neurodegenerative diseases. ALTO-101 has the potential to address the cognitive aspects of these diseases with a known pro-cognitive mechanism, while also overcoming the side effects typically associated with PDE4 inhibitors. We look forward to completing this proof-of-concept study, as we believe ALTO-101 may benefit patients with CIAS and potentially become an important therapeutic option for a broad range of indications.”
Alto recently completed a Phase 1 study of ALTO-101 in healthy adults, demonstrating positive effects on cognition and electroencephalography (EEG) measures relevant to cognitive function. Alto’s novel transdermal formulation of ALTO-101 exhibited greater systemic drug exposure than orally administered ALTO-101 while also demonstrating a reduction in typical class-related adverse events.
The Phase 2 study announced today consists of a cross-over double-blind, placebo-controlled, dose-escalating treatment with ALTO-101 and placebo in patients with CIAS. Alto expects to enroll approximately 70 adult participants between 21-55 years old with schizophrenia and a demonstrable level of cognitive impairment. Alto will evaluate the effects of ALTO-101 on EEG markers relevant to cognitive function and will also explore its effects on cognitive performance. The primary outcome in the study is the effect of ALTO-101 on theta band activity as measured using EEG at the end of each dose period.
Alto recently presented data at the Society of Biological Psychiatry (SOBP) Annual Meeting demonstrating that theta band activity has both the greatest correlation with cognitive function in patients with schizophrenia among a comprehensive set of EEG measures as well as greatest case-control sensitivity. The findings on these measures, which were prospectively replicated, support the suitability of the primary outcome measure being evaluated in the present study to demonstrate robust proof-of-concept of ALTO-101 as a potential treatment for CIAS.
About ALTO-101
ALTO-101 is a novel small molecule PDE4 inhibitor being developed for cognitive impairment associated with schizophrenia, a disease state defined by negative and cognitive symptoms with no currently available targeted treatments. Through a proprietary transdermal delivery system, ALTO-101 is designed to provide steady state concentrations to improve drug safety, tolerability, and pharmacokinetics. The proprietary transdermal delivery system for ALTO-101 has been developed in partnership with MEDRx. In Phase 1 clinical trials, ALTO-101 demonstrated human brain penetration, robust CNS-relevant pharmacodynamic effects, and was well tolerated across therapeutically relevant dose ranges.
About Cognitive Impairment Associated with Schizophrenia
Schizophrenia is a serious, persistent, and often disabling mental illness impacting how a person thinks, feels, and behaves. The illness affects nearly 24 million people worldwide, including 2.8 million people in
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 depression, PTSD, 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 “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “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 for the timing, design, and results of the ALTO-101 study, and the number of subjects to be enrolled in the study. 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 design, initiation, progress and completion of the ALTO-101 study and clinical development of ALTO-101; the risk that Alto may not achieve the targeted enrollment in the ALTO-101 study or that enrollment may take longer than expected; the availability and timing of results from the ALTO-101 study; 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 Alto’s Quarterly Report on Form 10-Q for the fiscal quarter ended March 31, 2024 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.
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Source: Alto Neuroscience, Inc.
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