Alto Neuroscience (ANRO) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Conference overview

• The event focused on advances in neuroscience, particularly the application of precision medicine to neuropsychiatric drug development.

• The presenting company discussed its approach to identifying the right patients for specific drugs using biological and cognitive markers.

• Five Phase 2 trials are ongoing, targeting prevalent disorders such as depression, schizophrenia, bipolar disorder, and PTSD.

• The company has dosed over 800 patients and is leveraging scalable, web-based and EEG tools for patient stratification.

• Top-line data for the lead candidate, ALTO-100, is expected in October, with additional readouts for other candidates in the first half of next year.

Precision medicine and trial design

• The precision approach aims to match drugs to patients based on biological markers, moving away from trial-and-error methods.

• ALTO-100 targets patients with impaired neuroplasticity, identified through memory and cognitive testing.

• The Phase 2b trial uses an enrichment strategy, focusing on patients with poor memory, and follows FDA enrichment guidelines.

• The primary endpoint is the MADRS score in the poor memory group, with a step-down test for monotherapy analysis.

• Rigorous blinding and external raters are used to minimize placebo effects and ensure data quality.

Clinical and commercial implications

• Data show that patients with poor cognition respond substantially better to ALTO-100, with effect sizes roughly double those seen in all-comer studies.

• The web-based cognitive test is easy to administer, reliable, and can be used by patients at home, supporting future commercial scalability.

• The approach allows for clear patient stratification, which could transform clinical practice and improve outcomes.

• The same precision strategy is being applied to other pipeline drugs and disorders, including bipolar depression and ALTO-300, which uses an EEG biomarker.

• The company operates its own trials, achieving higher data quality and capital efficiency compared to industry averages.