Alto Neuroscience (ANRO) Jefferies Global Healthcare Conference 2026 summary

Strategic approach and pipeline overview

• Focuses on precision psychiatry using biomarkers and machine learning to match patients to mechanisms, addressing heterogeneity in psychiatric disorders.

• Pipeline includes ALTO-207 for treatment-resistant depression (TRD), ALTO-300 for adjunctive depression, and ALTO-100 for bipolar depression and cognitive impairment in schizophrenia.

• Multiple assets leverage novel mechanisms, with upcoming phase II-B and phase III milestones over the next 6-12 months.

ALTO-207: Differentiation, study design, and regulatory strategy

• ALTO-207 is a fixed-dose combination of pramipexole and ondansetron, targeting TRD with a unique efficacy and tolerability profile.

• Demonstrates higher efficacy (Cohen's d up to 0.9) than standard antidepressants, with fewer side effects compared to antipsychotics and esketamine.

• Phase II-B trial (178 patients) is powered for an effect size of 0.45, with phase III planned to start in early 2027, aiming for regulatory alignment and NDA submission.

• Strategies to reduce dropout rates include ondansetron co-administration, modified release, and titration schedules.

• Rigorous patient selection and compliance monitoring are used to mitigate professional patient risk and placebo response.

ALTO-300: Adjunctive depression program and trial adjustments

• ALTO-300 uses an EEG biomarker for patient selection, aiming to improve consistency and efficacy in adjunctive depression treatment.

• Phase II-B readout delayed to first half 2027 due to enhanced patient quality filters, improving recruitment accuracy.

• Interim analysis led to modest upsizing of the trial, with effect size boundaries set for futility and early success.

• Safety strategy focuses on 25 mg dosing to avoid liver enzyme elevations, supported by meta-analyses showing no efficacy loss at lower dose.

• Patent protection relies on method of treatment using the EEG biomarker, with granted IP supporting exclusivity.