Cybin (HELP) TD Cowen 4th Annual Novel Mechanisms in Neuropsychiatry Summit 2024 summary

Regulatory landscape and agency guidance

• FDA applies the same safety and efficacy standards to psychedelics as other therapies, with specific guidance evolving in response to recent cases like Lykos and Spravato.

• Issues such as functional unblinding and expectation bias are now more clearly addressed, with new requirements for measuring and managing these factors in trials.

• Exclusion of patients with prior psychedelic experience is now an FDA requirement to reduce bias in clinical trials.

• The agency maintains open communication and supports developers through regulatory pathways.

• Abuse potential and drug liking are closely monitored, with comprehensive data collection and standard FDA methodologies applied.

Clinical trial design and methodologies

• Evidence-based psychotherapeutic support is emphasized, with most companies focusing on pre- and post-dose support rather than therapy during dosing.

• Expectation bias is measured using patient questionnaires, and large effect sizes are expected to mitigate its impact on efficacy and safety.

• Trials are designed to be reflective of the general population, with strict criteria for prior psychedelic use and robust sample sizes to enable meaningful safety and efficacy analyses.

• Durability of effect and retreatment paradigms are key endpoints, with some trials allowing as-needed retreatment based on symptom recurrence.

Company program updates and timelines

• Atai's VLS-01 is entering phase II with an oral transmucosal film formulation, targeting robust, repeatable exposure and a patient-friendly protocol; phase II will assess durability over twelve weeks.

• GH Research expects to complete phase IIb enrollment by end of Q3, with top-line data anticipated by year-end or Q1 next year; primary endpoint is MADRS at day seven, with a six-month open-label extension and as-needed retreatment.

• Cybin's CYB003, a deuterated psilocin oral capsule, is moving into phase III with two doses three weeks apart; first phase III data expected in 2026, with NDA submission targeted for early to mid-2027.