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

Regulatory landscape and agency expectations

• 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, emphasizing evidence-based psychotherapeutic support and addressing functional unblinding and expectation bias.

• Recent regulatory feedback has led to stricter exclusion of patients with prior psychedelic experience and the use of expectation bias questionnaires in trials.

• Safety oversight, abuse potential, and drug liking are closely monitored, with comprehensive data collection and reporting required, and methodological challenges acknowledged for human abuse potential studies.

• The FDA's approach is seen as consistent regardless of political changes, with bipartisan support for advancing psychedelic therapies.

Clinical development strategies and trial design

• Companies are refining trial designs to address regulatory concerns, including larger sample sizes, stringent criteria for prior psychedelic use, and robust post-dose psychological support.

• Functional unblinding and expectation bias are managed through patient questionnaires and careful informed consent, with subset analyses planned to assess their impact.

• Trials increasingly focus on durability of effect, with endpoints at both early (day 7) and longer-term (up to 12 weeks or 6 months) timeframes, and retreatment paradigms as needed.

• Different compounds and formulations (short-acting vs. long-acting, oral vs. IV) are being developed to offer diverse treatment options and address patient and logistical needs.

Company-specific program updates

• Atai's VLS-01 is entering phase II with an oral transmucosal film formulation, targeting robust, repeatable exposure and rapid return to baseline, with stringent criteria for prior psychedelic use.

• GH Research expects to complete phase IIb enrollment by end of Q3, with top-line data anticipated by year-end or Q1 next year, using a rapid-acting antidepressant paradigm and allowing as-needed retreatment.

• Cybin's CYB003, a deuterated psilocin capsule, is moving into phase III with two doses three weeks apart, aiming for reduced variability and data expected in 2026, with a staggered second study and NDA submission targeted for early to mid-2027.