COMPASS Pathways (CMPS) 7th Annual Evercore ISI HealthCONx Healthcare Conference summary

Clinical development progress

• Phase III program for treatment-resistant depression is underway, with robust phase IIb results previously published showing significant efficacy and balanced safety.

• The first phase III study (005) will report six-week primary endpoint data in Q2 next year; the second study (006) will report 26-week data in the second half of 2026.

• Recruitment challenges included stringent documentation requirements, DEA licensing delays, and logistical issues with therapist training and site adaptation, but these have been addressed and recruitment is accelerating.

• Discontinuation rates are below expectations, data quality is high, and site performance is closely monitored with nonproductive sites being culled.

• No single site will contribute more than 10% of the total patient population, ensuring data diversity.

Data disclosure and trial integrity

• Only the primary endpoint (MADRS score difference, p-value, and confidence interval) and a DSMB safety statement will be disclosed at six weeks for 005 to maintain trial blinding.

• Both phase III trials remain blinded through 26 weeks, with 006 data to be disclosed only after full blinding to avoid unblinding complexities.

• DSMB reviews suicidality data qualitatively, with no preset quantitative thresholds; no safety signals have been identified to date.

Commercial and regulatory outlook

• Commercial rollout is expected to be less complex than trials due to less stringent documentation and broader eligibility for support staff.

• Real-world administration will allow a wider pool of licensed professionals, reducing bottlenecks seen in trials.

• A clinically meaningful MADRS effect size is considered to be above two points; studies are powered for 3.5–4 points.

• Durability of effect may allow for two to four administrations per year, with intermittent dosing and high adherence expected to reduce patient burden.

• Regulatory learnings from the Lykos AdCom have led to more conservative data disclosure and a comprehensive clinical pharmacology package.

• Ongoing positive interactions with the FDA, with breakthrough designation and clear guidance that both phase III studies are needed for filing.