Definium Therapeutics Inc (DFTX) Canaccord Genuity 44th Annual Growth Conference & Private Company Showcase 2024 summary

Clinical development updates

• Advancing MM120 (LSD) for generalized anxiety disorder (GAD) and major depressive disorder (MDD), with phase III trials launching in late 2024 for GAD and early 2025 for MDD.

• MM120 demonstrated multi-month durable effects after a single dose in phase II, with about 50% remission at 12 weeks and some patients showing benefits for years.

• MM402 (R-MDMA) is in phase I for autism spectrum disorder, aiming for early phase II efficacy studies.

• Three phase III catalysts are expected across 2026, with ongoing regulatory and clinical milestones anticipated in coming quarters.

• Trial design includes up to four open-label doses of MM120 in both GAD and MDD studies, with primary endpoints at 12 weeks.

Regulatory and trial design considerations

• MM120 program avoids psychotherapeutic interventions, focusing on drug-only benefit to mitigate complexities seen in other programs.

• Addressed functional unblinding in trial design, using independent raters and third-party diagnosis verification to ensure robust data.

• Phase III studies are powered to detect a 0.5 effect size, with interim sample size re-estimation to maintain 90% power.

• Monitoring intervals for dosing sessions are being reduced based on data, with patient safety and burden in mind.

Market opportunity and commercial strategy

• MM120 aims to address both GAD and MDD, which have high comorbidity and overlapping symptoms, offering a broad treatment label.

• Durability of effect is expected to support an episodic treatment paradigm, with potential for long intervals between doses.

• Pricing strategy considers annual treatment costs, benchmarking against products like Spravato ($25,000–$62,000/year), and aims for attractive economics if few doses are needed annually.

• Commercial challenges include evolving infrastructure, need for more treatment centers, and overcoming the historical perception of LSD.

• Strong provider and patient enthusiasm, with expectations for a paradigm shift in psychiatric care if clinical data are confirmed.