Definium Therapeutics Inc (DFTX) investor relations material

Clinical program and pipeline updates

• Lead program DT120, a proprietary LSD formulation, targets GAD and MDD, with three pivotal Phase 3 studies (Voyage, Panorama, Emerge, Ascend) reading out in 2026, building on strong Phase 2 data published in 2024.

• Phase 2 data showed rapid, durable effects: a Cohen's d of 0.81, 21.9-point improvement on the Hamilton Anxiety Rating Scale, and a 48% remission rate at 12 weeks after a single dose.

• Dose-response studies identified 100 micrograms as optimal, with no added efficacy at 200 micrograms but increased adverse events.

• Phase 3 studies began in late 2024/early 2025, with adaptive designs, open-label extensions, and centralized, blinded raters to optimize real-world relevance and mitigate bias.

• DT402 (R-MDMA) is in Phase 2a for autism spectrum disorder, with first patient dosed in late 2025 and initial data expected in 2026.

Efficacy and safety insights

• DT120 demonstrated rapid onset, durable effects, and statistically significant improvements in anxiety and depression, with effects double or better than current standards.

• Clinical outcomes showed significant separation from placebo, with no evidence that functional unblinding explained results.

• Adverse events were mostly mild to moderate, limited to dosing day, and resolved quickly; no drug-related serious adverse events or suicidality signals observed.

• Breakthrough Therapy designation was granted, and regulatory alignment with FDA has enabled a rapid development timeline, aiming for NDA submission within five years of IND opening.

• Intellectual property strategy includes issued patents for DT120 ODT covering formulation, manufacturing, and treatment methods.

Market opportunity and adoption

• GAD and MDD affect 50 million people in the U.S., with 27 million currently receiving medication; current treatments are often ineffective and poorly tolerated.

• Commercial strategy targets rapid adoption, leveraging 7,000 interventional psychiatry clinics and high enthusiasm among healthcare providers and payors.

• Surveys show 70% of healthcare professionals intend to prescribe DT120 for GAD if approved.

• Even treating 100,000 patients would represent a multi-billion dollar opportunity, with annual revenue potential per 100,000 patients ranging from $2.8B to $7.0B depending on pricing.

• Existing healthcare infrastructure and reimbursement pathways are expected to support scalable delivery and access.