Definium Therapeutics Inc (DFTX) investor relations material

Key clinical program updates

• Pivotal data readouts for the lead program DT120 are expected in late Q2 for MDD and early Q3 for GAD, with a third phase III readout later in the year.

• The EMERGE study for MDD is a randomized, double-blind, placebo-controlled trial using MADRS as the primary endpoint, powered to detect a 5-point difference.

• GAD phase III studies mirror the MDD design, with a single dose, 12-week follow-up, and a 9-month extension; sample size re-estimation for VOYAGE showed no need for increase due to lower dropout rates.

• VOYAGE is now powered to detect just over a 2-point difference on the Hamilton Anxiety Scale, with over 99% power for a 5-point difference if interim parameters hold.

• Durability of effect is being characterized, with phase II showing at least 12 weeks of benefit from a single dose; more data will be shared with top-line phase III results.

Study design rationale and interpretation

• Significant overlap exists between GAD and MDD diagnoses and measurement scales, supporting confidence in translating phase II GAD results to MDD.

• In phase II GAD, 60% of patients also had MDD, reinforcing the relevance of efficacy data across indications.

• Higher baseline severity in MDD phase III is expected to allow greater room for improvement compared to phase II.

• A 4-point or greater delta on anxiety scales would position the drug as best-in-class for GAD; exceeding 3.5–4 points in MDD would be highly competitive.

• Prior psychedelic use is tracked but not used for randomization balancing, as its impact is considered minimal.

Commercial and market insights

• GAD diagnoses and treatments are growing rapidly, now estimated at over 10% prevalence in U.S. adults, up from 3% two decades ago.

• Existing treatments for GAD, mainly SRIs and benzodiazepines, are often inadequate, highlighting unmet need.

• Having both GAD and MDD labels would allow broader market access and address overlapping patient populations.

• The commercial model for clinics includes E&M billing, session-based codes, and a buy-and-bill option, with consolidated treatment days offering efficiency for both patients and providers.

• A high-touch support model for clinics and patients is planned, leveraging operational expertise from clinical trials to facilitate adoption and access.