Definium Therapeutics Inc (DFTX) investor relations material

Key efficacy and safety findings

• Three phase III studies (VOYAGE, PANORAMA in GAD; EMERGE in MDD) showed significant efficacy with single DT120 treatment, achieving 5–8 point improvements on primary endpoints versus placebo at 6–12 weeks post-treatment.

• Response and remission rates at week 12 were 43%/14% (VOYAGE) and 32%/15% (PANORAMA), with odds ratios up to 4x over placebo, indicating strong clinical differentiation from standard care.

• Safety profile was benign, and functional unblinding was addressed through study design, including a 50 mcg control arm in PANORAMA to confound treatment perception.

• Durability data from Part B show that with open-label retreatment, 60–70% of patients achieve mild or remission status after one to three treatments over six months.

• No a priori predictors of response have been identified, but ongoing research aims to clarify responder profiles.

Study design and regulatory strategy

• Studies included a 12-week double-blind period followed by a 40-week open-label Part B, totaling one year of observation.

• Retreatment in Part B is protocol-driven, triggered by moderate symptom thresholds (MADRS ≥20 or HAM-A ≥16), with up to five treatments per year and at least one month between doses.

• Real-world retreatment cadence is expected to mirror study conditions, with most patients needing one to three treatments in six months.

• NDA filing strategy for GAD and MDD may be sequential or combined, pending FDA discussions; a second MDD study is ongoing.

• Regulatory engagement with FDA has been collaborative, focusing on safety, access, and clear labeling for real-world use.

Operational and commercial considerations

• Median clearance time post-treatment is six hours, with 97% of patients cleared by eight hours, supporting outpatient administration.

• Discharge-readiness is assessed via a checklist developed with FDA input, ensuring patients return to baseline function before leaving supervision.

• Commercial distribution is expected to start with more white bagging, shifting to buy and bill as clinics gain experience; flexibility in distribution models will be prioritized.

• Practice standards and adjunctive therapy research are anticipated to evolve post-approval as clinicians gain experience.