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Definium Therapeutics (NASDAQ:DFTX) outlined its clinical pipeline and upcoming data catalysts at the Leerink Partners Global Healthcare Conference, with CEO Robert Barrow and CFO Brandi Roberts detailing progress in the company’s late-stage program for anxiety and depression and an earlier-stage effort in autism spectrum disorder.
Rebrand from MindMed to Definium
Barrow said the company changed its name from MindMed largely for practical and positioning reasons as it moves closer to potential commercialization. He noted that “mind” and “medicine” are common terms used broadly across wellness and therapy groups, complicating brand differentiation and even basic digital presence. As an example, he said the company could not obtain the mindmed.com domain.
DT120: Phase III program in GAD and MDD
Barrow said Definium’s lead asset is DT120, an orally disintegrating tablet (ODT) formulation of LSD being developed for generalized anxiety disorder (GAD) and major depressive disorder (MDD). He described three ongoing Phase III studies—two in GAD and one in MDD—with a second MDD study expected to start “very soon.” He added the company received Breakthrough Therapy Designation for the program in 2024.
He characterized the GAD and MDD pivotal programs as “mirror images” built off a prior five-arm Phase II dose-response study. In the Phase III program, the first study in each indication is a two-arm, head-to-head design (active dose versus placebo). He said the MDD study (149 patients) is fully enrolled and expected to read out in late Q2, while the first GAD pivotal study is expected to read out in early Q3.
Barrow also described the design of a second pivotal study that includes an additional control arm using a 50 microgram “intermediate dose” intended as a functional methodological control to reduce the ability of patients to infer treatment assignment based on subjective effects. He said the intermediate dose control does not affect the statistical analysis and is not intended to be a key comparator.
Phase II results: dose response, durability, and remission
In discussing the Phase II trial, Barrow said it was a five-arm study with placebo and 25, 50, 100, and 200 microgram doses. The goal, he said, was to establish a dose response and select a Phase III dose using an MCP-Mod statistical approach. Definium concluded there was a dose response and selected 100 micrograms as the recommended dose, which Barrow said balanced maximal efficacy with a more favorable adverse event burden and a shorter treatment-session duration than higher doses.
Barrow emphasized that functional unblinding—patients recognizing they received an active psychedelic—could not explain the clinical response. He said about 90% of patients at 25 micrograms could correctly guess they were on drug, and “virtually everyone” knew they were on drug across doses, yet the study still showed a clinical dose response.
On efficacy, Barrow reported that patients entered the GAD study with a mean Hamilton Anxiety Rating Scale (HAM-A) score of around 30, which he described as severe. After a single dose and 12 weeks of follow-up, he said patients receiving 100 micrograms had an average 21.9-point reduction from baseline, which he called the largest anxiety response he has seen, and a placebo-adjusted improvement of 7.7 points. He also said that half of patients who received 100 micrograms were in remission at 12 weeks, describing the outcome as “no detectable anxiety” three months after treatment.
He added that symptom improvement appeared early and remained stable through the 12-week observation window. Because HAM-A is a one-week recall instrument, the earliest HAM-A timepoint was one week post-dose, at which point Barrow said the effect size was similar to later timepoints. He also said the Clinical Global Impressions-Severity (CGI-S) measure showed improvement within 24 hours of treatment.
For depression-related measures observed within the same Phase II study population, Barrow said baseline MADRS scores were in the mid-20s and the average change was an 18.5-point drop, about 6.5 points better than placebo. He noted that many patients approached remission levels on the scale, which reduced “discriminatory power,” and suggested a dedicated MDD population with higher baseline severity could potentially provide more room to separate from placebo.
Treatment-day logistics and commercialization considerations
Barrow described the treatment visit as a full-day clinic session. Patients are dosed in the morning and kept under observation for eight hours, regardless of whether they receive placebo or active drug. He said a monitor is present for patient comfort and safety, though intervention is “very, very rare.” Definium’s trials do not include concurrent psychotherapy, which Barrow said is impractical at higher doses because the experience tends to be internal.
Definium begins assessing readiness for discharge at hour five and checks hourly thereafter, with Barrow saying most patients are able to leave by eight hours. Roberts added that the company is planning for a potential REMS program and is considering how to support sites with REMS requirements, administration workflow, and payer processes if DT120 is approved.
On market opportunity, Roberts said there are “50 million US adults” with GAD and MDD and cited what she described as a significant unmet need, including that no drug has been approved in GAD since 2007 and that GAD incidence has increased from 3.5% to over 10% over the last 10–15 years. She also said Definium estimates roughly 7,000 providers currently support patients with GAD and that many are familiar with and certified to administer Spravato, which she suggested could provide a starting point for adoption.
Roberts said pricing is premature but compared potential DT120 economics to Spravato’s annual pricing, which she characterized as roughly $28,000 per year at low dose/low frequency up to about $70,000 per year at high dose/high frequency. She argued that if DT120 requires fewer administrations—citing Phase II durability out to 12 weeks and ongoing Phase III follow-up out to one year—payers may view pricing in that general range as acceptable, especially if efficacy is strong.
DT402: R-MDMA program in autism spectrum disorder
Definium also discussed DT402, the R-enantiomer of MDMA, which Barrow said has completed a Phase I single ascending dose study and is now in a 20-patient open-label proof-of-concept study targeting core symptoms of autism spectrum disorder—particularly social communication. He contrasted Definium’s approach with “session-based” models and said the goal is not necessarily disease modification, but rather functional improvement while the drug is on board.
Barrow said the company selected the R-enantiomer because it has more serotonergic activity and, in preclinical work, appeared more associated with MDMA’s prosocial effects. He contrasted it with the S-enantiomer, which he said is associated with more dopamine signaling and more stimulant-like properties. He said early clinical pharmacology observations aligned with this rationale and supported moving into an early efficacy study.
Upcoming catalysts
Management highlighted three expected clinical readouts in 2026:
- Emerge (MDD): data expected in late Q2
- Voyage (first GAD pivotal study): data expected in early Q3
- Panorama (second GAD study): data expected in the second half of the year
About Definium Therapeutics (NASDAQ:DFTX)
Definium Therapeutics, Inc, a clinical stage biopharmaceutical company, develops novel products to treat brain health disorders. The company’s lead product candidates include MM120, which is in phase 3 for the treatment of generalized anxiety disorder and attention deficit hyperactivity disorder; and DT402, a R-enantiomer of 3,4-methylenedioxymethamphetamine, which is in phase 2a clinical trials for the treatment of core symptoms of autism spectrum disorder. The company was formerly known as Mind Medicine (MindMed) Inc and changed its name to Definium Therapeutics, Inc in January 2026.
