Definium Therapeutics has drawn up plans to submit a New Drug Application (NDA) to US regulators for its psychedelic medicine, DT120, following a third successful Phase III readout.
The biopharma company is teeing up regulatory submissions following the positive results of the Phase III open-label Panorama study (NCT06809595), in which it pitted a 50µg and 100µg dose of its lysergic acid diethylamide (LSD)-based medicine DT120 – otherwise known as lysergide – against placebo over a 12-week period in patients with generalised anxiety disorder (GAD). This was then followed by a 40-week extension phase, where patients had the option to receive further open-label treatment.
During the study, the highest dose of DT120 triggered a significant, 5.1-point placebo-adjusted improvement from baseline in anxiety scores at week 12, meeting the trial’s primary endpoint. This effect was also rapid and sustained, with changes in scores seen as early as day two and maintained during the entire first portion of the trial.
According to Leerink analysts, this result clears the 5-point bar set out by investors for the confirmatory study. Investors were also happy with the data, with the company’s stock rising 3.5% on 14 September, from a $38.89 close on 11 September to a $40.25 close on 14 September. Definium Therapeutics is listed on the Nasdaq exchange with a market cap of $5.41bn.
Patients also tolerated DT120 well, with the majority of treatment-related adverse events (TRAEs) being mild-to-moderate in severity and occurring on the day of dosing. Researchers observed no new safety signals and discontinuation rates remained consistent between the treatment and placebo arms.
DT120 scores a hattrick
The Panorama results closely follow the positive outcomes of the Emerge (NCT06941844) and Voyage (NCT06741228) studies, which were looking at the drug’s potential in major depressive disorder (MDD) and GAD, respectively. In Voyage, DT120 triggered a 5.4-point improvement in anxiety scores.
“With Emerge, Voyage, and now Panorama all positive, we view DT120 as clinically de-risked for GAD and mostly de-risked for MDD,” said Leerink analysts, adding that the conversation will now shift towards the logistics around bringing a psychedelic drug to the commercial market.
Meanwhile, Jefferies analysts believe that DT120’s 5.1-point anxiety score reduction constitutes its “best-in-class” potential in GAD, leading them to “conservatively assume” $1.5bn-plus peak sales per indication. DT120 is currently in development for MDD, GAD and post-traumatic stress disorder (PTSD).
With positive results in hand, Definium plans to file an application for DT120 with the US Food and Drug Administration (FDA) for GAD in the first half of 2027, following a pre-NDA meeting with the agency taking place in the final quarter of 2026.
Access remains key talking point following FDA hearing
As psychedelics edge closer to potential approvals, the FDA held a hearing to seek public input on how this new drug class should be safely and effectively rolled out, monitored and studied outside of the clinical trial setting.
During the session, attendees placed a sharp focus on access, as some questioned how these drugs will be administered in the real world. While several biotechs looking to commercialise psychedelic therapies, such as Definium and Compass Pathways, have opted to explore the supervised treatment session approach to ascertain the benefit of the drug alone, some experts are advocating for a psychotherapy-based approach, where the medicine is given alongside therapy to improve outcomes.
With the supervised treatment option picking up steam, however, experts also raise questions as to whether there is suitable infrastructure and know-how to administer these drugs on a broad scale.
