Definium Therapeutics’ LSD formula has gone two for two in its phase 3 program, notching a win in generalized anxiety disorder mere months after knocking a depression study out of the park.
The 214-patient Voyage trial pitted a psychoactive session with Definium’s DT120 ODT against placebo, with the psychedelic coming out ahead by reducing anxiety symptoms at week 12 by 11.6 points compared to a 6.2-point reduction for placebo. Symptom severity was assessed using the Hamilton Anxiety Rating Scale (HAM-A).
“We have never seen a pivotal study deliver greater than five-point placebo-adjusted change on the HAM-A, and we did this 12 weeks after a single dose of drug,” Definium CEO Robert Barrow told Fierce.
For patients with anxiety, DT120’s 5.4-point improvement over placebo “is a sea change” in their treatment prospects, Barrow claimed. “This opens the door to an entirely new expectation.”
Barrow was similarly effusive in June when discussing DT120’s phase 3 results in major depression, where the CEO argued that the candidate had demonstrated unprecedented levels of efficacy.
“If ‘pretty good’ encompasses the best data ever seen in a pivotal study of depression, I think we would say pretty good,” the CEO said at the time.
Anxiety has not seen significant medical advancement in close to 20 years, since the approval of Eli Lilly’s Cymbalta back in 2007. The historical standard for treating anxiety leaves much to be desired, Barrow told Fierce, with benzodiazepines bringing risks of addiction when taken chronically and serotonin reuptake inhibitors better suited for depression.
“Many of the patients I see have not found relief despite trying multiple therapies,” Brian Barnett, M.D., vice chair of psychiatry at Cleveland Clinic and principal investigator of the Voyage trial, said in Definium's release.
“A single-dose treatment option delivering meaningful benefits for 12 weeks is a promising step forward not found in today’s therapies,” Barnett added. “If approved, it could give patients and their physicians a treatment option that works through an entirely different mechanism than anything currently used in clinical practice.”
With two phase 3 trials down, DT120’s next test is another anxiety study that is set to read out next month. That trial, called Panorama, includes a second control arm of a lower dose of the LSD-based drug.
“It's just there to basically try to confuse people so that if they feel the effects of the drug, they can't be sure it's the real dose of drug,” Barrow explained.
While Barrow thinks the Panorama readout will further bolster DT120’s chances with the FDA, he argued that Definium has enough definitive proof that the psychedelic works already.
“We now have two very compelling studies across anxiety and depression,” the CEO said. “These are high need, high priority indications.”
“We feel very, very good about the constructive dialog we've had with FDA and where that leads us going forward,” he added.
Definium's readouts come at a time of renewed enthusiasm for psychedelic medicines, including support from the Trump administration and Lilly's $2.8 billion acquisition of AtaiBeckley last month.