A psychiatric drug unlike anything currently approved in the United States just cleared a major hurdle.

Definium Therapeutics said Wednesday that a single dose of its LSD-based tablet significantly reduced symptoms of generalized anxiety disorder within days, delivering a major Phase 3 victory that could put the company on a path toward the first FDA-approved LSD-based treatment for anxiety.

The drug, DT120, is a pharmaceutical-grade form of lysergide — better known as LSD — delivered as a tablet that dissolves in the mouth. Unlike conventional anxiety medicines that patients may take every day for months or years, participants in Definium’s trial received one 100-microgram dose under medical supervision.

The effect appeared rapidly.

Patients receiving DT120 showed statistically significant improvement shortly after treatment, with the benefit becoming evident within days following the single administration. The study continued tracking patients for 12 weeks and found that the treatment advantage remained at the study’s final measurement.

That distinction matters: patients did not take the pill for 12 weeks. They took it once.

One Dose Produces Major Phase 3 Result

The Voyage trial enrolled 214 adults ages 18 to 74 with generalized anxiety disorder at roughly 35 U.S. clinical sites.

Patients began the study with moderate-to-severe anxiety and were randomly assigned to receive either DT120 or placebo.

At the study’s primary endpoint, patients receiving DT120 experienced an average 11.6-point reduction on the Hamilton Anxiety Rating Scale, compared with a 6.2-point reduction for placebo.

That produced a 5.4-point advantage over placebo, comfortably clearing the trial’s statistical threshold and giving Definium the positive Phase 3 result investors and regulators were waiting for.

The individual patient results were equally striking.

About 43% of patients receiving DT120 cut their anxiety symptoms by at least half, compared with 16% in the placebo group.

And 14% of patients receiving DT120 reached remission, versus 4% receiving placebo.

Definium said the drug was generally well tolerated and the trial met its primary endpoint and all key secondary efficacy endpoints.

A Different Way to Treat Anxiety

What makes DT120 potentially groundbreaking is not simply that LSD reduced anxiety.

It is the possibility that a chronic psychiatric condition normally managed with daily medication could instead be treated with a single supervised dose producing rapid and lasting improvement.

Many conventional antidepressant and anti-anxiety medicines must be taken every day and can take weeks before patients know whether they are working. Patients may cycle through several medications before finding one that helps.

Definium is proposing a fundamentally different model.

The patient comes to a qualified medical facility, receives a single tablet, remains under supervision while the psychedelic effects wear off and then goes home. No daily prescription follows from that treatment session.

And unlike some psychedelic programs being developed elsewhere, Definium is not requiring psychotherapy to accompany the drug, potentially making the treatment easier for clinics to administer and insurers to reimburse if it eventually reaches the market.

There is currently no FDA-approved LSD medicine for generalized anxiety disorder.

That means DT120, if it successfully completes development and wins regulatory approval, could create an entirely new category of psychiatric treatment.

The FDA has already granted the drug Breakthrough Therapy designation for generalized anxiety disorder, a designation intended to accelerate development and regulatory review of medicines showing substantial potential improvement over existing treatments.

Investors Send Shares Higher

Wall Street immediately recognized the significance.

Definium shares surged in premarket trading Wednesday after the results were released, reversing much of Tuesday’s decline as investors reassessed the likelihood that DT120 could eventually reach the market.

The company also has considerable financial resources behind the program, reporting approximately $1.1 billion in cash, cash equivalents and investments at the end of June.

And anxiety is only one part of the opportunity.

In June, DT120 also produced positive Phase 3 results in major depressive disorder, meaning the same one-dose LSD tablet has now generated successful late-stage results in two of the largest psychiatric treatment markets.

One More Anxiety Trial Matters

Definium still has another major hurdle before it can declare the anxiety program complete.

Its second Phase 3 anxiety study, Panorama, is expected to report results in September.

If that trial also succeeds, Definium could have the pivotal evidence needed to move substantially closer to an FDA submission for generalized anxiety disorder.

That is why Wednesday’s announcement goes well beyond another biotechnology trial result.

For decades, LSD has been known primarily as an illegal psychedelic associated with recreational drug use and the counterculture of the 1960s.

Definium is now attempting to turn a precisely manufactured pharmaceutical version of that compound into something entirely different: a regulated medicine that a patient could potentially take once and experience meaningful relief from severe anxiety within days.

If the remaining trials confirm what Voyage has shown, psychiatry may be looking at the beginning of an entirely new treatment model.

JBizNews Desk | New York

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