We speak with a biotech CEO about the potential of psychedelic medicine – and the challenges of working with stigmatized substances
In recent years, psychedelics have returned to the spotlight – not as cultural curiosities, but as serious candidates for next-generation mental health treatments.
MindMed is one company working in this area, with the goal of developing psychedelic-inspired medicines through rigorous, modern clinical science. Results from a phase II trial for an LSD-based compound have been extremely positive – and the company is excited about what comes next. We speak with CEO Rob Barrow about the therapeutic potential of LSD for generalized anxiety disorder and the challenges of developing a stigmatized substance into a therapeutic medicine.
Why are psychedelics so intriguing for therapeutic drug development, particularly for mental health disorders?
When I first got involved in this field, I started digging into the historical data, and honestly, the potential of these compounds is remarkable – not just the magnitude of the effects, but the nature of what these compounds seem capable of doing. We're not just talking about symptom suppression here.
Historically, psychiatric treatment has focused on managing symptoms and helping people to get by. Drugs like SSRIs can be effective and life-changing for some, but they’re often an incremental improvement rather than a transformational change.
There is an enormous mental health crisis in the world today. We all know somebody with anxiety or depression. Psychedelic compounds showed promise decades ago, but were sidelined – not because of a lack of scientific merit, but because of cultural and political forces. It wasn’t the science that failed; it was everything around it.
It’s time to bring these substances into the modern era with rigorous clinical research and development – and potentially we can deliver some truly transformative treatments to people in need.
What are the stigmas and challenges surrounding psychedelics, and how do these translate into research and development?
When it comes to psychedelics, there’s both a historical and cultural weight to the conversation. LSD, in particular, has a very storied history. LSD played a central role in early psychiatric and psychopharmacological research before everything was shut down in the early 1970s. LSD was the most visible and researched substance – and the groups that latched onto it and other psychedelics like psilocybin outside of medical research led to a backlash.
Different countries and regulators responded in different ways. In the US, LSD was made illegal and placed in Schedule I of the Controlled Substances Act.
In recent years, however, there’s been a renewed openness to the therapeutic potential of psychedelics. Biotech development is a marathon and it takes time, but we’ve made tremendous progress. We had our first meeting with the FDA in 2020, and by 2026, we expect to have pivotal data from two studies in generalized anxiety disorder. By biotech timelines, that’s actually pretty quick!
How did MindMed come to focus on LSD?
We took an objective look at the science and became increasingly confident that LSD is uniquely positioned. It had a compelling scientific profile, and from a business standpoint, there was less competition and more room for innovation.
Our goal was to take this decades-old active pharmaceutical ingredient and modernize it with new formulations, delivery methods, and clinical protocols that can meet today’s rigorous regulatory standards. That work led to our first meeting with the FDA in 2020. It took some time to get our first trial launched, but by 2022, we had enrolled our first participants.
In March 2024, we released our first phase IIb data for our MM120 (lysergide d-tartrate) program for generalized anxiety disorder (GAD) – and the results were great, with both primary and key secondary endpoints met. A single dose led to strong and long-lasting reductions in anxiety, with nearly two-thirds of participants still showing significant improvements three months later. About half no longer met the criteria for an anxiety disorder at all. The treatment was well tolerated, and the results were strong enough for the FDA to grant it Breakthrough Therapy Designation – helping to fast-track further development.
The full results for the positive phase IIb study have been published in JAMA. 100 µg was determined to be the optimal dose, which is delivered via an orally disintegrating tablet, and is now being evaluated in pivotal phase III trials. MM120 was generally well-tolerated study, with most adverse events rated as mild-to-moderate, occurring on the dosing day, and being consistent with the expected effects of the trial drug.
We expect to see topline data from our phase 3 studies in 2026.
Maurizio Fava, a member of our scientific advisory board and Chair of Mass General Brigham Department of Psychiatry described the study as a turning point in the field. He said, “For the first time, LSD has been studied with modern scientific rigor, and the results are both clinically meaningful and potentially paradigm-shifting for the treatment of GAD. GAD affects 26 million adults in the U.S., yet no new medications have been approved since 2007—and first-line treatments fail 50% of patients. I have seen firsthand the devastating toll GAD takes on patients and their families, which is why it is so significant that a single dose of MM120 delivered rapid, robust, and lasting effects. These results highlight the promise of psychedelics in psychiatric medicine and represent a critical step toward expanding effective options for those who are suffering.”
We also have earlier-stage programs in the pipeline, but the centerpiece of our efforts right now is GAD and major depressive disorder.
How did you feel when you saw the phase 2 data?
I remember sitting with our Chief Medical Officer, Dan Karlin, who’s been part of this journey from the beginning. He’s a psychiatrist by training – and just watching his reaction as we pulled up the graphs was unforgettable. We had gone in optimistic, but the magnitude of the results exceeded our expectations.
At the 100 microgram dose, about 50 percent of patients went into remission from GAD twelve weeks after a single dose. That kind of rapid and durable response is incredibly rare. Even with a stronger-than-usual placebo response in the trial, the drug still beat it by more than double and by more than what we see with current standard-of-care treatments.
And it wasn’t just anxiety symptoms that improved, we also saw dramatic reductions in depressive symptoms as well. In fact, a majority of patients experienced remission from both clusters of symptoms. What’s been even more encouraging is how consistent the data has been. Every time we’ve re-analyzed it, the results hold up. No contradictions and no red flags.
Now as we move toward our phase III readouts, expectations are high.
Why did you choose anxiety as one of your key indications?
If you look back at the history of psychiatry, anxiety has been the predominant focus for much of history. In the early 1990s, when SSRIs came onto the scene, there was a “diagnostic drift,” where major depressive disorder became the dominant diagnosis. Depression started to absorb what had previously been considered more broadly “neurotic illness” – essentially a lot of what we would now think of as anxiety-related disorders.
Fast forward to now: the last FDA-approved treatment for generalized anxiety disorder came in 2007. During that time, the prevalence of diagnosed anxiety has skyrocketed. Even beyond formal diagnoses, awareness of anxiety symptoms and the impact they have on people’s lives has grown tremendously. When we looked at this space – both from a scientific and a human need perspective – it was clear that anxiety represented a profound unmet need.
Mechanistically, we also believe LSD has unique potential here. We know a lot about how it behaves in the brain, including its pharmacology, receptor interactions, and so on, but what is most fascinating is the lasting effect it can have.
What’s involved in manufacturing the drug?
We’re working with CMOs for manufacturing. We go through all the same rigorous qualification and compliance processes that any modern drug developer would. It’s especially important to emphasize this when dealing with compounds that have a history outside of the traditional medical context. When companies repurpose or reintroduce such drugs, there's often a legacy of stigma or misconception that needs to be overcome. But we’ve seen other successful examples of this in the industry.
Take sodium oxybate, for instance; it had a very negative public perception before it became Xyrem, a breakthrough treatment commercialized by Jazz Pharmaceuticals. There is also Epidiolex, which was the first FDA-approved CBD-based treatment. GW Pharmaceuticals (now part of Jazz) brought this product to market and, in doing so, really opened up the medical community’s eyes to the therapeutic potential of compounds that had previously been misunderstood or underutilized.
To get there, those companies had to follow the same rigorous manufacturing processes and drug development pathways as any other pharmaceutical product. And that’s exactly what we’re doing. We’re not cutting corners or doing anything outside the norm. We are working with top-tier vendors, qualifying them carefully, and building a robust, compliant supply chain to support our development and eventual commercialization efforts.
How has the regulatory situation for psychedelics changed since the early 2000s?
I feel very fortunate to be working in this field at a time when the regulatory environment continues to become even more open and engaged. We've been able to make real progress because there's now a broader understanding that these compounds – despite their history – have serious therapeutic potential.
One advantage we have in central nervous system (CNS) drug development, particularly with LSD, is that we already know it’s active in the brain. That might sound obvious, but in CNS drug development, it’s actually quite rare to start out with that level of certainty. With many drugs, you can spend years just trying to confirm whether the compound is doing anything meaningful in the brain.
When you had your first meeting with the FDA, how did they react?
By the time we had our first FDA meeting as part of MindMed, I had already had several meetings with the agency around other compounds in this class through prior work. So, I had some familiarity with how they were approaching psychedelics.
The FDA, to their credit, has been incredibly thoughtful and engaged on this topic. They’ve been giving presentations at conferences, talking about the therapeutic potential of psychedelics, the regulatory history, and how they’re thinking about the future of this drug class. They’ve approached this field with a real sense of objectivity. They are open and they are listening.
One memorable aspect of our meeting was just how many people attended. I don’t recall the exact number, but there were a lot of people there – many just to listen in voluntarily. This isn’t something you typically see for an early-stage meeting. But this was LSD – and it captured people’s attention.
This level of enthusiasm and curiosity was really exciting for us. It gave us confidence that we weren’t alone in believing in the potential of this work.
Science is about looking at things objectively – level-headedly and with as clear a lens as possible. We feel incredibly fortunate to have constructive dialogue with the FDA. But it doesn’t stop there. We’re now seeing that same engagement from congressional offices, the executive branch, and really from just about every stakeholder you’d hope to have involved. That kind of comprehensive support is a critical piece of the puzzle. It is what makes progress possible.
About the Phase IIb Study
The MMED008 phase IIb study was designed to explore how a single administration of MM-120, a pharmaceutical formulation of LSD, might help people with generalized anxiety disorder (GAD). The trial enrolled 198 adults across 20 clinical sites in the US, all of whom were living with moderate to severe anxiety symptoms at the start of the study. To ensure that the drug’s effects could be measured in isolation, participants were tapered off and fully washed out from any existing antidepressant or anti-anxiety medications before taking part.
No structured psychotherapy was provided alongside the drug, making this one of the first large studies to examine LSD’s therapeutic potential as a stand-alone treatment rather than as part of a combined approach.
On dosing day, participants were randomly assigned to receive a single oral dose of MM-120 at one of four strengths (25, 50, 100, or 200 micrograms) or a placebo. They remained under clinical supervision for the entire dosing session, which lasted several hours, to ensure safety and manage any perceptual or physical effects. The experience was not repeated: each participant received only one administration, after which they were followed for twelve weeks. The main goal was to measure the change in anxiety symptoms four weeks after treatment, while secondary measures looked at outcomes across the full three months, including safety, tolerability, day-to-day functioning, and quality of life.
The results pointed to the 100-microgram dose as the most effective. Four weeks after dosing, participants in this group experienced a far greater reduction in their anxiety scores than those given placebo, with an effect size large enough to be considered both statistically and clinically meaningful. By twelve weeks, about 65 percent of patients in the 100-microgram group had a strong clinical response to treatment, and nearly half no longer met the criteria for GAD. The higher 200-microgram dose did not improve outcomes and was associated with more intense perceptual effects on the dosing day, while the lower doses were less effective.
The full results have been published in JAMA.
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