COMPASS Pathways plc (CMPS) September 15, 2026 Conference Transcript

Hello, everyone. Good afternoon, and welcome back to H.C. Wainwright's 20th Annual Global Investor Conference held on September 14th of 16th, 2026. I'm Patrick Trucchio, Senior Healthcare Analyst at H.C. Wainwright. It's my pleasure to welcome the management team from Compass Pathways, CEO Kabir Nath, CCO Lori Engelbert, and Chief Patient Officer Steve Levine. Compass is a biotechnology company that's pioneering development of a new model of Psilocybin Treatment, Comp360, that's intended to address serious mental health disorders. Comp360 has generated positive data from two Phase III trials in TRD, and we are expecting an approval next year in 2027. So first, maybe we can begin by introducing Comp360, how it works mechanistically, how it's administered, and why you've chosen to pursue it initially in TRD.

Thanks very much, Patrick, and thanks for the invitation to be here. Just a reminder, we will be making some forward-looking statements here, so you should refer to our many risk factors in our various findings. We have had breakthrough designations since 2018, based on some of the early work and the resurgence of interest in psychedelics in 2010 onwards. Actually, TRD was chosen because that's where both regulators and the founders of the company felt was a very large unmet need. At that point, there were actually no drugs recently approved for treatment-resistant depression. And in talking to psychiatrists and patients, it was very clear that there was a large gap there. And hence the decision to go into treatment-resistant depression, which we believe was exactly the right thing, as we'll talk about from a commercial and scale-up possibility. Steve, I don't know if you want to comment briefly on MOA and what a COM360 session looks like?

COMP360, which is a synthetic form of psilocybin, is a psychedelic, which acts very differently in the brain relative to conventionally known antidepressants, which are typically taken on a daily basis. We've administered COMP360 one or two initial times within our Phase III trials, and ultimately this will be a very infrequently delivered treatment on the order of one to four times per year. The compound initially acts as serotonin receptors, 5-HT2A receptors, and elicits an initial reaction that requires it to be delivered in a supervised medical setting, but with elements of an experience that may be correlated to the positive therapeutic outcomes, which may lead to shifts in depressive patterns of thinking that are durable long beyond the action of the medication of the body.

So following two positive phase three trials, COMP005, COMP006, can you summarize the key data?

Sure, no, happy to do that. So COMP005 was placebo-controlled, single dose of 25 milligrams against placebo. COMP006 is two fixed doses three weeks apart and compared against 10 milligrams and one milligram. In both cases, we saw that at the primary endpoint of six weeks, we saw a statistically significant separation in change from baseline of Madras of just under four points on both. What we saw was rapid onset, measured as soon as the next day in every case. And with the single dose, roughly a quarter of patients with a clinically meaningful response. With O06, with the two fixed doses, 40% of patients with a clinically meaningful response. We now have data out to 26 weeks, and we ran these trials fully blinded to 26 weeks, which I think is probably unprecedented in depression. And what we see, therefore, is that one, two, or perhaps three doses produce sustained durability out to at least 26 weeks, because that's what we have at the moment, that we do have some open-label data, which we'll come to in a second, produce sustained durability. So the combination of rapid onset, sustained durability, limited dosing is unprecedented in depression, and therefore is a potential complete paradigm shift, particularly given the only other drug approved, which is Spravato, is going to be.

So then where exactly does the rolling NDA stand today, and what are the final items needed for the expected fourth quarter submission?

So we have submitted virtually, I think, all of the modules except clinical at this point. We do now have all the data we require for the complete clinical module in hand, but these two phase three studies are close to 900 patients between them, so putting together the integrated summaries is clearly a significant amount of work. That is the final piece, and that we will complete in quarter four, and we're still on track for that.

Can you remind us through which week the phase three trials remain blinded through, and then as well, what have you seen on durability, and what do you have in anticipation of what the expected intervals would be between dosing sessions for patients.

So the blinded period is through 26 weeks, so that was through our Part B. We did just release Part C data, which took us out to 12 months. And that was open label from 26 weeks out to fully one year. And what we saw is exactly like Kabir said, is that with one or two initial doses, you saw a very dramatic, very fast, very rapid onset of action. And And I just want to quickly just remind everyone, this is treatment-resistant depression. This is not general MDD. This is treatment-resistant depression where these patients have, on average, been in their current episode for about three years. So they have tried multiple products and had no success with these products. And we were able to show, after one dosing session, a very rapid onset of action within 24 hours. That is unheard of and unlike anything available for treatment-resistant depression right now. And what we saw is that with potentially one additional dose, that durability lasted out through the blinded period of 26 weeks. With the open-label data where patients were all either blinded or on the placebo arm or eligible for an additional dose at 26 weeks, when they received that additional dose at that time period, they were able to sustain that durability out through the end of the year. So this is really incredibly promising data. The only other pharmacologic product that's available for treatment-resistant depression right now is Spravato, and that would, on average, take about 25 to 50 administrations in a year. That's 25 to 50 days off of work that a patient and a caregiver would need to take to go and have this product administered, whereas we have shown data in this patient population, Again, a very, very, very difficult and hard patient population to have efficacy after somewhere between two and four doses on an annualized basis.

Right. That's interesting. And you've also received the national priority voucher. What does that mean? What does that actually do to the review clock? And does the rolling review mechanically change, or what does it change about how the FDA engages with you?

Yeah, so the National Priority Voucher commits the FDA to a target of approval in 60 days or less, though they are very clear to stress that it's a target, not a commitment. But more importantly than that, and obviously that at the back end would be nice to see, much more importantly, it really has transformed the way that you work with the FDA. So we are already, as you noted, on a rolling submission and rolling review. And first, the rolling review is actually happening.

So we are getting IRs, we're responding to IRs, there is dialogue.

Other elements that would normally come later in the review process are already underway, including a draft label, REMS framework, the eight-factor analysis, inspections have already started. So it's really not just at the back end where we obviously hope to gain that time, but it's really in the way we're working now with a very engaged psychiatry division. But just to call out, they have been engaged and supportive throughout our development program ever since Karate Gospray through designation, but it's really that way of working now that is a significant change.

So the final FDA psychedelics guidance was published in July. Were there any significant changes in that guidance relative to the draft guidance? And maybe you can talk more broadly about just engagement with the FDA and just sort of the openness of the FDA and how the the FDA is viewing psychedelics in COMP360?

Sure, so the draft guidance in June 23 came out shortly after we had finalized the first phase three studies in classic psychedelics, and it was a pleasure but also not a surprise to see that the description of an acceptable phase three program described our phase three program in that draft guidance, and that is carried over into the final guidance. Yeah, there are a few changes. Some of the same concerns that they have remain there. There are, we all acknowledge, open questions still more broadly around durability, around the role of therapy to supplement the effects of psychedelics. And just to be very clear, this is not about whether therapy is needed in the treatment. We are getting a drug approved, not a drug therapy combination. But there are clearly questions that the FDA still has about in the broader medical context. But no, there were no surprises for us in that. There was one comment about guidance on 12 months blinded. in psychiatry really expects to run a 12-month blinded study in psychiatry.

I'll ask my psychiatrist friend here to comment on that. Well, just to also say that, as said earlier, we have blinded data out to week 26, which is already far surpassing the length of the blinded period of other psychedelic trials. And so we feel quite confident that that will be an acceptable study design and, frankly, would be even unethical to blind studies out to a full 12 months, particularly in this population.

Can you walk us through operationally what we should expect from the filing to the DEA rescheduling to potential approval and launch? What will this cadence look like?

So I'll cover the filing of regulatory briefly. So as I say, we will complete the submission in Q4. We would hope that there's not a full 60-day acceptance period, but obviously we don't that is the agency's prerogative. Our assumption is that there will be a 60-day acceptance, that the MPV clock will only start at that point. Again, we think we may be issued a six-month PDUFA with the agency's intention to beat it, but candidly, we just don't know. There aren't enough precedents, so I'll turn to Laurie to talk about rescheduling and so on, and then we'll come back to the timelines we've put out there.

Yeah, so we very clearly have stated that we will finish are filing in Q4. And given where we're sitting with not only the national priority voucher, but also the executive order that was issued about the same time back in April, the national priority voucher, which Kabir has referenced several times, had a target review time of one to two months versus typical priority review times of six to eight. And so that is where we are expecting to gain in terms of timing. Then with the executive order, the executive order encouraged the DEA and the FDA-controlled substance staff to work closer together to pull forward the DEA rescheduling timeline. DEA statutorily has about 90 days to reschedule a Schedule I drug after an FDA approval, and they typically hit it. And so we're working in a window if it could be anywhere from one day to 90 days with a DEA rescheduling. So there is that piece that needs to happen. Once the federal DEA reschedules, then the states need to reschedule. We've done a tremendous amount of work as a company to make sure that all states, as many as possible, about 93% of the population now sits in a state that will reschedule within 30 days of federal. And so we are sitting in high uncertain timelines in terms of when we will, you know, actually get an approval, when the DEA will reschedule. But we have pretty good timelines on when the states will reschedule. Go ahead.

Yeah, that's why, Patrick, our guidance right now is launch in the first half of 27, because we think it's likely we will land somewhere in there. But we've also clearly said we will be launch ready by the end of the year, because there is clearly a very unlikely but outlandish scenario where both approval and DEA rescheduling happen by the end of this year. Very unlikely, but we have said we will be launch ready, and we will be.

Right. Interesting. So then just on the commercial side, you've pointed toward more than 8,000 existing interventional psychiatry centers offering multi-hour treatments. What does a site actually need to deliver Comp360, and what have you been doing to make sure the sites are prepared to administer it at launch?

What do sites need? In short, what they've already got. And it sounds facetious, but it's literally true, in the sense that those sites that are already capable of delivering in-office multi-hour treatments like Spravato are prepared. The rooms that they're using to deliver Spravato today are the rooms that will be used to deliver Comp360 at launch, and the clinical staff that are currently monitoring patients receiving those treatments is the same staff that will be used to monitor Comp360 sessions. That staffing, just to give a little bit further detail, is typically team-based. It's a multidisciplinary team. Typically, there is at least a single prescriber on site who may be a psychiatrist, but it could also be a nurse practitioner or a physician's assistant. And the rest of the multidisciplinary team may be made up of medical assistants, technicians, nurses, sometimes therapists. And they work as a team. They work by committee, monitoring multiple patients, having treatments in multiple rooms simultaneously that today include Spravato along with treatments like TMS, which is transcranial magnetic stimulation. And they have built capacity ahead, anticipating that Comp360 and potentially other psychedelics will be approved to be ready for that.

So I know there's been a lot of effort put toward, you know, making sure we have the proper codes in place and reimbursement structure in place.

Maybe you can walk us through that process and where that stands and just level of confidence that all of that on that side things will be sort of ready at launch yeah so while we will be selling a drug and we will apply for the appropriate code a j code to make sure that drug can be reimbursed and immediately post approval we are also aware that if this treatment is not economically viable for providers to deliver that prescriptions won't be written and we fortunately are in the position to follow J&J's launch of Spravato and see some of the challenges they had early on, because there wasn't a reimbursement framework in place for those providers to bill for their observation time if they would deliver that treatment. That really led to a lot of confusion, delays or denials of claims, and a very confusing situation that has taken multiple years to iron out. And frankly, even today, although it's clearly viable because J&J is currently selling Spravato at about a $2 billion run rate, it really still isn't optimized because the codes that they're using are generic office visit codes that really don't capture all of the work done in these visits or the extensive practice expenses involved in complying with the REMS, ordering and storing and handling a controlled substance, all of the capabilities these sites have built, but they're not really paid for that time. We've been able to take a note from that and take a different approach, which was a few years ago to apply for new CPT codes that are specific to the administration of a psychedelic treatment. And that means they will capture all of the value, all of the work done, and those practice expenses. They are currently in their Category 3 form, which is a tracking code. Post our approval and the usage of these codes, they will be progressed to a Category 1 status, And we will have field reimbursement teams out at these sites, helping them to understand how to appropriately code so that there isn't friction with reimbursement.

So, you know, the durability data is quite compelling, especially relative, and the number of doses and treatments per year is very compelling relative to Spravato. So I'm wondering if you can kind of discuss how we should think about, you know, a six-hour in-clinic treatment, but with far fewer of those treatments per year, and how the clinics are going to view that relative to, you know, 25 to 50 Spravata treatments per year?

Well, first, these centers have capacity. So particularly initially, they're not going to make trade-off decisions between the profile of one drug versus another. They'll be making selections as what is appropriate for patients. But in any case, this will be incremental revenue for them. Any treatments they deliver are incremental. Beyond that, there will be potentially a point down the line when they have multiple options to choose from and maybe be, you know, thinking more about the differentiated profiles of these products. Speaking to your question, most importantly, something that's overlooked sometimes are the challenges for sites having shorter treatments where they need to turn the room over frequently in order to keep that room full, because if the room isn't occupied, they're not getting paid. That comes with additional administrative complexity. It also comes with time they're not paid, because there's going to be gaps between those treatments as they're turning the room over and cleaning it and so on. Ultimately with these new codes that are reportable on an hour-by-hour basis, being able to fill that room for a longer period of time and having the confidence that you'll be paid for the entire time becomes an advantage for these sites.

That's interesting. And I did want to ask about PTSD. So this program has now moved to later stage development. Maybe you could just walk us through the data you've seen in this program so far. Why is PTSD potentially a compelling indication? And then maybe walk us through your late stage program.

Laurie, you start with the need.

Yeah, this is an incredibly exciting space to be developing products. And the last product approved was 25 years ago. And there were only two that have actually been approved. And so there is a tremendous unmet need, I'm sure everyone's heard about PTSD and I'm positive you've heard about it in our veterans, which is an incredibly important patient population. But they actually don't make up the entire patient population or even the majority of the patient population. The patient population is about 13 million patients with PTSD, about 60% of them are women. And only 15% are veterans. Again, very important patient population, but they're not the full gamut of PTSD. So the unmet need is huge right now.

So, we ran an open-label phase 2 study, 22 patients, principally designed for safety because there were clearly some concerns that the psilocybin experience could somehow be triggering or trigger new safety events in that population. We absolutely didn't see that, and what we actually saw was profound efficacy in that open-label population with the vast majority of patients with a deep response and actually close to 80% in remission with 12 weeks. Based on that and based on the fact that there is now a lot of smaller studies with psilocybin and PTSD, we have designed a late-stage study that is now underway. It's a 300-patient study. Again, three arms with an intermediate dose, which is very much the same design as we use for treatment-resistant depression to manage expectancy and functional unblinding. Two fixed doses, we decided that based on the experience from 006, that was the right thing to do to really kind of maximize the likelihood of efficacy. That study is underway and, you know, it's been designed, as I say, as a very robust late-stage study. And, you know, as we see the tailwinds both overall within the agency around some flexibility, but also specifically in psychedelics, the fact that the safety database from TRD is clearly applicable to PTSD, our current hope and intent is this would be a single registrational study but again obviously that will be a review matter in due course but we're very excited about it we actually see it as something that uh investors in general really haven't started to get their heads around because actually we think it's potentially a bigger opportunity than trd and beyond trd and ptsd how should we think about a broader comp 360 opportunity so it's a question of choice i mean there are a number of areas i think an area that's increasingly coming to focus but we haven't made any commitments there, is SUD in general, but particularly alcohol use disorder. There is one fairly robust phase two study. There are some other proof of concept studies. And clearly, from an overall public health and cost burden, alcohol use disorder is very significant. And there is a lot of interest potentially in looking at that. Again, no commitments at this stage. It's just one of the areas we could look at.

Maybe just as a final question, what do you think investors are missing about the Compass story?

I think what's about to happen in psychiatry is unlike anyone seen in many, many years, and certainly not in psychiatry. The data that we're producing and psychedelics are producing for this patient population is truly going to change the way that mental health is treated going forward. And I think, you know, people believe and always give that first mover advantage or somewhat disadvantage when it comes to investors because we need to knock off the warts, so to speak, of getting something this revolutionary to market. We've luckily have J&J's infrastructure there, so we're not starting from scratch. And we have one heck of a team that has come together with many, many years of launch experience to really think about all the different avenues and ways to make sure that we're successful once we get to launch.

Well, and alluded to just a moment ago, but I think also really not giving credit to the PTSD program, which we are really excited about.

And as Kabir said, is potentially a bigger opportunity even than the enormous opportunity And I think, I mean, just to build on both these, it is the fact that the infrastructure does exist, and while it's not a perfect plug and play, we have done an enormous amount of work to make sure that these sites are ready for Comp360, and I think people just miss that.

All right. Well, Steve, thank you so much for being with us. Compass team, thank you. Thank you, Patrick.