atai Life Sciences co-CEO details VLS-01 trial success - ICYMI

atai Life Sciences (NASDAQ:ATAI, ETR:9VC)’ co-CEO Dr Srinivas Rao joined Proactive to discuss the company's latest clinical trial results.

Dr Rao detailed the positive outcomes from the Phase 1B trial of the VLS-01 (oral DMT) program, emphasizing the drug's excellent tolerability and potential as a best-in-class treatment option.

Proactive: You're out with some exciting clinical trial results today. Can you tell us about the VLS-01 program? What do today's Phase 1b results mean?

Srinivas Rao: Yes, we are very excited about these results. So, we started with this program a couple of years ago with the idea of having a product that was very easy to use and well-accepted by doctors and patients. Something that could fit into our paradigm that J&J has established, for example. That's something that was very important to us. We've been tweaking the formulation over the past couple of years. We did a Phase 1, reported some results in the fall of last year, learned a lot from it, and updated the formulation to test that new formulation in the current Phase 1 trial we're reporting results on.

And what we found is that it was very well tolerated. The formulation actually exceeded expectations in terms of pharmacokinetics, the amount of drug actually put into the body. It did better than we anticipated, comparable to intravenous administration. So, we're very excited about that. The tolerability was really good, potentially best-in-class. Many psychedelics are associated with some degree of nausea and vomiting. At the dose we're moving forward with, 120mg, we saw one instance of nausea in 14 exposures, about 7%. We had no vomiting, low incidence of headaches, and no local issues, which sometimes occur with intranasal or oral administration. So, we're really excited about that.

We're taking that intermediate dose, 120mg, forward into a Phase 2 trial, a robust trial for treatment-resistant depression. We've had discussions with regulatory authorities about the design, and everyone is very comfortable with it. We anticipate results from that trial around the end of next year.

Maybe to address the elephant in the room, the FDA made a decision on Friday not to allow MDMA [-assisted therapy to be approved for PTSD]. What are your thoughts on that?

It’s important to note that there were several idiosyncratic elements to that program. The folks at MAPS, particularly Rick Doblin, deserve lots of credit for all their work over the decades to advance this program. But it was almost generational. The IND was submitted in 2021, so it has been a very long journey. The trials were small, and the way the program was positioned with MDMA-assisted therapy, which is not under the purview of the FDA, was unusual. The trials were tiny, with a total of about 180 people in the two Phase 3 trials.

This is distinct from COMPASS’ Phase 2 trial, which had 233 subjects, and their Phase 3 program, which involves over 800 participants. They did some other things differently, focusing on therapy, which was not standardized, giving therapists latitude in treatment approaches. This raised concerns about functional blinding, with therapists possibly knowing which patients received the drug. There were also data integrity questions and other issues that were raised at the advisory committee meeting.

The FDA did not reject the program outright; they acknowledged the promise of the product but required another Phase 3 trial. This is somewhat positive for the rest of the space because it shows that lessons have been learned and are being implemented by other companies, including ours. We are conducting trials with large sample sizes, thorough adverse event assessments, and standardized approaches.

Are there any other developments that investors should be looking out for this year?

Yes, a couple of things. We also had our earnings release, and in terms of financials, we have guided that we have $103 million in cash and cash equivalents. We're reiterating our guidance for a runway through 2026. Another thing we mentioned in the press release is our second program, which ties into the discussion around Lykos. Lykos is developing MDMA, which is a racemate, consisting of two molecules that are mirror images. We wanted to focus on the (R) enantiomer, which is thought to have most of the therapeutic effects. We conducted a Phase 1 trial on this and found it to be a really interesting drug with features that were psychedelic-like, very different from MDMA.

We're moving forward into a Phase 2 trial in social anxiety disorder, with the trial set to kick off towards the end of this year. We anticipate a readout towards the end of next year. So again, really excited about that as well.

Quotes have been lightly edited for clarity and style