Health Care

Navigating the Current State of FDA with Praxis Therapeutics

Mar. 12, 2026

Guest: Marcio Souza, CEO, Praxis

Host: Ritu Baral, Managing Director, Health Care – Biotechnology Research Analyst, TD Cowen

TD Cowen senior biotech analyst Ritu Baral speaks with Praxis CEO Marcio Souza at the TD Cowen 46th Annual Health Care Conference to discuss FDA functionality following recent leadership changes and destaffing, approaches to working with the agency on complex datasets and implications for the broader biotech sector. Drawing on Praxis’s extensive regulatory experience, Souza notes that despite chronic FDA understaffing, engagement with the Office of Neuroscience has remained stable and highly productive. He underscores the value of incorporating FDA guidance early in endpoint and trial design in areas such as essential tremor, where regulatory precedent is limited, and emphasizes that frequent, senior-level interaction and a patient-centric framing of complex data can help drive alignment and constructive regulatory dialogue. He adds that investors can gauge the quality of a developer’s relationship with the agency by looking at the recency of FDA communications and the level of senior leadership engagement during recent interactions.

This podcast was originally recorded on March 3, 2026.

Full transcript

Announcer:
Welcome to TD Cowen Insights, a space that brings leading thinkers together to share insights and ideas shaping the world around us. Join us as we converse with the top minds who are influencing our global sectors.
Ritu Baral:
Hi. I'm Ritu Bural, one of the senior biotech analysts at TD Cowen, and I'm here live at the 46th Annual TD Cowan Healthcare Conference. I'm here joined by CEO of Praxis, Marcio Souza. Marcio, welcome.
Marcio Souza:
Thank you so much.
Ritu Baral:
I've got some questions for you about the biotech sector and its relationship with the FDA. How do you see biotech developers viewing the functionality of the FDA right now, given the staffing and independence concerns that have been in the news?
Marcio Souza:
Absolutely a super important topic for us to discuss, since we're all impacted by the FDA. Very prudent as well right now to talk as we start a new year at the agency, and we always expect what changed, what is still going on. I prefer to take a more historical view, I think, to answer your question too. If you look into the history of drug developments and the FDA action, what you see is there are times it gets a little faster, one could argue more flexible, others can argue a little bit more aggressive. But when you look into the recent time, I'm going to take the noise out of the equation here ... there's a lot of noise ... and really try to look into the signal.
And of course, I can speculate on the states for the industry in overall, but I can only really speak about our experience with the agency. And in our experience, we have four drugs in development in the clinic, many others that we are going to be bringing into the clinic soon, two NDAs submitted, so a lot of regulatory interaction, very focused on the U.S. It's been actually fairly stable and, I would say, very productive, very healthy discussions with the agents, that we are uniquely positioned only on the Office of Neuroscience with two of the divisions there right now. So it might not be the case for the entire agency.
But I think it's worth mentioning, since you mentioned the staff, that the FDA has been chronically understaffed for many, many years. There's been discussions, as you might recall, and hearings during many PDUFA reauthorizations about the staff in the agency, so I don't believe it's an acute issue. I believe it's real. I heard recently, I believe the commissioner is saying a thousand scientists should be hired this year, so there's an acknowledgement by leadership as well that something has to be done. But so far, I haven't seen that impacting either the speeds or the quality of the reviews, which for us are the things that matter the most.
Ritu Baral:
How does a company best approach FDA in unique situations, or with unprecedented but complex data sets?
Marcio Souza:
We have, I would say, both, and I would say unprecedented because there has now been a drug approved for essential tremor in the case of ulixacaltamide. Hydrofluorates, that is one of our applications. We had to get advice on the endpoints, on the trial design, on the execution, working throughout the years with the agents here. I think there's a healthy balance between us executing all of that and the agents advising the best that they can, and for us distilling that advice and really doing what is best for the company, the drug development, and at the end of the day for the patients as well.
What I've seen along the way is that when you take the position of what is best for the patients ... and that's always the position we take ... it becomes a lot easier, the conversation, versus what is best for the drug, what is best for us, what is best for the agency, in their case. That common ground is how I would arguably advise one to approach, is that just trying to find, as anything else in life, when you look for common ground is normally where the solutions lie versus if you look to self-interest.
And interact as frequently as you can. Use all the elements. Use all the types of meetings. Don't use only the meetings that people normally use. Use written response only as well, which takes less resource from the agents but it still gives you advice, so let's say go very wide. The current federal code has so many options for interactions with the agency, and we're proud as a company we use pretty much all of them. And again, as I said, our biased view is that it's been quite productive.
Ritu Baral:
So given the investor focus, what questions can investors fairly ask management teams to determine that a developer is responsibly navigating this new regulatory landscape?
Marcio Souza:
I think it goes back to the last comment I made. How recently have you actually interacted? What is the level of seniority involved in those meetings? We're talking about more staff or we're talking about more senior involvement, and are we following most of the advice or not? I think it's fair to try to understand what is the level of alignment. What are the main areas of disagreements? We don't have to agree on everything, but what are the main areas?
And look into the previous reviews, if one exists on that area. Look into previous interactions if those exist, because the agency is pretty good about following precedent in general. I think they've been very forward lately on publishing the new guidance in the future, what they're going to focus on, like the plausible mechanism, for example. We first had blurbs and then we had a few paragraphs and now we have guidance, and that evolution I think was telegraphing to all of us the evolution of the agency.
We personally, on that one, we actually asked the agency in between, "What kind of application, how are you thinking about this," informally. In a meeting we had, we took a few minutes and we asked another topic, and it was incredibly helpful to understand how they were thinking about it. So I think oftentimes when I talk to my colleagues, like other CEOs or other leaders in the industry, they're a little bit shy, almost treating the agents as if they're not humans. They're humans. Treat them as humans.
Maybe the last thing I would say is in recent years, I didn't come across any one of the agents who don't love helping patients. They always do. They love helping patients. And take that angle, really better share that angle with them, and things are going to be better.
Ritu Baral:
Great. Thank you, Marcio.
Marcio Souza:
Thank you.
Announcer:
Thanks for joining us. Stay tuned for the next episode of TD Cowen Insights.

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Ritu Baral

Managing Director, Health Care – Biotechnology Research Analyst, TD Cowen

Ritu Baral
Managing Director, Health Care – Biotechnology Research Analyst, TD Cowen

Biography

Ritu Baral joined TD Cowen in August 2014 as a senior biotechnology analyst and managing director. Ms. Baral has more than nineteen years of experience in biotechnology finance, including over sixteen years in biotechnology equity research. Her coverage has focused on rare diseases and neurology. From August 2006 until June 2014, she held a series of positions in biotechnology equity research at Canaccord Genuity, including senior analyst and managing director. Before that, Ms. Baral was an equity research associate at JMP Securities and a senior associate at the Trout Group. Previously, she was a research associate at Columbia University’s Department of Medicine, where she participated in neuroendocrine research focused on appetite and metabolism regulation, and completed graduate coursework in immunology.

Ms. Baral graduated with a BA in biological sciences from Barnard College. She is involved in a number of rare disease patient advocacy organizations, including as a board member of the Everylife Foundation for Rare Disease and the Industry Advisory Board of the National Tay-Sachs and Allied Diseases Foundation. She was previously on the board of directors of the Pulmonary Fibrosis Foundation.