Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Good morning, everyone.Wonderful to kick off our healthcare conference, and excited to have Praxis Precision Medicines here with us.We've lots to cover over the next 25 minutes, so let's get started.Want to congratulate you on the epilepsy announcement today, and we'll get to talk about it-
Marcio Souza
President and CEO, Praxis Precision Medicines
Thank you.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
-in a second.But maybe let's start off with ET, and then move into epilepsy.I think the first question on Essential ET, that you're kicking off, which is amazing, is I think during the last R&D Day, this late fall, you talked about how you have 600 patients that have been identified and ready, and they have been in the screening process.So could you maybe talk to us a little bit about how the screening process is going?
Marcio Souza
President and CEO, Praxis Precision Medicines
Mm-hmm.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
What percentage of the 600 patients are in the queue being reviewed?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah, yeah.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
We'll start there.
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah, absolutely.No, thanks for the invitation, again, for being here, and always, it's very nice to talk to you.Yeah.The...Maybe I will expand a little bit further-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
On that question, right?What we mentioned on R&D Day, a few months ago, was that we were able to, through a consenting process, like,
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... what we'd call a small study, that was on request for a number of patients-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... and over 600 of them-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
engaged with us on how was their tremor, how willing they were to participate in a trial.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
that we would sponsor.So it was very specific.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
Praxis study, and a number of other questions that would help us understand how many of them would qualify-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
to this study.Now, the source of those 600 patients were a larger database that we built on the last,
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... 12 months or so, as we're enrolling Essential1 -
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
As we continue to engage on interest on clinical studies from qualified patients, coming primarily from healthcare providers.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
or Praxis that believe patients would be eligible, and from the Tremor Foundation.That totals to a little bit over 2,000 patients.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Wow.
Marcio Souza
President and CEO, Praxis Precision Medicines
Right?So that was our base.So our recruitment strategy on the beginning of this study-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
was going back to the 600, of course, and then slightly-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-uh, up that-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
to the 2000, and now we are already there, and beyond there.So far, without giving specific numbers-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-we're going to keep that a little bit close to the vest.We're being very overwhelmed-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
like, positively, with the interest, with the engagement-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
I'll, I would argue, as well as the suffering-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
that goes through here, right?This is a very serious-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-disease, been neglected for so long.These patients are very engaged with us.The process is going very well.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
I'm incredibly happy, absolutely not satisfied-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... which is what our team here, every day, the number one priority for us-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
Is to screen the right patient.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
To get the right patient for the process.So the process is severalfold, but the first one is a formal screening and then the-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-eligibility review, for these patients.That gate-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
makes sure that the right severity, the right diagnosis.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
concomitant medications, lab results
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
All are wrapped up, so they, they can randomize.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
So the commitment we had is to get this right enrolled by the first half of next year.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
And I'll tell as it stands here today, I'm very confident.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
that, that's what we're going to be able to deliver.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Could you maybe talk about the process of the screening?Because some investors that I've been speaking with lately, they have this thought process that you get a patient in, you screen them, and tomorrow you can get them on drug, right?So I think it's important to understand that's not the case.There are many steps and assessments that are put in place, and the average...So if you could just kind of-
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
... capture what truly goes into screening.What's the time range it takes for screening just one patient on average?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
So people have a real expectation of these processes are moving.
Marcio Souza
President and CEO, Praxis Precision Medicines
So from the time we engage-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
with a qualified patient, and I'll call him qualified patient.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
-someone who already has Essential Tremor coming from us, like a verification system that we have, to the time that a given person-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
could randomize, is about a month.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay.
Marcio Souza
President and CEO, Praxis Precision Medicines
and that goes through both collection of medical records-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-which we'll review, and a few other things, assessments, screening assessment for, for different endpoints-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
- and things like that.We do have multiple assessments-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-for baseline.So the, what you're trying to learn from the previous-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
study that was done in this space is, probably getting more than one assessment before the baseline.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
Is a good idea to reduce variability.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
and that's what we did here.So all that process, about 28 days-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay
Marcio Souza
President and CEO, Praxis Precision Medicines
to get the patients on this study.So not overnight whatsoever.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.No, that's helpful.And then, who is screening?Is there a central committee that reviews all this and then-
Marcio Souza
President and CEO, Praxis Precision Medicines
Mm-hmm, mm-hmm
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
And then assesses eligibility?How is that controlled for?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.So there's a number of steps, and I'm not going to bother you-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
with all of them.But the most important one is when all the information is collected, is the same group of physicians.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
like there is an eligibility review committee.They're looking through every single-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay
Marcio Souza
President and CEO, Praxis Precision Medicines
One of them.That comes from a standardized exam-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-that we developed.It, it's, I would say, a little bit more advanced than the regular, exam to measure different-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
aspects of the disease... to assess stratification factors, and so on.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
So that, that's what happens.Once they give the okay, I'll have to assume everything else-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
remains the same, then the patient can be randomized.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.Okay.Sorry about that.
Marcio Souza
President and CEO, Praxis Precision Medicines
Okay.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
The other question that we get is, you've also publicly said that you will announce enrollment updates.How do you visualize sort of the cadence of sort of the disclosures over the next first half of 2024?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.I think we're trying to balance, give enough confidence-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
To all of you, who
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
are being, and we're very grateful
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
investors in the company to...On the other side, right, to the certainty.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
on the ends.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
So what you're going to see moving forward from us is more increasing the certainty on the end of the process.So this is not going to be, like, every couple of days-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
-or every 15 days, but, we're going to give periodic updates.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
Probably more in large increments.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
So you can see really it's moving on the proper direction here.But, if I can measure by the start-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
of the process, I think we're going to be all very pleased to how we do that.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
When you say increments, increments of what?Increments of the hundreds, potentially, or?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah, that wouldn't-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay
Marcio Souza
President and CEO, Praxis Precision Medicines
... be unreasonable.I think we're going to see the time of disclosures-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
reg recalls like this one.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
and others, and how we can get there.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
No, that's, that's, that's great.The other question I want to talk about is, you guys, once you connected your phase IIb, you really stepped back and really reviewed the execution of the study, and you really implemented many aspects in Essential3 to really ensure success.Very briefly, like, maybe talk about three or four of the main strategies that were implemented.We talked about the screening, we talked about the committee, the eligibility committee, but what else has been implemented that you think is important for investors to understand?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah, well, the nice thing about the way we operate as well, quality is not negotiable-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... at Praxis, right?So if you look into the way we run this trial-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm
Marcio Souza
President and CEO, Praxis Precision Medicines
that's never been in
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... the way we operate, it's never going to be like-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... like a quality issue.So we keep that quite fixed-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... as a parameter.So everything will go back and say: Can we do something-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... even better here?But that, that was never a, an issue for us.So the other aspect, you just talked about refining-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... eligibility, refining, and by refining, I mean reducing-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... the number of people who touch the process and keeping the same group.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
So reducing the intra and inter-rate variability, there was quite important for us.We learned couple aspects, for example, family history-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... as an important covariate.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
Now it's a stratification factor.Intention tremor-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... was an important-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... covariate, so it's a stratification factor.So those learnings were put as the-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... arguably one of the biggest learnings, that we only need one dose.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
So that, the one dose-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... going in, into the study as well.So all of those came to play.And maybe the biggest of all is the-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... the fact that we know how to recruit this patient-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... very well, that allow us to keep the trial moving quite nicely.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay.Maybe just walk us, I think, just as a reminder, your primary endpoint, powering assumptions-
Marcio Souza
President and CEO, Praxis Precision Medicines
Mm-hmm
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
... and what's considered clinically meaningful in this patient population?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
So, just to remind everyone about Essential I-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... there were two endpoints that were combined.The largest proportion of the combined endpoint was the TETRAS-ADL-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... and then a smaller proportion from the performance scale.But those are two endpoints, right?
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
When you separate the endpoint, we had a number of conversations with the FDA about the performance of-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... the performance scale, not on drug.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
The performance of the scale, and I think we got to an agreement that the scale-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... really doesn't work
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... for studies like this, so we abandoned that.So if you go back to Essential1 and look exactly what we collected for-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... the current endpoints, it's incredibly clear, right?
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
It would have been like that sick, like clinically meaningful-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... and so on and so forth.So we're getting... and I'm not talking about-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... the population.I'm talking about-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... the actual intent-to-treat, modified intent-to-treat population.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
There's no change to that.We just use that endpoint now-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... to power this study.Essentially, it was powered at 80%.We obviously learned-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... a fair bit there.We're powering this study at 90%.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
Give a little bit of a hair there as well-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... for anything we might not know, so higher them.And I'm saying now, remember, there are two studies.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
There's a parallel group study and a randomized-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... a withdrawal one.Both of them are powered at 9%.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
For what treatment effect?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.We also published something at MDS on our website-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... you might be able to see it, showing that the minimal clinical importance-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... difference, is, two points-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... for these patients on the Modified ADL, and we are aiming for larger than-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... for the criteria.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
But one could argue the actual separation on the meaningful-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... that starts at 0-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... which is quite interesting-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... because it means they really need an improvement-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... to be meaningful.We don't see that in many diseases here, but.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.Okay, very helpful.Could you maybe tell us, is there any secondaries that it's powered for and what the hierarchy of the powering is in the secondary?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah, we haven't disclosed hierarchically what we're looking-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... but, it's a sequential-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... testing.We, the TETRAS-ADL, by definition-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... is powered, if anything, like larger numbers-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... difference there, right?So one more item, the Patient Global Impression-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... is quite important, as well.And the Clinical Global Impression is quite important.Those are the four things, together with the primary, that we should be looking into.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
They're not formally powered in a sense that, that we are reserving alpha to them-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
we do expect to be successful.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.Given that your product looks highly safe, is there a third - and you talked about the 2-point difference, and the-
Marcio Souza
President and CEO, Praxis Precision Medicines
Mm-hmm.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Modified ADL is considered, you know, clinically meaningful.Is there a need to show that type of a treatment to file for approvability, given the time and need, or is any statistical separation sufficient to-
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
to file?Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
Pretty interesting question.We actually had that conversation with the agency, and-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
our interpretation of the-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
... the conversation is, they are probably more interested on a separation-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
on the parallel group.I think for the randomized authority-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
It's slightly different because we have a threshold.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
They have to match the threshold and then be.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
-withdraw.Because we suggested, and I believe they were happy with-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
The combination of two designs.When you put them together.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
It becomes very clear.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
if the patients are benefiting, so there was not as much of a-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
- obsessional call-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
for that one number, one study
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-uh, there.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
That's very helpful.Another question that we get quite a bit is, I think there's a competitor program by Jazz-
Marcio Souza
President and CEO, Praxis Precision Medicines
Mm-hmm, mm-hmm.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
that's also going to be reading out.
Marcio Souza
President and CEO, Praxis Precision Medicines
Mm-hmm
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
In 2024.Like, what is your expectation from that program?Remind us what the differentiation between the two molecules are.
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.The mechanism of action is the same, right?The number of isoforms-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
There are three isoforms of this channel that both drugs have.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
They're similar.I think there's a number of details that we wouldn't have time,
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
-right now to go through, including the, the fact that, the other drug has a very significant-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-active metabolite, for example.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
Which is not necessarily an issue, but it's a factor-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
to consider.That seems to be-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
an impression out there, that selectivity in terms of the-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
states of the channel matters, it doesn't.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
for this disease.Those are bursting fires-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
not open
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-closed states like sodium channels, for example.So that is a little bit of urban legends.We have pushed the dose all the way to double of-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
the dose what we have right now.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
There is no additional benefits.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
So what I expect to see there is an effect.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
The mechanism work.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
So we do expect to see an effect because it's diluted in multiple-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-dose, is likely smaller-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-in certain geographies that have not been, actually any trial-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
or a potential trial run before.I think there are a couple areas of variability-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
that might be expected, but I firmly expect the-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
The study actually to show quite promising results as well.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Great.Maybe now would love to transition to the epilepsy program.Before we go into specific questions, you announced multiple presentations across the epilepsy programs that are going to be presented at this- in the month of December-
Marcio Souza
President and CEO, Praxis Precision Medicines
Right
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
... at significant conferences.Is this gonna be just an opportunity to educate the physicians, or is there new data gonna be coming out at the conference?
Marcio Souza
President and CEO, Praxis Precision Medicines
I think it's all of the above.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay.
Marcio Souza
President and CEO, Praxis Precision Medicines
Right.It's a scientific conference, so-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
We're trying to focus.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
The discussion with the scientists.Some of those presentations were late breakers.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
As you know, for AAF, specifically for our conference, we have to have something new.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
One of that is our predictive model for drug activity in the brain.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-using EEG.It's the first time we showed with three molecules-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
-not only one.So we're gonna see, in a week, we can project-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
Whether or not it's gonna have an activity in the brain using something we developed in-house.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
Quite exciting.One of the authors-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-and I'm gonna be there, helping present-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
that and more data on 628 to increase the confidence.A little bit more data on 222 as well, like the
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
final four patients, and so-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Oh, so the two... all four, four patients-
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
are gonna come also.
Marcio Souza
President and CEO, Praxis Precision Medicines
Are gonna be there?
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay
Marcio Souza
President and CEO, Praxis Precision Medicines
On the, like, the wrap-up of what-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
We expected the trial to be.I think in general, what you're gonna see in a week or so is confirming to improving.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-the confidence-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
in the drugs we have in epilepsy, and we're very excited-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
to participate in the conference.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
No, that's great.So let's maybe, like, go through each one.
Marcio Souza
President and CEO, Praxis Precision Medicines
Mm-hmm.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Maybe start with 562, since that you're gonna show 4 patient data.
Marcio Souza
President and CEO, Praxis Precision Medicines
222.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
222, sorry.
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
PRAX-222.I apologize.On PRAX-222, so you're gonna show four patient data.What, have you had a chance to engage with the agency to discuss, or is it just more the whole data set that we're gonna see among-
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
-each patient, to assess the next program?
Marcio Souza
President and CEO, Praxis Precision Medicines
No, it's just a wrap-up.Like, we had three.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah, now 4.
Marcio Souza
President and CEO, Praxis Precision Medicines
Now we just-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
finalize that for 4 dose, 4 patients.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
3 dose before, 4 patients-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-4 dose now.That was what the cohort was meant to be.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
I think it's very straightforward, and as I'm sure you're gonna see next week as well.It does not change whatsoever-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-our view.We have, I'm gonna give a little, two-way answer to your question.Yes, we have engaged.No, we haven't had the meeting-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
Yet, which are sometimes different things.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
We're very pleased with the fact that.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
There is a pre-engagement and a meeting, which is scheduled right now.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
I'm not gonna guide for when.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
We did say we were trying to get this meeting as quickly as possible with the FDA.We will have that.It was as quickly as they could-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
give us a date... and we believe that they're gonna, like-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
-part with us on understanding, the benefit for these patients-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
and work together for the next steps.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
For 2-2.That is a gate for us.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
I wanna be incredibly clear, we have very high expectations-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
-from those engagements, because any investments in any-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
program has to be justified by a very clear-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
path forward.And,
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay.Maybe now on focal epilepsy, PRAX-628.
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
You have noted that you're gonna have POC data by year-end.That's gonna be year-end, that's not gonna be at the conference, right?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay.So separate data disclosure.I guess as we look at, you know, I guess, what additional data are we gonna get at year-end?
Marcio Souza
President and CEO, Praxis Precision Medicines
Mm-hmm.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
What is sort of the next steps that you need to take post that data to really come back to us and guide sort of the-
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
phase 2 design in the first half of 2024?
Marcio Souza
President and CEO, Praxis Precision Medicines
Very quickly, what we've shown with 628, right?
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
Praxis, in focal epilepsy, I was gonna say best in class-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
but really best in focal epilepsy, therapeutic index.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-on very predicted model.If you go across the industry, if you look into other drugs-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
Everyone is using the same benchmark.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
I would argue that it should go and compare-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
those multiples to the ones we are showing.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
They all tap out-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-between 2-4 folds on the MES EC50 equivalent.We're talking about 15-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-18, with 628.So we're very comfortable there.Then we show it's safe on the health volunteer study.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
It's present in the brain-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
It alters the EEG.So one could argue, just jump straight-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-into a phase 2, 3.We chose to run this-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
PPR study.The original cohorts was planned to be increments of 4.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
We over enrolled a little bit.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay.
Marcio Souza
President and CEO, Praxis Precision Medicines
On that, so we're wrapping up as quickly as possible-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
but we're really gonna be-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
Try to give as much as a full sum, or-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
I would say, excited.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
about that.That, that's gonna give us a further guidance from activities in patients, right?
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
This is no longer.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
-a biomarker in healthy volunteers.Now, we have a biomarker, translational marker-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
-in patients, so, stay tuned for that one, but not at the AS.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Okay.Maybe moving to 562.
Marcio Souza
President and CEO, Praxis Precision Medicines
Mm-hmm.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
We hear quite a bit.You shared really exciting data at the analyst event this fall.So, and I guess the question now here is, you're gonna have more data in this population, given it's a high unmet need.
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
You're also gonna engage with the agency.So could you maybe talk about the type of data that will come in the first half of 2024?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
What is considered, like, further POC and validation in this?
Marcio Souza
President and CEO, Praxis Precision Medicines
Mm-hmm.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
How could you work with the agency to maybe find a very innovative way for path for approval?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
If you could tackle all three, that would be great.
Marcio Souza
President and CEO, Praxis Precision Medicines
So 562, we're testing right now, it's a phase 2-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
in two cohorts of patients.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
That's in SCN2A gain of function, that's in SCN8A gain of function.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
They have to have a high seizure burden at baseline, and then we're dosing them for 16 weeks.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
Four of those weeks on placebo, for a group of patients, and the other one's on drugs.So the... what you expect there, and they're only standard of care-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
best standard of care right now.So what you can expect from that study is a comparison, not only with placebo.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
But what happens when they transition?
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
in a blinded fashion, right?So a crossover-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
A one-way crossover there as well.We're excited because these patients have nothing.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
And they have a very high-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-burden at baseline.It's a relatively smaller study.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
It is a phase two-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
-so we're trying to learn as much as possible here.But what should enable us, once we do meet-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-with the agency, is to get a very efficient-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
Confirmatory study that leads to approval, shortly thereafter.So that's what we're looking there.First half of the year, we should have that in our hands.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Is there an opportunity that this existing dataset could be just increased in a cohort and you could deem it as pivotal?Is that also one of the optionalities?
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah, I think it's always an option.We need to unblind-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
the study and then look into what is happening there.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
I think what the agency being very clearly indicating-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
They want more treatments of these patients.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
The vast majority of these patients, if not all of them, die before-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
-like teenage years.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
Uh, twenties.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
So the unmet need, this is not an epilepsy.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
This is a fatal disease.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
This is comparable to many other fatal diseases out there.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
So there is a very high-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
Need from all sides perspective.So we believe they're gonna work with us to accelerate it.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Another question that we get quite often now is: Given that you have a pivotal study reading out in the second half of 2024, you have pipeline updates across all epilepsy programs in the first half of the year.When you start these discussions with investors, could you maybe quantify what percentage of your time is sort of really digging into epilepsy versus—like, in, into essential tremor versus epilepsy program?Like, sort of what's, what's the hierarchy of—
Marcio Souza
President and CEO, Praxis Precision Medicines
Yeah
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Discussion points at this time?
Marcio Souza
President and CEO, Praxis Precision Medicines
So from an internal focus, it's pretty obvious, right?Like, essential tremor, it's larger-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
... more complex, more important, larger inflection.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
There's a lot of, like, disproportional-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
-amount of resource there.The other programs are not suffering from a quality perspective-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
But there's less resource allocated.The way we look financially for-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-for the resource allocation, if what we have right now, in the bank covers-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
all those readouts and obviously some
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
Because we, we need to get that on the other side.But we are very serious with the game.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
Right?So covering to success.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah.
Marcio Souza
President and CEO, Praxis Precision Medicines
And then we make a decision-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
—like we're not taking risks for
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
-beyond the-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
the proof of concepts or the phase 2 readouts for this.The priority is
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
to readouts and bring new source of cash.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
Our key initiatives right now are deals with-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
Strategics, and I think we're gonna be able to continue to supplement the cash runway based on that.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
When you speak about strategics and partnership interest, is the thought process to partner in some of the epilepsy programs, or is the part to potentially entertain the idea of, like, actually give the, you know, ET programs?So, what's the hierarchy of interest in terms of partnership on which asset?
Marcio Souza
President and CEO, Praxis Precision Medicines
The highest likelihood, I would say, on a short term.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
is, regional deals with
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
our ET, for example.They're pretty high on our radar.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
Right now in conversations.I think what you're hearing from strategics about ET is, it's such a large market.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
that it's hard to reconcile-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
the potential up front or even
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Yeah
Marcio Souza
President and CEO, Praxis Precision Medicines
full consideration with market cap right now.So, we're trying to balance that interest for a larger deal.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
But for the rest of the portfolio, it's very productive discussions to...We are not too precious about one or assets-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
or another.We believe there's a lot more from where those came from.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm.
Marcio Souza
President and CEO, Praxis Precision Medicines
We can continue to move the company, and if we can bring more dilutive-
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Mm-hmm
Marcio Souza
President and CEO, Praxis Precision Medicines
-source of capital, the better for all of us.
Yasmeen Rahimi, Ph.D.
Managing Director and Senior Research Analyst in Biotechnology, Piper Sandler
Great.Well, we've come to the end of the fireside chat.What a great discussion, and really lots of excitement in 2024.Just wanna say thank you on behalf of all of us here at Piper Sandler for great discussion and participation at our conference.Let's thank Marcio for being here.
