The following is excerpted from the question-and-answer section of the transcript.
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Question: Ash Verma - UBS Equities - Analyst
: Yeah, that's great. (Event Instructions) But kind of like a 25 minutes fireside chat, and we discuss another key programs for Praxis. So maybe it will
be beneficial if you can just kind of give a brief update on the business, like what are the key highlights of some of the pipeline programs.
And just if I can sort of pinpoint to one specific area. I mean, look, I think there's a lot of focus on using genetics and translational tools in guiding
drug discovery and development. Just like -- as you look at your approach, right, like how do you use that in your pipeline? What are some of the
facets to keep in mind across your platform from that perspective?
Question: Ash Verma - UBS Equities - Analyst
: Yes, that's great. Yes, there is a fair bit of excitement in this space definitely, especially like anything to do with using genetic advances in drug
development and discovery. So thanks for sharing that. Maybe I guess like if we go over some of the key programs that you have perhaps like
starting with relutrigine.
So here, if you can just kind of explain -- so sodium channel modulator, but like you -- I believe you sort of position that this is differentiated versus
some of the other competitors or prior attempts at this. So if you can expand on that, like what's the mechanistic differentiation for your --
Question: Ash Verma - UBS Equities - Analyst
: Yes, yes. I mean I think like the data, like you pointed out, like this proof of concept on relutrigine. It shows pretty promising efficacy, like the way
that you think about it? Is this more of a sort of like an efficacy play or is safety, a differentiation factor for you versus the -- like the standard of care
or competition?
Question: Ash Verma - UBS Equities - Analyst
: Yes, yes. And then in terms of the Cohort 2 that you have right now, right, so that's the first half '26 expected readout -- just if you can help me
understand like what are the differences that you have, if any, in this Cohort 2 versus what the data that you presented late last year?
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MARCH 17, 2025 / 1:30PM, PRAX.OQ - Praxis Precision Medicines Inc At UBS Virtual CNS Day
Question: Ash Verma - UBS Equities - Analyst
: Characteristics, those type of things.
Question: Ash Verma - UBS Equities - Analyst
: Yes. Okay. And then in terms of the patients, so this is a little bit more geographically diverse, I believe you're using like US and Europe patients.
Does that have any like bearings on trial outcome there?
Question: Ash Verma - UBS Equities - Analyst
: Got it. So I'm assuming that like if you are targeting like first half '26, you are at some point like first half of this year, get this trial enrolled completely.
Is that a fair assumption to make?
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MARCH 17, 2025 / 1:30PM, PRAX.OQ - Praxis Precision Medicines Inc At UBS Virtual CNS Day
Question: Ash Verma - UBS Equities - Analyst
: Yes. Is there a possibility that the data can come this year, by the end of this year, let's say?
Question: Ash Verma - UBS Equities - Analyst
: Okay. All right. And then, yes. I mean, look, I think investors generally tend to kind of like set bogeys on where, what the trial is going to do based
on game [right]. And you have shown pretty decent motor seizure reduction in your Cohort 1. Is that a basis that relutrigine can generate that type
of efficacy in this Cohort 2? Or any sort of like nuances to consider the outcome?
Question: Ash Verma - UBS Equities - Analyst
: Yes. Yes. Okay. All right. And then just like broadly speaking, taking a step back from SCN2A and 8A, so you have this broader DEEs that you're
studying in the EMERALD study, right?
Question: Ash Verma - UBS Equities - Analyst
: I think I heard at one of the recent calls that you say you have the regulatory inputs that you required, so you're ready to start that?
Question: Ash Verma - UBS Equities - Analyst
: Yes. Okay. So I wanted to just switch gears real quick to vormatrigine. So here, so I know you've talked about the mechanistic differentiation and
Question: Ash Verma - UBS Equities - Analyst
: How much of that efficacy benefit is sort of like replicable you think in the focal and generalized epilepsy space?
Question: Ash Verma - UBS Equities - Analyst
: Yes. And these like upcoming results like for RADIANT or POWER1, like what should be like our investors consider good data to be?
Question: Ash Verma - UBS Equities - Analyst
: All right. Okay. All right. That's great. We are almost out of time. So maybe just talking about like essential tremor. So yes, I saw the update. I think
like you mentioned that IDMC recommended to stop the study for futility. Just anything that you can just kind of characterize like what happened
there? What are the next steps? What to think about the future of the program?
Question: Ash Verma - UBS Equities - Analyst
: Yes. No, that's great. That's fair. All right. Perfect. Good. I mean thank you so much for this. Thanks for your time and looking forward to staying in
touch and learning more about your pipeline.
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