The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Ritu Baral - Cowen Inc - Analyst
: But I guess, let's start with your some epilepsy programs, relutrigine. Can you review how your sodium channel modulators are differentiated from
those used in the standard of care and the competitors in development?
Question: Ritu Baral - Cowen Inc - Analyst
: Well, that's what they do with status, right? They give you propofol and put you into a coma and seizures.
Question: Ritu Baral - Cowen Inc - Analyst
: Toxicity means sedation?
Question: Ritu Baral - Cowen Inc - Analyst
: Something that actually a couple of the experts brought up in response to my question yesterday at the epilepsy panel, one of them said that it's
important -- there's a dose response curve. And you need to get up that curve, but you can't get up that curve with standard of care because dose
limiting side effects. So the question is, is there another option to help you get further up that curve?
And then another -- the other KOL actually weighed in and said different drugs bind to the sodium channel different ways, and therefore, can affect
different types of inhibition with different characteristics. Do you -- can you talk about that aspect of it, the binding?
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
MARCH 05, 2025 / 2:10PM, PRAX.OQ - Praxis Precision Medicines Inc at TD Cowen Healthcare Conference
Question: Ritu Baral - Cowen Inc - Analyst
: Great. So your ongoing Phase 2 study in SCN2A and 8A, how is enrollment going in the registrational cohort 2 of that study and when should we
expect top line data?
Question: Ritu Baral - Cowen Inc - Analyst
: So things like site activation, IRBs, all that's going well?
Question: Ritu Baral - Cowen Inc - Analyst
: So how should we be thinking of what potential top line data will look like? I mean, you saw placebo adjusted 46% in motor seizures in cohort 1.
Is that setting the bogey for cohort 2?
Question: Ritu Baral - Cowen Inc - Analyst
: So minimum is -- do you believe that that minimum is still like the 30%?
Question: Ritu Baral - Cowen Inc - Analyst
: So 30% ranging up to sort of that 45%?
Question: Ritu Baral - Cowen Inc - Analyst
: Yes, 30% to 50%. You're also planning to start a registration of all DEE study by mid-2025. How are those FDA discussions going? And what are the
key considerations as you talk to your advisors for the agency?
Question: Ritu Baral - Cowen Inc - Analyst
: Because if it was a non-functional channel, you couldn't modulate it?
Question: Ritu Baral - Cowen Inc - Analyst
: A part of that. Can you review the market opportunity for all DEEs versus just the 2A, 8A?
Question: Ritu Baral - Cowen Inc - Analyst
: All DEEs together?
Question: Ritu Baral - Cowen Inc - Analyst
: And within that, the biggest chunks are Dravet, LGS?
Question: Ritu Baral - Cowen Inc - Analyst
: When will we have that final -- the final indication on that strategy and that trial?
Question: Ritu Baral - Cowen Inc - Analyst
: No later than mid-year?
Question: Ritu Baral - Cowen Inc - Analyst
: So you'll let us know upon study start or near study start. Okay. Great. I want to move on to vormatrigine 628. This is your sodium channel blocker,
not modulator. And this is in the Phase 2/3 energy study for focal onset seizures. And you're also looking at generalized epilepsies. Can you review
for us the three different studies and what they're designed to show? So you have RADIANT, you have POWER1, and you have POWER2.
Question: Ritu Baral - Cowen Inc - Analyst
: So you mentioned the bar in focal, but in RADIANT, you're also doing generalize. How do you think of the efficacy bar in those two populations?
Is it a different bar?
Question: Ritu Baral - Cowen Inc - Analyst
: So you could have a lower percentage reduction with a bigger clinical impact?
Question: Ritu Baral - Cowen Inc - Analyst
: Much more massive? Got it.
Question: Ritu Baral - Cowen Inc - Analyst
: No, you didn't.
Question: Ritu Baral - Cowen Inc - Analyst
: No longer.
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
MARCH 05, 2025 / 2:10PM, PRAX.OQ - Praxis Precision Medicines Inc at TD Cowen Healthcare Conference
Question: Ritu Baral - Cowen Inc - Analyst
: Got it. I do want to ground back to your essential trauma program. Last week you disclosed, they pre-plant interim, hit futility on study 1. But you
noted some underlying assumptions on the statistical, what they noted, underlying assumptions of the statistical model may have influenced the
outcome and suggested exploration of other statistical models. Can you walk us through a little bit of what model was utilized and what other
options that the DSM may have been suggesting? I believe you mentioned they didn't suggest anything in particular, but what are the options
you're considering?
Question: Ritu Baral - Cowen Inc - Analyst
: Are you going to change the SAP of the withdrawal study, study 2?
Question: Ritu Baral - Cowen Inc - Analyst
: Great. Well, we still have one epilepsy program in a minute left. This is 222. This is your ASO. And this is for 2A, SCN2.
So you recently disclosed you've got global regulatory alignment. Cohort 1 is going to look at 1 milligram against sham. What is the sham procedure
that's going to be conducted? And how is the -- I guess why was this selected?
Question: Ritu Baral - Cowen Inc - Analyst
: It's infection risk.
Question: Ritu Baral - Cowen Inc - Analyst
: When are data catalysts from that?
|