Praxis Precision Medicines Inc Q2 2024 Earnings Call Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Yasmeen Rahimi - Piper Sandler - Analyst : Hi. Good morning, team, and congrats on kicking off the ENERGY program. Team, few questions, first one, and you alluded to that, I guess could you comment whether enrollment, I guess it seems like it's still sort of finishing up. If you could quantify sort of where you are in the cadence of getting Essential done? Second is, you spoke about an interim analysis. Could you maybe talk about what this interim analysis is, you're referring to? Because this may be a new word that we just picked up on, would love your thoughts on what that analysis is and when it will be unveiled. And then the third question is, could you maybe talk to us about, given that RADIANT will read out in the first half ahead of the POWER studies, how predictive is going to be the RADIANT study in terms of read-through to POWER1 and POWER2, given that it has maybe a more broader population and how you're thinking about enriching that group?

Question: Joon Lee - Truist Securities - Analyst : Oh, yes, congrats on the progress, and thanks for taking our questions. I think interim is part of good housekeeping, but if you do decide to resize the trial for any reason, that could potentially push out the timeline for Study 1. With the randomized withdrawal trial, which I believe is Study 2, still read out by year-end, I don't think that there is an interim for Study 2, I believe. And I have a follow-up question. Thank you.

Question: Joon Lee - Truist Securities - Analyst : Great, that's a fascinating setup there. The follow-up question is, as we look forward to the relutrigine data in DEE, help us set some bar for what you think is a good data from SCN2A and SCN8A and are you say that the opportunity in 2A and 8A could be just the tip of the iceberg for future application. Can you elaborate on that a little bit? For example, what other types of DEE did you have in mind? Thank you. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. AUGUST 13, 2024 / 12:00PM, PRAX.OQ - Q2 2024 Praxis Precision Medicines Inc Earnings Call

Question: Francois Brisebois - Oppenheimer - Analyst : Hi. Thanks Franc for the questions. So just, first of all, you mentioned the 20% to 30% seizure reduction would be great, especially with this size of a trial, but should we be expecting a P-value here? I just want to make it clear for expectations into the data.

Question: Francois Brisebois - Oppenheimer - Analyst : Okay, thanks. And then just in terms of the study design, just differences with others in terms of Essential Tremor, can you just help us understand, maybe the differences in the thought process between doing parallel and randomized withdrawal? And just overall, just maybe going through the differences that make you feel comfortable with the readout or more comfortable than what others ran into here? Thank you.

Question: Yatin Suneja - Guggenheim - Analyst : Hey guys, thank you for taking my question, just a couple of clarifications for me. So with regard to the interim analysis in Study 1, could you tell us what would be the sample size that will trigger it? So that's one. What exactly are you looking for? Is there a particular mADL11 delta you're shooting for there? And then is there an alpha loss there since you do an interim analysis and what sort of an alpha loss? And then I have a follow-up on the other program. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. AUGUST 13, 2024 / 12:00PM, PRAX.OQ - Q2 2024 Praxis Precision Medicines Inc Earnings Call

Question: Yatin Suneja - Guggenheim - Analyst : Okay. For the final analysis, or let's say for the study readout, is there -- what is clinically meaningful in terms of mADL, both on the absolute side and also on the placebo-adjusted delta?

Question: Yatin Suneja - Guggenheim - Analyst : All right. Maybe one more multiple part, like a classic sell-side question. So, for the interim analysis, this is an efficacy analysis, not utility analysis? Two, how you will disclose or what exactly are you willing to disclose to us when this interim happens and when exactly is this happening? Is it a Q3 event or a Q4 event? Thank you so much.

Question: Ritu Baral - TD Cowan - Analyst : Good morning, guys. Thanks for taking the question. I have got one last question on ulixa and then a bunch on 628. So, for the Essential, the withdrawal study, I know that sometimes FDA imposes an official or unofficial requirement on the initial response rate for those patients going in that are defined as responders before the actual withdrawal portion is conducted and analyzed. Have you spoken to FDA around the design of the withdrawal study specifically on that initial open label responder rate, sort of what they're looking for, even if -- and if there is even a hard requirement or just a soft, would like to have?

Question: Ritu Baral - TD Cowan - Analyst : But the original sort of open-label response rate going in, there was no criteria for what was required around that?

Question: Ritu Baral - TD Cowan - Analyst : They're all blinded already. Okay.

Question: Ritu Baral - TD Cowan - Analyst : Okay, understood. Moving to 628 and the RADIANT studies, first of all, can we confirm that the RADIANT and POWER1 and POWER2, those are placebo-controlled studies?

Question: Ritu Baral - TD Cowan - Analyst : Got it. And then what doses are you using? Just given the very good safety that you've seen, what doses are you using in RADIANT and POWER2 for 628?

Question: Ritu Baral - TD Cowan - Analyst : Is there a minimum percentage of generalized epilepsy or maximum percentage of generalized epilepsy that you have in mind for RADIANT? We're asking just because when we did our doc checks, some of our KOLs were particularly excited about the potential for 628 in this population.

Question: Ritu Baral - TD Cowan - Analyst : Got it. Got it. And then, final question, thanks for your patience. Other than the dosing, do you see, in the design, are there any major differences between POWER1 and POWER2 of note?

Question: Ritu Baral - TD Cowan - Analyst : Understood. Thanks for taking all the questions.

Question: Joel Beatty - Baird - Analyst : Hi, thanks for taking the questions. A couple on Essential3. The first is, what's the next update for us to expect from the Essential3 program? Would it be an announcement on the completion of the planned enrollment, or would the next update be the implications from the interim analysis? Then, as a second question, has the data from Essential3 been able to be looked at on a blinded basis, perhaps to assess things like whether the variability of the data is in line with expectations?

Question: Ami Fadia - Needham - Analyst : Hi, good morning. Thanks for taking my questions. Firstly, just a follow-up on 628. What is the gating factor for initiating a physical program for generalized epilepsy? And is RADIANT meant to inform the dose that you would study in that epilepsy type? And then I have one or two other quick follow-ups.

Question: Ami Fadia - Needham - Analyst : Got it, that's quite helpful. With regards to PRAX-562, can you talk about how age and baseline severity impact how much seizure reduction we could expect to see? Also, how should we think about read-through into other DEEs based on the data that you will share with us on the two DEE subtypes? And then maybe I'll just plug it in here, a quick follow-up on ulixa. Is the interim analysis likely to drive a statistical penalty or not? Thank you.

Question: Douglas Tsao - HC Wainwright & Co - Analyst : Hi, good morning. Thanks for taking the questions. Just maybe starting with RADIANT, I think given the PPR results, there was a lot of interest in terms of 628's effect in generalized epilepsy. I'm just curious, why not just do that study with generalized epilepsy patients? And then also, I'm just curious in terms of the dosing between RADIANT and the POWER studies. With POWER, you're starting at 20 milligrams for six weeks and then moving to 30 milligrams. Why in RADIANT are you going directly to the 30 milligram without sort of a titration period? Thank you.

Question: Kambiz Yazdi - Jeffries - Analyst : Good morning, team. For PRAX-628, in terms of focal patient enrollment, how will you prioritize it between POWER1 and RADIANT? Then maybe you can go into more details of them, POWER observational study. And then for another program, Elsunersen, what feedback did you receive from global regulators to initiate a pivotal study in Brazil and what remains required to advance the program in the US and Europe? Thank you.