Praxis Precision Medicines Inc Q3 2024 Earnings Call Transcript

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

Question: Ritu Baral - TD Cowen - Analyst : Good morning guys. Thanks for taking the question. A couple questions on retro gene 550 for tee specifically, as you think about the 80 patients in the expanded cohort for 80 patient, expanded cohort sufficient for registration. Will the enrollment criteria for that 80 patients be any different than the original 15 patients? And it, so do you expect it to result in any or prospectively expected to result in any changes to Fathy or safety? And then the second part of that question is you mentioned that you are seeking alignment with regulators in the first half. Can you talk to maybe any more specifics around that timing? How you might expect cohort to change based on feedback and any specifics on what you're asking on? Emerald? Thanks.

Question: Ritu Baral - TD Cowen - Analyst : Got it. So I want to clarify, you are going to genotype these patients, but all they need to have is a genetic mechanism that's rational for good.

Question: Ritu Baral - TD Cowen - Analyst : Okay. Got it. Thank.

Question: Yasmeen Rahimi - Piper Sandler Companies - Analyst : Good morning team. Thank you so much for all the thoughtful comments. A few questions on the interim analysis. I think investors were just wondering I think the interim was expected to happen end of year, maybe just some color around why it got a little bit moved into one Q2025. And then it appears based on the remarks you've made that we're study two will not read out before the interim or at the interim. We'll get an insight about one and two. I just want to make sure just to go ahead and if you could just walk us through sort of the disclosures around both of the studies that, that could be helpful. And then last question is I think you guys noted that upon the outcome of the data in A T, you would reinitiate a Parkinson's disease program in 2025. Could you maybe comment on like the success in A T would move you into Parkinson's or what would that be a phase two study? Phase three study? Any color around that would be helpful and I'll jump back into the queue.

Question: Yasmeen Rahimi - Piper Sandler Companies - Analyst : Great. Thank you. And I'll jump back in the queue.

Question: Joon Lee - Truist Securities - Analyst : Hey, thanks for the updates guys. Just a quick clarification. Will you be including Lentis Gesto in the broader DEE study? Because there's, you know, genetic basis for L gaso and you seem to want to stick to epilepsy based on your response to the question. And I have a follow up.

Question: Joon Lee - Truist Securities - Analyst : Great. So it's a true the E study? Great. You know, for the interim analysis for essential program just wanted to clarify that you when you say refer to interim analysis, you're referring to the study one interim. So your withdrawal trial topline will depend on the interim from the parallel comparator trial. Is that correct? And then is, yeah. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 06, 2024 / 1:00PM, PRAX.OQ - Q3 2024 Praxis Precision Medicines Inc Earnings Call Yeah. So, and, then number part part two is that or is there no inferiority analysis baked into that interim analysis?

Question: Joon Lee - Truist Securities - Analyst : Right? And then last question, you know, as we look to the, you know, very likely approval of sugen from vtex in January, it's actually impressive that it even works at all because it only targets one of the three voltage gated sodium channels in the peripheral nervous system. So it's actually interesting that you're advanced looking at your, your formal in in pain as well, which targets two out of the three pain receptors or not receptors, but voltage gated channels, any anecdotal evidence of pain reduction from your phase one or any other. Thank you.

Question: Joon Lee - Truist Securities - Analyst : Thanks for all your answers. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 06, 2024 / 1:00PM, PRAX.OQ - Q3 2024 Praxis Precision Medicines Inc Earnings Call

Question: Francois Brisebois - Oppenheimer - Analyst : Hi, thanks for the questions and thanks for kind of going through the, the potential scenarios here and the complexity and the dependence between the or the the impact of study one on study two. So, but in terms of, you know, what to share on the interim, is it, you know, could it go into data or that's actually a really good case scenario where we stop the study because things are working out or is it more, you know, it, can we be assured that the actual top line of study one will be after study two that might come short after the interim. Look just any any help there understanding the timeline of the top line versus the interim for study one.

Question: Francois Brisebois - Oppenheimer - Analyst : Okay. Thank you. And then on the de commercial front, there's a lot of different ways to look at this market. Can you help us understand, you know US, X US, how you think about the commercial potential here?

Question: Francois Brisebois - Oppenheimer - Analyst : Thank you.

Question: Yatin Suneja - Guggenheim - Analyst : Hey, guys, thank you for taking my question. Just a couple for me as well. Mostly on the ET side, could you provide us where you are, at least on the enrollment from like how many patients have been enrolled in study one and two if you could and then, you know, we understand this. In turn, could you also talk about and maybe put some numbers around the possible sample size adjustment ranges based on the pre specified plan? Like how long would that take at the maximum if, you decide to increase the size and, and let's say you go with the maximum of patients that are allowed, how long will that will that take, will that take?

Question: Yatin Suneja - Guggenheim - Analyst : Thank you.

Question: Douglas Tsao - H.C. Wainwright & Co., LLC - Analyst : Hi, good morning. Thanks for taking the questions. Just to confirm what you just said. So I think you indicated 30 patients. Is that enrolled and randomized or is that simply enrolled into the study in the scenario that you expand the patient population for essential?

Question: Douglas Tsao - H.C. Wainwright & Co., LLC - Analyst : Okay, great. That's helpful. And then just if you could provide a little more color in terms of moving from trigen into these pain indications and what type of work you're going to be doing or need to do before coming out with a more sort of expanse or sort of coming forward with the full development plan.

Question: Douglas Tsao - H.C. Wainwright & Co., LLC - Analyst : Okay, great. And then just one more on (Elsa Urson), I think with last quarter, you indicated that the first patient was being enrolled in sort of the Global Registration study. But I think today you indicated that they're sort of being added to Embra. And I just want to understand if there's been any change as you think forward about the global registration program for that drug.

Question: Douglas Tsao - H.C. Wainwright & Co., LLC - Analyst : Okay. And so with the patients being studied in Brazil, now, are you, you're looking at some additional doses? And if the result, I mean, is there a scenario, I know you've been very confident in the 1 mg per K dose? I mean, is there a chance that if if results warrant that you would potentially look at higher doses in the US and European registrational studies?

Question: Douglas Tsao - H.C. Wainwright & Co., LLC - Analyst : Okay, great. Thank you very much.

Question: Kambiz Yazdi - Jefferies - Analyst : Morning team a couple of questions for me. The placebo response was pretty well controlled in essential one. Essential three has a more innovative decentralized design. What steps have you taken to control placebo response and study one in essential three? And then as a second and a set of questions maybe on verme gene. What is sodium channel blockers demonstrated historically in PGTC seizures? And what would a registrational program consist of in a generalized epilepsy? Thank you.

Question: Kambiz Yazdi - Jefferies - Analyst : Great. Thank you.

Question: Ami Fadia - Needham - Analyst : Hi, good morning. Thanks for all the updates. A couple of quick questions for me. Firstly, on Alexa, can you comment on the enrollment and and how what percent of patients have completed randomization relative to the target enrollment for the two studies? And was there a slowdown in the enrollment rate that caused the shift in the timeline? Maybe I'll ask my next question after you.

Question: Ami Fadia - Needham - Analyst : Got it. Okay. And then on Vormatrigine in the RADIANT study, can you talk about how many patients you're targeting or, or sort of the mix of patients between focal and generalized and, and how that might inform your plans to develop it further in generalized epilepsy?

Question: Ami Fadia - Needham - Analyst : Understood. Maybe just a last question for me as you design the MD E study, what assumptions would you be making with regards to regulatory performance in this patient population relative to the data that you saw, you know, in sen two A&A?

Question: Ami Fadia - Needham - Analyst : Thank you.

Question: Joel Beatty - Baird - Analyst : Hey, thanks for the updates. The first one is on (eex callide) For how if the interim is successful, could we get final results at the same time that we learned the interim is successful? Or will there inherently be some amount of time between those two events?

Question: Joel Beatty - Baird - Analyst : Thanks and then in, DEEs, should we think about routine as kind of just working in, in patients who are already responsive to calcium channel blockers? Or could you talk about the potential to work beyond the patients who are responsive to those agents?

Question: Joel Beatty - Baird - Analyst : Thank you.

Question: Laura Chico - Web Bush Securities - Analyst : Hey, good morning very much. Thanks for taking the question. Two clarification questions for me first on the interim for let's call them. I I understand that the timing for the interim has extended to the first quarter 25. What I'm what I'm trying to clarify though is does it, does the actual at which the interim is executed, is that also changing? And I guess maybe asking it differently is if this was expected to be conducted at 50% for example, does this now shift to 60%? Ho hopefully that makes sense. And then I have a follow up.

Question: Laura Chico - Web Bush Securities - Analyst : Okay. That's helpful. And then I guess I understand your comments also about with respect to study, conduct and integrity and the ramifications to study too. My understanding was that these are conducted under the same protocol. So I'm trying to understand the the separation of the release of results. And have you had any feedback from FDA on how they would like to see result communication? Thanks.

Question: Laura Chico - Web Bush Securities - Analyst : Okay. That's helpful Marcio. And maybe the last question for me, I, I think I missed this and I know you commented that earlier, but could you expand a little bit more on the rationale that we start in the Parkinson's indication? I guess I'm, I'm trying to better understand what would be the mechanistic read through from the ET studies to Parkinson's. Thanks very much.

Question: Laura Chico - Web Bush Securities - Analyst : Thanks very much.