Praxis Precision Medicines Inc To Host Corporate Update Call Transcript

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

Question: Yasmeen Rahimi - Piper Sandler - Analyst : Good morning, team and congrats on the data. The first question that clients are going to be wondering as how does this data compare to maybe other current approaches that are in development? Obviously, it's difficult to make apples-to-apples comparison. But if you could just put it into context of what this data means from a competitive perspective, that would be great. That's question one. And then question two, if you could maybe allude a little bit in regards to your plans into a broader population. What maybe the steps are necessary to have to engage with the agency? What would that look like? And how soon could you come back and communicate that with us? That would be really helpful if we could start on those two that would be great.

Question: Yasmeen Rahimi - Piper Sandler - Analyst : And then, Tim, I apologize for sneaking in one question -- one last question. Given that you had eight patients in the 28-day period as well. Long term, you had two additional. Could you talk about the homogeneity and the response among the small cohort of patients. And I'll jump back in the queue.

Question: Yasmeen Rahimi - Piper Sandler - Analyst : Great. Congrats again.

Question: Joon Lee - Truist Securities - Analyst : Congrats on the strong data. And thanks for taking our questions. Dr. Petrou's explanation on the functional selectivity of relutrigine was really helpful and can understand how it could be differentiated from other sodium channel inhibitors. So the question is how prevalent is the off-label use of sodium channel inhibitors for DEE, number one? What would be the washout period for the trials that you have in mind? And does this mean that the trial could in theory look like a randomized withdrawal? And what I mean is that if you are -- if the patient is on stable sodium channel inhibitor and then you wash them out, is that like in essence a randomized trial, withdrawal trial? Thank you.

Question: Joon Lee - Truist Securities - Analyst : Fantastic. Thank you so much. If I could ask one quick question, follow up. I was a little shocked that you actually don't wash them off of existing sodium channel. So you're actually thinking that you will let -- you keep the patient on whatever off-label sodium channels they're on and add-on with relutrigine, a better sodium channel. Is that the idea? And so in the trial that you just reported, how many patients were on baseline sodium channel inhibitor off-label?

Question: Joon Lee - Truist Securities - Analyst : Thank you so much. I'll hop back on the queue.

Question: Tina Chen - TD Cowen - Analyst : Thanks for taking my question. First on tolerability. How much dizziness or sedation was seen? And next on efficacy. What was the onset of effect of relutrigine and how fast the patients achieved that seizure freedom?

Question: Tina Chen - TD Cowen - Analyst : And lastly, what was the severity of somnolence or sedation?

Question: Tina Chen - TD Cowen - Analyst : Got it. Thank you.

Question: Yatin Suneja - Guggenheim - Analyst : Let me add my congratulations as well. Very nice results. Just a couple from me. Mostly clarification on how should we think about the placebo response in this patient population? Our understanding is that it tends to be a little bit higher than 2% that you have observed in at least the single, the 28-day period. So that's one. And then help us understand this lot transformational -- transformation estimate that you're using to drive placebo-adjusted efficacy at week 16.

Question: Yatin Suneja - Guggenheim - Analyst : Got it. Very helpful. Very helpful articulation. One more question if I may. And this is a specific question that I just came from an investor. Could you just talk about your approach to DEE? Because our understanding is that, you know, sodium channel blockers are contraindicated for some populations like Dravet. So how would you be able to go after broader DEE? Would you just not go after certain indications where the sodium channels are contraindicated. Just curious to understand the dynamic how it plays out from the regulatory and development standpoint. 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. SEPTEMBER 03, 2024 / 12:00PM, PRAX.OQ - Praxis Precision Medicines Inc To Host Corporate Update Call

Question: Yatin Suneja - Guggenheim - Analyst : Very good. Thank you so much.

Question: Douglas Tsao - HC Wainwright & Co - Analyst : Hi, good morning and congrats on the results. Maybe Marcio, I think it would be helpful, just be curious to get your thoughts, how you're thinking about that 562 in the context of SCN8A versus Elsunersen where we also got some very impressive results. I mean, how do you see the two therapies sort of sitting next to each other in the market? Who would -- do you think one would be sort of the initial treatment versus 562? Thanks.

Question: Douglas Tsao - HC Wainwright & Co - Analyst : And if I can on a follow up. So obviously, I think everybody's initially going to focus on the seizure reduction numbers, which are really impressive. But when we think about the potential of the drug. I think what seems very compelling is sort of the other benefits that we saw in terms of patient, verbalization and awareness et cetera. Just maybe if you could provide some context how that compares to other treatments on the market and the standard of care in terms of the rate of improvement that you saw with relutrigine it involve? Thank you.

Question: Douglas Tsao - HC Wainwright & Co - Analyst : Great. Thank you so much.

Question: Francois Brisebois - Oppenheimer - Analyst : Thank you for the question and congrats on the data. Just wondering in terms of the specificity here and the strategy behind the prespecified, the prescreening patients to 74, then 21 and the eligibility of 16. Can you help us understand what goes in? Could that -- just making sure that you have the right patients and why -- you know it from going to the pre-screen number to ultimately the 16, why -- what is so restrictive and how does that translate into the market down the road? And then I'll have a follow-up. Thank you.

Question: Francois Brisebois - Oppenheimer - Analyst : That's very helpful. Thank you. And then in terms of the read-throughs, are these patients -- in terms of DEEs, are these may be the hardest patients, the worst? Or are they all -- they have their difficulties regardless of the population? Or is it like, look, we got an SC and 2A in a day. So the odds of this working in other DEEs is actually a lot stronger because this was a very high bar. And then maybe if you can touch on the read through to 628 as well? Thank you.

Question: Francois Brisebois - Oppenheimer - Analyst : Thank you and congrats again.

Question: Ami Fadia - Needham - Analyst : Hi, good morning and congrats on the strong data. A couple of questions. Just with regard to how we should think about some of the sort of design elements as if you think about the registration study. How could sort of the efficacy results differ if you initiated all patients on 1 milligram per kg. And I'm looking at slide 17 and 18, where we sort of see. On slide 18 response rate from patients with -- who dosed up during the double-blind period. So should we anticipate an improvement in response rates if all patients started at a higher dose? And how should we think about impact? Should patients not have other sodium channel blocker on board at baseline? If you could sort of help us think through that and then I have another question.

Question: Ami Fadia - Needham - Analyst : Great. And just one more question, if I may. As you think about a broader clinical development in other DEEs, you talk a little bit about Dravet syndrome. But outside of Dravet syndrome, should we assume that you would think about a trial in all DEEs other than DS? Or would you sort of -- is the work that you're going to do to sort of identify which specific DEEs subtypes would be more suitable?

Question: Ami Fadia - Needham - Analyst : Got it. Okay. Thank you so much.

Question: Joel Beatty - Baird - Analyst : Congrats on the data. And first question is how much of reducing or removing of other medications did you see over the course of the study or in the long-term extension? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 03, 2024 / 12:00PM, PRAX.OQ - Praxis Precision Medicines Inc To Host Corporate Update Call

Question: Joel Beatty - Baird - Analyst : And then could you provide any more details on the one patient in the placebo group who is now eligible for the efficacy assessments and why that was?

Question: Joel Beatty - Baird - Analyst : And maybe one more follow-up. Can you provide any context on what type of enrollment speed to expect and a study that's just starting now compared to the study that is reading out?

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

Question: Kambiz Yazdi - Jeffries - Analyst : Congratulations. And then maybe just following up on kind of the breadth of the program. Would you consider development in non-sodium channel DEEs. You saw some kind of anticonvulsant activity and some PTZ-induced preclinical seizure models. So I just wanted to get your perspective there.