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: Ami Fadia - Needham & Company - Analyst
: Perfect. Marcio, thanks for the overview. I have a few questions that I wanted to go over. Now, you -- right after the data from Essential1 had been
announced, you included FDA's written feedback as part of a 8-K that you had published where there was some feedback from the FDA around
the inclusion of performance scale, subscales, spiral, and handwriting. Can you share with us some of the context in which the FDA provided that
feedback to you?
Question: Ami Fadia - Needham & Company - Analyst
: Can you maybe remind us what other companies in the industry are doing in terms of the type of endpoints they may be studying? One example,
obviously, is the Jazz product and they're in the customer running the clinical trial. Can you remind us what endpoint they're measuring? And how
that compares to Essential1?
Question: Ami Fadia - Needham & Company - Analyst
: That sounds good. Can you talk a little bit about the patients with intention tremor, how easy are those to diagnose and really form entry criteria
in clinical trials around? And did you have conversations with the FDA around that subpopulation in the past. Can you kind of talk a little bit about
FDA's -- or it is your view on what FDA's thought process around that might be?
Question: Ami Fadia - Needham & Company - Analyst
: Right. So as you head towards the conversation with FDA, what are some other scenarios that you anticipate following that meeting in terms of
how a trial design may end up being structured? And some of the variables really, I think, in people's minds are the performance subscales, the
spirals, the handwriting as well as the essential tremor subset. What are kind of some of the realistic possibilities in terms of what the trial may end
up looking like? Maybe we start there and then I wanted to go back to some of the data.
Question: Ami Fadia - Needham & Company - Analyst
: Yeah. Fair enough. And I guess just to remind us, some of the permutations around when you did your post hoc analysis of Essential1. One thing
that seems sort of somewhat clear and easy enough to me is to not include the essential tremor patients. And even if some of the performance
subscale items are up for debate and for whatever reason you end up including them in a Phase 3, just going back to Essential1, just help us
understand what the data looked like if you took out the patients with essential -- intention tremor and then -- but included all the endpoints?
Question: Ami Fadia - Needham & Company - Analyst
: Yeah. Okay. That makes sense. So once you have the meeting with the FDA, maybe just what should we expect in terms of the cadence of
communication with the Street, how would you share the update with the Street?
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APRIL 17, 2023 / 4:45PM, PRAX.OQ - Praxis Precision Medicines Inc at Needham Healthcare Conference
(Virtual)
Question: Ami Fadia - Needham & Company - Analyst
: Okay. Let me ask one other question. We started a minute or so late, so I'm going to maybe take up a minute or two extra in the end. Just if you
talk about PRAX-628. This is your PAN-NaV blocker which is being developed for focal epilepsy and Phase 1 data is expected very soon. This is sort
of different from your other molecules, PRAX-562. Can you help us understand how it's different from a therapeutic window perspective? And as
we look to the Phase 1 data, what data are we looking for and what are you hoping to see which will determine your path forward on that asset?
Question: Ami Fadia - Needham & Company - Analyst
: Okay. That's very helpful. I should probably let everyone go for the 1:30 session, but I appreciate you staying on a little bit longer, Marcio. And
thanks to everyone for listening.
Question: Ami Fadia - Needham & Company - Analyst
: Have a good day.
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