PRAX - Earnings call transcript Q3 2024 | Praxis Precision Medicines, Inc.

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Operator

Good day, and thank you for standing by. Welcome to the Praxis Precision Medicine's Third Quarter 2024 Corporate Update. Please be advised that today's conference is being recorded. [Operator Instructions]. I would now like to hand the conference over to your speaker today, Dan Ferry from LifeScience.

Daniel Ferry

to and Precision Business welcome XXXX Third and Results Financial Update morning, the Medicine's Praxis Quarter Good Conference Call. being section website is on call webcast can and This be the Investors www.praxismedecines.com. of at Praxis the live accessed

plans, Please financial statements contain statements note future about Private Litigation the within may XXXX. projections. of and the forward-looking Reform of company's that Act during clinical meaning These call lines, made Securities remarks development this time the may expectations include

taking practices views be is associated represent most discussion statements of today, representing business. for While not company's not refer statements these a to future, with the with Please these certain Exchange so. as to recent and risks they should may the Commission an relied of the but forward-looking Practice's in the update as company's uncertainties on upon future. the views obligation do in filings Securities Praxis and

are Practice; session. key the Chief will to today it's on over and our a Marcio programs, President Kelly, call to pleasure my After that, updates Marcio. call Officer. question-and-answer brief Tim the Financial be and Joining providing De'Souza, of With there Officer turn Executive Chief

Marcio Souza

morning, III well. Conference The ulixacaltamide, our we and in opportunity. for our tolling the Thank essential you. to X program, gear year past have multibillion-dollar as Quarter remain lead to in continues substantial to Phase XXXX programs up registration to Good we -- Third Call. laser Praxis progress pipeline focused welcome a advancing quarter, This study on EssentialX for next

months, to for success are and interim board the have are and both because we engine interim XXXX. that timing well for for from a coming the plans outcomes powered well analysis. Study each all decided as the the range there's Both We aspects will QX X an have share on as review Study recommendation the once have we confirmed the updating we studies evaluated for update analysis only study of analyses and in results controlled of the interim in X now the

report SCNXA-DEE. results versus in In seizures to an we patients X/X results, Based and and after seizure-free prior the in initiated those excited study, cohorts XX% screening trial with weeks just motor the relutrigine a the of started SCNXA in registrational in second an patient placebo, study, of our QX, EMBOLD EMBOLD achieving XX Phase impressive we're the completion on the has positive other very In for II top pipeline, assets demonstrated of unprecedented already patients the Vormatrigine, reduction cohorts. which status. line

launched strong as our this study X,XXX common The innovative registered patients In starting in all the first of consortium comprehensive Praxis, in of observational period previously interest a time, of its the attracted epilepsy relutrigine short the out energy were gate third clinical in in study. is Empower, known with In program. PRAX-XXX quarter. kind study of collaboration the of over this areas

POWER energy the learnings X next II the line results trials The Phase and key expect We II/III Empower on the RADIANT top impact Phase entire for for are track to program. year.

agencies portfolio, ] we the our EMBRACE continue patients sheet, our for study, therapies function in to [ deliver and out we funded in plans SCNXA vision finalize patients. CNS for our disorders. quarter in as continue fully to development pursue Rounding the be engage to begin With the strong to balance in the U.S. with precision dosing patients second elsunersen regulatory we Europe Brazil in and

me focus some Let more ulixa. on now

interested Phase recruiting III in of seen conducted and is of about for today. thousands have X Our We tens innovative and Essential most IT the ago X program X program biggest just patients the studies began year participating. in comprehensive

the for patients millions over survey They program. said without This not their advance vibrant needs perform the current therapy patients patients, up are The are to symptoms a participation their looking will respondents and we defined visits treatments. with XX% impairments. a XXX who daily of significant conducted essential In conducted on be to to of more that of tremor with for we managed separate seeking that we tremor focused this for physicians as needs allow are in patients with treatment with Essential highlights a in continues feel activities caregivers do XXX the XX% with In treatments. their unmet ETF they physicians. survey, shared our

ESSENTIAL look and forward is we the an with the to need market. there the bringing an completing to study option shortly incredible X Clearly, here, of goal

on III a program is Phase studies used X XX primary clinical studies with activity parallel being weeks group X-arm they Both and than has is randomized ESSENTIAL both study. the Study at assessments, As quick the of run rather a entirely as X home patient's [ in as at ] decentralized concurrently. refresher, XX and study dry simultaneous the are change XX-week day two placebo-controlled living, a the science. in modified

first and well expectation us who consider in the the operations in operational the throughout of quarterly number the in to completion the analysis study it's we consider point of end if completion complete sufficiently internal study to when The Based impacts should a the the be we study, of the for considering to XX% completed seizing as the our the success. of interim study whether our board that analysis steady last interim quarter or to on XX% shared met X. call statistical cleaning the the enrolling testing we'll this by XXXX. sufficient analysis ensure additional as patients XX-week primary preplanned We trigger continue decided patients and finalizing execution the of independent patients will is analysis have data to powered inform for of the

review time we we'll of readout Study in X boards or until the file range XXXX. for Given informed not speculate to and interim the which of expected better Regardless, preparations update. Study timing X we do at the NDA be to provide as from an outcomes, scenarios continue on hear

[ highly differentiated moving a epilepsy a common as a We are small on eplaxis both shift way that as this next-generation tolerability. for oral with developed lacking with in treatment treat to the options is functionally Now differentiated ] to know believe Vormatrigine Vormatrigine, previously treatment merging selective efficacy plenty. disease. provide and PRAX-XXX known once-daily for molecule portfolio. in being paradigm of profile our adults will we And from highly

next and Vormatrigine ambitious quarter, is advancing Last we our strong trial and for differentiated XX we Vormatrigine. to focal of a in base the points [indiscernible]. build Three I'm glad to months of energy achieve trials are our Energy goal data to efficacy safety. profile introduced generating of multiple are the clinical X program aim that evaluate studies, support for the to comprised well. of And over patients energy multi-study to broad share aiming the while

study of The first of with for generalized is or our RADIANT. enroll X focal that open-label a messaging receive epilepsy will this weeks. X An who either safe follow-up with patients weeks

our XXXX, better line on understand to first the levels messaging pharmacology We population. of results in of patient half are the on the should us its in which in top help deliver effectiveness track

the XX-week studies results end we and POWERX II/III anticipate and of underway towards XXXX. patients are in POWERX is onset focal top with studies seizures. line X Phase The POWER

begin X of of expected first studies approximately the stagger XXXX. We combined globally. XXX will initiation to slightly POWER The in the are to recruiting enroll patients half

that an activity it could we in in clear important play an pain As X.X and pharmacology where fast-acting role profile with important role, Vormatrigine areas that management. is and other consider its can NaVX.X, coupled play sales

about We and are more we'll of potential concluding sharing the near in pain, our the role our in assessment Vormatrigine future. be

state our being and in group this, delays with and epilepsies for there approved relutrigine, functional a one of [indiscernible] And is -- a characterized are most that by refractory modulator of is formulated the pediatric SCNXA treatment. we to currently SCNXA with no turning of forms this. developmental severe severe Now use

and pediatric reminder, these designation a two rare orphan relutrigine As indications. has for

EMBOLD unparalleled SCNA thrilled are to unprecedented releveraging a trial and the and impressive We in of where number last in humbled X XA cohorts demonstrated Phase data share results quarter, observed II we and points.

Patients weeks. over the in achieving to option robust the observed. seizure-free with arm continue periods placebo-adjusted population. was for seen With was in and of X the never was releveraging aware minister periods. a which neither over were placebo XX X other investigators weeks patients XX% period patients had status and the period patients out study an the seizures from on the the or completed that This and for X-week XX% patients patient XX motor label notably was before expansion placebo X-arm run period were one four placebo. after severe in XX reduction open XX or study, A

other well-being study and In leading in meaningful severity, improvement with the addition, and encouraging of patients in these clinicians with It's impact available also areas disease, lack not of and of very measures. alertness we in seizure disease-modifying only finding caregivers noted severity saw the overall and given also impressive but important in intensity, the both of currently leveraging areas a treatments. the to by improvements

from dose no And reductions proof with the concepts, These potential enroll physicians for we XX was which patients relutrigine cohort caregivers, moving following a and for to or the up and successful best-in-class therapy study, adverse results goal us drug-related further Lastly, screening the interest those patients. and set has our well treatment. events initiated required generally X severe relating of of first receiving during precision potential serious tolerated as of to closer bringing been the aims study.

the more this, When to on of a in protocol across we working in are people which block. the the GEs. data see diligently regulatory the potential already believe we EMERALD a GEs. is affected which all patients sudden across broader southern nearly a relutrigine action, finalize nitrogen, the mind approach there for channel XXX,XXX XX% of With currently used the from addition, all agencies In all on for with U.S., study to we're XX% target mechanism channel that

of and initiates finalize in to We end by this expect quarter XXXX. the

for more Southern of of relutrigine, SCNXA the expression population. part out and the designed selectively come effectiveness target calls Brazil. to examine elsunersen directly protocol by severe clinical Last excited program early in our disease a in very and decrease of China the the the the progress XXXX. a epilepsy ASO, EMBRACE data, Vormatrigine gene in Part SCNXA-DEE. to important of there's provide We're This and very control lot our both continued A onset study safety of quarter, elsunersen, potential Running underlying first and in will our is in we modulators seizures and the

XXXX continues transformative year. for an be has been a in time exciting This practice to

in submissions and number to in Tim points, NDA to our Kelly. on remain ahead me call XXXX. the Officer, look turn first mind, that Tim? We of the Looking rigorous to With forward we XXXX, now let Financial have our focus inflection of potential we a over execution. many Chief

Tim Kelly

summary and programs. $XX.X expenses good and today's and thank for joining QX, Thanks, provide the Marcio, quick of our our clinical and movement reflects million $XX.X remaining I'll with million call. an a increased R&D our third everybody, activity in on $XX.X quarter for operating amount financials. disorder morning, In epilepsy that you G&A were million of and

quarter, operating $XX.X Praxis third similar focus on it cash, reflects and the XXXX the During quarter to working in second million capital. our of spent

Praxis million equivalents in which increase follow-on with to to and primarily $XX.X cash QX offerings due the this ended We earlier at $XXX.X from the XXXX, December year. XX, marketable public with compares cash cash, net million securities, proceeds of

Our cash their of and readouts. into discussed that includes it through Marcio supports today programs all the funding runway XXXX

I'll it over to Marcio. you, Now pass

Marcio Souza

Thank Q&A. you, will the Tim. for open I now call Operator? now

Operator

Instructions]. [Operator

Ritu comes TD Baral question from first Cowen. with Our

Ritu Baral

expect as the cohort think in be original to any A couple or of result on patients XX for registration, in cohort enrollment expanded specifically, criteria And that -- expected XX prospectively for any patients the changes the [indiscernible] you will you patients? so, any or different for do about safety. efficacy to than expanded result questions relutrigine, if in the to it sufficient

asking maybe Can timing half. might question that on that that seeking how mentioned the you talk and then of to EMERALD? more is, specifics on regulators you are cohort you're specifics around any feedback any with to in the second alignment you part expect first what change on And based you

Marcio Souza

Thanks, good. question. Ritu, for the Sounds

enrolling on study So second one, on the now. the are right additional the first that patients cohort XX the for on

patients. So active number are like one, there

news [ in been those and which The start patients So major study we've randomized is the good is study, getting that actually dose the into view. the ] for this outcome to very eye drug. screening our patients difference

per day per was per X kilogram So per starting kilogram one at milligram on is the X.X previous and day. milligram this study

So it's straight believe most up efficacious. into the to be the one we

light or and real effect maintain the is It's just a seizure-free no faster, in fast there. of number So may we say, deeper number believe the and that. of terms the on or expanding on effect of a I possibly be impact major to inclusion change even going patients separation criteria. days

we're And here. patient expecting not to do see so EMERALD, timing on for then different So the from population perspective, protocol. a one a a have we

on traditional would more little weeks. call, maybe inclusion patients. is XX, there. specifics those the it's aligning parallel what like a of a bit are aligning of We to I would I really we're are XX as expecting run group What we say,

seizure sensitive what define #X the a we be that like So with our their high geology. that efficient. believe is now view burden can patients as for have to the long not and impact genotypical, we can And we that #X as consistent typically mechanism, with of our should and independently position are right they play

a So the we're checking going and and just details that end how beginning terms and be of next of by year. size randomize very year be like number that, to the like to very the should to double in of things done able which by operationalize then small of like

Ritu Baral

Got it.

going want that's you I to is have these rational. So need are to to but genetic a patients, all mechanism they clarify, geneotype

Marcio Souza

right. is That

Operator

question Yasmeen Sandler. next Piper Our comes Rahimi with from

Yasmeen Rahimi

Two the questions analysis. on interim

XXXX. it to read sort disclosures X the wondering expected the of be if year. could then both end to get moved I around interim just through sure interim it bit think remarks or you've the happen investors the to on want got I the to were just into make appears the a helpful. insight walk get not XQ of and will the an just just before that And you based some out were color interim, us we'll studies, I could that made steady little think why Maybe about X. of around was just

into reinitiate last question Any data AT like helpful. II the that guys then study? Could color the on maybe you of is, Parkinson's program a comment be I'll in noted disease a I success study, would jump that be Phase would back the or that you Parkinson's think Phase And queue. outcome XXXX. upon in And the move around into III would you would and

Marcio Souza

confident start Sounds So our every interim, in in like the saying that for on the good. increased by a execution Thanks, the like right? monitoring aspect trend so every will I just successful we're very discussion confidence on execution.

success we is the Of be, would will we're course, begin with towards main these making driver And back that So [indiscernible]. the our file the trying to here, step I and can program right? and can a for it up biased do wrap we what if a take clear to to like be deliver hydrochlorates to efficacy NDA an and of ulixacaltamide incredibly successful patients. sure say, the that safety program

is important into of on of members, as terms analysis that factor the argue That to the question, which analyses number the either GMC stature. that the look interim were the is of influence in that, is quite their in here second expect. exactly a work part your the possibility would most of of you When I And availability we eventuality, right? conduct the things ability there bumping up the data. a cleaning was

result negative Study -- by the of to I same So a make X is influence after study #X. But wanted influence, very there positive readouts. no that influence shortly sure time about we on to too, mean the with and

for that in safeguarding combination is is make more combined view package MDA the the as this, positive end. rationale on which the main the of studies, like a Study our our the So our results in it its is slicing and so made when to into program, and X overall that us right, view sense you're looking for XX,

Everything far. is so progressing brilliantly

so last that on grows for terms incredibly want be alike the to from ourselves, time had time essential on is study. to they important what of to for the this them off strategics into expansion I the On our confidence that study, make open the Parkinson's We I We outcome we sure and need some our on asset an Terremark, think to ready business. of disease an see like in got would forth so indication -- feedback ground. and FDA on gets PG the really

So very as program good a in a we significantly advance would Parkinson's to have Phase that this idea design II/III study well.

planning. that of just terms in the restarting we So

we to So time, and at kick that restart. off already,

X. So we of Pollex have a instead portfolio just in of indications

Operator

Joon with Thank Lee you from next Truist Securities. question comes

Joon Lee

broader including genetic seem have relutrigine on there's clarification. Just question. you want extended the in to and study be Ritu's ] to to follow-up. response a your [ quick And a to epilepsy you stick because I [indiscernible] EEE basis based Will

Marcio Souza

good. Sounds

include reach will are will we information. much as providing the to attempt answer you genotype there patients So, we LGS who to to on again an collect before attempting

[indiscernible] terms as claims know, we one there. the defined and our actually think as [indiscernible] for a highest you're final projects [indiscernible], patients in diagnosed to with on in when the believe But X like define not now, a for these be clinically Sometimes, we drugs. of mechanism spot use sweet data right the data is going have you of which to typically looking that of of the issues highest are

this time, having inclusion yes, discussion be about will now. doing. at criteria discussion of part the that and So like is we're But is the entire same measuring right

Joon Lee

Great.

[indiscernible] a great. study, So it's

I analysis X to interim just Study referring you're your analysis, interim that referred wanted to for you interim. the program, the essential For clarify to

trial? comparator Is from randomized you that line top correct? will parallel trial the withdrawal So then... And on depend interim the

Marcio Souza

sorry. Joon, ahead, Go Yes.

Joon Lee

Yes.

or into interim already that X -- there is -- analysis the number that no So is analysis? is inferior -- and there part baked

Marcio Souza

Yes.

I X is the here. We of would Interim between Basically, don't right? on X. analysis terms not only, mentioned, talking there's events be Study appropriate interim claiming the for an multiple alignment necessary like database, or an we're believe in that that Study So

that same the concomitant pool you of those So recruited could is as patients, they because play, influence some right, are studies are the be and studies. know, from to and water randomized

well. why So internal the the be outcome that's of influence depending some as on could there on

We our will [indiscernible] that monitor like to rates of course, like X, thereafter be the that. reading is on trend out could and study argue on shortly base things event Study

what that inferiority -- question sorry. on the bullish was -- about second of where begin quite third So to part the the

a it the end on lower that. conditional Right margin on is there the futility like of So interims power as standards.

So fairly standard in they terms [indiscernible] inter-analysis say, I zone studied the or was reason of the with. wide, very there, the a designed to begin bond why because would that's

And which the that, as the fraction overall course, all spending to to on other work that well, for come. and perspective successful of a the toward top considered all overall balance from hand, our be wanted as and drive if of from execution new should perspective efficacy information

Joon Lee

last Great. And then question.

As to [ very X voltage-gated sodium we ] [ it of impressive channels that targets actually January, the X even of all system. is from works the perpenervous ] look [indiscernible] only it because sugetrigene likely at in approval in the

So anecdotal it's you're any voltage-gated of receptors, other out thing Any or can but X the reduction evidence formal or which I targets study. your receptors at looking actually advanced interesting Phase channels. as X well, your pain that from of pain not

Marcio Souza

Yes.

execution, a we you excited evidence It's seeing. us and into do beforehand mechanism with acute is believe something while. mechanism, we're what's and preclinical we're yes, potent as the generally that to bank for appropriate we we finalize in but of for everything very tens very We excited what chronic that, important strong standpoint. some that duality paying is thought [indiscernible], in quite think chronic this a look And we models as capital we're strong like of does. from super in made a mean, I looking well, know, have most just the paying for not it like general, allocation on priority us, So now -- the about that competitive

of sure make of year the well, our then be in top As would with and all you. competitive early that plan

Operator

Next question Oppenheimer. with from Francois comes Brisebois

FranÃ§ois Brisebois

scenarios potential Study thanks for of, kind one the and complexity say, dependence the the between of here going and through the impact X. on

where there Or the So of could look? Or Study -- of because -- top it can line X be the is time short be line terms after help it X -- interim is Study in case is the really go things are actual of Study but versus that X. to data? after it interim assured working -- more scenario good the that's study we stop the for a we interim what actually into the share understanding out? the that might will top line come Just any on

Marcio Souza

think if Well, Sounds I cans here. ball think I that like about in areas good.

the mentioned, case, then you [indiscernible] is that at stop quite time, It's the so which same as package would and point and complete one, let's time, at studies X obviously like and that having out, right? just Study will for So the like in both base X to play be be that potential results on. but overall not obviously positive, the Study

that increase think to Study size as now an X. is well of I the opportunity

And well. Study we're quickly have be the as conversation having study the moving and X, in like should will very case, we're results forward, that we the or for increasing size as

is larger is highest the rate store, when the zone. will year. That of on power that the is So dissociates happen that our the just probability on widest priority conditional it this because that's that the

impact, the the terms in the that's X studies so highest say should right, think operationally, other having like I around it So first on. making we wrap to sure with the like up most I'll of ready and plan the

wanted So look how considerations the being we interim. why into for ready and we the that's

FranÃ§ois Brisebois

to look potential U.S., here? ways ex understand Can this about then help on of a DE front, at Okay. commercial U.S., how you commercial you there's lot think And us the market. different the

Marcio Souza

say, business, States. majority vast in United dollar the the of perspective a value is and Yes. from would I The

looking U.S. talking bit of completed another revenue. into XX% in about of Our shy the back for about the in XXX,XXX [ just relatively U.S. modeling the the like the like When consider the gets And GEs? utilization shows a so you quite around a it's that of We important then potential yet figures mechanism limitations the little here to in epemiology the refinement in of small XX% outside and U.S. -- terms ] on. We're multibillion-dollar of and PEC. in and market the share patients. even big U.S.

And well. then it's as to becomes important call the more, the access outside revenues. to quite of the but U.S. bit as to from opportunistic ground business going obviously, drug I'm not to off get a a perspective, meaningful patients important little have to get the and

So how of U.S. been about the X/X the modeling. in X/X outside That's we've

Operator

question Our Suneja next comes with from Yatin Guggenheim.

Yatin Suneja

would that long to you couple prespecified the some the around a like ET at decide also side. are, the ranges the enrollment how then how maximum the sample front, us let's and X you number understand with as adjustment long take? where you and Just based record, well, Could at talk in enrolled mostly X take not, say, patients we on this and size in turn. of for least maybe Study patients that how possible provide plan are about increase many put that been me and in if that the have numbers on will max the Could you size

Marcio Souza

Absolutely.

now we preserve a give us in we I'll feel give could right order for the So much optionality to as lot. like that as

week. look per way patients randomized we on but of on the screening, being the So would the of state, right, is the not into current the patients, study top I say,

between look randomized when what per we maintain is into patients you week and XX can XX anywhere new patients. confidently So

can a could be an X that of and XXX increase So an like in between XXX achieved if fast I'm to to of potential here, [indiscernible]. scenario X scenarios going where you weeks use increase forward like that, you of anywhere patients so X see like to

study, need course, very that. actually study to XX fast weeks terms of if needed. accruing of is Of the completion after in patients new but it to you

Operator

comes question Douglas next Wainwright. H.C. Tsao with Our from

Douglas Tsao

as just Just you to confirm did. what

enrolled simply study essential scenario is expand think indicated that patients. the that for and I population enrolled or Is randomized the XX the into you patient that So ESSENTIALX? in you

Marcio Souza

Yes.

we we're one on of very we, the a randomized lot we easily would because that that is comfortable achieve in can not say, patients that organization a say I'm relatively pace goes a our I from to like there's given going week. So very, this very,

Douglas Tsao

little type helpful. going Okay. into doing just or Great. what could coming color be of more do And full of if a with sort pain to terms coming Vormatrigine a indications out moving then need sort work and you're these to in the plan. with more development of forward you That's provide of before --

Marcio Souza

CEMEX paying, of What this cetera, biomarker important thanks right? last So There's pharmacology and preclinical us relationships for would [indiscernible] moving are for lot like is week confident in like were and others certain of say to is we is potentially space, like us terms of for relief things, a what types we're year Like a done few type similar for et and data, with So you enough of couple into the that into say. working, we that as ideal the one of the itself, right? a when is our I thing. can [indiscernible] what have last get confidence right? working clinical we on quickly the early years a point a been that's to were in When package, have to would as well. incredibly could I look data,

both and other impact from here developed disease. aspects things I And the then understanding like really being talking central differently about peripheral and of we're what think the

how that study things we So this more show mechanism expect can and and like much at, cost that. from again, in [indiscernible] that study to

to showcase to you. more speak. in So very And of lot we a literally work's the think earning of and that's be to the that now good year. all the A being to going year as we're done the finalized be position between I end

Douglas Tsao

then one And Great. Okay. more on elsunersen. just

I I last you think patient any that indicated I [indiscernible]. about of the indicated global today, of quarter, if sort they're registration added you you as to there's think want And that first with program understand in for sort registration change just been But global the being that study. forward think enrolled the was being drug? to

Marcio Souza

Yes.

as So aspect that's very a no, important well.

into way the twofold we've here. elsunersen So are been looking

consider assessments Brazil cohort to done explore, lot or to patients been or dose like a to those exploring can placebo particularly be that of those or realize drug patients the drug at you levels, doing one imagine. we So that are of the sequentially and to is want you other range case. as all right on We're on who to well, important second a different or on the in safety what's increasing now exposure sham as

was actually from to good as separated we milligram for cations what X important it believe a for exposure global long-term dose very study. to be the keep month per So that us the

good our year have needed We meeting do which what meeting very still on from pain their we in the already occurred. this slightly was expectations But finalizing have changed a FDA with there. is general, the alignments -- end on the on was when

bit initiate it's confidence of need we So little there, nothing fact the final time can it. have problematic just to the make a sure on kind more so drove that we calling but we protocol

one. that's So for bottom that line the

Douglas Tsao

Okay.

I at the some and patients -- would per the if is result at higher And that are been there in a registrational I X in there if you the that -- the chance mean European is with in mean I results being so look milligram U.S. studies. doses you're you've warrant confident scenario Brazil potentially kg additional And now, a looking doses. studied know very dose.

Marcio Souza

warranted. like if whatever. the either different we must that to U.S., got open higher position meaningfully case, see than ] took Australia the in the now. far meaningfully we drug phase that dose meaningfully global milestones things development follow science, not are the be on phase yes, control which to are we've Europe to of the the [ in we its in cohort and and gains on after the Tier it's different And So remain and it's so right label in be terms that's would all a in that in complements in going if U.S. Yes, actually even a to the patient are what continue possibility that's patient open the faster like different taking the right? in on and control

by see with be dosed terms the like the here going are patients, we for the global So time original the study. in lines we that driven be lines enrollment there it they're but will the have curtailed of results to time X

So it in complex terms patients. possible shouldn't be a for how of results [indiscernible] those

Operator

comes with Yazdi Kambiz Jeffries. from Our question next

Kambiz Yazdi

response A a The in One. well EssentialX have Central X for pretty the innovative Study couple was to centralized steps response control controlled in has what of design taken you placebo placebo consist questions Essential What more demonstrated in a second in X? registrational set Vormatrigine. a program And have maybe And in then of questions, generalized me. of sodium seizures? kind would epilepsy. of what as on PGTC historically channel blockers

Marcio Souza

Kambiz. Thanks,

as you already was pretty to to when to home controlled, said, EssentialX, one well throw add placebo on was we that. So further wanted the EssentialX, there aspect you're looking that

on I sorry, patients a or but at So assessment what slight, a -- stable [indiscernible] what that is important assessment streaming -- in there study we're by on low would on chains baseline a is were to EssentialX a that assessment. are seeing I mean baseline. And say, there is

look they you be bit when patients that on tended vary little a to So placebo. between higher those more in into those

maximum similar premium right? the patients So chose Now start can we on end very controlling question to [indiscernible] actually not don't sure successful want from visits was, variability We the tighten similar, ones. make whether formally like effects. on that long and you wanted to the We potentially making the we smaller that what a the right? to visit be to the cohorts due is were monitor that ask to we or the between it's only patients but to day, believe want parameter to EssentialX, of not but we study, again, that drug from we influence effect, want ammoniation the one, drug so that Xx to depart rather the those have the to to for takes sure going working know how are add from to on, going be it study we at placebo than because is

of then very surge really that to think primary is are on drug. question potentials in is I very, partially patients. a the blocker physiological that being norms selective very action more -- isn't on put your that and And historically. terms block the expanding results that these with channel really that the second lot mixed measures but And as and generalized lot earnout, works we not good for partially these on like in placebo we're selective control, on control I'm can a confident there. So it's a hiring that going the very on a loss pathological of because the like the place to really or more events, the selective call

[indiscernible], we would enough, off we've been So lot conversations ground having conversations got the a the that say, excited. get like that are prices of and I the interesting

amount there more much believe A generalized they are surprising bit huge a actually important lot me severity of was as excited a those are there, and fatality [indiscernible] for is of happen patients. equally and well people and I terms well potential a the on seizures or when from generalize to is so there incredibly as because excitement impact about that obviously, it's little those Vormatrigine, in

as generalized we registrational to would kind be I say, RADIANT to of as a can would going is And to us there. what we well. we're springboard to enough, for RADIANT, what and study. So phase kind serve from have going soon a for a feedback of expect see design

Operator

comes Fadia next Needham. from Our with question Ami

Ami Fadia

me. A from quick of couple questions

line? studies? ask -- time enrollment after of the for And in Can the Ulixa. on X the target how slowdown Maybe percent one. patients on you have And shift the completed enrollment rate relative the you was a I'll there question next what my Firstly, answer in to that this comment caused the randomization enrollment?

Marcio Souza

Sure.

is talk from about in that slow expected press down So in remarks patients Ami, tell or the But can we to proration you screening. going not we're and what release. I what the right no as on you right, mentioned, now, on that prepared

we we and been the saw coming intentionally interim. for right the like that see think best positive a size the of the managing can continue I fairly to that I the increase like to [indiscernible] so we in are if patients historically, decide after two we study think we of QX, through expect robust get we continue studies have study number to

a us not lot course, on patients things So and of it's and the meaningful. influence been but scaling of like are that, which

Ami Fadia

it. Got

on RADIANT generalized to mix you're patients sort between and Can about then plans focal And further many might it your the inform how how talk and epilepsy? in that relutrigine you Okay. of targeting or develop of the in study. patients generalized

Marcio Souza

Yes.

expected about is goal agent. patients are we're seeing, is the on the days, So again, early have to what XX% focal XX seeing on generalized XX% interest. what What we but

I because that like, say, a a on were, than to of the didn't the of again, in selective significantly of got general, that would here control interest, like we lose we terms and in I'm particularly and relatively the of huge want to For, target say, is, here. of going more expect we size number number completely, sites we patients

that bit the little terms of a or so patients that we tight to enroll. in kept we So expect

as adapt or generalized open go Focus overall end. and the needed maybe we to in So and to sufficient the That label of continuously going But of based at across, we're on seeing study, are expect what as right? total should we number be from and terms patients reducing the increasing an is it's interest. like see

Ami Fadia

from me. just last a Maybe question Understood.

with what that you design to the assumptions to saw in you relutrigine patient this As regards would performance SCNXA. study population in data embed be the relative you making

Marcio Souza

Yes.

incredibly of is in reductions mortality but right, So when yes, [indiscernible] Chile, treats, see hardest Xx were definitely were arguably, there infancy. are look [indiscernible] those higher difficult that others than incredibly a in like conditions to hard across the example, you to the those SCNXA so board, few to see for

higher So bar. is like significantly this

above So we be study for that, think that we're patients. at powering of the don't that these little seeing, would unreasonable we're conservative, being it's efficacy bit this the course, assume a for more expect or levels I not best-in-class but to

Operator

with Our Beatty Joel next question from Baird. comes

Joel Beatty

between that if The amount is of time those first events? interim we successful? Or learned final results one we the time get is on some same there the acceptable, could the interim at be X will ulixacaltamide, inherently is

Marcio Souza

Yes. No, Joel, [indiscernible] is for that. thanks If successful.

an efficacy equip crossed, So is the at buying have results same it's the will time. grid for we

Joel Beatty

those And to should patients are blockers, then calcium agents? working as already patients could about relutrigine just beyond DEE, to kind DEE the responsive of to we or work you in about potential channel who are think in who talk responsible

Marcio Souza

Yes.

interesting. quite it's So

to that like does preclinically prior did, we like are the is looking I not to. think or patients you're should it restricted it or who look had expected work could be -- response that all when to and

example, published into then we're first-generation priority our efficacy -- some population, that The there as work the on are would therefore, to in and outer ideas with If expect seeing over syndrome dynamics is here. case, better The which -- general back. would a preclinical expects you you neurons right? the in look and well. have So not all we is that, models, compounds point meaning did two should results significantly actual, are have there's models, the outside the maybe clearly that towards the that on of among work put efficacy the the unexpected on wouldn't we little animal expect quite a for blockers bias at than a few other to

channels a When figure sodium a it. if to going happening, neuron, crazy is a we very are right, way like in term could observe late describe

etiology activity for brain is important that or seizure even independently anatomically. stopping control So very origin the very, in of of general the or seizure the

here we So wide efficacy. of quite expect range

It going I clusters. the example, question do one others to count to who clusters always, some is how can't patients be, think the don't seizure because study. right? consistent is ask and for similarly, what wants to have we have You're have

So put we together. in shouldn't them

So that's more of registration anything a else. than

Operator

Instructions]. [Operator

with next Securities. comes Laura Wedbush from Chico question Our

Laura Chico

me. for questions clarification Two

now XX%? I follow-up. And guess, then And expected the it at interim differently, to has for the maybe to ulixacaltamide. that sense. the is though, First example, asking also this executed. clarify, I'm a conducted I have Is trying to XX%, timing be for XX. is shift interim Hopefully, changing? for makes that quarter to on the was understand that does which extended at I actual interim the if this first does is the What

Marcio Souza

It sense for Laura. the lot makes a of question,

for gave XX%. and XX% your was we information I that the understand correctly, range right, that's if the So fraction, question between

change. So does the range information for not the

is we it not a day by but range the on did change depends the transfer it DMC, data exact because the the course, calculation that. Of of

Laura Chico

Okay. I that was helpful. also the same your to ramifications then, study That's the comments are these My and understanding conducted I understand conduct integrity guess, to X. respect And Study protocol. and about with under

release on of separation how any see trying And like to communication. understand feedback the I'm they to would the FDA results. had So results from have you

Marcio Souza

Yes.

is. part So the last

opine don't health be separately. So we another analysis. often I they rate just think from the the studies data yet which and analysis they reveal plan, communicated, results though is did then the for how on plan did should And there's another internal not, analyzed

it that reviewed that. back received FDA the months from we So on and many ago. was

a So on call that.

So definition the like of sensors, you're influence is let the operational IT on correct. meaning absolutely operation,

they more study a on know or own to X. tube rhythmized or wouldn't on study a influence other. their study they one the to finalize potential as are patients by randomized meals potential failure are studies of Study patients impression It's study of The are the the who by since

be didn't by impact to potentially unduly a qualified expect or perfectly that in we tact a to create influence a case, study be quite placebo we on honest here, positive study. creating successful So potential what

but overly entire program. trying end, to of again, the So gardeners may our cautious be on set

Laura Chico

Marcio. question That's for helpful, And last me. maybe Okay.

I Parkinson's. to think mechanistic I rationale read-through know trying guess but to expand a missed understand ET the Parkinson's the more you on and indication? from and little in would this, the earlier, restart it be the I commented on the I'm studies you bit what better I could

Marcio Souza

absolutely. no, No, Yes.

stopped that for to scientific Parkinson's. I a in actually a strong we to study to when The last do do very So little our lot be stay in and is time Parkinson's rationale very availability terms had of with with the results from capital. expectation of think

weaker, be got as Study we X data our the expect we the more Parkinson's and course, get time ID-X a they fast we scientific positive think forward, are for then I not rationale. excited stronger of to significantly have from essential standpoint of do we data, safety front

be reinitiate actually [indiscernible] we results don't using want a significant time why results, So the of to essential reinitiate Parkinson's. capital like ready X at before amount to the of like the to

a necessarily So more than the of use there have and not maybe that's delay mechanism. the to message

a X% movement far the I there specialists other patients, go-to-market while patients. with those to of strategy where more both there's patients is about -- the is think XX% only a of Parkinson have X% here to XX% coexist lot

that strategy kind a is in leaders. of important. market general is of is that the engagement the So the penetration And just other in

think goes have patients without we it can we that saying as a well. impact course, Of meaningful on Parkinson

Operator

call turn closing over back I like the to would remarks. for to Marcio now De'Souza any

Marcio Souza

transformation everyone you over the with of Thank you programs one X joining our year the count went months XX was potentially ulixacaltamide. or I last a so evolve NDA and much. call quite appreciate months, of and midyear terms going really and on for quarter. lot be company registrational in for continues the to to next as what the staying positively the so company if us far submitted it's the

and of value in of drugs created all be As those patients of be us positively dollars we will billions will progress to to continue impacted by millions shareholders.

be So future it to appreciate we looking touch. you're more going near much. updates sure providing forward in Thank in to and I'm the you so

Operator

Thank the concludes your you This participation. you. Thank conference. for

disconnect. now may You

Praxis Precision Medicines (PRAX) 6 Nov 242024 Q3 Earnings call transcript