MRNS - Earnings call transcript Q2 2022 | Marinus Pharmaceuticals, Inc.

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Greetings and welcome to the Marinus Pharmaceuticals Second Quarter 2022 Financial Results and Business Update Call. This time all participants are in listen-only mode. After the speakers' presentation there will be a question-and-answer session.

Now it is my pleasure to introduce your host, Sasha Ellis, Vice President of Corporate Affairs and Investor Relations.

You may begin Ms. Ellis. Damouni

Sasha Ellis

Officer; good Executive from Commercial Officer; Joe Chief and Scott are Officer; With Chief Hulihan, Chief Pfanstiel, Braunstein, Christy Medical morning. you Marinus me Dr. Dr. Financial Thank Officer. Steve and Shafer, Chief

XX-Q programs, and remind statements. or risks with our the turn from forward by I statements results, in with including of significantly both looking those future making are expressed statements to to are and clinical the implied over under are call risks XX-K, that forward-looking and business uncertainties such some and the Before CEO, associated X-K. forward we risks statements achievements like I'll uncertainties securities Securities Scott or looking reports Braunstein. everyone cause to begin, today would filed development that the Commission, the These now and could we company's our laws. performance described and Exchange our substantial These Form differ

Scott Braunstein

you to the exciting our to Thank patients of older. with for ZTALMY disorder FDA received associated CDKLX first that prescription the patient pleased receiving deficiency age our ended forms. approval the completed years with Sasha And enrolment in call. of news now quarter seizures We be two welcome treatment first and and and

of Our has National and excellence Epilepsy centers educating and Centers. CDD force sales as been in engaging the physicians field

ZTALMY. We are the strong encouraged in the interest by early

the publication the to addition Marigold of April III the results In Neurology. Phase in Lancet

publications June. survey in which international and published individuals supported participated the momentous expert families shortly. CDKLX for thank panel consensus for researchers, respondents for investigators milestone. deficiency of clinical the to recommendation all of the who include and disorder international they will in for was Christy discuss results would to in Frontiers Neurology timely launch, with of in help I seizures and again the this the ZTALMY in more the with are publication offerings an of patients, achieve detail have the clinicians assessment treatment done management which and Both all like CDD. the in associated once First program us Among

We of updates. number have other operational a

And be the start to sharing first next during dataset our we like the development generation program. call. will I from would So with product

We can drive we which oral goal our blood are makes have believe bioavailability steady seen next Key ultimately initial we Phase believe desired showing efficacy. improve which compared first I and suspension, study The Joe profile enhance is second will dosing. the achieve higher promising features in the which our that will of call. results formulations, volunteer of detail in our generation several levels state in discuss on differentiated formulation our us chronic current utilizing the of to PK goals to to generation later healthy ganaxolone,

We need Syndrome. suited also for as dose of upon areas we believe unmet that well address think potential to are frequency it will ZTALMY's the new indication including be important additional to Lennox-Gastaut about improve

share. important clinical several I late-stage have on excited to We that updates pipeline our am

supplies, new production the preliminary BARDA the trial with modification epilepticus. status RAISE program. have future ganaxolone The Marinus' the and the our the productive FDA agreed alignment changing also ganaxolone clinical system. transfer it We've IV interactions to IV reached for is including of manufacturing principle IV ongoing facility FDA the acknowledged the of and a formulation plans acceptable to ganaxolone start in me IV had that Let specifically change, proposed in formulation on with

shelf greater ganaxolone stability drug room are results promising support new life of initial our Importantly, of assessment us confidence temperature. shown this alignment that provides IV XX using filing batches likely meet new application. of months upon the will new necessary requirements buffer the that to at a FDA Early has a than

We regulatory expect teams with this formulation. and extremely and a NDA both productive our dialogue utilizing for our continue the manufacturing done site proud FDA. CMC batches exchange and by I'm our in of to buffer producing the scientific begin quarter to year, new IV of our fourth the work

on RAISE Let the trial. me Phase comment III

As previously protocol, of the the eligibility accelerate communicated, be to expanding is quarter. The criteria implementing end we sites are changes to third adopted the important by the across enrollment. to amendment of protocol majority RAISE expected clinical

to first our dosed. TrustTSC has Phase trial been patient we randomized share complex, III pleased tuberous the Moving in to sclerosis are and that

this the patients screening in continue sites track quarter U.S. of year. at European third with to begin We activations on

targeting the are Europe, Canada clinical in and XX sites, predominantly Western U.S., We global Israel.

the believe will look on Phase the titration data and quarter anticipated of tolerability that XXXX. forward the to and a We continue improvement efficacy in III have schedule to readout new meaningful first

that for to have need option during our with continue After interactions with for Conference. potential refractory and there significant recent a an been these We TSC the seizures engaging TSC, the actively team's including both important our community, patients believe World associated TSC has participation be clinicians unmet and is we the families, with suffering to ZTALMY with patients.

authorities end launch. European the in efforts both of Marketing Marinus significant first to Orion the There how Finally, other In families quarter result Authorization regions. XXX XXXX. been CHMP Europe, submitting and the targeting plan patients list by advance of to some ZTALMY put team globally. to In EMA's we a activities, questions ZTALMY Application U.S. continue in by parallel to partner, and our work responses with November, the Day commercial and from to anticipation are comments we we have to commercial key of of complete regulatory a to on would opinion the strategic CDD Corporation, which

opportunity to we to is help ex-U.S. broader patients that commercial global a there are believe further addition, expand exploring and footprint. In ganaxolone's alliances

to like ZTALMY. for I Commercial commercial on Chief turn Shafer, to the Now the Officer, launch Christy call over our updates would of

Christy Shafer

potential of commercial Scott. see ZTALMY and Today, in ZTALMY continued from high that the the reflecting in for share the you, both physicians a need enthusiasm the to in ZTALMY the level our first could of to from approval two launch the and are by on Thank U.S. and of base prescriptions. of in this caregivers, ZTALMY. provide launch forms early I'm Since March, the we've has excited field community actively enrollment enrollment prescribing from feedback management. encouraged early infusion Initial of launch, fielding volume strong and strongly we unique are a questions completed weeks patient the In regarding confirmed interest physicians progress

CDD and early In interest Centers accounts. encouraged key referrals U.S. centers, from outside from addition also to national we're Excellence our epilepsy by very of the coming

lives U.S. and relationships see representing the coverage have payers to support establish key of begun criteria to a access. already we continue Additionally, with covered published positive to large being proportion in patient

Drug that Administration, reminder, least which a in As the shared June is by restrictive the we received V designation schedule. Schedule a Enforcement ZTALMY

option pleased as are designation, will strategic treatment age an patient fulfillment. our older. caregivers which to efficacy important are of providers providers The seamless with payers disorder with and seizures launch further ZTALMY through and healthcare years CDKLX profile to from and continue of commercial enable parents, on healthcare use major We builds X deficiency and in ZTALMY this of associated access confidence our in for educating objectives prescription how compelling patients and safety the

We are benefits which tools patient throughout of on Physicians experience, ztalmyhcp.com patient about of provide strong aligned developed is single pharmacy information the prescription access website and indication caregivers a journey. ZTALMY. important pharmacy safety These at track professional it believe educate and the team caregiver providers available our to record at information and specialty to specialty and information to that the disease prescribing ZTALMY patients can important based individualized critical fulfillment healthcare website support communication CDD suite and community. process. have healthcare marketing ztalmy.com has the their to Orsini and provide is dedication Our We caregivers HCP to website. rare selected with and a the about

prescription education treatment patients part patient prescribed the to ZTALMY includes Marinus' are As benefit program patients terms and that helps conditions meet coverage prescription. support who eligible low need, delivery, throughout they This facilitates copay have support ongoing and $X insurance that savings commitment a gap as prescription programs support no and insurance, financial patients limited provides Orsini program medication helping support pay a and commercially launched per the fill collaboration ensure One, support ZTALMY as program with prior receive ZTALMY the drug the authorization comprehensive or journey. treatment we for to in the with in access of and who for

with In team ZTALMY the pharmacists available coordinators Monday patient support Friday, through XX/X. care addition, available includes One

in ZTALMY also and targeting field large for educating In centers, education on physicians Our open-label efficacy Marigold are of us of expanded XX epilepsy label the practices parallel U.S. allowed are distinct study has supporting representatives the payers. and profile of discussion advance to access of those who including on XXX have of focused epilepsy we each to coverage also who circumstances participated now close the outreach, the patient. access mindful experience program, X ZTALMY and disease epilepsy CDD being centers individual XX availability excellence to key team rare The the national safety patients significant the approved hospitals, and extension the with centers. to our and ZTALMY, of our

with covering managed top a fee-for-service top XX% coverage patient commercially will the the and relationship we expect being both of continue CDD access we Medicaid national and reminder, U.S. the with regional lives. payers, As payers. to through most government managed with approximately PBMs establish Along XX% population remaining commercial and

early tone be the far patients and treatment recognize indicate level limitations and within based CDD able as this prior believe to a families, with to specifically drugs. landscape agent of on of diagnosis the coverage have conversations access broad should engagement, who in we support associated disease X the years thus on of pace payers the we antiepileptic and impact first approved our Our achieve failed expectation, seizures X X with or patients CDD. ZTALMY profile for Consistent older CDD criteria product confirmed and

states The increasingly November is of pays established priority into practice Recent that CDD XX% ICD-XX database of initial being XX XX% CDD suggests on largest healthcare excellence centers is was located. to code, we the data are physicians. where Our the concentration and for implemented by , that the with believe believe include patients that on is the usage recognize in XXXX will ICD-XX claims period. of increasing years code are usage unique determinations the and help or in take consistent of XX shows year-over-year during patient claims. can in XX% We patients launch this XX% pediatric visits alignment coverage older. Early of growth time ensure with illustrate patients patient access the payers. We which code

the be are published of majority within commercial Medicaid and to X X expectations state that months. policies Our will

medical formal payer requests of from publication every necessity have to However, prior we policies. the expectation field separate and to

to prior established a a criteria. As ZTALMY One to as reminder, part our help coverage commitment access patients, eligible support of will patient

road with are the to patients, We physicians over to months. to I'll and caregivers, hand call Chief our development payers to work continuing Officer, look confident coming in forward ahead Medical our Joe discuss the Hulihan, now the and ongoing program. over

Joseph Hulihan

for increase were and suspension Thank peak doses the hours the administered and Phase target new with to controls. contrasted adverse by XXX exposure as twice XX believe The in following like XXX dose Total with where in administration. the proportion of Christy, from when the dose ascending milligrams without and of upward supports the increase a such or curve, escalating plasma begin The promising clinical was formulation ranging being concentration, results, to healthy XXX between to initial an data suspension. allowing everyone. exposure a the adverse profile believe The elevation volunteers was to to in the AUC the dosing. franchise. formulation the doses we levels area an greater efficacy milligrams predictable XXX I this could is allow demonstrated from future XXX milligrams which we've includes in effects potential second-generation adverse relationship with of approximately reference concentration, Study for effects consistent participants new current Sonance. severity. significant seen more which excellent rate and and administered doses ganaxolone the this formulation of in effects. AUC profile of PK could with majority given increased a the The efficacy Cmax. in overall under We to current development. from single amount ganaxolone you, study formulations enhanced hello, studies in concomitant the goal prior with improve pharmacokinetic a provide rise was ganaxolone results. oral vast titration dose-related further through as Initial individualization encouraging could low relative I'd single measured a our above dosing daily be of for Plasma Cmax-related received mild oral with in of Tolerability further without the XX% measured linearly

evolution in exposures concentration Next higher can volunteers single additional the studies healthy doses step to determine with Phase in milligrams include further increases XXX of above this I relative achieved. program be to peak whether in

be should patient. Lennox-Gastaut we steps, allow provide We for rare, each will modeling many syndrome study structural individualize an anticipate Lennox-Gastaut We second-generation also that refractory, more for currently can the encephalopathy epileptic oral doses. to to and a It's to response selecting exposure treatment syndrome achieve PK next the performing or ascending optimal from including causes. consistent a predictable begin highly dosing to multiple need and is to the study physicians one will a result formulation for Phase which II determine in XXXX. in genetic believe ganaxolone, with ganaxolone formulations of new

that Additionally, in several and studies targeted have selected individualization. Phase prodrug with improvements prodrug further Lead IV there been and ease offer are of additional dose data candidates I XXXX. for and IND-enabling efficacy tolerability, candidates could for oral manufacturing

the status mentioned to and help bit Scott we a enrollment. I'd our into to that dive drive moving programs will epilepticus, how protocol this believe like IV amendment deeper and Now RAISE

First, from refractory results I'm share Phase status in in were pleased epilepticus published that Epilepsia to II our in June. trial the

will patients least progressed study, had this anesthetics the IV during and X in mind, to participants the were optimistic be incubated the to third-line Phase data, in treatment study all ganaxolone I'd the III enrolled leaving extabated. sites like potential reminder, the of study. in with discuss this None to protocol epilepticus. to the XX the of within change XX clinical that support the across X successfully prior the Phase which refractory this status out failed expected trial XX hours, XX and amendments, AED. IV a We're quarter. adopted of patients As majority primary paradigm finding Of patients third out endpoint key in line changes note, the to follow-up XX the second II at or for validate With raised who were is in of the

to protocol had those of status. hours Phase transferred were received enrollment criteria IV during patients amended amended the of population current who important particularly to patients outside from RAISE anesthesia or First, anesthesia, IV for under were department. included up emergency be many protocol. received the allow had II excluded can inclusion episode who original hospitals inadvertently These an patients XX part the the now of The and

failed AEDs protocol The involves X IV in required more The than of least failure at benzodiazepine second protocol required prior treatments. X. X number change least of IV treatments failure X second-line of prior key the at or amended The rather and initially total. agents requires

line of involved be IV control The underlying investigator issues. the rationale change next seizures. that therapy for likely X treatment fails anesthesia to the this with must would Additionally, indicate study

advancing the anesthesia First, to of initial IV IV AED. are more failure after second-line centers

with without through Second, receive doses setting ICU because sedation. multiple patients already usually for in a the agent benzodiazepine, may had progressing second-line received they benzodiazepine treatment

appropriate study reducing This study potentially sensitivity failures, changes who this As qualify of would with this allow Phase response. anesthesia III by of the the We're will will the IV drug believe anesthesia enroll in increase to to IV patients the high placebo rate for confident Phase in fostering thereby that pool to change, failures. We study a these enrollment. profile drug treatment of similar study. first to the progression a and with II of the those enrollment in will expand clinically support progression patients X

determination the to clinical now minutes. quantify The allows protocol determination. with of usual better burden which third to this to seizure than established highly about feedback also and workflow. clinically for more of epilepticus potential ICU enrollment II for increasing appropriate We've study a care an from which Phase in paradigm should the the eligible change study EEG significant range immediately new sites patients. protocol. Rather of received consistent treatment and be facilitate change requiring XX qualification transition will diagnose we the protocol take our for the number the of adopting This this aligns EEG treatment, sites an study protocol. positive administered, we've under hour the of amended an more then later development the meeting administration amendment, minutes to XX and patients with investigator holding will discuss closely prior month to status physician study XX aligns the in a this leaders outreach review initiative executive an Furthermore, can smooth This the patient be with and to is scientific to to by investigators affairs ensure our clinical changes

the Looking of broaden support what's of further and potentially enrollment recruitment well continue trial, additional how to future can the working monitor patients the refine RAISE actively criteria we to the we label. will to ganaxolone and

trial For older. and enrolls example, currently patients XX aged the

may shortly receive include criteria us broaden that will allow We even to the younger data entry patients. to

support epilepticus of could the study incidence higher enrollment. this children, Given further status and change timely

XX option full for the beginning new X/X we the when advance, would have or completed in enrollment. continuing site Canada the conduct we study in of interim anticipate participants U.S. time, to third for would sites RAISE continue the initiating disclosed, the committee recommend to and an quarter. XX previously to the have activations monitoring efficacy patients. stopping independent trial in which study, analysis At committee be an approximately that data As

In the as epilepticus, exception XXXX. consent study We established community consent Phase informed continue status second to underway. in well for the expect the known data is II top in of process, half RESET line

the and to the site of and of further Also, pleased enrollment X critical is ongoing, approval we have the to to potential the protocol we U.S. designed expect RAISE understanding as gaining on European focus to We for are Site on are U.S. of IV track a enrollment finalized. the site plan activated selection in the to or X investment. X, also This indication second to in in to is larger limit believe strategy, activation it making half XXXX. of we is and initially piece to now half a the that serve the trial enrollment ganaxolone begin trends share first before second year. comprehensive a has We broaden the begin

Now Pfanstiel, I'll over with the our call Steven turn CFO, to financial you a will who provide update.

Steven Pfanstiel

Joe, good to morning and Thanks, everyone.

reformulation Oaktree, critical to pipeline, monetization an have facility, of including is Before well efforts diving announced which position, a agreement of over certain related PRV, synthetic PRV, sources, of position proceeds provide the ZTALMY. into voucher under to including disease we the have under been Orion non-dilutive XXXX. financial milestones. runway on XXXX, prior option trials expected of agreement progress funding entered ganaxolone Inclusive commercial commercialization $XX as well credit for launch opportunity our have would funding of strengthen over into $XXX our rare the agreement review of to from into European from and execute that from extends months the quarter and our sales financial million enabling and of few third our we a definitive have our within sell further our us fourth and we we the as federal $XXX These to subject close draw to call. million. Phase the the I financial quarter highlights In of and on achievement our funds quarter sale we clinical with III results like The significantly million our credit July, the raised contract. Furthermore, of second Marinus' believe BARDA strengthens our last for agreement since priority additional the U.S. to facility the cash XX earnings pediatric the our the to to through to royalty the cash a Oaktree with have

will we on and XXXX key resources to balance in programs, and the we Phase and focus specifically As investments and trials of to on and want III programs the our into as launch for PSC ZTALMY. prioritize look well our delivering remain XXXX, our funded ensure they These commercial future and track critical of shareholder to are Marinus' are RSE success. value. We as

additional As we Phase in the have sites for trial invested and an example, TrustTSC III trials. resources both RAISE global and

earlier-stage overall we continue other lower perspective. While will these pipeline time funding line to our will be advance investments from our as a such priority a and programs,

Christy ZTALMY. discussed, about of excited the are we As launch

reminder, for previously expectations is our communicated and acquisition we As XXX-millimeter cost ZTALMY. ZTALMY's a bottle. or dosing pricing X,XXX wholesale

the XX on years acquisition XX%. to translates average sales discounts be and kilograms. expected the wholesale approximately and we of Our average and This including expect is government focused X.X be pediatric cost gross initially, annual force population patient mandatory XXX,XXX to the net to weigh an approximately deductions, in low old projected are

note, in estimates population and our usage projections. age any to our with weight a are population these representing upside As the expectations adult within on an based pediatric

advances, the patients Initially, our total enrollment forms, including to forward focused providing progress. will patients on launch we therapy. patient enrollment be on we and new patient on updates active completed therapy on As metrics, look

with prescriber for treated look the dose-related along We metrics provide relevant will and patient population, to average also metrics. payer

to a the coordinating utilizing with we will expect not a closely single and For we as be stocking significant occur are do specialty pharmacy, we launch, impact them.

long X to complete, months Additionally, coverage determinations once to process. initial now results. enrollment as I'll as patient financial to are our move take may X forms

to received $X.X million XXXX, Orion payment of in prior contract and a, recognized same the upfront associated Corporation. the the with revenue European For the both in by of periods increase Orion our to and in XXXX first the Revenues year. we as and the was X months collaboration. collaboration of quarter for X in $X.X BARDA in and driven of result are The recognized with $XX our revenue previously XX, $X.X a $XX.X the June revenue ended each compared million XXXX, of million million second related federal total respectively, as million one-time XXXX quarter recognition

The company the and for federal million same the for contract R&D to was due Excluding months million XXXX, $XX.X XX, of prior revenue, months recognized X year. revenue in and and $X.X Orion and were XXXX, headcount $X.X X compared for $XX.X change additional prior million primarily to BARDA III for and $XX.X respectively, trial. $X.X to for X X million of periods General the company's ended as and costs TrustTSC million preparation operations. X respectively, associated and $XX.X ZTALMY respectively, June expenses Phase the $XX.X the year. to primary million drivers the prior as same the the to $X.X increased administrative scale-up $X.X XX, and the start-up period XX, the million periods Research million $XX.X increased in increased June commercialization change X the for million million with the The million ended and and of as in the year. and in ended $XX.X for months development and and to expenses June the compared same for million XXXX, compared support

X $X in Additionally, from and of respectively, and fee June cost of period totals XX, operating months Cash XXXX, first the recognized XXXX compared XX, for same June Therapeutics. in million the to net a $XX.X period million for in compensation respectively, June months patents million as $XX.X X license associated cash in the months year. used million for and the $XX.X ended applications These expense the same the ended was for and June patent IP as The for net of the million for prior million onetime XX, of as stock-based million $X.X million prior $X in-licensing XX, include the year. activities XXXX, X the noncash $X.X of used with $XX activities in XXXX, expense Ovid quarter $XX.X and $XX.X losses during same the of to ended months of compared the year. as was X the losses in of compared operating million million prior $X.X to X company period million and ended and XXXX, reported X

cash will cash of $XX.X As XX, and balance the facility. debt equivalents minimum of cash we of operations our required of under XXXX, be XXXX the sale fourth We proceeds coupled expected net the believe into our while sufficient this balance, maintaining June quarter had to with fund PRV when the million.

compensation. includes was projected $XXX approximately operating of noncash to million GAAP to slight providing $XXX BARDA fiscal our stock-based million, which year revenues $XX including our compensation. GAAP XXXX, of with $XXX the range million million, $XX to expense to the between we and inclusive stock-based to guidance expense which to $XXX $X to R&D of be be $XX For noncash reduction G&A are represent a expenses These in million values updated expected million, the in estimate estimate, million and of range be previously approximately million operating of

to call provide turn will who concluding back I'll Now remarks. the Scott,

Scott Braunstein

Steve. Thanks,

ongoing We and excited continued Phase are development program, our and launch the very ZTALMY advancement foundation commercial built. about second-generation the of X of first results strong the from trials we've III our

first the allow to many we'll to can patients streams suffering this seizure and of to to We now you prioritize these continue to is are work from critical more help disorders. open launch Operator, questions. come, the confident call us

Operator

[Operator Instructions].

is Tsai Andrew with question first Our Jefferies. from

Andrew Tsai

the on Congrats the recently. launch progress, Okay. especially

first ask still been phase. be ask past or because a indeed on up? of them many on one few CDD first are have coverage sales thinking just I coming the over the is how form I'd weeks CDD bulk one in we thought treated should curious maybe I patients QX how So about

Scott Braunstein

Andrew. question, great a it's Well,

prescription prior kicked everyone, to fill launch out physicians off the X reminder, we weeks that formally about allow a As forms. forms ago authorization and

numbers bit prior little with X As basis But coverage. more be to will of we're Steve a starts important Steve recognize all, in going we for months I'll those up about and of reminder But future quarterly it and a round the talked as authorization a in talking and the share I'll terms Christy you going turn approval want approvals. to let starting guidance. a a forms turn talk commentary to kind then to to on you we've let get And that, more about, and from you what to patient that most insurance follow. a about little talk that terms out about take expected really to to that's to numbers. bit new we over metric talk Christy, next guys quarter of Steve, you're to maybe, little

Christy Shafer

really, One. really the in past great from into enrollment again, have, standpoint ZTALMY weeks, seen X We and prescription enthusiasm a

every expectation our to that significantly now. We us start payers very evaluate have will right

We several have simultaneously. period, in months near it necessity requests fielding policies But between we'll that medical that be process. on future. get And the this that already. be having we in will patients therapy to again, through think X again, early believe take and X we We'll published

Steven Pfanstiel

this Andrew, is Steve.

therapy, Just defer We'll question. of to the think associated metrics therapy, enrollment well. the metrics other to right kind providing the committed patients patient kind of and enrollment absolutely until end that we're and appropriate to new the on of providing patient we active completed will payer patients But do and metrics cadence. total kind metrics of be provide on as specifically terms quarterly enrollment patient I of we'll through on in get a forms, address quarter, it

on basis. So that we the routine a on look forward doing to quarters

Andrew Tsai

you it a sounds recent just FDA great shifting had the discussion changes. about like follow-up, and gears to Fantastic a quick RSE,

IV latest study more not with as modified the clarity confident greatest, needed the us a formulation bit you or remind maybe so little great? and would be bridging ganaxolone whether a are So

Scott Braunstein

Andrew. Sure I'm happy to,

call. proactive past, the the I we -- our we very we with the as have done mentioned been on interactions So with in FDA. have

a for communications team, then the do terrific those a in guidelines that, this any light by tremendous with our this batches at product a presuming buffer the And on not in we III they've Phase see They we're new ability first changing do, as all And and that it's our We've product gives addition in regulatory a leadership been that do They to right? agency us -- team this and We current Phase new need time, new a that So amount green guidelines, the and us their given products. specifically study. side to our we III. buffer specs very bioequivalent in we transparent fact, about filing approach. sNDAs their why using going a is hit with confidence, study, product, team. manufacturing and incredibly see though have use in the our system of don't point reasonable only using us. reference new commented new CMC to facility reason current product what their to as to want great the system, with be the

a lot a very just absolutely to what So we And in is of commercially viable. new of confidence everyone, have has early on. data the reminder like looked current team, system this manufacturing buffer buffer the

shelf to want don't launch we a However, life. with X-year

over bags period So moving to shelf the in goal final as steps of of ultimately, this the from X-year and process. development IV perspective a perspective, make is new buffer to interesting lack we system X really see life our much commercially a more again, refrigeration the or --

So of I and with have a confident will CMC the very transparency the that factor agency IV ganaxolone approval our the we you, for filing we're we manufacturing gating piece that sharing program. be feel the of not think

Operator

The next Fitzgerald. Charles with Duncan Cantor from is question

Charles Duncan

line you what or interest the expected my and my is or characterize as Exciting see had on expected for by in Is -- first of community I'm terms congratulations Scott as it you both of prescriber with if ZTALMY? initial the what response quarter payer for you is the question a in had first beyond wondering that you team, could community? Christy. progress. to well it great first in launch

Scott Braunstein

I'll few over it just then me and comments, Christy. let give turn a to opening Well,

discussion, think into fantastic feel we've a III study. any that we got editorial payer got I going supporting that. Phase from physicians independent We've published

public ZTALMY. are in the the guidelines also have supportive We use of of strongly that ganaxolone, domain

And think these a going all of scientific so into robust we've got I package. discussions,

over it And we high me turn her let had certainly Christy but speak to so and we had expectations, let more specifically. --

Christy Shafer

look, great a plan the too. high commercial day expectations all give bit comes. for I a think that a it's actually work for we supports when person And perspective some But little of question to some the

the day say community seen of enrollment our of on enthusiasm. be excitement very also high has I pretty been efforts. today, world I'd is we've didn't we if So commercial XX, now, a but the payer remiss side not X had robust that our through with which on that just the of level day the right is only supportive

CMC from have and X We sides of is a testament several work the commercial medical all to that it side. years' business, known published was worth policies. the of really And done the just

at we're this So enthused point.

Charles Duncan

and ahead. good Very go

Steven Pfanstiel

thought too. effort. a an please, that here, I put that's pricing add, critical would a we work, organizational think of just piece into was I the And and lot

think I you payers I think that the sorry, for critically for is question? I And drug very respecting your proposition value as we next ahead go give important these priced think I'm don't the well are -- fairly that the why us patients.

Charles Duncan

be that Yes case. the seems to

to interim that program, -- program So about to But you and the would IV period occur that to of with and XX:XX:XX? wondering if through now the specifically have trigger us 'XX. when second could between or when [indiscernible] on rates, just could result patients interim of and you sense XX a the RAISE that half required is moving regard possibly and the obviously, give I'm just

Scott Braunstein

great a question, ask way That's to the Charles.

the the trigger I the flexibility pulled FDA, that to discussions think importantly, interim. most - the us with the gives on

running now would protocol several running the coming importantly, I We'll up knew May it we get like up timeframe. vast full initiated full We now had feel year. really have this the we've take the We think at year. in of or most amendment currently. majority sites and and -- to sites weeks are trial

I decision we the by important overlap. going about to we're organization will really with I TSC where quite months how we as some to want make we'll And year, next much corporate forward a next there our sites to enrollment enrollment fourth study, yet, the early what looking not we're we're And a backup as And think the decision we'll September we monthly few study And -- I Canada to as we're we of studies adding studies. feel winds think, -- on have of when do. both of and back and third so, really by the the terms us in anything X the But trends in quarter additional from for those studies creates stands an on study? just add well. the an the we in Joe, this patients for as what's year-end, commentary where to this timeframe RAISE like to our think to having monthly again, a and want by for you really make going case, flexibility lot be strategy. have enrollment, be of stand in of can right do to investors as clinical opportunity in overall the about terms for

Joseph Hulihan

been that a the Yes to positive. boost of out some extremely confidence recently to implementing And there's sites amendment enrollment. and actually, sites on protocol response lot help them going to that's I've that we'll of be part the is the

the be end in going. by that And year, the we'll of so I'm very -- able are see and the a confident trends how boost this like Scott, enrollment to

Operator

The Wainwright. with is next from H.C. question Douglas Tsao

Douglas Tsao

medical just what launch the raising in into about of and the year plans for at I'm curious the your meetings remainder the year, the particular, of there? move and you through the as presentations just are ganaxolone, profile AES and just And rest

Scott Braunstein

team talk Joe over the side, to it on then we'll talked happening scientific with your about give affairs update. -- Joe to and Alex's a team, Christy commercial and why don't pass about what's you

Joseph Hulihan

Sure Neurology in have to be care an there's Epilepsy presentation status the posters. And -- then in submit a to on on the video Pastananite submitted page, in additional author of clinical website presence his We're a CDD X on epilepticus the Society guidelines see ganaxolone AES, the Neurology well, posters, we X use international X Child were an publish meeting. glad presenting corner for its the ZTALMY on recently discussed while on topic we've society American article lead other were AES, conference experts. published going its we'll June that. Neurocritical we'll Lancet in CDD, to CDD epilepsy where in on abstracts mention And And in at that neurology, September, Society to clinical for consensus X chose AES. Frontiers the in the we're of also in abstracts with I'd we in at of X Salzburg corner on late-breaking paper, treatment

should ganaxolone asked respondents yes. All CDD for of when the offered be whether responded,

we back publications. the presence addition getting opinion to have in to overall it So was a AES, unanimous a expect about ganaxolone. using And big there

Douglas Tsao

Just as an follow-up on.

Christy Shafer

Doug. Sorry,

Douglas Tsao

go no, sorry. No, Christy ahead,

Christy Shafer

from Yes, perspective. I'll jump just in a quickly commercial

of will kind so conference our is that coming this commercial have out AES for year be party to going booth. that imagine, robust can you As commercial we're first pretty including ZTALMY presence our a

as significant will We data provided will theater also and review clinical have the KOL by you of really by top a could a from way imagine, that product a commercial engagement team. then KOL. be And physician

Douglas Tsao

is people there And I'm focusing of Okay the what engagements, generally what's commonly just on -- are great. the ZTALMY -- then Christy, from I'm most if curious, questions product? asked of clinicians? curious early is sort the just sort the about accepted there and just about awareness And of

Christy Shafer

question. a great That's

push-and-pull with going a that is we twofold this is are on guided community CDD. In are XX% this are with this treating living the think strategy. XX% kind by children to of for with I families are that and at the physician know point. families guided CDD, of focusing physicians that by that caring be or and We

deserve. you And be kids access. process their major arduous get to talk these so quite when dealing that an families now, their families, need medicines can for and are Right concern multiple the the kids, these they about medications is and with it

ZTALMY sure a we that's and that that So appropriate access One robust ZTALMY the make program that is the built very for medication. it seamless is to why gets anyone

They really I to already this to understand talking want they've could they an than you're physician what action. be this patients is think previously treated to how how want the additive potentially see about they're if presence, and effect on. how mechanism of our Now different

basis. are those pull, address a So push in we X field and the and things questions major on kind daily of that the

Douglas Tsao

time NDA? shelf the Okay to -- life Scott, terms or later can formulation in at of you IV stability that walk be the stability one Would the would through ready you be X-year of us X think incorporated into data, great. maybe filing how added on that quick data do would just that And -- on? the just, years a be

Scott Braunstein

that One, a the question, time have least quarter have X months too you vision. more we worried would that the data remember, of big year, showing of data from us to which we're used by begin about. stability give model. reason when the at And case. least we It's that different data so have and in the right? file end make X-year manufacturing the are strong in not stability And we're to sure you a year agency want things. we've we'll data So of -- accelerated this we at accelerated a formulation, will one already run Well, of We've we few the have typically, with fourth the going of that have that new time filing be models to this NDA, But hand. a NDA would results. that filing, great And with X discussing have year that on ahead very which the is also we've we tested Doug. with agency the -- the a and that's the by forth stability of some back batch

will as next And cetera. discussions stability filing so et shelf or take having proxy data as example, agency they with an X-year those the be months, accelerated we'll a for over life, XX the our accelerated

that minimum a the would right buffer. would different shelf amended mean give use X this can the logarithmically we now, us accelerated different would are really the which plus. actual that I months solubility few be approach, years curves for we the can approval, XX a life occur, again, have I to expect of of data, strategies So time of having or quickly new

don't pretty major got a we've of as for early the a convincing all data package so at it by And time approval. concern and see us

Operator

next the Thome Cowen. Joseph question from with is And

Joseph Thome

TSC the of on study, you bandwidth terms make I are point? make are that the go one look and for to be you guess or first take to the some and to a into able in on can help see the of coming of between unblinded decision look to kind interim enrollment really that between patients that study Maybe for readout criteria when just at it just decision or that data on RAISE to decision now the Is going what's going the RAISE you?

Scott Braunstein

certainly I of data be I can -- it's course, talk it's would now, right be say, reviewing that corporate we'll be blinded about in think have sets. that. going Joe a decision. we But to it, but place our DSMB by driven a that's we'll to

And reimbursement haven't the Joe, very the we really flexibility. don't about I around we'll view into we trends, now we from is by the se an drive course, the started if the program feel and commitment decision. of again, study, the gives so, data going the study -- this to launch the corporate II. this? based TSC. asking our do see our blind we to endpoint advance, can't to any key RAISE monthly this This power in is be agency lot that would us affect secondary of in was conclusion we And to I time have with that's ventilation, do know we choose to good allow the at that important think really interim, we are all an difficult us look my really and make hospital so really time we to talked environment. be But a those the run And can changed hit where date we decision the to what think I mechanical certainly, well mind, What's of factors enrollment this XX% all the my a in incredibly is -- I for decision. this a which per this And think on so is,

and to early mind, decision a my that's the RAISE we be X have what about data this big to now will in how our data set. of we over also, so, double-blind placebo-controlled needs And bigger generate. on need of years trial And launch, it type influence

not I side. But say push but know, those to decision, about on set can't I going thinking don't to all see hard imagine one clinical way So itself. things. else there's a the comment going we're we're a And certainly, if anything from it's want of again, data we're the to to you us to make from Joe, lot

Joseph Hulihan

It's do does they to takes an enrollment interim. independent in it. the studies got I accelerates that has and some time along. cleaned all Yes, The mean as statistician it typically be data go no to

be final a there'll And just substantially about the got so wait But of said, more done, consideration data. the rate would enrollment. that And case, in time enrollment. it for we likely by be in could the interim the it's really that Scott as we

Joseph Thome

to And maybe or you into are doses then do just And you higher LG go think think going on you can much you formulation, a here? Perfect. push the you're look that dose do now to down you study? Are maybe going forward? specifically follow-up run novel multiple take how what be to to forward the able for at to looking

Joseph Hulihan

take that can one. I Yes,

in we higher. a a We is along the now, dosing prodrug. the increase be exposure points do we've promising on to and very way. that and the of and are go reformulation that going. patients the and we single able there tolerate and of there keep early go can milligrams, and individualized the other dose candidates the got dosing, drug blunt lot be epileptic and Runacasteaul we could XXX to Most potential that hope We've highest over a So well to Right seen decision the overall Cmax particularly choices, results encephalopathies. hope lot go is

at allow efficacy patients human if current go to waiting so Our to we're the would PK of daily us kinetics per still the and label it. some we'd X,XXX that -- And caps see day hope dosing higher and milligrams tolerability need data.

that it's don't step ascending to go X-arm want III. yet in against We'd Phase what take And for repeat be single arm see dose few decision. Phase clinical a the Phase and we placebo, III, even with and ascending inform want completing take dosing really we're to the straight data studies to a or IIb but For potentially next, going study path a to the development example, we to weeks, first decide have months then we to single dose what reformulation candidate. with II until know multiple

Scott Braunstein

Joe, Hulihan formulation. able could and/or of to at Joe envelope when I add prodrug the our from started the formulation is our And goal have above back a certainly I'd nanograms was go only a this that thing to that. to we levels ml that per blood least be that reductions And with program critical has been what deliver blood correlate we that has this achieve the potentially and modeling believe with think to and great XX steady-state of reasonably. mL ml. XXX as tolerability. that to that And -- we've think seen per reductions showed quite and nanograms nanograms poorly, XXX steady-state that these see a would can test level I per in what And our we and us and

on that they bit tolerability. as bring day to Right twice really be. formulation. we feel a think focus now, create around graph. levels within is And much in Certainly, that. certainly we terms want land? release we How to our dosing about a is really trough So We SMAs make the data be now where maintaining can levels modified And a we to to feedback formulation. we're harder I think have to replicate it. confident well with we where decisions getting to think in this once-a-day encouraging a about do of should think And able want now, trough and we daily little me a to important may a think I truly I formulation well some

generated is is we nicely program. this line this it's out what like very data -- we my in that So to to exciting of with just in the to-date perform mind, incredibly the

drug strong drive that in clearly But remind our to of believe efficacy. a program, that a believe Marigold strong I drive levels of very plasma higher high I imagine that, X quite IV That is steady think but efficacy. the we this incrementally everyone, and higher be we blood was data genanograms. X blood probability levels concentrations a certainly state, Certainly, has there's XXX well-tolerated in Marigold. nanograms efficacy and in haven't driving between very at signal we proven very everyone possibility a going can are blood Now a

but about good disclosure, we of today. feel full stand the that's So we pretty success the where

Operator

Instructions]. [Operator

Truist is Our Securities. next from with question Lee Joon

Joon Lee

has code Does the this diagnosis the based use comedic of clinical drug? CDD records on suspicion of a ICD-XX for is and required CDD or diagnosis approval of another payers the --

Scott Braunstein

want you X, first do and Christy, take to the to you take the welcome you if second want?

Christy Shafer

the some I'll published a we earlier Yes, that seen second label. very our address them line mentioned with policies very, in early first. is minimum directly I at and each of have

least good a years a patients test of is failure line at would X we that older of diagnosis and CDD genetic So label is inclusive confirmation set AEDs. expectations be with policy our of that -- and some in that of inclusive a X payer

that that less is test. policy policy a published and restrictive that, genetic seen that we've of certainly in have with We of line is that, including seen

all And have will genetic test a we to every -- a handful a think going again, that may few we now, component determination that have published have published of policies. policies right currently, forward So it. be we

is a across be that component from that, little included. likely would used However, highly a United is, knowing of we've seen both genetic testing very the It States. again, that it bit

Scott Braunstein

for where to And our little for just been strong a groups for advocacy That we're more ICD-XX back fortunate an CDKLX. the of as in have Joon, be bit than place. good in clear, made and just push really XX ICD-XX code reminder, a work call, for to ISDR months. on to was That's Foundation up everyone LouLou place a an the

so in is first with was to commercial and their the our field MSL team what's now interesting Marinus before And us our out MSL so been team.

that code numbers. physicians use are We increasing seeing ICD-XX

education that provide been strategic our not on the -- And would want with and commercial like in centers to where would really centers the ICD-XX planning excellence continue regard. to that sure list, finally, we able align if increase, have ICD-XX codes our is from meeting And that with. of additional key and code, track evaluate. invaluable places make that we team somewhere we and we see are else will to to about being that go, how so ICD our to we certainly to XX we has that expect us code we for But epilepsy

that data for use in it's commercial toolbox tool So more our it's a bit really simple of we think complement a reimbursement the sets a team. And the nice for. add -- to little tool to a that will it's the will I relatively

around business. revenue. the ultimately, codes are average or the And implications adults. the will ICD-XX patients seen is what really adults -- right And drives have course, And So interesting us and identified about weight young obviously ICD-XX via for of also thinking XX% identifying has to code important what been now, the that that some that our way patients spectrums. we're it of is about all across the we've

Joon Lee

coded you what might CDD hint follow-up, give quick that that are differentials go code that a top Scott, might to are the want next? you to of as some being ICD-XX where maybe some alongside

Scott Braunstein

jump -- to The is in to would for fantastic tool program look I start think used I'll place today. doing I her start shortly. But let going our refractory June, we the on are speaker that as the sets at KOL other honestly, the commercial in way team is logical mean, to Christy's most data that a We're I work and call, for anti-epileptics patients the think me. team. quite

value proposition talk that a you to have helping we told the I get boost. great ganaxolone. think Joe AES about going we're us of already

refractory busy targets organization to be specific to said of make excellence thinking incredibly So maximizing the with months, you data to 'XX can we with I sales think we number the a sure is think great anything way centers, us that by really I Kristy, continued the right available to meaningful add? to to X we're we're have And the this are vital do in of but got about only sets think we've this we're successfully that that marketplace. that all way we that small The to the going epilepsy our is X what these success along. going physicians. centers we've a want

Christy Shafer

informing we And it's on because a also our other the deployment inform Just further. again But even a had continued great have of we've our of here today. conversation the that such guides used Joon, to accounts, conversations on we're help in accounts, But strategy go data but are use in quite here. one thing some are the being not understand code. weeks, we us small unique and that to team. regularly our our to where that learning across interest It additionally, only commercial team are non-targeted few the targeted educating past codes mapping

So again, we on that even of guiding plan being have very other education further. our use But codes used a also are mapping are the the that learning distinct ICD-XX. we're then again,

Operator

with is Olson question Jay next from Oppenheimer. The

Jay Olson

the all on progress. Congrats

enrollment data result question you you the data? in Phase from you that to received any about awareness as Phase the rates physician on on have feedback seen II Phase talk publishing published can was the related of II Our RAISE III Epilepsia? RSE increased is a And any impact

Scott Braunstein

the we out discussing into the positive. the amendment Yes the of RAISE I for And that's study, And sites, on context because said, changes up Phase protocol, in the visiting again, study these Phase as better. data. with II I've the feedback it in been II has I mean, the the coming the been do much in always and reflects population study get really

patient have incubated Phase X all about amendment, either get And got do intubated the in extabated. of for of enrollment. were do with they we up. I the that when And in intubated the discussions population patients the way. study, the And along came we that but were the because feedback RAISE positive so study, lot one when A to amendment patient about the every follow-up mentioned, bring we alignment I features and the of came in II in they was as the mean, and

-- that haven't framed that's finding, positive it we way. quite that's incredibly so to me, an And and

the forward, the way brought been response And discussions so so where in few up has incredibly positive. the in moving far, in I data that

so and And and population Phase this II will recipe as the amendment, mirror success. great a -- study, that we for again, in see the

Operator

from next SVB. The question Marc is with Goodman

Marc Goodman

little be a pretty look you clean mentioned all Europe? gotten it here able the response Europe, be it's bit, Are have set you'll we from does but approval? to going curious, like it you to get and Just

Scott Braunstein

were to or we're preclinical about terms back pretty the that formally requirements. the with of -- actually joined we'll some the questions of working so by early think We've -- And European Marc, That fourth was the Joe actually data. was all we in talked risk done syringes. Yes, biggest had almost third quarter. the big additional studies We and at a list additional our going I the the respond has so job impact. of -- great in work XXX-day quarter of a looking it, it end on additional --

impact response As that incrementally XX% in in we well Europeans. Europeans the on statistical data rate the to Marc, interesting more Marigold phase the important to is about really get the maintenance some we study. you going know, have are that And

do enthusiastic So questions done year. early the the we seeking we're that the next are I answer can and we have think everything Europeans outcome to about

Marc Goodman

And regions regions? discussions other to you license some still having with other out

Scott Braunstein

this overwhelm term this we're really but have more that our them markets skills a demand off whether table. but But efforts they're support in think mine we Yes, what have going authorities in way patient efficiency the teach And indications certainly, that a short think for have I with region a know now but economics a And the from but be creates so meaningful company tough at to we And us, bringing a that demand, our R&D Marc, these lot China. that a we're will -- we or with most we Japan in and we're confident the geography, number orphan team. certainly, Europe, of one time, I we and work -- We -- on next Orion one mean, forced is the in going do really deals lot in start just I trying and disorder. pretty place feel strategic to lot to a interesting Japan a are to other to some organization. like, strategic to regions standpoint, well. knock CDKLX just to are same, to and And one in a the that regional partner value allow love another commercial continue by This work do we trying of it's economic and meaningful creates because year there's in efforts we organizationally to fashion as and our of a, expect place good doing. bring handle them some that there's certainly us are China will for And we're that in can the drug. think all to and the are the payments the huge another real doesn't I be important as that and players, to things to partner it's year-end. significant would because in some for region I expect measured

discussions. very yes, those -- about So feel we

Marc Goodman

if potentially in the Is even Also, that I still Scott, considered. even necessary royalty past, about something doing know you've anymore? deal. it talked a didn't was

Steven Pfanstiel

the Yes, that I place. We're it contract of And end the at frankly, gives PRV excited we and number really being the this really with in options. critical. that some at of time to in flexibility we've flexibility the options, Steve. look I'll answer look, a is one. the very to royalty still think sale, Yes, feel get bought play. much was like there. mean, that the high We're But with reassessing range, I us PRV

Operator

with from is The Skorney next Brian question Baird.

Brian Skorney

far One. hurdle the is that at you revenue revenue Steve. hoping can pure How average I or from are co-pays the conversion to my sales. mix, a it effectively just-in-time sort the here. see you be to do able pharmacy through going? booking for reported walk what get you you us payer there was receipt demand specialty the as on guess as and And a if prescription revenue that's of based as will much And combined one recognized Is held the through and delivery. actual ZTALMY of the of offset sort Christy I as maybe inventory ZTALMY

Steven Pfanstiel

piece, start allow I'll so the as then well bit and Brian, I'll recognition Yes, to little Christy here. of with of revenue kind a chime in

to sell distribute specialty will pharm patient. to who the will partner We the then

So pharma stocking net we'd when it appropriately and for gross we'll to as coordinating our have We're with expect, expect mentioned, we to be partner. to deductions. significant going sell estimate very account would Obviously, able gross-to-nets I to the closely be we recognize that metrics to not impact. to those specialty them. any in

So or hand. need It from will that. but for really inventory kind time, order that there's of no to them excess not in on far of have any just quite not be

Maybe over gross expectations we'll of tightly inventory Christy it to and now So through conversion to to process. manage the to deductions net able talk I'll that very and be very really turn I closely. think understanding the

Christy Shafer

ZTALMY we enrolled necessitate patients from for ZTALMY in is One, in for would line sparks the conversion it the clean the the start into which what be fulfillment, really benefit naive the One. For and We'll to that's patients. very of prescription a a need once investigation enrollment out-of-pocket simultaneously

Orsini. any need Once patients we will also have investigation, that that for be services So patient through initiated start. no after for process a simultaneously will And for auth need is prior done, co-pay. benefits commercial that insurance or support any insurance that assume the again, then all authorization. prior patients the BI will for Again, limited who

Medicaid plan, months, then co-pay through, are minimal about X out-of-pocket -- So this that that determined out-of-pocket. we need. will Again, will seen And once is think patients X when take what considering the minimal plan any period that that that drugs that, be those anywhere again, one we we X PA average patients, a market. available for think are you've one and we early buying to be who will you've be We think we're new seen will in between will a talking just down patients. we commercial it our for be for for our And to

our one moving are services a where pretty patient with . again, now line under So clear right pharmacy specialty partner

Brian Skorney

that's helpful. thanks Right very

Operator

Thalmann. next is Higgins The with from question Ladenburg Michael

Michael Higgins

to question try and congrats the limit with ZTALMY, progress topic, one amendments just here you to the and Behave on manufacturing. my and again are

focus that are underway evolving there the for of information today? going percent is the ZTALMY the at reach a a have a today? in And if is lot centers noted remind with is evolving ICD of You've states, you're expanding your here, what With launch How could where that You you us looking timing on. you've is are XX new reach how excellence. having -- the of that plans noted? your what what that your outside and states

Christy Shafer

inform I today, certainly -- into the reps need an certain our again, happy that that about reach that's that with ICD-XX increased We increasingly don't launch are where probably has to in that I point data other I used today, that, Michael, earlier take we estimate, think have at personally being XX% X to early Currently, that. in that believe confirmed that that. point. with we to weeks we'll any reach used, we'll the codes have situations think we'll have my being I'm that. again,

accounts, top our we are target being that Again, with both but about again, Excellence, Center community we're from smaller We very we targeting are force that to CDD sales the have that a epilepsy able of centers, easily down centers have. XXX national also today, we reaching accounts. to

Michael Higgins

Appreciate that.

Scott Braunstein

also say, can I We world and today, to a need the right? a physician find is You drug. a mean, help salesperson this family different don't

already. that had We happen have

the was already that got We've see and data. launch the So physician, very has clear to I practice call those the materials that which we're didn't to great I straightforward. think available. And confidence it's prescriptions. very us lot are not that a that years gives actually rep we ability or mean that be right? the is of critical seeing on not and force an wrote nice, but in by job team done last to need exciting for And make sales this for right-sized it's success having a capture that XX% for today, we sales amazing They're I

Operator

The Jason next from question with Securities. JMP Butler is

Jason Butler

failed of inclusion you speak the change those one level II, can of you the said, so with IV have the Phase we associated confidence patients you amendment some confidence But your prior as in there. of protocol anesthesia, on to Just obviously, around RAISE had IV just level the agents?

Joseph Hulihan

Sure II Again, And taken about patients I for patients AEDs didn't we that, have difference in a well, well. in reasons, of Phase that too. who fewer think a IV more lot as yes previously. versus parallels the study we're see confident

ventilator for ICU, couple failed dose IV of other is, or enrollment a AD. a it's to progressing that the one. gotten anesthesia of help AD progress piece second-line as because propofol, They is while get treatment going the The midazolam so after not to the right a they the they've patients earlier reasons, may the protocol. are in first other in because is of IV required And more in of to benzodiazepine straight may sequence That's management. centers second-line much IV whatever current

X requiring to be second-line line patients. agents So either more benzodiazepine just will and X X by capture or second able AEDs IV

anesthesia it protocol The specifies other a be that piece current of IV is contraindication. the mustn't

That -- couple with change now would likely rather of than that next it that the of IV treatment sensitivity appropriate the needs that the anesthesia also -- would sequence increases severity anesthesia be increases appropriate Now treat second that the and that we and since this making the to the want strengthens depends be the decreases considerably. sure earlier investigation the treatment to actually And for it. also chance with because agent, inclusion we're for or intervention in going would besides co-primary consider. the consider not a intervention treating drug, we're It's patients it next very does think were and on things intervention patients. endpoint they study next IV they study

actually the on with study. these strengthening some -- and about the together, in the all of enrollment things further, so, study the terms I'm And we're of effect that changes, optimistic bolstering

Scott Braunstein

the And think assumptions. we've that rate have closer Joe's to going this progression even talked that Jason, talked XX% I'll on XX% already just of as We've we placebo these be is changes add, great. to the patients powered ability believe the around And to lower powering Joe, to our believe to publicly I number And study, point, and likely that are mechanical ventilation. about number drive anesthesia. to general also was some protocol be a much really the because that more placebo we that to we these

get easy either status, So go a their having if very into III being positive I run anesthesia, launch. lowering to on decision going you on going study a certainly, this thank and be about for we're call. drug a placebo and around very late. turn still everyone question. they questions placebo. the only we they right? they're agents, of or aesthetic their feel good up So down crank to about when If status, going potential the placebo it's they want get in to happy That to progress be new our lot on, formulation We're just the for were last overall. the we that excited was trial. super know effect we Phase We've making to got I the take a and our effect

back and to in, you. dialing for Operator, everyone, thanks, day. have So good a

Operator

gentlemen, and you Thank ladies you, call. conference Thank this concludes today's participating. for

You may now disconnect.

Marinus Pharmaceuticals (MRNS) 11 Aug 222022 Q2 Earnings call transcript