Earnings call transcript Q2 2023

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Operator

Greetings and welcome to the Marinus Pharmaceuticals’ Second Quarter 2023 Financial Results and Business Update Call. At this time, all participants are in a listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. [Operator Instructions]. And now it is my pleasure to introduce you to your host, Sonya Weigle, Senior Vice President Investor Relations, Human Resources, and Corporate Affairs.

Ms. begin now may You Weigle.

Sonia Weigle

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

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

Scott Braunstein

Sonia us rich you, and this call. and We've regulatory the well to and the for programs front, our progress as both on clinical positioning ganaxolone advancing commercial our quarter IV as year made oral ahead. well catalyst strong welcome Thank

franchise. believe XXXX report year are Commercial ZTALMY support continued our ZTALMY Christy for The launch million. Chief $XX be growth we Shafer, sales $XX.X and sales key in We momentum will to ZTALMY’s for long-term of of the of will million initiatives guidance pleased reviewing range has to are we as Officer XXXX that continued the some quarter second the $X.X of raising million. a the result,

associated be more Investor outcome sclerosis for a of successful in with September will the an Day important that This that commercial detail see ZTALMY indication one be during significant will discussing on in potential Additionally, XXth. offer would tuberous topics we we synergies. complex, following seizures our

for patients around access to making progress, meaningful ganaxolone the are We expanding world.

deficiency we approval application On a approved support access, to raised team the effort marketing European the technical application questions marketing tremendous XXst, we disorder. respond To clinical and This that announced the Marinus Commission our further the process. during CDKLX in review required global EMA to the many authorization the authorization July recently in UK. ZTALMY submitted for the by from

the Our ex-U.S. community. reach partnerships are our to global central of commitment a part patient

the early reported, to we Middle previously into the and Tenacia entered exclusive XXXX. with in expect Africa available East of our Marinus significant agreement near Biologics have China, As hope in an selected our CDD made submission In in the markets the MENA and region and patients in partner ZTALMY NDA term. distribution for we in progress, has

had has a clinical Raise summer the our of enrollment the for you. on Status right phenomenon sure the has which our turnover has to in move site the activations pipeline, have particularly of top while I'm much work is we a the recruitment. believe time months majority clinical of in remaining start an Epilepticus, second I in which trial a Phase Total with continued update impact completed in many and anticipated, resulted personnel, direction, to in want Moving slowdown X previously been hard mind the to bigger quarter. than however, site continued of This refractory team for many to

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successful trial in outcome. this believe While that for are continue to important it's disappointed right delay, patient population we critically to the we enroll a

putting for clinical measures. study a rate well us creating placebo team and efforts position within expectations, diligent in the sites We will a are opportunity demonstrate utilization by healthcare result a our that multiple benefit across a screening our drive the Raise and meaningful to clinical confident

The organization NDA execution. and filing working future commercial our preparing towards expected an is

of We support to and year. filing the a submit help goal an commercial stopping a Raise and NDA II label sites Raise it Importantly, size, plan allow subset to interim shortly. review and we to later conducted, delay our valuable the Event the study. Raise to label us enrollment impact a quarter European upcoming timing these an launch with II shortly the expansion. our an one for data to providing extension, then trial overview steps is next may transition interim discuss if alternative filing the Christy timelines expected our a the to track enrollment is bottle of This driving is transitioning next open forward and to with criteria, meets to following of which analysis of the smooth as on will to will this update and our and of the analysis The as potential not on improved sites Analyst When milestones the interim month. look during and begin begin completion U.S. majority rapid well to Investor

to Let clinical with and development a me oral program begin trial. the on quick franchise move TrustTSC update the

rates tolerability to discontinuation belief top supports mid-XXXX. been in had We in enrolling the deliver patients are dose global improvements Blinded following our Phase can titration clinically trial refractory and continue to a meaningful line study in this adjustments actively in drive X results TrustTSC made which the expect that outcome. low, and data

TSC critical of trial to advocacy Alliance. One working elements our is is Trust of the at closely with that the the success partners the

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in Both equally ganaxolone to exciting, to franchise can to are the dose our formulation saw the our would belief over that serum refractory this the trial, We program will I'm we've on that for potential ascending are by concentrations first the in Finally, be twice achieved share critical using oral broader our attributes day like and Chief then that that, single the second -- ganaxolone generation We other dose physicians will to of data multiple study population. and an end. titrate and happy I turn this hopeful preliminary the and of of its a suffering to second enhanced Christy study LGS improvement will believe enrolled year to future oral the to today. Officer, epilepsies. track from these target that ability reinforce and dosing solution has can With higher are allow dose MAD to the ascending Shafer. generation provide in and patient be offering our call Commercial to the patients continue use refractory in be oral pharmacokinetics meaningful formulation,

Christy Shafer

I'm in commercial on we've quarter. plans revenues therapy, morning. coverage second patients for commercial active Thank with Allstate the coverage the plans to and XXX grow strong ZTALMY and extending extended progress million our second quarter the the pleased of have coverage we at approximately labeled underlying access and Scott good present positive of made payer QX to the performance continue X.X XX% you, to of ZTALMY grew U.S. brand. product the as with continued end In and to Medicaid drivers ZTALMY. net

guidance out we a exceptional increase and day early million and change to want thrilled $XX XXXX worked a serve. the are launch meaningful of $XX.X ensure patients commercial range we revenue ZTALMY successful passion in million entire demonstrate performance of day to I the for to they tirelessly bringing to With team for who's thank dollars. to to the of ZTALMY, our

The XX more a Vanderbilt Montefiore new is families. the International the which of U.S., has CDKLX and centers of for provide multispecialty Foundation of reflection centers to bringing two that for need COEs Research established direct for total CDD CDD a excellence at care

with collaborate Centers access to Excellence to them community continue and they the closely connect our to of HCPs We treat. patients with care helping maximize local for

executing addresses potential on the This continuously to phase team seizure focused As of invest better start, that is each identification caregiver journey, community, the launches, care needed patient the key experience. as the strategy enabling enhancing identification, standard launch, To data full in patient building two are and CDD Insights in strategy on potential TSC. the more patient its for four in a is we our anchored of party and of to look reach continuing ways. for current increased the to activation third core comprehensive our of capabilities while and areas; analytics. commercial establishing informative future including autonomy management, and we're next phase

than the First, we've to cases, see CDD time ICD not the of cohorts where gained patients coded CDD on look to beginning testing, genetic at misperceptions both to a we providers our number accessible who to last In The with And This treatment patients insurance CDD alike the which been call patients enabling are care. this visibility and into widely call education known our with and providers continuous in ICD have improvement year. identify under patients these with and on CDD option focusing of continuum could genetic delivery of we're meet message therapy targets as appropriate nearly XX they coding. and or second, most suspected coded reimbursed insights code. diagnosed driving yet plans. XX two expanding state, patients times includes points ZTALMY are and for dispelling cost testing, in is ensure with are reps and us disease access for we're

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commercial Epilepsy for American Annual forward as underway Meeting look second initiatives. Society our to we is planning these Finally, where a showcasing organization,

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successes With our commitment programs. ZTALMY and look to building suffering patients Chief our to refractory discuss foundation, of new rare innovative the forward deliver disorders. call as I from our Medical to Hulihan hand seizure on to early to I'll options now ongoing epilepsies Joe over development genetic launch treatment Officer the our

Joseph Hulihan

Thank we to you, If criteria, Starting now my the of plan refractory IV Christy announce provide made of analysis with meets Epilepticus, ganaxolone in programs that and interim top time. and the first protocol. in expect overview predefined hello, pipeline results progress XXXX. development quarter line mentioned, to we since its pleasure an our the as everyone. Phase an substantial It's Status Raise to stopping of Scott our we've analysis at of status X conduct first the trial

headwinds fellows among to and recognize top summer first turnover these trial to and with have tackle staff. that In address new slowed Raise research study transition the virtual time of many the in challenges, other States, is is patients that education activated first pleased busy results these we've and July continue recruitment, of XX institutions onsite have Raise three residents already in sites We factors the and United the share successfully industry quarter delay recruitment, believe We these line and trial centers our clinical affecting the year for resulting the to a across and our and alone, are study of champions Australia, To our supporting Canada. at enrolled we XXXX. countries new Marinus to through in June Canada. then

by and to dedication, encouraged look motivation are the and forward Raise the enthusiasm, site collaboration of to complete We the study. continued staff

complete double fully focused on activations extension. for of to final at the label the point Raise the month, site We initiating expect completing which this open phase the and trial will be team blind

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I'd resupplied buffer integrity is a formulation conduct. a operational to does refrigeration monitoring IV are with the safety of on study. XX-month shelf new review ganaxolone. require citrate now committee Now and being have expected updates Also, and like the the new of provide to life. the few formulation Raise sites trial to This data not met recently information

they review, study continuation this Following of the recommended without modification.

we reminder, we endpoints will that enrollment drug key two are interim. participants patients. the study thirds of of co-primary There endpoints be two The endpoints significance to it a agents no anesthesia on the and SE conduct interim need reported hours drug to study co-primary is endpoints. key initiation two two hours. hours. criteria, XX stopping plan secondary with The at with participants are analysis IV an achieve analyzed are and XX XX progression cessation time endpoints meet minutes IV As both to and proportionate secondary anesthesia proportionate and progression XX status or to for no to within XX off for To where statistical

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those are We the will those continue that to X. X. from mirror gives and X characteristics closely This similar study entering Phase monitor have the seen Phase us patients baseline patients results Phase the of the enrolled from to that in largely optimism

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have you EEG reviews we -- our further detail the central on well from a and on the an with eligibility. interim Day information analysis provide reading will At addition, Investor In upcoming we validate as to study an demographics patient patient basis on independent as characteristics ongoing plan. blinded group baseline that

Our to of interim analysis, for navigating refractory seamless in Raise trial study and activation quick begin this a other and expect a to expect a Epilepticus The registration later status to portion would new following we study Raise site meaningful year. team trials Raise enrollment II done our we II transitioning By process. in sites regulations progress. Status in great make clinical a European successful job Europe the continue has

Scott as Super Refractory mentioned, emergency ganaxolone Epilepticus Reset interface physicians cohort we've established year. in under or of Epilepticus, the request and SRSE. IMDs study first trial Status initiated we're expect the complete upon by to Also, to to Status for In addition, the screening end supplying

extremely cases a new treat to specifically for dosing in than developed study. we designed Raise these one has team like the rather using a paradigm the difficult regimen, XX-hour clinical Our recommending were

with regimen, patient most am all a So treatment. I responsive off to far, been three three a with with new treated poorly that's one to condition resolution anesthetics new their including or patients report were status. Norse, onset the have of of SRSE pleased the Status Epilepticus, weaned IV Refractory patients

can to trial the we we're X track next sclerosis global data patients tuberous middle Moving complex, that and year. in actively that remain on the enrolling I for franchise, Phase TrustTSC oral report of our by in

expected discussed, we By in we previously have address than titration lead improve we potentially Even titration, a efficacy for we've and tolerability higher As TSC schedule. few schedule believe we somnolence blinded the a titration well. Phase in though the developed and our Phase to of seen rates study, that had X our to rates initial saw slower using [indiscernible] new reports X. as can have low discontinuation study confidence bolstering the date, remains of we

is which enthusiasm are We also collaboration in as begun encouraged and enrolling research investigators. region patients of level that sites report pleased China, important as visited high from the partner, us. for an engagement well some of expertise the Tenacia, of the and I'm to I TSC recently China clinical our we've by in their study

Finally, our initiated to in enrollment turning dose development second study. product generation ascending multiple efforts, have we're pleased to our

to year the we of We're we've including far, support the XXXX to IND the pleased first data quarter appears of MAD study. from anticipated with design a clinical Extension. study Meeting application the also b.i.d. at the in the Annual continues so will of plan preliminary with advance submission Development of day. to Open profile parallel, the Label seven that targeted pending expect results an of Society program quarter of to of by abstracts for ganaxolone the We Epilepsy dosing pro-drug the feasibility confirm finalize Marigold end. syndrome Lennox-Gastaut In once pro-drug American in presentation two-year December, that XXXX. also submitted share for dosing glad fourth for We're data the

catalysts of I'd on patients call programs to now focus our months financial CFO seeing provide year. look could successfully forward over refractory hold like and turn the suffering executing data and XX The a who on our we'll Pfanstiel, horizon on next impact COO, will to Phase conclude, and I continue the the with from remainder update. To to our work efforts throughout to have families seizure positive you great numerous the Steve the promise disorders. with our X

Steven E. Pfanstiel

key our XXXX. I Thanks, and Joe pleased our everyone. details business. to am the of quarter provide for I'd to financial able results diving of the financial performance, like into few for morning updates good to share second Prior to be the to a

XXXX are to operating fiscal to increasing the increase an we to net guidance I our to be The million, ZTALMY the in end of and guidance. projected the net prior update represents pleased For which revenue, range guidance $XX.X revenues higher expense with million the $X.X range. our year am revenue on provide the million $X lower an end of $XX now million and on of

execution launch. the mentioned, our Christy the of reflects increased As continued guidance strong

on the of million to our million, based commercialization cost of investments reduction revenue, range of The and We which BARDA ongoing the is to of and drive range to onshoring are be of $X revenues which of we guidance the $XX and ganaxolone prior range GAAP For expenses, million of we and SG&A $XXX to of $XXX in R&D by IV our prior development. This million is also guidance, an ZTALMY compensation. $XX in be a project result be activities, X non-cash, expenses management inclusive a driven $XXX Phase continued our increasing focused million to increase $XXX guidance approximately ongoing million increase to from the now from million to $XX prudent is now efforts API ongoing with stock represents to operating million. progress $XX our and trials. projected million, expect

cash credit into that and will equivalents, million minimum our of of fund XXXX second agreement. of required cash maintaining cash, be short-term the under expect current our the $XXX.X million investments to our inclusive half We operations of balance sufficient $XX

milestone year patients throughout EU ZTALMY marks for of approval a CDD significant Europe. The July in the of this by

for of obtaining there, has ZTALMY announced for required Our launch processes commercial in countries. approval preparations the it including the recently pricing reimbursement engaging European begun various Orion the and partner

environment that While label of as brings in a challenging evidenced patients, two-year profile and safety Europe, data. number CDD ZTALMY be the can durability attributes of differentiated for by including the the reimbursement meaningful we believe open effect,

eligible As commercial sales receive under of EU with approval, and a royalties result and to collaboration milestones also is our the Marinus agreement Orion.

reminder, a the digits under range rates low As royalty double the to agreement teens ZTALMY. the Orion upper towards from

into move results. now I'll financial our

For XX, and product million primarily the the periods API second product million quarter months quarter three We by associated in X.X sales, in year. of with increase million and X.X X.X XX, million driven revenues million onshoring of XXXX, the six prior as Separately, of million launched June which startup same the June X.X X.X X.X activity and of ended initiative. The ended for to revenues the of compared the is for for XXXX. ZTALMY and XXXX. recognize consist three and of months BARDA six revenues third we was These recognize XXXX. our

headcount. million Research for and expenses in increased RSC three with million six change onshoring and June trial to compared TSC months the due to million increased development year. million and XXXX, same XX.X increased costs our XX.X was were The and as activity, the XX, prior periods XX.X API the XX.X associated for year-to-date effort, clinical and ended and

the by of reminder, a funded onshoring R&D increased in first the BARDA so API XX% half As approximately revenues by XXXX. reflected expenses that partially is the BARDA offset increase effort the is in

XX, and for Interest X.X the of three The June administrative and the U.S. and those XX.X increased and year-to-date overall primary The headcount million as increased same less million drivers million year. months in was yield million XXXX, equivalents, change launch were XX.X million increase ended in and increase X.X by the for on on periods as to driven and six compared in the were the income XXXX year. investments general, related ZTALMY. XX, cash, of cash same months and for prior basis a million expenses six for million compared X.X income the periods to interest XX.X in was XX.X June three the the than to and balances. short-term the prior ended Selling,

and in under an Interest expense ended interest ZTALMY compared XX, the XXXX to a company income taxes XXXX the March June the as in credit and XXXX, a for was million increase is the XX.X six Oak three for Tree the periods additional by drawdown X.X million The FDA X.X the million months to for six agreement XX driven prior and million million net approval XX.X X.X year. of three X.X XX.X months ended loss million interest million upon compared year. June The same before XX.X and non-cash and the same loss of our expense related for Sagard. in for revenue and prior reported million financing and with of to net as of million periods XX,

the in X.X million X.X to for the one-time prior XXXX in X.X our with based XX, associated Orion used six include months million for expense of operating of three in as the the of million XX.X ended six and totals June the million loss These in year. gain note, Cash the of activities a million cash portion related payment prior periods X.X and period XX.X to compensation compared ended includes same months also recognition XX.X operating of a XXXX the the compared As for upfront million same and as for non-cash stock the year. XXXX June to activities net XX, partnership. million was used

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

Scott Braunstein

With of for growth as against in patients to a seizure milestones with Marinus are a of rare success our continued foundation Thanks refractory options and Steve. treatment leader drive into near-term innovative we epilepsies and confident the solid expansion, to next execute for in ability genetic disorders. our the stage development

in September in commercial many be we'll I deep person. a TSC. On mentioned, hybrid to be and able join Operator, We for pipeline the there and XXth, New we've call open of to look a Day and As in you you now York into questions? our for forward and will City you dive Analyst can seeing hope hosting preparations Investor launch RSC

Operator

[Operator Instructions]. you. thank Yes,

Your line Abrahams first of Markets. from the Brian comes RBC of question Capital

open. is line Your

Brian Abrahams

guys, continued the quarter ZTALMY. progress congrats Raise Phase seizure you're Hey, the little a the And the has good actually, more morning. the of like a the mentioned lower burden in could being burden looked characteristics. the It seeing down question with study. taking and for bit and -- seizure the nice the I last baseline on gone looks since that you my versus expand quite all to Thanks ask cut the if bit on it like characteristics you X. wanted baseline

those bit, also the of So Thanks. if on differences patients the actually I how with study, be that more along And in features I'm IV endpoint? the how progression I'm fits well? to there's prominent with stringency placebo patients so potential rate then any motor has you've come guess criteria, that maybe what a anesthesia the could to dovetails the up in the of wondering enrolled as and screening into the maybe in recently types lines noticed quite proportion impact wondering the that also

Joseph Hulihan

Hey Joe. for thanks Brian, is the question. This

guideline mainly response. baseline is American XX% identical on remarked burden endpoint response or the the -- while status and Phase that this for mentioned to yeah, diagnosis published the is believe either drug influence I think, Neurophysiology that by don't X. seizure baseline in from the as Clinical it's going a to the Society changed placebo And don't of and inclusion driven both severity, placebo actually I has as The which we I either be criteria, seizure I status. burden, for

that the complete the the difference study high in in or regardless had of we severity outcome, burden So of baseline the make all group, saw persisted the a that status the that Phase in but status dose of X we was their something drug seizure was patients may resolution virtually stopped. after

motor outcome be So could the effect with burden, status motor going baseline to nor features, motor status either think motor prominent focal motor type that features. or that That don't have anything, any bearing non-convulsive is be The either. have shouldn't could any of the on features. activity seizure any I

have to the it's we patients in from haven't don't looked difference. to a differences the there at baseline characteristics, that been whether think I think time, So have going really able there. of the those I make characteristics anything see And been change over regardless any of

Brian Abrahams

Got it.

[Multiple Speakers]

Scott Braunstein

were it Brian, the high where of seizure we at very the seizure the was, first too X was I'd kind burden I data, burden think is right. though Because, how the thing Phase to XX%. add only the similar the as surprised looked

XX% and down That surprised So by study that not come really our overall, expectation be we've the somewhere it. number into XX%. expected going we'd that we're between was and to

Operator

you. Thank

question comes of next line from Joseph Thome Cowen. Your TD of

line open. is Your

Joseph Thome

going for Hi Maybe is be taking morning. what dosed. my this patients goal question. maybe XX% confidence you in And what the gives the then there, and seeing good you updated the XX to thank metric, of updated just that QXXX the are timing, interim over on you're getting under

you. main a a reason is that screen X? patient what's the the seeing maybe in So Thank why Phase failure you're

Scott Braunstein

Thanks, Joe.

then for and timeline a first bit the pass I'll little more question on it Joe over take to me color. Let

we beginning for investors, of basis a the of steady were Joe a the on us And activated. and months. first since our sites met more seeing enrollment are the doubled the of several year, As monthly the at go of almost now timelines the double we we open study. January very lot on if That half the completion look in year, of confidence back numbers. and with gave number that Actually we've and the than

liaison throughout right all really the feel has that in we've his Canada, teams, mentioned science impact. of now very expected we Joe degree had a as place we've think to prepared summer. clinical and high medical over the we U.S., be underestimated our such a ops slowdown turnover confident we We in saw surprised as different teams, a really short-term were So remarks, hospital, And our to the the study but that we Australia. the talked in I that sites

we'll are to timelines see if we can quite accelerated QX of that and to that our participating, enrollment. for using of we back normal levels their certainly, confident hope projections baseline up. terms So the the we're are of excluded. normal year, level would some activity half move in We that relative see the now sites we're first And the of that

Disappointing resources over short-term the But a the So lot in or I'll us second to a on doubled it talk a it's be from Joe, this really study. about study an ZTALMY’s reason nine last summer. we question. have We've to our perspective, enthusiastic. turn of six overall to on confident months hit. bit you to little more

Joseph Hulihan

Yeah. Hi, Joe.

come potential responding which end looking of think we up and for right at hours. that I one anesthesia perhaps reasons XX the another been first say within screen to enrolling Not have gives in the yeah, second the then responder. major the study, us don't the actually AED, intubation the is on patients, into patients when failures So within or confidence XX but IV endpoint, hours endpoint, necessarily placebo a could

And being by main the is screened and criteria patients far the in that's X, Phase the think out. stringency of that than so, reason are greater I

Joseph Thome

great. Thank much. you Okay, very

Operator

Please Tsai question ahead. next from go of comes Your line Andrew the Jefferies. of

Andrew Tsai

Hey, good update. Appreciate morning. Thanks for taking the questions. our

is you quickly, So interim what And percentage on to latest we patient line QX? top for this should what the your reanalysis an been individual about based has he XXnd just in line announce wait be enrolled, reanalysis, release, patients secondly, Thank when release thinking as or baseline will the you. on it have top data the press really of is whether think then the could on? itself

Scott Braunstein

second say a my would today generally question Andrew. We've bias patient. Thanks, that Yeah. the the top would and line I we is lot gotten

we given We've really timelines cut. recent investor baseline I a owe our last talk you really but updated This weeks. was have the to characteristic disappointment numbers number, bit that are September. going few us. the community absolute to not the data, going little more in it think a we to shifted is and We're to that the a to about that give

I tuned. anything stay there, Andrew? miss So Did

Andrew Tsai

Nope. very All clear. Thank you, set.

Joseph Hulihan

now of was a amendment. think talked enrolled took really I about really, a questions data been get at their with sites updated patients with protocol this to that took we've made important say, effect amendments you IRB's. year for of protocol and show I number have thought through those last the while since to large to summer, We would importantly, really investors lot a the we set gotten we it all that And end, as it

Andrew, So initially I it's really this the that critical I a that X. is would to Phase that's call well, there as in we intended think mixed it study the a would really piece population, we argue population although

Andrew Tsai

you again. agreed, Right, thank

Operator

of Fitzgerald. the Your Duncan of from line Cantor comes next question Charles

open. is line Your

Charles Duncan

commercial on Yeah, all, and on I question to two-part Raise the a and ask first I. wanted quarter, approval. congrats progress good Scott of I quarter you morning good team, a had EU recent that of regarding

assessed DMC that have the mentioned color being recently some continued enrollment. the regarding You and question you Could observations trial. second then this that the on And assessment? DMC TrustTSC recommended met were provide is the that part within of

in you're You mentioned discontinuation lower are? rates and blinded color rates. give topline basis, us what on you Could mid-XXXX seeing lower discontinuation some those Thanks.

Scott Braunstein

to want DMC? start you Joe, the on

Joseph Hulihan

the for analysis efficacy, right with see time? Sure. they that an looking interim and in at there's imbalance obviously, to the look one going And indicate if just safety. drug. of at When And now, really look They effects at. TrustTSC see could they data, I'm repeat the more DMC data they'll study, safety was that but terms Yeah, all safety. adverse looking at they're sorry. the convene at unblinded that's something look they you on

Charles Duncan

seeing, rate rate is discontinuation Yeah, the far? reduced and mean, rate helping great thus in it you're that terms I the what sounds what discontinuation is reducing but titration of are the -- really you

Joseph Hulihan

terms Well, I single the digits it's it's extremely in low. discontinuing. patients mean, in of

mean, thing basic adjusting the think from the I we manage of terms very there to low physicians mentioned, for to rate. answer I in do But discontinuation other that's very titration calls with that titrations too So or effects. that question is reasons few of about what the side discontinuation. yeah, And the

So we're that very titration about schedule. optimistic

Scott Braunstein

again, add, discontinued to we XX% acquired tolerability if aggressive you discontinuation trial. one in discontinuation patients TSC had There the Charles kind Phase the us of rate, of only rate. with dosing. blind double who Scott, the And standard a of And XX% because the persisted throughout thing adjustments. really our are as was recall, XX% In a the XX% this than who study, I X. one surprised Phase achieved benchmark about less saw patient that dose would really we And Marigold, about X patients

very blinded blinded of the Phase in patients much baseline onus sets data a I number effectively more to where that we TSC seeing adjustments now really titration. So rates us confident trust. and what discontinuation a dichotomy are We're be the enrolled the was amending dose in X two for data feel And we Marigold the to characteristics, think meaningful titration than and in appears similar rates, dose discontinuation set. dataset with

us of efficacy giving confidence, the the both I think signal. that's a lot and safety

Charles Duncan

notes. the for Thanks good. Very

Operator

Your next the of of Joon Truist Lee Securities. question comes from line

Your line is open.

Joon Lee

difficult you. Hey, given opportunity thanks you to RSC, Thank the is in would in it arrays, X And Phase ganaxolone why in, differ gives confidence how enroll that been what commercial on mean, it for enroll that taking to has situations? questions. I challenging commercial find our in to patients

Scott Braunstein

patient we add be next X who've physician. value physician's pass a Joon. I'll the over using to different things, than it to A a I few but kick to we're then had be different and want think, versus let me you it Yeah, off if to the color, therapies, to where Phase give to And mind any X, In IV of really you. Christy, happy the And particularly study wanted to anesthesia very slice clinical population the like hospital the physician setting, and narrowed show really any allowed think -- Phase really strict in anesthesia. this IV the we to I we wasn't small a anesthesia therapy. had we criteria of IV that patients proposition enroll in RSC needs multiple need necessarily several because our the where a population ready

think a as we've criteria. inclusion kept this narrow a that of as very, result very I

to driving the that rates. the Now, placebo critical low that's we of flip think is side piece

really more shift that multiple the really that XX X study, So that Phase member you ultimately I hours to interesting will of have ill. sometimes we Epilepticus looking difficult physicians the And believe which study. for are is them observing a AEDs XX would is very is who I Status be -- paradigm we the family me want one the think, time Phase is what in subset here patients, average smaller in with of status to these look retrospect, means two than It's no, ultimate at. think AEDs, enrollment X a at in quite treating we really as in shouldn't population today, and are hours, the

so, makes we'll faster And quickly Raise of and to general AEDs. think And, But of time will complications. start rates, and showing the other in do we II trial those important. with dataset move about drug response the being very more this faster, used Raise brain very the more anesthesia, and avoidance severe all is which which, I importance is the

me powered I question, pass think I think to part we to marketing the think Christy, we People story. the it the all the Raise On critical on choose the you. on did your the we to a why of Raise are very critical. the than And it's answer. get to over time, Joon, one hand, at as other sufficiently I market becomes me analysis interim II ask look do trial. let more

Christy Shafer

come over here. side, encouraged us Scott. bring is certainly this expect through value hospital the about out And to how But Clinical efforts to get prepared that differ We've initially, patients these this. love hospital ganaxolone amazing these the what our from commercial on we really in for about data that Scott commercial Sure, touched the bit say on spoke for Joon, system, the the of as that is and the that thing Thanks. data by think to them. little the what efforts selection patients given very back move thanks of it question. they been can sort been trial we through I have thinking was to and I'd that same that the of cost a progress has and remarks the system, promotional we

on it perspective, will feel that the in directly able is promotional is working affect be wildly the we to that a confident value team from but hospital. again, selection present different So clinical the how we

Operator

Thank you.

of of Partners. Marc question next from Your Goodman line comes the Leerink

is line Your open.

Marc Goodman

talk was There you of are Yes, CDD good you guess number what from coding the Can is CDD you've that patients about number earlier the it was a there about I patient’s the evaluating. is morning. believe number, been that U.S., comment maybe what a the net, number, the pricing where give these the revenue to and the in days? us the gross in quarter just old their just patients was of got what's sense Thank how new number the that you. probably

Scott Braunstein

more both going to numbers. IFCR flip on for our I'm everyone, and to talk advocacy were of code. it XX Christy creating over reminder the the instrumental Marc, and And Loulou ICD Foundation to ICD groups, patient you so just a that really in

us be share for with really we've data sensitive. Chris, had to to Marc. try available the changed numbers physicians and code three about we've So years, ICD you feel to our sources more to that only free actually and But

Christy Shafer

Sure.

the has pediatric of all patients, our dictated that that's population And XXXX patient that addressable number. modeling we is our stay and forecasts. continuing an of to inform really firm So number about on

did. patients, We that ICD XX that ICD efforts very that code numbers. However, with in dataset the our certainly a We confidence would tracks promotional increase grow a had when it. of strong doubling coded usage earlier we I and in early nice invested spoke about on XX the really and our it saw

capturing to is more data team as However, even we that further patients. coded invest decided, of in amounts a

then with so physicians initial patients, patients, United treating size dataset has increasing States. us that that unique actually unique of so these we from target our across and that which has physicians purchased And the we're to getting number led doubled the

easier. XXXX a this them has we on stay So are us firm still that to pediatric enabled patients that bit find approachable now and little

Operator

you. Thank

Your next Tsao of line H.C. question comes Wainwright. of Douglas from the

Scott Braunstein

second Operator, part Marc's I sorry, question. am the answer we of didn't

Operator

apologies. My

Scott Braunstein

the to no, Yeah, problem, Marc. on Yeah, provide commentary launch. additional happy no

quarter million. So were revenues in the X.X

XXX on in therapy, of on big end No saw as the commercial the you assumptions. QX. therapy patients detail, commercial about had actively we at think, I changes in

would average number been some maybe Our four age age old, had could be basis, around that's discounts upside. that had an slight be that a to the average that average where of gross or our upside From an closer seen have seeing annually we year. takes for thought our around around median indicated XXX,XXX a XX%, actually old. little that years to WAC we're projected WAC We though, nine to years five we average net to Initially, more.

is and happen us about investment We're gave as of track after a patients, breakeven we again, that of us that think patients. XX view an this the what this about even, between XXXX. and team by commercial two-year build million, investing a driving these the we really sales annualized this been that's small market we the that window. saying as along what quarter I confidence XXXX continued in us rate confidence access, our and have we've of we is get middle of At much going build million kind launch giving steady, run within kind in CDD XX reps, think all break annually still to our very on to of

Joseph Hulihan

little, asked question I know Marc, there a want I a direct question. you your to And, answer just

that coded Apologies somewhere XXX for ahead one, get that number year. you XXX we at growing now between is the patients. seeing And every XXX and we were operator. CDKLX, well Go didn't year ICD we seeing patients So to data of earlier. we're and when that looked north

Operator

Douglas line your Tsao, is open.

Douglas Tsao

the morning. question. Thanks good Hi, for taking

now about for the I'm of of that the behind, it's Just one far we franchise? the terms on a How generation. Scott, overall question that that pro progress drug. the second oral are in see me. development that you of lot not And curious, thinking asset

Scott Braunstein

the being than marketplace. I the solution more second in I keeping has was always our think in to And depression very expected, least we the know formulation question space. little really we happened all once frank, a our on It's the where that what unfortunately, shown initially a saw that day pocket a given great the bit at early in depression it really to this gen Doug. reverse week, fact the better space. And the characteristics look at back we be pro drugs was of

looks levels the we honestly, a was make It nanograms in majority investment to very moving the aggressively day, blood to strongest But about get to intellectual be vast think property. mind LGS. very with twice be think I almost one given the very should able formulation into looks our up new XXX that brainer to of that a the patients quickly. no to like XXX of and really

take what that and data, So compound X clinical I second half the I we efficacy accelerate that is X to also think feels strongly our in team world which seizure would from And ganaxolone Phase we that quite can only exciting FDA pretty single leverage the require incredibly unique us long perspective. study, based a is package really approval. an on potentially volunteer, Phase multiple we progress, into we see think into of the in X, healthy this to in ascending Phase see good as continue as to for XXXX, me dose to

to I in great from is sure point preclinical of feel strong. exactly I a now, what really equally as little our and about our sorry, is We that the generation for us So I bit this at epilepsy, a we had us be kind look year Doug two drug the a right it'll The move think lot problem take or think the we now of forward at staring partner, a us. we thinking I'm across going pretty refractory really the can -- we're to other drug looks a programs. And we role to and development be second a fit on looks think we're drug look ones second alternative. But it a to great of take will here board may to as exciting not a believe with it face so but… safety new in. the And we going the open are have. this say I'm strategic have that where pro how to into we slot And, it's to expand gen going program, of in. long and that, is thus the pro work in there profile generation I program a and way very success I'm about formulation. better dependent second potential The good really be time, pro answer would in And how to a drug I accelerate how there. from far broader minded

Douglas Tsao

that you. mean, provides that's No, helpful. very some I clarity. Thank

Operator

of Our from Olson Jay last line question Oppenheimer. comes of the

line open. Your is

Jay Olson

event Hey, Raise that guys, next the thanks stopping commercialization? also, criteria scenario? the year, steps walk that the incentives of taking us through can meet in specified next for the and secondary the And commercialization you In for how pre first in important does the question. quarter are just timing

Scott Braunstein

a Well, bit Jay then and timing little secondaries. we I'll about the can let's start with talk the

roughly in -- talk think study Christy incorporate looking launch review will file the we launch. when those at lay out XXX middle would for on really expect for And of times of would priority would put timelines, And our ready expect into that the the XXX, years. you. set will of XXXX. we little file together timelines for can launch and going Kristin NDA completion timing for We But supplemental the QX, NTAP that talk of this the in approval are IV I general, planning into timelines Rudisill, approval, our XXX. that in in And NDA. at we then about think, of the the in And historically middle really that now in more September full the up a we'll September, about to type would bottle for we're expect that three we bit a those NDA lead the we And thought team with program. ml a XXXX. program as fashion our not guaranteed. strong be incorporate we that. talk think the the lot and with We again, a September. With certainly launch had soft all we mentioned, those And XXXX reimbursement we'll And with the we think to us certainly, get in rapid can in that look the NTAP launch, all

endpoints think mean, secondary we earlier. a Are big -- the I how originally smaller the we is is are we we've thought. questions The on critical. this market gotten than market much

to narrowing key and here. field are us I primaries secondaries if key The we do the in really truly the want and right is drug answer that. secondaries key getting we I the believe think -- do the approved. value really us again, are that. by critical this of have proper the we no to the for reimbursement study But the think patients And designed the We're to show economic hospital, we drug. enrolling

those -- to secondaries use we as so, the think can those many to I public larger put to into be secondaries our as of going we're that strategies. the health And publication into second data the our basis make And the a those of exploratory folks, economic exploratory set are reminder arguments arguments. very we enthusiastic domain,

anything complications, that rates some it's that of morbidity, ventilatory I is going complications, really High combination itself and for us two. on? know XX hospital So, to of mortality, for XX has from the you high lots outcome I that, of anesthesia stays typically benefits. to wanted on add a of high patients. hit didn't think be the ICU in low Chris, high We so GI of in cardiac a these to bar longer to costs days show meaningful support, rates pretty that neuro

Christy Shafer

work to pieces really No, now what's just larger by that being did I that is of the is informed right we finished. work note and you important think that

cost to we're spending majority Scott's what now. time point, value where of of secondaries right And are most the And that those is is care driving and book. of coming our so out of

Scott Braunstein

Want to seeing we're Great, conference and of We're to out we thank for forward on September. in do a Raise bit everyone support, to little time a and dive look baseline deep also a dialing many characteristics. you giving on the going thanks, your We'll And call. in, operator. more of look to trial. really end dive, deep live forward we the for you

about, the As go talked numbers. we'll through enrollment we've

of and on as well. dive our the deep to on commercial really a to for a You'll plans doing been team going franchise has we're hear a get The job that. quite bit great to spend oral time prepare

So we look forward to seeing you then. Thank you.

Operator

This conference call. concludes today's

now may You disconnect.

Marinus Pharmaceuticals (MRNS) 10 Aug 232023 Q2 Earnings call transcript