Earnings call transcript Q4 2022

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Operator

Greetings everyone and welcome to the Marinus Pharmaceuticals’ Fourth Quarter and Full-Year 2022 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]

It is now my pleasure to introduce your host, Sasha Damouni Ellis, Vice President of Corporate Affairs and Investor Relations. ahead. go Please

Sasha Damouni Ellis

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

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

over Officer, turn Executive to the Chief will I call now our Braunstein. Scott

Scott Braunstein

the call. continued commitment our of and the team. and driven approval was launch in cross-functional welcome the mechanism Thank of advancing U.S. important cornerstone the forefront action by At IV of ganaxolone in XXXX, and landmark subsequent oral and franchise. potential to ganaxolone’s differentiated In July. to the you, validates our the progress achievements make programs commercial of grow the ZTALMY Sasha, our our our ZTALMY and represents of we approval March, ability Marinus the rare U.S. late commercial of its franchise epilepsy belief to in our

an which on progress, demand will on patient give payer of are to provide meeting this Christy and report our underserved update coverage. robust of including us Importantly, further the full and are first of pleased details metrics launch commercial we sales, needs quarter key our are confidence population. we reflecting

proposition for both with believe critically formulation advancing and formulation the second-generation broader trials parallel epilepsy our important focused We pipeline, of is program value remain two and ganaxolone launch, progress that we significant of important that on oral our for franchise. first made second-generation an including pivotal our piece product rare the a In clinical our IV expanding puzzle. and

pleased are look our X seen program to which advance seeing Joe Phase shortly studies, dosing we data discuss in the have will We forward clinic. through early with and this the

short now In and see we time expanded ganaxolone a a for oral investment very the period with a focused new of pathway franchise. and

on recruitment adjustments developmental X enthusiasm Although number it Furthermore, second-generation IV and met epilepticus and is trending support of that positively in are a by with believe this refractory we've in been made success also to the enrollment the are participating sites. with status track early, a replicate we trial Phase formulation. to growing we RAISE

Boston Tampa, XXXX. and and condition. month, protocol investigators in live are I the participate with motivated need continue amendment key met this from fully the Baltimore been to Just they expected be results and to top this given The life-threatening implemented line has medical second-half highly of unmet have in

forward. for to European the like and in programs RAISE continue registration acknowledge status to other in RSE I would IV reset that press also established II

can While the investments pivotal U.S. all primary U.S. three we focus to launch. and have will begun we trials the our efforts on program that progress, continue believe successful RAISE, drive commercial a help

TSC believe will activations Alliance clinical forms additional discussion the is over to oral hosted Canada, population. and is awareness which to in participated trial, these a current Marinus that site its X TSC the by TrustTSC about first and Australia around on community refractory we this and of Israel, Europe educate working raise to continue community Phase Belgium, we in will we highly months recruiting the in of Italy, the franchise, the to coming move Let's weeks and expected week, use are the panel with continue the in TSC China. and closely kind types Last now with U.S. trial. a One clinical

and the are are first confident tolerability refractory track us key Phase top to CDD, we've And As remain the schedule to We XXXX. we quarter in data the opportunity population. success for attributes epilepsy with that of titration disclose outcome. the line maximal X to a learned in successful efficacy give changes on

in update a Europe. material, in November. XXX list to to on the our the XXX The like day report, of in to choice in authorization medicine patients recently marketing outstanding including number a issues received be in ZTALMY to would expected the contained of major which our starting the including of objections, provide status the formulation We January. responses an in the We I The of EMA ZTALMY submitted bringing the Europe. application material. same day contains this starting list is hurdle of U.S. regulatory approved biggest

the on the a remain to with this XXXX quarter opinion a We result Biotechnology the MAA XXXX. combined in Corporation for and expected EMA, regulatory second our to advance front efforts, the to the include Tenacia trial. The announced with in ganaxalone collaboration advocacy TrustTSC is working committed of plan medicine. CHMP a There for China. Tenacia within moved November partners sites commercialization remains has and opportunity quickly of and the Chinese in of We the global present Orion at its ganaxolone. closely important development we community to on our broad as

also are Development maximize opportunity. to the RSC underway plans

XXXX. additional ZTALMY explore region in including footprint to continue MENA We Japan the expand to commercial alliances, expect of and commercial ex-U.S., in the

second the IV treatment will IV This believe for newly of Christy, We regimen for I hand the to our our in on additional the claims issued status portfolio using related U.S. I received to status patent from Xst our epilepticus for ganaxolone. program. March therapeutic We the IP would allowance franchise. clinical it claims allowed of be patent want and a news. exciting notice some office trademark strengthen our Before to share over epilepticus

organization the has been Steve a Steve ago, share an over agreements know, played He within Officer Operating the addition you of his our launch integral leader now I years the to himself to has taking and Officer. joining CFO. to will sheet of strengthen of nearly planning. as in that and number helped am many as as since pleased important Chief since as role important Chief be organization contributor established ZTALMY balance has driven an our strategic a two role Financial taking expanded the Pfanstiel as on in Additionally, responsibilities

background in Board business corporate acumen neurology to January. an capital make financial in rare her strategic and and development, Audit risk and strategic extensive Finally, pleased announce addition we management. of in and the Christine of and compliance are expertise with Directors her outstanding planning, experience Christine a Committee deep our diseases to the markets Silverstein to appointment board. combined Her brings

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

Christy Shafer

Thank pleased continued to an ZTALMY. execute our progress launch you, we the Scott. I'm commercial provide U.S. of update as you on with on

$X.X first launch, XXXX we fiscal for a the In the ZTALMY ZTALMY generated As third had $X.X revenues July XX, net of quarter $XXX,XXX following revenue ended total year quarter, launched fourth initial reminder, on quarter. million, to dollars million. the full product December XX, the and in net in the bringing XXXX

forms over prescription of enrollment We the XXXX forms in CDKLX the clinical year total trial fourth enrollment XX disorder and quarter received over and XXXX, transitions CDD XX, XX December completed fiscal prescription for naive commercial inclusive completed deficiency patients. of patient ended

prescriptions prescriptions commercial of and Centers from base more continue to large treatment a from coming Excellence, and healthy of of distribution and physicians, naive unique growing We accounts. with see diverse patients than build epilepsy XX a centers a community steady of prescriber local

end made the market of the access. enrollment particularly CDD More Since less. to than prescriptions with receive encouraged forms has were reimbursed within of were by launching these have the with therapy team XXXX. XX-days of we ZTALMY, most patients filled been And or able completed by progress the XX%

payer to coverage both XX lives, determinations close for ZTALMY programs. are of pleased million XX, XXX XXX commercial XX% representing announce from represent government ZTALMY total that plans. has payers February lives, approximately as We coverage to which increased favorable million received commercial XXXX, and commercial including to of

first-half statements We the remaining expect XXXX. in coverage most of the of

and confirmed this medical and patient access been their and million approximately Washington well XX We as as Puerto representing the our on D.C. in an capabilities, the need early States U.S. success CDD unmet believe impact speaks an has as market access of families. for Medicaid as services to patients lives. Rico, all of well disease strength the appreciation and

physicians. a of from prescriptions continue COEs, and local healthy steady large centers and of build distribution a patients commercial see to naïve epilepsy treatment both community We

supporting to regarding journey. community We with patients from pharmacies, their the white throughout glove and received families patient delighted and have feedback approach, dedication are we Orsini specialty and treatment response the

label above dose our that average Thus the structure. in expectations. which in initial titration with have XX% place warrants far, maintenance dose, the we a our our Based the experience to is and us anything not to-date, forecasting seen patient change market upon approximately is line

of above. the two a seen and in have treated, adult adult young broad handful uptake and We being with patients consistent patients

of field attendance. interactions of of steady ZTALMY engagement robust Our by patient education efforts Bureau our healthcare digital see approach. momentum great with supported of which confirms the our our awareness The launch. strategy execution of continued received authentic ahead Speakers' and launch have has websites, benefited from that a significant programs number been rates of effectiveness and and marketing tools, the and publication our the centric marketing strength provider following patient are and the We on being well

clinical diagnosis. diagnostic We of for clinical a plan data, early more CDD ZTALMY to and importance the indicators voices peer-to-peer treater expand to the the filling CDD Bureau add of the to Speakers’ ZTALMY community education need

established, in and grow promotional we foundation well the several strategies XXXX. excited positioned With to in our ways have solid we are extremely

our meaningful diagnostic the in of diagnostic one continue think tools, paradigm determining next we the U.S. families offering expanded providers solutions. steps diagnostic and journey healthcare education, support CDD and inform insights we unencumbering where support our current can of CDD to will journey our and testing CDD and for genetic These to further offer resources The help is the understanding we and area

series Planning to their their features into for These that that has CDD educate stories to with webinar and the loved experiences of providers, one community a for share along also branded experience profile be also and healthcare who meant program with of have to clinical caregiver on incorporated the are can ZTALMY commenced has ZTALMY. what ambassador a planned who families. activate promotion our initiated treatment families

to many We throughout families to committed grow are brand and feel we are CDD supporting in year. there opportunities our meaningful the to ways continue

pleased CDD patients on the for and launch our We are progress we impact making and very are are believe grateful with families. early we

programs. development hand now I'll ongoing the to Hulihan Medical Chief Officer, discuss our to over call Joe our

Joe Hulihan

Christy, everyone. you, hello, and Thank

progress, generate In addition deficiency commercial consistent we compelling and XXXX, of its ganaxolone’s data and mechanism action and two-year label in that patients, to strong profile continued the data potential to made we've efficacy safety to including CDKLX of benefit differentiated the in disorder. open extension highlighted

our studies PCDHXX research. were medical were Over major and clinical of annual the in oral the recently, Neurology Care course the portfolio were in Society, X from meetings, of of related Lancet and data American in at ganaxolone published year, epilepsy. including Phase epilepsy Society. including presented Epilepsy results top Neurocritical results the the Also of Society Child journals, our placebo-controlled of study Neurology the tier Most epilepsy published meetings medical

generally reduction a well to seizure was and to and led greater Ganaxolone frequency median tolerated. compared placebo

the scope the in a and patient If we clinical did of several this the for significance, reduced to concept ganaxolone. study keep you results believe the achieve recall, Though study data important community continue statistical to advocacy not we of reasons. proof about generated to with trials informed it's

share from I'd a results programs concentration. the announce for dosing generation on of second in generation healthy and effective in to our I studies, potential a level to our ganaxolone. developing longer with some final volunteers. saw like efforts Now BID I'm recently dose utilizing ascending range the percentage with up milligrams exposure that to second Phase and exciting linear doses relationship the dose at completed a minimum has profile this to blood development clinical single higher ganaxolone up a a updates of formulation pleased in dose our time a third starting X,XXX we end where encouraging above We trial, a cohort

quarter. MAD dosing in Phase multiple in impairment X ascending the initiated is step begins BID PK clinical at epilepsy looking the form which generalized The tonic of we study dosing, intractable be X. than a defining with atonic, pivotal Phase dose will second will trials. next is some repeat will neurodevelopmental do Lennox-Gastaut to and to a dose additional in be to we'd straight The that move able with our having study rather Ideally, and initial important ranging syndrome, in Along be childhood clinical pursue for severe seizures. modeling, indication

overlap the etiologies this CDD the as in that Given such development potential, to targeting We're disorders in finalization this half of promising We and continuing patient other second where with clinical believe therapeutic of the LGS represents the seizure types in population. a plans need and has ganaxolone opportunity unmet for TSC. year. address

We shortly. to also administration. and we And has a for ongoing. An prodrugs candidate IV ganaxolone prodrug oral the IV advancing is are been development of oral preclinical expect selected lead and candidate both select development

we XX and actively U.S., that TrustTSC our Phase targeting and Europe tuberous expected enthusiasm in data first clinical X site the anticipate the and in with enrolling share for to in sites the in with sclerosis of Moving we're to the XXXX. and in additional screening We're patients, activations of medical complex, Canada, this predominantly high-level top trial are pleased China. community encouraged quarter trial Australia line

IV programs. Now on to move I'll our

Australia. importance the our updated we held of emphasize investigator and educate U.S., and on expanding meeting for been in patients broadly protocol the to study epilepticus the centers refractory We're the adopted at our rates as RAISE for the as participating study in sites the November, mentioned, Scott trial trial amendment In X U.S. and As this protocol. clinical well of has the Canada an to number Phase status sites.

emergency considerable the have ICU. greater sites study particularly the centers protocol transferred the Investigators a recruitment potential of optimism about of number patients other of to amended eligible room expressed the patients, allow from or to to from

where closely trial. We execution. changes site the monitoring enrolling replicating support and at timely working Phase to X identifying support provide additional well appropriate for we for engagements patients recruitment level what's with These and of and working X study can are sites a critical study in to site success trial enrollment, the are protocol the Phase actively

members of site affairs have Scientific responding the engagement about Marinus engaging study addition, intensive and development with protocol been and fully site RAISE group road scientific and to to enrollment personnel. In the maintaining has on our principal and teams supporting questions dedicated procedures. and other Affairs study a clinical investigators

COVID being of sites the in and these and uptick a we're and enrollment on With on seeing on the reduction systems, data healthcare global track more remain XXXX. enrollment an scale initiatives, activated a in impact of for half second

we and RAISE is of reminder, with years based for age FDA, currently a a the be enroll will the expansion that As on The in to of also conversations trial age There XX eligible for is positive potential of study the the range U.S. previous older. patients sufficient expect designed filing patients.

patients However, and the of epilepticus XX expand eligible inclusion in FDA of which pool is with We're enrollment. we our status the be the below there recognize required high the to studies, incidence of currently working that could a to patients further of what support information would in determine support children. age

the we an data analysis independent previously As two-thirds option there mentioned, for have conduct XX interim monitoring the completed the of patients when is committee study. approximately participants, an to

We a move later will with whether we year. in make second determination the analysis forward an quarter interim this will

to continue analysis designed power should on is analysis effect interim a an of fully trial. conduct have affects statistical RAISE it study full a the a enrolled of study, of the for efficacy interim The minimal often the outcome While enrollment. to

on enrolling of a is of of to and year. stability the in continues months. it X formulation We second ganaxolone at to refractory believe for targeting lead shortly. a modified trial shelf the RAISE quarter registration, will have RAISE life European second-half expected we in We're of to share the are least into formulation modified placed study XXXX. that which formulation new expected also are of XX with Registration batches Planning a successfully IV in this for be increased change begin to and buffer this the happy patients product status incorporate the expect stability to manufactured

status X year. U.S. RESET to Phase on to study this in on we the remain begin Moving enrollment track established epilepticus,

of study of eight first and of five status cohort the determine established The for designed regimen will with each from sequential cohorts to to ganaxolone phase each the to include used is the the up optimal patients determine in dosing results next.

late programs expect excited our We trials, complete stage first clinical continued CDD. going as focusing the on this the cohort to of support as momentum of XXXX end our by of for We're the ganaxolone, second well our with ZTALMY generation formulations and year. into

R&D, cross regulatory with studies the teams from clinical, communities. scientific advocacy executing I in functional the engaging our scientific our and and and appreciate efforts affairs

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

Steve Pfanstiel

to quarter good our as am able guidance well pleased Joe, to to afternoon and Thanks, for for I XXXX, results financial everyone. share and XXXX. fourth be the as initial full-year

financing Before results, funding. a offering million what going we completed briefly the to fourth was very quarter touch successfully I'd active of front. into follow-on $XX.X In on our the like raised on quarter, net equity a

We pleased of investors as bring in part are story several the a the to very new Marinus offering. to

which for including on agreement completed future interest sales a U.S. million of Healthcare net with in return $XX.X payments we Additionally, Sagard Partners, brought financing upfront ZTALMY. a revenue ganaxolone

over a of our our Additionally, for commercialization and Tenacia when XXXX $XX million a balance $XXX three balance $XXX.X collaboration cash in which million sheet, to part we These our received results Chinese of in market. a added with end payment them gross existing cash with year upfront the cash from of development as deals combined million.

operations inclusive of of $XX under into our be required minimum the is required XXXX, projected credit sufficient million maintaining the cash balance fund our agreement. of second to cash Our to half

revenues In our million the into XX, and $X.X results. move product of XXXX, three months XX recognized now ended December we $X.X and financial million XXXX. I'll

this of BARDA million $X.X of a three XXXX and Separately, product and note, to recognized $X.X each as in compared consists and million ZTALMY in million $X.X U.S. revenues As XX same revenue in $X.X the XX, the ended prior we December year. for respectively, the months the sales of million periods

$X.X of BARDA range million. the between was our million $XX at and XXXX guidance actual $X bottom revenue Our of of million

Tenacia. of related with We quarter fourth of collaboration associated $X with our revenue million XXXX recognized upfront in also agreement to the the payment

$XX.X expenses respectively, for trial XX development to and was to activity. three the same and and primarily to million Research XX, million headcount clinical year. due $XX.X and compared $XX $XX.X as change R&D ended increased for XXXX The December months million in and periods the the million costs with increased associated prior

Selling, the to XX, headcount $XX.X U.S. the and as the million XXXX of The drivers for months to year. general expenses of $XX.X compared December ended XX and and in change for million primary million million were support launch same and $XX.X prior the for administrative increased $XX.X three respectively, and commercial the ZTALMY. periods increased

full-year the was million below million spend of was were onshoring XXXX, SG&A which of million, $XXX to $XXX the and total expenses and timing driven For management. our our general initiative API by $XXX.X and inclusive R&D operating activities of guidance including certain

XXXX, the to The prior the company loss $XX.X months and for three losses million net $XX.X and XX compared million the December same year. in $XX.X XX, of $XX.X million for reported periods net and of respectively million as ended

XX, for as same of Priority loss three and months in in million in non-cash $XX.X includes of gain sale QX and compensation XXXX. $XX.X of prior for totals $X.X million XXXX $XXX.X Review expense million the the XX As respectively, compared the million one-time $X the These to also a our include Voucher net ended stock-based million year. the periods the note, XXXX, from December and

year. ended million $XX.X of activities the Cash cash was the December in months same XX, operating used for XXXX, used prior period in to the operating XX as compared $XXX.X activities for in

of million For to revenues $XX the in million projecting U.S. range $XX fiscal ZTALMY the of to $X BARDA year to million. and XXXX, in to $XX we be be are the million range revenues

EU in be $XX of compensation. million operating of million several non-cash R&D which our initiation launch by as and we expenses range operating approximately $XXX and costs. to second the is onshoring related trial raised to million our GAAP project of critical a CMC We incremental annualization driven funded API expect investments which to ZTALMY the commercial The activities, market support XX% BARDA, stock-based of our program, expenses $XXX for including in by inclusive SG&A increase to full-year expenses readiness, be and IV of reminder is the

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

Scott Braunstein

Steve. a our encouraging strong are balance transformational for advancing with sheet, the XXXX a development. we second data XXXX and year to and enter clinical Thanks, two product team positive thrilled our momentum surrounding generation Marinus stage programs late was

patients may to expand committed remain we will that and from ZTALMY. always, value to to As delivering shareholder benefit continue opportunities serve

would for open With like mission. questions? to employees dedication and Operator, thank advancing work that, call to the our now our hard to can their I you

Operator

Certainly. [Operator with Abrahams Instructions] We'll hear RBC first today from Markets. Capital Brian

Brian Abrahams

to on just the Hey, RAISE progress. guys. elaborate you're little on (ph) Steve Maybe thanks question. on is Congrats -- guess the follow-up. now could study. you and the guess a to potentially therapy, a what And if seizure my for of pace regards your to then taking Congrats wondering I or [RSE] bit I would patients proceeding thoughts going with the the burden? some regards you was of enrollments had the expanded I on with seeing on latest your to on an Good then more all of starting I protocol afternoon. and to role not of the interim? on whether And how types modifications? sense take

Scott Braunstein

together room pass to a one and comments Scott, and or of me, it's Joe I over This I'll kind it is just I'll time, -- with the in Marinus Brian. this Maybe fun. first so in the a have is Joe. Thanks, time team I'll all two long is conference make

DSMB. we'll our between of plan that fall we'll around time again was really RAISE to the the trends And the the -- a and It year comparison data. send the that of to the when early X data in last gave Phase we do kind enrollment

most And updates think enrollment on to is we're so And as be regular us specifically comment I going active going updating to to this a the can I this have expand, not on patient be continue process realistically don't certainly, basis. with and think to that we very weeks. in recent something any a is see you're we

I to the most think from appropriate to anesthesia whose that those focused being like patients than team's importantly, And are guaranteeing that still in patients I quality And enthusiastic the site we're next on right still seeing the think for type Joe confident more move study. rate a enrollment hard. certainly numbers. placebo or in perspective, very enrolled. as think on really where drive we're patients are about investigators, are expecting and The we treatment low will of the patients, and the study. would a anything going And sites really I call, general the about I both working we drive we've today. We're got that right said feel high generally we the

Joe, any specifically? other make to comments you want

Joe Hulihan

just I'll about asked add, seizure No, burden. you

about patients. X but intubated the the patients into were actually study who patients couldn't were Phase X in there the Phase misunderstanding closely and intubated And X was on Phase that the trial replicates the the trial, so There closely burden protocol in in more amendment Phase replicate patients even the in -- the even far, closely X population bring seizure come entry. should half this more were

The hospital in more that's more from transfer on the facilitate coming will And enrollment or one patients of intubation patients. allow so to ER. amendment that's the thing from

Brian Abrahams

for of then indication and guess sense the that upside? oral that data if mentioned. Got interest Can could it. baked recently hop had you the be all potential maybe a me are us that of Thanks. amongst into you the physicians you expectations use follow-up PCDHXX give back use the there, on in I the label publication Will in And queue. your any And or for I the reimbursement guidance? of tell sort I'll to much how off

Scott Braunstein

number about need to overall. community PCDHXX, just been spoken epidemiology from of is we've open is numbers in CDD the reporting CDD. patients the for pretty who have physicians intractable from different, who suffer a quite although unmet very a We've compared small dramatically the seizures. a Yes, Brian, our only to few similar subgroup PCDHXX And and really there's standpoint,

think So, the smaller publication and and it that We a review and certainly the that is that's payers public the about we at X compendia We as important. think think can I think something data in Phase data the look opportunity. see much domain. market in

it's to don't seen part have We a future. near it story a prescription as prescriptions, meaningful spontaneous in the that But of but the few understand we see certainly, nice know is that these patients, -- benefit there potentially rare who potentially available have retractable ZTALMY. them. access that that's think I from And those cases, important in PCDHXX could to really it's payers

it's So, really nice to see.

Brian Abrahams

That's really healthy. Thanks Scott. again,

Scott Braunstein

for Thanks the got Brian. it, You questions.

Operator

Securities. now We'll hear from Joon with Lee [Operator Truist Instructions]

Joon Lee

taking terms updates to I has of what have and to ‘XX? net? an questions. and And stage provide It's follow-up. can like the quarter in gross fourth the that early And quarter and guidance gross for net been impressive reimbursement the at you're of for in overall the for reimbursement expect rate What our of rate first a thanks we such Hi, able launch.

Steve Pfanstiel

deductions low-XX% it's Steve Medicaid think having Difficulty] I'll net have discount. half in few XX% payments. with gross Joon, We to Separately, expect Hi, Pfanstiel to gross in the Yes. on we consistently right the did of driven launch. be in know XX% for QX. right we discounts we here. commercial gross [Technical copay touch there. patients our approximately to around said seeing second-half a programs expectation. net were by more the piece, still And a coverage. around XX% or back that's last upside the see to We have so We're a that's think We kind really still that's net year, dynamics I of

patients lot as the are simply for don't need through of is copay, they a so One support much that.

The other which have to now piece all State Medicaid simply programs, is do. All access didn't we we

year Medicaid have around kind deduction. we that of stabilizing We XX% see this expect that to in. now gross more So net coming it to

Joon Lee

is, opportunity or to interim due have Thank Great. look, trial And to utility? trying when the you or will rates? efficacy if follow-up the and Basically, to the for an do extend an either trial the understand you look interim question you. the of stop goal

Joe Hulihan

the futility is so efficacy. to Yes. not that point. is for at goal Stopping for for can hard being or primary The the to is stop general demonstrate, potential we stop for really efficacy whether done

Joon Lee

you. Alright. Thank

Scott Braunstein

operator. Joon, -- add I'm I'll sorry,

Joon your Let me to question. just color add a little

the study, primary think efficacy we on we endpoints. our limited really secondary very very both a I XX% powered initially when data We had delta endpoint. about on had assumptions key conservative

interim our that we between to a secondary confident we think interim And have that's delta will one placebo hope now at show be look key into I be than key and that at days our XX%, expectation. ICU, goes that when specifically greater significantly elements in at looking the a of to the the that belief benefit. an continue two we or day an power more is XX% certainly

meaningful it add the for the which to I of Joe's only interim hit is going point, a gives but can we the lot of be to benefit, analysis, critical those think really so show drug. And confidence us up economic a not health launch you and that at pieces efficacy

Sorry, I'll you. to back turn operator, it

Operator

SVB to next Goodman. move We'll problem. Mark No Securities,

Rudy

new Hi, Maybe how long the moving Rudy Thanks. just process? trend through us for so And into walk call can this prescription? you fulfill of talk the for now, Mark, is question. takes Maybe for it about starts my to XXXX? the taking patient on the thanks

Christy Shafer

the another there. we've we've XX the with fact very Thanks moving into QX seen QX, forms new be Hi, the we're increase XX pleased and enrollment that Considering we in then that at With QX me, prescription seen in to -- for question. about this XX% in continued over total or into at QX. progress momentum see that tracking excuse progress, time. and prescriptions QX about

Rudy

can how like long fulfill And provide it now? for the you more takes color to prescription

Christy Shafer

Yes.

to continue for reimbursed see that process around tracking get are moving forgot therapy XX those right with around initially now enrollment right of have to now, QX. through and going that into through I that pleased planned Right XX-days QX the curves note. our we and apologize forward for at Sorry, we and now fact we're fulfillment that. XX% I received days. prescription we very patients

Rudy

That's Thanks. very helpful.

Operator

from Jefferies. next Andrew hear We'll Tsai with

Andrew Tsai

I the we sharing Thanks on it is due to Pardon my but updates. clarification one interim, did all summary, progress. of the study it possible have stops the if hopefully interim efficacy. that you did I completely analysis. hit is question correctly Congrats these for on the overwhelming captured Thanks. on

showing anything. The is was that scenario as of right unknown thinking opposed it scenario analysis, about? would continues for the kind be be the be as final drug whether we should other trend Is but a perhaps to the not may study there

Joe Hulihan

Yes.

just analysis. to by of they efficacy say would we direction continue monitoring if trial, So, know and or the there's committee. at complete won't the the with modification study, the clean not the but we be anything, made That the do about anything do see It won't that of is to If they'll patients, and the trial The show takes direction they'll blinded just would If months at know without results. conjunction in to us conducted they two continue the to data it point, be data modification. the notify stop interim. whether the not about decision XXX then statistician efficacy we'll without a efficacy, trial. then the

said the that we interim. completion. effect don't statistical I And I'd substantial this in prepared really the a It's the to if power pay point think remarks a doing minimal goes on for out I penalty too, it and

Scott Braunstein

would data -- additional why DSMB, or an interim cleaned we should Andrew, those while the enroll label And the within one being we view overall in by as requirements safety FDA to to will that continue the for do analysis just important that reviewed We for ultimately period, and set clarifier, would the time. is of the patients that patients during choose we collect well. period data

XX, five, or to final the would double Let's the portion we on day comes XX one, more day patients company, analysis -- interim enrolled of the to blind assume be the with XX that we evaluate might an choose at to study do XX critical have DSMB when we that FDA and back filing.

Andrew Tsai

is you just for the in XXXX, to kind $XX the what clear. to guidance Thank as ZTALMY follow-up, of so $XX clear. are much. terms secondly, assumptions share extent million can curious Very million guys you And a of Very question a the being baked of and in is raise one I root type the or such on year? end. can the you conservative a this realistic of you. lower and guidance beat is Thank upper guess that it throughout the

Steve Pfanstiel

in bottles I Andrew. five some look increase gross net we've about think as range. of age. be of feel Look, launch talked naive address reasonable the early. full those pretty a that and it XX% the just have really confident over deductions tried that assumptions at in past, It we just when still far. do solely we will patient one think about think reasonable question. number in that to being I over Hi, forward. look But We we're I'll This that with feel the to that's we pretty population, per is script quarter time so is Steve. seeing tight and patients we've our as We we

We're buildup situation. going We a seeing a complicated that lot bolus a patients; patients know these steady in. isn't factors on, of a of a lot it’s have decision, this

drug in There are an see XX% after label more And a reasons. right time going over therapy But expect the Phase then data, of will attrition still build for six we So, patients some other we to that the reach time. X months ganaxolone be call won't Marigold over the kind years. the the standpoint, attract steady of think three open patients, to just two period. will level or are initial with but we early, still so patients for that blood know long where from the very they stay again, single-digits on at the to of vast that using for on annual it period XX% or of attrition the of majority

the of range. of to the $XX So give million broad kind million came outlines $XX that should to how we you

Andrew Tsai

again. Thanks clear. Congrats. Very

Scott Braunstein

Appreciate the Andrew. questions. you, Thank

Operator

with Duncan Charles And Cantor Fitzgerald has our next question.

Charles Duncan

on Okay, questions. on a RAISE taking super. thanks questions all second then on for and I and the are my congratulations ganaxolone. generation had the Yes, really main our progress question

on I or And adopted changes broadly been less not in or there's quickly the do how among some guess, the about population? feel a in whether that timing? RAISE, the to more for relative trade-off heterogeneous just debate protocol patient so you investors And result

Joe Hulihan

Thanks, introduce Yes. to X. Charles. it's I This into going of we're population. is Phase Joe the so heterogeneity far more range don't in The Hulihan, seeing etiologies so think

common treatment a more pathway although etiology, status. ultimate really Than very etiology. the on closely obviously We Phase of dependent is of X. outcome are on final And depends patient

So, next next we that don't thing more protocol the much think that's is more mentioned see And that that flip contributing the powerful treatment IV the the on the anesthesia. change would be to the I treatment, amendment the heterogeneity. is side, likely Scott

going sensitivity of treatment detect and again the study. difference Phase And that also on second in that that in terms patients get I is to consistent mentioned are but X severity, the to, their think, a I endpoint the increase us patients mirror as

Scott Braunstein

lot And I was affected the independent of all Phase in state was X, predictor every the X etiologies. a would world etiology. I Duke Phase that amendments in Joe is six quite -- be to like is a we bring at of X X, and I was general status patients in lead that's loved there about saw was interestingly next treatment. is our Yes. underlying worried major And in see really what said, I we Phase would an whose anesthesia. success. on sure mentioned sites get the the Joe particularly the glioblastomas by critical definition glioblastomas that seeing has making protocol Charles, real think we're Phase that a was X, piece still by why as a of Because little Ganaxolone that evidence status on we Phase five and spending or the And heterogeneity, disease that disproportionate the disease heterogeneity. energy a I'll add step no we're as that of status these And epilepticus didn't. number in

I our team. we're are very patients in when screened. with medical They fortunate contact constant are think

Joe of Gasior, Henri who who the enrolled of is X, away communication really Phase to to be believe was every aren't with far the are many many we are and the going the medical who only one Our Phase aware these calls. one constant those to not and in patients those add calls, patients, many is on comment. the protocol we Maciej our is are in talking from investigator the so Gasior, team continue Vaitkevicius, those of, know them need investigators proposition trial, are but physicians, to of those value X wrote of drug. we you who quality what of we to show of lead Dr. the

Joe Hulihan

at Yes, in score. called other the is thing seems score we they're me severity we status saw what And STESS X. one occurs that Phase treated, be before also mirroring do to to epilepticus baseline and that the a that

number of probably good of So clinical that of on heterogeneity than based it’s more the diagnosis, a status, a measure so factors. is the severity

So that score is so X. far looking similar to Phase

Charles Duncan

Very helpful added color.

if may. to that forward I data, Looking

if of the think terms then in regard a BID additional I'm sense could generation the color with intrigued that wondering that. of ganaxolone size And X Phase on with in second be dosing? any to follow-up any one the you why you the Just for Lennox-Gastaut? really provide trial could

Joe Hulihan

seeing goal that I to the -- are exposure, of dosing. So, the Cmax. AUC with your increased we reason mean to the part dose really and first studies the relative kinetics support the of single Charles, see question, that is increased ID

dose dosing, and some pharmacokinetic seeing the And the we so favorable. very then what we're in a plasma We're with think that good dose. a slower target at concentration. bump looks plasma we've BID the modeled good seeing done maybe from AUC And results concentration single the minimum modeling the a of decay

it a Cmin the limitation twice focal epilepsy concentration. drop so minimum in day. when below would and was early The especially that a gave effective -- And they studies the

We reproduced with comfortable current modeling the the very we to studies the results. so both the similar actually epilepsy In suspension giving one did we're modeling, by and suspension modeling the the fact, of formulation reformulation saw for modeling the in BID. actual focal results that and was

Scott Braunstein

the MAD about do the comprehensive with foods, probably in would study. think, seeing to think what go those Phase my as a be able pediatric curves, X, we important from think concentrations we us we which understanding think will part I just But does the different product actually -- so leapfrogging critical having we Charles, need population. that I choose And X different hope not add, to only two seen. MAD of really blood that to how we I'd for Phase And be when is we've study should target to an study, so to

for active their I So Phase placebo, to similar has very think Phase Epidiolex X two arms, TSC would a but a that seen you've in their study, what X with and example, again, early about arms three days, study.

think we I study ability single some be whether have the or a have regulatory studies. two it do to interesting discussions

I would think required and what a we perspective. be about safety, safety very database are hard from thinking

to a decision. final more So on come that, we and haven't certainly made

than given call we hoped I that that makes study. is from the what quickly to to Phase a a a with move into we more X, if And signal consistent see incrementally that for. better a communicate of MAD this think it kinetics you potential we -- had bit are wanted it

a it its levels chronic increase believers an on really very things favorable we're never product. exciting, program that have this can really is franchise the that oral with of efficacy already seeing before. blood And seen we So basis are higher

question. So, all. thanks got it we it. Hope for Appreciate the

Charles Duncan

good. Thank you. Very Yep.

Scott Braunstein

Great.

Operator

Wainwright. We'll from H. Tsao Douglas C. hear next with

Douglas Tsao

Hi, good for afternoon. Thanks taking questions. the

interim Just improvement terms some of you secondary terms I terms the ICU. of think endpoint in to the clarify, of days of in said the in analysis in

something that for on subsequently comfortable given long the the of be economics powering analysis? the just hit or comfortable this that in additional that that terms more that in you to given interim powering the ICU how the just front numbers patients felt done you work will feel So understanding was have you've able are some health more you

Joe Hulihan

Thanks, This Yes. Joe. Douglas. is

on the we're secondary. hit yes, key So we’ll answer short yes, confident, is that

We point the get ICU, endpoints is hierarchy in first clinical second the that hierarchy utilization actually analysis. and primary, a of days built into have in a It's of utilization actually key of analyses. is the them built primaries one in. that stepwise number analyses. of is two healthcare then the end One health Get get primary,

be So end would of and is if be all and for considered be they it evidence. a will significant, that evidence utilization promotion level will level statistically appropriate and point that a of substantial high healthcare hit

the looked for at that we that's and not powering in confounded secondary. ICU endpoint on is and but so for time may And days epilepticus so ventilator, less assumptions status beds there's the whether And the healthcare to just key be utilization the on. primary, on confounded pressure due by

we on looking lot in think Adam adequate ventilator happens endpoint of gotten time on who's but Germany, we're And days Strelzik very the health the versed on be one. in look have see economics. work at, that we'll will that a also in much about what more and a but as well. sensitive so, difference And I is who's we've to advice neurologist also someone, this power with a And done ICU. a show to from this. that at

Scott Braunstein

about not think first would play. credit, when was I of we at endpoints, where and lot team Doug, thought would terms really to out to of key these a and included literature, in And did on look the an this patients only need lot thing his win who really spent primary see a and with key Aimetti really us I'll didn't outcomes. improvement of scientific helping time we we come about what add, study Alex the something the these powering into give but FDA is the We And we the team. met have

hit example, for at were to end went certainly to before perform on, we interim, an vetted discussion, the very end and we significantly could confident FDA but secondaries and looked this of win the to that to an thought powered XX to about asked we we about when So what and specifically of XX XX%. our still lower, our we primary, the drove team felt ability

and secondary end was spot that XX key sweet analysis. of from primary So those the

Joe Hulihan

XX% endpoint. on ventilator the we interim, at have Even the power time on on that

Douglas Tsao

to do interim NDA you data just look, then the interim Okay. you filing based set? would as quick be you. Scott, full or the still clarify, patients And the ultimately Great. would on Thank would enroll if just on follow-up. be a the so based and it

Scott Braunstein

today. where be one all, basic submit pre and label. Normally cases from as expect been is choose you negotiated, an that that is interim, or do if all assumption agency our it Yes, things like the piece blinded interim would there based only a company. of data, those it data is to on said is wouldn't there of the mention collect the we the so a remember would would are safety have in safety would two the that seen there potential you've blinded We end included But primary has I will that today label. burden, we we it the

continuing of we only not for efficacy number double to So, are should patients on we be trial expectation. blinded an can enroll which would interim, an additional to that stopped have evaluate, our but the be

collection discussions about continue had We Should all keep as our what in or collect data requirements expect these look? safety we've another to we label of But for open additional the to part we probability to that venue an safety take like. for use label registration. X as RAISE would arm look

we be would controlled would will on have said, that it agency the submit patients the it look that on as based the Make analysis. of case interim patients base the package. sense? to primarily placebo being blind of think another is But data we part set I -- certainly that will in double

Douglas Tsao

much. you Thank Great. sure. Yes, so

Scott Braunstein

Operator?

Operator

Jay next we'll Oppenheimer, from move And Olson. to

Jay Olson

you seen ZTALMY. Hey, question. for What the Thank are you drivers diagnosis? rate increase are to Have launch diagnosis congrats And volume the factors you on the the all plan the progress growth And in the the rate? in diagnosis you. how CDD taking XXXX? limit for ZTALMY do and of of increase what thank that current since the any largest

Christy Shafer

part Hi, there. question. This is Christy, this I'll take of the first

the is diagnosis more on code at usage promotional supportive not efforts And identifying much only have end point, code of into of utilized CDD analysis these them do United a what XXXX the we've is of patients, properly ICD-XX which from coding States. on then realized the therapy. XXXX, So we the them our correctly lot a that of getting and across

from that and currently So finding these diagnosis standpoint, how is we're a triangulating patients.

are I'll remind excellence across these assumptions eight some where centers we you have CDD on made are found, being that we treated. of United states. there Currently, patients the

do a we these months more excellence every then However, more and basis they're to centers home that go limited one their seeing of sometimes patients on know six, routinely every XX these physician. at

these increase rates increased diagnostic the of home excellence. the at and So be centers if will, these may physician in rates at you limited, the then

The areas, of don't that get rural these to a is the have that more center we thing capabilities in other excellence. to patients know

utilizing not now. the right are best code So, the they Probably of at their capabilities?

distinctly. educate some So more in only XXXX, We're our have to increasing not we able to efforts programs. speakers’ be

a patients, diagnosed distinct We therapy to new get that so awareness in population. through effort on patient specifically have to to families, to increase there's have planned partners, used for code increase a patient patients very who ultimately the education partners our awareness, through increase more this and have lot advocacy XXXX speak

Jay Olson

Great. That's helpful. Thank you.

Operator

next Baird. hear with Skorney from Brian We'll

Brian Skorney

on the Thanks afternoon. Good promotion congrats Steve and taking for question well my deserve.

hit around a an some you've get inter powered you comfort you're to the the what based wanted sounds of guidance on also excellent your should additional interim. be thoughts on getting for just seen like Just that lot

assumptions analysis get hit output really I for between output. to point full end have handle what the the a for So the the a you primary sort on P-value differences without versus of the wanted to delta

trouble just on take If And like versus obvious decision seems you calculation, the about XX% just delta to a do take that the why case. it's interim a guess having I'm and in it's delta. interim it I not XX% understanding an my

to is, factors question my consider why to what you So wouldn't interim? as the the are take

Joe Hulihan

for this Thanks, the question. is Well, Brian Joe.

The I the about the assumption analysis, we hit that we endpoints. by not it So, The we the data now if healthcare we delta was same. think got is enrollment fully interim be And as to on just the utilization and secondary reason would the gangbusters. well to have powered, do be the the key on would is study interim to powered with the comfortable Scott looking is study endpoints time wanted the be enrolled. were mentioned done performing would more decide the

be And be to reason there'd going interim. consideration, So, would we're no do it. to otherwise, that the do

spend -- assumptions versus the use there's Now gets the (ph) well, as little into know, at interim into bit an at There weeds, build an [half] How is how but lot a -- a end? interim the you analysis. there to the alpha of can interim analysis. you spent much is you much

you the alpha patient a interim outcomes having look the and if endpoints, between at is would fully we at study on performed difference actual However, the the X.XX a enrolled an the of one a end in outcome. difference have

And same is end the XX% still very have well lower it a an interim. end hit to the powered. P-value remains penalty though you but for proceeds low, and pay The if the there's so are actual it at even the the interim after assumptions clinical to delta -- you

Scott Braunstein

it's potentially you, is I delta XX%. think XX%, Look, Joe. potentially don't or expectation XX%. study, XX% jump me one this think We the outcomes. your have That things ICU be being Let I days other health economic small said, totally patients potentially do a in going think or to wonky could two to any study going in be to in, I'm with Brian, that we

be outlier the So in outlier. could patient to the would me, on number come XXX in five. patient study come way that of could patients reminded it same risk Joe more reduce number has XX general, or the have bias my an on to

the impact I the saw this the comfort gives and in has little I utilization our And the to us buyers of gets But we and only anything a That more think treats site So, of normal. better X, as now and view a patient, if endpoint. said, protocol. well. not bit point, we in patient keep on led initiates think with of as and minimal going I two bigger bit number out the every than a They're to time that's be equally the that drug. in a think Phase to success little of primary terms comfort into really certainly else and consideration comfortable three, greater that on

our activating participate a now There sites an to So, and is comfort level a are the of of busy sleep give that is powering them sweet a just always element the the into starting particularly any study. get placebo I months to there clinicians losing as are small get lot that side. on several key of end spot. we really don't assumptions. But It's the creates in some risk, think us on unknown

is I So, in by the to terms the baseline a of you study it's characteristics of of types et cetera. nothing nice data think to than practical. surprised shared more than with what theoretical points, But is that, we've understanding patients while and more being ongoing it have not enrolled, lot being

have So an it interim. luxury take support we choose and what is the of balancing that's to we the if -- we're we're we feel FDA that that fortunate

Operator

JMP Butler We'll hear Jason Securities. with now from

Jason Butler

questions. Hi thanks for the taking

novel on more couple a formulations. Just the

you forward you into we do the there X about data, And MAD be the could there how forward? interpretation will meaningful have -- a of LGS, should you that the secondly, think move the from And data one somnolence a Thanks. First quickly then first the use make expect Phase to with in reformulation if of to incidents? prodrug? trial how strategically utility enough moving

Joe Hulihan

somnolence, first the about Jason. on Thanks the question. could the I comment part for

the low is at to increases current the They rate. then the rate a and of going and the up expect neurodevelopmental study, be the And of, to and aren't think read major be I TSC Okay, they think concomitant have them major escalations of problems. end the that's titration get going don't dose, somnolence, titration. the from the on on on, to do factor low ramp cognitive these we'll but titration idea the we wouldn't with so somnolence going I think volunteers in We've healthy in factor medications. slow that's study, and a the increasing kind a detailed the I Cmax affecting adjusted and so MAD some I level toward see Start are rate ongoing impairments affect approach. the somnolence.

about idea really get it to clinical that. takes the good a So, trial

I’ll of prodrug, And Scott, of in the prodrug terms let piece the can you comment it? on

Scott Braunstein

me TSC of seeing I'll let that of study. share similarly lower MAD the on going And study And Sure. I the that that have Phase And at behaves discontinuations. ganaxolone. of and to we really work I we think, X meaningful we rates to it the a titration create the the just Joe lower has feel impact somnolence, improved doses the tolerability, this schedule, seeing done are as believe we're up, new to Jason, opportunity novel formulation, very use to be blinded though is back think in as in we TSC continue what to our rates

So we would of the type expect use same almost for to identical titration, the titration formulation. the new

some milligram X,XXX from get today. drug, it will Certainly, very fashion we're mild to additional we the again, certainly in the But don't insight titration, in only the Cmax’s. via we going And we titration you but doing as study dose. you much reasonable MAD see more believe at MAD bigger issue study, are when get a an absorbing that on some are some

not it's or schedule, an -- it’s MAD a study. an it's titration truly a So study, titration

some those frank so to there of be And clear, are just differences.

need a start use would a drug our our the side. has Captisol. partners It I'll very to And although work limited The really different at Captisol, prodrug the IV to work lot -- few on propositions Ligand it’s with eliminate It's value helpful. have to in been of we to specifically in refractory of certainly it to today the have in have And limitations amount it's important status. to GAAP us going it's limitations a for can tying give opportunity very patients. ganaxolone result. young expand day going super an It's some without in

that So is piece future we it investing an are really our of important in today.

that for the think a that a is PK day And exciting. profile prodrug have to think side still look IP goods. on now the I now, we then have the side, is right And And interim, right the of very profile, has the a the I seeing the potential programs prodrug On lower what a oral lead IND. from weigh we given potential with candidate we're certainly fortunate formulation. -- incredibly potential current unique for of what certainly once significantly new plus cost has a away has two our the for we're year dosing,

and mentioned feel of to the reasons along I down that road move compelled for think the we have we to moving potential the rapidly earlier. program all benefit the of prodrug

So, to dosing this in have is blood compounds and should for higher can problem deliver greater solve be great we to fantastic problem which life we drive ultimately our for. really patients, efficacy. think future that It easier choosing will biggest that levels a

But will tell. new John has point to behaved the it we're right your the real preclinical how But models. see in. that thought better moving actually reason again, rapidly Time now we and via that's program fits Pro forward formulation than we'll

question. the So, for thanks

think a really good I one. was it

questions. excited a operator, to going really that into With incredibly organization. Appreciates the an it your Again, call night. for Thank we're the going support. all we for are you everyone important year

commercial things. Our continues to team do great

And team we the appreciate best Operator, team their point to our a really time we call. major inflection you along. feel we'll our operations clinical tonight. Thanks and end so much. clinical is move all of we're at making clinical the trials doing Our and

Operator

Thank again, that conference. you. today's And concludes

You may disconnect. now

Marinus Pharmaceuticals (MRNS) 7 Mar 232022 Q4 Earnings call transcript