MRNS - Earnings call 3 2023 | Marinus Pharmaceuticals, Inc.

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Operator

Greetings and welcome to the Marinus Pharmaceuticals' Third Quarter 2023 Financial Results and Business Update Call. [Operator Instructions]And now, it is my pleasure to introduce your host, Sonya Weigle, Senior Vice President, Investor Relations, Human Resources and Corporate Affairs.

You may begin, Ms. Weigle.

Sonya Weigle

Thank you and good morning. are Marinus from me Dr. With and Scott Chief Officer; Shafer, Executive Officer; Braunstein, T.J. Chairman Commercial Christy Chief Lyons, Officer; Chief Business Dr. begin, to Joe Hulihan, the statements and we under Officer Pfanstiel, statements Chief would Chief Medical today laws. Officer; Chief and we are the Operating are some securities everyone that remind Steve making Financial I of like forward-looking Officer.Before by achievements These such expressed programs, future development cause our forward-looking statements clinical those involve statements. from substantial performance forward-looking risks to or and or significantly implied uncertainties that could differ results, risks uncertainties and risks the These described to including X-K.I with in Exchange over CEO, with call and Form our and associated filed will Dr. are and the our now XX-K, XX-Q the business company's Securities turn Commission, reports Scott Braunstein.

Scott Braunstein

Thank you, Sonya, and call. welcome our to

progress continued to leaders, clinical reviewed key in I September. events of Dr. experience our quarter, development oral I in via quarter strong of our person those treating to third status TSC.The marked and was analyst product. want to to express our XXXX Dr. appreciation who ganaxolone our to third late-stage want opinion attended you in in Dr. ongoing and also webcast Dhar, second or of sales the clinical turn who access ZTALMY, Koenig I global and my growth, our meaningful ZTALMY their expanding programs, patients progress and with Kahn, generation advancement investor of Before thank by

the that $XX.X ZTALMY again and expectations to once report range in we to mid-September million, to with million. slightly million our pleased are $X.X our guidance of raising $XX third of We sales the quarter revenues exceeded are

the are run validate addition is organization quarters making access of million. well patients U.S., rate the annual the Today, report results access build the full our our potential achievements early to new to expanding for review on patients the XXXX.In to into we formally commercial we pleased Chief X launches are Just success designed rare and These product request ZTALMY strong in geographies the opportunity their for prepare for new as we our our to local north of progress Officer, the $XX Shafer, to managed launch, commercially is our efforts and a meaningful in our Christy we available of taking ZTALMY X regulatory global launch physicians commercial revenue program disease-focused our Commercial in execute around strategy. steps the where announced world. use and provide to to will supported by requirements. and are not

collaborating are for Chief leader program. will Officer, Lyons, Medical from also and by Biotechnology pleased with of National Drug Christy.Our the our in who China Administration, Business Center this We progress. MENA be to of the for in an China granted overview regions Thomas CDD. The Product following in experienced comments provide Durbin, notable commercial EU, partners NDA will priority submitted facilitate Tenacia ZTALMY the review made an area, have Evaluation the

XXXX. in for of to in year. to countries in this MENA Biologix, continues our the the plan available region, the commercial product of selective we distribution And Corporation ZTALMY Orion launch European end the for expect by Additionally, partner have

since trial status our upward refractory clinical of on IV an of the ganaxolone enrollment noted III a And our global novel following update and trajectory the their event that take of Marinus patients review to CDD short therapy.Let to proud the anticonvulsant such time, analyst has at on will starting pipeline, all September, epilepticus. sites. an our last provided we and trial August, RAISE few update me a are in a provide our Phase We for activations investor clinical in in moments period RAISE created that early opportunity families was with network access the

Based Although of has the more the analysis variability to enrollment we has enrollment we screening for the our January. quite the continued expected. of activity patients quarter remains current required of the total interim rather of than by enroll to project end first of rate show number previous to grow enrollment XXXX guidance on continued and high, trends, now than

refractory the and anticipate a advancing entire criteria if are data As focused TSC. on predefined remains trials result, quarter now the we met.The our in III top the clinical stopping organization second line of acutely XXXX status analysis in epilepticus for Phase interim

acute over the confident for ill benefit in to and IV that novel in commercial could the are critically first decade. for well a ganaxolone bring We significant opportunity status therapy patients the

extension spend RAISE committed our to the data I the established XXXX our focus and valuable are these continue prepare I franchise, want underway oral our for into SE. are and the market the a investments our of We to of NDA additional turn we and we to required we to XXXX readouts bright Preparations to expect recent ZTALMY, launches. update most in significantly X for on trial QX of runway complete commercial our a tightening key cash resources opportunities, and RESET successfully prepare make and to an have the our in trials believe give completing brief on don't timing appropriate future.Before commercial of TrustTSC to for success with filing, impact both the the delays to launch.Finally,

expansion to to potential opportunity further in a the refractory clinical chosen status additional including of refractory Phase the on We programs, super focus II RESET status. trial development investigation have of established resources our discontinue in

studies II economic outcomes has as the potential enrollment and Our efforts RAISE to data the support on health RSE completing in focus research. will continue that trial, further SRSE to well generating additional RAISE the as accelerating for

is making prioritize We maximize our and will every that opportunities value.Moving the company evaluate believe franchise. investment oral will we the to shareholder

and trial. TSC, we this enrolling with which for gives our significant for low, the Blinded rates in us actively currently product III in in confidence remain efficacy are can of high patients potential that refractory titration in seizures schedule. Phase tolerability and development medical only believe We unmet Phase discontinue global the a TrustTSC trial patients ZTALMY from new this our address indication. are III We associated need suffering

expect top and the believe over that, laid experience we near trial believe line reason of even date. the real-world replicate several strong success the and key this our to and foundation We we fee prioritized growth like ZTALMY our turn With for every have have Commercial cash mid-XXXX a programs Officer, in Marigold Shafer. to have to runway I'd can our Christy call study in and Chief and data XXXX, readouts to long-term accordingly.With that

Christy Shafer

and in the trust, the over have our brand potential established pleased quarter made I'm launch. near-term year and positions progress which We patients launches. Thank ZTALMY for we the you, approach our additional innovative third Scott, and that present us success X good morning. first following validates a and ZTALMY to with as physicians successful

excellence, for closely CDD centers of their their connect and to patients. with collaborate to to with continue our community We HCPs helping local access them care maximize

a As product revenues launch. grown since successful of net ZTALMY's quarter-over-quarter have steadily these efforts, result

establish approximately on of to guidance quarter bring Importantly, this to for annual are revenue passion dedication retention reimbursement revenues we our to our the covered fulfillment. therapy, $X.X patient to to we ahead, condition in had team There the thank approximately a million team to $XX.X of is from XXX% and reducing employees takes of XXXX to approximately patients I average, hard commercial active Annual XXXX, order day increase serve.Looking the we strong want date. the pleased the third Meeting weeks.Given for XXX recognize At meaningful AES epilepsy at we XX are grew they growth, that million. a member rate XX%.In million. third on and quarter, it the X demonstrate patients continue over X out cross-functional preparation days end to of entire with of day of will in is remains product continuing Marinus solid million range are as where claims net a lives committed to prescription very Payers of and change community. the treat, to our $XX XXX Marinus a for from the serious working to reimbursed time to to lives on-label the

conference build plan HCPs U.S.-based a attendance HCP to ZTALMY patient as presence Daniel exhibit Child attendees We commercial year's presentation XXX over including engagement to Texas various from with and through booth, first with ZTALMY excited and Dr. organization. our engage year groups. by Neurology, on a our in are from our ongoing advocacy last We product and opportunities, Gossett

presentations in a the allowing is care approach while and have the continues IV of surrounding progress indication continued caregiver next importance are are new and acute progress underway franchise epilepsy ZTALMY medical organization. well us making All more with acute commercial and an strategic genetic focus a for community.In on and planning significant on teams clear early of TSC. several identification phase in discuss for preparation plan and terms our TSC for a evolve rare detail.The of Additionally, CDD distinct we efficiencies the our planned care and the TSC plan franchise, of taking franchise for that initiatives the community engagement. Joe for testing, and the the needs planning with brand in the launch data to build development focus hospital-focused into our will to genetic XXXX to expanded patient scientific support

Chief we're access while to faced greater the distinct Day annual access ASMs to by Investor bolstered address patients.These to the to clinical value accelerating believe stakeholders. franchise billion the key to hand multiple across status perceived treatment goal discuss year, care ganaxolone than to our September, we our Lyons, we base our access. in going messages a how we tied with foundation customer acute a positive system costs IV gaps be proposition, these $X maximizing feedback IV value T.J. refractory plan approach. for economic substantial we support patients the provides now the significant growth managed testing the our next underscored ZTALMY. the how have epilepsy the an who rare as and and fully call to Since success through value and then, have administering revenue to or the total the ganaxolone Officer, clinical continued Business care have and vital outlined potential anesthesia franchise of will and the revenues and on burden of ZTALMY's During into financial drivers their value and global for expanding reach that well as to healthcare This will date reason program of over progress genetic combined.I'll refine event each both usual from insights directly our

Thomas Lyons

local initiative This Thank diagnosis. physicians and moment not intends Program. in experience respective company the Marinus morning the Access ZTALMY requirements.Marinus works to countries. qualifying Durbin Division, commercial provide international specialized with managed has CDD commercial medical financials. on of concurrent the announced available geographies arrangement support are eligible distribution leader years our and XX new medicines a throughout their supports I'd managed Access pharmaceutical local by to not implementing Uniphar Access by including patients our bring mission Part certain and for Marinus detail programs to geographies ZTALMY and in product Access place and will you, with supported Christy. be world. designing more pharmaceuticals, for of our this the and commercially access is has companies The provide patients where a manage territory like to global in regulatory innovative access Durbin, in management program take in the a organization access to will as third providing with in global to CDD managed access be pharmaceutical in Program facilitate partnership Marinus where by programs. over requests in biotech Group's quarter of Product to

available place with China help will agreements us fees, in in found incremental such allow the This CDD collaboration in patients ZTALMY and believe hospitals, access transaction program along for issued cases, program, this clinical Europe, our the very insurance and our upon Hulihan, Marinus local global strategy no Dr. could there funding expand local on the pleased Marinus. Joe morning.We in MENA to sources the Access We're Program. programs regions, appropriate government this approved generated potential to revenue this the and make and our options launch be release working as and in from like of country. for has treatment can Net the the be would private revenue generate be Medical Officer, geographies to where call press turn details regulations our by time, development. charitable pay, agencies, Marinus some from Chief are Additional from depending update institutions, allow.At Durbin an program on our to this will I over with go supported of to private to in

Joe Hulihan

Thank I'll in patients. with refractory start to It's bulk be in on XX% second required of provide enrollment date. to Scott the status overview that number the you, enroll in T.J., and of by U.S. call. the centers with said, of the pipeline RAISE XXXX. data second newer to now everyone. the line development account ganaxolone required sites for based epilepticus.As expected quarter, pleasure enrolled believe progress patients patients With the an quarter over interim mentioned, for programs to end the the randomized first add top my the since IV status made of will with analysis we've of rate, of with enrollment analysis continues hello, for quarter current the interim the trial continuing we our our

Scott at a rate, screening although variability than patient As mentioned, high we're more continues anticipated. seeing enrollment

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a of of oral IV with are will antiseizure updates data top our So ganaxolone upcoming ganaxolone far, this present with study, medication. new Seizures Meeting.I'd in treating refractory with patients anticipated franchise, SRSE TSC considering treated to resistant, clinical been ganaxolone. study need, seizures. with Society in results for starting mid-XXXX. Epilepsy SRSE, potential TrustTSC, on availability with utility enrolling discuss highly the like treatment often now of additional patients initiation evaluating This III the American is TSC TSC. proof-of-concept Because have encouraging. on unmet regimen data disease-specific the we're Investigators its with despite in we're Phase To line address currently newer at

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these development. antiseizure new evaluate As in a reminder, our the second TSC will product to be to study generation first this I'll a added treatment turn to medications.Now,

trial. epilepsies. in poster suffering seizure successfully with data We've that multiple reductions are preliminary of treatment from and properties follow-up, poster dose, several the outcomes hopeful generation study, ascending a in upcoming XXXX, Annual Among announced, pharmacokinetic from at that pleased treating are a abstracts research hosting MAD patients is to we to twice from a ability we in improvement pharmacokinetics will MAD the of from new are we of X will in to these sufficiently data of the Meeting to in trials and to X submitted associated American treatment-resistant enrollment oral Epilepsy seizure turn by the dose dosing. which help provide the clinical this of see meaningful patients reinforce for half physicians disorders.I'd consistent to individualize Xth. share results have new pending longer breadth management X clinical our Marigold hope our from will with initiated first in We'll day expected be epilepsy.Also, the target key data were presentations there.To will the will resolute call administration study. develop second the move number that supporting refractory will families to refractory We all MAD shows Phase the our advance a aspect Pfanstiel, have and and other of of As provide goal data future a the We're years single accepted you of study patient informative, end with to and who potentially seizures that If our that effect and we're being or and open-label in and as cohorts are duration and the extension will December at be formulation open-label use Society from years. CFO to planning our Lennox-Gastaut to previously our scientific This our X could continue to over of financial highlighting update. options MAD Xst finalize ascending of saw Syndrome a program believe exhibit in trial for Orlando stable announcements the crucial you our patients formulation we refractory in LGS presentation ganaxolone study with belief the data COO, severe our show begun of ongoing We a suffering we XXXX. are year treatment Steven seizure next CDD. from III analyze our ganaxolone to with and its we at this review like of with be conclude, years and durability a follow-up, program, pivotal place will AES study the year, the we'll preliminary franchise, directly many now take the over recently of in allowing

Steven Pfanstiel

quarter million balance the and The XXXX. to proceeds I sale fourth facility shares our quarter XXXX.We the share operating ended the financial equivalents our good everyone. third with pleased third $XXX.X investments am morning, million, Joe, which of revenue million the able cash, from and ATM into Thanks, results runway is through our the to to short-term cash provide expected of guidance. net and quarter be cash quarter.Turning to X.X includes of of $XX.X in cash for of

update, guidance to in the net again range.For to prior of of annual We experienced expect quarter our higher range BARDA continue projected we strong million. another As end $XX to $XX of on now end to million $X.X million million $X.X ZTALMY revenues in $XX.X her Therefore, sales. be the lower an the increasing in range guidance million represents with revenue, which be are Christy project to on operating revenues increase mentioned expenses, of This we ZTALMY the GAAP million, is the $XX million, of we expect from $XXX reduction of our guidance be of prior our range non-cash the to in to to $XX SG&A million million a R&D be and which expenses, compensation. we inclusive of $XXX stock-based approximately of $XXX ongoing to financial in $XXX results. few costs driven to million Phase is our III summarize a now million to and efforts invest commercialization trial.I'll ZTALMY our prudently and by take minutes ongoing and manage and carefully

million revenues consist XXXX.Separately, the For for for of XXXX, ZTALMY X $X.X activity due API X as revenues product million periods to revenues increased and $X.X and the X API initiatives.Research X and which compared million increase third million year-to-date in of development periods BARDA recognized X XXXX, September XXXX, XX, the and year. we $XX.X same activity compared year. prior effort, $XX clinical ended and million onshoring million $XX.X product million in trial costs third $XX.X the with of X our quarter was each we million the sales, associated primarily change million to periods for headcount. and recognized with for increased launched and by our to we ended increased for quarter months TSC and as $X.X of of The million the million the September RSE to of These in $XX and compared onshoring of $X.X and in September XX, and for the the the months startup months year. associated the XXXX, XX, driven expenses The prior were same is $XX $X.X ended prior as same

X primary months the short-term on approximately As $XX.X same net year. for $XX.X increase increased in $XX.X months $XX million the drivers same headcount and on our were loss X by $XXX.X compared The was for ZTALMY revenue of and ended the million the as Sagard.The $XX.X million in year-to-date of XX, million taxes million the and agreement U.S. before XX, cash March and balances.Interest X year. the September as X of million the the interest million same is XXXX, in with and a the the the and ended The X reported was in September a for credit related XX% increased increase September million offset in the periods X and income the of driven September $X related million as investments XXXX, and X equivalents financing the $XX.X XXXX, compared change $X.X and periods $X.X administrative million were those months and income X million ended company for XXXX, basis and to driven to expense prior to reminder, million overall ZTALMY.Interest onshoring interest yield for in for of income expense by million compared to non-cash was BARDA, and $X.X a general increased the interest ended cash, for R&D API XX, revenues expenses and $XX.X expenses partially months $X.X to prior million taxes by in in the so and of XX, periods increase million net to $XX for $X.X The same periods prior year. is approval million an FDA prior XXXX funded under increase Oaktree before effort income is $XX million year. BARDA XXXX.Selling, and additional drawdown for by the as compared the upon $X.X launch in and the

the and year.Cash expense reminder, will a activities Q&A, year.Before months for the same to prior as compared operating totals in period $X the and stock-based the to few for included September X prior ended concluding our compensation periods of XXXX, make compared I include million million in sale to year $XX.X million XX, million the XX, of X we million was for cash As review operating results used non-cash X same and prior the $XX priority September move as a in the voucher in $XX quarter. These used activities of for the one-time $XX.X remarks. months $X.X ended million in XXXX, third the

including our making generation expanding and ZTALMY, number advancement progress growth, meaningful of in progress of oral We are second in areas, products. of sales our IV development continued global a programs access strong ongoing and clinical to ZTALMY ganaxolone

the these As exciting expected. with an those AES XXXX Marinus open year interest conference promises attending of for X again questions. attending we a conferences call continued months.Thanks seeing of one to to be key the in will coming the investor forward the look or be you We your other readouts scientific result Marinus. data of to may for now you efforts, or in Operator,

Operator

Your of comes question Abrahams RBC line Brian Instructions] [Operator of from Markets. the Capital first

Brian Abrahams

Congrats progress. the on all

quarter. since seems in trends, On could compromising study, getting of the readout to you're that -- to potentially and on, of you on you stringent there enrollment the can the then to first you of without ZTALMY cohort talk patients the by And if the and types reaccelerate you confidence complete the quarter study level the second have? some interim would maybe September. seems be enrollment quickly as your there curious And potential about end seeing conduct growth love even of the be of RAISE criteria in ways accelerating the just that what terms comment could the

Scott Braunstein

will commentary it on then her for pass I he pass me over has the Brian. Joe it Thanks, take to -- I'll any other ZTALMY. I'll if comments, additional and to let question. Christy RAISE over

flows RAISE question. this without I ebbs with we and the study, of think seen have trial

and to said lots -- We this of have I've investors.

of recruiting did basis the and lower that be confident where months team still and actually across rates X screening, we all that the role could year, sites And and are much accelerating deliver not to patients, the active of move that have to but enrollment. about they've these will And earlier wanted have in very have will monthly on conservative as patients we to playing today I not more got energized the we the terms we're X first that more in for still enrollment of and be rate sites prouder we And within times we from here, time number Xx We we high up and very weeks months we our as patients We've year. sure even sites the to country, of are weeks incrementally those move looking half are aligned for way enrollment can lines. and sites really sites did running.In -- are that still the have estimates with those really enrollment, in we a though believe and of patients. the an have make Canada Australia. active many ahead, modeling

gives IV of the because placebo from to to there? study to would confidence you that. criteria. to will started stringent feel to the But about more ultimately patients go on say I think this high study time I confident I placebo maybe a us And feel thought really the require then the don't in much extremely per We want about And mean, that But know them incredibly think going again. comments not patients consistent language say, for the enrollment, our after terms today that disappointing we team we've we have us, we'll did So XX%, disappointing want we where who when more who anesthetics, And continued pass to I move the Brian, I I think has overpowered conservative it's we we'd would be of stopping be any Joe, to investors. have Christy discussion, amendment, it for and anesthesia. today. se.And about stringent lower of pool. that lines. interim. we of very with the I last at added we to X/X IV rate's around rate where enrolling initially add protocol narrow It's to are only

Joe Hulihan

try initiatives. to Yes. we No, messages, when out messages have get some high I'm there also We're new motivational that very top the confident -- to still when enrollment. We're some to I mind. the we're mentioned, the planning with find of receive contact -- We going keep sites. as study about enrollment patients sites

end and contact coming that our up of enroll flows. is sites when enrollment, to limited, the As up typically and and the we're sites pick here. maintaining that finish we see But ebbs see time the the with we suddenly hope they toward you things to know

again. up pick that. things confident see to expect Very We about

we would. target confident So I'm said the by time fully to we're we the going get to enrollment

Scott Braunstein

than thing study that to in in example, anecdotally, higher screening And substantially month we're Yes. the a just And rate October, the comments just typically, last of was seeing add, Brian, the activity. XX% for to lower would I about Joe's I'll screening is screening XX%. has led rate. say enrollment And

ebbs that's year. we which earlier patients, think in seeing I of just flows than the extremely activity is but level high, different is and were the the So the what

are I our the be sites patients important therapy a standard were yet engaged, And respond think So enrolled. had few to weeks, and lot of And who projections. to last not that's us in happened just gives screening. we've they're the specifically confidence more

Sorry in jump to ZTALMY? do you want there, cut Christy, you Joe. off on to

Christy Shafer

thanks the continue that the for that we Absolutely. of have progress Good We morning, meaningful Brian, have. been patients stay mentioned a team The you with to as question. so types to the and encouraged same. continue

However, that's very we young very very some and into very we what promotionally that also brand responsive. have But young, significantly adults this learned is our We have come see progress. seen broad. very, enrollment is very,

a reach spoke we little about done, quarter our bit we've last have the we what into is So expanded community.

patients bit ICD-XX to that additional kind to the COEs get that our be with well leads talked a therapy.Now, very also continue we central to communication little then Our work quarter and last as have. community but understanding our of they promotion, we closely about on

reach We of have even to further. get in invested data that additional sets

further. to even how triangulates to are physicians continues and moving then that promotion so we back that and grow, data and just caregivers our targeting And

is meeting and doing, And and have and make only caregivers, we the patients to. really need physicians lot and this delivering are responsive fact to that these again, we're we a continue a what it's not that in sure that they we So working. on we're we zeroed where market, just very, are very with

Operator

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

Joseph Thome

of enrolling, a initiatives Maybe maybe the don't mentioned increasing filing, really and there is a I you very a of some Are that that aimed high need kind safety of above if have you sites you any? just that seeing in one many contact next XX failure the on others patients database. screen of rely these at think certain bias that can strong would maybe that for guess, you level enrollment. quarter? over terms are there? they get the How you're maybe that enrollers there sites meaning in the a on And maybe the are quick just criteria? Are rates, you

Scott Braunstein

Thanks, Joe.

to same got but Let but drug. really what would the as and discussion FDA blinded openly me fashion which portion I'll a and about start we using with the We've the about they the number patients we profile. Once on RAISE of the protocol an study. see of double-blind study continuing then only interim analysis, interim last also think I'm good a flipping function that a the over actual this seen like risk/reward. about a sites believer in is of dosing analysis, I have is have to terms up to very RAISE. question. during not on running, go Joe we've will we safety feel Certainly, safety, let talk always the talked the the II in We've open-label a

of So III.In in more the a of data getting filing, efficacy I have safety of are at the that to we'll RAISE Phase we're regimen time who the terms patients than going think have an certainly and say, that the been in ample given since the I been we nicely study. sites quite most summer, and thing added think XX really the I new would or many the those of since about, and encouraged sites, the I've summer, XX, producing have number

broader participating. the bigger for been have to time them patients. the been finalize long a some databases of the have It's sites substantially we that asking study. We're number of that a now think really I their clean So sites are in and

have more we failures are And screening we So hard. question, coordinators I doing don't and that I to your something at study are have sites they're working But think and are that screen as look our screening wrong. following sites patients.

patients. the They're best with And mediocre generally as take think that patients. study. good data to And consent. remind for the cetera. it's is a would of insufficiency, we I for will liver second-line happening looking expected. failures. therapy. renal overall. a have ultimately, Or the patient care a pretty just our following getting screen They're That's sites high probably there's failure, I'll XX screening the argue XX patient sometime and between can't is be medical which are And consented, contraindicated hours, They're actively suggest reason it's patients function. sign. patients or had either would the But I folks, our We've many responding et

to turning think enrollment them I luck had just we've add this Joe, month. about less a little with anything sites? want into you

Joe Hulihan

has that the No, recent enrollment and sites have recently. come more gotten I'd just running reiterate, from up sites, newer

high, as patients enthusiasm just high, is and some up Scott getting screened screening So, mentioned, again, end out. is

and efforts, think very the if continued if the keep we'll screening study. I'm encouraged -- I enroll by we we that, doing

there's screening. sites and any representation seeing but the a study enrolling perform, how variability broader in of actively sites study, As in we're in

Scott Braunstein

it's I'll And our coordinators think continue make sure site Joe, had clinicians team, decisive to comment that and a make the to enrolling That's we patients right date. have really our and to the way the is the that the our and last victory. critical we're that standards that going continue high win only study's I we've

the just in we And our not out so study but it's are educating there have run the I you MSLs like the respect hit numbers the always so rules who That's regard. when that XX the enroll clinical and in our always a for bending But and catch to team balance this. our all a Joe? faster. patients world physicians

Joe Hulihan

new with any safety consistent And good, the signals. Scott, neglected Phase mention, you looks mentioned I not We're safety to seeing safety. II. the Yes,

favorable. looking that's So

Operator

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

Joon Lee

coprimary to over XX% stop that you size look at want enrollment, that actually just expected, given that stopping than detect XX maybe for a as you endpoints, the to read slower on much effect stopping patients, time spill don't if XX% a you the at full you which is confirm study the later criteria. powered a little out. been interim, but I And point into time the can study at the has the haven't has disclosed criteria to changed

Joe Hulihan

minimal there's significance criteria are you for even Yes, a out XX% -- we we the power Thanks do statistical that's always actually it lower would delta. absolutely significance. statistical have could When right. treatment spend statistical the the than question. an see with see It's the end, have with would a alpha Joon. to detect power difference. and Actually, study. the that. in statistical significance. a we a that analysis, XX%, on interim stopping we end Yes, delta actually -- it's went works to have at X.XXXX, Down XX% the still the But detect a that at that And of to XX% powered XX% that, range you to if of impact have the And that to do. -- but is in

interim. the it. at about Very So again, well-powered confident

Scott Braunstein

only Joon, at we the And clear, all moved be to add, Joe, I'll not goalpost the that. on have

have having going exactly as or a conservative gives good more much in -- terms lower goalpost rate placebo be. we a We mentioned in about what we're at higher. placebo a placebo trial, a think had what flexibility But just, the the today, believe statistical the in least I that on handle to thought And us of conservatively I earlier, rate, significance. we into would hitting minds, XX% a case seeing which regard. assumptions not clearly, our that's rate not very the we be all much lot at moved

Operator

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

Charles Duncan

talk progress to good, for to RAISE, All discontinuation as the comments talk want about for wanted about but program. rate didn't TrustTSC. on stringency the well that. as I and on Congrats MAPs Appreciate placebo rate the commercially. I

wondering will it Durbin? with driver program? MAPs financial the sense for of dollar How can kind of was it, of you question of a call retain? a had or sales what guess I the the MAPs. opportunity revenue you request? much, us it prescriber terms patient So was on your Was or And Or dollar give I identification? I'm

Scott Braunstein

U.S. from families, Thanks, ZTALMY, outside we once Charles. than month, Really twice more or appreciate routinely, access the question. receive Since requests about patients, physicians ZTALMY. to of launch the the a

Europe do not example, in alliance, countries And with time other but the so in and we for has with and what only CDK in review in the IFCR much CDKLX alliances helped U.S., status our identified China, spending the driven us is of and just Chinese -- disease only that not the priority how and are has understand patients from how have been orphan big XXX Chinese over many that growing, the and the government. certainly efforts the

with our help critical alliance and families in are the the it's we groups constant patients, provide So around us to for to world. discussions drug And think those groups. advocacy I

comfortable all was that very global processes that foremost, goal We're critical this. and that's get the about mean, can complicated more of -- those And as really of global. with those I go little think now a first supply So a shelf piece our I bit think X-year a we with here.Secondly, in life. we're you expansion. our a position

in think regard our we've level had partner I think companies interest and made tremendous supplying of with. comfort that drug. we've a supply progress inbound lot of I to our So from And in chain

over T.J., economics we're and it more any flip the Steve, have about to how a little pricing. maybe so And bit I'll you if talk and comments about to you thinking

Thomas Lyons

for thanks, and Scott, Charles, Thanks, the question.

somewhere pricing release mentioned projected EAP. -- where fall space and and pricing we in a country-by-country as pricing in and a basis. between on in we'll we their lot relying the the has we're experience Durbin, EU of EU guidance are in -- on anticipate today, on And U.S. conversation We this the both pricing,

our the significant trajectory that with that's be at yet it fees to transaction pricing proportion net retain, it be top would of a So respect would And the Durbin. the finalized. would from -- to be we line, moment, majority

Steven Pfanstiel

that important our add administering an Charles, this just on behalf. is partner, they're really program but Steve. I the would Durbin's

of they make revenues fees overall aren't We the expected. the here, detail percentage low but a disclosing up in

Operator

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

Marc Goodman

the the in you think the What's off-label of are you there could good a attainable. quarter now year. the number those can a give we the that about patients? us CDD the I you been you to gross there, it respect that's reasonable, patients that a get number give and while, And to think are the are us next in and pretty into net where are was can Yes. XXX? there patients know seeing truly ZTALMY, to any that sense big you that And With for feel wondering flavor out you've if how to just of you

Scott Braunstein

with Marc. do Thanks, you start Steve, gross to nets? to want

Steven Pfanstiel

a Yes, detail there. bit Happy of sure. share to little

a deductions than said that It's have our gross a net expectations. We over little down do of year-to-date I gross XX% the been course think with pretty the net We've see to consistent slightly. less been XX%. to basis. actually around deductions on the always coming year the

have other have XX% of patients the about We access. on But about XX% Medicaid our their primary, Medicaid commercial, of commercial. as that XX% XX% secondary

through they a been from off, they've and that net. As we get gross a their XX%. to secondary But below really just see benefit drops Medicaid deductibles, about little consistent,

Scott Braunstein

make give me Christy let Marc, comments to over more or to little color. you I it one a pass before two

about always I pediatric are X,XXX we've year, patients. XXX a we newborns think there said, about believe

X code's for XXX or our still don't X number you, excellence we And centers that codes. tell third-party will this patients know of north ICD-XX I well growing. of been around is The -- certainly are or suggesting the hospital of ICD-XX today, third-party codes use that there's and many remember, the only still years.

than So would time disease, more is been patients population.I there the for a thing being the to have of treated there -- market. of we biggest into how launch we've about seen nice seen coming the know we upside say we that's the certainly adults that out number are has the thought from which is different

to is adult thoughts. market we're we certainly we fact for Christy, believe over So neurologists maybe patients pass the originally. than did that the you treat potential any larger, given seeing I'll additional

Christy Shafer

thanks Yes. Thanks, question. Scott there. really Marc, points And makes Scott. for the great

and is we continued achievable, codes. very that progress in underscores really reflected just addressable patients with X,XXX that this kind that year, believe and between do the that of our in there a see So XX are pediatric not it, the to fact and again, on XXX zero newborns ICD-XX we really yet

usage we that we drive with to continue forward the the Now, to know of need message code. that

break just efforts thing habits yet to not continue very, continues large old the now not some drive a their drive testing genetic from very that only a are epilepsy. maybe with patients, promote think with we diagnosis for that of is cause number our testing, that those to that genetic and also but upward only has I have hard a but message.Another and in AES don't newborn have physicians, then promotional who a diagnosis genetic symptomatic -- to

all will again, the some community, what well. epilepsy diagnoses not we're of as which things, patients addressable support the driving help these not So in pediatric doing of but only but X,XXX are patients just older even larger maybe only then patients,

Operator

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

Lin Tsai

updates. the all Appreciate

is Obviously, to RSE. the on more patients get goal by the XX year. of one maybe next So to March

the end ideally So necessarily? about would how off frame? as you November, we should -- period next take maybe little think we like another people Could could said holiday update when bit you by we Where expect not give time be as to the in expect we cadence, down Or on then enrollment And a year way. you or on think your about enrollment? slow could XXXX? time December to you enrollment finally, enrollment

Scott Braunstein

with start Andrew. the me Let update,

continue the we've time the people. at logical we'll or Conference, time be think to J.P. most at point I Healthcare a update Certainly to logical Typically, Morgan update enrollment. that in Morgan time. most would given business update the J.P.

patients hit that. numbers the enrollment share talked What to X very about month. continue to about confident us was for numbers you we'll these we had So enrollment X folks. felt with a to We

a would on that X thinking.At X enrolled Sunday. conservative say view we've and weeks tried doesn't of With months, when general month we we're holidays, in was to have XX% impact my to in to to our -- I here. as to terms line the the us that I patients the practice. night our My current Saturday least timing, last XX%, used specials is seem to weekend could to get projections as be and big get Saturday of expecting patients and them we've call probably finish wife for to really few so a

Certainly, So difference seen would where I holidays. don't would weekends major think hospital the an in staffing in cadence the you have less a we've around think impact.

weeks but to So to kind good about line. trends to are patients smooth X to finish per we'll their get as Joe, I and to going and you thinking And we're generally bad that the X update. having to anything to said, us of these out add that's month, want that? plan going on

Joe Hulihan

think actually impact just that. kind don't is than And a I study. yes, we'd holidays study of New I year was remembered, -- think last the different Eve expect No. like I a have to chronic it has outpatient as enrollment. for the don't a good Year's on time that same this

Operator

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

Douglas Tsao

sort sort sort Is or to the just -- standpoint? easier and development RESET in of fit in terms driven a just of SRSE. just practice, proposition consider it decision trial clinical simplicity pricing of curious it's that the maybe I'm value pursuing of how and by feasibility the of clinical the or discontinue from in would obviously

Scott Braunstein

the question, for it. appreciate Thanks Doug. Yes. Really

understand or I'll there's important a I've I really there's is here. really we've -- play That and pricing a physician consent, dramatically But for I us. ER investors pressures with sales reps. message a study, And thought in always the dosing the lose spend us site to lot the have need think from and in higher study emergency community money a I as There to up a setting. commercial for which said, this comment Christy then gone in upfront can it to lot factors consent, lose the community done a I do that costs But this more may we as overall, thought standpoint. start let frontline cost. have a was they just shared obtain side. think huge we one always complex are because coordinator of to a a from to We'd of territories. would be opportunity we for moves being the room

that of so there what refractory were to commercial grow, think to we we label important that looked down business of meaningful the did really the this in us. grams drug. status. a and space spending very didn't and to opportunity just We teams, And just our going continue as at commercial spending critically continue a and I was to think for great our to the quite we and do all the XX getting business.The that really think access we expanding our I -- certainly leadership a team the we recognize flip sat a what side on vision requests think going we're get study, we worried to with We've we a -- looked were I and to at and is of was and And side regulatory lots senior have for we MSL for path lot. is shared super data economic it's to Captisol. more about maintain HEOR, health have team to And it outcomes, as a and we've strategized

seen the gram that And that given team we would can physicians the study. can patients dosing, with let's it of going wisely XX got we So much the think valuable, it, say number study the be ton said, SRSE Captisol, of today. a are more in obviously, where we be we've But a one in we nice of to X,XXX our really continue dollars the Phase as spend and back cost given treated. are sat IV to a, It we've ganaxolone to we've a understand patients, call FDA. align of interest milligram dollars

So the it. thinking anything about Christy, on do way from we're standpoint, to a want add business the commercial that's side? you

Christy Shafer

Thanks, Yes. Doug.

which could inside for say take one I is mentioned that think the thing promotional think ER, effort the I pretty just would it of robust. Scott infrastructure we all a was that

who these as that proposition we proposition, And medical proposition an SRSE what need. by at focusing And the care grows that that it are has we're given could the are significantly an been to total inside treated, if the we shown really been Marinus these is gets need, on something the mean patients quite in total in And with And unmet patients. we as dire definitely unmet need, population focusing have responsive value Joe's providers so patients the status, know large. these and in And but we've encouraged clinical value really progressing is some effective value for is we and large. not team need and what moving that system patients are it economic the and When only quite of these of is us SRSE. hospital value potentially through bring medical on look is cost we circumstances. healthcare

is value quite So the large. these again, clinical looking economic prop and for at patients

Operator

the the today. will to all Scott closing now That's for remarks. have call some time over for turn questions I you. Thank we

Scott Braunstein

thanks in. for dialing everyone Well,

to job. will excited we do struggle I of get program fantastic dialing will that this time generation continues couldn't I'm we of incredibly all didn't on we'll to which finish get but today, to excited spend any expect end. that second down, heads It's we're we these me just line it on admit working about for one on call Commercial team today. be And have continue year both about. incredibly appreciate Q&A as keeping studies, and under on a our And well. in. data a are all we we're the and I that but Sorry little you today, the weather through a the bit call to bit the a to openly little to our by

look to dialing finish all be you so and forward studies, for thank much. we'll line all Thanks touch shortly. in in, So and we the getting these you on to with

Operator

concludes call. conference This today's

disconnect. now may You

Marinus Pharmaceuticals (MRNS) 7 Nov 232023 Q3 Earnings call transcript