MRNS - Earnings call transcript Q4 2020 | Marinus Pharmaceuticals, Inc.

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

Operator

Ladies and gentlemen, thank you for standing by. And welcome to Marinus Pharmaceuticals Fourth Quarter 2020 Financial Results. At this time, all participant lines are in a listen-only mode. [Operator Instructions] After the speakers’ remarks, there will be a question-and-answer session. [Operator Instructions] I would now like to turn the call over to Sasha Damouni, Vice President of Investor Relations and Corporate Communications. ahead. go Please

Sasha Damouni Ellis

Affairs; good Q&A Dr. and President Hulihan, With from Commercial to Kimberly of me Medical Also and Financial Thank the Officer; Christy on Braunstein, morning, Regulatory Shafer, Chief of are call Scientific President Vice Scott Alex in Joe Vice Smith, Executive Marinus Affairs. Chief Officer; Officer. the you, participate Aimetti, everyone. Ed is Officer; McCormick, Chief Chief Dr. our

that Securities These I some could associated certain Form I begin, like are would will and made in forward-looking remind our everyone subject with over uncertainties cover the and now we the turn are today the XX-K in Scott. of Before be as company’s XX-Q under call to statements the part forward-looking to as termed the Exchange that and securities and risks to Commission. laws. business with statements filed

Dr. Scott Braunstein

morning, fiscal for another very our tremendous for Good shaping quarter XXXX to was providing everyone. period welcome XXXX Sasha. be to up update. is fourth Marinus. exciting financial momentum strong end you, and progress, year Thank The And and what of quarter a fourth business

on patients already status of our XXXX, and we trial clinical fourth this CDD the did exciting, CDD a at base, December of milestones. me announced Before this If $XX I Annual as States Program focused at several Society we as have the launching from meeting incredibly from well important an both look research you quarter, year ganaxolone offering who raising for treating super-refractory some on the use Access equity in let in trial. enhancing touch has presentations as Phase million our with Expanded the United which been not participate X share and investor and suffering included year, in clinical the highlights epilepticus, compelling in American Epilepsy

the topline my in epilepsy, Phase X enthusiastic PCDHXX-related review data Violet share are from which to Alex we Firstly, promising following remarks. will trial opening

complete ongoing evaluating that treatment report track expected of or this year. Next, we tuberous the adjunctive will in happy study II are to trial enrollment topline with patients on The results week complex this and in sclerosis in are TSC. efficacy QX and ganaxolone of safety Phase

Importantly, of with X an by confident Phase to X that this after discuss the for to with are report NDA on happy submission design that evaluation CALM interim Phase FDA the in trial mid-XXXX. randomized the moving for global the track is conjunction registration the is meet this trial but in by supports hope in will ganaxolone Joe to we CDD-related analyzing data open-label the trial. review our epilepsy we of placebo-controlled more of Phase a the double-blind X TSC, to middle Study, QX. news, use In are detail,

having of the We our reported clinical the of the patient first treatment in X refractory epilepticus. enrolled trial for intravenous also status ganaxolone, RAISE Phase trial

activated fully have from to screen in sites of been that first the and are on half RAISE the that challenges the the additional by for majority trial and believe in We we topline track continue of continue vast to we the Despite look XXXX. study enroll by to the year. COVID-pandemic, middle data this patients created of have the

Dr. epilepsy. Affairs with would Violet Scientific study trial, PCDHXX-related Head like to on Alex over of to the in expanding patients Before our I to our in discuss Alex? the our results Aimetti, turn proof-of-concept call ‘XX plans,

Alex Aimetti

PCDHXX-related to I’m placebo-controlled Scott. very you, Thank X with you our in epilepsy. topline the share clinical excited Phase from data study

of XXXX, a announced the of As the a as truncation reminder, decision. study strategic we May in business

results between in decision, directionally the ganaxolone of result treatment results and very of are demonstrate observed as significance with powered encouraging study further adequately consistent effects a generated well-controlled believe statistical are was day the from times not this to dosing. a CDD were the validating they we study the a arms. As Nonetheless, X

of levels, hypothesis, ganaxolone median ganaxolone X-week observation which independent XX.X% remind preliminary period. patients The to that evidence frequency then baseline to endogenous and broad added in the tracked the allopregnanolone-sulfate frequency, across improvement placebo XX.X% study the study wanted period treatment effects the may medications. treatment of Patients provides in baseline compared seizure anti-seizure in X.XX. allopregnanolone-sulfate an reduction study patient anti-seizure be the randomized broader may receive designed baseline included ganaxolone reduction you XX-week study design. track CDD frequency. then baseline standard-of-care seizure ganaxolone evaluate was in across period seizure a was finding for directional primary to during This to p a was The to or to percent I may originally the have XX-week in confidence was effects hypothesis relative to Contrary with providing placebo First, response experienced the to were consistent Patients ganaxolone this their patients arm. a further our This ganaxolone endpoint in endpoint of arm. value in XX period This patients the observed randomized refractory and in further response. ganaxolone primary PCDHXX observed predict the primary XX in epilepsy. patients treatment patients that resulted endpoint with randomized. population. levels the that in arm frequency XX were placebo in efficacy change was all the arm the the Seizure

in size. With call adverse Lastly, well-tolerated the Scott to back the all, event. only out to study we that, remarks. I’ll small very his generally round discontinuing are data to patient ganaxolone was with sample one despite with ganaxolone these study encouraged turn the All due an

Dr. Scott Braunstein

Thank you, Alex.

our PCDHXX results families study. physician team, through with Marinus our anti-epileptic supporting We suffering this their are with would our of compound journey. to I thank broad patients ganaxolone for in us the expectations investigators, data from as belief pleased very with highly are consistent and The Violet like applicability. the support the an and

for as and of With a seek help would meeting at expect the as this to team, medical data we present Alex publication. well year, a journal scientific later his

submission As this double-blind placebo epilepsy. remain for look suffering in turn from the we we opportunities fill to over envision to epilepsy sustainable by ganaxolone that a address to patients developing work rest unmet oral scientifically of use is of guided should important believe is first Marinus, a and need. reminder, back step I medical as the the of meaningful long-term. be sound NDA in the to in and events our our the that franchise we forward the CDD-related controlled like both treatment next We would other principles the organization’s PCDHXX-related the for basic that beyond, the meaningful XXXX trial a

intensify and for the indications. drug a planning our ganaxolone intravenous XXXX epilepsy and future believe on on a progress efforts to CDD, intend and our evaluate we ganaxolone path endeavors, other X CDD While opportunities focus in trial continue TSC to we early reformulation in success PCDHXX indications. for commercialization interim to-date, in additional of that our may pave RSE we potential maintain orphan oral additional front in Based launch, analysis pro progress our registrational midyear Phase our

organization. dosing paradigms. team disorders. examining a a that important be bolstered rare Marinus patient data, to range clinical the build ganaxolone ganaxolone strengthen we driven focused believe will trial and evaluation in potential the our studies, design, drug our of growing We opportunities, We as epilepsies, as of and seizure commercial an thorough dosing these by other set of Our support clinical, has on well the regulatory investment become efforts, scientific the the previous data PK and rare decisions rationale using legal, personnel. commercial, in operations to To other continue TID

team, top Development has Hunt Vice as brings Strategy. space. ranked President of added team, Ian the to President, officially a Clinical Market Joe Ian in Vice developing including Ian patients a reimbursement been several to had the considered worked field market opinion Miller Hulihan. key and epilepsies conducting organization with and Dr. strategy with in been decades organizations both research rare be Christy Ian advocacy several building quarter, the rare treating Shafer, the has the reporting several will has leader as joined of franchises. Channel the Access epilepsy to During her for we out Dr. who reporting leadership members and commercial

We that are that has to joined the we’ve scenes, on out pleased to organization strengthening relationships, Behind critical are success. identifying Technology. Fred key Marinus announce Halperin centers MSL to the continued President, equally presence our advocacy our the our our been as and build focusing Vice messaging future patient had and

stop to in extremely have quickly X tirelessly entire is strategic obstacles out Marinus feedback a logo coming resulted the encompasses, Sasha, innovation our fantastic overcome Marinus trials, our many Finally, company COVID-XX. our Phase I RAISE specifically give by battled and want community. team. for with kudos to presented the mission, a clinical helping align and creative have leadership and to and and commercial to which trial. new the Their for moment quality the They shout and up efforts cards thoughtfulness commitment, the of on team the positive

As markets sites be the pandemic ready we are see and calls begun world. interested will We has major now signs of believe and clinical both therapeutics hope programs trials. yield to that that the involved several IV ongoing our to in and own important oral in inbound our fielding subside, we around

turning provide detailed over highlights more IV call our the quickly So before the let to Joe of discussion. a trials and oral for me

expectations in will of ganaxolone TSC our Once announce met expected goal from to to of safety first for adjunctive timing X potential the safety with CDD data, Looking I initiated, am shorten at patients to X patient pleased confirm window our our with a treatment between and quarter. our and open-label Phase interim TSC and first oral topline is the launches. data enrolled evaluate efficacy the to that the in to criteria the fourth trial significantly Phase is the our proceed programs, the the be we trial trial tolerability to

TSC Marigold in approval drug support sufficiency We III January, data drug will appear filing the we sufficient Phase received of to year. CDD be a application. a designation and ganaxolone also as an for during new response the our this support Study FDA that of the the the And the determined indicating plan efficacy data from of to use in later reminder, FDA, the request of submit a to safety from positive In review orphan the application.

shortly a a will well year trial. this the February for suspension, data ongoing Marigold open-label through the formal of from one-year update have oral as as We stability

a deficiency the XXXX. as expecting forward CDKLX the approved this be strengthen Keep package to followed from year. EU first will that make if of submission EMEA balance by the specifically NDA look This as be QX used in Rare are to ganaxolone of end medication Disease by the We voucher by and and indicated QX disorder. Pediatric of potential complete the submission. the our planned a believe for result middle this NDA our of expect We We sheet significantly FDA. in to treatment seizures a receive approved, we mind could voucher the FDA

we outside summer. the a drive and We approved launch, we strategic European the targeting of the IV that European and are critical if in ganaxolone XXXX. half in can also have partnership be second franchises, specific for is oral a U.S. over both This to will for enthusiastic the partner place are

continues of refractory status for trial, RAISE make epilepticus, as program good IV progress. mentioned, the treatment the Our to

the expect screening reduction high either to quarter. be by enrolling now early in quality the vast opening or are sites, encouraging COVID-XX gradual sites in restrictions, trends We enrollment. patients majority we opened of second the to be the and seeing With

of are trial a for the As result, a in continues data Europe half topline we believe RSE to to to track an trial planned on first we EU first to in of half the be The registration. that is in conducted XXXX. commence separate Planning report support XXXX.

to we most then IBM status in and ganaxolone that treatment uncontrolled to requests super-refractory believe in where to ganaxolone to We status use rapid durable receive physicians and an statics. consequences effect most action onset of seizures. play stage of In advanced the patients patients epilepticus, from forms the continue a of failed firmly can of preventing respond with addition, devastating the important potentially can role in it severe SE provide the a of have

part our pursuits. valuable a future team speak I him CFO, been Smith, turning has he I the and development. in company Ed his our thanking to for thank other management over pursue Before to is call meaningful the Marinus discussion, success wishing continued who more in We’ve Joe made business progress him contribution the opportunities. of and well clinical career for his detailed our for Ed leaving since joined to to want

Steve more LifeScan. Steven We new to joining finance that recently Financial Johnson, professional join to turn mid-April organization to Johnson in We now like and our With Chief in Officer. experience are leadership our & of timeframe. strategy roles the majority at and organization him Medical diverse having happy his in forward the as Hulihan. Chief the announce would Optinose that, career had to with Officer, over a will at be Joe has Pfanstiel call look the and Joe? I the

Dr. Joe Hulihan

and improve advance good there’s deficiency to further disorder and Scott, is ganaxolone filings, a regulatory for year, and epilepsies. CDD. treatment meetings high and submission believe and you, This the where orphan an strategically and defined activities of where significantly Thank CDKLX everyone. NDA program of treatment true formulations need These of several potential devastating in focused unmet well ganaxolone outcomes. data will morning, targeted for or we rare Marinus on has we including key rare the patient expect readouts epilepsies,

to at to franchise. least or Turning tuberous pleased targeted Study, we the sclerosis eight we at analysis X. current next ganaxolone CALM oral complex enrollment achieved enrollment interim for patients proceeding that had XX efficacy expect at within the to where TSE, first we’ve In called an are Phase on our I’m few sites. open-label conducted a has X to targets our showed Phase study conducting Based study report the we goal reach that safety and that days. enrollment,

track across available second efficacy, for Phase quarter seizure Based of year, in XXXX. as QX as including adjunctive and patient in fourth and with third medications, the interim first X the provided end a meeting treatment meeting with when Topline support enrolled EMEA quarter interim a efficacy data safety is analysis from to the as well in ganaxolone the newer with results, of on Afinitor overall on as the to are the to in types, currently available used trial AEDDs, and we addition an X the In such and planning plan be EPIDIOLEX. in the of Phase have this quarter. FDA study

with placebo patients proposing three trial, lasting ganaxolone to genetically seizure or FDA study four baseline phase. obtaining year double-blind the a double-blind remainder for phase. complex. placebo-controlled study We be data period multicenter enter period day to X,XXX six milligrams a the maintenance a weeks, a lasting doses XXX will be Then to titration After and After randomized years XX-week enrollment age one with the confirmed will are enter the they weeks a on of in or matching at participants frequency, to receive from divided double-blind planned sclerosis of that ganaxolone placebo. XX up dose of of tuberous will a to per

Following opportunity this, in the ganaxolone. an enter extension they will receive have will open-label all to participants which

effective to the seizures to dosing filing. studies CDD our In the pleased with fruitful CDD, encouraging studies Medical single our ongoing and the open-label in and positive is to studies disorder We key from the planning failed our X could by in FDA evaluate effective Study in of all that Alex extension Phase The NDA included and The of CDD, of seizure We other encouraging of time. X and importance of of long-term enrolled we we very X presented efficacy franchise, the adults, Analysis CDD. in our data this the times in plan characterized that A to EU demonstrate be the was with follow of of both to discontinued TSC filing. refractory other that analysis different study us from profile trial review site follow Phase data insight report a day to announced epilepsies. times that over to generalized. the advance are gives data the we and ganaxolone XXXX. the study the a discussions treatment data Marigold believe filing particular, demonstrate efficacy NDA in efficacy. and information to submission. was PCDHXX concern types, great patients. in ganaxolone twice extension in FDA of positive confidence the and had CBD, we’ve we’re status CDD from of I’m we submission. focal spectrum our In the We that of and that will first quickly onset NDA believe Study the slowed data additional the that in the be Our AEDD regimen the dosing and of have versus appreciated in durability of of demonstrated IV anti-convulsive a data agency consistent of diverted a day satisfied open-label seizures In the resources across supportive of epilepticus the focal robustly from refractory Returning startup, NDA efficacy a hospital broad data study that’s The other the results the of the COVID-XX in older loss our continue the in Study interim the X focal ganaxolone Marigold Marigold Study and in the this phase a enroll ability support use the in as PK/PD that what screen Study results ganaxolone daily early from activation. RAISE patient trial. pandemic in Marigold the in that we the

hospitals the and most the for back we expect quarter. coming by of study sites online However, second accelerated to has pace end have of enrollment the activated

on Based readout on track early enrollment, and study to the about first and for this are patients. topline a of feedback pace data in of XXXX. enrolling we begin half and building. eager momentum about clearly the the positive getting signals see remain excited We’re We investigators the

to published the in ESETT patients status is presenting we’re experience community seizures. in quarter, earlier England ESE year continuum in of of lower convulsive status, the one control line status for known to treatment treatment will that the the responsive the the a last status emergency patients of of stage who with have first epilepticus. epilepticus. why year we’re successful of U.S. centers and centers emergency or urgent In where as study. maybe study, study trial ESETT than trial XX,XXX with non-convulsive epilepticus, targeting have many rather the engagement studies ESE, This with patients status Medicine. and same involving status evaluating a refractory trial anticipate departments who and that non-convulsive ESE parallel of obtaining status population, Journal likely necessary It’s the benzodiazepines. patients the will RAISE of earlier design the than seen of in is in the initiating New dose trial The of the the and consent per estimated cases ganaxolone that mentioned we this in believe to of approximately the in that those to failed patient care have ganaxolone room, in in more primarily with conducting We That’s study. XX,XXX means established the We patients of that with RAISE participated As compared stage enrolling the epilepticus treatment keys last convulsive enlisting be in

with progressing dose completed important It’s this in ganaxolone, plan exception the therapy study The safety duration The the the doses placebo. X enter a the of have note of design who required. and patients sequential informed for failure. versus carefully We thinking RAISE to be IV initiation ESE we’ve following as propose assess to design several a the after AED double-blind that Phase are would of novel and optimal at FDA to early the benzodiazepine the time refractory process initiate status a we utilizes XXXX dosing the the plan distinct epilepticus, and study condition first with to that same from study the from durations to to who those qualify infusion efficacy trial involves trial. of of ganaxolone So about will trial we consent.

to So established up epilepticus, immediate supply the status on ganaxolone super-refractory continue emergency this study. we the the our use medications a same to trial consider of can on formal in sites in for in about compromising horizon the though enrollment status emergency IND condition Quickly following response for RAISE Scott’s trial. without requests, is comments we not

neurologic ganaxolone and We’re about to a dosing thank to the again, who significant and their lot the throughout all knowledge these of making Through the Dr. is support, trials. this orphan suffering for patients in of potential in that, condition, lives Aimetti. I’ll families which paradigm progress the in improve very turn efficacy participate our efforts treatment these debilitating patients from Once, to like we’re call your families continuum informing status our dosing With learning and Alex the epilepticus. over serious disorders. Alex? its I’d about

Alex Aimetti

Joe. pipeline, Thank achieved share progress as development Scott ahead. entire clinical I you, have and our promise about Joe’s that for lies the what excitement the as across we well

some provide those me let of So respects. into insight each additional

broad clinical for First, indicating additional the efficacy signal Study across from potential CDD subgroups, the American Society communicated patients’ we at Marigold data presented Annual ganaxolone across the in demonstrating meeting. the population. in multiple for Epilepsy December, CDD we ganaxolone consistent a There data

this the longer improved see key forward correlated frequency in ganaxolone suggesting average medical evidence patients these a concentrations one need aims times of Marigold show effective day These of a a demonstrated of further trough day the the a XX.X% median X open-label lastly, reminder, drug motor evidence patients. are supports of first compared medication X that we demonstrate of said durability enormous And anti-seizure patient which a reduction ganaxolone to open-label In a we extended presented Phase anti-seizure including that when anti-seizure frequency that is have Consequently, durable can at broad this before, to important data ganaxolone ganaxolone as well suggestive effects reductions provided other data community on improve day X preliminary times need, ganaxolone year. and confident TSC, appropriate. where we dosing. CDD evaluated X the of a term effects ganaxolone of addition, went anti-seizure and dosing, as data and appropriately differentiator we CDD data, meetings. with And We ganaxolone, the in to findings times times seizure indications Study durability in dosing. analysis rare believe as for that would sharing We major as a high of experienced seizure to potential epilepsy effect approximately dosed therapies. which plasma the from upcoming with ganaxolone existing concentrations levels we additional X to demonstrating look higher meaningful study be support populations, effect chronic feel believe to additional and as unmet was be future epilepsy other a day dosing a provided to

Marinus call me excited broader populations deliver oral In consistent a financials. aim headed. formulations, to could efforts that, need. CFO, higher, our in Ed to opportunities of very to findings that progress recent has the patient direction which levels, With improved expand addition, our review about more support ganaxolone scientific Smith, clinical for in to and our develop these let I’m future ganaxolone over our turn ongoing pipeline the Again, the

Ed Smith

everyone. morning, and Thank you, Alex, good

fiscal and for reflect commercial ganaxolone support from operations results capital initiatives preparedness of results in development Our XXXX activities. and

the months like XXXX, months recognize $X.X XX, XX ago. year federal in million we contract, and a $X.X revenue ended December for of There and million revenue respectively. As were a the three no result contract BARDA

expenditures in increased also the $XX due in potential million in status including of CDD, based on X million to ended $XX.X activities and expenses the XX, of $XX.X net drug most increase equivalents million period initiated recently for The a year. and December At ended The $X cash $X.X month to increase operating same investments a and realized NDA of compared enrolment million options base prepare the potential expenses will per our million into in in $X stock-based activity, RSE. million, X million over three million be to $XXX clinical and enable capital prior XX, BARDA. million reminder, a in operating well XX, for million filing a $XX.X include may milestones, by for was success $XX.X company’s compensation year for preclinical which losses Phase non-dilutive XXXX, XXXX, approximately through and tick up was General million resources preparation December As and year for do million increased of the contract headcount current respectively, the the fund reflect could million periods December December in refractory RSE initially up months and in month to fees period in our of per the three manufacturing activities the of provides will for for and and XXXX, company’s trial range these million the over associated ended $XX trial. development primarily program our operations which as $XX.X and to as months revenue of the reported increase respectively, be voucher of costs increased $X as from XX trial, initiation had for with for third to XX development XXXX. or the company three directed million funding $XX.X that prior support Keep quarter with $XX used Research in XX $XX.X towards month year. the cash, consulting priority the year and studies the period same scale the approximately CDD us scaling BARDA to expenses ongoing the With costs administrative XXXX, million XX, as million. from offset months, same compared to we $X.X mind increased million periods primary XXXX spend a a and legal and in partnership. the same year with to $XX up to European through $X potential and spend of second increase ago. million, the to and the ended XX for fiscal trial We we to million rose ago, the compared could XX, XXXX, ago expenses of Phase $XX.X $XX.X over expenses TSC to of projections project This million. current the funding drivers December $XX.X in quarter, ago. million year were and the year-to-year to The not quarter Cash the personnel also the Compared the associated million year months in epilepticus. of their respectively, $X.X review non-cash quarter next by monetization compared $XX million commercialization. increased the and $XX

Before closing some for to confidence Scott for Scott I thank will be like our mentioned, and handing would programs. remarks, leaving continued in the call support back as investors I Marinus. to our their

call of be Steve, While now and who working at will an am excited and that of from now a to be for I proud I’m friends over journey. the the turn the in and some the Marinus and future highlight as organization many efforts for to Marinus to exciting with expect advocacy to future. colleagues be I’ve this and I transition will will I what of of the miss back turn and enjoyed next will leg Scott Sasha. I what Marinus, promises and lead Marinus’ finance smooth a to branding. rooting bright rest Sasha the the Damouni team promises sidelines shareholder,

Sasha Damouni Ellis

striving we their with the organizations thank strengthen to we’ve who We beneficial epilepticus that build to to epilepsy the groups can help communities. an care. to existing meaningful awareness and to broader to. progress and effort we to recognize organizations value bring believe communities Ed. field organizations education aim more disease working the these increase the work efficiently improve We together. new alongside We of tuberous for and within quality by the partnership overall PCDHXX, serve change in and of partnerships efforts and considerable CDD, collaborations Recently, status made are Thanks, patient key relationships sclerosis,

mentioned in Marigold ganaxolone principles also for campaign not the access Access we closely we advocacy and logo did mention are inflection to in stakeholders, awareness communicate patients, principles and at broadly a launch should are company’s potential for patients the rolled we With As families, that, call. Program, history important our have the working of our our turn these believe community, call providing epilepsy believe groups to participate to including community we our treatment with the I’d and portion point earlier, branding with guiding who Study like first out like the would investors. has community. that I patient commitment, highlight to Marinus Scott We of the to that our and our Scott? Q&A and to throughout we an before their back enter medical community. innovation new the of the Expanded the launched

Dr. Scott Braunstein

again, future his best of Marinus, you, here to endeavors. Ed on the I in everyone Once, of Sasha. success at Thank behalf want wish

questions, final to thoughts. a call few let just the me conclude Before opening with

incremental we oral the Importantly, development of for that to PK efficacy. hopes dedicating future ultimately improved clinical resources achieving could lead properties formulation are in

a recently efforts. series filed ongoing of patents these that have support We of two research

goal clinic least in one current in second-generation XXXX. formulation have the Our is to at

expand our and continue to we Additionally, organization. strengthen

including legal We name hires regulatory, throughout few. have quality human resources recent added clinical operations, the high a to team, and just

our to CFO the team join next thrilled month. Steve Pfanstiel, have are incoming We

finally, call as the everyone tirelessly families, how that are of dedication the everyone when been and feels at as efforts study we who by well commitment let for through grateful want we of continued sentiments team say, ongoing the thank and open the commercial our me head the Operator? patients And leadership We echo has in that, for to as site these Shafer personnel, teams the deterred team expect to round been for worked trying sales And and thousands like to of their of I this has with and will calls Marinus, Christy not company’s organization. investigators, our of by questions. have headwinds the what marketing supportive here the now hired of by Marinus through created also the out zoom times. pandemic. senior year, have the both continues middle her I

Operator

comes Your and Joseph [Operator from from Instructions] Company. Cowen Thome question you. Thank first

Your line is open.

Joseph Thome

and rest there. you to team questions Hi, for some the of Ed progress. Thank and congratulations on taking my the great

just on First TSC. me question for is

and out. you met analysis the safety laid that targets indicated the efficacy You interim had

If detail about what pricing a that? efficacy has little of and are some that target was as decisions that how how into gone second, into initial move gave more TSC bit here, just -- you you you approaching what could give us Thank kind maybe maybe CDD And on information you. data positive have to the and thinking confidence you Phase X? then, are

Dr. Scott Braunstein

This Joe. Thanks the Good is morning, Scott. for questions.

handle the question. me Let TSC

remarks, were in a patients having criteria So, mentioned a response. go important to decision. looked efficacy his we at number that Joe looking X, and prepared PCHDXX in meaningful as view no is consistent we and we’ve what We clinical an CDD for at go, for what now similar as signal looked we the of Phase overall overall seen and several

of we a seizure and in and we see types wanted Secondly, -- lot expand Joe looking I’ll to differentiation Joe that. spent seizure in at time on let different a types improvement

Third, patients we important are would are finally, important either -- patients a clinical analysis failed for we what for standards-of-care, patients responder response patients were and we’ve had are it us. we who standpoint see having XX% efficacy Epidiolex, refractory of the that XX% meaningful think, study, we or responses. And I several in was from typically had And Epidiolex. rates to looking go having think, that at about an looking the who where critically were decision Epidiolex who a Phase were all is think percent I on who including X was in and we commercial --

in So, our all factors of those played decision.

to more going any not on We’re specifics give that.

to over -- our more that TSC bit to to our on on a turn anything little on I Christy you -- decision? want talk else Before about Joe, add pricing,

Dr. Joe Hulihan

types call drug efficacy the so wanted do drop showed in seizures, across seizures, -- make predominantly TSC often major Yeah. In from the same we we just we know patient’s generalized saw trend the And lesions seizures focal in that seizure and sure have has CDD, they good because efficacy Scott as seizures, motor Joe. the Well and in start in the attacks seizures the hi, one seizures. we focally. we what in mentioned convulsions. Those generalized focal location to signal brain, there. and are

So that was part of the decision to proceed.

Dr. Scott Braunstein

and little Christy, pricing. want a to do you bit in talk about jump

Christy Shafer

a for to Yeah. Scott. Absolutely. It’s meet you, Joe. the question. Thanks, pleasure Thanks

value and can both for significant oral IV to franchises. dedicating time of pricing imagine, we’re construct suspension the and a our our you amount As

hurdles CDD, for bit value, done a that’s So specific work access journey, patients. our establish patient to our we’ve understanding to and our their been a any quite there are of landscape to dedicated treatment

lot and understanding optionality. for be CDD a will of indicated that So only first the we brings

thoughts our we’re However, designing indications. around both

CDD and launches out traditionally orphan points. look defined TSE both in of space the reference to and have into and disease both model fall to So prior we as

So, nonetheless, we in highly differentiated. address ganaxolone unmet the have significant fact the and ability confidence it’s need to that

So on where that. price we ultimately reflect will land

Joseph Thome

Great. much. so Thank you

Operator

Wainright. And come will from question your Tsao H.C. from next Douglas

open. is line Your

Douglas Tsao

questions. for Hi. the taking Thanks Good morning.

curious, evidence affect focal TSE future epilepsy? up what potentially in seizures. to about a gotten thinking that of I’m How that as -- mean as Joe, the you as with we’ve follow Just well in well effect data, obviously, that some onset Scott, does of general, development? now both as your just does for as the comment, last across terms development

Dr. Joe Hulihan

question. Yeah. good That’s a

broad about the agent. drug a it us gives think spectrum confidence I being

remarks, failed the Phase studies seizures, larger in it Phase one X although, the it Phase did to As X, focal not. didn’t efficacy I study study mentioned of show older in in X the

then now levels important about day. gives the it’s PK reinforces dosing this seizure formulation X also this to of adequate a confidence further it throughout with times us types So and day with aspect the have

it I So as about us well. that something tells think

Douglas Tsao

advancing also down, a sort Thank good sort what’s of balance how Scott, of to advanced perhaps or having of And with the think about could that slowing potential obviously, effect quickly be into you the lot the those how you’ve current weigh you and formulation, better formulations, advancing clinical you. commercial of versus just that do sort formulations could a clinic, quite new have effects? seems which but be do between spoken sort

Dr. Scott Braunstein

the for Thanks question, Yeah. Doug.

that to somewhere what think we’ve trial our PCHDXX certainly PK, seeing shared of we and patients out serum clinical bioavailability, having clinical least I a is think drug consistent between in tied XX signal meaningful meaningful response eight the the you, response absorption seven and look at data, we’re of clearly and with in TSC the at concentrations. and a early CDD trial,

a we Doug, research meaningful would drug. absorption. if very comment, oral ever can I our suspension market good a day current your X all very effect that and we and XX in So dose And convinced our these nanograms meaningful effect said, that’s are having. titrate that felt best XXX the I’ve have like have that’s patients number we from XXX feel durable a patients, physicians and make certainly have know if important to per we dosing. the see to a being better. this about That drug nanograms good patients, blood nanograms XX on think, we having physicians to times ml say, quite patients incrementally between opportunity having let’s nanograms physicians that ml, somewhere to -- I’d the are per per have love titrate and ml per give level absorbing to are still that could to ml seizures seizures would of I

we’re We of different targeting expensive. the amount place. So We’ve not certainly Doug, those formulation few a -- will made about They’re parameters two two a it our in we programs in we improve believe a tremendous of it we’ve have drug. really talked and incrementally progress. plans second-generation lot, the efficacy that

in novel at series now we’ve a commented As filed call, patents of formulations. least I different on two the

incrementally we’d We’re our two hope excited possibly ‘XX, in we pretty in can to the to and steady in I the of quickly. the of you’re peaks to move smooth clinic of looking about would troughs compounds and drug. again, a doses. think is efficacy be hurt for think drive state And looking these that the And drug ‘XX, never and that one it we have

stop me let there So Thanks, and operator. Doug.

today. to we’re little time on Let’s the because tight question move on a next

Operator

Okay.

Cantor. from Young question from come will Your next Alethia

open. is line Your

Alethia Young

and the my with taking you you. for Thanks wishing and Hey, Ed question working great guys. all best

decision event So low refractory seen there’s for asset, the protocols to was of Is And more color Europe? well bit to it’s anything a characterized different think study make there? attributed about talk was a with pretty little do status? ganaxolone? Thanks. I that know just you rate, potentially relates you mean, the to it that to about there to a PCDH couple little safety kind there’s another make it just get you a but separate to seriously in that one, treatment any then and study in there as just I can want is due the talk bit can your question, RSE,

Dr. Scott Braunstein

could I those. Alethia, take Yeah.

in a felt see much a treatment for And behavioral separate brain. having and patients different in is and status number study. was IV the seizures characterized So a take drugs be Psychogenic it I sometimes not of particularly that the called U.S. before there’s for It’s than no serious event. the something the status. as severity admitted more for to electrical first. was adverse we symptom I the characterize what in that a event non-convulsive that, and terms one and to patient minimizes the to EEG because monitoring to rationale step terms, to the of was meaning versus in non-epileptic it’s as non tend hospitalization, in of. means the for anesthesia a of progress the contemporary that -- refractory are of The moderate. Europe, event, discharge that less be seizures, The -- serious treatment attributed adverse characterized event, initially say to terms U.S. It drug. anti-epileptic think They’re was inclined won’t use Europe. needed psychogenic investigator taking that seizures to for different the anything

And different so study feedback. a over It it considerations and into design based evolved our time. evolved on regulatory

to it they differences have U.S., duplicates it complements study. And study the other that In complimentary actually going a study. design make study least U.S. the I got one. think that U.S. IVDs are rather least fill than two at be we’ve EU to at there is and the a now to

are with pleased things very So we’re going. how

Alethia Young

Okay. Thank you.

Dr. Scott Braunstein

Yeah. Sure.

Operator

And Truist Securities. your next question comes from from Lee Joon

line open. Your is

Joon Lee

taking our Thanks for questions. Hi.

of many meeting see total is Have set data statistical for did TSC its see full any Allo-S, in that stratified full interim work so, with meeting opposed that For to and interim to patients the the on strategic lastly, in of data? not broad reason would are you data any on TSC XX part planned suggest And program, disclose look? that in any changes did the as that just if the there the quarter with is quarter able the you you. And were done second third the you how of with expected? no further also and study -- also difference across based the baseline sort Thank based Board? FDA

Dr. Scott Braunstein

and So, for it’s jump can about add we a regulatory great to if then and want talk have Alex anything has in the signal? and to just question, Joe Joon. then you strategy the Allo talk TSC maybe about Kim,

Kimberly McCormick

Sure.

plan made. study to been the study. to with of the not Phase FDA request to submit completion entire until of submit new takes the have There X a cut, in documents once -- So that discuss the we’re waiting terms briefing is we time do the

by end subsequently -- and on Europe then of that target So second is meeting the in that. quarter after shortly

support the program Phase interim we forward. data our X, robust to plans the to to moving is to that support the FDA So are take assuming X the look that the enough feel Phase

Dr. Scott Braunstein

So, final asking rather the more interim was cut than Kim, little cut. why maybe really the specifically, Joon, a bit

the you talk a little terms agency. bit maybe with about So meeting of and our safety in should goal

Kimberly McCormick

Sure.

the our I the well study, that cut based think data the we supported agency. safety for largest the support I our CDD as important is to as X, on that the have of enough and signal the from other most what’s very, is Phase interim the agency database feel strong we’ll a take safety by strongly think and

Does the think and to don’t Phase the on to it’d be study have order collaboration question, get full I in X data that design. answer set Scott? agreement So the required

Dr. Scott Braunstein

four looked same efficacy thought weeks. our reminder, XX-week who is of the just been a looked at And the really received study when have at the the ganaxolone complete [ph] Joon for -- Study, it periods we I population least the Yeah. to the half at we had is drug as discussing as at the four-week at Marigold period. about

at study were we half able take four-week a set. data about least biomarker? talk a at to and look at Alex, So the about to little the want look bit the you

Alex Aimetti

guys, Joon. Sure. for question thanks Thanks, and the

just it in talked in on the I TSE, to I use PCHDXX. quickly about before So of the sulfate wanted comment allopregnanolone

appear of levels to that can for day appropriately higher predictive a proper suggestive Again, have a we biomarker the response. dosing recognize is epilepsy effects design, and seeing case. that probably PCHDXX think, is effects, of And a these ganaxolone’s on whether positive CDD of data that I to value. everyone’s As not sulfate with we’ve endogenous And beneficial the not ganaxolone in a although predictive broad the does allopregnanolone hypothesis high degree broad surprisingly be result as here, response. study now today with times of shown better was population that I’m I the population predictor in X a the a clinical could that potentially test say confidence aware, trial the effect designed that

is your enrolled. study primary that not endpoint question, baseline specific to the are in levels sulfate Now but are in going to we patients, stratified all explore they patients allopregnanolone TSC in in efficacy

Joon Lee

you. Thank

Operator

Goodman Marc SVB from come will question from next your And Leerink.

Your open. line is

Marc Goodman

of talk testing, be sure there about the can when And is that? and baseline behind kind it? you. thinking increasing just you’re hits you’re partnership Will of European Thank just is and you just be product of testing the optimal to Will us Scott, then us with CDD? for about that? economics can testing to you TSC, going how to market? just give us complete what for there so just understand just What some on we deal about how remind make and the scenes secondly, thinking your

Dr. Scott Braunstein

terms bit the as market we’re the where testing of some from pass Thanks, a well, a Marc. of And our trigger maybe because little of over I’ll lot of to feedback Christy diagnosis. research some in partnership, in the with have several on focused the vast are Marc, great Europe. specifically majority of early terms organizations, Europe, in are we’ve points some But discussions outside reach several had getting of

specialty focused who focus, to on We and of some They us. are a business. certainly, orphan folks disease they have have that’s intriguing of -- the are more primarily

We and to level different have hospital have interestingly strategic certainly and interest both of an other the who that orphan in strategic a us. has interest partners space

what’s Now IV rather for and -- than We we the opportunities certainly have commercial good sure from that take later. of sooner on full the confirmation program. the in want needed Europe make about we thinking to advantage pricing that

we IV important we’re there’s to ourselves. think think development the us, continue the the IV going a rationale partnering a the still ahead for I Marc, oral strategic rationale partnering either So for There’s would European the program we global and I effort both of way, program lead And research TSC program. and just single programs for partner. of which decision both really have to program

thinking really the of we’re NPV about of economics, long-term the the terms In business.

We in have a the of lot confidence business.

payments balance Our will sheet research it certainly reimbursement than certainly upfront is around stronger was. runway. some But much our development and extend and

would in us But interested really of and as grows. are focus are to think, for So reaching are is parties. that commercial upfront, And, a the are us. going to certainly, like, we both key research the product combination I important the for really again, NPVs as expect, equitable reimbursements look royalties particularly what you

-- did missed, -- was question have another So I I your I think did there that question, I you covered Marc, apologize.

Marc Goodman

Yeah.

testing? genetic Just on the

Dr. Scott Braunstein

sorry. to jump Oh! want you Genetic testing, Christy, into take that?

Christy Shafer

Sure.

six in and few years and present acceptable these used the within to past genetic tests. that months over to to seven first a When physicians specifically are it’s CDD. for it widely early widely have there that CDD become for patients life, these acceptable the panel look of testing So life, years typically do several has genetic

in diagnosis on. CDD something rely that is will early we So,

we of know, years different their As tend cycle first these to anti-epileptics life. in through few patients all

of these us the age they have be two, refractory. time XX% to considered of patients the will by they will at get So

high us a the So population. early the rate bring will diagnosis large part we believe, patient again, refractory and of

Marc Goodman

Thanks.

Dr. Scott Braunstein

Thanks, have for on last and also question trial, little take Marc. we one posted a new we the the design. the And TSC and time have and as of our slides a set of we just investor wrap PCHDXX bit do slides Operator, we website Phase more remind those our community trial more question, that to wanted before well as include analysis detailed X

for So an we to Operator, our just individuals. as question? last don’t FYI why move

Operator

Okay.

from So Jay question Olson from will final come our today Oppenheimer.

is Your line open.

Jay Olson

feedback SRSE study? from and or one Hey. you the Thank are that Oh! the might table study for not at a some that design thanks you compassionate this clinical decision anecdotal a But not all physicians Can work consider that in of time. who share question. SE? do use didn’t stage on progress and future the in or you’ve the to would influence lessons from patients to think on the Congrats in your for SRSE And of what super how you. out taking that you or I learned study received gotten a is ganaxolone refractory said any trial the

Dr. Scott Braunstein

thanks, Jay. Let me --

clinical we to drug a If support an Let community. we going on going question bit requested provide the little with to drug certainly, But pass that can. super we certainly status, are for me and experience. basis patients are of research over refractory I’ll to Joe EIND when is take for the

at going we’re dose been day, up of be milligrams these can extremely that Phase patients up per Captisol. that are learning difficult. going to XXXX where to physicians already XX We’re or X grams We’ve over XX higher doses patients our dosing

the the know are there or RSE we so population. ESE real population in differences And to compared population, this

said, really continue right since we mistake good and don’t design. get setting with as of we I others to want even the beginning are and consistently, about we make very we the consider before could same matters have acute to want dosing support of I think research program, and made. So where RSE. IV type we trial the But any understand that differences want to the to in our feel we

in years clinical different taking well. We’re result now say, few unleash be we are that And I formulations, over the to us a on of as of may down SRSE we Joe ESE, new it Captisol turn call. you and on ultimately, creating currently, to a me but road some ability for will path. But ahead well, as development the which linked before wrap approach let the super our refractory different in status to approach a

Dr. Joe Hulihan

about with getting of a enough Scott. experience there main dosing agree in thing think No. initially. levels The is The we’re and higher have I using Yeah. dose. high learning I yet. we importance don’t

think look seven I types, eight think or emergency treated have I age. don’t experience patient been patients have we it’s at far INDs. been so enough to under

was and ganaxolone learned may and and to a dosing the may a from Some high Sage is the constrained there some so role of tougher lesson treat. level, blood bit to PK levels main the etiology, it’s patients get from study allopregnanolone. the doses Scott patients and were have very that’s quickly, And I important think, differ probably be responded they the -- done very well, the mentioned, so as

anyway, to continue drug explore of So, and status. -- continue we’ll super the dosing we’re different respond to refractory in continuing ways

Jay Olson

Thank very Great. much. you

Dr. Scott Braunstein

I’m like providing to and for I’d you. and going today coming and your back up to the thank wrap joining Well, Operator, support in updates to everyone look to call confidence us and continued forward we the months.

Operator

call. everyone, conclude will you, us Thank joining conference today’s for today. This Okay.

now You may disconnect.

Marinus Pharmaceuticals (MRNS) 9 Mar 212020 Q4 Earnings call transcript