MRNS - Earnings call transcript Q1 2021 | Marinus Pharmaceuticals, Inc.

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Operator

Greetings and welcome to the Marinus Pharmaceuticals First Quarter 2021 Business Update Call. [Operator Instructions] And it is now my pleasure to introduce to your host, Sasha Damouni, Vice President of Investor Relations and Corporate Communications.

You may begin, Ms. Damouni.

Sasha Damouni

Thank you. are Marinus from me Dr. With Executive Scott Dr. Braunstein, Chief Officer; Christy Commercial and Officer; Hulihan, McCormick, Chief Pfanstiel, Chief Officer; Medical Affairs. Officer; Joe Steve Shafer, Chief President, Regulatory Vice Kimberly Financial

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

Scott Braunstein

start. This financial Sasha off to you, business XXXX Thank and tremendous welcome quarter and year is first our update. to a

rare our progress The our submission, to our to agreement exemplifies management potential future believe ability the Board confidence and support our XXXX. We our end development access credit priority next and well with to as in that financing with as deliver would programs the and in to specializing our for have firm through alternative support granted anticipate be of programs cash advisory clinical completion asset capital Capital our use assists Oaktree to as middle European launch, research, management voucher of sustainable provides disease and The that runway of the NDA deepened investment submission first have our opportunities generation our envision year. a milestones, global us approval Oaktree’s CDD research by This million announced and continued We $XXX IV This ongoing preparing the intensified or efforts is we To capital a with that is in by to and being have our commercial address financing team, well deficiency providing this extending on formulation review made have launch. this today, of preparations we Management, partners, commercialization upcoming we next monetize strategies. make board meaningful financing to ganaxolone support track clinical potential pediatric funded headway as on over additional discussions NDA programs, step ability first year. of strengthened research company’s while is of in in CDKLX for the potential ganaxolone Directors. commercial continued across exciting for for this scientific the oral oral disorder, in this the long-term. franchise the developing progress,

ganaxolone’s early the sufficiency display and As to that The to process. strong noted Marigold approval review preclinical profile dataset open FDA from determined safety has a efficacy the since will Marigold been study showed that a reminder, favorable support the and data durability continues be of label the studies. during study

the a Let quick and submission with ganaxolone had remain The on give European of track me the we end has quarter. Europe. on Agency third oral EU our you by update to company a planned authorization surrounding encouraging Medicines submit application the interactions pre-marketing

a beginning weeks the in potential ganaxolone coming be if Europe over with commercial European partner to the to year. and expect We available to continue have have approved commercially months. in oral We would selected of partners next middle to hope constructive discussions

filled has first U.S. as call been Officer, Chief CDD from well efforts Shafer. team will This launch the in hospital Christy our arena. the as with We in both Commercial have space of been potential planning is the remarks fantastic orphan hear XXXX disease experts intensifying our front you Christy in that the prepared building commercial a midyear

for with to liaisons Our diligently educational their science efforts working the clinical are medical prepare community.

the Our characteristics message of of will binding. focus from and on and of efficacy, its based on extrasynaptic modulation ganaxolone on to and ganaxolone We durability. results have Christy. commercial unique safety the differentiated receptor GABAA of mechanism the that will action believe come and that profile plans More a our

our Let TSC me move to program.

line We XX around expect with the middle the community X third to from investment share the study data quarter. patient Phase of top the

design data study interim early complex, TSC of the trial the in completed based expected X have in on the Phase and third FDA ganaxolone or tuberous planned meeting analysis end-of-Phase with for is sclerosis We an an our X quarter.

are As for the in site anticipated to patient of the first fourth of the a quarter summer, the while latter Phase for planning trial portion is initiations XXXX. the begin we enrolled result, X in

We are of timelines of by our by to share Phase the for study end we completion expect QX targeting the end site selection of and X September. the

believe strengthened the advice We for To in with both and to with quarter that leading scientific advisory and X experts our of ganaxolone to today clinical several the our of in meet later this drug the announced to plan support clinical use play request disorders. and obtain have appointment efforts, development the TSC scientific the ganaxolone We fourth in early We we board critical trial EU the EMA the in an epilepsies. year. treatment pediatric orphan to designation a seizure on program. Phase clinical also further U.S. chronic submit plan of we in neurology potential has role expanded refractory

and Neuroscience I the Neurology section at shift Knight, at Pestana Head a the unit Joining our in of to Clinic; Elia Medical in Harvard Children’s Rome, General of to Bambino Dr. move Nicola SAB and Department Dr. School; Epileptologist the Pediatric Epilepsy Elizabeth new Epilepsy of Pediatric and gears Dr. are IV the like Specchio, Italy. members, Cleveland Massachusetts at Thiele program. from Professor Gesù Hospital, would Hospital

continue are know of trial refractory epilepticus, and status that to both hospitalizations year the of thankful for several encouraging RSC, challenging a tracking infections in COVID the significantly clinical U.S. the these we the of rate many hospitalizations numbers, and has robust. showing numbers below been RAISE in almost which that this for months, you are in news. first have I treatment Phase the Despite to the X trial ganaxolone patients interest continued XX,XXX new the COVID over is Our continued advance. past hospitalization of or right and IV time progress, be direction. U.S. to in the study move continues

the clinical to open a of this job sites centers major has Our team done academic operations in middle pandemic. tremendous in

to site We evaluate and open accordingly. continue enrollment

report site line in early per expectations. with the interactions development numbers are the that from It diligent Dr. We still in clinical Thanks are including total Henri patient happy is tracking [indiscernible]. team, our to to study. currently our

a affected meeting very this third be COVID describe where the European EMA, the trial despite the quarter. vast majority past with top first line have are dialogue sites headwinds trial in further at that for the a by was in open half begin RSE X named open the time, first XXXX. Joe half recently X activations More However, in data Planning will trial for separate A the of readout of XXXX in also we engaged by continues the end of the detail. persistent held to we months. are constructive RAISE RAISE expected reiterating trial recently and to the with half for site the that quarter, the the than second

appropriately a the European ability second Our oral and to both franchises encompass on our strategic would bolster believe execute trial. IV registrational we powered discussions and a partner IV

market. from our fortify education. registrational Our only X a have for to hope for site trial has efforts, We pivotal a More and the potential around that approval, is we that in but help European selection execution believe the will RAISE serve come implication to clinical place the an to specifically Joe. important currently have would not as partner trial medical global

financing amazing been to strong major ability and we team Steve his continue team is and CFO, Pfanstiel. credit the update and a build newly jump strengthen in business completion first leadership a As to not appointed way. be his the the contribute at entire been for but could meaningful Steve Steve skills weeks, this organization. has of and better organization, us effort will the call in in few for has contributor the conference a come with thrilled leadership with a time right only have the the a

Christy now tapping has and expertise we been approval over Equity we commitment, to her our is managing achieve culture planned and at involved us pleased pleased for potential innovation the to continue like welcome corporate into to and We Sara Chief Commercial call the Board first we success have and rare provide Finally, Pharmaceuticals’ X months Alnylam turn and Directors. of are Sara Dr. our drive I commercial in experience community. brings novel plan our to and Sara early thoughts build as our Christy serving development diseases. ago. officer. currently for drug on as am values as more joined as launch. core to would Diversity, regulatory her drugs first I Inclusion several our of global on her Shafer. Officer Marinus, to chief Nochur in to tremendous

Christy Shafer

Scott call you joining our thank and Thanks, everyone for today.

solidifying continues mentioned, capabilities the our commercial to and fully plans. commercial team strategic we developing plan. Scott are strategy its and in launch engaged As The external internal by broaden

senior the and construct operational leadership chain its turn finalized are sales the plans. marketing, developing by Marinus welcoming in leaders access, and QX, operations tactical who During supply commercial market and

team of as most addition Alexion. the Our joins who to President Vice is from us Lejuwaan recent Lisa Sales

commercial approved. a XXXX a continue of in epileptic commensurate associated of with team launch treatment the midyear to will deficiency NDA for and with ganaxolone disorder CDKL-X the submission We seizures if grow planning mid-XXXX

Our strategy launches. basic of is principles with orphan consistent disease

account the plan X specializing X XX to keep Medicaid. XX to organization payer we on and managers with lean representatives, First,

of on marketing and focused most and will efforts opportunity concentrated facilities. sales be commercial is high a in of number as Our influencers the traders limited top

underlying We have pieces strategy. and that informed completed go-to-market is several assumptions the market conclusions our core research to of

finalized work strategy, segmentation, messaging, and key given platforms. the has and such build our upon critical structure. journey, in elements our market We digital patient several have the foundation to process, print us which This now and branding, account size as deployment

a Marinus final a an From be with is and agency, our an platform emphasis the perspective, decision our made CBD design want selecting We of on external ensure of marketing we unmet the are needs proposition stages cohesive that messaging the pricing patient team, important in to collaboratively prioritize value will targeted amongst our cross-functional population. to reflected.

As those can to outcomes potential any access our managing economics complicating are we the mitigate efforts, and This ensure research and to includes payer reimbursement restrictions. part value bolster we timely and landscape generating of health proposition. assuring

to access Additionally, experts monitor we the continue and and landscape market engage the Europe. across U.S. competitive

efforts patients will them. these that to characterize true confident like the that our physicians We CBD, for their value with now families back the would to are turn and for living treat Scott. call I

Scott Braunstein

Two is comments as over but to-date in with both activities, our the deck. and I updates EU Christy. confident call Timing study you, I preparations to XXXX Joe. data analysis are turn The priority. of and for on track FDA the our and the and NDA diligent with our our Kim and clinical will the affect not in interactions May Joe from you U.S. me does before submissions, have discuss through Thank the a we the will corporate regulatory which additional statistical our top CDD data been regulatory am that EU final walk reflected conclusions the of authorities. to

to reformulation to in the into still move at first pro-drug ganaxolone and formulations We we in opportunities refractory these endeavors and least the half XXXX. Finally, for evaluate of progress one other indications. epilepsy clinic oral expect our continue additional of

afternoon, planned in as we all formulation. As the middle on with potential if this of X or announced Phase we XXXX initiating LGS new a a Lennox-Gastaut Syndrome, planning and are in trial proceeds

population, Officer, meaningful would Given market data With and the in differentiated I and TSC investment types ganaxolone Joe? with well the competitive Joe as the encouraging clinical specifically where turn highly in call has Hulihan. patient enter the is best believe is we to warranted. our profile. look market overlap to that with over the a chance like now seizure disorders, understand of the the Medical LGS early the other future We Chief potential to etiologies shown CDD would as that,

Joe Hulihan

to business you, Scott everyone our and welcome update Thank call.

majority or the of get in I quarter. sites have sites of RSC. COVID-XX our the vast me it. the the RSC we with into trial, end more will the start by of middle with RAISE the than with year, status right delays, epilepticus, program open still Let even refractory open third the expecting are to half In by

we rate are that are of to patients to-date. pleased We the and are enrollment screening with see appropriate encouraged sites

screening control design carefully with that Our rates very have we response arm. we experience conviction study about reinforces thought the right and our in the

Let patients have a The that protocol requires second me line failed explain AEDs. bit. that two

failed failure We of will they believe have AEDs the response after to of they two asked second failing quickly can another. be the sites enrolled to so study first that requiring candidates line lessen pre-screen We standard AED, after line second care. potential

In fact, patients likely some in responded those been patients had placebo we enrolled AEDs. of actually they have the second would to those it’s responders. the have trial, If

study. strategy is believe we AEDs requiring So, line in two this defining the correct second that population failure of for the

with and next but U.S. and RSC were approach failed And benzodiazepine, criteria could the EMA RSC influence of the must first a of trial RAISE trials. for the initial the Both studies continuing some plans both, have for key very have is are we in placebo-controlled major in are establishing have The to had hear and agreement to treatment a We version launch such design an Midazolam. patients in the European status pivotal endpoints. designs, which Europe, treatment that guidelines trial studies named European our dialogue XXXX. of one for similar targeted Lorazepam and pleased half productive with there X epilepsy. Agency and registration of We as features of Medicines on our studying leadership treatment epilepticus, study are double-blind differences. enrollment Since the reached goals in we study

study, AED. study, However, in that AEDs. is failed in to as must will at IV patients fail the of ganaxolone started they then is The time this have mentioned, as U.S. second the standard placebo trial, IV X, second for European need U.S. one. RAISE unlike least only in two I line the care at the But same the or in reason

We are placebo thereby higher response. better the a aware RSC care response of of allow that course could a treating to standard earlier in producing

for two early However, that placebo trial hours maintain possibility intended other to other the treats rate. the nearly and of response Europe we care anesthesia in treatment U.S. for at to response. of progression a the within the physicians has features don’t co-primary AED have The U.S., IV endpoint be have anticipated of any into of Since IV prevention the study modified the use we or to durability looking endpoints: low at IV much assessing the European incorporated status as as response design, and that anesthetic escalation anesthesia I XX one, status. trial in durability whether an another

cessation is no EU combined hours. within further the success a for RSE. of having increasing endpoint, in XX of the within the broaden Importantly, a U.S., The each the i.e., other, defined as preferred instead analysis. program, endpoint now study having are is of to regulators in potential chance co-primary we care use that differences claim efficacy responder the A responder to and minutes of and that in XX status trials design overall the IV ganaxolone these endpoints and complimentary mean for escalation

a patients failure you Next, as like continues following I on status and status of treatment epilepticus with of exception defined RAISE and would FDA. to who ESE the ESE status than to patients status who the would and note status. the important in type enroll earlier or XXXX, The have RSE epilepticus. trial plan trial from in distinct alignment from gained than from have have study We qualify the convulsive is process update will initiate departments trial, completed consent, continuum those is to status for It’s epilepticus an our that ESE. study enter of we establish informed predominantly the the who will early non-convulsive rather of the ESE after give with benzodiazepines. emergency non-convulsive

of initial line without trial as which study, is our for ESETT intended to In same an standard we ganaxolone RACE consider established the The the believe be first rates ESE IV and hour. in care, reduce the So, to the the establish effective than XX% study sites the epilepticus to standard potential response is AEDs that to enrollment durability second We adjuvant showed care the of can status of ganaxolone and will response status AED. has of standard study. time that less act increase to the within care AEDs. IV the compromising

of same the following The durations and a assess to and tolerated AED is proposed study this of and safe in the trial a design design sequential safety dose the efficacy novel doses infusion at ganaxolone, study The Our to of the failure. first time well FDA goal to the benzodiazepine involves as of ganaxolone. utilizes ganaxolone. initiation find several

X the progressing versus Phase blind dose placebo. double a to With regimen optimal study

oral would to related an medical like a from a give PCDHXX scientific update to for meeting quick presentation study Now, and in our and I still submit ganaxolone upcoming results for Phase you our the on X and epilepsy, In we plan journal. publication programs.

We is due study the intermittent and to condition of nature have this seizure variable a recognized one difficult that to activity.

in will we any in patient offer are mentioned, to submission in mid-year wishes we filing quarter. who FDA to study the third with on ganaxolone a track Importantly, treatment. participated as the the continue EMA for and and CDD, Scott for continue And to

meeting data We including for from December, submit abstracts the X extension also Marigold open the in the study. plan year of upcoming to a AES label series

and to ganaxolone reduction we open year XX during XX.X%. are seeing of had from patients placebo months on XX.X% of XX data who percent of treatment, who blind. in So X label Those percent far, and been reductions patients double transitioned achieved baseline ganaxolone, on who on and label reached XX a open with open are label

statistical change major ganaxolone group across prior placebo, in approval. of U.S., diary than and have data are and top the from of there data with of were with entries. in work centers potential early FDA secondary for top quarter. changes With end discovered in resulting endpoints, the point outcomes. expanded primary The conclusions This less reported or top to the fourth of a XX.X%. X.XXX. the a were The percent X% U.S. duplicate committed data seizures data recently study the presented available program. study. we showed a regulatory the in planning access was to reduction set we ganaxolone with and continue X.X% there X.XXXX. clinical X% line that with These motor And a seizure was We the final several small third of not seizure previously the value shown entries or the who the We analysis the with data data reduction line between patients changes in the submission. for that And to X some analysis affected with EMA making we XX.X% do p meeting ganaxolone, total transfer it error. CDD And Phase differences show the statistical included regard diary no previously to validated XX,XXX TSC, for over an other interest placebo p value key endpoint Though to for affect the line that safety of are quarter

advice with are receive to We our the meeting on FDA second a submission. IND anticipating scientific also

that first XXX And We in a enrolled with will the the quarter This least We taking failed enroll of used AEDs. the EPIDIOLEX. will medications. expect prior study, allow EPIDIOLEX concomitant study range And of the failed two X be and everolimus, taking will Phase who that at or inhibitor XX% seizures, which trial anticipate study fourth we tumors. having our enter participants also TSC associated study of treatment the enrollment is for to taking patient of roughly this our Afinitor TSC patients patients mTOR for study year. will expect unlike patients of initiate or

We are this Lennox-Gastaut also new Syndrome. including exploring indications year,

our diagnosis that etiologies. genetic a is other can several of had different complement epilepsies this of TSC and epilepsy thinking represents have underlying current diagnosis, pediatric patients Our a LGS studies, work number in LGS. who CDD clinical the we and that Unlike carried would syndrome our have as a

more of will year. a share sometime about thinking our later We this trial clinical

supportive thank always, advocacy the been groups of closing, As to efforts. and I our patients, who would families, professionals in like medical have so

call regulatory for would a to I turn the over review. to Now, like Kim

Kimberly McCormick

interaction EMA, for receiving Thanks and XXXX an the the wanted Joe. of the pre-NDA August in Marigold with community, FDA Since our we CMC been regulatory from investment uptick have meeting in we study. with questions to the of held was a communications discuss FDA

the and the X legacy epilepsy the meeting, meeting the format a the achieved. in most FDA the to proposal, had in CMC adequate NDA Marigold FDA study submission agreement details dosing with trial, provided that we one our demonstrated PK a in overall to to CDD. we meeting the the study, NDA discrepancy study reviewed efficacy, ganaxolone explain aspects the on in was that show We addition in efficacy outcomes During the a of CDD and adults. in to review information provided very that Type feedback seizures request, of trial. support Phase the January, of Prior content of support for company the failed the constructive section has X differences of that and particular study focal the would our A FDA’s on studies and Subsequently, believe conducted in notably, that key study Marigold be at in Phase Phase by to X we

to the and pre-NDA FDA with Response March responsive, noting NDA in first from meeting filings. what been at Overall Rapid our the the and information data study agreed the of the information the We study The Marigold achieved provided, worth the co-rapporteur meeting end the final discuss they are the and and be on during U.S. see adequacy format. by very pre-NDA what the that data FDA of for submission had MAA. a would engaged would meeting to being meeting NDA NDA completed is quarter. for potential the was quickly that We analysis we any our review for NDA approval process. the determined FDA. in remain FDA of in and EU on the to be provided feedback set adequate the up requests review and wanted Following and data the that the Teams fully and to that with respond support of filing, track supports the believe has Marigold we the regulatory It’s the agreement pre-MAA XXXX. on of format with in May. we and end MAA EMA content successful to rapporteur with the planned A the of the constructive

like Steve over I Now of financials. review would call the turn our a to for to

Steve Pfanstiel

I results. share to you, pleased financial to our able Thank Kim. be am

first first with the from revenues contract are in federal For in associated contract no September of million there BARDA XXXX, so $X.X we to year. of XXXX, this the quarter revenue prior the was recognized quarter related have contract. This the signed

expenses million clinical prior success million. The contract with year. based base RSE prior for XX this was our extends associated million and million extend and XX, $XX the clinical the same our drivers reminder, XXXX versus increased within million prior expenses in ended of with administrative prior based reported period year. $X.X non-cash same non-dilutive million in BARDA and to contracts for X for the totals to loss total months approximately the the These $XX.X potential change XXXX, primarily scale million increased for $XX on support expense a through $X.X funding increased March General respectively. March to change in to expected a stock-based a compensation further operations, ended million to net X $X commercialization. of the for increased year As the exists months resourcing XXXX, to ended a milestones, period to as the of BARDA March year of for funding company The costs and for of and activity, were and The the months compared including XXXX, as preparation X Research the to million development the include potential as company’s primary is of million due ago. $XX.X $XX.X the compared Phase over well third trial same $XX X increased XX, $XX.X total the time. up The the over compared of to up XXXX primarily teams. provide CMC startup QX of of XXXX, the quarter in year period and

The for XXXX CFO. our XX, severance a months to of a included to $XX.X compared million compensation $X.X based of related stock used for million the to with first quarter March $XX prior agreement X the operating ago. Cash same million in XXXX, activities increased period ended year

Oaktree to credit balance, the quarter As scale net upfront capital and expenses the of company’s enable second us of fund of agreement of expenditures March XX, This combined XXXX, operating will million. we had cash and proceeds $XXX.X cash through equivalents current with the XXXX. of

our business Phase X our we work executing the look the support to X CDD of RAISE focus on a investments accelerate and As credit key and result associated an approval, the several including the ganaxolone. and within review voucher priority plans facility, a additional to trials, partnership formulation anticipated monetize to activities with related to TSE European European approval,

$XXX $X $XX revenues includes million As approximately for and BARDA between estimate and between million these we this, million fiscal $XXX $XX This the operational of million XXXX. for year expenses, Offsetting estimate expenses total of the a XXXX. of compensation. stock-based fiscal we million result of year

we with completion the Capital upon In successful quarter million be addition certain to XX, the agreement, credit $XXX details first to subject down facility tranches access financial to Under XXXX. additional this financing Oaktree results, to signed to the I credit certain ability on milestones. to conditions in like of provide of would up taken May have of

received remaining The a already million funded NDA initial have with FDA FDA connection in payment available based clinical, each $XX at certain and on indication, the with one million $XX achieving the additional be milestones. our CDD and of of million Two upon the We financial at acceptance upfront progression signing. discretion second of occur commercial $XX our filing approval. funded upon successfully will tranches

see runway Importantly, potential to the With excited not be ability to a part review extend Marinus potential me Marinus rare experts. this just few encumber of voucher. does commercial and priority facility top been the a let such incredibly epilepsies. excited on significantly of our a I cash leader and first weeks that, alongside CDD to monetize My based indication say notch this I in to am our am provides have group productive, company work alone, and at the talented and become

to turning Scott, Now, concluding back call will provide who remarks. the

Scott Braunstein

Thanks aboard. once welcome Steve again and

Before who significant prepared progress to has the I we have remarks, entire team the want continue and been bring to challenges. concluding ingenuity thank responsible to for solutions and to strive our made Marinus

company’s milestone hit has now and headcount to be Our about the next could can I a more stages of questions? the to Operator, enthusiastic history. close not in employees, the XXX call you open

Operator

Thome Cowen & from first Instructions] Company. your line Joe comes [Operator with And of question

Joe Thome

can be there exacerbations any on worry you any of for that and ganaxolone taking interactions with is study AEDs in Thanks. morning potential could thank drug the X RAISE AEDs do AED? in ganaxolone? Good And Maybe one any there. used consideration combination when or the other my there for questions. you be Hi just which conjunction design. of specific Is there

Scott Braunstein

investigators other It’s The AEDs choice. No, on question. limitation many. the your AEDs. isn’t no number for thanks is of that there Joe, available the

that we randomization balance but will are drugs reflects we get what will standard out expect so, do of care. used and And

the oral drugs. In is on expect interaction to in see efficacy trial you high use study, is terms enough. other inducing of of will drug didn’t potential interactions, there any the wouldn’t CYPXAX especially would based in effect, drugs. be dose any But an with ganaxolone all. which We I the we And difference the used any at use. see of think factor interaction, no, epilepsy don’t in a enzyme that

Operator

of the And line comes your question Fitzgerald. Young Alethia next from Cantor with

Alethia Young

congrats question been... This taking for thanks has Hi, my the progress. and on

Scott Braunstein

Thank you.

Alethia Young

competitors? comparable additional versus you will see? And the what about For of data TSC full how what is we think readout, data do Thanks.

Scott Braunstein

it? take you why Joe, don’t

Joe Hulihan

I today, else let to anything going work. do all To do am everyone Yes. the

key Alethia. study So, Yes, of are focuses safety X data. Phase think and this hi efficacy kind I

there differences So, study is if we obviously, data. or other combinations, usual the this the with in – with seizure disorder see interested the and different at seizure of ways be we one drug things in different ways will seizure also those how types. EPIDIOLEX we in then Afinitor look than particularly some The any to safety some see And types this data, CDD. in are are

a X the patient So, outcomes the range we the on other but are Phase at. those In reported data of will things looking we are and key we the are have assessments, main Phase study X. from focusing

Operator

Securities. And from Joon question Truist comes your the with Lee line of next

Joon Lee

taking our updates. questions Hi. and the for Thanks for

there if For draw? each the additional loan terms with any flexibility And the are interest from for Oaktree, doesn’t any expectations? are what is for funding call-back reach certain the provisions further outcome And indications? there

Scott Braunstein

Joon picture, hit the question. and the So before on for Steve thanks to me I just turn big over let details, for the

think quite in deal. thrilled is forms And to could a walk But of think you many in will partnership time. over through a partner. been are we different that they all, the They have I taken have a team. time specifics fantastic And lot have great. do to with I this I of expand thoughtful Steve the They we that Oaktree believe have this been deal deal. of this this First be

Steve, Joon? to for have as why partner. through happy details all the deal So, them you don’t the walk of a But

Steve Pfanstiel

Yes, walk Yes. you certainly happy to Hi, Joon. through.

have rate the couple based royalty of So, financing as a a we funding, that take that million it’s we do we The tranche a is it European clinical, and think interest CDD years million a for filing up do interest-only mentioned funding alone. out, able financial, indication worth million starts the calling to PRD, The acceptance, days ability we to U.S. partnership. I worth after monetize can things from ticking still XXX noting, the synthetic are still tranche to X targets. I can It’s the is first then have $XX remaining and well as is million tranche the obviously X on driven maturity $XX the $XX relative of fee is first to $XXX XX.X%. there the of $XX CDD think it’s first years. commercial we There full million by it’s after

a So, key those of number kind the those things. of are are

In a go this further, we we further something as with terms expanding can look that’s as of think and Oaktree. we relationship long-term I at discuss along relationship, a

Operator

And your next question SVB comes from the lime Leerink. of Marc Goodman with

Marc Goodman

LGS can this made little what bit Joe, indication? you hi. Yes, you Thanks. that pursue to want about you proof-of-concept a talk have data

Joe Hulihan

Yes.

LGS specific etiologies. not It’s diagnosis. have did diagnosis. a multiple It’s we a is genetic So,

CDD of some patients we carried LGS. that study had a So, also the in diagnosis

– data. it of think a many right number gives confidence in large But to the that without are we we started so giving too specifics, study, but heads an patients, I direction. not analyze LGS And that us it

have number study previous in data is And developmental could but use so way seizure of And a that to multiple had capture really LGS there study didn’t types, patients there we a proof-of-concept. the epileptic that, we that small in see whether a encephalopathies. in in was a it

me LGS the gives largely that are thing types similar other confidence The CDD. to about is seizure

us to so other better likely going it, more we would did expect to CDD. efficacy with etiologies give reformulation. I’d the And in a It’s same it exposure and we some if And the as even be pursue drug. to see in

too. I think So, a plus that’s

Scott Braunstein

and Marc. And additional Joe add I one Marc, comment will

performed day it’s Alex We we onset PK that think company. the performed now focal We differences and studies that were that by have updated are failed PK our we that slide clear three slide at seeing Joe dosing. previously course CDD titration signal in we we LGS is terms are a consistent Marc. function of seeing Xx a when look which over at study. think And study Thanks than of back the very we think I different dose signal in a for the question the the seen deck. have just the have And in study. what you the included of think a And levels month, in least analysis have inconsistency on PK, I deck, in or

Operator

next from H.C. Tsao comes of Douglas Wainwright. line question And your with the

Douglas Tsao

to to Thanks the Have Hi, engaged expand curious taking I for am drugs. potentially good or you whether about sort FDA of X label and trial just U.S. of terms I with opportunity plan the of you would sort FDA sort of RAISE potentially of engage do have utilization the move the in know questions. inclusion morning. mentioned you of the the paradigm? to standards the meet up in with terms that the treatment

Scott Braunstein

So, want Joe, do to you start?

Joe Hulihan

I this over Kim no, it. regulatory Yes, the of for one could turn piece to maybe

Kimberly McCormick

that. Joe. can Sure. I take Thanks,

discussed potential the will have So, the for FDA. we X with we our what study is The current delay plan that from have the and yet are RAISE options time. not then the what we discuss data once how spending that study, FDA,

Scott Braunstein

just Yes. a give me And let color. more little

I we a say, agency. step with have we European we – understood require regulatory to we think the knew would and hey, I think path back different

with the well. that the think us we I with have to that for have we to so in do is the agency. would good the sites what wouldn’t study gotten design, have interaction study very European given as a regulatory to it place U.S. in to U.S. doing love agency. are it that And in logical believe the We complimentary be we

wouldn’t we U.S. itself sites RAISE perspective, timing initiate only was the a enrolled see the as from would conflict. when that we in those think significantly trial that I a

pretty So, or all agency we to think we terms do where But we be it’s I love us filing Thanks a the going for in we have their question. a the are today. additional know always potential logical And for studies, with it’s a a label discussion certainly would expansion. discussion of we from to have. anytime to think buy-in as

Operator

comes of Jay question Oppenheimer. And your with the line next Olson from

Jay Olson

our Hey, guys. thanks progress the on and Congrats for taking questions.

payer pre-launch do if could. XX or frequency that reduction plateau from months a talk or seizure XX% the then any that feedback the a for OLE had And other Can CDD about We months are about activities? XX think follow-up saw of improve curious you you CDD. could in I you discussions question, effect? you first have is beyond And I gotten

Scott Braunstein

you Chris, to to right do that? want take in jump

Joe Hulihan

Go ahead.

Christy Shafer

Sure.

Scott Braunstein

I sorry, to Joe. going on was have question. Christy I commercial the start am

Joe Hulihan

yes. Please, please. No,

Christy Shafer

morning. Hi, Good there, Jay.

and So, view from research amount significant that CDD they we perspective, payer a how a they just done expectations have marketplace the the understanding have. of

phase been will the discussions have We of moving first have and this discussions positive. of just more and the plan. very, say move be very strategic research that finalized will how we because and very, research into only, to a internal into though, we are our that I payer

for population. this a have we reduction incredible there with that based research in an XX% secure is medication that also for market will the is So physicians, we are standpoint. say these patient increasingly more I in value our proposition appetite that patients that and payers new on done over

threshold lower So, certainly that communication. supports our

clinical perspective, a certainly it I over turn from Joe. So, will to

Joe Hulihan

Yes.

of need the to Patients So the And moving potential yes, through. still long-term in now. what longer patients terms there we follow-up, is follow-up happens. a with more I are to right see term see think

in improvement. the possibility there maintain potentially an see a overall opportunity, seizure could see can become minimum, change some we we efficacy. especially or they is I think continue the patients median, a think is there to no, will free could But I at but

Scott Braunstein

go we next the why don’t thanks. Operator, to Yes, question?

Sorry.

Operator

line question your problem. from Brian comes next Baird. the of And with No Skorney

Brian Skorney

label and the all interim I call. that an walk just on for FDA meeting are you it factors kind questions color I the the at of everyone. submission said the analysis. under study. for through guess comes of transparency good both you when if TSC, indication, the wondering NDA you an Hey, are upcoming to be will I for based the for can too? thanks just taking Thank CDD morning, and And PSC kind the division gating it’s CDD the was on pathways here and neurology us what the you of and open guess, review last could

kind from be little study of an give have so the of moving if maybe in analysis X you So, have about idea discussing talk to into into you sort confidence you already the you the learned interim bit what and can if Thanks. anything will far, that meeting? of a Phase

Scott Braunstein

add to Thanks, wants in your Brian. he NDA And meeting? and where kick anything, can on want TSC we after filing with you. Joe then if you Kim, the do thoughts the the about to off hop are

Kimberly McCormick

Sure.

submission So filing end NDA the for of the to process in targeted the we by are actively the June. preparing regards of NDA submission, in

but timeframe a on In are have submitted the month. and of to everything we are time for with the work an FDA this to track study. study, X still data and with and submit is the lot slightly study TSC end preparations, use of from as August-September end for ongoing of the discussions. timeframe study will the meeting well parallel in cut in support label our discussing that in Phase of the open those planning be agency there the regards So hopefully staggered European meeting request the also I having lot initiation to at we a to X of to a delivered by Phase the to July the

Joe Hulihan

of And on the yes, EPIDIOLEX. think looking what terms comment we recent Yes. for. I I be I the TSC in would mean, benchmark can are most

label, effect study. obviously, to similar that. so like see single an open to of And It magnitude we arm is would

the of interim placebo that. the we are things we present it’s seeing are head look, And to we of seeing terms So and have the effect? the should will dataset, the right direction control, the we response, full I think direction we the in predicting this historical really, later have but in and magnitude use in summer,

Operator

comes the Ladenburg with Higgins from question next your of Thalmann. line And Michael

Michael Higgins

you. different European to Certainly we now Question days. looking I execution. U.S. I progress. guys. out you that design departments. what for seeing Thank a am discussed RAISE of your on poses of Congrats these little for differences the response Can the in morning, regions, and the X? a going the some challenge, the versus help time would the bit RAISE you. mentioned. coming forward of continued rates have the over X Europe, between And Good us continued on in expected protocols Thank that’s that because lot ED in about driving but placebo some two be also study you difference understanding you think there, RAISE for efficacy

Scott Braunstein

a complicated. it’s Yes, bit Sure.

are different. I think the endpoints

in by to the EU, It’s use really IV neurologists stage not that in practice as to of mentioned, anesthesia nearly I the the gets much. tend ICUs As status. standard they it time

rather has the of well there you response. of early an power think that. study of they think escalation have escalation EU, that co-primary analysis to the In that. I have two itself care, has endpoint And they of patient And accommodation And to each that to is so, I And so also of have actually in That’s the actually, the increased combined endpoint escalation is will – because early anesthesia. I have second the have care the that we because to actually response in endpoints, own. to on as it’s obviously to made U.S. a part any of IV or a as responder given durable response the hit a one can superiority it’s show than the particular their think different endpoint care standard alone.

overall effect same the magnitude U.S. the the And is so, of the in

delta do But reasons, were to sample U.S., We placebo. the the parameters reflected that see size. different for study Initially, a ganaxolone and are in going different are the a for XX% and that’s we with between expecting is the combined design, the patients, smaller sample XX the identical same Europe with this can rather done in on study But but achieve own pool being with is plant its than the patients. data. power much of we complimentary to number a

the with reflection that’s again, increased responder so power a And of the endpoint.

Michael Higgins

Thanks, it. guys. Very helpful. Appreciate

Scott Braunstein

you question. Thank the for

Operator

your of with And from question Jefferies. next Andrew Tsai comes line

Andrew Tsai

and the morning all thanks good progress. Hi, for

we you And wanted an question Adcom review is Should should a standard suggests Thanks. investors XX-month in to epilepsies? you what just rare do assume expect on file CDD. submit? once confirm once President panel my So,

Scott Braunstein

all don’t the to a going take is why keeping for the all guess background. typing question? Kim. Michael Andrew. the We I us. was We I turn toes. was our heard question, over in on Andrew. am who it it you Thanks, Thanks bit it thought to us little Kim,

Kimberly McCormick

Scott. Thanks, Sure.

planning. there of the as NDA determine is FDA need an part for Adcom if will typical So any actually a

we preparing a actively the are FDA from Adcom whether or will confirmation hear we potential not or for We until won’t.

review if hear as that otherwise obtain that able disease, duration, time the versus this time, priority very to are part review of in to be are the we XX-month the So the typical as preparing In one the standard this getting unmet will medical are review of orphan NDA be high months for therefore, have going and as reduced filing a an very submission the we at we priority need, filing. regards confident until X will requesting rare this point agency. in we to we is from

that that timeframe So, the by your XX-day review we plus to targeting filability of question? you of first on X-month end are next year. do consider we anticipate have quarter answer the approval Does the when

Operator

with next of And your comes JMP Butler question Securities. from line the Jason

Jason Butler

taking and the Hi, question progress. thanks on congrats for the

kind formulations? the the the formulation on you in how of of on the can profile Thanks. are you you first indications and bioavailability could target us one formulation dynamics your Just and are thinking terms into for just thoughts PK doing, work prioritizing you that about give move with different

Scott Braunstein

Appreciate Jason. Thanks, take will I it. This is Scott. it.

So approach. bioavailability. multi-stepped we is the I current recognized of current it with will And the And a starts variability think understand the basics formulation significant. we program the formulation be the that

titrate the level. as what to physicians ability patients, step and replicate a do physicians to so give want blood we TID really allow and is a first level And therapeutic obtain dosing all that blood to potentially

we we a drug better ml. or a is we a clinic flexibility standpoint, dosing very XX we or standpoint, And very which know I next And our dose think clearly but physicians don’t to any that the are have But is from Cmax see much titration, delivery QD better BID more now, from and programs aggressively I go into more paradigm. After to members Cmins. and mentioned below I about bioavailability, But per be and TID remarks, want stretch incrementally could think of think really importantly, TID stable that three the administer to will formulation formulation family nanograms BID ultimately week Marigold I importantly Cmins patients to the as can Cmax go we technology good I will prepared to having about even will sure we think right what so control And drug to formulation platform our gives to QD. minimize work make in don’t Cmins. on dosing easier all three replicating that, create patients. either delivery want for from ultimately we tolerated, on TID

So excited we move for are have we quickly, that’s to-date thinking to about progress we to try about And which are about very and the we are a made long-term are but we a the bit. how we quite also it. thinking going franchise, have vision

Scott Braunstein

are we operator, to have question dialing was in to X:XX. We to great that there the to Certainly, you last was digest. release everyone want with lot going that press for coming all. thank in. make know that’s the We a upon it So

this our on am Oaktree have moving team great calls. team of move where myself, and available today us ball Christy for we us Joe, the is Steve, will just follow-up the have having and thrilled call I didn’t Kim, with the hear be from on to They thank done forward, thrilled an organization. to again. I So, about just And board just Sasha as team helping and Alex we job a forward want today.

everyone soon. thanks So, we’ll talk and to you Operator?

Operator

Thank call. conference today’s concludes participation. this for And your you

You may now disconnect.

Marinus Pharmaceuticals (MRNS) 17 May 212021 Q1 Earnings call transcript