MRNS - Earnings call transcript Q2 2021 | Marinus Pharmaceuticals, Inc.

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Operator

Greetings and welcome to the Marinus Pharmaceuticals Second Quarter 2021 Business Update Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. [Operator Instructions]. And now it is my pleasure to introduce your host, Sasha Damouni Ellis, Vice President of Corporate Affairs and Investor Relations.

Ms. begin, may You Ellis. Damouni

Sasha Damouni Ellis

Vice With Officer, Chief me Officer, Dr. you. Q&A. Christy McCormick, Marinus, Pfanstiel, on Affairs. the call will from Officer, Dr. be Braunstein, is Senior Kimberly Executive and Medical Chief Financial Scott of Joe Regulatory Chief Also Commercial Thank Shafer, Officer, are President who Hulihan, Steve during Chief available

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

Scott Braunstein

we've IV you, to across welcome to our Quarter, Financial Update. significant Second make Thank clinical and Building programs. in Sasha. our busy First XXXX oral Business progress our Quarter And and continued

announcements. Just important week, last we made X

team Tuberous partner Orion First, we the and with disorder, exclusive for sclerosis epilepticus. Ganaxolone deficiency believe the an Orion collaboration that complex Corporation entered an of franchise. refractory into be CDKLX for European excellent Ganaxolone in commercialization We will status strategic

filing, days from should the FDA. the two with third notification of application only Ganaxolone that these the receive runway collaboration continue significant we but a milestones our capital business. drug the to on they cash to end European of advancing draw to our FDA in CBD. activities. and acceptance of treatment development occur to across the events Facility, of Credit continue the have letter an expect Europe. Oaktree submission, seizures us for important to allowing the filing submitted pre-commercial evolution And of NDA to The of opportunity announce Not provides communication meaningful FDA accomplishments, provides also for the quarter. a represent associated are expand our That prior the extending we Ganaxolone to Within XX We for the new the

a addition, on number of milestones. In organization is the regulatory executing

the we our alignment that QX, Phase indicated overall our TSC to FDA, in plan. they submitted design the clinical During with X and Kim. the details were to will leave development trial I agency

the Trust Alliance for that TSC forward. Fourth an X Phase have Quarter first thank whose been to prepare program to in has this support the driving enrolled TSC we like this continue instrumental I named the expected trial year. We patient of would TSC in with

U.S. the The this with RAISE organization by the key patent potential Ganaxolone meaningful on We to update and for The patent move IV me later application completion method action X the Let the Franchise. NDA date Patent registrational RSC following and We the are initiatives. other the an on their the CBD trial. When IV XXXX. formerly for dosing U.S. expect the We a an continuing work method the is application the aligns RSC the create We run First to notice highlights our of Quarter launch dosing for of Quarter. treatment Office. XXXX. received of end of patent the towards allowance in we look and granted, trial. important for of shareholder for progressing. FDA granted the study, Plans to of from regimen anticipate the patent believe Phase value patent to has X treatment be through Trademark and Third the the Phase

result As for scheduling FDA a followed 'XX mid a period launch. approval in by reminder, would a DEA X-month

Europe diligent a diseases After looking have leader Corporation. be neurological for. in signed presence as rare hospital process, European Finland-based A and the significant commercial A disorders. strong with that all were we mentioned, European desire in the epilepsy, Western ICU, agreement Orion and and across collaboration extensive to orphan a As we an competitive boxes as we expertise checked well believe the the a in infrastructure. that Orion

low they together cultural Orion to strikes XXX million sales be as of commercialize future the UK Importantly, and been milestones. Ganaxolone net received Oral this million fit and Switzerland, could granted payment and and to an up and achieve XXs reimbursement has a all structure approximately the pricing upfront exclusive area, oral forward development, for and reimbursement, as right R&D on approvals. look We the to working eligible the strong to that will the and We've formulations and our believe as the the Marinus upfront economic payments, well and of European tiered to success responsible program. deal the offers payments long-term $XX Orion shareholders on allowing where in organization, in commercialization Ganaxolone from IV high towards approximately that we teens to benefit rights franchise. for the royalties, royalties future our for programs IV reach balance are

commitment of Importantly, oral supports and launch Ganaxolone CDD patient European our underscores for this CDD the to mid-XX a agreement community.

are track in high-quality QX of this throughout to We the desire availability patients, collaboration the on a observational of designed provide CDD. development CDD to a variety commitment an research XXXX. inform marketing better our advancing Ganaxolone therapeutics near application to the a history Loulou broader outcomes including Europe. living and the observational refractory families we database see keeping clinical CDD having Ganaxolone of unmet term, severity, geographically to to disease file the sponsored Foundation, in new organization support This better plan end and a describe expanded access understanding for committed dedicated potential program. to future for provide collaborative alternative and community, Both to organizations for are the In on CDD CDD, support to by of a CDD nonprofit patients by patient European we study the natural study to In important with improving is to to trials. authorization with need, the a

are resources excited are contributing community. this We necessary and to the a part research of critically CDD consortium be committed to to

on Phase to to program, trial open-label topline our data the Quarter. TSC from remain Moving track the X in Third we announce

and appropriate meeting the to earlier, to the noted First of overall reached XXXX. path. expect occur We're I alignment on the have believe obtain now expecting Quarter we that As in FDA we advice from an EMA regulatory also that with scientific

to site be continue to Quarter the We in planning with patient Third in for initiations XXXX. Phase of first Fourth the the enrolled begin X Quarter expected

providing are EMA. targeting interaction on We We regulatory plan our Trust later with the timelines initial TSC sites U.S. currently will in the this trial following for sites and EU enrollment add year.

COVID-XX participating impacted delays due major devote clinical the with caused These the to most delays. patients Turning medical enrollment. Second fatigue acutely IV site Quarter resources to significant staff in We've and COVID-XX experienced primarily trial. COVID-XX. We believe site to need sites priorities centers in challenges several programs, for to by these turnover RAISE our to initiation are academic due and enrollment

initiation open these are Quarter, the improving, have our RAISE we the site Consequently, giving issues numbers now We've expect vast seen July the second this already behind site Fortunately, timeline. to of us. of operations readout end with half Trial our clinical confidence majority the in our of team majority top-line data XXXX, of by believes that us XXXX. Third in

we'll additional and We our updates provide day. are actively October with R&D during our site engaged

or As launch This is XXXX. in for the IV the The begin a U.S. track enrollment reminder, to X is ESC, to half in Status planned trial X Established adjunctive planning European trial the of been to first half continues of registration in Ganaxolone Phase on RESET RAISE absalon first in ROC. of next trial year. support of Epilepticus

Syndrome the CDD call Marigold thinking on share LGS note the LGS. wanted study. an Phenotype with to who Of our turning Before Kim, to or in treated over Lennox-Gastaut I patients X

for that Epilepsy the meeting. submitted annual Society at have We presentation data American

higher We blood titration observed an LGS majority anti-epileptic and not competitive that our those to have to LGS including the blood also We that the in similar for regimen, believe Company's therapy. noted the did concentrations the and lead serum best study, highly Ganaxolone. from that patients schedule, possible in dosing achieve study. Phase should the seeing an that earlier appropriate families reviewed Marigold of said landscape suffering are X looking is TID That study LGS concentration current we're adequate patients prior development of and

these with tied next-generation next-generation work to dose deliver steady-state investment to LGS in for oral indications. more in a improved and We Our or formulations efficacy. believe reliable create program. drug we and that in committed be these Phase candidate ultimately, team prodrugs is should novel right bioavailability, addition studies higher ' solid incremental Marinus closely will evaluate plan that our the front, titration, formulations oral new clinical plasma concentrations, the Ganaxolone. to the evaluate X and XXXX epilepsy for platform continue new several potential IV technologies in to formulation and refractory Further and We on

in We more two have will clinic expect year. the to likely to it's next we into at formulations that believe formulations currently these least that advance of And reason one advance XXXX.

and including on first step reformulations the brings career to our to second-generation We Board Marinus's academic our I More and a want distinguished R&D leadership investments neurology at in are in the come during department the also neurology Hopkins Dr. Dr. Hospital. that development, to the a enthusiastic Directors. Johns Day. to of are research Arroyo Santiago welcome Arroyo clinical platform.

now development, would over Kim. on new experience of turn call our he addition, regulatory Vice that, McCormick in extensive like an to update numerous President interactions. has Senior Regulatory drug for therapeutic Affairs, to alternatives. Kim With contributing the to epilepsy I In

Kimberly McCormick

Thank you, Scott.

Marinus CDD. may biologic or we that for the review is to third NDA filing, the an subsequent NDA priority also period. priority for priority end This seizures is period. party. as they The have the of a with action Within be priority the meeting Ganaxolone committee let review pediatric approves of accepted request submission, team granted, designation. for then in has obtain FDA to we or the early the potential transfer at for plan be the pleased designation the anticipate as disease days will advisory rare NDA DEA a NDA, us Marinus actively a if they approval. if to review FDA or If a it and may are schedule for a benchmarks, filing the sell recommendation of for granted voucher includes We Ganaxolone to treatment for an to PRV days whether the well review our date other produce To would know our associated FDA. could We as is The recently to XX-days used be notify meeting. FDA hold drug a the indicated targeted FDA scheduling submission If is system during preparing the advisory of committee announced XXXX. XX for NDA, - expected or March notify forward To PRV XX application, working the as to XX-day with human is look expected PRV. with within of would dataset, is a a granted.

anticipated review of remain Ganaxolone. several questions to for an preparing in focused As related metabolite had We've MX process, our we launch.

had on have reminder, we Marinus FDA the it. best metabolite the characterize with As in identified a discussions and how MX to XXXX

what the activity required essay in for the to of results interactions part respect NDA included recently with metabolite. with raised the the have the on We NDA FDA, was with MX As agreement completed was submission agreed commission as in pre-NDA and an metabolite the FDA.

the planning vivo this with XXXX. to testing of anticipate conduct in be first micronuclear complete I are in an common analysis. test, will quarter We that

be expect if toxicology a studies such acquired, additional that post-approval. are MX any package metabolite, We they on the full conducted as can

by the track U.S. outside authorization As a Third the the Scott Agency end remain mentioned, European with to on marketing we the are in markets CDD Ganaxolone of and dedicated application file for to advancing Quarter. Medicines

we part Phase our reduce CHMP expect health, plan, of are proposed for for the receive the the eligible the determines is regard TSC We of This review the of if particularly share public on the the our days Quarter EMA of product grant to major XXX. mean our of target trial. development as Applications associated standard clinical are therapeutic seizures that XXX of accelerated to CDD. to we indicated review B the to for from assessment could that for will recommendation from of the point X FDA If to Second with assessment Ganaxolone assessment a innovation. request alignment the view meeting written for CHMP XXXX. time to granted, interest pleased overall Type treatment could accelerated With TSC, responses accelerated

review, be of applicable following could it an matter will of a indications approval believe X would NDA. be oral the with pursue the study in a a Although will single Phase orphan single support Ganaxolone of be this we paradigm of to that to approval pivotal trial this CDD indication other rare pivotal sufficient Ganaxolone. We

In following a for a to likely XXXX. meeting. the is the on of EMA Quarter program replace targeted scientific trial addition, Enrollment meeting with First Phase begin and is TSC that X

the end designation and As the a requests We're have to Quarter. by receive expecting Third submitted to drug FDA responses of EMA. reminder, we orphan

Status we've our I'm Epilepticus with the FDA design trial. alignment RESET ESE ESE, in overall the Turning or share to Established reached to trial for that pleased on our

have the XXXX. this. and we IND begin expect our to be trial further Joe Additionally, U.S. There'll from first may received RESET to continue details the we notification that to recently half enrollment of on support in proceed,

the Joe. like Joe Officer, call our would turn to Chief I to Medical Now, Hulihan. over Dr.

Joe Hulihan

And Kim. everyone. morning good you, Thank

I been As I XX like end mentioned, vast next the start by extremely response half franchise. although control have is you've study meet a sites the with protocol top-line little the and That has trial, are we as opened right of RAISE planned. busy screening high, of a of in and expect quarter, months We the at heard, of now appropriate originally quarter. have study those highlighting first third In IV beyond. design patients for Rates requirements. course is the putting considered Scott on expected we would year than progress us majority this last for the a arm data detailed those sites the being our later carefully on believe as to only the who enrolled. rates study by productive, to

and in months. team is initiations study centers clinical the has enrollment Our expressed the next driving patient over and few site to outreach outlook for confidence

Our site, educational scheduled X, of continue training, the in RSC with for interactive regularly interactions first and studies, clinical strong to teams launch for our registration The RAISE remains have including case half XXXX. pivotal sessions. operations target European medical trial and on

of most will next guidelines. the leadership our RAISE a X influence call may RAISE we our RAISE If implication as discussed X EMA I establishing U.S. the for pleased studying clinical this As that could it epilepsy earlier treatment such to in global potentially be trial Ganaxolone the registration can RSC is the goals version complementary the RAISE but serve not hear Since approach also of important the trial the Epilepticus, the on notably, only last an that Europe, quarter, Status trial one of pivotal RAISE market. U.S. X. in trial to differs initiated in of the that have were data to treatment study course could in provide the from in in in RSC successful,

trial a second-line consent. our of the we've is X In completed to Quarter a Established for status Status begin time care As the of of less RESET alignment Kim adjunctive the which enrollment trial. Epilepticus. First is are gained RESET sites Status to ESET, in potential after of exception response known Treatment, response can in effect to the trial, those to the informed of in would standard same trial, alone. mentioned, FDA we Epilepticus first plan trial from consider s has ESC XXXX, AED stands established response of earlier to a hour. established within showed including qualify initiate to expected with target the the trial study the that be Treatment patients as care increasing have who the as trial. for act first-line standard rather and AED regimen Epilepticus standard in Established for from with to Status departments. believe enter initial an process in that the Researching distinct to rates, and U.S. Ganaxolone in continues trial, The Phase the conjunction status We initial type enrollment RESET AED. RSC reducing intended AED. is which second-line The with the epilepticus increase enrolled adjuvant will participation RESET than RAISE fails. from than Ganaxolone treatment RAISE dosing Status convulsive non-convulsive as will XX% Epilepticus. trial rates compromising the continuable will Ganaxolone than We initiated Ganaxolone The with of without status it's emergency And the [Indiscernible] the benzodiazepines The durability defined care the specifically of compared s shorter examine The Status from

in doses an well-tolerated the regimen assess dosing find durations for Ganaxolone ESC. The and design and infusion several effective, of to goal safety Our the effectiveness novel a in study Ganaxolone. sequential with utilizes is trial to safe, and

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

case from continue meeting. upcoming on In with for presented that Ganaxolone epilepticus we Investigation medical report to status addition, an additional new applications. investigators under requests optimistic an at IV for Emergency will We're field drug be super-refractory patients

like our provide on more to oral I'd color Now, programs.

frequency, for for in is patients submission those And double-blind for A endpoint receiving by with seizure that trial. Kim of motor placebo to to CDD. and in one-year extension abstracts Achieving clinical a the from XXX median series of NDA P-value enrolled compared supported discussed, suffering Ganaxolone We're reduction treated milestone control Scott for plan AES the we with We a placebo. the the submit medical The Marigold major from X.X% is December Ganaxolone's patients. As primary efforts. Those submitted on X X.XXXX. very data including this the meeting, those CBD. recently from advancing showed data diligently trial a open-label X.X% a trial's NDA an focused important Phase reduction development XX-day a study.

treatment, seeing XX.X%. with reached data XX a from far year open-label patients we're on percent preliminary of of who've So one median reduction baseline

X rest Phase Access open-label We Adjunctive Ganaxolone the extension. patients have to XX-week we're the Quarter. a in to CDD in our U.S. and followed a Ganaxolone seizure We're potential period across to in several later trial through of work also approval to in which with the regard with at global and X who've initiate improve committed through continue throughout data and who Third approximately to the the XX-week XXX with enroll CALM U.S. patients and to this interest patients, X-week treated X identifying the will Consistent With use We're least Expanded programs. compassionate placebo-controlled prior the additional TSC making top-line to more This double-blind baseline help been prior is from control we're the world trial, and lives including safety, evaluating TSC. in tolerability committed to Phase with opportunities available the TSC, shown treatment of to by of study, expecting participated collaborations the effectiveness, trial program. period, patients patients a this a inadequate centers expecting Program, AEDs. year, who present have had treatment

for patients of EPIDIOLEX. the EPIDIOLEX patients TSC most a Unlike recently treated study, allow We AED have taking enrollment expect range been approved the of full concomitant of will our TSC with that will trial XX% medications. our

patient fourth to expect the enroll We during the of quarter XXXX. first

syndrome, like mutation, will CDD Since would single a is representing as of our it complement pediatric etiologies. good the options clinical our a for underlying carried regardless etiology. can CDD, a those TSC we in LGS several we which epilepsies have are in who've believe had LGS. LGS our occurring studies than reviewing much seizure to types and in diagnosis LGS Ganaxolone Syndrome, due for candidate a Rather genetic several that phenotype of clinical development also clinical other expect we've We be in are work Lennox-Gastaut development patients

and one of more an plans trial provide formulations consistent to Our our pursue to to LGS exposure Ganaxolone. with new a are greater

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

turn Steve to a to call I Pfanstiel would update. over CFO, Now, financial our like the for

Steve Pfanstiel

to Thank you, am of to first our for half year. Joe. able financial be pleased the share I the results

of X we XXXX, X.X For million recognized BARDA XXth, months and in respectively. contract federal for revenues the the and Second million X.X Quarter XXXX ended X June

Company RSC the the expenses XXXX, trial, associated the the change June are change X corresponding X XXXX, increased for and to and contract As XX.X X.X million increased and periods administrative the submission, increased respectively, General and to the operations, XXth, X activities X XXXX, of million losses million year. as compared the same expenses the the the and June months as of with was XXth, the in commercialization. compensation for XXXX, million months used to XX for for and the primary XX.X XXth, in XX.X respectively, respectively, of and operating year. June totals million start-up X year. for for XX reported in million year. X increased months of The same X.X prior the periods the X million XX.X prior ended million primarily million million XX.X and to X associated BARDA development as the X period million compared respectively, there for expense activities as regulatory R&D for this periods year. ended prior to million XXth, signed months with no June from XX.X million losses XXXX, the net same prior and the X X.X increased Company's million net for Phase the include million to and as prior X XX of The Research in in activities million X of the X and September XX.X to and ended year. support scale-up in stock-based used periods and in and were million CDD for the of same million XXXX, as June compared and headcount. in compared million period XXth, ended due same prior XX.X the in These and XX.X as well drivers the to non-cash, was preparation million compared revenues and ended months The X.X operating for to for respectively, XX and Cash cash X

XXth, June the Orion European combined of had required of believe As is we sufficient balance under the maintaining cash signed We with debt XXXX, for XXX.X balance the this XX to million. facility. and equivalents our with cash of operations months net upfront cash at recently fund proceeds the least collaboration minimum while

FDA filing million acceptance Additionally, as our we the have under upon an mentioned, of opportunity XX facility. financing Oaktree additional Scott NDA, to of draw the for

monetize strengthen We the an approval, if opportunity million including as as PRV to our sheet well have the funding awarded. tranche Balance to further Oaktree, from a significantly opportunities FDA upon XX of second

The million, the timing to which revenue a contract of million XX X million. million to to of the expected Separately, our of our reflects XXXX, XXXX, estimate million activities. operating XX compared contract BARDA year XXX of expense of guidance of our to in the value million remains updated prior and BARDA includes for million, unchanged Fiscal range at approximately, fiscal be range compensation. the stock-based XX to X unchanged total For revenues XXX XX are the year GAAP guidance we for remains adjusting non-cash which in million,

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

Scott Braunstein

our first Thanks, XXXX. months, the we of of has to and our of and to dedicated been advocacy half their Steve. the over the can possible open employees without forward the for Operator, a exciting It developments this to been support call encouragement. None our would've look thank work We productive Marinus hard now partners. support the coming highly want of and you questions? shareholders

Operator

We'll roster. Instructions] moment [Operator a just the to pause Yes. Q&A for compile

question Thome first the comes from Company. line of Our & Cowen with Joseph

Joseph Thome

on first to Because wave in Maybe indication be to you progress. mentioned just you the assay congratulations good new clinical formulation it in in vito the be ought back of are CBD or one taking Is our and on this clarification, first-generation epilepsy be thinking ahead Hello, the How Is questions Thanks. a first prepared with the the just formulations. quarter new the was it? formulation in approved And of possible before morning. share. the just on new completed will decision you second, Thank may for about the TSC. next liquidates maybe activity essentially the the the bridge then indications of CDB to potentially NDA?. remarks, initial that necessary for indications? the and a

Scott Braunstein

but and of Thanks, Joe. Good the second at let MX, over had question. on we've Micronucleus plan not We the will we agency and time with morning. And for interactions me jump very just filing. test, likely bit necessary take -- the the the the provide them In Vivo

report. get it a a don't the FDA It we we'll it. discussion. be supplement happy And so we'll see will amendment, be year-end require major we've had as and that that should brief And to that relatively

step-by-step the new On work, formulation process. it's a

bioavailability new preclinical patients, data Ganaxolone and these TBA. a respond are steady-state new CDD, Joe, back is to go from to like we would going showing thinking thinking if concentrations. and there's substantially about to would we or with success. general of at formulations, we're say availability I those that that blood how to going of we're that lack odds new into first levels indication. foremost, that to success is now about an both maximize our how trough, and a CDD. start patients really some bio in a increase formulation certainly given feeling, new prior that right indications are the believe certainly don't and seen we're Ganaxolone. with to to this My bring patients know And excited are think obvious formulation higher we and And formulation opportunity peak of really achieve those and XX% tied early we formulations in X that least XX% to than the that really understand We've restudy we're in today. humans First where And

there good with And So I we some second as CDD lot where about what patients we have enroll We'll data several are like a importantly, indications thought we'd are shots opportunity -- think of practical of opportunity can in to with next study? Most exactly to front understanding the I thinking well very Joe. year. new the really on as real the and we've about challenges would And time. on the mentioned, new certainly TBA, I'm say I have this Marigold CDD TSC the that, excited first given and a we to goal at think from foremost. we formulation. achieve I in are the

the don't I'll So go and stop why question, Thanks to the there. for next we Operator, question. Joe,

Operator

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

Joon Lee

Hi. Thanks question. for the

trial, the I can you what And enrollment enrollment are For now contingent follow-up. and on RSC trial of complete in start RESET where a that enrolling RAISE completion. by you ESC trial with year-end? and confidence the And you gives have be the on

Scott Braunstein

Joon. Thanks,

X-X about we're on site expecting, expect monthly on and our timelines And trials, all between RSC the year. sites very this, enroll active per patients based trial, that on the -- will are our a per expected RAISE somewhere -- Our been of are basis per we've transparent intuition. we patient,

been the expected curves site to some enrollment We really up their in and thus line really -- of we've in per have be -- new trial, sites we a in particularly far, hit enrollment where our key sites have that sites high enrolling. seen have getting expectations, snag, with enrollment that on our QX, their early the

already the on last expect by about sites Talked of the continue of second-half and we then in X to very And QX, really enthusiastic we new of the is And feel of enroll have majority And of and our our that then per we about team open. 'XX. aligned be X sites much August July. As course, those the to clinical key those sites open to sites open, end year. getting I between September. somewhere mentioned, night, better to with patients how majority to timing we

really end nicely had for site which but I 'XX, the by a QX 'XX the Joe driven really really mentioned through of and early very call, on is in the COVID, change the worst as took initiations, turn both started I We those and lifestyle. double-digit more are academic investigators caught off site site their changes. personnel us coordinators. It people changed really their but guard, now They catch playing And up. as than centers lost think changed major several life. So

Before your to Joe, to we there? want go anything second question, add you

Joon Lee

Scott. No,

covered you've think it. I

Scott Braunstein

quick a we I questions. because follow-up other the make to get Just want sure

Joon Lee

Yes.

RESET question upon RAISE. the whether the actually getting moments trial the was start be -- So [Indiscernible] of included contingent Yeah.

Scott Braunstein

Yes.

the is partner, Authorities, X, that. European about. which about to going important that study strategic an to Regulatory now see we're Sorry our talk the with got alignment them Orion certainly partner. RAISE we We as

that alignment about Orion said, love we're that with opportunity That in, sites requested group study good had the alliance being sites the through have design. EMA using, on already CROs, we discussions to with they progress. We our experience have speak to the and that those we've

very Remember, have have start will U.S. this we're every add end of of And so in we a intention moving all. expectation is forward. the see but as at European driven quarter, conflict and will likely with X RAISE potentially X European strong Orion expecting 'XX, to don't RAISE key some largely the U.S. in in We'll that alignment certainly we X sites. be RAISE RAISE my by by is sites sites,

that as which will our U.S. Joe from running, as issue really about, And ESE in sites some talked For are we the and but where feel remember trial, we U.S. coming as RAISE populations. through those perspective, -- trial in in room an is very see study, were initiated of study, certainly sites reset have study for We we curves the handful by be. the will our patient 'XX. up beginning a enrollment them and in a the should ESE emergency mentioned, patients overlaps time in the have they different

there chance of may X. space, a sites fortunate be Status are RAISE some at or And sites of we certainly, in terms be enrollers to impact are opportunity to patients. RAISE and be That don't may the there overlap than Epilepticus certainly I potential with in create active timelines from in of to X expect we RAISE the not we by feel overlap but work X QX trial. would more going that the very trial active So that them would have that just all. one a 'XX, will with any think

RAISE So Is between avoid that's clear, X. Joon? that would certainly something we and RAISE

Joon Lee

very Yes. much. you Yes. Thank understand. Thank That's I exactly wanted to what you.

Scott Braunstein

Absolutely. Operator, next question.

Operator

Yes.

Your next with H.C. question Wainwright. comes from Douglas Tsao

Douglas Tsao

the morning. Hi. taking Good Thanks questions. for

the been so to gear you and if Company activities up online, the launch. what maybe on you ahead for the you've an since put -- accomplish potential able far, Just Thank Christy pre-commercialization provide you. and of update

Scott Braunstein

jump to jump why do right you Did don't question? in? that Christy, hear right in? you you Christy, want

Sasha Damouni Ellis

I -- thought she's

Scott Braunstein

some think issues, I we're having Doug. technical

So would you --

Douglas Tsao

Okay.

Scott Braunstein

let's Do want get see. me -- to you

Douglas Tsao

take can You it.

Scott Braunstein

have on a across 's hired forget side. our team. Christy chain. ops commercial add to great sales, now leader, was one great I our the want of We first supply and I'll clinical that, to a across anyone, don't hires marketing, leadership Sure. reimbursement

We're leadership our be positions will now sales territories. leadership reps. Those So leaders sales responsible actually for our we in key have place. all the for recruiting hiring X for our

would into approval. plan offers And make on great those shape. team Our a early perspective, sales to XXXX, contingent we're in from be

flip the territories. almost the our on MSLs across science our five with complete medical on we're side, side, liaison Just

practice, we on finding who little we're MSLs key and interacting And we do Our significant are and KOLs, our understanding bit CDD numbers now to in of their what's more market physicians of are building Doug, much treaters patients those have interesting, with are. been who the side I expected. is relationships research would many than have really continue

really AES. like to great certainly a we're have we're hard, the in to really CDD very degree. working commercial base large have shape. identification feel the busy And is We'll going forward of We're launch. looking the to solid So we a patient team

team MSL have see major Our live. is abstracts. We'll if we're working on at a AES. program We'll

little going what's suspicious We're with certainly I to Delta on that education given talked forms ready team in fact, necessary If live Chicago, I'm going the communication meeting. of will a about preparing is for inside, in all more to come be regard. December. bit for a be online yesterday In we virus, but

today. you happier audio great to Christy a still her a of account? So is give grief I'm is I commercial down lot job. the doing couldn't with is out be going because team not today. are their Christy, to where

Christy Shafer

I not. I on. now am am back

Scott Braunstein

Okay. do. Well, I'm about did that, sorry my [Indiscernible]. I rambling that. add best Anything you please to want to

Christy Shafer

So Scott Doug, that on I'm sure everything. hit

launch how that, physician look that an support strategy. functional our looking mid-XX of of perspective, all obviously, finalized a about a we've a one enterprise-wide should Marinus We excited readiness this that the approach with And functional board. some priority agency for review, excited balanced a will departments. our pieces X anticipation of the and the launch full areas organization of launch. like as we've marketing blueprint our have high-level think to at community. really the is that we're we're now onboard to marketing filing, to then executed the within launch a new on but very integrated From about, on things the I first, in all heels And we're be we have largest with strategy work fully of really would product caregiver

as And so will from the from heard marketing being interactions. a and the we're access both to from and chosen have really that get access will for launch we leads just leaders out to market one we get important continue help really a sales and are and I support first-line with into it that, can CDKLX, encouraged early sales. that payers with These these research perspective, our our to bit insights continue about we really strategy, think be strategy. little our that KOLs getting will there Scott the on that talk that communication continues the be from we're with doing, how but inform on us one we I back

So having I duplicative gave with encouraged but wasn't for and that next Scott thrilled on are we're you, we hope in, are, where we filing we're year. what on track this

Douglas Tsao

Thank you so great. Okay, much.

Christy Shafer

great day. a Have

Operator

Your next --

Christy Shafer

Operator?

Operator

Yes.

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

Andrew Tsai

question. for I Great. question Good CDD. It's me. like housekeeping on morning a have and another thanks more having

of at what talk your can I general, relates In about guess case schedule point drugs including context, some stricter starts can for you DEA. other received what become X then uptake? from And expectation you does of the to remarks base a there? to gathers mentioned guess, the by have out an how, You scheduled I why epilepsy prepared scheduling the a issue? is talk kinds past, schedule Thanks. would it about in as non-issue your And be for

Scott Braunstein

and why it for Kim, commercial be thanks talk the why question. then -- for think for Christy we'll side. about Well, you the over turn a bit Andrew, first little where don't scheduled we'll you to

Kimberly McCormick

Sure, Scott. Thanks.

based NDA supported So in had recommendations as the compared And based the abuse clinical volume, on I support think, fact conduct that to that on to Liability of regards scheduling, required to study part at effect our to were as data, we similar and we FDA. non-clinical arable the the to and submission. to

clinical conducted that the our of assessment years, prepare the as too our of for. view data to studies to metric past some our led such for well submission the the As us from NDA we recommendation because additions [Indiscernible] has saw and that with much benchmarks, over [Indiscernible] schedule batch a we schedule that as data and make several to part Ganaxolone, as similarity

So that's DEA on we to that went kind how further recommendation. for it endorsement. And of now, will go

Scott Braunstein

want Christy you And about do talk commercial the about to that? thinking

Christy Shafer

Schedule the only our do point put here we physicians we're hurdles perspective, in the don't large anticipate Sure. very put well anticipated fully into The They're We perspective. channel. Pharmacy from when help into and any to have that from to to the IV where drug Specialty products. Specialty approval whether To versed to remember communication understand several Kim's analogs means are, from we get need which be it's what with to however, to Schedule conversations done with identification We've with slight promote market needs good when this a support IV. can to XX drug, have time, patients. scheduling that thing able days during we channel, the patients our we is that that during understanding officially that drug of the period, and actually the

So IV we'll for wait necessary period had Schedule we'll materials that then be RPI it really and when confirm update all and which XX-day -- that pause to are we will where scheduling that associated. that of a

Andrew Tsai

-- you Thank Great.

Scott Braunstein

want about to just remind and EPIDIOLEX their you comparator? scheduling, folks a do Yeah, as

Kimberly McCormick

that Is to Scott? me

Scott Braunstein

Yeah.

Kimberly McCormick

initially, side. schedule EPIDIOLEX, was

removed EPIDIOLEX. scheduling However, was from subsequently, a the

I a a not it's don't because know -- but fairly believe is drug, should [Indiscernible] drug, don't So have We're that's working. our update. barrier schedule I the anticipate currently or I a scheduled mentioned, there'll with there's antiepileptic to other barrier you and can be I commercialization consistent

Andrew Tsai

Makes sense. Thanks.

Operator

comes of SVB Leerink. the from line with Marc The Goodman question next

Marc Goodman

Yeah.

now, and work, really started of sites and for As give mentioned just you us research that thinking for sense can more you patients the a us of there on that you've as how may much year a and they on done we And -- Orion. many launch the where and U.S. you've the all the that? have touching ago a have then second more Give had to all, sense everything, of I Scott, number patients. was you've the visibility CDD, patients it just this are ready of in getting you're you how a and into there gotten the you found you you guys out think thought they and are just how know

done has off as Have partner. with epilepsy a remind really it them? make just a that Just to good before Orion that the us impressed Thanks. products they them some of you done really job

Scott Braunstein

Marc. Thanks,

in the expertise, second Orion have team with a they're leader I'll and orphan give second Europe. the so pharmaceutical and is about little flip Companies neurology the the on the in on Orion into it their rare of part have Precedex then in in they and products business. experience ICU have tremendous the side of team nice recently have they over flip Let Parkinson's I'll -- companies, is that thing of really me patients Christy. and bit commercial key orphan it take is Orion are area on commercial products, fact, disease, they on biotech other and to a -- the has of then global the both one who -- disease They background I'll with and and branched

from of about result, standpoint. parts from a European reimbursement And the had the they've market, to of both an alignment approaching access strong great We've really they been had we think standpoint. in terms with. puzzle So all X data really our how collection work as

reimbursement. from brought Christy's thinking value to they've they're and to market RSC about how some with of discussions team access of within think some a I already. team led members disable lot and an that's countries And Europe specific

they're already we of Orion our CDD. X feel number became we're the And and on in fortunate that CDKLX believe regard. very X -- from a So to of side bringing a like in On table. patients center to already the to a way, they've identified going to patients, CDD, and is they now center UK this we've jump trial a and just tables U.S. that had talked way lot changing nice done that in having of we're in that skill sets seeing We grow. by Phase around the XX side landscape a recent Europe good CDD excellence the

seeing U.S. the everything almost in think we're dynamic changing we Europe as So that in about, talked well. I

within year them, dirty, almost U.S., are in age, about year to and we given all XXX going every the for recommended of the testing. illness, diagnosed U.S. of born quick genetic the the with severity On side believe be CDKLX, are kids first are of their

global many those patient's of X genetic likelihood in data being age dataset, there's patients. X-years-old, the the a capturing that done aligning what's set largest reminder, number the of a average high CDD incidence as with So testing And very were our and a that world. Phase

the And kind being maybe confident, I'll of you. is to stop so for -- rest we being to identified, that younger and particularly there, centers population referred very turn excellence. just you Christy, feel And of over

Christy Shafer

Sure.

But market really, are X fine And So total when research our just it's continued on to KOL the it's and patients. U.S., closely excellence and deep finding they're key I get pediatric centers Right? we we Scott, key to across treat thought. what We've say, just of involvement really really as significant doing they're and to reminder, to epileptologist than XX really focus working a of there patients. centers. a assumptions. best epilepsy test CDD really quick number with these to better their call and local very So have has continue what is we're that neurologist

I COVID us. environment, think perspective that are will patients how interesting an the virtual which of treated give being supportive us in is of

sales team. will We have small a

with much great level it us for to locally to in concentrated are in large X epilepsy order these we're easier treated folks. how much, centers, XX these of well, be are. potentially and large in CDD really these centers Again, all are engaging centers in get feedback physicians getting patients overall, where really at as but them So will some

Marc Goodman

the think there in patients right U.S. you total how many in are So now?

Christy Shafer

The to about -- -- hard will really is the at X summer. remind to XX Code and the say. gosh. It's months you at Oh, only last end of time it I that ICD-XX is was the old. think I

So it's the sure, believe remind about at we're now. in I'll not pediatric population an XXX% XX,XXX. right X just you we're a we do but looking that

pediatric being. time So because right patients for undiagnosed this looking really and or now not we X,XXX those patients be X,XXX at adult, will probably between looking

Marc Goodman

you. Thank

Operator

next Your Alethia line comes Cantor with question Fitzgerald. the from of Young,

Nina

taking Hi, Alethia. good for morning. our questions. This for is Thanks Nina on

and update you design, could should share the TSC top-line in share for think the the We be you. will more expectations. also on Phase what are wondering Thank just how Can Quarter. proposed any on details we please X about if you and disclosed Third

Scott Braunstein

I Good call -- you. morning. the the over to don't turn question Joe, why turn

Joe Hulihan

And are in of different. which it's a seizure Most same major The not bit endpoint, vocal, The the fairly then CDD reduction the to much be the case like treated weeks case, X-week placebo, design, or titration types X titration, endpoint weeks a going maintenance. Yeah. and Phase XX in to standard the percent TSC same in little have, very be TSC. schedule. CDD. the with this a actual Sure. X-week baseline study, are have of the as seizures. Patients types a seizure on seizures XX it's for is study.

And be be X when see XX number XX in in thing we the patients look single-arm are there of things open-label, one it's we'll and looking that's to again, -- I data going it. interested So a at XX, we'll so think at. Phase

that patients there effect we've really magnitude competency the of effect, around We could will levels of just wide at number be not of the a lot variability. pretty look with seen,

that top-line as implications the but and response chance response, will has see So and if for be initial X. but within a design seizure type, long the for see good Yep. the results of variability we to we'll set then as a Phase with the explore by first the continue data data,

Nina? So question, does answer that your

Nina

Thank Yes, it does. you.

Scott Braunstein

Okay.

just that's me are keen cohort on be going well We on in the on Joe. add Let looking response who to as as very young AFINITOR on patient at entire rates are previously to EPIDIOLEX. certainly patients

the only we've had a add, patients. gotten responses to real line So a sure that and X And thing recently We've done in to will refractory we SAB lot we're I'd add about X. as -- we'll Phase Joe's I insights. happy very study, of I'm We this reasonable those would a commentary. and learned be Phase make a thinking see Board. We've really this effectively, want study, lot. an That third they

in have our background. and great our back thoughtful Dr. GW are differences go trying the some who design. that epilepsy about we're Novartis you just and Centaur be X study both to to and If these And Joe, thrilled for study appropriate the I'm for Dr. house look at design joined epileptologist, designs study, trial extremely there Arroyo, board with

the grants be paradigm more. say, And aligned going if but well, and with some we again, forward people as it's dialog, in really now ask have we've question, epilepsies, important the -- very we to that agency so an with awesome if was job, that and approval, have single-study to a board really about thoughtful why us we make I have study for we to us have the the to forward-thinking tweaks and a lot to one us. we FDA forward any move across as quite very and of with we Kim's continue paradigm constructive the we'll so we're a we we're a with a transparent, interactions happy I really feel if with happy we're like think need strategy of right? their of another but and in interactions certainly, go very CDD done agency And if comforting. design, nice rare general, registration we're our these don't with Operator, potential been time them, the agency

Operator

Skorney Brian from Baird.

Brian Skorney

fitting Good to in. the little me morning, Hey. was more thanks for second-generation everyone. I hoping color get guess a I And formulations. on

I think I heard remarks. in you prepared word the Prodrug the say

possible also, I characterize was any, the API, chemical what formulations? the kind Thanks. formulations, just wondering, chemical actual if that, in of So you utilize novel we about are development projecting And standard just looking at pathway acceptance you maybe the sort how think that? more and of patents. entity of XXXBX or structure that or are also new sort of in changes new references of to changes can on And to to kind

Scott Braunstein

the after And question. really one Bryan. question more we'll take you. Thanks, I appreciate

expect, past, formulations a novel take some s, made fantastic effort. additional and We we has from with work of -- pursuing much been standpoint the where preclinical and least the had at made There an prodrugs. building would both intellectual an We've we well we're that. work. We've in it So prodrug that some but progress. prodrugs we've with excipient portfolio larger are bit a are little could today certainly around very

As prodrug final formulation. a we to move

much the an I'm say, about, step in, being would think program -- second prodrug bioavailability think the a confident as we be the in with first And be would that this a set is where feel dosing formulations. the clinic formulations. 'XX, excipient-based have solubility, I'm that really improve patients. interested in -- I in likely of would our in and very hopeful of regimens honestly, the third-generation improved opportunity dosing either our we could actually thinking BID step. or first the in improve to, very will be a for one, step, to but have going I likely It's still s, process. we're dosing, step today the simplified I So that That TID prodrug is SR And utilizing quite second really we in today, technology prodrug

already can think -- the we work share early And building we're formulation, I -- in excipient with so some the we're an intellectual also of 'XX. I going to that property with goal. you formulation around with that it, we're Maybe we've clinic based to moving both to

very potential decisions So potential -- and the expectation. were -- X we the incrementally programs meaningful incrementally all a I about thinks on meaning There changes think has having and working today, now, will in prodrugs. right being a program that for But have I'm all Hope we're that we're to NCE fourth us or and hope enthusiastic that bit with all to your said, the a longer could take that's a that recent what familiar the with Prodrug property. still intellectual -- prodrug space. FDA doing work to to the was pursue do recent necessary And planning helpful. an are pre-clinical able guidelines. NCE point, we're for fact quite that lot with there's right those of been with path to way if just in And pathway, my our FDA now and has

Operator

Olson will line of Your come from with next Oppenheimer. the question Jay

Jay Olson

of what of Phase rare like a your in hey. maybe endpoints pediatric might Congrats that key just prevalence talk you the separately, thanks that And our in you disease about patient plans be voucher? questions. it progress Thank CDD. what some the about on LGS U.S. population? you. the needs trials? X all taking for are unmet review and Can And year some maybe Can look of efficacy eligible talk then voucher, the And and priority Europe? Marinus like seems assuming approval you Well, the would in for get in your next for

Scott Braunstein

little and for you voucher it flip we over then the to don't why LGS? have about Joe, a I'll Steve talk bit

Joe Hulihan

Okay.

Steve Pfanstiel

morning, Good Jay. Yes. Yeah.

that FDA, would we the part the CDD we from a receive pediatric expect rare as So of approval a would voucher.

that, that we use balance sheet, would I our of could look and at as think monetize to pretty us more that's look -- the we further and value and pipeline would we think to so quickly. at when the we something strengthen something we turn

So value, steady. if we got marketplace quickly approval at continued that, look that the to probably the early and out next balance year, development. that's a we sheet would try we When strengthen monetize value fund good in and the pretty very held there's use

So point. that's on pediatric use voucher this our the thinking of at the

Joe Hulihan

So --

Steve Pfanstiel

LGS? about talking We're

Joe Hulihan

Yeah.

XX,XXX seizures. there's of majority have for the considerable several are They're a and U.S. uncontrolled about they're or of in unmet there is range terms and seizure So XX,XXX Syndrome. in with The despite can the which adults of with the And types unfortunately, problems. disabilities frequency LGS, seizure terms in recent availability, of need different they developmental usually Lennox-Gastaut, in seizures, syndrome behavioral cause of children a Lennox-Gastaut the need slow still fairly wave. of spike patients supportive a developmental a needs of sometimes significantly unmet some range drop have as Lennox-Gastaut of injury, epileptic other they approved require lot encephalopathies drugs a particular other pattern and institutional and on care, still epileptic of EEG encephalopathies, treatment lot care. they have a They have called same like disabled

and there's a going compliment we be so think real available need could for be and to to there, a therapies to And the have other it adults. children Ganaxolone those available

Jay Olson

thank Great, you taking questions. for the

Scott Braunstein

team doing. really wins team's that teams you we're on I as for questions. had operator. thank our for and regulatory finish Christy, team Well, work And the working I great operations of to want going to I jumping some this want thank know I'm everyone calls. you the and The all out there. proud quarter. mentioned, difficult how in to busy that her think season building times clinical wrap, earnings just time it's the through

a Have everyone's look to appreciate support up I So live. forward following and day, good everyone.

Operator

and gentlemen, Ladies for conference call. you today's in thank participating

disconnect. You now may

Marinus Pharmaceuticals (MRNS) 10 Aug 212021 Q2 Earnings call transcript