MRNS - Earnings call transcript Q1 2020 | Marinus Pharmaceuticals, Inc.

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Operator

Greetings and welcome to the Marinus Pharmaceuticals First Quarter 2020 Earnings Call. At this time, all participants are in listen-only mode. A brief question-and-answer session will follow the formal presentation. [Operator Instructions].

As a reminder, this conference is being recorded.

pleasure Investor your Relations. my Ellis, Sasha now President, Vice it's to host, introduce Damouni And Thank you.

begin, may You Ms. Ellis.

Sasha Damouni Ellis

Officer; Joe Thank are you. Chief Dr. Dr. Marinus Smith, Medical Hulihan, Scott Braunstein, Financial With Chief Executive me Officer. and Edward from Chief Officer;

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

to call I will the now Scott. turn over

Scott Braunstein

and financial Good Sasha. update. everyone, you, to morning, business our welcome Thank XXXX and first quarter

today Financial have Joe I review mentioned, who Sasha our with who provide call, Chief the Chief our Hulihan, statements position. progress, the updates on our Officer Dr. clinical Smith, will company's on Officer, cash Medical and will financial Ed me and As

We the call. the will end of questions available be at for

off progress the to filing like company milestone that a for reflects first that significant nearing and NDA marks our data an made have update to and Marinus commercial This that would today's business call, this earnings important conference an trial pivotal with the acknowledge evolution call. Starting of launch. in both we I is potential is a the

disorders; FDA with more few implementation for program We the we financing a milestones, interaction have ganaxolone broader the to in will describe important continued range and unlock quarters shortly. promising to designed a and strategic that clinical plan, of potential several epilepticus you prior with across of clinical of encouraging seizure our an past in the from detail status rare the over including data our momentum year-;

up and an at to forward XXXX to keep be our exciting we shareholders as calls turned our opportunity well informed. Marinus, has out to quarterly time an having date and look already to

I'd been life COVID-XX been acknowledge moment take major colleagues we the we Marinus, many so have that, sciences. like have fortunate of delays the for a the challenging today, ongoing our to of that and avoided across to as impacts pandemic. At have

that Phase encouraged data II sclerosis trial pivotal CDKLX include deficiency trials upcoming which Phase or tuberous top remain the our are line clinical or from We initiation milestones, complex, on track. III our disorder, TSE, CDD, and our of in in

commitment This unprecedented They for us and am our to entire I has our has times. investigators. risen us, enabled dedication teams and continued a and have challenging difficult been grateful organization these our the navigate occasion. successfully the and of period for of to all

slightly share our summarizing for like Before roughly in a I been you highlights, our pursuing stepped moment ganaxolone. vision over a It with and has nine would to and, since months take time year role. executive CEO as program know, chairman I you to the full Marinus evolving as in

core the drive is and focus in trial forms skill These to a based strategy a the made the a our epilepsy, our both future leadership trial finally, opportunity with epilepsy space, clinical and clinical hires, be of our the throughout of epilepsy hospital. out meaningful focus include severe manufacturing a on and community. leader should needs driving the execution, to in treating have encouraging and key patients to set, strong our help demanding clinical that that series We changes unmet renewed broadening success. capabilities, design of on organization of see the science continuing We improve

to making strategy. help discipline and a appropriate the investments to to the while Equally will commercial successful continue important, financial we achieve apply us strong business, developmental

program brief that, before provide the Joe provide call, highlights. more I'd detailed clinical But on updates. like will some Later to

of to me our the status is damage in in near as lead to cases, brain rare prolonged off and, or patients that Let status permanent state some death. seizure can referred epilepticus are or with condition a to where Status program, a continuous also SE. continuous start activity

of from treatment Last of data primary year, with our the we Phase endpoint, as encouraging within II reported in dose-finding anesthetics, XX ganaxolone IV to progression of defined hours prevention study achieving patients SE, XXX% of IV initiation.

of we action saw of to minutes cessation evaluated. rapid time onset SE all five In with median a doses across addition,

no patients at having response primary the serum the dose and targeted time dose of highest of endpoint also at of ml, trend with We XX-hour concentration XXX status noted the our meeting and at numerical recurrence per a XXX% nanograms point.

available Additionally, of four we no week in visit. up follow-up, for the saw SE recurrence patients all to

has potential that capable a the status. new be for the addressing effective We are treatment of and to option, for safe powerful a ganaxolone confident need

rapid We believe in role uncontrolled provide a devastating play preventing of that and ganaxolone potentially of seizures. can a onset action consequences the

a including Phase plans have III progress, study Joe Phase excellent We FDA. and more constructive our provide meeting with the on have for plans the details later status end program call. a forward pivotal the made with moved will rapidly for of II our in

III of children CDD move refractory Study, in is our with treatments. oral enrollment double-blind, adults a of rare young our placebo-controlled, next efficacious and CDD. limited program. evaluating form quarter, ganaxolone announced Last epilepsy with trial, pivotal we CDD the global the Let's of Phase to pediatric completion Marigold

Importantly, trial on NDA year, track potential begun top for preparations line in have the report and from QX we file. the this remain to of data

build help commercial a have also strategy. our to started lead team We to

TSC suffering ganaxolone the Our possible work early opportunity evaluate the expansion in into use has commercial population. as fully of as to the begun the for well patients from CDD, market potential

greater We primary the those endpoint. detail meets should III will its plans Phase you sharing be with in

this We are looking the the forward concurrent to program our data set year. and about later sharing with you thinking

organs of lungs. skin, can we heart, lead announced expand TSC, common brain, disorder mentioned, many and epilepsy eyes franchise also non-malignant rare plans to is and affect As genetic tumors a cause ganaxolone have a kidneys, which into the our to of and that the

expand by to scientifically was informed sulfate, a of into new decision allopregnanolone TSC biomarker, our discovery or Allo-S. The

be Allo-S epilepsies respond important are ganaxolone. may treatment Phase epilepsy. our predict in During to with to genetic to believe an study II likely most rare We biomarker related which PCDHXX

have of TSC abnormal ganaxolone that indicated impacted by as may that the well from as the those allopregnanolone patients production are genetic one neuro be thus that rare of likelihood will analyses epilepsies benefit Our increasing treatment. other steroids,

in new patients benefits and allows the We need, an biology-driven to greatest which believe our chapter is ganaxolone, with this success. medical mechanistically of chance of strategic advancement approach on for maximizing relevant, us indications focus potential unmet important also while our clinical

PCDHXX to have pilot III careful proof-of-concept with a clinical ongoing decision Allo-S strategic to Phase made mutation. as the biomarker transition we our larger the of a part As study a patients evaluating plan, in confirmed trial

PCDHXX without disturbances related and a accompanying reminder, a variable disability. with serious epilepsy rare seizures, characterized and intellectual by behavioral disease early onset or As is comorbid, cognitive and cluster

that the nature factors overall the well for opportunity us of trial many a PCDHXX running limited potential associated were seizures resources study global the in There led with with enrollment, required study, the patients, the during challenges pandemic. including pivotal as the limit as COVID-XX to associated commercial episodic a global indication

that indications will allow us available capital by where transition is population treatments. this to significant currently on believe patient we there resources underserved our is a Overall, focus

be our that for will believe additional provide we provide resources. to for PCDHXX. updated explore therapy strongly the best will no substantially study. could and longer part to program will eligible of from as patients this details benefit We ganaxolone use clinical on But pilot commitment continue is opportunities that for of Joe to community, our this our that the

I'd updates, corporate I over detailed to little achievements Before a highlighting to quarter. the transition for clinical time call the like more spend for Joe our

have the and disorder experts. Dr. University globally recognized fortunate Hirsch Yale School Medical Dr. NYU scientific Hospital; Health. our of Rogawski University; Dr. Medicine; UC board from We recently and Davis Husain from announced seizure Dr. Vaitkevicius from Brigham Dr. the French Langone and from are Medicine; of Trinka formation Women's Dr. Paracelsus advisory from Health; from six to Duke

have want drug are consensus epilepticus. contributed field pioneered establishment list, the diagnosis of While steroids the long our status and of SAB antiepileptic multiple too to treatment on highlight of the that to the approvals, leading are accomplishments newly of members team I of and formed neuroactive to the international

enthusiasm to strategies leveraging forward and testament of outcomes to participation potential scientific and improve our and is guidance ganaxolone as epilepsies, the the a of advisors on that ganaxolone look to global severe their advance the clinical registration believe of and We through expertise clinic. patients with development we

Vice appointment Sasha experience of thrilled addition, as Corporate Ellis including media. stakeholders, Marinus to and visibility, of to roles as Damouni with brings and we the financial organizations will extensive look Communications. In communication, well key of Investor relationships care President in health Sasha and across Relations announce diverse patient to the employees, invaluable as executive strengthen and which issues as we advocacy groups, our management be investors, were

put to right we work. see, can Sasha you As

XXXX be development Marinus is of ganaxolone, next exciting phase of and include the at the to going clinical two trials. pivotal will with late trial which time an stage additional data

a life strong potential of unlock number transform and and year for severe the threatening plans to throughout execution the continued our expect of paradigm strategic the forward We implementation look treatment epilepsy. the of to to ganaxolone

the Officer, With Medical like to Hulihan. that, I call would Joe? turn over to now Chief Joe our

Joe Hulihan

Thank you, Good everyone. morning, Scott.

you mentioned, milestones plans are for and these exciting clinical update and on times XXXX key like Marinus, Scott beyond. to at As I'd and our

Last year, epilepticus. reported plans positive II enabling our rapidly dose we results, And our III Phase advanced data starting Phase we've registration based with encouraging So, status a these finding on off from study. trial. for

can patient we've primary Phase reached design, productive a including the and on collaborative trial end-of-Phase the population. paradigm dosing endpoints, the of We with and aspects that had the general report III and II highly FDA, alignment meeting our key

co-primary epilepticus by two reason status the – be outcome findings control. optimistic the endpoints, stopping informed The status was major these about seizure us rapidly endpoints study address II, in maintaining Phase for and to and Phase III. The give has selection of efficacy Epilepticus from in which the criteria

minutes, than but cessation XX was we II, the and median less patient. all Phase five minutes for to time it In seizure saw was one

with endpoint of treatments rapid needed. also alternative is without quickly to co-primary the of for first XX treatment. additional with III proportion patients endpoint if only physician the within intervene So, onset This reflects allows cessation minutes Phase it seizure not the action,

the anesthesia Phase the anesthesia within XX The was XX In is of primary endpoint of II of studies, at patients other the treatment, endpoint. progression for IV least percentage IV hours. initiating study's required co-primary none hours to XX which patients the without

control meaningful ICU drive is admission the for need measure Morbidity status and cost considerable, are clinical in itself. and of the health for anesthesia and and endpoint a and as of also IV stays epilepticus This care. mortality hospital and with is proxy of important prolonged economic an

III, on FDA this We controlling The Phase of for II ml hours at level III. Phase aspect eight. ganaxolone that Phase selection improvements, in study level a be the a eight will rather also was informed we status. completely based blood able to study. aspect of dose we hours formulation maintain the on maintenance blood for ganaxolone nanograms agreement our that per have XXX And target found of XX key least than for at from In

enroll then to and a have benzodiazepine line between of The to placebo. first With second-line study study. trial and drug respond who of will which to a detect medications, will failed power difference line approximately has the Phase status failed than treatments anti-seizure II XX% greater participants, that in second the patients the two average number number additional XXX was treatment with XX%

the in with the We to of the as dialogue look year. forward this protocol to trial quarter patient we continued third begin enrollment expect FDA and finalize

to I'm pleased ensure share which enrollment. will sites, rapid also begun have readying and already identifying the that initiation we of patient trial

in care emergency. XXXX clinically line that top of data to first and for half a and the will results catastrophic expect study guide We common the provide feel meaningful neurologic

Turning CDD. to

Phase Marigold pivotal As we and study in oral ganaxolone III children evaluating in completed young adults Scott the have reviewed, enrollment CDD. study, with our

completed confirmed and reminder, As a CDKLX double-blind, trial global, in between the XX, X placebo-controlled a gene of schedule enrollment patients has on this patients. with ages of XXX

than less of can enrollment I that open rate are high of be rate trial. label update discontinuation in the we Today, and the continued continues encouraged that XX%, the by extension the arm an provide to

to track and year. diaries been to access track in We through COVID-XX fast data on the from on the the this third patient us top trial of approaching seizure patient remain of keep line required disruption remotely. are allowing electronic our last able and trial assessments use We've and analyze data despite visit the quarter is report to

for We that actively NDA would have III successful outcome. preparations begun follow the filing a Phase

in step newest believe likely benefit our to the next TSC, or indications program, next Moving tuberous which we is marks biomarker-driven strategy patients. complex, where our to target clinical sclerosis ganaxolone most to

of a a can illness, complex aspects The are a TSC most that often leading the said, types diverse TSC patient's is change lifespan seizures of one treatment manifestations can and difficult epilepsy. multiple Scott cause of that of disease with be is As resistant. the seizure with across genetically-determined and

And response neurosteroid relevant factor potential our levels an a as has with TSC biomarker a causation. identified discovery of was Allo-S to potential as in especially individuals imbalance in endogenous since seizure been

target imbalance. would this Ganaxolone directly

Our discovery potential epilepsy of being affected. expect where range genetic a of us would Allo-S biomarker as other highest also ganaxolone allowed the we have investigate a to has likelihood determine for epilepsies of to to

while epilepsy the challenging, would also journey finding for treatment. of If that confirmed, those for patients searching while years-long most benefit, one the can an preventing effective likely patients focuses medication treatment, use to goal in this face often realize the individualized

open safety patients XX and Our of ganaxolone evaluate XX Phase from will to TSC adjunctive II seizures. the refractory in approximately ages with X highly label tolerability in trial

participants reduction to primary whether biomarker XX-week Study up monthly Allo-S criteria ganaxolone an receive frequency analyze a may an in XXX liquid endpoint and is in XX seizure we meet extension. will times to In levels our suspension a for who with our plan ganaxolone is continue day baseline. of between Patients association to eligibility relative oral weeks. three percent of The improvements. treatment seizure there line strategy, milligrams

open. planned trial data do anticipate with line beginning top considerable to We made third in the sites any of already screen in in enroll trial, six already XXXX. the quarter plan anticipate preparing during delays initiation of related from and We've We not and quarter, trial for study of to patients progress to the the this this report one COVID-XX. progress

we Violet call, related trial a study. decided the like the to proof-of-concept in Scott have Earlier touch III in on the that PCDHXX I'd Study, to Finally, to ongoing our Phase transition program epilepsy. detailed

patients groups each ganaxolone into stratum. within double data, This to they study trial Allo-S stratify consist XX-week a on two and levels will phase. collect to of followed prospective blind period based placebo seizure randomized by baseline biomarker a one will be treatment The or or will

to Patients for times a to up suspension randomized will titrate ganaxolone liquid day that XXX goes weeks XX of over weeks. three maintain dose oral of a four milligrams to and

top double the patients XXXX. XX of the of We the trial approximately line first with to in data expect to half complete portion blind XX and anticipate

we improve With unmet with these we potential have ganaxolone clinical studies, a has outcomes. program on to focused developed rare epilepsies high need, believe patient and real where

many months next our look of to will progress with milestones initiation stage study late you. and pivotal and new The the sharing our forward we few see program data trials, from with

despite that their continuity rapidly to to the the call turn care sincerely studies and COVID-XX. Before investigators personnel, programs health Ed advancing review new over could the yield financials, by posed to challenges our our I ensured study thank dedication our I'd like to site the whose of system has and to treatments to patients evolving

on that like thank Marinus, and trials. improvements their participated And patients would to efforts of of meaningful severe will most We're these disorders. behalf our lead families increasingly who neurologic deal in and for I who patients must such the challenges the families with confident to importantly,

turn to that, Ed. call the I'll With over

Edward Smith

morning, good And Joe. everyone. Thanks,

continued the our expenses quarter clinical Our morning programs of ongoing for development ganaxolone. this associated first released financial reflect the results XXXX for with

mostly most of CDD. activities with expense first the NDA-enabling advanced for support attributable of in same million program clinical in clinical development increase $XX This and quarter the research For manufacturing and XXXX, XXXX. in our expenses in to period were million the $X.X compared R&D is

costs incur status trial Phase our pivotal In addition, II have Phase in last a which reduced with associated commence of III to preparations the offset to by PPD by completion partially year. driven epilepticus, are studies non-seizure we costs our begun disorder

respectively. year General costs for which and and of $X.X consistent first were versus million are this administrative million quarter-to-quarter, quarter $X.X last the

loss XXXX the a in for million share or net $XX.X net per and basic diluted share and period $X.XX Our compared diluted was same the quarter for basic of per $XX.X XXXX. $X.XX with loss of or first million

As the of the in end approximately XX, approximately and XXXX, March at equivalents compared investments of year. had $XX.X $XX.X cash cash, million with we million

Our XXXX, runway inflows potential the provide investments any consideration by quarter cash Joe. into scale cash assuming and Scott to our current operations and of of into third taking company without the current earlier described as

We support want Marinus programs. Before of to through progress opening and confidence I for dedication the call thank for XXXX. to questions, management and momentum our the their continued to team for and I'd continued all also hard mission. our our thank to, course, the employees look work entire investors and in forward like

you. that, now we questions. the Thank will open And for with call

Operator

Thank [Operator you. Instructions].

is Marc question Goodman, Leerink. first from sir. SVB Please go ahead, The

Marc Goodman

Yes. Good guys. morning,

assume So, we as from should be program that far that this line the over basically you're to PCDHXX indication? from first is is, that the to get for conversation question about that TSC switch goal a pursuing going approval as a to

all, delays. there? how to that? on what avoided about like And press you're in issues? Can or that not to don't you've be just was seems hear release why I is? about some having CDD you everybody trials, But second specifically And supportive of Phase talk can COVID trial give clinical There there to. trouble. you something no more the Thanks. can it And delays, could talk you meant and you be it's color It plan just seems about these you great

Scott Braunstein

to we best everyone trying this Hope and and in kick we're you're mute our before off different are Sasha as locations. appreciate, myself doing we Good answer. morning, morning, Joe, Marc. as And our it phones well. can And

sure a we'll make there's everyone. we if delay, So, remind

strong TSC? in first assume of half Again, all study Yes, II. strategic tackle mind for TSC. like the to year me about plans next And should TSC, point through PCDHXX is we a in TSC Phase would be Phase in a that of in think programs we'd the III move you interested CDD Marc, I on positive that question, program that our initiating let our outcome first relative concept to rapidly our the if So, and proof a direction. very in with

efficacy TSC. TSC that to reasons. from at of substantially signal the about market and least in is ganaxolone equally data sample the in a strong thinking biomarker standpoint for We're the We think potential several is larger. blood We think support

there's huge drug of TSC study amount feedback the to investigator how with that we data community And a the in very proof-of-concept interest population. seeing Our our plays out. additional in interested are

On as you was that PCDHXX, study a the was took already flip side, I as Marc, underway when over know, chairman. for this executive

in market saw here. we patients research certainly in III, Phase about potential early much think opportunity. concerns the were but market were a trial market the a find do was that, to the there, enough some of days smaller clinical ultimately, the our could had I number this We that I of out we that work, concerned

with Many medications. and spoken handful really up between I've all would very XX%, a supplemental different that's with children it's than say, their on current it's XX% XX% fashion. only even very that population. CDD well again, wind a the I of anti-seizure needing And TSC somewhere patients, regimen. episodic of As And and, controlled you, PCDHXX are

trial resource of a move PCDHXX and Phase really from decision a is with our III strategy allocation. So, filing one away to

at hope at produce at data some that least of able to blinded at low being continued think III levels. from And abnormally in We'll be, would high programs Phase would patients least if programs we or proof-of-concept PCDHXX. a what now a our an multiple in consider in allo biomarker that, show patients levels would of positive a III run look results genetic in going positive to I orphan broader We're allo Phase of IV PCDHXX epilepsies, a to we a strategy true said, allo Phase really and about continue that very being a data, consider all part as our programs. that strongly see that data number our strategy Now to can study be Marc, good biomarker future included to I study think really and a show study. both the

PCDHXX switch the So, logic that's the from really TSC. to around

think we I we can think things is And favor. the significantly in our opportunity need as work said, as unmet several all timelines, really the is larger times condense And if well. market larger. I

lot to design. we make from is a minute Joe lot But well. electronic have at our standpoint, come few it to doing A CDD a relieves fortunate. lot that A is it fact move from is of is of the a serendipity. as ask timing. are clinical that an standpoint, of I'll visit it COVID efficacy to But trial in the issue, A a to that comments a home, patients system patients just lot our the been stress diary to data needing of hospital. trial

said, we at could our February, businesses really things some That office February. investigators. team in telephonic supply But of had in February, chain thinking that of looked very before got at early was do. really about and that here investors our the being US. about We middle closed we all the of minds kind the very talked our activity moving early visits started the move to to in in that were and We for much

telephonically in good at can we're The that And very data, shape home with that timing from or trial. about so, the efficacy the feel CDD of of we until trial. feel all trial standpoint really be end very the the patient done fortunate we that tail visits very confident the around

the of minutes. up, Status meeting should bit half debt. of and again. ability to into open our starts with heard wait-and-see, little summer quickly the fortunate world final agency right? We very the End as meeting that, II in the in the a Phase minimal of of to go is have expectation the second from III is And agency telephonically. our hope We're a terms Phase

at as that we're and our stand to trial challenge. today, in will for course, feeling where planned. But Of reasonably reemerges are open if real least hospitals that be we a ability extremely had confident we busy, COVID-XX

of that, sites now want trial. doing Our recruitment clinical a of sites ops with job numbers worse, of as very we feel who part in get our Those doesn't team particular now, III feel doesn't right long our should world in of we on as be Right incredible as on confident is the be though an the worse get are Phase working terms and the plan time. US to track.

terms trials, center there a many and of Phase clin this I In I a my the reopen. supporting like brought potential last actually a of Hall, actually you But – centers hotel We, hotel know, I several approval. about COVID. I put head the use ops, that placed trials a as by – They'll we're are goes Phase about heard will other Phase been for our as seeing are solution. CROs and into We've required if week, affected it FDA of all the then talked for have of be and closing. companies, Phase one that they'll we I all I Phase into in be patients starting to screened center Matt well,

And expect to the or a so, one filing, for or Phase trials work, is month a on our that which fall. but all rate will them I limiting expectation to summer we not is in our those track be delay, back have three NDA the our

or TSC, add? PCDHXX want about to that you trial Joe, that work general I any thoughts comments mention didn't that clinical on your either

Joe Hulihan

a for PCDHXX TSC, to study Scott. – we're and think Well, No, proof-of-concept. is I thanks, continue the a Yeah. PCDHXX going as

I terms of is to degree think of perhaps And perhaps think that biomarker interesting of more particularly kind and enroll been need enrollment. TSC hypothesis. the challenging of of lesser would I a unmet one that's marker terms in have of in rapidity

that the on For time Scott was enrollment CDD, the issues The enrolled enrolled mentioned team and trial track was problems COVID-XX – completely enrollment with around it hit. that again, serendipity. the

was earlier able screen PCDHXX And be needed Patients so, phase. CDD, we Patients be this remote need be patient screened had to an And But in get that visits. didn't in anymore the done done to were remote labs. We and patients. do can person. to remotely. could to the

things electronic to so, time as we much as And they remotely. can access did mentioned, seizure the efficacy data can Scott could everything again, on they done that diary, And get also, remotely. the so and we have team do

Scott Braunstein

to… follow-ups any Marc,

Scott Braunstein

you. Thanks. Thank

Scott Braunstein

Yeah.

Operator

The sir. go next is Oppenheimer. from ahead, Olson, question Jay Please

Jay Olson

on treatment the questions. this for Epidiolex impact and the progress study and ganaxolone wondering TSC. TSC? that is decision enrollment an for expect collecting? data Allo-S landscape, impact I There's up FDA assuming it congrats TSC eventually with your that approved for how expect year coming And thanks to how you the taking perhaps for to you of Hey, And differentiate do the later drug biomarker was you're

Scott Braunstein

prescription with through very last is access. TSC December. work off-label Epidiolex. we the is thanks and either some quite being a question, today. TSC are whom at around quite that for for And program comments of of Thanks the a The few appreciate And product patients Jay. feedback expanded from investigators, that already today, the nice comfortable AES and on lot bit of via our are you patients or there were dialing today in We getting used the a did

our using or can't are the program. Epidiolex design that time very new excluded expanded that's you a logical that to trial for sites the on they access, In trial the therapy. all of are seeing and GW from part That's clinical because add

begin already and the access We using they we interested And of later all So, we fact, of are the enrolling not being our expanded few in have don't have to some expect the Epidiolex in talked a our background in they all TSC about a of And certainly, as patients patients chosen are have of of XXX% us in II streaming of centers think in the shortly in been approval format. that we'll terms that are excited significant limiting have factor. that'll would the study year we've our Epidiolex number therapy. announced and in physicians, Epidiolex rate at study. be very Phase quite a this our where valuable investigators sites very be see And read. to opportunity told

we our So, think that all not impact will enrollment. at approval

you'd Joe, any add Epidiolex? around other like to comments that

Joe Hulihan

standpoint, still a clinical lot unfortunately, need. responds a From Yeah. to differently there's medications. Every of unmet patient

us, availability tell Scott seizure despite will be qualify of that the they investigators refractory. have study, that can other as the Seizures in Our Epidiolex medications. quite or any mentioned, patients TSC for

So, I the of be enrollment on we'll for study. think track

Jay Olson

seems confusion S-X preferred be some Great. could, housekeeping And you. the about and a if I the That's There common in squeeze very share and that about helpful. question to Thank you maybe week. components conversion. last filed

you please describe your maybe great. plans there. be So, That would if could,

Scott Braunstein

Thanks, turn you do Jay. to Ed, it Ed. to over want take that? And, I'll

Edward Smith

got that I it. did that filed that S-X we morning. as Yeah, stock we part the Yeah, financing good last week the preferred Thank of so sold relates we the And in to you. December.

that mandatorily a preferred effectiveness we convert count, the that both the we statement into secondly, stock of in that common will in preferred underlies and just December filed, share registration the So, sold stock. authorized it's the registration statement the large our preferred. common that And that covers essentially, increase upon in the part

either occur to month. expect this month So, we or would next later that

Jay Olson

for on again thanks taking again very the progress. questions. That's Congrats Great. helpful. the And

Scott Braunstein

Jay. Stay Thanks, well.

Operator

go ahead. a question from Fitzgerald. Cantor Alethia Please have We Young,

Alethia Young

kind of somewhere there? thinking you range do you the if kind with it Hey, you need guys. I question. the be the Thanks you in anything guess incremental guys can for How neighborhood. was [indiscernible]? to or couple. provide could you Again, progress. I And little lower, FDA talk a little my conversations of had was on a that that expect that taking would One, bit guys congrats about a in

of kind PCDHXX, just it's with is some biomarker with touched people I the your degree. [indiscernible] maybe not [indiscernible] in place follow-up. a the to And kind put of just I think it on maybe the somewhat – before, chips, then, then light best have especially [indiscernible] And of to

Scott Braunstein

every hope puppy bigger too. you're And Thanks, great staying to Alethia. hear day I getting your well.

then turn FDA and Let interaction. me commentary for little handle more the it on the a PCDHXX Joe to question, bit I'll over

agency And the forward that the to a And about very say, to with study. we just I'll move study. we we're much that are a got happy just aligned it's this complex interaction, way best on with the FDA place really

So, easy turn I'll a think for And Joe bit more commentary it it to said, over little as I wasn't there. interaction. an I

been Again, study is few that I that we started this the PCDHXX, would say has it. from challenging a day On a things.

going tricky. I lot study resources realized of type of And is to that particularly global a think it COVID-XX environment we in scale. was take a certainly, a on

would this We good on to at allocation I and aside, but make And I a as planes things our have people Dollars what having going company. ultimately is, that study to I a some really has organizational have the was great of get potential. market challenges one – on take individual standpoint had real of resources. our have had the then to an from a limited on lot decisions of said,

Now, still that biomarker is real. the being said, signal and very important very we think

all that in see in you'll in states clump, think population. come I all new morning, think three of our out that we're production what of these we really so speak, what our terms can to in see driven of TSC. you And deck to I and in us later seeing allopregnanolone young slide the saw it unequivocally disease programs, has will this PCDHXX this under

in on study we for with from question TSC. right able the III aggressively think I Phase early be turning question, are, we trials today, year-end as share be standpoint, And and a positive believe it other I that on looking proof-of-concept still a the forward have there. positive to Marc's see to drove we year, we'll into our heels of from to earlier. – but And question, see so, earlier where move And next program we real that concept a can of from is mentioned is we around think no Phase there a the quickly TSC very accelerate And we'll decision think But part signal a people us a should the be standpoint, No PCDHXX TSC that you to we should a program story. resource signals III can CDD. the inhouse we study to data proof company. certainly,

that? to Joe, the Let to FDA take turn want question Joe. you me over

Joe Hulihan

Sure. Yeah.

design. the study. I'll a trial outlines think, collegial – collegial the I about discussion it Now, FDA gained, we of the And had the major on with call we agreement

complex collaborative out But a a path. suggestions point. It's did with working think from this a at well-worn meeting we're you I that. was details It's trial. not about, it FDA the this consider and

pleased final do. And and details the But we'll I the look was meeting. hopefully, FDA. have have we'll to We're And very the once the of so, protocol think forward we the outcome all protocol. talk finalized we soon sharing I continuing the with with to

Alethia Young

[indiscernible]. And there that may [indiscernible] in is you trial then, as follow-up, a the

Joe Hulihan

status, For no.

size You asked a to that. I size. we'll that's in we're good sample COVID-XX think we'll then I able enroll. holding calculations. And true, sample recurrence, think think that we're there's I about hopefully – if Scott in shape to we'll as – the But mentioned, same comes Again, if be the have still Yeah, address – having regard. there's

Alethia Young

you. Okay, thank

Scott Braunstein

Thanks, Alethia.

Operator

Tsao, is The Douglas sir. go ahead, next Please Wainright. question from H.C.

Douglas Tsao

Thanks morning. taking Hi, questions. good for the

in the out patients to terms strong bit trying be initial go trial, benzodiazepine. little failed patients COVID-XX, was bit had of given sort It Just know Is of other that sounds like of other execute similar there treatment that in results anti-epileptics it's just of incorporate thoughts were to needing results, terms of some be given there of Phase in that coming might as I II, some to to thoughts Thank a just just the a to more trying in or as Phase of of the have a complex. terms very the a little a of on the going earlier. trial, III, incredibly status you. the sort thought terms couple study replicated with ability well

Scott Braunstein

Thanks, Doug. well. over I'll Joe as I'll turn kick and it then to off, it

think surprised somewhat Doug, failed XX patients intervention one out that mean cases, were II prior at allowed And we where X.X in treatments. surprised we Phase therapy, in and, Phase anti-epileptics those design the first benzodiazepines least anti-epileptic. the two were that prior after failed was one II, and foremost, failed about most I our of of XX

thinking III confident, in to experience is responding now impact. around patients is failed with thinking value agent placebo that particularly this anti-epileptics, feel have medical these really the our driving the low, if probability community, that's of public I proposition, the extremely health a all a our the really data and economic and to And benzodiazepine rate. we Phase our prior and the for their two given very fourth Part think want the domain of show we

we to So, placebo we can of a placebo as or think, And than of a in thinking. the – think setting. world about a critical see a we XX%, a I very that delta higher real rate absorb would expect I well think was, much piece

the the talk one second world what after of agent think typically that's piece, single class. epileptologists or a get is, you these other you'll real benzodiazepine, there. then I you different to a any neuro-intensivists, and a out of When agent a see

non-sodium as example. channel start blocker an channel may and sodium a blocker, You a to move with then

and real we the trial. real world. world so, living that's And want that think are very I actually the That's we to run in

really to want up. comments, up for important you not I that end we we're is point, to bring think I Before his a where starting where Doug, Joe turn over

significantly we drugs Slightly States of XXX,XXX. second So, three behind in response that, have United data somewhere those an north and It's more patients drugs rate. today, the XX,XXX at as largest between And the than none give stroke. are know used in are there a But status XX,XXX than the US that XX% example, when or refractory the take, we look that. the of larger below commonly ESETT that emergency neurologic market is

of higher and trial while patients. And registration And in therapy. as Phase That's emergency substantially a into on Phase a study. IV by and that's view, should refractory to sickest the do drug, study. that a ICU with be our run in have the second our drug neuro think move in III a up room believe And But we patients. so, different that to line really this we right success first a an build program we population the line ultimately, study, the and way, that then will has front line rate way is a second

So, other I'll stop comments like there. to that Joe, add? any you'd

Joe Hulihan

No, lot, people to not of the which on to point at in whole we're the intervening brink Scott. go IV trial, having will be The a anesthesia.

Phase population And can avert we is level treatment Scott is low. to the care. escalated the study the to II. patients who And rapidly essentially, anesthesia. don't IV as the the modified suggested, close similar so, they study if care response that at Phase is to placebo, respond, data we've II. be point mentioned, study to progression works, progression Patients very on But, population of in of etiologies, The slightly. The the very that next

Douglas Tsao

room of and for changed? highlighting that lot maybe. And in just improvement, Joe, Are terms that versus you any as a are you the that success have might results changes Phase that Phase of learnings given saw the you follow-up enhancing sort Not on a probability of worth of to there just what think that, study? based II II just of things but the types in just curious

Joe Hulihan

dosing drug question. your the is probably to thing relevant Well, the most

data thing And above, blood of hours. key lucky have the in us we for that terms III, target the maintained patients per dose, level, so three more total were So, groups. data, were level. the group ml who XXX efficacy, that was Phase that than One daily to target the or showed maintaining there maintain in blood nanograms level a four

it Another group eight maintained for hours.

activity. groups, group very in level hours eight at eight achieve continuous The blood controlled the The during did controlling EEG but we hour all in for the better infusion. well that group level didn't the at EEG. that had infusion Status looking all maintained better. third that And did blood EEG, was

on XX that continue breaking the that anesthesia that's care interrupt it the to to benzodiazepine for have clears new a you and break we "breaking of the chance board but And hopefully early us a because longer. kept it's So, abnormal and IV hope, forever. status allow will, we is extend it III, better status. other very Obviously, the late in can will end, XX on whether circuitry that's hours. to or don't the hours quickly, brain the the reset, given a we idea in or seizures," Any or Phase in care, actually the of formulation, give

keep status. the broken you've the for lighten it. patient a of and anesthesia time You under certain Hopefully, period then

that that we're X rather chance hours an hours the I So, better would even us And give think hoping than major XX that's intervene stop and difference. status. the to

Douglas Tsao

on to follow-up program. one maybe pursuing the just pathway? a the How like you're envision do from regulatory focus then the really going And epilepsy CDD, sound much standpoint? great. biomarker it on you after on Okay, Does that more Allo

Scott Braunstein

personalized it bucket. fits the medicine within that think I

Scott broadly developmental signal from CDD, and to more epilepsies, look across depending on the at the As biomarker the the study the may we epilepsies mentioned, genetic Allo-S hypothesis. further

think so, and inform where do of TSC, CDD developmental pediatric what us I And we next terms that'll with epilepsy. well about in between go we

Douglas Tsao

so you thank Great, much.

Scott Braunstein

Thanks, cognizant to be of Doug. this I noticed XX:XX. it's I And want time morning. the

caller. take didn't a take And we'll afternoon a we don't all So, and more more day call? available we to one questions later get if apologies question One to operator, be why more But my from calls. few today.

please. question, more one operator, So,

Operator

you. Thank

go question. Michael Higgins, sir. a Please Ladenburg ahead, Thalmann. have from We

Michael Higgins

morning, more Good in A on the despite Thanks couple conditions. continued Congrats the progress taking challenging questions quick development. guys. on pipeline here. of one for the

high rate is extension be? the us On Marigold, some label is, that more to you you might mentioned that give as detail high. open the how enrollment Can into what

Scott Braunstein

Thanks, Michael.

where we out for seizure those starting those think take giving nothing there's that seen And gives we have numbers for certainly would in results bad the to opportunities we've with study there to think open us side numbers the off, open I on not concern. would be not I'd that expect children we of treatments We're expect I say but specifically, lot reason any a a disorder, high. there's cautiously. label label or

at, was extremely I And that the know surprising important seeing also we things think very one child I the think happy anything that's of not lot we're the had and we side, on that's looking of dosing. design a around And effects one we're tolerability the there. and drug discontinuation well. side in flexibility we're the our that and again, an of

well. think And something comfortable investigator the I community is so, it's with very as

going the go right closing we're going but for Michael, just question. to And, remarks. all to say to that's I'm now, So, operator, thanks

Okay?

Scott Braunstein

treatment So, conference you to we providing be to for our we an forward confidence. you quarterly ganaxolone exciting, XXXX updates your and in call. thank all thank want rare all just will We us joining potentially the today And for continued to look as transformative for first year advance milestone-rich I seizure for coming Marinus. for disorders. support the months

Sasha wanted has with an that upcoming event discuss you. Sasha? she to

Sasha Damouni Ellis

Thanks, Scott.

updates our groups. and opinion day. we The with treatment our advocacy during XX, commercial clinical June efforts showcasing management, key be will event Marinus landscape, opportunities ganaxolone and and leaders, highlight we'll improve pipeline on So, potential to the virtual will presentations R&D expansion current include from

joining Thank the today. call you, for everyone,

Operator

teleconference. concludes today's This

out time. safe and there. be your for participation this disconnect your You may you Thank lines at

Marinus Pharmaceuticals (MRNS) 4 May 202020 Q1 Earnings call transcript