MRNS - Earnings call transcript Q2 2024 | Marinus Pharmaceuticals, Inc.

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Operator

Greetings, and welcome to the Marinus Pharmaceuticals Second Quarter Financial Results and Business Update Call. [Operator Instructions] It is now my pleasure to introduce your host, Sonya Weigle, Chief People and Investor Relations Officer.

You may begin, Ms. Weigle.

Sonya Weigle

Thank you, and good morning. are Marinus from me Dr. With and Scott Senior Dr. Officer; Lejuwaan, Executive President Braunstein, and Epilepsy; Chairman Vice Genetic Business Lisa Rare Chief Unit lead, Hulihan, Financial Pfanstiel, Officer. and Medical and Chief Operating Joe Chief Chief Officer; Officer Steven

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

over now CEO, the our will Dr. Braunstein. turn call to Scott I

Scott Braunstein

you, also our we everyone, of and and call, plans complex operational we're scale. team joining III sclerosis for tuberous a will morning. clinical this the second patients made ZTALMY the have readout thanks, in throughout on progress our us upcoming the year for for the discuss and On preparing global significant We'll commercial, beyond, today's highlight access that Sonya, Thank Phase to how expanding for including and half XXXX.

highly profitability a the of our no has success with ahead its and the a our U.S. CDKLX XXXX, team that our now accomplishments the we deficiency initial growing Starting in ZTALMY, in achieved particularly brand proven attaining be been first easy regulatory space in the in on of be has task, to well have product, orphan since but commercial globally. disorder. cannot launch investment quarter it of X is years new epilepsy expectations. prouder And the the I commercial ability commercial Launching effective in

made across the for to for was progress ZTALMY scientific I'm We associated world. bolstered have efforts and years a CDD the that we with of also that expand proud the believe first meetings age presentations treatment patients considerable number July, only year-end. announced will X medical ZTALMY In we as several seizures of patients older. by around these of to China be in access approved in by

for as responsible to as quarter China of the is in and Biotechnology expects of launch commercialization ganaxolone early Tenacia XXXX. ZTALMY first

collaboration a established As the Russia reminder, permissible and without MENA make Europe name China, were basis access regulations to Marinus a in program has distribution in commercial agreements under and agreements available local approval. managed on patient regulatory ganaxolone and

with on Russia our supply in those to patient achieved ganaxolone and in regulatory basis approvals to We partners plan distribution MENA are work markets. begin immediately to a named before

expect on engaged and Unipar weeks. Canada health countries, several and we care named Australia the our patient use supply the first patient in to have a Additionally, approved for a company including have specialty Group, additional ganaxolone within basis to coming

months in as second the is investment help has updated manufacturing second It achieve ongoing our facility us to projections. been mid- The as of approved with expected to now the the ZTALMY based for forecast to able well our on U.K. our have European U.S., expand Union, China long-range facility as place TSC for a manufacturing to forecast. in have is and meet critical demand important investments our CDD, be increased With capacity we several global made proceeding and planned. patients to and

this early market year have we ex in several or the launch off launches Tenacia of end XXXX. first of the by currently international to kicking Orion as with near into term, our expect next With anticipated year. the to U.S. In support partners, the Corporation the Europe, Chinese in And for launches previously, expects EU of commercial prepare ZTALMY select countries. continues early in to noted

are approvals in patients for local regulatory secured. We also assets broader anticipate and MENA once Russia

we Japanese partner following the TSC targeting half data Finally, first board to set. a Phase in III XXXX come our on of are

is U.S., United of forecast. incidence meaningful the to similar the be the outside our we believe global CDD to Since ZTALMY States opportunity that will the

example, was the identified. ago, to few where ZTALMY profitability genetic testing only new years. X,XXX already the therapeutic have bring achieving financial to CDD as instituted access patients years over larger objective support well provide China, Broader corporate a families their will a in For been and patients specifically the option over for as coming company's

business CDD in-depth our ZTALMY franchise the commercial including more overview Lejuwaan, our TSC of to preparing half heads will team grow to how the for provide of the potential and her remarks, next the is up who Lisa the in strategy second U.S. in plans a continue launch year.

to move pipeline. clinical me our Let

remarks, next our the ZTALMY As associated readout Joe Trust evaluating TSC milestone treatment his of detail the Global is of seizures TSC. for the major in will with trial

are we the reminder, a enrollment visit As last mid-September. for track on completed May in and by patient

the to in are the supplemental later of for NDA a April fourth expect review. quarter top data targeting the and than XXXX of with We report submission first half no request line priority

plans rate running. We the percentage overall the open-label ZTALMY FDA leverage to and a portion a and to the trial approval, discontinuation both the of to are the patients existing positive of high see outcome double-blind ground rolling a infrastructure the hit commercial low to pleased over extension. from team Pending

years formula and We our a adapt X last to positioned CDD are in the and to well learned TSC amount larger the have market. achieve tremendous in success

are XX profitability, now occur achieving which As sights following believe we launch. on earlier, our the setting can we XX company months TSC to I commented

analyst an event We our on on also we franchise. that oral investor will share wanted to focused host September and XX,

the joined patient landscape the TSC into to Our share and dive opinionators by be market team journey key TSC deep opportunity. treatment current a will leadership

still organization. the ZTALMY the some and review share Phase of this thoughts a will current We III in readout findings on successful its data be potential that make our can us to create new will growth continue confident that why unique study for

basis initiated of share claims successful were PGR that Marinus challenge been challenge issued the that USPTO. PTAB I Marinus claims have unpatentable. a ganaxolone the for XXXX, of its challenging with all that Ovid March on the were patent SC patent should in note, was not including invalid am to PGR added one the pleased 'XXX Finally, and in by the IV

that, Lisa now Christina be Vice Lejuwaan, who the our call update I'll With Shafer. Officer, over the President, our will to Commercial Senior commercial covering turn for Chief

Lisa Lejuwaan

and Today, activities and into years Scott. will commercial have potential for the progress morning, its Lejuwaan, to epilepsy Marinus. ZTALMY significant everyone. provide Good be you, made Thank genetic the expansion since an update I'm TSC. President Senior Vice we Lisa grow ago CDD of at Unit highlighting planning rare X brand Leader our I'll in launch on Business

on of patients to With ZTALMY active continue CDKLX after years growth approximately deficiency steady XXX we see of therapy, launch. and X in adoption disorder

product achieve year generated we of XX% growth comprehensive guidance between our vital revenue associated representing compared of full of $X quarter, million. we're CDD. over of second the on million, with role the projected million same to track believe ZTALMY growth the and highlights We the For XXXX XXXX. features to This period net management $XX in $XX revenue and of

believe, adoption a prescribing continue cautious way often patient treatments broader growing and these in is We a physicians treating about starting we and ZTALMY difference to comfort increasingly younger ZTALMY. for think the with they in signifying making HCPs hear more where We population a a patients. patients new that about changing parents patients' see lives ZTALMY from are

CDD patient in pleased with III of experience, therapy Phase We rates patients our are matches and highlighted the aligned our by greater very remaining from with active as trial Marigold launch. than with discontinuation This expectations. experience the low on XX% since our is

Notably, across commercial underscoring ZTALMY CDD see no have terms In continue been epilepsy. and CDD to brings both families reimbursement value of by acceptance patients to reflecting affected coverage coverage, we the patients refractory programs government widespread dynamic. to and date, favorable to no that

infrastructure is and community we've Over built to our and the HCPs, centers our that specialized rare settings, continued both We've KOLs approach and enrollments continues disease new efficient, resulting driving past is demand. in sales strong of X prescribers and in team that years, mutation working. and treatment growth reach lean an proven

brand maximize CDD the we're grow its evolve in and to impact several We implementing ZTALMY continue market, key initiatives. and the

By analysis analytics, paints ICD-XX and have with CDD with and First, in a patients who may yet build diagnosis. not have claims more do identified. the sources code data than identified third-party new robust we have patients data not have using but now we X,XXX CDD CDD,

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We continue over most with at conference, in CDKLX families International engaging for where recently the XXX Foundation annual their attendance. Research, were

planning the with potential the success are We're proud and and adoption to of fourth a of the CDD replicate ZTALMY in TSC of of growing launch quarter XXXX.

CDD from refractory we to of the also meet to of who ahead, suffering our needs proven the seizures. plan TSC foundation infrastructure build patients Looking upon are

potential With we approximately to growth population a the unlock X CDD, to patient significant to addressable have Xx the compared opportunity.

patients there remains our and a that unmet a the new and would welcomed. clear from in market alliance significant be it TSC need colleagues these Our makes that therapy research

has Furthermore, quality product with accessible applications, the and be life. when excited effective for to pulled, of meaningfully the it's used and improve an that safe other physicians potential anti-friction can about potential concomitantly easily are

launched unmet quick relatively When in medical CDD, population. we due this in the need to we had significant uptake patient

quicker for even uptake TSC reasons. anticipate we several with However,

often finding hurdle. the disease largest ultrarare patient an First, is

is confidence TSC through in first population neurological of inhibitors global prior identify HCPs needs physical underscoring III includes and rhabdomyomas much families and the community. in the However, and medications giving combining be through TrustTSC cardiac such and will this to trial that TSC Epidiolex code executed tubers well-known as and current and concomitant further the patient refractory seizures. ICD-XX identifiers usage mTOR and in easier Phase a

expect action other easier perspective, across expected From GABAergic place ZTALMY TSC In in the of of patients, activation the making of treatment the TSC. and control payer The changing and of and toy. ZTALMY from and differentiated broad CDD priority, unique to by Patient mechanism be supports is refractory ASMs algorithm. and is seizure straightforward. majority epilepsy the is we class from to the label medication adding access payers, main or supported protected a rapid extension

TSC will Additionally, in been time this randomized of physicians brand. years the the be completed global by confidence trial giving over have a X the will on market of even ZTALMY, which launch, the second for greater

is which uptake and replicate TSC, can opportunity. reinforces ZTALMY in a continued and larger we CDD market The significantly accelerate that growth of success the confidence our in

Our that highlight progress team commercial our further and looks plans we and focused our the on to Analyst at our TSC team September, work Day sharing in updates will forward Investor where the scenes insights has services patient been key This to prepare portfolio product doing from value expansion for key and behind study, our demand enhancements. include into potential positioning proposition, will TSC.

the Medical now like would our on I an for Dr. programs. Hulihan, update turn call Officer, Chief over our Joe clinical to to

Joe Hulihan

overview to our a Thank readout that genetic Phase our programs steps TSC, III an and next Phase you, share our recap for trial in the RAISE other Lisa. program. preparation upcoming includes progress data and Good pipeline III for which morning. of I'm readout pleased rare epilepsy of

TSC most skin, complex. caused cognitive behavioral of a or symptoms with sclerosis the or the Common other trial kidney is mutation genetic abnormalities manifestations. oral of lung among and the one in I'll ganaxolone impairment, difficulties TrustTSC start genes. TSCX by is tuberous common defect or seizures, TSCX of epilepsies include in

resistant Despite in XX% difficult need significant of XX% antiseizure the getting and TSC Epilepsy the of is unmet remain seizures treatments to year control. manage life. occurs Seizures TSC treatment. and with to are disease-specific introduction approximately seizure patients first for medications, typically there and effective of a to in still often to provide

with oral in refractory we're patients the TrustTSC need, evaluating seizures trial. unmet this in ganaxolone TSC address To

enrolled to line fourth continue quarter. to results a As with expect of randomized the the TSC placebo-controlled global Scott in associated patients TrustTSC seizures. adjunctive of in for enrollment which the trial double-blind, half completed report study and is mentioned, Phase first III ganaxolone we XXX top May

percent titration seizure difference reminder, to consists double-blind ganaxolone change frequency. the continuing reductions phase with a the the endpoint primary being open-label extension, XX-day of power Then seizure a to trial in open-label a by from detect study a there's XX% medications X-week between period, XX% patients double-blind to for ganaxolone. cross followed a The baseline placebo. X-week in the As trial XX-week and provides

to the patient or patients the relatively of and or clinical the by the responder including endpoints double-blind rate the trial reported additions their range secondary first allow study change mTOR the global impression medication treating percent seizure in the enrollment portion of XX% of is double-blind period, The during TSC scale Afinitor, and inhibitors, the management. are study maintenance frequency to TSC in caregiver. of in This newer the Epidiolex taking used clinician

in patients these XX% medications XX% rate, As taking Marinus a II respectively. a and TSC reminder, responder trial Phase have

approximately As reported experience patients study have anti-seizure of approximately in inventory current experience having having with or TrustTSC May, with XX% the medications mean failed Epidiolex entering with prior and a X.X inhibitors. XX%

XX median seizures Patients TSC-associated rate a had days. for baseline of XX

study titrated the the on associated decrease results, with been of based some this an III As a we discussed When Phase too increased to had reviewed II study, believe quickly, unexpectedly in we that the we rate discontinuation efficacy modified titration TSC Phase led XX%. Phase our schedule. previously, approximately leading ganaxolone information from to II

With schedule, in is the Marigold III the in below CDD rate revised study. the discontinuation titration Phase which the we what saw consistent study with is X%,

X date to to study due only patients the discontinued events. adverse TrustTSC Importantly, from have somnolence-related

suggesting complete benefiting seeing tolerating we over the from only drug likely into XX% study extension, III overall. but of not well the addition, treatment patients transitioning are also Phase patients who the are In open-label are that

medications, for of targeting demographics details forward rates doses. a analyst updated We're of of request XXXX supplemental to a look and submission April patient review. with on We September and sharing average seizure and priority including baseline investor in types, information use patient concomitant characteristics on event, MD&A our discontinuation

prior the goes data QX of goal a the a following in commercial with in in of line at we anticipate plan, early present results major fourth our launch. December, all in XXXX. Assuming the AES to the publication the according to PDUFA journal top date of plan announcement quarter We and to

broad ZTALMY range It the oral ganaxolone In which LGS refractory addition to delay the presence explore underway other developmental U.S., XX,XXX of trial to potential Planning expand epilepsies. to syndrome. Lennox-Gastaut the is of in we by its than TSC, plan approximately assess epileptic in a investment of is our clinical and encephalopathies, Xx and a would rare is in refractory the affects of developmental seizures. characterized patients for TSC treatment including population. larger for that treatment the

unmet We of be in approved of a top XXXX the data. half anti-seizure seizures ganaxolone Despite for addition valuable We there remains medications for given plan the to mechanism availability that first pending of options of overall, TSC several and unique need for DEEs in treatment. effective could the its proof-of-concept LGS, the considerable a a line action. treatment initiate trial LGS ZTALMY the to believe treatment with

design can help future The us and planned in trial a conduct the III proof-of-concept Phase study. guide of

to efforts our continuing product. we're second-generation Additionally, develop a ganaxolone

expected the to end of parameters PK are which for are studies prodrug, for initiated completed to efficacy, be optimize dosing XXXX. goals and IND-enabling Our frequency. ganaxolone We've tolerability by

trial approach. that III IV population. status our our ganaxolone steps refractory briefly share highly showed the cessation patient I'll that produced the Phase the in believe of in data of Lastly, RAISE next evidence of also We and a we'll ganaxolone trial RAISE epilepticus, results refractory status in rapid and of totality cessation and clinical the durable regulatory of recap

co-primary patients to the with status value a XX% of receiving less to XX of hours, p to that association Unfortunately, is achieve rapid XX significance. endpoint epileptic lack minutes anesthesia statistical second result significant within compared XX% Specifically, of progression of failed than effect statistically placebo, was a onset within with ganaxolone X.XXXX. was IV having for

anesthesia use to treatment the IV of to placebo. data by data specifically reduction demonstrated and analysis driven unrelated likely severity. of median seizure monitoring control was patients factors in clinical in for XX of absence needed status, durable of that XX% showed hours anesthesia of continuous the compared the EEG status burden contrast, EEG for continuous and RAISE In of demonstrated XX% ganaxolone a analysis EEG Our objective IV

proud for data on that next study this complex We're of was our a meeting success trial in team's the hopeful our completing program. this IV This enrolling set epilepticus. need. the the for program to set, and RAISE tremendous a support hospital-based and the in the Based refractory status can area treatment status. trial results to placebo-controlled to conducted of our steps FDA month We're of in assessment request the RAISE of for submit a unmet the in data with forward moving determine plan first ganaxolone we discuss

treatment we've patients for that applications. ganaxolone XX mention under epilepticus IV IND over also emergency I'd for of status super-refractory provided

have ahead, plan in presentation Epilepsy trial at results to Neurocritical October a been and RAISE the Looking platform Care Society meeting from data at additional year's submit this Meeting. for Society Annual American accepted for the presentation we

effective our seizures suffering clinical passionately team to to research committed In patients closing, of and than epilepticus. is safe, status bringing the treatments consequences refractory innovative

like now financial the and for a update. Steven over COO, Pfanstiel, to to I'd CFO Steve our call turn

Steven Pfanstiel

in the RAISE half first Thanks, achieved savings to to combined Joe, in selling, approximately our $XX III of into actions administrative expenses everyone. as took is of XXXX. our by update of good efficiently to be XX% for the now now and leveraged cost the structure second aligns be financial year general morning, as XXXX, in and second we the cash million XXXX share able from plans and also can We further remain upon to the half In to epilepsy quarter projected franchise, XXXX. This second with our cost which results our quarter on be runway, of reduced indication $XX.X of April as the financial and a reduction well project a and pleased Cost of in the R&D result TSC. decrease I'm $XX impact second expected significant the ongoing an of which of several quarter provide near-term XXXX. with were trial initiated this expansion extend to quarter. into focus full new oral between the million million third Phase

of with June Sagard our In agreements and this restructured Healthcare. Capital we both Oaktree credit year,

has minimum of payments from principal been and a XX%. with payment result, Oaktree both made million our to $XX the principal in have reducing to XXXX Marinus due million been reduced outstanding by to return, requirement a Oaktree million. liquidity amortization agreements In $XX $XX As onetime removed Oaktree,

result actions, XXXX extending our of ended these equivalents of second of cash and a June As into cash $XX.X we XXXX. million, the with runway cash quarter projected

As mentioned the in by to investments and support remain expansion capacity important potential a made launch ZTALMY Scott preparation and committed expand manufacturing TSC. in franchise of growing have Lisa, the our of we to U.S. and to outside

from we of to proud progress while have are We markets the opportunities meaningful revenue scale patient expand also the outside a on global made creating access U.S.

to remain to noncash XXXX ZTALMY SG&A year product and million million and in activities. end related upon approximately of the We regulatory see from from of expect guidance our of commercial Full expense remains $XX and TSC the approximately the milestone positive CDD receive year unchanged commercialization the expense $XX certain partners program $XXX with Access compensation eligible payments combined our $XXX before achievement Managed $XX between million, and of projected range million. including returns and to stock-based and revenues net several R&D million

ZTALMY of summarize quarter quarterly take of second the second results the and to million XX, XXXX. to XX% now $X as and This million for robust $XX.X financial prior our the compared of $X.X X product period recognized I'll of ended represents XXXX, and for million growth the June quarter. same million year. $X.X over We revenues for minutes few X months a the

X X Separately, $X.X million XX, the year. months compared we revenues million and for recognized the million the in $X.X of million and ended June and $X.X $X.X period prior XXXX, same as BARDA for to

the the of XXXX. XXXX API the period start-up quarter associated reminder, a onshoring activity the funding fourth initiative As base and first with was in of completed included

$XX.X XX, Research expenses months $XX.X X with in the as onshoring to efforts. million The million compared for and the to was associated XXXX, change and period due development X million for ended our year. $XX.X $XX API same prior June were decreased the year-to-date and million costs and

expense. ended were change for to commercial $XX.X prior Selling, expenses drivers primary general year. XXXX, and period million basis same on were and the June XX, X the The for million and X months the compared of increased the $XX.X million administrative in a as personnel and million year-to-date $XX.X $XX.X and

costs income were for termination decrease severance June equivalents months to X year. the was onetime assets. ended months overall million trial-related XXXX, the XX, June was the and $X.X Restructuring $X.X XX, X million $X.X million of interest these million lease reflect for costs cash, $X.X period in the investments. the expenses for million same and Interest prior and decrease and XXXX, income ended $X in as in short-term driven compared The by cash the

compared ended the and to the year. as million period XXXX, in the by XX, in $X.X was primarily compared totals $X.X associated expense net These June taxes $X.X same prepayment before The expense net Interest year, $XX.X million million as $XX of and million $XX.X with X the the the same for a for periods is $X.X million Oaktree. taxes months the XXXX, for to X before reported of the X for million and million million million XX, in the prior million $X.X loss and periods expense million $XX.X driven income and of as XX, X ended $XX.X compensation million company X loss noncash income interest months prior June stock-based XXXX, in X and to months and to June and and ended same for compared include million the $XX.X $X year. prior $X.X

June X XX, in in $XX.X operating of million the $XX.X Cash activities operating increased XXXX, to months in to cash compared as prior used activities million the used for year. ended

will I concluding make a Before the we to Q&A, few move remarks.

and on delivering to years trials. the development continued with for belief product suffering with RAISE about the development market, CDD achieve patients can Phase first and specifically committed epilepsy. proud make of driving ganaxolone ganaxolone what able through to remain we've TrustTSC strong in to the our III are past patients refractory we these X effect of We a We of from further positive been

will will Scott on morning be forward hosting an many as oral details Investor will Finally, webcasted Friday, franchise. TSC Analyst person broader follow focus epilepsy on that The our mentioned, September in and will attendance. More coming We of look weeks. and the we event be Day and the in XX. event of you in

Marinus. Thanks to again call interest continued the for open questions. your in may now Operator, you

Operator

[Operator of comes the Duncan Instructions] first And Cantor from with our question today line Charles Fitzgerald.

Charles Duncan

have Yes. pipeline in ZTALMY And on you the I'll with specifically, wanted congrats commercial ask I quarter. to and but progress did about one. question, stick I the a

quarter, you additions In color on about little Lisa little give us could a of that? provide Could the terms that perhaps you the XX% patients launch. bit in And persistence her drug could of active new a sense then remaining if add drug? patients little a or better sense some on more clarify on of around since statement bit patients have patients of bit

Scott Braunstein

I'll I'll off, to then it it and Thanks, Lisa. Charles. over kick turn

I quarter-to-quarter. seizure really on or therapy, patient I almost the relatively steady a population from seen that the things think new one But different paramount to than new this patients' patient this is every some in very we adds think always TSC quarter. started launch the a variation population think in a has get-go is is understand a with issue is one to that we've those often, lot growth of control months we get that plate lives. be on their going very really it's There's

time generally of think very I in there clinical And so patients the small franchise our launch. a to beginning launch. consistent growth of since was the the the with we certainly, from been bolus And have of trials

what terms back saw lost with but the we've to in of good commercial number effect the better one really been follow-up, a of about go our of patients in or X really of Marigold. we that on rates, persistence seen to Marigold, mirrors has saw discontinuation to X the XX% experiences long-term therapy, than nicer And if drug. the you experience incrementally years In date, we we

seeing comments at then still north north the on very Lisa's XX% So of Analyst in at population and Lisa, for it the of you're terms data to on some in the new you marketplace, to happy durable I'm durability both But AES. patient show in effect. starts We're additional persistence. put to we're also some TSC comments going Day persistence what and over

Lisa Lejuwaan

Thanks Scott. so much,

of market's think of it we're a there, the covered patients proud you yet significant to I done just add I'll has reach find. many patients team work extremely that most of the but portion to the CDD with more

it of fact happy a also with any, remained drug profile the complicated less is getting of of proud proud reduction mix these if interaction that relatively response. therapies, the community few, -- Our wonderful have the team And you multiple of very and this we're seizure patients, is side they discontinuation about very that these a signifying and drug-drug with on that XX% need has talk of such this rate, than that action patients a to XX% it's are drug. durability when this speaks ZTALMY the of this the unique they effects, mechanism

Scott Braunstein

me let Charles, comment.

the The second which first meetings some are and the of half paucity critical the meetings tends other of to for stronger really There's the lot AES in of year a of half the A certainly pediatric additional medical back-end really weighted, are us. to year. really messaging. be medical meetings,

for second meetings medical almost really looking meetings immediately ZTALMY. to half medical the year see we're following we of forward and and the So the that the interest

Charles Duncan

the office? countervailing. prescribing? are the take drives that Or does What out So it of prescribers Yes.

Scott Braunstein

prescription trends Generally, we've following seen very our meetings. strong medical

Operator

is of line the from Capital Markets. with Our next Abrahams RBC Brian question

Unknown Analyst

done study, their III and On Phase reduction can is analysis about extension gives you the for talk Phase end how read the the into therapy II the many until that you've confidence had for some that the for most How study Phase patients so the far? stayed for guess TSC, who consistent open entered study III can John maintenance seizure those you visit and This of of open-label was I ongoing you the label positively. last Brian. will subgroup tell us phase, on out the

Scott Braunstein

Yes. Well, September. patient to are visit over let the saw But II. now Phase about talk north we well to we patients to turn in me The I'll are it little open question, start last in with and XXX label. is what patients last close of that the then bit the into a XXX Joe

patient The the first or visit second study. week the in in September, last should we have

majority vast patients into of the label. rolled have open So now

And -- those think to XX% of label consistency open patients open who continue and label. high of study to your of to see completing into north rolling I question, the we We're patients open staying rolling are on label. into percentages of the

give in now numbers on we a well, discontinuations the what Analyst than we'll are at the trial. of We So in Phase very confident tolerating extremely different seen haven't in north early saw II. from certainly the -- Day, we but final patients drug the that bump feel very the really XXX

in As we a Phase II. had a reminder, discontinuation the rate XX%

saw to comps dosing done to of think much paradigm. highlights III. team and today Joe Phase Phase the we're the about the Joe, done has want I talk we reconfigure And some place our II in because you of work that has the into a different that when going in

Joe Hulihan

effects, X it patients -- as there Yes, Scott for Yes, side that Scott. mentioned, for was discontinuations as XX% were was Yes. so you mentioned X. side effects,

rate patients So we II, to adverse really, titration had due discontinued in Phase X the events. despite

the the about II open-label Now study the open patients from the that's one thing study roll Phase could into ongoing, current label.

patients still II a the but the Phase number we patients III the tongue, we extension not have have combined latest And so from we point have continued to the II. is open-label for my the of this Phase extension. at open-label tip have And and who Phase in

a we so And seizure the as to I greater I and a good you overall big difference. I had everolimus, makes and the mean about XX% at I response, in also we They patients types. titration at particularly compared everolimus XX% think whole. a cannabidiol looked think a the reduction really population then looked mean with response they And focal patients on better subpopulations. response, had asked population.

I Did So question? your signals. again, encouraging answer

Unknown Analyst

. Yes.

Scott Braunstein

Yes.

And X Let about what the in said reminder, of close a in terms we're saw to the blinded Phase types, what in we III the me comments XX% mark seizure add to XX% focal hit seeing that Joe publicly II. the X in nature. or Phase as are we of

So that's hoped see. exactly we what had to

robust strong Phase II, be issues, expect we in why which to even the So Phase signal point, that number to is III. the with Joe's more in tolerability

group, on And Phase are we And again, make into And mTOR XX% that than the responder XX% the with you'll inhibitors. from more Phase in study up population. our the III, cannabidiol saw responder number the patients number which half was the II. robust of the strongest coming a certainly, about

subtypes have the the in we right all Phase So III.

the right seizure were real that's of issues where the setting even in Phase some We have efficacy the tolerability signals types. And II. the certainly, in strong

question. the Phase the lot So going Thanks for of III. we have confidence a into

Operator

TD from is question Cowen. of with Joseph next the Our Thome line

Joseph Thome

frequency you to potentially that Maybe a a little ganaxolone on the your sort Or of of little and your more bit of question, little be Phase Phase data also the Phase is to in versus think experience from looks seizure II. Phase what's a there? it's than in these higher your Phase based the little of kind challenging patients a bit the going saw I to indicate Phase last a benefit therapies II, III? bit treat, are expectation a easier the and CDD the guess number that show like -- to in failed does the is the explanation prior in but II the higher baseline III III what of you blinded bit follow-on

Scott Braunstein

Yes.

or a I worsening have off There symptoms, investigators, their Let that over up state, who, which patients then like better right? yourself seizing critical in of tell and what's failure. set their at to experience, this through at will for would it I'll me medications to talking Joe. acute kick from turn acceleration lack we of it isn't study tend key our to a some of my a are an is least you for really steady

of Just progressing, therapy whether not or any can address the acutely is that. in disease kind

more minimizing really factor that that's at to for give the think stuck seizures enrollment the all and critical world criteria per Joe in I kept credit our guns placebo than I our rate. I So a And think he X month.

a X You only there's natural with and can if patient enroll the was imagine month, variability, that if X. to to XX% per allowed could be seizures placebo from response they X went

month, multiple driving do our studies. you than is minimize really higher us see again, keeping But rate team seizures success. which in and view what placebo we was Joe maximize at seizure is how to So in the per instrumental X

is us. who Look, older, a be surgery, medications. bit controlling multiple think gone little talking to through from have gone experience investigators that our we're for placebo to getting of my of have through a many I many patients going group is critical So for

this placebo it's think will a high showing, is market this effect. think again, phenotype as in population. burden least discussion, I classic really that's the very at and I just Certainly, to minimize research seizure

experience go enroller that GW about the that the So the feel CDKLX, second We XX%. very to in yes, placebo about we good study, seizure a very countries really or where baseline we a I'll went if we rates, but rate. of We the when high you that was not enrolled ask this folks -- very good rate Poland Poland had about feel type, largest remind was think only study. I unfortunate back was in. in efficacy. because with placebo you placebo

Certainly, the high suggested placebo GW rate. a study

risk out stay placebo. So European the Eastern to minimize we high of countries chose of to

than somewhere placebo So between no X digits, to which best more about that we good very win. us rate and chance single the high seizures in the digits, month, the coming low in the gives feel per double

to types the in on Joe, seen past that of or comment anything the you you want give blinder seizure data additionally else in comfort? that we've in seizures terms

Joe Hulihan

lot in seizures, into we still see things. the saw, seizures, expect to Phase where I'm the in variability The less III I mean, with of One, say, [indiscernible] than the the a response size. good atonic or and efficacy about good vast patients And have with the Yes. talking CDD. mentioned focal effect Well, seizure tonic onset seizures coming majority we patients types. of you a

the in -- effect in, with patients on is Scott very Still a focal less eliminating size, coming really not tag that with to really the not the low of but patients just baseline, patients there's linear those seizure seizure with frequency, get. just to the it's of able seizures. problem bigger patients what said mathematical that And number variability said was But good better. numbers get kind very terms the to Scott it's really those there you about being more response It's with in at much few seizures.

just is a X baseline beyond get seizures response XX higher seizure depending you once doesn't differ but month, patients. eliminating low the very on or But much that better, the rate, really

mention, is a came The coming of things. make thing other couple regression didn't study. the I'll One gives had they too, that we to disease patients so sure the mean want stability when have in, well, -- to the into they

the than the those into and at is So when X want them months have seizures free come frequency in disease week to certain more make stable months we of sure no X seizure baseline. a during the to they each

The is due lower lower somnolence And thing, saw the this somnolence. odd discontinuation relationship efficacy. too, titration We and higher other the somnolence. lower rate -- and to between the somnolence, the

somnolence, And me the to Phase in that reducing see about so going III. by gives also optimism the efficacy we're

Operator

Lee from Truist. is Our the next Joon question with of line

Asim Rana

This is Asim on for Joon.

somnolence on TrustTSC the to trial? Type Phase could on think And II the timing the FDA that And what be what what I out call in meeting? do is the was the scenarios trial? what rate curious come this meeting Phase you the want the was know then mentioned of C what but III due sure to you was discontinuation number Just and in

Scott Braunstein

Thanks, Joon.

we've from the issues. the the II XX% backwards, start tolerability them required certainly, related dosing related rate those II Phase are of the all is XX% in only discontinuation piece more patients puzzle issues. We believe to adjustments. tolerability than of that total to And of that other Just Phase the reported of to of were

dosing, completed. until trial the So was it they are were and of struggling until didn't was who with become effectively something patients very a really the we aware really

really be was medical get Day in One at what fashion from of will team a blinded the Day, share Joon, on our in III. dosing physicians he's from personal Phase I the Phase first to position forgot II think I'm struggled our quite Phase at really Analyst Analyst patients took a your question. commencing the regimen apologize. and who lot response myself participate you about good And least in I thus this a with had because he'll our and the III Phase he of that with II the I the far. excited to at

Asim Rana

the I timing of was FDA just of the curious Type meeting you come could about C and the with that. out generics of

Scott Braunstein

Yes. Absolutely.

a And no team regulatory the are durable and Alex, incredibly -- comments hard that going Joe's that putting and through was been to set really quite the efficacious question highly convinced Joe, had data. data So has drug working we there's understanding effect. IV the

experts and I is It reflected think drug. really unfortunately, what the the going the to. again unfortunate. the to that's study and durability what counterintuitive not medical and time practice, we endpoint that more by the great and variability did That's the told physician that talk really we really into of chose had believe time we reflect deal of community were a we

critical a going is robust for the objective certainly, hope So what the our on that we have discussion. really can more are endpoints the FDA data product. define very the durability really to is our we thinking meeting realign and a of will think the the goal with be we FDA about study we're That real that to much and piece design. share set with And

goal of in the month. And by and meeting review. XX-day hope is to get a end Type the Our typical filing the a is C

physician, how is think that the as patterns the us understand I we've show but this I in. data to this is early data a efficacy practice an me we're study it's in has looking ICU believe really gave quarter. And after I are part just set, the job set, physicians -- come where looking drug. the for mean amazing patterns. in fourth rethink of a EEG nothing which to And certainly, there have at helping distorting It's to the this our we us best lot physicians new study an efficacy learning unfortunately, drug. meeting at practice think pause of done of seen that So XX the hope hours, just of Joe

I think of drug. terms FDA with we make the align value the will can in and We no align to want of patient population. that significant we with thing this stratification is sure outcomes, variability show differences really and enrolling on in strategy the really are should other to about a that imbalances some we how there the think saw the FDA prop

a of note we'll data meeting in presenting presentation. last I'll it at NCS just be the this vast will the medical be majority platform is a meeting, October, and

question. FDA So folks of in as will all drug. of get and to seen Thanks terms we've we're of really the tolerability share well. the And the what the to all that of with going for see efficacy the certainly,

Operator

is Jefferies. Tsai of question line next Andrew Our from with the

Lin Tsai

it meeting, but on have And succeed say, years? done, also FDA not after within of run only the would tidbits, if study the you X anything to sharing ensure. to so Is top to you can there for it -- had of design X think you how another all thanks those to

Scott Braunstein

the for Yes. question. Well, thanks foremost, I think, and first Andrew,

I very about thoughtful the design. think we're to trial going be very,

protocol we had and and great the issues stability of some once resolve amendment able life were manufacturing product. our recall, our shelf we we we issues, that have think got to I and through product now

those got those study. study patient majority for eligible per is what of population in a the future once we would screening through and patients about quite be So we right think honestly, patients at we to the all vast XX were issues, month,

a worry tremendous don't amount I the enrollment. So about

are which know very powerful. think sites we I

in think study. eager another to involved I they're be

started that think, I to strategics where to have engaging to we talking the we think, with conversations But still BARDA. we and impact company outcome. about are our really fortunately, curious, our resources. in the want of multiple be to FDA think those that for regularly we about the vast data room, the We're majority strategics place us still really have the of I honest, data, in to financial But

unfortunate that under and never BARDA let's United they in unequivocally ganaxolone would have attacks within product this there so access that. the to available wants commercially hope the States, gas for case, is,

the ways really And another to about we of are with another hard finance party. trial help thinking so

from certainly, But And with within plus the is again, roughly right about we're I of doable million thinking window. agency, the a think now the the trial, which getting XXX quite in think range minus alignment think thinking size really I program. or we X-year be patient I a -- We really almost for program the going to that it but oral a also recognize behind resource want allocation resources standpoint, financial put all IV our we're future the have to to has we to see a smart investment.

meeting FDA. with So the how we'll the see goes

the we'll to certainly we we're to run really then these an some feel mind. way of figure out and think very time, a of the elements. can optimistic best finance reasonable with in we get I We shareholder that study interest key alignment amount in study on can

forward. away path to be with So this of relatively FDA that's where our think a clear with interaction walk able kind I And the from we'll we stand. with

solution we simple. think relatively think is I the

the think has partner. agency great a I thought been

forward looking we're to So with them to drug meeting market. this and getting

show to that's I recognize have we the critically to think have. plan. the resource But that And to environment, we a hospital able physicians it's for important it's our one be value.

will you So those with be to the months. that's share plans for the we're and in Appreciate coming really it. we forward, question. excited Thanks to how going move

Operator

Douglas of Wainwright. the Our next H.C. is Tsao H.C. with from question line

Douglas Tsao

especially the expectations it terms of sites at of that study? the had a in I'm of and seen your to IV of that program, phenomenon Scott, really behavior. individual broader and what just IV or sort curious, expectation physician site? a would in of treatment analysis advancing deviated understanding patients was results chance to sites challenges we've in there Were the in of have terms particular you anesthesia look obviously, patterns by just saw of by be terms -- from really sort your you Just

Scott Braunstein

drug sites. And analysis Yes, those we to think, And certainly, IV an anesthesia. more our sites high We're advanced quite physicians driven at there afraid much did not or honestly, moving that was factor I how that in stronger did. signal the for a sites of concerned commonly. larger was the enrolling our in bigger our care about by look as

certainly, placebo as overall study. So the a lower delta larger result, was rates there were than the and

think necessarily. a bit to that quite not matters fair, I be but

not would feel enrolling study the we IV to is where another advancement endpoint. think comfortable anesthesia I

the to physicians is with ganaxolone which told X failing at after learned who we IV what think drugs, time of us, regardless -- patients XX% what in X.X advancing we're would And move in received even the was they received for the of anesthesia I XX% that the were typically about or at in study, physicians case, drugs. group, X days. only scenario time. control this out and status in that care Typically,

and what patterns. necessarily this been I that we've patterns. and is time subjective -- EEG but may us So may our end -- survey. sites. very again. following It specifically practice did that all their and And regardless We to the EEG, big point remember, been too that we have time of We learned physician after a change behaviors have changing been think have went have the in of physicians just not told may physicians

variation more sites. that in I pattern much sites X a we in and our or would treatment And X enrolled say, those yes, enrolling a consistent bigger I think, And saw patterns patients.

certainly you have That are risk enrolling you bias. but run influencing when X nature, of sites much I here, do study mean site the their outcome that spend you the of a by That Joe, site have behavior add? is don't think I I X patients, you you or and data. a factor played so anything to the biggest factor. time study. the think want

Joe Hulihan

Yes, at no, It site to every we're study. the were another you mentioned, yes. But sites at some patients variability each not doing Scott. or was particular lot and as site. think to a think No, there don't I of kind do minimize I

patient in mentioned We've sure I stratification. representative get we a in to population, We at that to what a look endpoints population from study. respond of want make think obviously, and that's selection Scott wanting lot to the terms we understand a another and RAISE learned a lot. study,

So incredible off RAISE now from we I we've and an pay when to amount do think study. time come learned another that

Scott Braunstein

to with to we the big by the the FDA Joe talked that drugs who site SAB this the many there as I right the signals I and FDA several and placebo. saw differences differentiated between with has in is at that being logical that think, than think, for pushing Does powerful thinking clearly EEGs variability. have shared about of ganaxolone durability and set their them FDA. would even it's with alignment very care to can how I is I Doug, demanded received the agency. measures think really our nice this more very with of durability certainly right? what objective, but the And IB talk those discussing FDA, Certainly, with we we EEGs I that data that thing escalation avoidance about durability So We that have every agree they want share I think key have Unfortunately, with piece discussion approval is discuss endpoint. and what the our recognize expert we agency And patients of of NCS meeting. think other really meeting, durability feel really defined imagine the And with we specifically feel won't of anesthesia. a placebo. strong We'll reality I agreed data at think I is clear we need in never with you think what about puzzle, And But one, study. be we the we unequivocally it's the a upon of measures have should support. very every this to. really of we've think per member, and the from the we other se the

Douglas Tsao

can question If Okay. on one I quick TSC.

really tolerate some continuation you are amount able are discontinuations of time? long-term the the drug terms any to patients so the in the long-term once for of And extension sort far? In rate of seeing extension,

Scott Braunstein

It presentation. that that expect our data of investigators of to extension you Day are a submitted be we top that with a very been accepted line going it in yet, fortunate one we data share at AES. hasn't so our Analyst all right? Well, little early, presenting for We'll is It's long-term as but September.

not activity And we encouraged we We time the states across the is extrasynaptic believe are time something ganaxolone pan it's the we durability only sandbox about that rates, in durability, and research, robust talked to when quite that play other and about, nicely of disease really response it's very about Lisa do about and really drugs. and unique. initial is again, our see safety, with it's market as really ability

of we there. of a see you some seen and when seizure I points, think that of And a I comparing will right, but it lot drugs, and most tackled refractory wouldn't I are to really lot TSC measures think, certainly, apples states we X in, So into the think with it little a bit other have just this that you go oranges disease see in disorders that in of we -- we've we talking particular, expect launch, of durability. out what

an So that at important we Doug. we're the sharing Thanks, to message, think going to looking all be with Analyst Day. you data guys and it's forward

Operator

Marc the Leerink. line next from with Goodman of Our question is

Marc Goodman

secondly, year? you with for little any about seen talk milestones. And bit eligibility use you a this you've Can Can elaborate off-label you mentioned for ganaxolone?

Scott Braunstein

missed I question the on sorry, I'm Marc, ganaxolone. first

Marc Goodman

[indiscernible]

Scott Braunstein

want questions? Sorry, take a I'm do interference. little to those having Steve, you

Marc Goodman

use. Off-label

Steven Pfanstiel

jump Yes. Sure. in here. I can

answer I'll I think, first. maybe the -- you second Marc, one

You mentioned milestones.

from XX CDD. is upon So be commercialization of talked would EUR a that about the near-term million due milestone we've Orion

market. So a launch TSC markets later when with of XX first similar of months in their that the There's they or payment commercialization. major happens after type than no associated X

not Tenacia payments China nice also the to magnitude, And Those further well. amount a commercialization we little are quite same a out with then partnership. with as our and have but

off-label kind off-label, do on ganaxolone? terms you with got I use want touch of to Lisa. In Lisa, know of we've

Lisa Lejuwaan

by Absolutely. on Thank diagnosis, you, that payers just are need of CDD the a I'll most Steve. affected patients, add acknowledging do We've for unmet got that other our XX% significant DEEs. these not a patients therapy about here. and covering drug have they're

Scott Braunstein

I couldn't that think the a the and went hear in the the about -- a launch but drug. back add TSC, add have program I'll thing with into maybe little GW launch we've is, number I with lot they LGS, And GW members team a of I Marinus think heard we expanded overlap you, forth between substantial several their as their and TSC and on of part patients access team of already and

no today being in say that can terms developmental of think any TSC reimbursed know encephalopathy. are or to we currently with certainly ganaxolone confidently the that of of I we there we are do those patients on close not don't -- diagnosis

is market for not So of we day us on there's big business and really feel the like open entire our X, a part certainly TSC today.

Operator

with The next question is from Butler Jason the JMP. of line

Unknown Analyst

on Roy It's for Jason.

XXX have patients CDD, diagnosis. which one I over the diagnosis, mentioned do I for not just this, key maybe guess. answer and have what of I today patients but might quick a patients in, who a ZTALMY, a initiatives the Just and proportion guess but

Scott Braunstein

kick it then and Let Lisa. over I'll it to flip off, me

groups, Lulu than more the Foundation Just went advocacy remember, we effect and that IFCR little the very U.S. for a year into X in major fought code bit hard CDKLX a before launch.

X.X So years ago. roughly

in newborns To CDKLX. X, patient we to gone X,XXX right. in X they've perspective, XXX unique XXX XXX to a about codes roughly now the X.X years, put kind to And from And year. of that for last

genetically patients always and tested. those another adult be but certainly of is patients, talked won't as age below adult the XX we've about, patients So what we roughly of X,XXX expected, many X,XXX

being Certainly, ZTALMY. tested some are being some and with treated are

a line less of of have patients, sight sight think X,XXX we the good other So of of I line X,XXX. roughly lot

So think than that diagnosis symptoms I probably the with we been haven't there estimate more yet patients or X,XXX are with out diagnosed.

So boards code, the recognition clearly advocacy growth seeing new this the XXX pushing not just of exponential know a year, right? for in groups and use ICD-XX we're than it's more there's of code, we the physician the And the code all leading were not know, code, had our puzzle year as that also positions a of pieces But centers the those of around the to we of the excellence using reimbursement use appropriately. we some of code, code. even ago,

education think tried pretty XXX that that code fact find but already we're feel on the these finding I patients keep like So to around business. or are really close it's than bit patients a and there And therapy have good feel therapy that little the who certainly, good X,XXX about patients we prescribe is the are so out XXX don't there, feel that patients I the the additional we on more patients only of a code, think to have we that. to patients focused currently can or growing very really

more. can have you make over And want think XX this to last question, We've And to Lisa, minutes. we're gone about that the going Lisa, I add? operator. to you about talk anything else

Lisa Lejuwaan

an there patients the just add unidentified patients have is been towards thing approach the shift an utilizing code the a years, because X and in more increase the our that should XXX genetic testing really X past with done think in nicely. available so HCPs it support data-driven strategy we we been X we've out underlying disease. that increase only who you've that more it putting we've I for from There's and over covered are time identified. seen seizing the many are years, every genetically casas from ideology to an Society, in that test their general over American also physician data X,XXX Epilepsy I'll have

too. combination in really left patients that are has for in the diagnosis that to So and ZTALMY CDKLX the introduce there still out are driven general, we very us confident up

Operator

[indiscernible]

Charles Duncan

key that that And end with that follow I be TrustTSC to Yes. at guess the well simplifying out filed assumption you'll I'm relative make yes, works the approved 'XX. sNDA get there. going I the Follow-up, Scott. able to I assume is and on. that capacity of at the had question so to that's have up manufacturing you to is I question

Scott Braunstein

Charles. Sure,

us dedicate very ganaxolone. manufacturing good as shelf effectively shelf been hoping partner X-year expand of very branded has our resources We're price We've the and of who their We're growing to feel API shelf well. we the current to think our a about with our life life. ZTALMY. will much I helping that capabilities fortunate only

So to ZTALMY. have to manufacture continue we the really ability

And and launch. very for that certainly the off the we're feel internal internal preparing additional the supply these 'XX in and TSC But to expectations in we supply, in by need expectations TSC launches going kick 'XX, U.S., opportunity to our once of certainly, global good by our and start as the we by CDD during really I we're that throughout think Analyst very volumes share some very about dial world. is sell across equally market expecting Day. a expanding that of everyone numbers realistic, least the Europe, at those and the Xx I strong potentially in And think volumes as market believe large with China, strong really XXx we we volumes important,

risk. stage second make certainly, than getting And that want a sure to made prepared investment we and the XXXX, have shortly. enough The that doing critical equipment. think will unique more very process, have in for it's so and to in the stepwise start crop. And we're minimize we fashion and a And to manufacturing we sites we've build-out room

feel some for we're plans for good was let us hard in people decision place, we it fortunate And -- go. -- a that. look, are and about So to very those really preparing

In said the all. had at organization. the June, profitability. we've in all TSC shortcut we a along, think great as confidence a -- drive not restructure the franchise Officer. Christie's oral But is high Commercial it been always short-change We great And have she Chief or launch. we've And obviously, can

We've the is about global incredibly We're call You Lisa We're the thinking in place. regulatory this well. that got from about leadership as thinking qualified can manufacturing. great hear strategy.

We this ready are going to to be data. quite execute post

the Charles, call. for much really so it. appreciate thanks So

you seeing at Thanks, our the some analyst we dialing but September. look thoughts, call. sharing and to XX in We're forward for again in. thanks really about you great minutes over, And appreciate everyone. event joining the I of

Operator

This today's conference. will conclude

You time. day. disconnect Thank this at for have lines may and your your participation, a wonderful you

Marinus Pharmaceuticals (MRNS) 13 Aug 242024 Q2 Earnings call transcript