Catalyst Pharmaceuticals (CPRX) 8 Aug 192019 Q2 Earnings call transcript

Greetings and welcome to the Catalyst Pharmaceuticals Second Quarter 2019 Results Conference Call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded.

It is now my pleasure to introduce your host Ali Grande, Chief Financial Officer. you. Thank

begin. may You

Chief and and we joining and have Steve Head Officer Officer; everyone, Chief Gary On Officer Brennan, Medical Chief conference Chief today’s Dr. and and Operating Chief call, Pat Regulatory Miller, Officer; thanks morning, Dr. today’s Ingenito, Affairs; for of call. Executive Scientific Dan Good McEnany, Officer. Chairman Commercial

about comments to expected and be for I to begin, These and that of you are estimates we will may remind make results, projections, statements expectations, would we future not like guarantees statements the performance. which forward-looking in purposes future the federal Before Q&A of relate session, following securities and current in statements our the laws.

to They results involve in including only information Annual XX-K. predict, factors accurate. to our vary. prove may in on detailed described filings, Form in risks, should Actual be not conjunction uncertainties may and be difficult contained forward-looking SEC are which statements These the considered Report risk with assumptions the our that

time, the call Pat. turn this I’ll over to At

for you, you everyone Good us on XXXX morning results and thank second call. Ali. financial our Thank quarter joining

probably press we to commercial afternoon, strong our issued yesterday continue part our for saw launch progress you in LEMS Firdapse on see of this release As quarter.

plan. as Our in solid rock commercial, medical our management the accounting of facets teams execution well team been launch have affairs, Firdapse and as our all patient financial services, of and

to through have patients’ meaningful that only enabled we Firdapse. patients providers a to to the received on to with education support support healthcare systems assess we not who and impact lives LEMS within LEMS well. the us support heartened I’m as treatment, to and that has The but community work from proud deliver by continue receive the are community access

of previously based to kind have the an continue evidenced use a caregivers patients from and by X,X-DAP feedback to who naïve received encouraging. Firdapse, from humbled and Also, now we that transitioned of LEMS were receive in with the word therapy. patients are compassionate especially now that Firdapse I’m program been have are treated adult FDA approved form previously who being any we to has

reported, medicine to the of Remember, to a patients FDA’s had investigational Firdapse. an previously only in few access prior U.S. approval the as hundred

ability monitor the has needs adult patient’s Our efforts and We treatment LEMS us. all patients continually surveys. the through meet motivational to of satisfaction from for been suffering

They and insurance ease measure the are getting how Catalyst pleased in and monthly as Pathways patients receive and well coverage programs with efficiency of Firdapse. their services, shipments as

July, with responses of in XXX X. to a from score of XX% Catalyst survey Further, X or and on of with a of patients X, a end had patients X the we’ve responded completed rated XX% our scale of programs X. a patients Firdapse Pathways As an of average X.X

Today, three we the the we’ve that priorities the previously business will status discuss for year. outlined of calendar

strong of the launch Firdapse for First, LEMS. commercial continued

the of Second, additional indications convenient our friendly Firdapse. more for a the acting, third, patient and potentially And of in reformulation robust pipeline. development formulation longer of Firdapse

that suit address before business FDA. matters, against But several like law the has Catalyst I’d other to that, filed including the

a alternate an announced LEMS for application the pediatric treatment formulation we of filed new for Pharmaceuticals, we of previously have law Jacobus drug challenge patients. As of to approval Ruzurgi, have amifampridine the suit in

that Act Drug our the Chemical orphan Exclusivities product, drug under other Orphan the have Jacobus Firdapse. and Among approving New the has the we granted to been FDA that by believe claims, violated

in resulting that in provisions of also the misbranding FDA labeling Cosmetic regulations, Food, violated We Ruzurgi Drug, and the of for Act. violation multiple believe

and complaint we Ruzurgi. occur. a you on We XX Additionally, filed in vacating our the complaint June developments apprised of we were order keep court will a as seeking approval

what in legal comments are the giving we these proceedings, we limited somewhat and can ongoing Beyond discuss.

that announced we we include the license during Japan. our have BioMarin quarter, Firdapse amended Also, to with second

Our original Asia, certain most the and of America. in amended been upon license South as license add Japan, Japan has as for well Central America achievement include it expanded milestones of to to and and can North was further be

the world for and to approved reports LEMS milestones subsequent economy free there On represents with in in to you earnings we therapies in efforts commercialize and largest are second the Japan. no call, provide Japan will Japan. population the currently Firdapse on treat timelines status our

We quarter or to press recorded $XX.X $X.XX like and yesterday our a afternoon share commercial release share. to diluted million that in Firdapse of of discussion profit operating LEMS. return million per an net for of $X.XX $XX the I’d Now, the and second per announced launch for we in revenue basic

end our transitioned to commercial last Firdapse have the of On to to so the the LEMS XXX that we both programs by participated most expected that second early therapy. a in be their quarter announced of lapse patients we access in earnings single or call, without

by that XXX second the quarter, Firdapse. those end essentially are on the of all We today patients are to of announce pleased

Now, expect enrollments This goal, to orphan more patient that the measured disease ultra-rare was trajectory and expected to of from have it’s transitioned launched product in the we new which initial majority at the the continue these patients was pace. space. typical Firdapse, of vast the been steady, start commercial a

it provide this believe revenue time not be to to that continue guidance appropriate for We XXXX. would at

continue halfway expected providing quarter’s be almost this third new shortly. through information additional a at are say Ali as revenues quarter, that and enrollments will financial results patient will on pace I steady robust. However, we the

Firdapse now through track on the of that I indicators like key the daily launch progress to some you walk monitored performance would commercial launch. to our were

As been have XXX on patients XXX and those patients physician; of Firdapse, prescribed that of LEMS written unique had were their XX, have one least prescribers by active prescription. XXX Firdapse at we June

fulfill XXX Pathways launch to proud have and that fact and who took adult Catalyst of Firdapse treated development in to need. This a approval being of that already as in premise to today of when is continues we not there LEMS that And the medical need form this XXXX I unmet are patients FDA amifampridine now most approved that indeed of an could enrolled with prior are Firdapse. was on any an am our did most access Firdapse that our for validate

to to with goals easy We discussions of our that of LEMS principal all, and and all payers ensure patients is one We have is reimbursement Firdapse. and that regarding collaborative adult have Firdapse. reiterate affordable continue capitalized access to that underscored

closely enrolled personal is our field caregiver that obtaining coverage patient and make or access through and patient for a that Pathways the the work end, as office, program assigned or can who each and the to is is in To their in with or and Firdapse, him with patient efficient physician liaison to experience PAL and the meet sure is that patient possible. Catalyst patients as guide her stress-free the

shortly Dan details the provide will progress regarding of additional commercial our launch.

on Type As the ongoing further remainder muscular a I a pipeline X focus spinal of in is have in trial beyond trials, Phase the Phase syndromes, or indications X. Firdapse second myasthenic X our CMS including trial of proof-of-concept study development of LEMS. and the mentioned, a with our several a MuSK-MG, atrophy expanding use priority congenital currently We in

programs, year. label our MuSK-MG first the of results from from report expect results LEMS to the have X of Type additional the trial this Further, from topline the enrollment would We in in we neuromuscular and the results Pending indications. CMS to second Firdapse trial the expect in MuSK-MG topline complete to next approved we positive half half year. these to SMA the look supplement include for trials and

you to pipeline robust excited we about are programs providing development of these forward progress. our and with clinical we look on each We updates as

a to Firdapse. on in patient information few is Steve provide priority formulation friendly Lastly, minutes. our these more longer you third develop a acting in will more programs of

we quarter I for incredibly thank to entire possible. productive As an you this have which team would Catalyst have had like for making our heard,

bring and of are employees for And us we patients to exist. helping evidence-based lives. that treat approved of each our to we are to to diseases. We therapies It patient important LEMS to is rare today the most is enjoy the grateful that neuromuscular why importantly, FDA know the productive most our work is efforts proud striving

I’ll Dan Dan? regarding more Chief now provide over call activities. details turn to commercial our who Brennan, will Commercial Officer, the our

and good Thank everyone. Pat you, morning

we mentioned, about thus are and far. we the progress enthusiastic have strong another had Pat As quarter launch had

sales, it’s a and the in the medical, services, and great all together teams Our across patient office patient team advocacy, field well effort. marketing working are

truly the main lift their and they disease. debilitating as Patients goal this how to possible require disease even to extreme this help of leg, retirement like mundane too stand blurred of encounter hard most experience patients arms, workforce often daily them Our or enjoyable and reach is as an adult with muscle, in arm, to bouts as is. live help it many up, find and or they emphasis I to would caregiver all and a vision, double them LEMS neck severe activities. the leave debilitating forcing navigate fatigue to the support

some is so and having worst identify people the better to Catalyst, and hospital At able good the determine progress. cause their affected for days by treat spend are their all and LEMS this, our of proper of to doctors it suffering In and that mission we we trying to speak illness. disease cases, the to in believe change patients making are the their LEMS diagnosis with

QX. the the our end of me with comparing Let share some results of of QX end

FDA the approval patients As on Firdapse. there mentioned, compares end of Pat the XXX launched to U.S. were in XX, following This XXX had the second end the QX. LEMS quarter, who at been prescribed unique of March

adult LEMS compared QX, the of at to We at announce of were end Firdapse end that are active to treatment on pleased XXX XXX patients QX. the

had Jacobus those rebates were and a phosphate. amifampridine expanded QX own compassionate program who in of access in amifampridine and our or patients use majority receiving patients QX A or previously investigational been

of delay who proud to so existing did beginning are Our in the we lapse focused delivery. medication this that and Firdapse on initial XX those or patients without a transitioning on launch treatment January and year those transition

lapse pharmacy to goal. late their proud Given rooms our important the AnovoRx, to to these was homes. XX patients and stories to without I’m on from efforts notice or from specialty very without away to mobility Firdapse delivery therefore to fled heroics medication There important and were us. patients emergency disease our numerous examples respond personnel emergency our this achieve of transition of field needs severity smooth a in to immediate impact medicine, and very for and very the XX-hour

and on adult to The investigational hear from about is but or LEMS, life awareness patients truly second February launch commercial response their drug. changing our on with medicine. diagnosed to any without access to of reports focused phase this gratifying began patient X It

from generated many to disease that and drug an in Our drug approved remark teams need know an promote of broad availability communicate awareness about physicians continue is adults, across to treats the and how unaware well or the having are field the LEMS so approved [indiscernible] and we nation.

X,X-DAP XX today to by as As X,X-DAP. to of to XXX end have renewed we patients, or mentioned, Pat helping who QX, this reported that there were naïve were the XXX we Firdapse of call, end X, and and the progress QX, of prescribed of We naïve to At and LEMS May there new grew are with prescriptions. been of of call last was naïve XX patients with there patients pleased XXX, group patients. at this unique this as group QX, as X,X-DAP the the Firdapse end were amifampridine

able [indiscernible], other At general it to prescribed obtain who too compassionate to previously neurologist there aware XXX burdensome over or Firdapse. were QX, of or end have not the access non-neuromuscular of been X,X-DAP X,X-DAP no have felt was not specialists who the

their communication Firdapse Firdapse market uninsured coverage assistance access of charitable Another team insurance commercial in access all pathways has have efforts strong. received and to or coverage insurance from patient affordable priority patient denied launch patient Thanks been Firdapse. liaison and patients of the from coverage teams, insurer determinations our their catalyst insurer the the those to have insured XX% for medication. About been patients reimbursement free positive the and received and has

to patients compares had of and end of on $X.XX payments monthly QX, copays patient the a practice. at would XXX per The continues pathways has month. reimbursed medicine, access failed that has we due insurer, patients access medication, the our XXX we single end to to Catalyst in cost plan launch obtain product. had been unfounded the to the patients At average QX. around pocket, Out to of to unable which who concerns enrolled Not absolutely be in over from when announced

over heard and on out as XXX Finally, a survey have we you regular over X enrolled satisfaction survey a from [X all the online pathway. And on have a Firdapse XX% different for rated to patient in available X-point patients Catalyst the scale], we and had responses Pat, had of X. where patients phone rated XX% from X

six with continue pleased our results following number the half of we second So, months that year. and the and XXX access with of unique adult are new naïve previously. has Firdapse month XX in patients the have to the prescriptions success from of first X,X-DAP In to patients were the launch X,X-DAP commercial who in didn't XX new patients, the or to those increased of look XXX to July, from LEMS

disease patients. identification, We expect not awareness, the around is next the ultra-rare commercialization, XX of that now of top physicians enrolments with and patient tougher range during diagnosis, of raising which into mind in patients month this phase work an gets per or and

LEMS to medicine to an aspirational adult of patients’ Internally, the we by help the end access more year. XXX life-changing or this have goal

target, of we the the to diagnosed undiagnosed XX% patients motivated believe are in almost and We is reach LEMS are U.S. suffering which X,XXX adult this

Now investigational medicine Firdapse perspective. that patients updated our priorities transition of the to a is we commercial from from three strategic complete, have

high help Firdapse level of satisfaction. First, with Firdapse on remain patients on a

seen awareness raise about of diagnosis provider. diagnosed are timely already And LEMS patients. by healthcare to continue ensure that patients LEMS to improve third, knowledgeable and a Second, Firdapse they

programs opinion their will general a and LEMS that them reach support country. program in to include across help efforts commercial help diagnosis. no-cost neuromuscular physicians find test development to in New antibody and Catalyst tool a to find-a-physician patients to physicians A neurologists provide second conclusive are new neuromuscular the and that

We’ve of or that being to currently when our number providers. from diagnosed digital feel directed and campaign sometimes LEMS started fully education being just diagnostic even patients are delay that and We implemented, they recently programs awareness promptly address healthcare patients misdiagnosed. these issues a will

their who the been helping if insurance in prescriptions regimens, and the benefit based continue to always questions navigate there importance and understand patients and I’d have be have person even helping with or in also initial our highlight liaisons, instrumental and of in those patient coverage. to field like medication and regarding access patients insurance, their titration being patients

around come experts inpatient answered, help about that our meet-ups well as latest country each more healthcare fully exercise, for patients as them where learn will the disease. We expand reduction other, tips the recent have the questions and LEMS together to hear or nutrition, research on can can from disease, with their live programs tricks and from learn stress and

societies and with will also and like We by American continue Society efforts immunology organizations our educational patient well neuromuscular partnering MDA, important AAN, groups. awareness exhibiting events physician of and other impact specialty Autonomic explore Genes, the NORD, with the as as or MGFA, groups and like rheumatology Global supporting AAMEM,

that LEMS X,XXX this patients with upon our these you services, the from have to a we program, ensure this As to truly suffering see, access hopefully efforts. build satisfied and helpful of find can success for affordable initial launch way all look and medication adult to to patients or are

that move medicines rare help other successful that patients goal, be and to study successfully neurological with will indications we feel Catalyst toward in As diseases. and neuromuscular will and more we able more

it And to that, I’ll with Steve. turn over

Thank you, Dan.

for to and additional additional programs will developing As we adult for while continue other patients both launch are I on Pat and briefly also neuromuscular with investing have we Firdapse the of summarize to LEMS, in committed Dan Firdapse indications. for Firdapse be our indications. focused mentioned, with commercial

this under the X is for an proof-of-concept special an in FDA optimistic trial Catalyst we assessment. investigator favorable would gravis this MuSK-MG previous multi-site MuSK-myasthenia the First, protocol remains outcomes international approved trial have ongoing This being ongoing indication. trial trial confirm sponsored or conducted and from Phase that

and approximately complete MG generalized expects of subjects the of by MuSK-MG enrollment Catalyst to the XX end XX XXXX.

abiding to the international of first with expect complexity trial exactly protocol in We our in the of the due half the this results the and However, of report to agreement special topline importance XXXX. by agency assessment. now

of this these Pending Firdapse. results MuSK-MG with supplemental myasthenia our to to for gravis a FDA the care ineffective patients forward an look NDA the to already of of add potential current alternative. therapeutic to and for intend approved in standard patients, is trial, submit so positive effective we approved MuSK-MG providing The potentially label we generally to

be this clinicaltrials.gov. on trial details can Additional on found

from patients indication clinical until of for physician to patient and the the in expect benefitting and feels treatment Catalyst is As treat continue Firdapse part trial the to with commitment if is patients, MuSK-MG participate patient that extended approved. condition are Firdapse this we offered their all to the treatment to continue for treatment such opportunity the

trial conducting and Canada. to CMS. trial, trial types at a the X currently which States Firdapse United and of conducted treatment Phase This are being evaluate pediatric certain adult clinical is of we includes sites the Second, for around subjects

trial This label to important results expect therapies portion are of because unfortunately a years project symptomatic half for for We alternatives currently of and that has there the continue CMS, from for very an the second known no in approved topline and year. limited CMS provide this off small this for few several types. been relief Catalyst

to Additional trial available this with the details NDA further our we CMS submit label of outcome to supplemental intend for FDA agency, to add discussions the this of to trial Firdapse. potentially Pending already clinicaltrials.gov. on a approved the and are

support to patients the if physician eligible their Firdapse our expanded access with and all that treatment program beneficial. feel We also continue patient Firdapse, CMS in for is

the diagnosed evaluate symptomatic with ambulatory proof-of-concept treatment proof-of-concept evaluating clinical as a efficacy designed ambulatory X of is X-period, safety, study X-treatment, potential double-blind, patients with muscular a conducting in SMA in subjects. amifampridine study for spinal crossover, a The Firdapse, X:X, X. study Type tolerability also randomized and patients are we to as Type outpatient Finally, atrophy

XX from the results top-line approximately to currently and study The report patients, to we of XXXX. this study expect is half first planned include in

details are Additional this of trial clinicaltrials.gov. on available

pateints Catalyst continue Firdapse with complete patient is and the with treat As to from treatment. trials, this to their offered treatment other their be will patient feels opportunity the if benefiting the physician that trial condition

for territory original included North license Catalyst America, was Canada. which

the approval has Canada submission of new been received NDS LEMS a United with Catalyst have Firdapse file that Now, of in LEMS for drug for preparing or in to the States, Firdapse. we treatment

is progress. to the an we market, comment you our While date on too Canadian for as it anticipated will keep soon posted filing efforts

Catalyst and to Again, year, soon posted is also for begun on for to Catalyst this market, anticipated In orphan marketing filing a May filing NDA. data process filing of too Japanese the has with our BioMarin Japanese we designation an on agreement our it Japan territory. Japan of drug announced on to comment will already add keep the the progress. but in you

also long ongoing have complex These of and project our we form have of patient of this focus patients involve studies clinical to are new part acting requested develop form Many often is and of an new a Firdapse. to projects kinds than we we keep on the it you of are say, more too move we’ll informed still working that to project, in early as this specific any and Firdapse new forward. comments development this diligently Firdapse and is provide other dosage form

research. the this funding are Catalyst application this of and closing, Details drug, these from new request process accepts that Catalyst Should be applications provide our investigator continues be of also progress research to nature any tablets, on made catalyst of the on for other potential in also placebo investigational we indications, uses uses In on can Firdapse. of grand found will provide potential website. for other research potential may wish and depending sponsored examine conduct to material Firdapse, updates. type

Financial Grande, I will to results. financial to call review the over Ali our now turn Chief Officer

XX, Form of share quarter Wednesday, compared filed diluted per On and quarter or or second share XXXX. which reported quarter a on basic second for for we ending diluted X, of $X.XX million net XX and the share in of Steve. $X.XX million XXXX, X basic per our net to second the June XXXX quarterly as August for the of XX-Q income loss we $X.XX per Thanks report

was same ended revenue XX, million. and second compared development quarter million quarter in the X.X XXXX, Related was cost X.X of the as sales for XX.X Research second launch net of Firdapse For with were for million. quarter XXXX. X.X expenses the of the of the quarter million June from XXXX, product

well for consisted Access and expenses affairs, and our programs, clinical development as and Expanded studies, of and Firdapse our the trials from regulatory primarily expenses of quality quarter ongoing XXXX medical, Program. expenses second Research for assurance as

we Research and consisted and Access trials comparable studies of NDA development prepare expenses as Firdapse and of the of Program. the treatment for as LEMS, Expanded period in our submit well our XXXX our as in expenses Firdapse clinical for primarily consulting to expenses

our The Expanded Type substantial in to research SMA company expects continue throughout that activities trials will and and the and be for as we costs continue to Access development studies related X, XXXX Program Firdapse. clinical MuSK-MG, and ongoing CMS, our

Selling, implementation, to of associated including market to launch general second professional in the expenses, due administrative increased is quarter the to selling product XXXX expenses suit for expenses, $X commercial million and in FDA. the fees $X.X XXXX. when the of system The sales against access costs same primarily market law compared million of force and supporting and with increase period personnel, our our totaled research expenses, and as of quarter compared second XXXX

expenses administrative and patient the continue of XXXX, increase build to programs our The FDA. we to sales activities Firdapse company and in against in lawsuit general prosecute XXXX, our as in and selling, and commercial support expects infrastructure

debt. XX, XX.X funded XXXX, and of and June On million Catalyst no had cash investments

based planned be that sufficient XX months. assurance can available at operations there current no we to believe for our the will information’s, least resources Although on be these support next

Exchange Commission in our www.catalystpharma.com. and filed website can on information found Form the XXXX Investor the which Securities X, Wednesday, may analysis be financial page on Relations August be on detailed company's found the was and XX-Q, Quarterly with at and Report More of

turn now Pat. back the to will I call

Ali. Thanks,

this every we physicians, everyone catalyst quarter’s patients foundation We providing are involved forward and trials of are presentation. our lives from launch patients, our to would results including like We look this formal the year. neuromuscular and pleased ongoing later thank on employees, with in to updates you with additional building ends rare Together making this stakeholders. and This changing day happen, very our other this strong diseases.

I’ll over the for the now turn operator questions. call to

you. of Please Duncan Thank is question first go Cantor [Operator Our Charles coming ahead. Instructions] Fitzgerald. from

nice team. and I you a my and for Pat the better you patient quick how progress. Hi, wondering Thanks question the and had regarding quarter to be an a patients source questions in make characterize or, coming the it’s impact taking to and in quarter of starts, did congrats naïve of in terms or forward. a second that could I’m terms contribution naïve if know demand, this revenue make you that going revenue or the of would perhaps

want Dan you take to do that?

Sure.

into with as think this weren't call diagnosed and was data such. get again patients primarily this X,XXX X,X-DAP from out group claims is see study many well. get so just patients a already naïve So, last there been there to of have question already LEMS, what and a able patients, have that the The a I we are trying that to and perhaps been on

So, demand patients. pent-up saw actually, prescribing then physicians with out there those any of physicians new number reaching to were us, for but patients and and their we a also many was same good these initial

digital around to that was looks will a online our and the and of pent-up there I and XX starting like efforts sales field patients that reps, from our into the some per it in, see now either in physicians month reading come get so what And be about from efforts. or somewhere as our about again are to we're with patients that the mentioned hearing demand it

were are In that to patients XXX XX QX QX, of QX. of these now earlier, I at and grew as By right mentioned there the we in end XXX.

some as meaningful far concerned. are QX, in revenues as it had So, impact

We forms are under in In XX days. the delivered. QX QX, a through quick being patient enrolment benefits was actually turnaround PAs that on seeing relatively getting to and

that in think recognition move QX, I revenue certainly some patients from and there So, forward. even was as we those

with helpful. the that on naïve a great, for And but could of overall that you’re the efficacy you it regarding, if patients experienced you That’s color know response and drugs, surprise of provide not encouraging, be feedback quality you getting terms the a is so life? initial regards and to or more in feedback would some the impact tolerability Okay. patients, mentioned

want Gary, take to do you that?

Sure.

for to the on levels when the catalyst patients system, them their not I attention used as high think are that titrated to the again help that having think that access well they’re other appropriately of And patients been feedback, that’s the of from liaisons satisfaction naïve reporting navigate or as something patient been they receiving get medication. with have with has and the positive companies. medications I to personally

can So, GI titration phase, as with as if I well are experiencing tingling drug be Charles, especially there some physician some of issues, patients, paresthesia. initial some during the to work minor for might this effects the patient add period the to typical that it’s for important of this where like the side that are

I is through this the job there period. our get I the these that’s consulting and required titration think some patients patients. so, MSLs initially. Docs And and think and wonderful done patient PLs, that And they’ve with a handholding that helping between naive initial These our

to certain question regarding effect the question treatment the helpful. perhaps the characterize multipart, Thanks. program, decided seems you drug are of a positive. Last seem go for wanted to thanks those ask trial? CMS some statistical transition agents stage, in patients, That's you offering you That's approximately then part progress, about you size you for possibly like it could color. for the have significance just that’s see Dan, making MuSK-MG the data, Steve, that the there conversations next into second said an the the looking the for that And a found to continued being with the sNDA. extended were percentage would on win is upcoming after to treatment or you on

Charles. I Hopefully, you, questions. your will Thank remember Sure. all

with CMS, for that’s also but regards to gene see small trial. First, a there’s like perfect remember multiple we’d statistical relatively world, to In types significance, CMS. a

or one there to win a our of data are in think trial. simply from more groups see that CMS is I signal the captured genetic some

have the allowed discussion I after that discussion us, the is forward trial? in the that of very that mean does things completion call, to and are subject the will all have which we the what and about the about, is As mentioned the one the hopefully indication that would subtypes a with what trials, label. you asked moving FDA and with results be a do which the of for genetic be Tell on

your answer about question answers second of that answer hopefully, agreed therapy the seek the after the to of trial. me patients on extended your all completion the question. patients the first So, virtually number portion let and regard of that With have MuSK treatment, continue short randomized trial, that to is the the is

clearly with the drug those them appropriately. to and a feel to the they the to continue first way is your it patients sure on and favor regard some me if repeat decided you treatment. benefiting So, in just answer I that question part of Could do make MuSK,

of really would is the kind factor the MuSK-MG trial? in terms what it. add, that Yes, of gating guess timing was I the I

data it will the sites be of enrollment, sites at both and trial, large should I half properly close of number and a the clean half international the the the in large just many the all through be all next for up mentioned this we first pristine up complex sites all conclusion as States and that United of the as the carry sure which make because the and data, us will internationally into Well, is records, and it simply year. expect the locations it’s any the all on very of in trial we sites and clean the call, with of to cycling

Okay, thanks added for that information.

thank you. Okay,

you. Thank

Our is next ahead. from go Joe Piper coming Please Catanzaro Jaffray. question with

you could questions nice then guys, provide discontinued subsequently the my XX provided exact congrats percentage and along the provide taking patients you wondering could you’ve detail I’m there it of the wondering like thanks I by provided initiated those color bit insight, but patients treatment, about but were first bunch rate, those I’m if refills Hi that provided, you’re for in some guess if you why a numbers lines, maybe around treatment. discontinued, metrics of refill more some so or terms and sounds the the who seeing on question, little quarter. of a

Sure, is the The rate. is refill strong. can insight give to Dan. some I refill very this rate

immediate symptomatic the a the highly benefit patient. medicine imagine disease would you provides where to As in

for refill actually rate patients, the XXX%. about active at So, is month per our to XX%

active the So, a patients refilling are regular basis. on

would of The Charles did second XX% in patients goes the We in in on didn’t this well think discontinuation a coming well. question naïve see discontinuation, or some the bit I the asked in study and with expecting question being for the [as with as you the that board, work drug neighborhood somewhere approximately and for naïve little a on as patient it’s to effects side patients, neighborhood Pat patients try were we so discontinuation intolerable things] help for XX% imagine rate and and in we in with cases the of XX% some to mentioned, that.

in we why just discontinuation or some the to narrow help cases XX% benefit, is a therapeutic have titration the time not a the is, whether intolerable feels it’s XX% to but physicians, help that we the element is wonder that’s the there, and and patient. are much patient. do truly but concept, we not from again just to so in spend understand long-story neighborhood of or somewhere so if and and the new the the drugs, of see effort be the cases, going responder they short dosing we for more patient other it patient There the In window for we an

this is one will payers so before patients paying. the the days first and the for why free do is bridge provide to product that start we look reasons payers response know actually that XX they can And of XX a the

any a helpful. you’re Okay, next provide market the just physicians Ruzurgi, guess seeing my your detail color high maybe just are offer got question, you any have that? level, hearing Ruzurgi, at That’s you you I Have it. or on to regards with of what to can wondering from patients? players seen around and physicians’ in terms patients if insight you transition any anything in Firdapse

actually very ongoing little are and from representative, sales discontinuation little minimal hearing our as some, and but and patients? seem from it Catalyst little There thus seeing in from Catalyst Pathways our what Pathways both heard We patients with we level the far. and very as very is well our physicians have are very at or field our from

maybe the day-XX the XXXX we wanted going know, point, MuSK-MG just That’s enrollment do it’s in to analysis? you trial it. the to later? just got expectations sure expect on up make half earlier the in change more is, then take to the the the then speculating, time data question completes it's helpful. than And last you just of the XXXX data up a to your rather could Okay, of in time clean know, I is if data one And primary endpoint just following maybe the and first timing. and that clear around due timing topline

Steve?

you the have It’s exposure stage the the relatively is enrollment Once sure in is the completed, that collection and be year. earlier the Once electronic system next said, conceivable has could all that to half is all that queries get and data in of data all data complete, the period first as next patients been the answered. entered short. make the

for have the Some next investigators be the all getting than and in are all about the checked, entered, data investigators half of all And closed if could also to very it and fairly queries responsive been others. it year. sites quicker making early are are first

take longer. delinquent If investigators, we have can it

to it resources. and other investigators the And how well have going fast of somewhat on get as unknown so, an competing done. trials as of Many it’s investigators can

them to And so, make time we out the all them have trial. in our sure interested talking we’re and keeping to there that

the Got it. questions. taking for Thanks

you. Thank

you. Thank

is question Robinson next SunTrust coming Please Lee from go Our Humphrey. Joon of ahead.

This question. XXXX on whole calculation half talking new year. XXX the for – come you mentioned XX for XX A there [indiscernible] about then you patients Joon. patients quick with about thank and some coming that’s so for this in is in about our and that question is of second the month, the Hi, patients per coming

the calculations, on of be of they terms to how So, and [indiscernible]? that by the fair? end calculating are based too calculation at [indiscernible] end end many patients XXXX. patients Or XXXX up then conservative in the Is are is reasonable going XXX XXX of or patients

Dan?

Yes.

and directionally on think prescriptions where patients, predict rare trend. that difficult steady-state in there be in. can now, I are comfortable X,XXX on to I disease diseases, number you determine and and working that. then, always are that X,XXX that ultra-rate patients to what to another diagnosed following new think estimate under-diagnosed. we such It's an from It's be this the we with seems experience coming of see right is that that my what But hard

trial dose the And you. phase, then, my thank running is treatment the they findings was are screening that just can rate? in wondering If what’s success does well question on as basically CMS Great. so have a rate it which be treatment can the trail, so trial, second what’s the the as so all in screening CMS, CMS the that randomized and patients MuSK-MG study? in the we eligible subsequent

other but I in many on commented say seen just get past that randomized. trials. rate, ultimately other many what the neuromuscular in somewhere haven’t the are can we’ve We in – patients words, how it's can to I how in general, success versus screened screening

on you, great. guys, strong quarter. the Thank congrats and Okay,

Joon. you, Thank

you. Thank

Oppenheimer. coming Our from Gershell ahead. go of Leland next question is Please

as I specialists, a outlying deploy ask in patients more to who and terms more to there have you the of bit currently then how them have neurologists, strategies what those and you see accessibility patients much those group going areas effort to And patients? a terms regard larger you’re expense of, are what getting Wanted about get incur you follow-up. those you to being to not might know, than to obviously morning. of perhaps focused perhaps use a perhaps therapy. of a more to good out With Hi, population in on the general in seeing LEMS patients

Dan.

Sure.

So, neurologists these have these are as Well, see, on buy of physicians sales prescription and quarterly to and updates sales codes the purchasing prescriptions. the procedures oftentimes general the still when well, us diagnosis we diagnosis and we essence physicians data patients well. that claims and we but folks country, offices inside of that as we calling data continually codes lump on across hand our even new update, the and using leads over representatives, we those are that’s in the for

good we So, there purchase mix. then, the And or a rural, we kind physician from sales that’s hand have, data in the level, know, off our traditional same understand see whether the and a force. to do you and that urban of where is

there patients a are there many in the outlying that out but are the in few, know, urban are in oftentimes fact, where and areas. quite of XX:XX you actually think large would You centers,

our we’re quite digital reps the addition, bit a follow-up. But to physicians send sales in also communications hand phone we calls in and so, to And or and patients. investing offices both those

as that's half we And so, now about the and information starting that’s, second up the both year. really educational are and search you know, of well where such, LEMS, providing terms as Firdapse, here in ramp to

add that. me to let Dan, And

the their they about around educate and over to they the Firdapse, the country the we that to of clinical and next disease of their and and have that hefty longer-term, some think and fairly fellows, that’s associates. from various fellows an educate them Ambassador’s reaching back making the go about holding investment meetings institutions I for couple program to we’re out investment of benefits institutions years

in various XX various the in had XX had fellows. at May, we They with went have one think, institutions I and And institutions, their those back their I with fellows, of meetings Dallas other believe. to already

longer-term So, said, that to that we’re doing. reference part not the we’re pay effort other educational it of just but Dan doing that longer-term will immediately what augment is dividends probably, has

not here, and of run And giving the when study? on be Could when update helpful. a we XXXX? sustained an might might hear that release know be moving wondering that very forward That’s much formulation, perhaps in to clinical just I but in just then you’re position sometime Okay.

Steve?

assumption might reasonable a about clinical that continuing work that’s there’s batches. be start manufacturing of time completed this we that’s formulation clinical some at believe when Obviously, to then and need I work.

taking for Okay great. Thanks the very questions. much

Thanks, Leland.

you. Thank

Our question ROTH next Henry coming go is of ahead. Please Capital. from Scott

Thank you. Good really strong results and morning, Pat.

Thanks, Scott.

a Just couple questions. of

given XX% low running bit royalties cost clarity That think a would seem going that XX%. it the about what’s on question, Any of in – little there? looks goods modeling I there's total a sold First paid. around like being is

Yes.

years. launch so approval, that expense per our FDA guidance we, to as on of supply, manufacturer most So, R&D the before accounting prior we the

cost able of that to capitalize from labeling we were most So, and packaging. was the supplies launched only the

new that manufacturing and plead for [indiscernible] product a product start sales. we As

You of see more will realistic sales. cost a

the of cost we expect So, increase sales by. passes as time to

on Scott conservative we’ve basis a modelling talked in the use to about royalties about purposes, from the XX%. past, including

Thank you. Great. Okay.

Knowing that the royalties. is XX% of that

with regards the get event Thank for And look should be structure looking lawsuit, there a from for? clarity. should trying we or anything clocks a to sense just FDA any or Perfect. for line an to that ticking then, time any of you Are events there are we there?

XX federal suits glitch Yes. and sometime by We response to There probably think we delay clerical believe a will so that was this have respond. delay, that around days, that Day. that our probably agencies like be XX about filed days Labor will

can we the color So, moment that’s give at really all the Scott.

about Japan we reach market? any timeline have to the as and given then, sort kind That’s great. of Okay don't And what should be size helpful. that color know, of market you I indication, that of the to thinking

Yes.

right now. guarded little a We’re

BioMarin some XX% maybe would basis to U.S., that somewhere on X,XXX that had prevalence precise was Japan that and as the by and the patients there of patients. X,XXX been be with a the we’ve work – our regulatory more and group about that the to match actually some up working week LEMS we're it reports of on team between CRO, Our to be a believe a literature about going conducted is and timing. based until seen Japanese can relates guarded little regulatory prevalence so as last indicates it We in and of bit sort percentage of we XXX

so. or X,XXX So,

have we are… all although that’s we Scott at Japan, point on this So,

Okay. And then timeline wise?

Pardon me?

on comments Any that market? to it long filing the or take how on could to get

that year. information the on in should have later more we No,

We Japan will the a some provide have I get clarity, meetings be now we And position say, several year. in and the we in more and to end of timelines. more as scheduled between guidance would

That you from great. questions. do should Thank me. the for it Okay taking

Thanks Scott.

I’d turn time, to this you. At closing the management for Thank comments. to like floor over back

are so nice year operator. the progress the today's have Have I about Thank saying a We want progresses. far as excited updates in how further to call for and look you, day. we made forward providing joining today close that XXXX. you thank we to by

Ladies and you today's gentlemen, conference. for your This concludes thank participation.

lines may your at You a time disconnect wonderful have and this day.