Catalyst Pharmaceuticals (CPRX) 13 Nov 192019 Q3 Earnings call transcript

Greetings. Welcome to the Catalyst Pharmaceuticals CPRX Third 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] Please note, this conference is being recorded.

the Officer. to turn Grande, host now Financial Chief will conference your over Ali I ahead. go Please

Good morning, for everyone. Thanks call. joining today’s conference

Joining Miller, Dan Chief Brennan, members are Officer and including and today’s Commercial Officer; Catalyst Operating Executive on and Scientific Chief Steve team Chairman Pat Officer. of Dr. Officer; Chief Chief Management McEnany, me the call

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

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

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

a update Ali, it's our Thank Well, you of work joining Catalyst thank for morning XXXX and we and to across streams. months everyone today. for and have good third progress of an us nine you exciting been on and quarter lot various you,

some Let through launch our launch of XXXX. me the enthused our about strong third making start of progress Firdapse. about by commercial comments We the remain quarter quite

patients. of a as physicians, positive across We as see and from variety metrics, payors trends continue importantly encouraging to treating reaction well most very

unmet have days underscoring our continue underserved in and in the of As the need prescribing breadth this Firdapse expectations, early population. the largely launch, exceed our for patient discussed we significant previously of to depth

trending long-term we increasingly detail. completed, performance and quarters that of key metrics positively and potential are Dan measured four discontinuations in the greater daily three, now we go will the Firdapse. Patients starts into of With grow performance confident in

Firdapse for for conditions. with only our start LEMS, to leveraging optimism not is or off myasthenic neuromuscular potential to and great a ultra-rare we're syndromes help Overall, growing patients other but

the first million time revenue now stage $XXX year with XXXX guidance revenue calendar the year of $XXX this $XXX for we're of full providing million into of comfortable range At to launch, and guidance approximately for a XXXX. million of for the

As next to we more continue the gain for we'll of experience look XXXX. to forecast tighten year, range

team account As our be making investments an to X,XXX we to XX growth, to we achieve current managers. XX will commercial complement and to regional contract targeting that providers an patient that And stated expect Firdapse. could expect might LEMS adult regional up representatives. be we benefit budget treating add add neurology from To resources for to healthcare that year's next for goal account hire the be will we roughly inside managers anticipated of sales neuromuscular seven They our XXXX. to end XX additional to further

of As XXX-cX we provide patients. the am number Firdapse patients announce be being to will grows, charitable the expanding contributions independent treated support to in XXXX pleased I qualified that our that foundations to LEMS with

of going amount the charitable we dollar to that contributions foundations from in number both this support and increasing are We four. in three of

is needs we While cannot enrolled to support that main patients Pathway's are things directly. LEMS one Catalyst support support we programs can we we the cannot community. our to intended – who these the do work of to everything in LEMS to address contributions patients the adult support and It who

Austin, Medicine efficacy exhibited communities. meeting is the in Last physicians, American Texas, Neuromuscular important month us for Annual and engage of we and attended with and venue always Electrodiagnostic an at which to Association scientific

LEMS clinical stage for neuromuscular most benefits positive messages patients; physicians very company adult as our out neuromuscular year and Catalyst first important As to Pathways, this the assistance programs a fellows. educational for program to our various and our our attending commercial get physicians about was it of patient Firdapse was

in hosted Why in Adults symposium. attended Perry we presented Neuromuscular was a LEMS and free Misdiagnosis. LEMS. introduced Specialist a Insight program we more symposium during Clinical Shieh, by meeting Common that this this room-only. was It titled; a At suspected from our to I program Warrants than have was Closer noted new antibody testing LEMS standing that UCLA patients It Dr. for lunch might a XXX add physicians.

the about This shorten program to patients for Catalyst will accurate will out-of-pocket antibody patient this eliminated to diagnosis quickly LEMS journey ensure will lengthy an that hope to for as diagnosis others. insurance a covering allow cost concern LEMS testing possible that help the is this physicians LEMS and order otherwise without no We as test. for

however you signs of or the secondary showed know, endpoints we patient top disappointed not CMS-XXX improvement. that and the results clinical are our patients XX did recently primary noticeable significance on We from we did several statistical As announced study we this in observe achieve that the study. small in

Congenital very Myasthenic the a complicated disease an neuromuscular patients XXXX CMS afflicting estimated or in XXXX ultra-rare Syndromes is U.S. to

the findings as to as our next well scheduled the this the year steps. from meet with end to study FDA of are before We discuss our

have treat We small even number approved population, FDA attempting CMS an patient of to to patients. been therapy specialists by attempting very study neuromuscular complicated to in provide a commended for while this

anticipate we year XXXX. end study this and by topline of reporting half enroll this MuSK-MG and continues completing the of the in trial to results first enrollment Our from

small Steve will for for in half the trial quarter, new of Also past information proof-of-concept shortly line the first provide XXXX. to initiated we've and target countries. programs. several muscular are on regarding these two this study X of top spinal Type we more you with sites European announce complete atrophy in Over results the

strategic of priorities commercial to is our to start footprint One previously you as our stated globally. expand

have have with filed NDS priority and granted New our Canada Within the past a Submission review. weeks Drug few Health we been or

Canadian of with an evaluating Assuming commercial plan firm process six-month the the this application. efficient means your Catalyst to submission options launch is and with in of We're for or our accepted team. effective Canada partner with review also

Labor Additionally, application have Welfare, to and understand over and with data our met months the the data well and in Firdapse of discuss PMDA existing several as what submit authorities to Device a new Ministry as maybe Japanese or drug past regulatory Medical additional Health, including we Agency the for required Pharmaceuticals the Japan.

the to Firdapse We've credits. to applied first years tax us in if against also in recently for quarter anticipate generics review, designation, market priority and of meeting Japan. drug granted, give next XX would exclusivity register hopefully a plan the which orphan nine-month certain a finalize with year PMDA We of

the second Japan can expand that offers conducting very we in promising largest research footprint. believe to opportunity been strategic market free We've world, as there global and us be and for the very our economy a

external value impact and assessing late-stage well medical have for creation that the as We their for debilitating a drug needs as development clear potential drug through suffering acquisitions path began have are may process programs on believe to disease. that evaluating patients we rare unmet from

yesterday, reported with ended funded in third and million we $XX.X the investments no cash quarter approximately debt. and As we

the we term access Our believe that cash stock. see loan continues or Further, we nondilutive sale in financing as a bank a to promising our product we build such if a source of quarterly and we acquisition of alternative event conventional from forms lenders to have for opportunity. no that near-term common need

approve schedule June January. against FDA. briefing in and it as I expected and suit LEMS current late patients for to different a the here four lawsuit We grounds, which is state Lastly, in a called by that FDA's suit few will detail. we to the This be filed decision challenges the all pediatric filings Ruzurgi on about great made progressing for words

understand, you can we time as can chance get this a the more additional there's and before decision. get the is timetable that best case at a than likely this provide of delays. you eager As judge to are I But always this

keep We will on posted front. this you

execution very team. This nine quarter has another of been performance the and and Catalyst solid from months

We number FDA-approved, LEMS by in of the to made to naïve need that the an for patients necessary to we investments Catalyst Catalyst community. again bring illustrates have evidence-based continue enroll are the medicine to which pleased the required further Pathways extremely

the turn to I'll with to you on of over our Brennan, launch Officer Dan Commercial our further Firdapse. specifics now call Chief provide

here excited continue Firdapse depict for Thanks execution LEMS. metrics to of remain launch from the Pat, States All and adult an very successful suffering good strong morning of every. a about United in launch. ultra We drug orphan

availability patients launch this a is rare, on XX We've started many is now January percent commercial with patients, condition. coverage exceptionally having helping very patient that Firdapse, very diagnosed whether in the physicians debilitating or metric quarters total disease patients treated product of three affordability, satisfaction, insurance a completed patients and number of is with and

and and now LEMS a Firdapse received new of is the nearly launch, brought also In diagnosed as population indication very payors physicians, high result of is nine hurdle for that great prescription in XX% a recognize we months of improving standard patient for it Firdapse which the a has medication. adult of rare first LEMS patients that care and such. is This availability treatments the

help successful continue as one will we continue ranks If patients, use to to this LEMS and adoption more rise of the the with we to launches. level orphan of drug

X, call been QX to highlights patients what have QX never over I'd naïve alone. therapy end whom of with these the since now launch, key like Over share. treated patients had before prescribed Some initiated XX XXX Till XXX over and we of of to in therapy. Firdapse been X-DAP

our over transitioned the Catalyst X, more initially for access had on When never than to were it access you patients expanded Pharmaceuticals would not and there now from patients are if who consider Firdapse who program, XXX Catalyst efforts. X-DAP,

LEMS XXX Over of maybe patient. the amifampridine XXX recent them over about we the insurer potential asking patients space added reimbursed QX. use into unforeseen another And market of prescribed Firdapse and concerns LEMS product, with on adult there at that understand about and the Ruzurgi entry unique of and were some writers have many therapy end

a differentiated point I'd consider. like out to points few to key

not adult use is Ruzurgi patient. LEMS indicated for or in approve First,

spoilage requires and to refrigeration Firdapse chain prevent Ruzurgi doesn't require Second, refrigeration. and any supply

comparison Third, support programs for Firdapse. limited patient are by Ruzurgi with

we LEMS any especially and to education this company, fully patient disease category there for a where thoughtful a and committed market to needs and support are product education this entrance that important in believe and consideration. be rare like dose as finally, safety And in

is not about and and proper care insurers why taking understand That label physicians a believe use. should and to Firdapse with use and they drug we do patient good off physicians ensure careful having insurers we are safe and generally to be Insurers so. physicians are inclined and its that

October. we have seen and ones we then available and and believe some have commercially another of who Jacobus September leave we over to small love product. in said, compassionate switch August the we patients commercial try small mainly That In with insured Medicare a group group Jacobus early run patients saw patient study Firdapse insured use of September

to slow important that is note, most bit receiving Firdapse. in weeks. the physicians three the here we it this believe have quite However, last satisfied been and are Catalyst at seen fully We support a that for down patients they've with

I mentioned we As high to earlier, of patient's satisfaction. continue level see

customer Now X.X XXX average with rating remains satisfaction completed out stars. over and at five surveys of our

insurance government are for XX% and seven to rate with prior XX within is approved approval patients commercial insurance -- being Our over insured our approval authorization and rate most days.

all co-pay in In per a $X.XX across stood patients addition, patient. at month per the September average

small approved all we over pediatric why only relatively left LEMS this the XXX these to group have try Firdapse For patients is of reasons the believe product. a

been and point some expected XX% rates remain saw in reasons with these there lack the are other succumbed discontinuations reasons study patients for patients. in normal I side-effects clinical to of about But have response, than XX% out of like should the to line cancer. we who what discontinuation for that experiencing unfortunately also

the study patients from naïve of by LEMS on access program approval bringing which of included who waiting Firdapse. been X, Having met goal also receiving from transitioning from our end over our enrolments patients adult have the and X-DAP launch patients QX, Jacobus XXX initial expanded the our

extensive level commercial and While doing new strategies sustain availability, our awareness executing raising of to activities patient this the to population. initiating a adult a which focus launch are turning our and with momentum we includes next satisfaction, and commercial by Firdapse the neuromuscular patient expert raising affordability high neurologists LEMS patient, on to beyond support several of including the continued now phase general awareness, for

the discussion have AAN neurology other meetings and the regional very meeting, meeting conference, a experts various Society medical We of neuromuscular Autonomic and meetings productive fall local recent AAMEM the and including general physicians. American at

test. insurance we mentioned, this antibody hesitate suspect patient no approval program Pat launched test for addition, physicians so perform In to that no-cost submit have LEMS who may their as or that there wait our LEMS need diagnostic for and is to

a experience are physicians We LEMS, find-a-physician identifying are help have located near readying looking tool physicians treating can patients web them. those who for where we that

non-personal advertising increase physicians and mainly level includes digital promotion of our in patients. to an This communication and

earnest the calendar We turn end to are help process in the patients at reauthorization to also preparing comes and year. working upcoming of that and insurance each

largest high and target our like sales LEMS group XXXX contract from our LEMS all of complex of treating new retargeting we will XXXX expanding We physician success patient up help of and inside patient LEMS about list neurologists. level and or symptoms know our includes continue in believe and neurologists Lastly, sales most that commercial force with edition treat and these is that on to current a efforts adult satisfaction. identifying this and over

ahead milestone that impressive that LEMS of patients our of come. helping gravis, adult the happy To LEMS prospect say I'm population. CMS, I'm helping to weeks excited about for company patient. excited that patience adult adult diagnosed positive SMA with the and which conclude, as of to in idea antibody additional get Japan, also is an just of very we X MuSK XXX ago and what Canada lies for next few and potentially important the years ready helping Type patients Firdapse, we prescribed myasthenia XX% in XXX the a LEMS I'm cross

over I'll clinical Officer, Chief our the with call Steve? report who progress And on in Steve hand the Miller, that, will activities. our Scientific

develop clinical Thanks for indications. update provide neuromuscular now I'll there. pipeline commercial for the to update an on additional And our Firdapse

on our Phase or regarding myasthenic the syndromes trial touch First, news X like congenital to would for I CMS.

disease XXX about estimated XXXX controlled genetic clinical range treatment a representing currently was symptomatic patients spectrum Phase first XXXX produces CMS This variations of therapy. wide X CMS for trial genetically to genetic clinical confirm XX to to of trial double-blind different the called the There This ever in placebo the U.S. of are defects presentations spectrum be a response and of defects. over CMS. broad possible of

We patients to size received showed the this treated endpoint global in measure. trial statistical some to responses for both sample this Phase amifampridine, period trial secondary not enabling the and Because did was crossover positive trial control of treatment assessed function response endpoint two each in and impressions of primary subject X subject of and the or design, XX tripling the the individual to overall muscle given achieve significance be placebo their with to each patients a patient's so treatment to trial. crossover

variety four trial This reasons. clinical took almost years to recruit a of from

a physicians. of they at First, trial disease to CMS of has needed only hundred In patient EAP testing diagnosis patients years time. free is our for a with rare four the past clinical charge diagnosed the this network any provided with over Catalyst very and order genetic within few aid

And this despite foreseeable patients the provide this trial, outcome future. will continue for the of Catalyst to service to

clinical exist placebo. trial that their the in the child maybe are with their of participate many the to Second, where treated are many child and reluctant allow patients to children parents possibility

CMS nature XXX their to to of to often the be at of complexity existing doctor's that doctors were is trial and sites, to the included the it CMS clinical one trial. clinical referring understandably be the disease not so and be in sites pediatric reluctant Third, patients trial patient's the its unfortunately could new a due more and willing treating were physician

to make this of given in require already agree child Catalyst for Finally, this many families all clinical patient's a the their trials thanks of the a trial. time caring families participate have commitment that disease. burden cannot existing who with

the encouraged treatment. individual response clinical trials While outcome, patients some of we we the were remain by to statistical disappointed in

and year the meet the full potential of to FDA dataset the by continuing next the and results, are scheduled to treatment discuss are conduct the with to observe to of We end analysis the responses steps.

they become We will updates as provide available.

the was trial believe valuable outcome the clinical for their population complex, data information patient physicians. While and provides this of the we

international multi-site Protocol conducted is Phase an an ongoing Special conducting are SPA. also FDA Assessment or on We MuSK-MG which X under trial being

trial X continues following trial successful Phase This indication. for our proof-of-concept this

first XXXX. to topline trial of this results expect the report in half from We

the treatment. in population defects had lot heterogeneous As previously, many a very and due in trial response described patient of to genetic resulting to involved variability CMS variation a

However the homogeneous junction by is to population single like which should a protein a resulting MuSK-MG LEMS caused patient response patients, single antibody in trials. a across in consistency in between more neuromuscular treatment exhibit sites and to multiple

trial we are Due this is currently Phase success the in XXXX optimistic for about we there no remain of no believe the which outcome or and small to part MuSK-MG. with previous an proof-of-concept about U.S. patients MuSK-MG trial X for approved ongoing there treatment to is MuSK-MG. autoimmune XXXX cautiously

Assuming one forward approved a options to day potentially patients. FDA this to provide successful trial we an these able look for being treatment

ongoing Italy we X. and muscular have ambulatory efficacy safety, is study or SMA its of and with spinal tolerability is a Europe diagnosed patients trial SMA This X. Lastly, Type amifampridine and potential atrophy in and proof-of-concept ongoing Type evaluating the Eastern

to in look the enroll this the forward genetic severity. relatively to SMN of approximately topline in defects SMA to half result study plan protein neurons, XX on first study should XXXX. mainly is in caused from a patients by which We disease differing homogeneous and this motor results announce related

NDS, six has in treatment of to New for submitted approval expansion Catalyst Firdapse the Canada, cycle Drug to time Firdapse seeking now has months. plans the reduce review Moving or LEMS. which for this review market priority on been granted should a of NDS also Submission Catalyst symptomatic for

provide seven about months With the time response XX time NDS acceptance yet review Health plans the in receive Canada. a in Canada when and commercial priority of review update days, Catalyst regarding for provided activities our required to accepted will expects to filing and Catalyst in has start is review cycle activities not commence review plus over we those month. an of this commercialization Canada's the NDS

include announced the Catalyst begun have and pathway Welfare in discussions to Japanese with of an Firdapse approval seek also expansion Ministry Recently, recently marketing or territories to regulatory Japan. regarding we Health, the to Labor MHLW the Japan. of

file the designated develop two the been a Firdapse companies for the years this Japanese government ago, and actively Approximately NDA have MHLW approval of and for soliciting to they as drug drug. priority an

file will provide on of Access pathway be patients an Catalyst most for MHLW CMS or our an continues in access An Japan, required be NDA filing to for the providing we'll Japan. to Catalyst what to update Firdapse NDA and in the regulatory come in EAP. Program agreement that Expanded

assistance EAP We needed also their if diagnosis. the optimize each genetic strategy to tailor This provide patient. subtype obtain treatment obtain testing in of patients order an and accurate accurate to and physicians physicians genetic treatment CMS helps confirmation diagnosis CMS to the to

assuming diligently also MuSK-MG this add to begin clinical our many trial including and successful plan Phase MuSK-MG outcome work We with and a of treatment EAP treatment new symptomatic involved the in IRBs EAP X we'll the of to the protocol.

a requested doses their around plan eliminate have acting need to Firdapse. to of multiple version order patients daily long the of in Finally, Firdapse activities

in new updates actively this future and when characteristics finalized. now are product the have developing We provide been product the

candidate being in treatment acting release developed LEMS. the symptomatic stage drug and properties being studied are this order to At program, the of optimize are of formulations development their long

about to patients. product to develop as tend expand excited all countries the in indications, are additional for opportunities a well Firdapse better the current label into these we additional as Overall,

become any available. these regulatory provide as path clinical will updates We they on and

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

XX, of and for basic XXth, Form quarter reported third which third or share ending our income quarter per XX-Q the XXXX, million we September for net diluted of November XXXX. report the Yesterday $XX.X filed Steve. quarterly on Thanks we $X.XX

share quarter million $X.XX to a $X.X of This third diluted and net or per XXXX. compares of for basic the loss

Firdapse same XXXX, of For the revenue million. quarter September $X.X XX, the net from XX.X with costs at ended million product related the sales launch for of quarter

as medical, quarter $X.X our our expenses for trials consisted third the regulatory affairs expenses of million well expenses development quarter XXXX for of with compared assurance programs, on development access studies Research $X.X million and of in as XXXX quality expenses and the the ongoing from for and third program. quarter Research clinical XXXX. of third Firdapse and were primarily expanded

up consisted of expenses as access submit in development and in trials comparable as our and to program. NDA period of clinical expenses consulting LEMS of the Firdapse well expanded primarily we our Firdapse for XXXX studies of prepared other as our to Research treatments expenses

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

when setting primarily general million and that's quarter third expenses in implementations increased Selling, and quarter The increase fees to including against in professional the due compared same supporting expenses, the third personnel, our and market of period the cost force access of $X.X product launch expenses XXXX $X.X XXXX. of and sales administrative for of XXXX associated to expenses total the lawsuit to system million, is compared market commercial, FDA. our research with

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

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

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

on financial or detailed the November Company's be at www.catalystpharma.com. Commission XX-Q Relations and report information on website Investor and at analysis can XX, may found XXXX in Tuesday Form Securities the with Exchange filed quarterly and be More the page was found which

I'll now back turn Pat. call the to

Thank you, Ali.

other and stakeholders. to I'd who to our extend made everyone LEMS Catalyst community always has success patients, this physicians, thanks including like for possible our As employees

Our look the and better to and update we forward disease in neuromuscular people of programs of our future. commercial providing you lives near the with clinical each mission remains to on

over formal to our is I'll operator now the This call for turn presentation. the questions.

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

the quarter I market. on and and of progress kind this granularity helpful and metrics. launch to appreciate team. and understand Pat all couple one Good are really the I related Congrats in then that a pipeline. on is questions the oriented That's the of to had the the that performance morning, commercial

touch on so really things LEMS are he if with for inputs question the some what start is that the of when guidance? you the could I'll guidance drivers then and you And And key consider that commercial really the or market front but a your the more said little color patient competitive maybe dynamics, new You in if provide on could identification? wondering I'm few the and bit a remarks, prepared

Thank want your do and question. you, the Charles. commercial with for Charles Dan, guidance you our to metrics address

guidance Yes. remainder I this mean, in for the Charles, for you year as going? were far the as year of where general, next

a that's it's the year next to able provide that you're little this bit Really Yes. time. I'm see. to nice surprise at that surprised because

Yes.

key that of is, on. launch elements and One there's we mean really elements I and a a had think I patients couple fantastic over. the going transitioned

next it good when I such new to harder have QX a think launch into this that enrollments that we've and of QX been call in you becomes phase. get describing

we with XX see physicians be the it'll be and a programs folks per we're that list more the in with XX. in sales I think our of target so place in And probably do XX expansion month. can to that that the field the in we the with on patients that call and new additional inside force sales to in are going these in additional moderate putting And new enrollments

are all things that until got transitioning discontinue have mentioned and treatment about quarter not them first naïve you it's start all and a getting spoiled patients, like you patients the of I kind a of the is about second in there's to rapid And thing seeing when these some patients then that you these not little none launch the patients we of that respond. start

intolerable somewhere experience to neighborhood the of over patients product. approved trickle I discontinuations. patients Some And or in to you mentioned LEMS effects, have you of intolerable so then as and small a are XX% side -- pediatric going the as that XX% get

discontinuation. that patients subtract have those out to You have

a what more and get in into versus rate moderate quarters you see that one, So growth three. you helps. Hope two

Yes.

we amount more question, We or those. addressed around and small been which when believe, under of that I get patients coverage were when in And what typically, those color we competition, to two anticipated use seen in some have launch kicked most were small think already. most commercial Dan back product it the suspect other converted Charles, your Medicare about they little a and your head were the have Jacobus and lose on. the they've part program go able that insurance patients to part for there would July. the I'll call that to of each for taking their anybody I compassionate boluses they the product for wanted who transition they made we've

to going it's patients. to we as ahead in better And -- offerings a forward so comfortable more services and that feeling these retain we position we're and move think with broad our support we're of patient

additional at? is of the Okay. adding then, us you're sense you regard seven to can you're like a that And That to adding. clear where adding what XX the trying the should then you're the What wasn't and XX give case. that folks looking be sales seems with I expansion it reps. to

Yes. to Charles. let Dan I question, take that

seen data find additional than to by experiencing on So, seen been that we've are medications used diagnosed or neurologists doing able physicians XXXX of we've a this list LEMS LEMS that have with prescribing often that purchased patients. symptoms. displaying X,XXX use that which much higher existing LEMS we've and that some patients is are patients that And

that these often a is patients. using physicians used those types they're have when So of seen medication

XXXX manager face interactions force and we they them with going these these area. to to from to with then will we will patients the sales who and we're of a has about that officers that So that lead, they supplement the sales by that the account calls train are to force hey, with inside way then And essence up sales types office. confirmed inside of in one to an we to patients field come hand the essence to in face phone visit that go lead make our understand, out XXXX with and field of group over reach a to all regional the go once going get to in

and more effective efficient infrequently, lower of you a they Peoria, go general a more orphan way in that of ultra and is more to patient do these account in example, covering catch to the of when type and regional neurologist have including a Illinois need orphan decile there area right still ground disease timing but you're just the in right when go to office this near manager these is there. of an work especially It's an who don't some physicians patients when you will for

heterogeneity in I'm patient could regarding That's on I wondering then if provide could actual enrollment that in Steve. seems, Okay. but Appreciate CMS you I the trends relative that of be done A kind the general of question on all as well had transition terms to that of commentary helpful. for if number MuSK-MG? lot terms information pipeline MuSK-MG to the to symptom or groundwork as perhaps presentation. of But in

Or off X a would if And positive by MuSK-MG you sNDA? study, would end the then, data anticipate perhaps are second half kicked next second Phase the you in year? would next be the year year anticipate of an out of

all I sNDA question, trials; in the be is practical trial an of Charles. the purposes prior this With number trial the anticipate Phase the trial. indicated first well in approval indication. meeting regards will current I successful X year. enrollment by the going regard their successful to of the assembling And the this an the filing currently specific they proof-of-concept mind or of guidance we scheduled seek therefore Okay, agency with two And to the enrollment the file plans in keep sNDA indication. to your end any trial, ongoing say combined that and successful the sufficient after with the the that MuSK-MG trial With a for is that provide can't for upon we're completion enrolled and completion have on patients to trial. of to second complete for would clinical

in enrolled patients test? of maybe that whether trial of an and And targeted the be have just number they Okay. me all antibody to pass to remind

have for there we so Also The a be on do this that acetylcholine and testing patients determine XX that FDA group. patient enrollment enrolling some an those well the patients. there's we MuSK-MG as hypothesis requested receptor to enroll MG is XX test no patients tabulate effect well. data can XX to reminder any to patients eligible receptor for pass acetylcholine is simply targeting patients and they or not are that The as antibody whether that as

quarter. the Congrats helpful. Okay. for Thanks progress in added That's the the color. on

you. Thank

Thank you, Charles.

Joe is go of next Piper from The Jaffray. Please question ahead. Catanzaro

guys, for questions. taking thanks the Hey

XQ me. that heading Just that that driving going a couple for the that confidence Maybe What's guidance, Is trend that? into first more reverse to to that off the what transitioning quick we simply suggests Firdapse it if is look you patient's at is a ones XXXX? around Ruzurgi. gives quarter. going guidance so XXXX that down of be to And

have given the address Dan, a for we that that our quarter the maybe would we be of details correct, down. as should put to you we're into But, giving to for assumption get a not that guidance are range revenues range. approximately a demonstrated going this Dan million guidance maybe is that have without want to a the that out conservative that let bit on well. And little sure really, Joe. But $XXX be I'll and that at Well,

Yes. No.

discontinuation. and plus more success this just, start into settling three I partially stable think the it's that get into great the quarters, any enrollments you do we of see still we LEMS continued is again, discontinuation a the the in approved discontinuation well had you a for to subtract have that normal first transition, pediatric product. get past out we going as to also as trickle rapid reasons, and I mentioned mean, once may

have small of And so ramp be a is the a a launch quick this there's to seeing. adjustment going in market, early we're and period period, when in you what

success better going that flattening and flatten in the LEMS, the quite the handsomely, have is. you severe initial more it's to So out in

think initially it And and patients, what providing was now with in that what try patients, to was now much QX. the this we've And understanding we we got so, discontinuation it that And see happening. that's we the more some better what with on physicians higher XX%, than and understand studied discontinuations of is early messaging is originally do with lower down in we periods. in and to better had was and to we expectations a saw very with gotten What which XX% setting line guidance good dosing initial studies. our naïve

the with symptoms holidays see all we And this. And – in mentioned growing. and last the we'll next that always anyone So be sure those is then discontinuation the bit all cadence. of We just is there's million enrollments There's and on normal LEMS. that And now continuing, I'll range number. where we that, a XXX QX such expansion, point mentioned a of both in that will and colder. the end is by that actually into getting on with there's And the on low Pat want challenging, effect the weather don't programs days year, has side. with to that's say the stabilizing still less that have make we selling a the surprise believe around a honed seasonal that we patients and and that with bit a the

these And so guidance with we're and on you try make surprises low we that giving dial best no the to all sure the side. things

Could the prepared it XXX quarter, numbers at was what me, Okay. the of you the patients end of you remarks, on at end and provided you but said end of have say Firdapse the And QX. XQ? this end Got think previously at I the was of it. your XXX at forgive on may it in XQ

Yes.

XX patients So had that free of second we and includes at patients therapy on drugs. the all active end getting the quarter,

actually, Program patients. quarter, So overall it rate, Bridge the out same is the end our Patient the XXX and Program, third they or about about Assistance or that so had and at flattened of

new growth and outs has team bridge subtracting by and waiting payer again, just getting transitioned work so, that previously just our enrollments, reflects And really the good of that not but also getting reflects helping in over paid coverage, Catalyst addition the discontinued. of Pathways patients a therapy. which number reimbursed, our saw that patients we and anyone of the onto the insurance kind our only But were at for team

for last here. Got just it. And Okay. then me one maybe

the So, general, that around you that? to transition of mentioned doing Thanks. of in do of you off small that a the patients some group you're continue numbers And what's slowdown? And Firdapse patient's this that? seeing percentage that? on you Can to expect provide driver What's Ruzurgi. maybe,

another because information have really to exactly patients we physicians, call they and because that with to their communication It's such most many to to hard patients have know say we Yes. to just don't how discontinuing. many understanding patient going up switch why try a the Ruzurgi drug. But and reasons switched really different and of capture these do good and is I'm

commercial, So, believe of the study, were end And there XX seem patients that to QX I mentioned, as we of that on Jacobus the mentioned, them other we had compassionate this you be or you the made were about originally. to at that most the patients drug. know, as transition

three very few two now, we but past weeks. about right then, XX, the in to And see currently that

working patients their Pathways time, medication, Firdapse without throughout they we're worse these going to signs actually that if to pick to on expect that up return to some journey that where sure they back for they able reason, to realize they any these no And that. will can, the left patients addressing of Catalyst behalf. that off. at patients that they come so, we work ever it's know discontent be that, they're if of better lead we and And any continue as any we and when say And all and will Firdapse we best showing can make with do another or just they're and Catalyst they

That's Got Okay. for taking helpful. it. questions. my very all Thanks

Thanks, Joe.

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

Thanks morning. be? are progress on it on may good or previously and of the Hey, not congratulations on then the that taking had to the another the be on Firdapse, for additional a has all XXXX kind the Pat, Would that, part group focusing Question Year into also you have somehow diagnosed of you you XXXX fair could system you as of brought second declined New great the kind for so who my versus second XX sharing say commercial that follow-up. and XXXX who for detail. I questions, in into in characterize group patients would the as a and if any And reps Firdapse be who be proceeded bringing will far. launch that were

again, to happy Pat. take I'm that

Yes, Dan.

physicians a urgency on believe come patients so treated XXX come the So, With they're diagnosed areas of try ask a not already to that that now patients yet back the know, and saw Firdapse. those and board, physician to representatives patients We that talking there are about have the still opportunity. that basically different in representatives prescribed do additional with are still are hasn't being to of Firdapse. with LEMS or they all are not yet you XXXX are -- couple

XX%, But a some that that's market, get of done. it once said, we past you But as does still be see still target. work is diagnosed there to XX% become challenging. So that a do

LEMS. antibody this That's than LEMS. themselves, patients not And are headcount offices. some of we misdiagnosed. have about to we rare. test. patients those there it's you see so you XXXX this are and I'm addition, field additional Here, LEMS? no undiagnosed -- you're it's or physicians is what to both Why probably believe of Have in sales questions cases test to correct, in if the thinking well, if considered are types experiencing In inside be sure Do see not hunch is cost these symptoms? going the not asking just rather

test, force, well them offices We're field as they're the if patients with off the sales trying asking some up to table, like people the or our both our take seeing barriers antibody inside of those this. as like those hesitations calling the programs

by dose used basis that's regard symptoms Okay. a if been variability patients of Thanks. dosing a seen manage -- any commercial also that's as seen if the flexibility you Firdapse, taken, know you've comment on as With That's, kind drug with or to their And if wondering to has helpful. there's the standard you've could that Firdapse?\ question. to effectively be possible one-off of I but

the you and were end brought whereas the again, at at titration. end be of to the the new metabolizers new a that's XX and dose XX, with the and and patients metabolism that many XX, where But dosing. it's as of expectation transitioning narrow range some of from XX the as the the on and naïve being Jacobus of And quite the dose, that around in study others bit maintenance. higher all patients, has drug therapeutic between a some metabolizers, bit very longer Yes. a kind they came and There's milligrams of high the overall, sure or that patients require patients slow fast do and, how, milligrams quite we well patients pretty itself across milligrams they I'm XX on lower initial not day, average there are moved how Jacobus of patients helped is as variability some over and the very little the side-effects started at of firm of but the fast will initial the difference patients window, have were metabolizers even where, term of board some a around obviously, previously on that realized patients, careful

over on, explain You XX the milligrams slightly, have us the about all and a But average stayed that come patients with population have across overall of such. physicians for and the less. dose that slightly is have to prescription that

extra the Very for color. right. much All you. Thank appreciated

Leland. Thanks

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

you Thank morning. and good

morning, Good Scott.

of stability environment the -- general made pricing to seeing all at you net pricing? market Ruzurgi or a Has couple since the in are environment Just it changed price questions.

work for other a to additional all, all decide We did should patient and that changed was priced The priced of at provide, where on indications. we at what we're as all on we pricing the studies Scott. we be fair and based thought the conducting lot hasn't it services value this

been the the of other entry very stable has company the us so for pricing, And despite

that color. for great. you Thank Okay,

I in market say, talk there regards XX of would what know, it's not to getting? that? may Another I color of if patients? Just think have on question among sense I any know But but Ruzurgi, was with do you doing thought product QX. share -- you about you new do how you that is any kind switches have

give I'll and my as Well, well. then you Dan thoughts probably some has

in naive X-DAP that part we're of that really doing for as think work not X, aware the patients a are are I physician those most education, offices are in experienced physician the as not education. patient well

a on. of there's work going So lot

to more neuromuscular if with treating to the they I regional fact, And with Sales LEMS rare field, work out more it's find. have naive to are who There's required patients. docs account any the difficult pointed regard patients like so patients, that's little find think a by in Dan disease out, managers find or force there to the

market don't we you that as share. I impact So can a think of percentage tell their

there and the the out months. think that previously, about number again, the of it's And will patients Dan year out, end you have trickle, for by I will. talked the is of been large. pointed not about six product we And a somewhat of been as if

so anybody would was go think X-DAP I were experienced have the by they wanted and on end compassionate year. done So that X, already to in the use the back program if of

think, we a Dan, color in any market we can But maybe terms think this that? gain have talk share. provide part, experience we'll better I be I in as that's able you the So go be on for to point. most more do as of to all forward at I position

place. is better patients ultimately, think it's they a many X,X-DAP vice think those be I I people that kind to well, versa. was from these why, seems elements. me old that couple are all that -- a mean, actually patients some see we'll the -- I remember think do the I of it that left, the that the I then interesting. actually I well, of over because mean, working of say, for better are and hear bit that's thinking, think Firdapse my Obviously, working have other that X,X-DAP, we're with come back Yes. I drug of there's We and much on

a on makes I think the is patient that but off make it getting working think new mean, is they're we easier you And and have new why think which have have what up reviewed by -- to the physicians tell. a the something appropriate. patients payers it that was sense. for such. that's them new And are I FDA they're and me that reason We that's some hard to they well and are patients, drug I I on their little the data for And of is a patients. this. studied, prescribing the when in this that's bit this. the like, writing with okay, bring for is don't actually And line for these physicians saying, that's are like in that comfortable don't been I and office with a most patient half. on label, been many year a But, label. -- treating that's treating data, physicians It's kind alluded that a they don't necessarily but who that don't the new It us

Okay, great. Thank for that you color.

that future can at data? expect near in closer that just a question, how Another or we the CMS anytime get regards present with you to data, look to the do

Steve?

we the to speak to present that have forum principal data an anticipate at investigator. some the we'll would basically, appropriate at Well, time. would I

the publishing data. on plan also We

want high we at impact so the wouldn't certainly journal ability to well. a impact And as publish to

into will the available. sometime taken those all that be next year I but will data of publicly to So account, anticipate have made factors would be

Steve. great. Final accounting you, Thank question. Okay, an just question,

on at As number. modeling for number you us to quarterly be see expect point, you a to fully profitable When basis, EPS taxed would the a will more you just fully tax continue some purposes?

have hired NOLs an and look a our orphan separate tax firm credits. and analysis at to of to we our do Scott, credits

around year. hard that's Street. the being now. we our expenditures, get year, soon work as our right -- going to analyze R&D arms to say, as that, hard us so perhaps, to conducted sometime and now we the not NOLs And have this right provide next it's our that's that's impact to as we us for And Certainly, that will to

you there, for Pat color Okay, great. Thank thank taking and the for you questions. the

Great. Thank Scott. you,

end now and will session. to for of and the remarks. McEnany, turn And Patrick We question CEO over the the reached I call Chairman answer have closing

Again, you you. We to occur. for call. with updates look us on today's further they as forward thanks providing Thank Thank you. joining

this you your This your disconnect concludes and participation. conference lines time. at may Thank today's you for