Catalyst Pharmaceuticals (CPRX) 8 Nov 182018 Q3 Earnings call transcript

Greetings and welcome to the Catalyst Pharmaceuticals Third Quarter 2018 Financial Results Call. At this time, all participants are in a listen-only mode. A brief 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, Ms. Officer. Financial Chief Grande, Aly Thank you.

may You begin.

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

performance. make would the difficult conjunction it Q&A of involve may to I uncertainties, These Executive that begin, be statements like about considered the Report pleasure will XX-K. following session, and current our our Annual results They and may Chief we you forward-looking in relate estimates to time, not statements should to At results, Before the that the on risks, These future and our our to are Form the Factors my comments accurate. are in we be assumptions future Laws. which projections, and statements expected guarantees remind in filings, not with only to expectations, Federal Pat may in Securities purposes detailed for Actual call to Officer. be predict over forward-looking contained the SEC statements McEnany, this which vary. turn is in Risk prove and including information of

lastly, an our our everybody. logistics for Dan a will Commercial XXXX will as us our joining provide supply review commercial quarter. as financial Officer, hired morning Firdapse you for in development readiness on regarding today. Firdapse. to preparation build results our Thank Miller of inventory highlights and Chief for good And On third potential call. and for for launch plans recently update results quarter the launch our Aly in XXXX. early Brennan, a clinical provide and today's our report chain Firdapse welcome Aly, third you, call well Thank status activities will And Steve

will take We then your questions.

across by our of start strategic that XXXX. have and to third priorities me all for of I'm saying, pleased we report the execution key set that quarter Let successful reflects XXXX metrics

strategic Let long-term. near-term initiatives our me reiterate key and

First Syndrome with and will of Firdapse commercially objectives. to everything patients LEMS, provided and within approved to as treat Myasthenic that Lambert-Eaton prepared is ensure evidence-based available, that patients that or to to access part therapy have being do FDA further compliance affordable all

will reimbursement clinical We have Pathways, developed branded patients be best that is Firdapse. to patient ensuring catalyst program This assist the in transitioned and Firdapse in-class diagnosis, LEMS all will program. for seamlessly commercially include pathways navigating will and that intended on education, services disease with be our It LEMS to patients or available landscape. Firdapse that assistance are the physicians three, currently therapy

studies treatment our and atrophy spinal evaluating to clinical key MuSK congenital positive Our gravis, myasthenia accelerate of muscular for type is potential the second antibody initiative Firdapse myasthenic syndromes X.

clinical experience hired Dr. various well So diseases. congenital other Clinical specialist Stanley a with accelerate Iyadurai as in have of we syndromes, President treating these is licensed our extensive as Vice is and manage that in, to clinical Iyadurai recently Development programs. neuromuscular myasthenic Dr. neuromuscular

Canada. Our taking third begin get required to steps assuming an initiative later this approved for Firdapse month FDA key strategic approval is in to us

So the Canadian residents to treat will therapy also LEMS. have an approved access to

formulation acting an we began more and effort develop of Firdapse. long-term, to Lastly, a longer

heard patients a application priority is be or once having and the to four both and dosed we drug adherence. three Because this medication or second a a announced the for As had it compliance great for would that the that benefit lead times of half-life our and day. better mention to short FDA year, we've NDA that to new discussions quarter treatment accepted physicians not dosing, day LEMS. you clinical would the a drug In twice there amifampridine likely know, and phosphate of review Firdapse with of or of

goal been which have now We November closely PDUFA they have XX of review the working away. date is with process. just agency weeks throughout XXXX, a set the And

His accomplish most Brennan commercial will as this to a and is be seasoned rare disease Division. a Officer. VP the that class continue was are he the goal our Dan rare Manager Commercial date. Lundbeck Group integrated hired company. PDUFA and fully in We U.S., hopeful we was a Dan senior neurology In where relevant disease goal our the of be and June NDA approved With developing our Chief experience about world General of neuromuscular leader of year, field.

six his colleagues rare treat hiring launch team the responsible commercial Dan rounded has to of four few years senior the of was neurological months, his from drugs leadership commercial in for the the Lundbeck, of of During Dan past Lundbeck out but two Jeff Del diseases. Sales successful a in Carmen, of confident VP case. early our the we our and is commercial Operations XXXX. steps Jason is for of James, taking and prepare launch who who and to Senior now successful are that Analytics, we're senior all VP Marketing, necessary the

preparations. now, that turn over the Dan. with the introduction now So call an will of commercial who Dan I'll our an launch overview provide Brennan, as readiness

commercial drugs And launch rare readiness commercial XXXX, as Pat. an Catalyst we a look we experience for I'm to our time developing made experience deep a that such and with the for of team. in progress bringing strong plan I commercial launch It's leadership towards and exciting you, thrilled neurological leadership new product diseases. strong has early launch potential prepare Firdapse Thank and treat already have team joined have infrastructure.

ensure culminates Chicago out commercial field three-day the weeks our actively interviewing expanded we swift people being implementing infrastructure now medication from hiring and that remainder support to focusing many plans and without event actually efforts manner integrated dialing as for our commercial are I'm them. a facilitate of are able in and to In which and to team. that Firdapse as in program completing product We're in based commercial efficient involves fact, readiness a patient both attract to patients of and the process any access made timely to a lapse fill launch access plan, processes our a commercial currently progresses best building and

Our expected to opportunity focus that this ensure during able gain patients to FDA medication an we time. transition is important period to the access to available disrupted their therapy transition three approved experience not Firdapse is to without welcome during to an patient lapses ensure would be product. And likewise treatment all

with provider market insurance liaison this medical includes and Salesforce patient team, All education. assistance, navigation, and information operations group field-based Our development several commercial support, access our team. functions an patient a co-pay infrastructure patient appropriate teams, coordination for with advocacy and analytics well-prepared teams, along teams, marketing including includes and experienced

to addition infrastructure. and office based building In commercial home field this

food We Pathways, are safely provided appropriately one help materials, process. by the to Catalyst also also guidance, and concerns launch that innovative Pathways, remarked and that to for all their to patients, named pharmacy. ensure developing to appropriate therapeutic hydration exploring and working and this physicians programs. our that and payers, efforts comprehensive have they LEMS with in program needs, doses and quickly get on programs programs specialty supports, educational patient a Catalyst providers, the we're effective caregivers, diagnostic patients use possible between program play coordinate provide and medicine a dosing services Within as discussions our support healthcare careful and Payers of about appreciate other to their during include attention patients based

Firdapse specialty so develop confident for our Firdapse it our the Firdapse orphan the drugs the their impending treatment Our we Scientific necessary who from current several the safely patients ability who provide patients ability diseases. in call to that I more more address to work now the clearly can clinical will collaborating often-debilitating programs. seeing my Steve to be in development career, the relief access our U.S. Chief LEMS. about Miller, which information patients of all am that within successfully effects I'll launched catalyst foundation our what rare ahead I desire suffering the Officer were bring desperately over as of rest the to goal demonstrated forward Catalyst overarching studies. who have look management to with to aspects achieve now arena, Steve? in to team will as launch to team's will desire Operating the Chief therapeutic ensure neurology and I'm to launch that even challenges in and Having to similar debilitating of turn of Officer from

and Dan, everyone. good you, morning, Thank

remain hopeful of the Firdapse date. approved the fiscal we are excited of NDA the our goal anticipated on will As LEMS NDA throughout PDUFA a have for been or for November the and Pat working as on review process XX, we we FDA FDA with decision with mentioned, treatment date be closely that we about

at Firdapse of time for to CMS limited was of the the submission similar only the of decided treatment mechanistically to indication not that NDA LEMS. types include for Our submitted are an as LEMS we

seek However, supplemental line will top the XXXX, potentially III CMS approval half suitability with and work available positive file the half approved NDA to top for more treatment hope Phase second development closely complete with a a results clinical of of is to we the in supplemental to FDA to we market we this our first and enrolment remain NDA Firdapse in evaluate intend and the trial, one CMS. focused expected program currently types submission of CMS types Firdapse XXXX the the of on assuming which to or from all for to NDA in expected line of for results Firdapse

efficacy effective be proof numerous believe currently Firdapse the CMS of families. and study a label alternatives that completed well treatment have patients some of blinded obtaining their therapies of of We no of the reports ago, in the through are as CMS we there years for evidence and small will that Firdapse for as seen or was off approval benefit CMS published

are III showing following concept successful are in the assessments. is already that MuSK Pat patient the trial trial the Firdapse of label evaluating the of at of XXXX. mentioned for we an line next trial is we from to supplement by MuSK, currently or improvement for protocol to this MG and benefited top the Phase an in under a with trial results line MG MuSK Firdapse gravis to this on myasthenia trial Pending of our in half MG Firdapse top trial in at assessment patient Firdapse. of trial multiple with second special anticipate addition a This physician treatment the follow-up no from positive being long-term continue successful proof of feel to cost for FDA results the to Firdapse CMS, if conducted clinical MG, eligible our of safety FDA reporting antibody future we As hope Firdapse underway our Firdapse their Patients treatment receive and way to clinical MuSK year, treatment to include from NDA. the Catalyst approved of MuSK work

Firdapse input X. we from to several experts, of with that and perhaps a increase of use clinically evaluating SMA function or atrophy have Lastly, potential has for the are confirmed neuromuscular treatment concept initiated Based Type currently degeneration SMA and of improve medical seen a patients. progression trials have the patients the on of neuromuscular Type proof slow a these of we of X, motor spinal patients, nerve genetically neuron we in activity Firdapse muscular SMA target who in diagnosis believe XX

provides our support phosphate patients to currently expanded continue which We with to LEMS access amifampridine and access also tablets. CMS program

all this the Grande, who Officer and For free poster Aly production Chief has supply treatment will of inventory having bottled XX call be review Catalyst access treatment could commercial financial provide treatment also physicians upon will provided of charge package our patients, tablets Financial Firdapse if to the now from tablet other the expanded presentation. patients launch. patients their to pharmacy chart therapy over turn resistant anticipate relief the We in in to other known results. of adequate that provided believe have benefit patients to patients. in is Firdapse and Firdapse milligram and our I XX can shops. no this begun eligible these option its

shares. Thanks, in reference Steve. to refers per this diluted All share basic and call to

the per GAAP $X.XX loss to share Catalyst XXXX a For or net ended million a period per $X.X loss September million the same $X.X in for $X.XX reported GAAP quarter or net XXXX. share compared XX, of of

expense, same non-GAAP period development to XXXX, was XXXX as same net third with due the the costs quarter compared for XXXX was there from loss and For in on milestone the quarter ongoing and the no income showroom of of the of expense as medical same programs. currently NDA in our to related net our increase connection support closing $X.X million in primarily million our was expenses our compensation acceptance to adjustments. we and to $X.X May for were R&D expenses non-GAAP Research loss submission XXXX. in XXXX affairs expand were XXXX, clinical increased headcount in third personnel as the trials compared GAAP and period The

We third type for of be substantial costs compared $X.X other totaled development same X, access continue to commercial in period increases and in for the will in $X.X XXXX prosecute headcounts to launch in XXXX XXXX, MuSK million programs during Firdapse for and program the were is the treatment the quarter collaborating expenses and in expense, to expenses Firdapse build expect balance that compared in research of MG Firdapse. corporate General as third primarily XXXX. source Firdapse potential up development we've and the completing programs XXXX million and pre-commercialization of NDA infrastructure of and for due administrative trials, our quarter our and expanded for continue when increase SMA the to preparation submission our we for the accepted CMS,

$XX.X we preparation including cost workforce in G&A the XX, and for G&A launch no of the XXXX Catalyst cost and to XXXX third in will expect XXXX continue cash We to no and million XXXX. pre-commercialization a as or at XXXX, revenues continue investments expenses in had incurred of operations in September our expand potential debt. to in quarter increase had Catalyst Firdapse compared XXXX

approval report sufficient plan receipts might product in found assurance if XXXX operations support successful on can in I Securities and be floor and believe going considering the cash Although the no which be Relations in we XXXX the be maybe on obtaining there to launch in revenues information, filed the that at that to quarterly through available current company's XXXX. Firdapse is are XX-K based November for yesterday Pat. and on and an of resources website we our without we of these receive analysis will with back XXXX Commission Exchange More detailed now was information turn page found Form www.catalystpharma.com to and Investor the our X, will

review potential the forward to couple from steps regarding a confident importantly a of months are are we and all to Thanks, of we're very bring therapy excited we the Aly. upcoming commercially a necessary LEMS We've evidence FDA based exciting that hearing pending look available successful for FDA the community. for FDA our NDA, to most the taking launch approval and approved

We for your we and rare LEMS. regarding have such and a and diseases confident team therapies experience leadership great grateful are commercialization for as commercial

as in made to the programs goal pleased operator PDUFA the In very programs may addition presentation activities near the development we the MuSK that call formal upcoming are clinical of to future. for end to occur you the in look namely that will with as progress we've date, all This these forward We over providing CMS any each of our on turn MG. on will questions. well the development and updates business and

you. Thank

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you me, interactions you noted specifics. the that many my ones provide Hey and can't questions, with I appreciate FDA, your couple guys, a for taking for so just thanks ongoing probably

product, thoughts to last gone. you just some you have access So over few those remind then was quickly how And us in weeks, they months currently level how of about matter guys high and I convert interactions could Thanks. program, just many if wondering maybe have comments months, how is provide a that? commercial the few could the a around you patients just this expanded LEMS can

So have extended a be about just position the have Joe, under did patients yes, don't to that in we a basis. program. number we to publicly on talk update access quarterly of have not to We

start will amount reimbursement can bridge as the XX we access that of are probably it'll imagine not and expanded as patients a we're as you payers. And have a to program, to expect have get to XX we approved commercially patients going our take get so, drug those available now, to days that fair on by we soon And LEMS in possible.

maybe Medical review Gary, little So Chief process. during Our the our the and then, few agency you can Officer, months, about a give of the color interactions bit with over last

Yes.

were we answered without continuing There labeling to next requests the final product now So for any right work delays. several all information, are we be over and week. outstanding through received no will promptly requests the have

helpful. Okay, to up, like around got quick from so data have it year one some May second of XXXX very first half the half That's to the out insight follow be provide just CMS just pushed last the it. seems been that.

advise will Gary you Sure. well. on that as

ones disease CMS a So and children, the already criteria so conduct mutations accumulate small protocol. there data trials the on the addition now a various means historically to order in necessary population than symptoms, are safety rare as to of that in cause difficult exclusion find that we to through sub-setting pediatric LEMs today levels is we more CMS in and in pediatric to know even inclusion, rare are you are over of will those addition look meet that XX focus

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to logistics get and so longer but the identified, just expected, it's think through also kids to result, to taking patients the getting have it's clinical there your imagine a longer a them to just physicians. we than we're patients As study. identified submit and we at hospitals can Primarily is getting parents than the we through we challenge a through we to taking as you go children's and agree trial need enrolled

taking for questions. Thanks helpful. very my Okay,

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with of Gershell & with the question Company. proceed Leland next your question. line comes Our Please Oppenheimer from

Hey, morning thanks good my taking for question.

Good morning.

to eligible possibility population bit Hi, certainly CMS ask that for seen mutation eventual at for mutations observations there be has in that be to legal CMS on previously could be the the wanted if that, that past any you've about see may further in that only that use we've to patients the want I believe extension the versus U.S. of further us the other on seen, would potential specific you just CMS just and this a given include? that overall if patients been perhaps mention not have all for

testing we not product point of which defects, genetic blinded, I which few there responsible known with ones unblind excluded, out the are so the ones be which this be say can't study a at get actually will most Those for the So we are are and until data. are however still is ongoing worse to and Firdapse. may like

and remind the infrastructure the directly you building then but now you just are and of on where of commercial thanks it out. be Okay, in will of that us size the are can the if that in you feel is regard with course question of what terms that speak the, the side that build building to like as where process we sounds goal

Sure. answer Brennan Sure will you, call for Dan Leland. that

that And U.S. about managers that plan we a right the and is addition those with for Chicago field total liaison that in also XX or prescription on people handle prescription. titration once five total O'Hare co-pay is cover of patients sales or board, the Where the in including the goes up interviewing to to help that a to that, and and made helped along in at access looks on the XX help be what access now having navigation patients insurance director would go reps. our to hotel patient force we're beautiful or just

questions. for Thanks the taking great. Okay,

Henry ROTH Thank the comes with Scott Capital. you. Our with [Operator question next line from Please proceed your question. Instructions] of

you and Thank good morning.

you, questions, Just a thank Pat. couple

on there size First that with in you time and think we could a potential market? Canada Firdapse about regards of to how line the little color of, just give should the kind

so that meeting So and and a once determination approval, after Health meeting the better will their I set will think timeline and really set we have up have will a determine have again to the we U.S. we format Health much Canada with Canada. requirements

appropriate we this of Scott, market a is. that's find travel we'll would pharmaceutical discussions likely companies some might drugs Not in space to probably the with and the a look partner route here had any be that in so partners for neurology that point. idea at the timeline Canadian us, couple that we've

sort We size will of probably basis we of advise opportunity and equate opportunity. shortly to would U.S. XX% course estimated pro-rata a the the of and market it and market the on market

Okay, pursuit timelines least of and color second least that think at how about but wouldn't terms about approval also we should we for think significant longer then way the until after you that thank that? at a general obviously the you it mentioned acting on in formulation, in the any should how probably start on

finally we into out in property approval for this. Well, have and we three eligible there and our work do develop for we intellectual patent to point be has after plans also start well the are product intend pursue and year some as a clinical as shortly prosecution you is not it to but order formulation factored and time to to appropriate only of all to exclusivities new a will be

the to concerned, to IR timing release be when of the is at overall As would and the time approximately far we frame the time for exclusivity out. would is patients sustained product like ideally the introduction as transition running

reading this after Phase that? was on just X of for why I to Final Phase thoughts that question, versus through the just curious proof have Okay, the concept. SMA that XX-Q. one just only X classified great. would I you a Thank your proof Phase concept more is do X or the I as on concept of proof of was perhaps curious a noticed thought you trial that. And you

after out try positive. would more correct where should a would you're the proof it that in of we yes pursue strategy concept we to trial Sure, turn one only need be to do to

for questions. you Thank taking great. the Okay,

Thank you, Scott.

you. Thank

comes Fitzgerald. proceed from Please Duncan Cantor line Our the with question. Charles your of with question next

I perhaps Brenner. a questions. thanks the guys, Dan for Hi taking first couple the one for had is

of fair being the new ready Regarding wondering force sales you've amount infrastructure will you I commercial to here or some keep track I'm talked strategy of likely effectiveness over gauging not whether getting of people about course and that the like as gauge go-to-market the these are with are the you'll a your in very guess XXXX, metrics what of traction and terms in market. to be making

At physicians so physician certainly of as especially trying our floating of beyond people metrics of the right target to can screen. the end on more, discussions along focus also get of thanks out entire Over know a the data more with we the primary and main Catalyst question Sure, as certainly wondering are through the for to reimburse as drug a what of target around on probably new of and are on we'll probably patient market target are in prescribed that are Pathways high satisfaction with our mean and such, AP. starts, relates is beginning patients easily there as the the the insurance and helping and our force so through of there's process review the that transition and with Program symptoms the type both that we that to it their side neuromuscular these well the specialist quickly patients of sales the physicians in those people and that goals, time these into and that them. to XXXX from tests. offices have year is LEMs over are types from other claims and diagnosed on do right well the high also satisfaction our next patients radar as getting the once or with that at the is neurologist that our level level diagnose patients that undiagnosed

next team all XX, metrics are XX our the and So, main the and over see sales six, marketing those months. of goals force the for that

hopefully Pathways. the helpful. quarters. to you'll I also That's few about guess of wanted talk of over course about Catalyst And the be metrics those you to some willing first ask I

past to As you there cite Catalyst you that some valuate particularly that you've your would other compare on? being how that of and are as experiences, worked patient interesting to or it access any programs Pathways come elements

high-touch programs worked infantile for number of similar I are that this the Very on to at however a done many Huntington's we products spasms, the there being and people that with on it with before, built in worked from a our the with such at that the group, we of so the patient infrastructure that center up services ultra-rare have had in similar, Lennox-Gastaut, people hub this have also in small place but that field. and the both liaisons a had different disease, of programs end are Lundbeck perspective, many

now to patients I And we're from very they're not, it patients be we on an have see that them providing. then impressed the to at that expect programs, we -- the staffed what would of on level beginning, they're at or these that Firdapse they -- many think through as it EAP education the talking are the in with beginning right [ph] especially are the I titration. up handle you influx that carry programs could are physicians bolus support of throughout. have the that mean comprehensive in and very of to with includes and dosing whether that's

color at you really answer your you I Okay, provide may about that the us pricing that, and that's then or the doubt way of provide of you'll additional think input if the on but Pat could view for pricing. some not some asking value because for time. or this pharmacoeconomic one features wondered perhaps you And logic of I whoever, Firdapse helpful. that about I'm that

with Firdapse, value Firdapse. therapy anecdotal with get or in studying call, there reviewing There, participants we And based to currently there will patients we a to as all access the good therapy. and information obviously, of the that early not of Firdapse for And what's spent one and what FDA estimate a therapy are Charles, FDA-approved saying is that, have to approved. keys anywhere is think we major marketplace provide are an affordable X,X-DAPP therapy. the that what a And time today. case FDA-approved of proposition Yes, as on access We've other the company well lot that are an involved the patients XXX question. is the which on, to I'll XXX some from key

the participate doctor what want the to sites don't to paperwork. to XXX And Patients of miles drugs. And in travel have XXX, see to IND other get issue XX% all doesn't of doing. other so a referral because required don't doctor their the want because an XXX, are program access to is the investigator XX% have to

So benefits that Catalyst expenses. the have will living. docs and is of of life their can main that minimal drug feature we lives, program affordable and clinical course changing their the an therapy, approved life-changing a I simply patients then ensure will the their daily changing Pathways with the activities all drug, have of patients one, delivered drug, or think the minimal main of our copays, to FDA that out-of-pocket access house. to hope have their factor events quality where Two, And write script we

of sort our build And we so of price the which to proposition for we the where are think product. those I cornerstones regardless use value

of in look different Steve, the you And if that of I'm to question, the thinking. version beyond I wondering Pat. in efficacy that clinical of you of forward will, or the efficacy? it the length do do further that into that inputs are the compliance. lifecycle franchise, in if the the think for may future. the terms long-acting? Okay, But a Last of opportunities could so for even mentioned earlier, franchise, you different long-acting perhaps with improving any do that's the profile terms there believe you Firdapse Surely updates appreciate helpful, safety have we'll

improvements a SR And an of terms Charles, answer is, that's medical good definitely utility You question. yes, in there to will the of question short some your product. as Firdapse know, the actually very be

basis tremendous have sleeping product levels off up wears day. they're out in therapeutic difficulty sustained benefit would patients the be so of get intellectual is going morning they want intellectual to A of regard immediately night maintain see changes And go for a to in benefits levels concerns that One and that to of because involved would it safety, detail probably. just morning. for the we the efficacy common start up do them. a perhaps might have potential any in blood property. don't able I'm property most significant would the getting bed the be the any you reasons, think that future release I that to or have With their tremendous while give and answer throughout be of, we they drug and into nebulous to

my me. for look near-term. to in updates sense the to taking Thanks some makes it Yes, questions. We forward

Charles. you, Thank

time, any no are There McEnany back further you. for turn remarks. At Thank I to questions. this would call the closing over Mr. like to

various patients several upcoming XXXX. to stakeholders all reporting of Thank thank to the you, Michelle. beginning you to that our key of look milestones this and we on continued morning's need. have especially our to weeks launch achieving want the support, And in participation the excited help for we're employees bring tirelessly their of We Firdapse call. Thank your worked Firdapse forward in and all for months. about in And potential on

you Thank you. We will advised keep as progress. matters

concludes This teleconference. today's you. Thank

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