Catalyst Pharmaceuticals (CPRX) 9 Nov 172017 Q3 Earnings call transcript

Greetings and welcome to the Catalyst Pharmaceuticals Inc.

Third Quarter 2017 Financial Results Conference Call. [Operator Instructions]. It is now my pleasure to introduce your host, Ms. Ali Grande, Chief Financial Officer. begin. may you you, Thank

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

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

financial report on of our Ali forward providing morning. pipeline opening the third will our third as I to few today a Welcome quarter comments will results status activities. well XXXX. you and quarter Ali. you, this the results and about After call. months for in to our development recent Thanks We development results for us nine Thank Steve make updating review our a our provide nine as look joining everyone XXXX month three

which LMS-XXX questions families completed call treatment to significant is Catalyst waiting a approved trial that start at in for as LEMS. for have a tremendous who importantly I'd program the treatment our FDA long this more This Firdapse I'm for then and end X Phase so is a want saying the will such in patients families and for your our have for Catalyst take by important time enrolment pleased patients second cross accomplishment functional well potential very diligently we like is milestone participated. call. evaluating completing the for this today's work thank who had trial to I as the We of LEMS. the investigators teams enrolment who that at to sbsut of their been very

As we LMS-XXX have early we previously December. from to top expect the in results announced report line

to they package. XXXX. meeting January we FDA top meeting the confirmatory hold with as it's results positive the In Further expect quarter LMS-XXX proposed first we expecting LEMS NDA meeting filing Firdapse, of XXXX. to from our are the soon request NDA have trial, our granted LEMS pre-NDA the As line in a we assuming plan discuss if we to to in for hold currently an resubmit that are

or MuSK to Phase patients We're sites. trial clinical the Firdapse advancing LEMS Myasthenic Myasthenic Firdapse evaluating with are MG. with logistics also trial our positive progressing in the addition antibody participating trial MuSK trial CMS In or we for in new initiating patients Syndrome evaluating for a by Congenital Gravis forward X

trial line the ongoing XXXX. first we the from Firdapse data As in clinical of evaluating treatment our half top reporting previously anticipated reported of for CMS

our Firdapse, similar NDA submission in those LEMS. types CMS We for to for also of LEMS to limited mechanistically that are include hope considered

LEMS. submitted that at not may pre-NDA inclusion that slow the a review to intend meeting with However any for FDA confirm will FDA's in down for Firdapse any of the abundance of granted caution be we the such NDA

our with we quarter quarter MuSK points the patients in to anticipate assessment and Phase a treatment and enrolment MG. taken an We on analysis statistical Firdapse of the regarding clinical for Gravis starting about I trial in in as trial design take reached registration approach with end approved no neuromuscular XXXX this expect We be XXXX special FDA enrolment. the a agreement upcoming protocol there protocol X XX will This in is evaluating first my form particularly this of months to disease it of patients for complete severe as the opportunity XXXX a U.S. is a for to this is that currently believe very effects promising skin which about Myasthenic FDA that us therapy

our include and Sabril refractory Lastly, we partner to efforts our programs which of generation the for next GABA-XX spasms. infantile CPP-XXX GABA-XX inhibitor continue CPP-XXX, allogeneic inhibitor

from hear and information our Chief now who more will Scientific development Officer pipeline. Operating Officer Miller, Steve will We our about Chief provide

Thank you, morning Good Pat. everyone.

just outcome moved trial amifampridine As in of know known this the This has X or Pat we touched that use in phosphate positive. on Gravis very recently are from and systematic familiar SGI. and enrolment have LEMS. completed clinical Firdapse Subject QMG second Impression assessments, Phase we're trial forward our of treatment we as we're Global the with for power. of with CAD early completing or optimistic co-primary our line XX trial the trial this the employed clinical endpoints of of results the to were statistical and the is were These data expecting trial although subjects Myasthenia calculations Quantitative until endpoint enrolment endpoints previous co-preliminary provide will this trial won't December. this the of power on The in cautiously investigators based we be unblend adequate and top expected was that it proper are

trial. as clinical X Catalyst stated the clinical of results in sites December size which relatively clinical previously a for geographical database fast The small locations. enable data lock designed in data report small complete is this trial top trial two a early trial and trial was employing this will we path final As monitoring, three to line Phase

these Phase abuse for by will this of abuse information studies have physical Phase there abuse FDA. no LEMS no results first studies the liability drug until drugs liability a liability be announced including these quarter a analysis Phase known assessment liability which will potential. in studies these does although complete also analysis a XXXX. both liability the clinical events already X the study, we're of drug trial, and not A and discrimination I'm three In studies of will signs the assurance abuse NDA the the study we an that studies We Firdapse the trial requested to an if well a X include targets of potential showed no submit as assessment confident and we FDA abuse submit only assessment. needed Firdapse trial evidence adverse three both year. has. other dependence confirmatory Firdapse recently also target three of In we as X with indicate is liability successful confirmatory not studies the of have Catalyst pharmacological abuse of abuse from remain in preclinical NDA also can planning the the for self-administration abuse abuse required These three results and that study but our results our liability FDA the completed As sorry any of be that pharmacological entire reviewed has next

safety Firdapse Phase in first a parallel between submission similarity clinical the as mechanistic NDA CMSROX potentially the X to suffering LEMS from support the treat subtypes using link LEMS Syndrome even the line types for receipt CMS. a LEMS Myasthenic results half also known include On the enable to of discussion We our continue would This XXXX for CMSROX Firdapse CMS for to CMS. we're of before Firdapse and the the of of working types of [ph] those trial CMS efficacy certain titled patients to and findings discussion Catalyst Congenital approval of top path final in data. to expect certain or to

and down or of plan acceptable to the review we with MuSK this Gravis. meeting discuss its in to international slow not to approach the to type the Myasthenic we will Myasthenia NDA any MuSK Firdapse ensure placebo with pre-NDA and or about with the have as known evaluating trial endpoint MG States and LEMS. X% However XX be Myasthenic MG. of assess this special agreement that and Patients that will be Gravis in in the be would inclusion the NDA daily on will FDA our X% may Constitute a approximately agency with of in patients Firdapse MGADL overall the order FDA multi-site population the assessment of score or to Myasthenic are MG. diagnosed endpoint of approximately for estimate QMG. granted will patients received Gravis The MuSK we Firdapse registration of also controlled there antibody blind positive for this United with living safety score a MuSK a the study double from the in evaluate diagnosed patients Gravis activities will trial Gravis trial Recently utilize proposed Quantitative efficacy in This secondary XXXX we primary Myasthenic protocol that XXXX that

year In first future a so marginal safety treatment The in Pat XXXX. with trial set very of will the proof Firdapse. as very limited this enrolment Catalyst compelling were with of is very treatment for MuSK announced March Firdapse the MuSK with this or about of treated The of for quarter As outcome Firdapse concerns the trial exciting for mentioned a development there our no begin subjects MG successfully we with of concept the options significant of results of and of Phase most symptoms seven long which results currently patients. the for are MG in promising of MuSK treatment that optimistic [ph] symptomatic in a this the anticipate trial X we're were with efficacy MG.

Ali patients the financial quarter call to phosphate results provide access amifampridine review disease patients their to XXXX. I to qualify who access free will our first months treated for nine other options last improve continues no to treatment for expanded for who the turn for Our this feel now program charge of will and could have the of physicians condition. over and tablets

Steve. Thanks,

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

this of references share on was warrants query, during May fair the made value results there expired basic our XXXX. Since For diluted to and quarter the change refers to of no warrants shares. in XXXX all liability-classified liability-classified in call per third

So in the change for or value third same the our warrants. quarter quarter share. Research $X.X the per share, same GAAP both for loss million, results that is in In million, $X.X $X.XX of to the was net compared XXXX or a of XXXX. $X.X for fair was as excludes loss net loss per our million, third which for expenses non-cash as non-GAAP in non-GAAP results comparison, $X.XX were quarter of $XXX,XXX the X.X and the development liability-classified XXXX of period million was

XXXX will XXXX. of expect administrative General We of our throughout substantial third that and million totaled be for the balance spend compared into of continue the $X.X quarter XXXX. R&D to quarter XXXX $X.X the to million in expenses third and

We to expenses expenses consistent remain pre-commercialization the expect of for balance G&A excluding XXXX.

to G&A Firdapse. expect launch a the also we XXXX reported are late XXXX potential of which and began as expenses for balance into as year of XXXX to We the increase of part early for pre-commercialization prepare

XXXX either in of As Catalyst revenue development no biopharmaceutical a the third XXXX. quarter stage company, or had

During approximately of the $X.X no had warrants Catalyst debt. [ph] million. XXXX XX, proceeds of quarter received Catalyst million September excessive of cash investments XX, At $XX.X and ended from September and XXXX

Securities which the yesterday XX-Q Pat. the will XXXX Exchange resources call financial found our Commission support there will on no the be can the at sufficient through Form XX page on website to information now be at quarterly of November that and may our least XX, planned be to report with and and back be assurance, More turn company’s was Investor detailed can next Relations analysis these on we Although the filed I months. operations www.catalystpharma.com. found believe

Thanks, Ali.

with look to the a first forward progress in trial this made early are in line that We submission and top to the Firdapse LMS-XXX NDA December of quarter. potential We XXXX. the we subsequent pleased quarter very with have results reporting to for

to updated as and trials keeping well provide activities. I MuSK are of LEMS, believe they questions. Firdapse will need. formal on the we'll stakeholders we available the look as the patients in the as to of CMS for Operator updates development forward end will it the verge over bringing This as of our turn to of continue We be meeting become MG our I other to our on goal and portion our

[Operator Instructions].

go Duncan from ahead. is Jaffray. Our Please first of coming Piper question Charles

for my questions, Thanks Pat. taking

I'm you've know know average having with with any the to conduct completed You patient? or to the the you of characterize perspective wondering way enrolment trial diagnosis that on obviously but you the also a know say at not experience some relative data patient Firdapse either with to protocol regard any the of if characteristics looked

me as were all this invited the for is trial you the of expanded patients, Let a the state Firdapse that on design time. characterizing have recall of been of patients first all about from that access program. What period means from of the patients our will

the time overall trial because with trial. were were the for very and both investigators the of all this the We appeared the from the this of visits up was investigators that of investigators were others and experience the been have the no but that showed We were well medication periods overall good, dropouts a very clean. have long did trial of treated of we're stayed execution this we trial, clinical medication it of in was to on so successfully on execution very the very Some trial. for regard should there with execution the previous was done periods previous and and the being taken thought for they short medication patients in execution had times, taken by trial they felt it very as all their all rescues experienced time With it. they the we're from no two rescues in pleased trial had benefiting and

trial this the in Phase any you interest program into of seen terms back you in new expanded those wondering since they've that I'm you've of the access starts have of any if X to experience expanded program relative off trial. gone with out if the having and patients access patient come change have program them started

program access enrolment itself don’t but the program. expanded I patients our always and trial a of clinical know patients. Well has expanded the stream patients into to accelerated access enrol coming enrolling if the continues we've been actually had

of the is go go Firdapse. patients expanded and program patient expanded With answer into continue they or program? to regards outcome of access the opted single did back to back access on upon all yes, the LMS-XXX the completion The into

in including relative Okay. slow to you or And talk the information agency that the you strategy would some guess but to initial wondering then whether the would regulatory not just and agency about why you it initial spoke might be data I'm consider on CMS actual or that. following CMS discussion breakthrough strategy, complicating easier NDA therapy of to with this already? the topic pursue given [indiscernible] this designation the is regulatory the wouldn't I LEMS

you. will Gary answer and Charles, of CMO Ingenito, Regulatory for our question that Head

the to terms done from So there symptom being [indiscernible] perspective and discussion which encouraged believe would to and FDA with that to medication. of have request the argument is no like for access review Charles, is with those I inadequate mean LEMS and mechanistic again the like they be and see an lot causes of a approved from I them CMS this have us the are there of similarities also who in in that are to population drugs of has CMS release a this acetylcholine which with of a because types diseases actual approved we submit FDA

with will meeting the expect that Charles, FDA to January. in an additionally we in vet we pre-NDA have opportunity this the

that our your an later filing will that point if file it's So it appears to exclude and complicating SNDA.

least have outline I supply turned the you Firdapse quickly there could question and activity know final had the initial you the be thoughts terms back do drug, past commercialize NDA hop in how started and the you but in of at I might initial then around me, and you commercial relatively commercial to sense Should and secondarily which steps or any plans I queue. will then you started in there. believe regarding makes not have you the some That

as lot will were we work plans class research, net a summer our together when program sales be Yes, in that access, been with 'XX the two calculation. of we put but of years unfortunately has pricing, services the delayed would world that sizing, about force commercial regard to already our know ago expect to patient we Charles gross market launch and plan thoughts conducted market we you

So the pulled so that shelf. playbook was ready we've off the

the We off it's two if and through in refreshing, it have information over to that any all now dusted way the of see we're going exercise years. of last changed

and very we ourselves So the think the be playbook the We employing the bring we anticipate regards a to being to and and as reworked. sales force intend a can will get lot reach or we've we changes this review that can year be to. and plan specialized position what last than a the XX know we inventory in first good of the couple we which good are review will shape and market in would of believe launch. we we'll in actually the is and of that months with priority nice we in the you in to if We ready we is if shape being full supplies ready launch be so you lastly and drug prescribers to and and by quicker XX over marketplace five about with [indiscernible] learned swing really now anticipate

Instructions]. [Operator

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

financial A couple of questions.

filing will the thoughts would I you an to that. good I Obviously it have the look through expected assuming on decision? have QX your but in and is on the think just track could than like you started possibly is data you to cash wanted and cash to approval you do less get get earned You question is

that's is know a have extrapolate little this because quarter, cash look we year the which burn a of the million to budget during discussed that for to and warrants new for to can't to will projected the outstanding have we you would is are $XX cash, will answer quarter the decrease as quarter we through year for anticipate XXXX for and that in 'XX I operate we we deceiving approval, on of some where calendar us extraordinary a about have year-end we enough or The you we've which we to say third based our were number cash an finalizing I that the budget we to did get be, speak. do but so but year expenses that we but yes think about exercised for

to and so. are all to will for the question to currently and the QX I early pre-commercialization We us in get launch the preparation to cash for activities kick-off see your will the sheet expenses close balance To we underway. which call which we believe I'm have additional [ph] have I'll extraordinary cash not sure we MG answer burn down enough is to what get will an MuSK a for launch, more want new that study expense that instruct burn

trying way on the $X should we how about just mean financials, be time $X should first of And I in million, staying the I'm the went pre-commercial million to around. we recollect QX, to the think it cost about amount thinking

Just we on any into fourth should quarter. color that ramp the how

done that previously. earlier mentioned really are work for the large there pre-commercial activities Yes, was are activities. quarter as there These that expenses aren��t I fourth in refreshing

million fair $X say million for QX to a would is I probably activities commercial and $X pre-commercial number. in So

about to for months, up assuming And Gravis enrol should a in confirm file, the quick similar Myasthenic trial just that how XX pretty just follow from to but wanted final trial, question we that that. on turnaround LEMS I'm it's think the then

we can trial approximately Well as stated XX in we months. enrol Pat anticipate the

The additional about to happens a that clinical other seek asking file what indications thing the to that follow an I that an as supplement NDA with will you're we to trial regard answer and is think NDA. up to after is approved the

like would turn closing Thank to time over comments. I any or to you. floor for At management additional the this back

thank you. Yes,

updating path the I nine saying be call today's that forerunner a to neuromuscular of to conclude confident in innovation the progress deal for in this Let developing me Catalyst diseases. year on great we're on future I'm first forward that look months and right rare for you calls. deliver and to our by treatments We've on a accomplished

thank employees our their As Thank always for you. and I'd like dedication performance. to

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

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