Catalyst Pharmaceuticals (CPRX) 15 Mar 182017 Q4 Earnings call transcript

Greetings and welcome to the Catalyst Pharmaceuticals Inc. Fourth Quarter 2017 Financial Results. 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. pleasure for your Catalyst my Chief Ali now Officer Financial Pharmaceutical’s is to host, introduce Grande, It Incorporated. Thank you. Ms. Grande, you may begin.

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

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

to will full activities fourth and on on call. Ali. will for status year. results Thanks fourth And for We and today development you to the our and activity. review joining development welcome and milestones our forward our financial updating Steve you, report year Miller Thank a good everybody. our and XXXX look Firdapse afternoon, and quarter Ali us quarter pipeline recent provide end results for

we our at and we regulatory overall our study resubmit the questions X Myasthenic for for prepare achieves be secondary the we our which endpoint. of as our this proposed The In as in well Firdapse, that NDA we proposed briefing resubmission. November, a our a package Firdapse from productive was Phase liability the very track second as which we month. for in Type NDA your call. included on results on Lambert-Eaton FDA data few topline this FDA trial take endpoints, of as abuse later After year feedback our activities weeks. in with our briefing advanced January clinical December these as the the advised C In quarter statistical meeting we results, of the line and next significance FDA the to studies. submitted were and top to based Syndrome, known our end This well In co-primary the our then LEMS. NDA announced us from meeting will We we the in advance remain as from the held resubmit positive documents to should patients as FDA with sufficient to for receiving

we have LEMS, our neuromuscular these as for progressed indications with well. Firdapse to in and addition In platform activities patients development other

conducting currently Firdapse of for known CMS. otherwise Phase a are We myasthenic congenital X for syndromes, the treatment as study

with indication, subtype past antibody indicated the positive for have forward assuming data. myasthenia gravis, the look and forward quarter of MuSK-MG. patience complete with for the half first positive And to the trial working to this to of quarter, second advanced bring in Firdapse of results otherwise FDA collaboratively year expect enrollment this from We in in we studies MuSK our also this this topline trial of XXXX. known report we Firdapse This as

for expect this and to and take announce granted a our trial Phase We of we we with begin roughly the agreement the months will few within X XXXX. FDA SPA protocol announced, previously complete trial special have half enrollment for XX to next trial MuSK-MG first As we results for believe the topline been expect weeks. assessment this to in

enrollment treatment X. which to affects Firdapse looking for study with myasthenia we’d muscular ambulatory is there SMA commencing gravis Type are X or evaluate to atrophy the no Phase subtype gravis We spinal myasthenia approved recently as the currently about of a X% forward initiated announced to population. that in We total patients X% proof-of-concept

anticipate We patients XXXX. in the of enroll second study study the in results to announcement expect topline XX this in of half we approximately and the

launch activities As we potential of pre-commercialization in previously have of a our many stated, Firdapse. advance restarted of we have

plan updating medical our force affairs logistics research, and and distribution communications, access, current regarding sales market on sizing. based commercial are We reimbursement, market field

very develop a are affordable Additionally, as to patients providing to Firdapse. working we committed to patient we access comprehensive services program, are all

refractory to In activities on our continue efforts for spasms infantile development addition Sabril. to of program advance version our Firdapse, and we for to a generic our CPP-XXX advance

partnering Sabril. deals CPP-XXX both reach to effort and our continuing also for are We generics

these programs they available. on updates provide as will We become

life proceeds. Finally, have we completed new with raising public of in a successful and were offering investors, both almost November science existing group million $XX a great to pleased in net

Scientific call details of a ended Miller, more and our of with will over Operating status last to investments the result Officer offering, year pipeline. about our cash no who million I provide now $XX of we Officer, and will As turn development Chief Dr. debt. and Chief approximately this the Steven

Thank you, And Pat. good afternoon, everyone.

or have reviewed or for announced to second Myasthenia endpoints, we NDA Phase defines peer of improvement of journal. conference significant co-primary and a quarter endpoint primary secondary well significance in future as global SGI, Firdapse trial topline the relates the Catalyst mentioned, of the trial the from as at our clinical the X global results to statistical a that results the impression the impression made this of on secondary remain the in LEMS. also first year. the of We plans the prospectively were present track in results The was scientific to Pat Both CGI-I. achieves to plans As or Quantitative We physicians treatment endpoint Gravis as progress QMG the subject positive LEMS. in trial. and November in publish endpoints of this submit it

Pat we studies the submitted having studies. review meeting our not of in second to confident comprehensive our FDA including as discuss the trial accept data be the from as that does abuse in from to resubmit submitting the sufficient a NDA quarter. data abuse The to be the data potential. FDA FDA information subscription this Phase As NDA, of X package well we we Firdapse submission a mentioned to expect our our liability the to LEMS, the end have FDA and will that for liability are data provided abuse C from that these it, indicated will before with After FDA type to and NDA this for all our

Firdapse topline CMS We trial our protocol. and XXXX. for to continue CMS-XXX syndromes of our in Phase results the quarter the The trial to the using of we expect XXXX their protocol reviewed from first clinical this to complete before comments treat this suffering the FDA X as in myasthenic have and into report incorporated from trial titled enrollment congenital patients to end known also

those were considering we reported, that generally CMS considering - mechanistically LEMS are limited similar to in including types filing previously for have considered As are of we us. initial our we

review that using after thorough seek Catalyst we Firdapse date. is to population a it the submission assuming for our the is indication the FDA and This CMS an initial results with and internal addition the also ongoing will of indication after a it to at concluded patients enable However, our we our to by a CMS broader have successful. CMS with for from labeling second later CMS to a indication add for trial, evaluation, of overcomplicate best not NDA of LEMS the plan discussions

first study We the trial the also Phase Italy. in This sites double the will in which U.S. XX blinded about trial MuSK-MG be patient future. plan include X to the outpatient enroll and will a near in

needed. MuSK XXXX supported this FDA in patients priority X. this gravis myasthenia and We they the half therefore will in suffering take for if XXXX XXXX. approved expect or sponsored, SMA approximately multi-center high expect to XX a topline of approved supplement positive spinal months Firdapse initiation disease support the the NDA myasthenia of results results of Firdapse to as we patients clinical to a study announce trial approximately for This the study that hope ambulatory company is tolerability in complete the are is symptomatic an there to and X gravis proof-of-concept well We and to FDA atrophy previously controlled an for first announced submit we efficacious agreement sub-type demonstrated treatment an of for a We trial X development Type is safety, a recently for effective from of of The U.S. and Type this muscular were be Firdapse we of MuSK-MG in adequate for protocol led treatment of in a conduct the granted the patients. from sponsor these treatment in indication year may investigator symptomatic FDA of in trials last treatment new of that and evaluating MuSK-MG. that from the trial. new are desperately support assessment - this for August efficacy an We trial of Phase suffering - treatment special SMA investigator Firdapse us, the

and development a degradation the following neuromuscular SMA this Firdapse meeting from receive the could relief be also effective neuron believe designation such study we for schedule results are if in drug to the orphan XXXX these with discuss the positive second further FDA expect [ph] junction to an that the and indication. We FDA also patients. slow the to provide symptomatic of results for plan SMA half which transmission topline of application to motor submit they in can and We enhancing for

continues qualify our condition. feel treatment results. program for the now provide accepting tablets and which charge the to financial turn patients physicians patients diagnosed of treating to will could with and to phosphate improve I The to disease for access program their CMS. expanded patient’s Ali, to call and free this Our amifampridine access currently grow LEMS is over for review

or for net Thanks, million share per compared December net refer call Steve. $XX.X per XX, and our per $X.XX XXXX, ended GAAP share GAAP to months loss the as share to XXXX. diluted of $XX.X this basic we for XX period same the in a Today report All or loss to million in shares. $X.XX of references

of to XXX,XXX million of net a loss of loss $X.XX XXXX. or excludes change liability-classified In change in $X.XX non-GAAP or per net attributable was non-cash for loss year the XXX,XXX share warrants. warrants, $XX of gain million per value non-GAAP $XX.X the attributable fair fair which non-cash to a Excluding share, value XXXX in was liability-classified comparison

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

of changing to $XX.X $X.X million For million the Research liability adjustment. same net and million million and the year $XX.X the the loss for fourth were share, of XXX,XXX and the GAAP non-cash expenses or non-GAAP quarter net for was respectively, quarter they fourth full fair value was classified In excludes million for comparison of as non-GAAP quarter loss $X.X loss year XXXX. XXXX, $X.X which for compared development in per quarter and non-GAAP XXXX and fourth full XXXX the fourth $X.XX were warrants. of as as gain no net

Firdapse gravis if our million substantial expanded manufactured Firdapse program myasthenia and to in launch for fourth in expect compared SMA be respectively, fully the quarter and and to XXXX clinical spend quarter expenses XXXX. that continue R&D and in will programs $X.X year million MuSK CMS, million we totaled year Type continue X, our supplies. fourth $X.X access and and administrative of continue our seven the $X.X $X.X for and million General full XXXX We

we increase as expect G&A the up prepare to infrastructure for the build expenses Firdapse excluding commercialization. to expenses We pre-commercialization in XXXX

to XXXX. launch We also in we potential of expect increase as of significantly for reported pre-commercialization are in which prepare part a expenses Firdapse as G&A XXXX

of XXXX development company, Catalyst biopharmaceutical fourth or XXXX. the full quarters revenue or a years no As had in stage

we in proceeds. December net the of investments approximately in XX Pat XXXX million and XX, closed raising cash million $XX As public no earlier, mentioned November offering Catalyst At debt. had

on for we XXXX. revenues earlier the More detailed are XXXX, Firdapse today assurance, might to at cash will Securities in filed turn resources receive be to we can sufficient found and believe and the call report approval analysis considering our back in are XXXX we Pat. through our I Commission these company’s website there and financial be be if Investor now in in information planned obtaining operations found XX-K Form which annual patients successful the Although be the Exchange support with that and of launching and receipts we of may no Relations that can and an the was product page will www.catalystpharma.com.

from Firdapse currently to Thanks, and in with we Ali. LMS-XXX With toward bringing a positive productive type Phase our C FDA trial advance data X continue patients meeting need. confirmatory who are to

We and be patients next with to the currently meeting within option. do results Firdapse milestone few who are have our weeks. not significant a resubmitting for approved with This FDA our for pleased FDA Catalyst, for just the from forward we LEMS also treatment will an look the not the but suffering from NDA

Firdapse several be other we a progressing are could indications In with believe addition, promising we treatment. potential that very which for

will to they our of the that turn This trials it and forward We for we'll questions. over meeting will the provide and end portion the become we to available milestones continue programs and come. our look be hardly of formal on updates as

Thank line with Jaffray. Our from Charles Please you. Piper first [Operator question question. of comes Instructions] with the your Duncan proceed

Hi, on the in guys. for last Congrats questions. quarter. Thanks the progress my taking

So [ph] Firdapse it firm, then of NDA what for working within that the to know steps ask to consultant kind are of are you you call giving you NDA, Wondering final about LEMS. And all you the handling I did submitted. are this in-house? also preparation want and

the that Thanks I’ll Charles. let Steve answer for question.

Okay, Charles.

using actually to with us the firm a are NDA. consulting We help

quite large. As know, NDAs you typically are

critical one XXX,XXX parts Certain and the written us. work to been XX,XXX Many other been closely and somewhere pages. and In share between review documents have NDA key by information. we fact, by we of parts have written other this expect consultants very be and

quickly know, on data. enrollment that It wondering CMS at longer moving timeline. just that than am initial I recalled filing. bit for not NDA had the going little to and all to concerned not that then then seems you And in timing, about Okay. I a But include you're CMS

And so their you terms little in us you, patients a does get what thought CMS? it little on on does does or broader - patients more that of give Could you you it get get characteristics? color set a of baseline

to which from if recall a previous has a trial of patients a office four requires naïve many that visits over - month CMS number the and addition so are period, for Yes. office it's in been involves you'll which visits. of to patient a design run-in period presentations, the a have crossover also of and amifampridine they

study. As more has a result of that, it been complex to enroll that

so fast hoped. is gone as of that it And hasn't part had we quite as enrolment

a that, In a mix of including representative good CMS genetic that to as addition to patients get and very and also adults of pediatric both patients defects are ensuring good well. trying mix we we have

some to And activities. Okay. your just then pre-commercial on moving of

you're of if you've that [ph] old wondering burden that for models. mentioned You've to you pharmaco future? consider dusting the the economic thoughts, I I looking the you're patients. guess further have [ph] anything, off disease or I'm key drivers any and know value are LEMS things talk probably to at pricing that the from identified that but in about what don't want you

on economics with about point. regard with to We're And suspect working like more life understand conference just the are prepared talk premature talk the We're next call and if the it's things this would to not of our a research that. have we by still we now. issues, little to at studies lot And a doing drivers We contractors value about of we'll would will. some pharmaco you quality that. what think market the of Charles, I be. value that

Type that. really X, question on intrigued Last SMA with Okay.

that think the subgroup you're I working with. ambulatory that's

stage said and look of I data you that patients? to ‘XX think younger other - way then half there's wondering First, second to I'm the in earlier if for I'm earlier subtypes at you a wondering expand approach - an

make for Well, conduct it for this trial. X the in implemented this The Type current trial - trial we for endpoints ambulatory to designed the the the have specifically the that easier patients assessments is much of patients. use

severe, there's are the So in really ambulatory to participate the of as which the opportunity patients of most a severe Type patients Type which more result for that X an less X is much not trial. or

limited now is inclusion So Type criteria X the for patients. to right

Is some you it [ph] if the case forward that's expand going above get might core you that data that?

from we trial, X, only it acceptable trial SMA get SMA types appropriate to also agency them want might for data a done Type Well, whether be not well. for or proof-of-concept the conduct very other we how trial with that's expand favorable that we we will and to as this if of part could not what but it's to after discuss the is is

Okay. It’s very useful. question. my Thanks all taking for

Thank you, Charles.

appears no at back closing to further to time. floor for There questions turn this to like comments. McEnany the be Mr. I'd over

thank I’d to all the unmet for patients everybody I'm on you. promise helping suffering medical their very today the like needs. deliver I'd to participation Thank in call. you. for its thank XXXX. pleased Thank your stakeholders forward Catalyst treatment like also that transforming to on support with accomplished in look more from even rare an I XXXX. to we advance for our

teleconference. concludes today's This

your your may lines You Thank time. you this for disconnect at participation.