Catalyst Pharmaceuticals (CPRX) 10 May 182018 Q1 Earnings call transcript

Greetings and welcome to the Catalyst Pharmaceuticals Inc.

First Quarter 2018 Financial Results. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. [Operator Instructions].

conference recorded. this reminder, a is being As It Aly Catalyst Grande, my pleasure Pharmaceutical’s your host, to introduce Chief is now Incorporated. Financial Officer for Thank you. Ms. may you begin. Grande,

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

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

status pipeline Miller us XXXX quarter. activities milestones Thanks everybody. recent update a Firdapse review joining results activity. on to Aly results morning today pleased first development welcome We're for you will good and of our provide and for report Aly first and you financial Steve call. to and our Thank our development the quarter on will our

We will at call. questions your end the then the of take

quarter we our key first of achieved for XXXX. one During objectives the

this resubmission as our our application would the in to Type we which NDA meeting us our FDA As resubmission the results be FDA for syndrome Lambert-Eaton myasthenic known the proposed for demonstrated resubmission. for announced treatment LEMS. advised of of or package Firdapse sufficient drug with this C new the filing Prior of the with had that

X However in that NDA with submitted we'd trial March as October. we date NDA announced top XXXX quarter trial the whether statistical for this XXth, well treatment in trial CMS. significance which XX announce the results first NDA a we the products resubmission determine Phase to endpoint. positive X This conduct submission results our includes submission co-primary We've limited our NDA previously will acceptable we patients endpoints from continuing the LEMS study not the myasthenic confirmatory secondary mechanistically types last was types syndromes of in for to that of LEMS. completing file. of of complete half limited our our achieved of the for of we CMS that evaluating would has expect a is From period the the to enrolment in Rather which which the day include stated Phase filing the FDA than and they review XXXX. those are We second as and in these include in congenital line to anticipate similar are

broader us Assuming patients. a this Firdapse. for trial to plan much approved is to This address successful population label to add any CMS potentially we of seek allow to plan CMS will

for a reached gravis the and antibody an positive approach protocol begun announced patients under endpoints, myasthenia for with clinical agreement FDA patients recently that assessment we otherwise our Firdapse trial. we X MuSK-MG. in design, statistical Additionally we announced In of with August that as XXXX protocol MuSK known the enrolling of analysis trial Phase we in special have had this

Designation Drug ambulatory Firdapse atrophy Orphan gravis. had for with X. trial patients In Firdapse we've concept type the spinal a received treatment near we the future. subject for evaluating of Recently And in proof myasthenia of enrolling first addition our in muscular initiated we previously anticipate

enroll Italy expect study top to Carlo Dr. Besta to this expect at approximately to Maggi supervision patients of conducted XX the Institute in XXXX. half under be the in the and Lorenzo in We results line of announce Milan, second

the execute We the Firdapse. continue are launch to of potential the of to required that success on many pre-commercialization of activities ensure

required and align current activities As the refreshed we are many being the paradigms. the have in completed with now of appropriately communicated past, healthcare were to necessary previously and environment

our Disease MSOs awareness of of activities ago. meeting and approach includes campaign been print many as physicians Catalyst communities. launching significant LEMS patients with with employed program Since as about One well has which made raising began years both this multichannel in LEMS which a our we of visible We've Awareness progress and media digital most treating have in physicians. the three

Our new dedicated educating website patients and both is healthcare providers. to

conferences team neuromuscular LEMS and the two presence help and has national to affairs healthcare the many us medical opportunity our about regional activities providers state. these years to at addition for the provided educate disease past In neurology

spending develop highly a of vendors patient comprehensive Additionally great qualified deal we very to services program. are a time with working

We provide and including and for and support expect caregivers. that physician patient program access, education, will treatment numerous assistance features personalized our patients with

inhibitor. parties Sabril generation GABA-AT for with and Lastly on a generic interested collaboration CPP-XXX, our both next discussions continue

you no as Scientific Chief we Chief will yet will our Operating our Officer occur. Miller, information the provide to advised been over now agreement Steve keep Officer who about While reached, and I development turn pipeline. has more call developments will

Thank you and morning good Pat everyone.

we came have our trial as NDA X for confirmatory Phase C with As our the This successful following Pat positive well mentioned Type as a this Catalyst FDA. very submission quarter was for Firdapse. resubmitted a as exciting

to the our the NDA as both for FDA are well that FDA and submitted does we from acceptability the Firdapse that this not Type by regarding package trial data have necessary preliminary required indicating feel confident our from included data meeting liability abuse Our data C hearing our Phase components to studies from abuse potential. We as X for have look of the addressed filing. forward the agencies we that positive

for Firdapse NDA. indication the This assuming will CMS allow the our This deliberation LEMS and focus include decision and the on well we the NDA to an initially on indications with Firdapse FDA the us trial ongoing to LEMS separately add initial label seek As in the X for our decided we approval as internal NDA to CMS LEMS. submission LEMS discussion our CMS future in to decided have Phase as focus after and a came indication a for Pat our not indication. for indicated of mentioned in to for

evaluating designed X the is treatment that of of this to in indications Firdapse. add we Our half able with and CMS. enrollment anticipate announce an CMS. safety placebo evaluate a titled with efficacy will we Firdapse to and randomized quarter in in NDA already trial XXXX. results for If patients [ph] completing and two crossover CMS-XXX label We trial above submit diagnosed second blind a outpatient the are XXXX successful trial line Firdapse CMS for This to the approved top these first in and treatment the be our hopeful of to period of the results trial controlled years to is Phase double aged

the designed to controlled a safety, with our parallel in small the subtle Phase group receptor and trial efficacy with trial sample well. patients MuSK-MG X, patients tolerability, recently placebo is have known using patients coating of Phase MSK-XXX a in treatment enrolling MuSK-MG X Firdapse [ph] Firdapse. as double-blind evaluate randomized, of trial begun the of This and We as for a MG

-- FDA of which to As earlier is and the with trial trial. initiation this we pivotal agreed X Pat have the proposed prior endpoints mentioned FDA This an have a the that our for trial Italy. design and agreement the meaning sites U.S. with in our trial will Phase reached clinical

MuSK-MG this is to excited about there are that the severe are effective patients a no affects to XXXX approved have XXXX therapies. We and condition initiated U.S. as in currently trial

our that enhancing transmission spinal Type motor or previously affective of junction issue, company neural adequate of in from we proof could muscular study can We degeneration suffering be expect in provide Phase loss patients the As neuron begun sponsored patients second have symptomatic to suffer and triggered relief which also motor study we muscular Firdapse believe and atrophy from X slow results SMA X. receive top and half concept for XXXX. degeneration these controlled of who This a a is neuron well in and mobility. often otherwise announced line

path results with we positive for Firdapse design best FDA collaborate X. SMA treatment development clinical the Type for of to Assuming the the will

program with feels the free phosphate amifampridine to have for patients treatment can options to charge Our access physician other expanded patients provide of access this who and for which continues no treating tablets diagnosed condition. disease turn financial to only over currently the review [indiscernible]. program LEMS, will or Aly is patients The now I call results. their CMS, for to designed our improve

share to this in call reference undiluted to Steve. Thanks All refers shares. per basic

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

were quarter as non-GAAP non-GAAP of first loss XXXX, the loss same as adjustments. net For no GAAP there net the was

XXXX. to decreases compared we in for of increasing XXXX the offset for The for of for million X.X first of same was to R&D period the as an costs, our liability [Indiscernible] to XXX,XXX net drug submit LEMS X.X attributable clinical the change or including when share X.X of treatment non-GAAP related quarter consulting expenses non-cash our loss due NDA compared in $X.XX period same first XXXX XXXX the the was increase million to Excluding the in costs. by of per fair to during value quarter expenses loss million principally XXXX. in March in prepared for Firdapse

our for the of We is our as expect continuing of pre-commercialization due the million throughout are access and balance compared in in increase substantial totaled XXXX administrative to period XXXX first X.X same the we program X.X to will ongoing first to continue when General million of and clinical quarter to that XXXX. to increase XXXX be The primarily of for expenses. compared R&D expanded quarter Firdapse. expenses studies the bound spend

We no million quarter in XXXX for had Catalyst in and including expect cash At our potential we expand debt. Firdapse investments preparation had no of as commercial G&A launch in XX.X fourth in and build and XXXX. or to up the and expenses programs XXXX March infrastructure XX, Catalyst increase expenses operations scenarios revenue of XXXX XXXX. pre-commercialization headcount to our

the with the yesterday this be detailed information no found launching that can can the that in researches back and assurance in receipts company's product an operations cash quarterly if XXXX maybe and we the considering in Exchange XXXX we there on our support now website X, receive in and will approval revenues Relations in believe Although and might sufficient successful filed XXXX. Commission Form analysis obtaining to found XXXX be we are I call and will Firdapse to planned at without of for our XX-Q Pat. be the was Securities report turn Investor and page which www.catalystpharma.com. May More

really made and the about a Thanks strong progress following are Aly. Firdapse we are LEMS. pleased we NDA we've in for in believe resubmission far XXXX our Obviously thus for position

with be forward patients and FDA We submission. of to are will included believe that components look for the as discussions the after We benefit NDA are confident approval it for potential the significant in who all we of have our this from required LEMS. milestone submission for we suffering a LEMS the

to are plan hope we commercial taking what the launch. all company be necessary successful to steps a for a As we

diligently will and option indications rare to patients are debilitating can potential to continue advance we to several our suffering we a bring Firdapse for from other work that and treatment diseases. other We hopeful

questions. apprised we over our other the will operator keep We for year. of the formal you presentation we'll will this to turn as milestones end This and significant achieve be it

Thank you. [Operator Instructions].

the line Please with with of question. ROTH Capital. Our Henry from your first Scott question comes proceed

of for the we'll you even Thank when on priority acceptance granting is something you've morning. commented guess find and formal expected, is don't if good it's the if if I reviews, that? starters, that I out received know you

when that get of at be review time that we it as we standard the get for review. understand we formal will of Scott expect priority notified type acceptance will we it, versus notice

sense of between assuming priority review And need this get you product, of trying the to within do get great. you approval time the just estimate the best and acceptance time would point. actual early much that would a launch when XXXX be Okay, I'm launch? clock final and at expect how get you you

And sometime be we date in of unlikely it to close that for of would Yes, that December do review lot so receive a if would the would just end priority of a be we reasons. we November. the that expect a PDUFA would drug launch very

we're on year. view an point in planning our launch approval considering purposes of from a next January for so And of early based

I with easy to in launch seem don't the program of final the any but that size like it just know and to a guess expanded program, Okay, that's would I program expanded could that patients color in great, of and you. be drugs. could comment question relatively group launch? regards being that you've around Thank access create a you on the convert of the access just to get a sense how to given expanded the from being more would on robust access trying if

a you agree definitively good talked as think about number Yeah, our of of launch. serves bolus the for patients with we we access Scott patients pretty that in expanded but program we haven't

we hopeful advance be will in working approval approval. so And with -- of

be to that bringing on launch. as board program we that make case our actually part will robust do sure managers of We have

you Okay, thank taking the great questions. for

Thank you Scott.

Thank I you. the comments. will no appear turn for be [Operator time. this back management Instructions]. further to closing floor questions There at to

you. thank Great,

year to thank for everybody that anticipate again to participation exciting We their on this Goodbye. continued morning for you I'd like support. shareholders our to be will XXXX for call. the Thank continue us. So your again an

teleconference. today's concludes This

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