Catalyst Pharmaceuticals (CPRX) 8 Aug 182018 Q2 Earnings call transcript

Greetings and welcome to the Catalyst Pharmaceuticals Inc.

Second Quarter 2018 Financial 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] As a reminder, this conference is being recorded. to Chief conference Financial like host to now Grande, Aly Officer. would turn over the your I Please go ahead.

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

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

everybody. joining results today us morning for and our Thanks good call. XXXX and second to Aly quarter you Thank welcome

will on financial Miller for pipeline for to activities review provide pleased and on We are Aly second report And quarter. results developments. recent update you our our lastly the Firdapse. will of our a status milestones Steve development

With us the well on chief will Officer, Commercial Dan highlighting from we activities. our activities. with as our that, will expected join take second you calls, let me We quarter by future the takeaways recent provide about start commercialization your key updates Further Firdapse Brennan then questions. to new as

report is in of symptomatic to such application that by had for treatment are the review or of the year, syndrome effectiveness improvement accepted new to quarter has provide myasthenic During the significant that safety this prior treatment the review Lambert-Eaton granted Priority we pleased for condition. FDA for second drug Firdapse LEMS. for our the FDA both or as address the a of of Firdapse the and potential drugs for

them We are review process assist working a we of year. to goal with have XXth and in closely date of FDA the PDUFA November received the this

with Phase review currently endpoint. which trials and submission co-primary significance the active patients previously both NDA the trial announced in of achieves results November III is the LEMS. Our for LMS-XXX statistical secondary endpoints positive and our includes This Those confirmatory under from XXXX.

our continuing and other Completing X. next rare the ensure Preparing them LEMS all NDA for evaluating positive and review CMS myasthenic the underscored the that launch to patients NDA, and for patient spinal early access clinical for MuSK, acceptance Firdapse ongoing expanded Firdapse congenital myasthenia with atrophy the and type to all of access treatment program our indications is have the currently now With enrolled gravis comprehensive potential review in antibody services other and FDA year. syndromes, neuromuscular who focus with assist closely of Firdapse Pushing muscular a potential convert quickly for Firdapse possible plans to trials exploring FDA disorders. as our our forward working for as this of process patients on of program. the

diseases. XXXX. Firdapse experience focused companies Dan Chief neurological as has prepare recently ones Commercial As for previously for Dan sales, and announced, Brennan appointed efforts leading of in particularly on Biopharmaceutical potential at Dan Officer. marketing responsible leading commercial we extensive we commercial operations rare is of have early as launch

his utilizing number have We for key of launch the added therapies the of knowledge and for Patient treatments rest Director President Sales will disorders. same launching Operations the are see rare excited an Monday, our the Services, in to and rare a has senior This leadership These as of several Senior and neuromuscular Catalyst potential team we for extensive look Firdapse. of forward progress integral effective the bringing Dan of made our commercialization Senior of and to we to President passion Since disorders. Vice of have experience him shares our in Efficacy and Marketing, joining suffering Engagement. in on-board Vice Director therapeutics of already On us includes, commercial for which safe great announced Analytics, and Senior team. patients from building of of diseases. that June, Dan be rare positions Patient leadership neurological extensive commercial Senior talent operation, Commercial part plan and has

physician three Senior Affairs recently wealth within Additionally, on neuromuscular hired have Medical Science of currently to community. additional MSLs of a brings our we This working have number the Liaison five. experience and Medical Team that a

as approved. Lastly, who of that manufacturing services have to patients finished have assure plans to we access our patient our part all program, Firdapse assuming

positive once the developing are assist To begun our ensure are have we the patients launch extended also transition program who in and we an transition already the with access to experience, is to We commercial the to requirements and that the smooth easy Miller, a product Officer to progress Officer, Steve our program a product, information Chief all over provide will who the expanded of turn patients. supplies call meet Chief Scientific programs. more adequate development commercial Operating of product. about I our manufacture will now of these

good and Thank everyone. you, Pat morning

As LEMS date priority and PDUFA this symptomatic NDA The the under NDA hopeful Catalyst be active FDA Pat call that time. was treatment is the approved on review was of or about NDA review the will remains this mentioned, of assigned was XX date. granted by at of the for November year. accepted and this our previously goal

our did we indication conducting Catalyst an Phase limited trial March evaluating safety currently adult continues to syndromes or for of are mechanistically that myasthenic half in of CMS both the filed to NDA Firdapse is not in with collect only submission LEMS include top-line CMS in and and for Firdapse data Enrollment in patients the the announced, the the work CMS congenital and subtypes similar LEMS. pediatric order As and we that to for for we diligently first the efficacy ongoing previously of CMS additional and was in trial patients. X results anticipate XXXX. treatment

CMS CMS top-line to label the such a results, include Firdapse in to the of successful the a content of pending with outcome Catalyst NDA clinical to as structure indication successful general Catalyst for of hope to the supplement the meeting the discuss Assuming indication. amazing well suitability results, the as additional supplement anticipates filed agency a its

look include an trial Catalyst We has a proof-of-concept We MuSK-MG. is early the that continue of half evaluating under with trial that our being in a SPA. hopes and previously X that the forward conducted second supplement way in Phase and the Catalyst hopes results its is previously gravis enrollment safety adequate This for provide NDA. on the of was confirmatory it of successful of positive expand indication announced already future the the in to XXXX. is treatment special This clinical of also confirm in or and continuing to myasthenia protocol trial that trial confirmatory efficacy for hopeful, X together Firdapse MuSK-MG patients expect XXXX. trial results reported top-line the positive Catalyst to label assessment will trial Phase will study MuSK or enroll results that Firdapse by approved is reported to FDA proof-of-concept the evidence antibody

reported, myasthenia Drug previously Designation been Catalyst treatment the As gravis. Firdapse, granted has Orphan for of

in approximately initiated recently we patient atrophy to SMA conducted And Europe XXXX. top-line in dose the on half patients. have enroll results second be of This forward trial spinal or trial X. expect of type this in We a to from trial second We and the half expect look of XX muscular announcing to in will proof-of-concept XXXX. Firdapse first

relief In to tablets cases, Expanded of provided LEMS that we Financial amifampridine the now to which to these to our could tablets phosphate amifampridine continue results. Program, As charges, phosphate will with Chief no their are have Officer and support other provide I and patients. treatment patients our their physicians the Grande, financial Aly your over Access option believe review call patients to CMS. if turn provides this free treatment

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

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

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

quarter due related our Non-GAAP in million the Program warrant. the and submission quarter currently Expenses XXXX. change to liability trials headcount compensation net was compared share same X.X consulting and expense, XXX,XXX NDA $X.XX with of to was XXXX. personnel Medical programs. acceptance of million gain and fair in non-cash The of the expenses for our for development attributable a XXXX million XXXX. X.X the per X.X to of was period classified for value second Research in to loss second increases expand our ongoing increase as same from principally in in our we to compared period expenses Excluding XXXX R&D Affairs connection and in costs, when in milestone support the of May or

million the research trials when balance that up X and in completing XXXX. that XXXX and million the will we and Firdapse for of continue the this and costs programs type and primarily infrastructure SMA Firdapse XXXX. to corporate administrative commercial during LEMS. of XXXX Expanded and trade in expenses XXXX potential program quarter expects recently in development MuSK-MG in quarter X.X as expenses we the be expenses, preparation second headcount and in is process as Access The period Program evaluating off Firdapse due build compared nation accepted towards pre-commercialization to to for substantial with all General the are to for of and increases for We increase treatment CMS work development launch second X.X same totaled compared our for continues

and to cost cost the continue will G&A no compared no quarter cost XXXX of to we of XX, G&A the set $XX.X or our have expand in in a continue the debt. had investments in That second for in million We neither cash XXXX At XXXX. incurred pre-commercialization with as always in preparation have XXXX, increase Catalyst XXXX operations potential revenue, including XXXX. June launch

found XXXX these will if be can to cash our [indiscernible] be related assurance no we XXXX might launching there resources through and that in More in and detailed are be on Company's Although Commission to we Relations information with can support and we planned yesterday Pat. successfully updating XX-Q which when to the product was received our on I on our back revenues Form XXXX. operations without comparing at sufficient X, page received Investor quarterly www.catalystpharma.com. analysis report the now in maybe the website of call Securities turn found Exchange XXXX August filed in will the and the

is team of Catalyst The NDA highly motivated Aly. a acceptance with receipt Thanks reviewing. prior day its of the and our

are and LEMS community therapy. and treatment FDA our our of the to NDA their review the Firdapse closely potential of forward of We the LEMS FDA approval assist and approved look for to working them in with treatment NDA we for bringing

We are for about the making Firdapse we also in indications. are additional pleased evaluating progress

are commercialization diseases. for therapies the these and and hires rare for to leadership grateful neurological commercial excited Finally, have our bringing new are we expanded for team activities experience

FDA of confident the Firdapse we are launch commercial that approval. for steps a pending taking are We for LEMS necessary successful

for also business presentation forward over development turn will pipeline additional the look activities available. call any to providing We with our This we they to questions. and end formal become as and will operator other on update the the you

time will go [Operator Thank you of this be today Edward comes session. first Our Robinson we SunTrust ahead. a Humphrey. sir. Instructions] from At question-and-answer question Nash conducting Please

welcome see have And to you Thanks But so you to (Ph)? [Adcom] about getting to discussed Nice guys that to team. PDUFA could an have now the you Dan how your before. see are my start still that? Pat, onboard. Steve we you won't I for I call. maybe know this much a Great. taking [Adcom] to I thoughts there are be on little as we or - bit Do maybe do the wanted - closer or what want feel

on is believe with the be don’t an is and Adcom to communications that FDA. based there We the going

So let’s way. it's it that put unlikely

what both? timing you for can regard to the in the us with then the could remind and patients you number the update appreciate per And us for just again, I - CMS points end remind Okay. the But trials. that on the you just enrollment each primary the target of MuSK-MG

Steve, like take you would to that?

Sure.

XX for gravis For and Function MFM and The And the patients. is enrollment enrollment is the the CMS trial, is daily the about Measure, Motor of target MuSK-MG endpoint activities SGI. trial, the is XX MGADL myasthenia target or primary endpoints or living. patients

the And this said start second year? SMA they great. in Okay, will the you half trial, of

Yes, will. it

will or and could based you a Okay that positive data that that is data there that that on to accelerated approval? would to still get so need that assume trial from second filed study, you trial. on based do trial I move chance any be for potentially on

we believe will disease. not assess treatment design SMA include would Firdapse The of No. duration whether progression that second or the actually does long not of is that require enough reduce the that trial. a trial of to the

findings? CMS the would NDA then in MuSK-MG Okay. off. I or be last regard of supplemental those the indications to jump supplemental And question my filing, both will With

That is correct.

that indication. the that Any NDA be FDA indications being is already would review only currently NDA. we LEMS by supplements approved the Our new add to for is

Okay. guys much. Great, thank so you

Edward. Thanks

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

good you Thank and morning.

question First Pat.

Can risk a of kind filing? that that of that to Now Is terms bed Firdapse puts accepted. them we has you NDA being does a that beating the jack Is by assume, clear? to now whole accepted? bus in been

technically, Well, believe is it don’t Scott. I

are again, think they that a company. held privately I

are know don’t in where - they they all we are at NDA. So if pursuing

your set And not so until match. approval really receive you it’s game

it’s the there from they only public are really much and in on and is again KOLs and calling we experts. the there about is who The anecdotal LEMS So get year very MSOs information where out are. this information not

we the expect will and we ahead approved so, PDUFA And be moving are that we by date.

expensed the we model, then we with Okay in And may great. fully QX it How to I regards QX. about in should that Thanks amortize. the be of will for clarity. the gather that or is that I see milestone X.X million? thought think million X.X possible

with renegotiated shareholders to the the the in XX-Q would parties the Scott who actually that [indiscernible]. have to We are license who [actually] (Ph)

changed. are so And have milestones laid And the renegotiated queue. those the in that and detailed out

And so hit a $X did that and pay --. million did we

In QX.

to QX amendment occurred a we signed expense. reserved an Even that is when and That it agreement. though

milestone Yes upon NDA. if the you are referring the approval of to

We will reach…

talking to is $X.X million. He the about acceptance

QX. of recorded Yes. only And portion a We paid that. that been has on

Scott for us. renegotiated favorably been It's

Yes.

then at look much a the much at Okay. would see how XX-Q. the selling that Great. selling I as G&A, the of color appendix as And the far I in take is you of approximately did will at steady expenses that any end. state? sort And XQ? how on see

I fairly you that I Yes. that's Scott. know but don't from you expected that [indiscernible], have

we will G&A.So expense. was have X.X, $X.X expense G&A that once X.X six the have of out expense. we So the selling selling the that outside to Of million shift for that we you number. that approved backed in million months, then $X.X in not were That got and selling if

taking for questions. Okay, you the great. Thank

Scott. thanks Okay,

from comes ahead. question Gold] Please next go [Arrie Oppenheimer. Our Instructions] [Operator (Ph) of

taking Thanks this on question. Arrie Gold for Oppenheimer. at is my for (Ph) [Levin] Hi

Hi, morning. good

you drug to Good are legacy assets. looking understand morning. your monetize I

how Just wanted been progress on that to know front? has

report don't any to We this have new at information time.

or and likely been seeing The we with making it a resolve a deal while, the it. of generic this to front. We fairly for makes we very then progress on that Sabril over that the we are a will it that we have be monetizing to of partner the a of great license monetize think currently as do future. least And other in And that near position Northwest are asset we in one regulatory bit in a more we our are of hoping we but dispute those amicably little at assets. close are

Thank Okay. you.

questions to to There are over further would for Patrick I this no closing McEnany time. like the at turn call back remarks.

participation in Thank morning's you for your all this call.

for thank as pleased balance with are an support move the you. XXXX our forward us. and the year with anticipate shareholder progress. achieved Thank you be progress And have We again we of for to exciting we your

This concludes conference. today’s

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