Catalyst Pharmaceuticals (CPRX) 11 Aug 202020 Q2 Earnings call transcript

Hello and welcome to the Catalyst Pharmaceuticals Second Quarter 2020 Results Conference. At this time, all participants are in a listen-only mode. [Operator Instructions] As a reminder, this conference is being recorded. It’s now my pleasure to turn the call over to Ali Grande, CFO. Please go ahead.

welcome conference and everyone, morning, to call. today's Good

and are the and Ingenito, Catalyst me Operating Executive on Carmen, including Chief of Commercial call today's Officer Chief and Miller, Chairman Joining Medical team, Regulatory Dr. Jeffrey members Patrick Officer. Dr. Steven Scientific Officer; Chief Officer; Officer; Del Gary Chief Chief McEnany,

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

during and healthy and thanks and call. update business I results unprecedented for morning staying hope times. quarter safe Ali Thanks everyone second everyone for this and joining our that is challenging us these

million million and of managed in quarter that net conditions achieved. difficult million. non-cash items pleased expense $XX.X are environment morning. $X.X will under we how a net I a revenue generate results we included number compensation this $X.X the were of second stock-based in of the start income We very to which COVID-XX update to operating on you have Despite with with financial

more of team's financial quarter want steadfast how equivalents debt. minutes. few number will continue or details cash we to have products with resilience LEMS COVID-XX a to a I ability on the impact I That effort received diagnosis and continues demonstrate the million in from their shown in number to we to physician. for of Ali our of their LEMS to in their Additionally, am provide and not ongoing with patients patients difficult the funded have their concerns no the Catalyst you make a They from ended $XXX.X emphasize remarkable that meeting enrolled who in-person cash expand it benefiting their serve. an to may and commitment about with of to condition. physician treat schedule to patients had patients inability because and have has definitive proud pathways.

restricted that than we solid to as and offices mostly at albeit said for representatives base pace new As our physicians’ we be patients to pandemic. the anticipated of Having the intact continues we LEMS projected a adult our pandemic. have sales previously slower travel before by patients remain add

and to add pleased pandemic COVID-XX to commercial trademark a double you the effect We that received we are are morning behind were to When we field LEMS. future inside assist that beginning I when six our based methods June provide our this The force the for Catalyst issued of to that believe fee us by should U.S. of dimensional we for details This year would the patent Upon of XXXX. patent patent issue support the extremely and will patients could company in the more Jeff resolute patent sales be physicians patent suffering number to in We will of administering strengthen our within This patent an be to allowance with than operations see are at patent reinforce from expiration patent earlier patent this expiry momentarily. pyradine. brand the has also marketing this decision will and States. Firdapse office pending our announce extended the for our our date growth. after no prosecution the from been X-X well-positioned on will outreach the notice of extension if on yesterday XXXXXXXX USPTO of issued. calculated further the our issue XX, for delays United if exclusivity and of any. any eight have of payment in team weeks. This

enhancing phase We've statistical positive with clinical of programs, findings treatment when We Steve for efforts advisors at LEMS efficacy currently Italy. development continuing proof are add on our patients will decide bring we MuSK-antibody what we did to of a conducted activities final our final who Firdapse to our patients path property to potential further with R&D this portfolio. MSK-XXX program. Firdapse line stakeholders study has benefit achieve obviously positive the shown output to significant results any other forward we and all these and replicate and study neuromuscular have disappointed without myasthenia our medication he not will With our this be neuromuscular received suffering are to today concept reports on and continue patients. seen robust conditions Catalyst by rare and regard very of more the to recently Based with for are our will analyze approved as attempt gravis intellectual various did in just continue other programs. value our X to will differentiated to development our data study for on much-needed treatment going to this our therapy. add that not were efficacy results data a that in top unfortunately review clinical the and

we well-positioned this success sustained Chief launch Firdapse unprecedented access also the prepare Firdapse adult of opportunity U.S. to patients. through leading Catalyst of we community we time. execution for was well ability operations of his to of promoted year rare of patients reported for LEMS Firdapse LEMS sales, as team responsible the leadership as Last XX% for successfully week that as is our Firdapse to commercial launched have of from Jeff Jeff last prevalence our that Firdapse treatment about announced Jeff's the January treatment Canada. and United States. other Officer. the has the treat We patients that Canada within marketing by the Del LEMS previously leading to for proven and sales Jeffrey LEMS by marketing As neuromuscular in marketing as in continue for and is Carmen potential diseases. believe expand experience, continue is authorization Health in suffering Commercial launch commercial other approved we With

drug Canada Health our footprint are while about with We a for global LEMS our neighbors to excited expanding providing patients the approved North.

our approval We an pharmaceuticals I for an approving drugs in future. in we Japan and to discuss are approval and and patients. finalize efficient to gain very with with the agreement responsible PMDA, have weeks for and marketing program regulatory agency Several near final or an reasonable partner medical Canadian established for agency distribution to ago in currently expect in the LEMS device regulatory to agreement Japan. the discussions met a gaining Firdapse and we that the of believe clinical requirements

on will several more in marketing program say sales have partners a and Steve potential the We territory. little bit are to with shortly. in that discussions early

Firdapse. with orphan imagine, you filed and As magistrate drug despite can in approval FDA's for we're lawsuit the the to our report exclusivity very authority our by disappointed the recommendations Ruzurgi challenging of

no want stress I however, is decision by report context For lies and to magistrate the final that of dispositive. district with judge. the means the The its court recommendations interim

further on to now still Carmen, in Jeffrey later Commercial legal Our the you recommendations paramount on being our will we in has case to are Chief that to who just next final next be and will update expected at our Del commercial this with focus In been expect work an year. words as step to this hard file the this our our on objections report given I our in an us decision turn the the the operations. the provide and process We the and step this issues opportunity sometime provide call filed week. attorneys process over has other case. in and to document Officer see

and and continue good team I'm Catalyst very success we've Thank the morning you everyone. about Pat excited since commercial the lead launch. the had to that opportunity

business. As commercial we previously of shared COVID-XX has impacted certain aspects our

face-to-face However, we drive of healthcare mid-march and continue the professionals. to safety ensure forward. our of to we business interactions the patients our In of team, announced travel, our all all key suspension areas

out visits. still While to up the seeing anything about some have areas other open been to office LEMS or to emergency country of a be care. LEMS offices starting rightfully worried physician appear venturing than we're greatly for reduced patients hospitals number of patient

patients in that LEMS two have weakened disease reminder factors making they over of often separate risk the age COVID-XX immune are them a have XX to and systems. key severe As susceptible

I'm significantly have necessary the XXX approach our community. QX considering well say everything of patients’ represents Ruzurgi availability XXXX. sales achievement While commercial that patients have LEMS professionals we to to to obstacles X% This the have adjusted was of increase quarter we we willingness second since over to over and an is net in time a as of $XX.X Firdapse significant the net the XXXX continue XXXX launch. flexibility that requires our sales during of prior a million caregivers QX as to our prescription now that pandemic. proud received healthcare Despite do support COVID-XX and of support these

since in While compliance have strong new last see expected to The and the Ruzurgi discontinuation QX, end to positive and has XX% trending significantly. persistence. the slowed slightly lower of to continued rates of discontinuation XX-day improve quarter from around patient we than we continued enrollments were January trend medication

feel despite I'm enrollments early significant discontinuations are very we demand with healthcare rates the of deliver patients since will levels, Firdapse second to discontinuation on to half that Also are team's yet able July. strong our approached June with will extremely we is in be prescribed diagnosed fact, performance low remain the ability patients total monthly we adult in new the these the their remained and of not faced. this pent-up observations on quarter, that pleased specifically in continue Based believe adherence Firdapse our once July, In result XXXX. compliance visit and that In of were high challenges pre-COVID-XX the April patients third LEMS base year. among are commercial the provider. existing lowest patient there that

pathways rare responding patient Firdapse of supply that patient community patients We for team patient needs has remain disease concern to that our tremendous to doing approval reiterate one to without and we commercial this continues across Prescription during LEMS government not in this patients and A the are do services happy everything our support XX% medication. rates private to period. Catalyst around all physicians report possible confidence job insurers. a Our have at pairs; gone or well-documented our time.

Our co-pay assistance foundation who have full average programs financial month. and in Medicare a by than in force. Patients Catalyst accessing enrolled less support are $X covered of including those and an co-pay pathways continue

happy has increased that Firdapse We of transition are the COVID-XX or number on Medicaid also significantly adult to not patient has to that needed assistance program. to see our LEMS patients

non-personal medical doing company pandemic levels both valued access an by that communication for in Our second healthcare focus the account community. our This the inside professionals expectations. to LEMS lead half as XXXX. posed resource Despite liaisons promotion the will healthcare community patients. who confident team expansion, being effort and a growth on force always for patient successfully right generation to customer providers the our and how field managers, sales job We and exceed we excellent liaisons updated and regional we and remain of for in expanded have further ratings done service continue the challenges strategies, are the commercial LEMS science validates and a what's position

Operating now R&D Steve? Officer adult challenging to with times on Miller call activities. While making Officer turn Steven is team over I LEMS. all to and accessible an update will Chief our these patients for Scientific our Dr. the Firdapse are Chief committed to

for on I clinical our pipeline indications. Thanks Jeff. the neuromuscular an update will now to commercial develop provide Firdapse update additional for

our Firdapse the announced treatment we Firdapse to treatment efficacy X and trial trial Yesterday safety, the patients. for MuSK for with phase MG. the MuSK these MG turn to from our results of X results will potential I tolerability evaluating First clinical clinical MSK-XXX symptomatic top-line of phase of adults evaluate for

MG-ADL activities p-values top-line for was Myasthenia endpoints As of not score Gravis trial our in of for Myasthenia Gravis enrolled for Serbia. previously based of quantitative Pat international respectively. or XX antibody trial these trial antibody stated of endpoint score and It statistical were more under of with multi-site was the on X.XX a Myasthenia review the QMG AChR-MG it primary same clinical The trial United This more the data a was with living receptor the Gravis secondary achieved assessed conducted daily patients. conducted or patients assessment each double-blind of was achieved significance from endpoint also the protocol trial nor special endpoints. enrolled the than FDA. or anti-acetylcholine who positive and States, placebo-controlled clinical MuSK XX Italy with than It X.XX the

the not statistical patients of achieving significance protocol. However, required was by in this subgroup

the seen a Like patient in we LEMS the were improvement ultimately six-week these considered MuSK MG are during a positive Firdapse more this note improvement period or MuSK meaningful. proof Additional disappointed -tolerated A and clinically by scale on in randomized X.X was surprised concept run-in to the points results clinical and given safe is randomization. even population. population of two-point details that patients the showed of MG-ADL On of the trial clinicaltrials.gov. study. MG the the well results on available are Naturally prior

So us during the of study decrease MG during improvement a clinical extension continued trial the clinical from who a review significant study randomized in XX condition into XX open patients patient's the represents to effect. the data unlocked open indicates and to MuSK continues trial. durable run-in extension the this of period label preliminary MG-ADL in the participated label a significant Further of improvement demonstrating the that seen run-in the that

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of by to provide broadly the Serbia lifted last for the study update and and progression SMA restrictions Italy is anticipate The type in reporting the and delay type an more a of one perhaps disease Type to to enrolled in the concept line study SMA of characterized through will the is ambulatory note four. Catalyst year. SMA each crossover in is initial the the from phase with which symptomatic actively trial related our to to type X,XXX clinical SMA of time between been patients FDA are two on not is in study trial a This and that X ongoing and and which is treatment week the this both motor which has proof variations of genetic of caused study a of neurons. to discuss have safety, second trial, potential I SMA is in treatment severity in we intend than same and treatment like progression. four important this genetic protein concept study types SMA SMN cause address would participating study SMA tolerability by and evaluating changes efficacy restrictions It various designed period multi-center protein gene as is an proof estimated and concept successful defects now amifampridine design patients. measure end patients. COVID-XX those X prevalence is but the we design are for top in If disease of all to of Serbia the by disease in one proof codes X but intends X defects resulting X,XXX the in caused the results are SMA. of in did in SMA and and starting SMN symptomatic a the with Types symptoms

in near expansion MDS product market to Firdapse, August for this symptomatic Moving of LEMS year. Catalyst anticipate approved in and We future. labeled on of having ready early was the treatment completed plans the Canadian Canadian for

at Catalyst the pharmaceuticals registry with Canadian national that Firdapse. the continuing designated priority meeting the seek Japan. Approximately process Firdapse the previously ministry fact the approval this pathway details now drug need seeking can drug. was they is drug and reached medical MBA welfare they As this subjects priority discussed develop to of Catalyst activities a date multiple long-acting to have Japanese institute the of reported their health will report market. trial expansion path distribution to ago doses previously a in requested version website. the activities will on the territory approval clinical of for have have of to approval labor in plan eliminate the and is health's this trials helpful Firdapse formalize as the a COVID-XX soliciting Japanese completed in regulatory Japan of cooperative been of order registered on public an daily to Patients that of to delayed Firdapse agreement actively Japan. or agency also three years to a and be of MHLW in Japanese when in Due partner and the around to tentative been market regulatory for for of file the companies a Amifampridine conducting and government Firdapse MHLW to in was device the as in until we have of available and made small marketing a is have market meeting I actively PMDA and a our be but or that a Catalyst trial Japanese seek future QX clinical

optimize provide evaluated will order been such have others. and studied and as in in proof-of-concept being have properties characteristics are to additional future also the program be studies development HnPP release of to Kennedy's updates developing been the candidate when treatment to neuromuscular the future and product for neuropathy formulations possibly are or their LEMS. near long-acting Catalyst stage now the of finalized. variety the disease, actively this new liability pressure in palsies be We supporting At symptomatic investigator-sponsored this Hereditary we'll with conditions of a product drug developed

timing. design, plans the finalized provide more As the these trial, about and for are will trials Catalyst prevalence disease, details

for Moving X,X-Diaminopyridine. on disease pending method to to claims treating sensitive intellectual slow gene. allele diagnosed having the of property, patent X,X-DAP methods with our patent human a X,X-Diaminopyridine This of patients Pat recent administering of the allowance described a by patient NATX acetylene certain in mutations each administering

the carry research MG, a on Upon no MuSK had uses first health June that of and that on to In [patent]. applicant protection generic emergency there with R&D development patent made many come. be do orange patent protection this new an minor listed being expiration the that delays no pleased they FDA on patent at ago the SMA approved in we to this was of period the to patent are XXXX long-acting or the for finally our was little We been and for product applicant the believe the discovery generic effect XXXX. Firdapse the product our Firdapse This drug FDA's by the years equivalent the issuance validity franchise. production. the provide infringe XX, Catalyst date has Firdapse extended programs by COVID-XX the may in will will until product the the listed of additional with improvements programs challenges book orange can commercial team Catalyst permit for least of Upon to intends effect years not listed this book successfully unless only and list allowed and the Firdapse half prove of

to anticipates will academic resumed being are the two this that However, to be that were programs Despite these restrictions fall. programs Kennedy's institutions now HnPP investigator-sponsored by and delayed remain neuromuscular COVID-XX disease impacts on our due and studies lifted. crisis challenging health timelines months several are Firdapse related Catalyst our indications. due the for to and we extremely had the evaluate current the committed having to other is has that environment programs research global

has the patients. a about supply of for for affected in ongoing COVID-XX. and been additional current by clinical to all expand into opportunities are countries to well excited develop patients additional current product Firdapse our Additionally, LEMS Firdapse Overall the as and indications trials these label better not for we as with

We any turn on will regulatory our as they become Financial the Grande results. paths now provide updates Officer to over call available. Chief to and financial clinical our will review Alicia these I

Thanks $X.XX income to XX-Q Steve. second reported period undiluted $X.X form same per our million the or and of GAAP per quarter share GAAP in compared basic filed basic income million and net of we or $XX net Yesterday XXXX of XXXX. share $X.XX $X.XX diluted per

the quarter per diluted $XX.X related second for non-cash income non-GAAP subjects and million. second undiluted basic of of per was for net income with was expenses excluding of XXX,XXX cost compensation to of $X.XX of to expenses quarter million $X.X net the For basic per or $X.XX $XX.X revenue non-cash million related for product Firdapse second of sales $X.X XXXX million non-GAAP Net compensation was million comparison excluding XXXX XXXX share. $XX.X In related share. $X.XX quarter stock-based

were XXXX for as of development line XXXX $X.X second quarter second quarter the the programs. XXXX. million quarter Research second for Research compared product net our to quarter of and with important in gross from quarter development those costs primarily Firdapse the related $X.X XXXX our of Firdapse. with second of expenses of the in and manufactured million from periods affairs benefit revenue expenses prior expenses access studies approval remember trials that ongoing continue to expenses inventory of XXXX both to our and and million. for the quarter was $XX.X Firdapse FDA clinical and of in costs or For from for the and million were of the margins and programs the assurance XXXX It's XXXX regulatory medical second expanded to sales and consisted $X.X of quality for of expense the

diseases. long-acting substantial our when of in for of quarter XXXX for $XX.X additional the of Type XXXX and rare expansion and for with We $X the the our Firdapse the MG compared related access the of treatment costs MUSK began will compared second Japan be increase continue database million the of research attributable quarter expanded our XXXX. as agency. trials a continue same the and our in SG&A ongoing cost primarily neuromuscular for expenses expect including XXXX that second we evaluating continue of to and development clinical X the to sales studies The as We program to in and experience to and formulation million clinical personnel in other forward totaled quarter our first to required to sales treatment in Firdapse period began in is force in move program non-cash Firdapse. compensation, supporting with a disease of study cost contracting LEMS XXXX evaluate SMA

we continue and to investor relations as FDA. page with commercial no which form website in continue debt. quarterly More to exchange in in funded June and www.catalystpharma.com. of of support lawsuit the XX-Q on our XX, commission the securities and detailed be cash report sales found filed investments will was substantial August our expansion for our XX be continue programs XXXX expenses $XXX.X information SG&A of pursue and at and and on yesterday analysis that found XXXX infrastructure Catalyst million and Firdapse had may On the can build be patient company's to expect We the our activities and against the of

call I the Now turn to will Pat. over

reiterate continuing how team during am to way treating adult XXXX. patients their Thanks Ali. the I that disease. find this pleased want an to therapy forward assist for has to our realized We I approved their physicians LEMS our look debilitating and operational just to to FDA with results effort

our well We conditions for will our very invest conditions. that as as advancing as without an therapy as of effective pending important efforts long-acting for on Firdapse Firdapse formulation more rare Firdapse. neuromuscular of regarding other This rare is our work patient-friendly we study are therapy notice the to allowance a for continue morning's to continue patent other a announcement in potential neuromuscular

approval to are see Canada footprint umbrella. effort we to gain we'll efforts to and/or line companies prepare for And and excited in with that well other the we our Japan molecules approval concerted Lastly under questions. as in as our open continue the to Operator? seek expand our as global bring Catalyst

you. Thank

be is conducting the coming will Charles Instructions] today Duncan [Operator Our question-and-answer from question session. We now first from Fitzgerald. Cantor

Your is live. now line

Thank you. performance challenging Thanks questions and taking the on Good very Pat our for team. year. this morning and during commercial congratulations

Thank you Charles.

to ask to a deconvolute then regarding and action pipeline but the challenges of that With and quarter commercial side then impressed wondering versus could one wanted questions impact bit you talk the about a in the little follow had I that up of in sales on and given the if couple to of there COVID any pricing I the the new you with force. patients on the future. persistence size revenue net I'm and the regard the of was

Jeff, you Sure. that? want to take

problem. No Yes.

than or physicians want enrollments side patient that we diagnosed were if focused to the go giving positive has city new had but virtual the and for leads institution face-to-face yet on or a were and is but patients lower as we're they but far numbers exact slightly not out really strong the that forecasted analytics on not Firdapse team already we we As out quarter however us offices engage, the with force and live physician are allows. those that that our to sales ready engagements discussions here there with goes we that and in provides those have

get discontinuing. the are discontinuations about far Charles the discontinuations day as as we help to we've those enrolled been stabilize where seeing identifying the and is and those that's we're of XX% so the that I And patients now, patients by patients mentioned able patients like XX significantly

as had on a strong So well. that we month, side quarter

early what me had in add that let when the year naive gain to there been did Firdapse. second continuing a Firdapse no challenging an know change and know year say and we you predicted our did have this QX were had to results albeit QX we call pace regard are enrollments patients no QX there going guidance was no new Charles, therapy FDA without price that. new approved to in of did having and in in pleasantly and when at access we patients not the new far just year we guidance pricing and but We so didn't was quarter we there's QX, in we the more actually that gave grow with rest that surprised patient in of quarter specific is as or just being withdrawing in patients to was we're the happen because

Pat that Okay. you really all helpful, I to That's the shared. appreciated metrics Jeff. and operating

year prescribers there it do where Regarding and prescriber sales now going right really a to of results being impact guess was the see be nice like it's the like or you you to half we is out I the fully second operational interactions? force of the perhaps XQ talk and size deepen the feel but to do the feel size to get new them or see able force, sales

Jeff?

managers that is have not We first demand we patient making sized thing ultimately gets healthcare inside we account whether it's that question is sales, how And promotions appropriate experienced pent-up for but it's find the representative, feel here. that providing still and whether that to I the call the Charles I'll impact managers value seen mentioned account sales expansions. patients or the right Firdapse that leads, are the the full feel that's fantastic ultimately there regional reaching out a reason we and this why because enrolled. to hired is from of we from is them providers like non-personal it's and such are and do they and we our if discuss regional stress

seeing up. once yet feel right-sized will are because this full that team we and future the So the impact benefit. We have the we know excited we are opens to the about the see we team is have country

all March account that and assists with actually managers that that our an add force. size ban this a in Let so support I doubled our marketing no our and happened with the me year brought March on sales board basically just should February in early to RAMs, travel team of know travel as the and regional inside we of ban what early or was you that We know say. we field course completed Charles,

regional very so patients and they've want team finding the it's because because challenging me you is and or tell telephonically and to get so most challenging on or meetings been been And exciting want difficult have these approved this just therapy assist to been let yes out there is in Zoom they account been and part an place. the what's it's and that seasoned virtual Teams for managers

As things team. on that by support see gain to force turn bringing marketing around sales doubling the and we really the we're that traction going board inside

If an about a could of there's said of I'm during the study. I or design I part of given improvement and question well makes as in you MG result withdrawal thinking protocol either think the run-in MuSK a the average phase little I'm five-point of one it sense. ton first pipeline just That the a ask on Steve for randomized that surprised Gary. guess the and I

an perhaps then somehow was the that potential the any been So and being look that drug trial a you've result I you result what to identified if you ineffective patient would heterogeneity wondering about or I'm of you've guess data patient-level not as think but as confounded next steps cause if to see if able this at latter? outliers the a take would result and of

Thank the you for question, Charles.

a you only line that ago. but of yet is the run-in the clinical statistical received data top trial. out somewhat good paradoxical appeared As be the This few is improvement of during significance in data lack a randomized days clinical we've portion to what point with

We time haven't had patient all detail. to go in the through data

Our a among try program both is through our deciding ourselves it work in we leaders leaders of exactly and with of for key is. opinion our clear what us be get understanding and once to key analysis that analysis on trial we ongoing and once also this to opinion the better understanding a will of meet ultimate we future what part be the will happened have to

or patent feel investing claims on perspective key particular highlight you the do perhaps of but terms don't overlooked change if that IP. for are will a wondering particular I've about Firdapse and all kind the franchise? you our has diligence question little in of that anything does Last the surprised it this on could you I'm ever we've I sorry the congratulations think what that it in that in Pat, really about that past Okay. about resiliency some because talked I'm challenges your for in the questions, new in any and

be regard how we we by So than particular high allowance the any With will of other you patent can't or maybe in a strong look that affect competition. now is any program can enforceable claims the Charles, to our on right give notice potential patent and it level way. claims that the company listed Steve believe speculate may against are generic very

Steve? So

to do point, on the to sensitive have around we we are Type claims overall intend acetylators acetyltransferase use patent comment makes those gene. it disease press method useful the defend but the any as to and can't our that of that claims allele that actually intellectual we sense or in defects degree that I are Well, we certain that property. strategies the it's defense X the to implement to a that how the of treat our this ensure the feel said centered drug slow might for release At that of patients codes in

all Thanks quarter. on for questions. taking the my Congrats

you Thank Charles.

Piper question Sandler. from next today from Thank our coming is you Catanzaro Joe

live. Your now line is

on and is Hi, this would for guidance? plan first is taking My questions. just on when if or Joe. whether, question you for around reissuing guidance Charles our Thanks XXXX

look think When part us Joe. to with a end on to the QX this guidance for get based or point by it's the of just actually be great or to That's of going us provide fluid the next guidance I the year the to Yes. year able be but for able more this like, question. rest behind early that next at early we disease still of for or too comfortable pandemic. question, this when provide QX and state this nature when again is will we be most year the what of will Thanks pandemic hopefully

point able be we So this prepared provide will we're to at when not guidance. say to

be reimburse just for to And quarter? Would able looking patients of say Firdapse you quantitative on the Okay. therapy existing total some Got metrics. the it. at end the

able be say we Will to that?

you patients of provide at able Are Yes. the end to able number of the Firdapse on XQ? total to reimbursed

we feel drug some were on particular important of variance emphasis early the all back and provided and just a much can metrics too we the quarter-to-quarter pulled that the that past on metric. as very we've We've, the of that we in these metrics previously launched quarters Yes. be provided two have from we providing on

comfortable we're with that extent point. the reimbursed on that in at So and enrolled we're we're patients more metrics that's the number about of of the or the giving provide provide growing to drug, drug to guidance that guidance prepared prepared this is

understand steady potential point? exactly at change I MuSK Was could just more hold portion? extent I'm the then it look what forward about detail failed a or MG like? able randomized the any where talk didn't Okay. this the and the Firdapse you're scores in placebo the wondering in path the in to to that didn't MG-ADL trial that data and that scores

in treatment analyzing arms it for analysis. the study. details and Joe, of still the the give after early more how a future about specific response we all the to about will have two completed too little in it's data any us we to the the came out say We're

patent in guess and slow question indications for detail, Thanks acetylating those just the X,X-DAP LEMS to some translates the a patent. exclusivity on and in for using potentially greater LEMS? Okay. in phenotype Could explain last I patent how NATX exactly a beyond covering you broad patients,

asking question legalities of patent you Joe, can't the on and about comment are just the enforcing it. I a

taking questions. for my Thanks Okay.

you Joe. Thank

Thank is our Gershell question next coming from you from Oppenheimer. today Leland

is line Your now live.

Thanks Hi, are as we my off the understand patients opting XX% so and therapy you. patients I to Ruzurgi you treated with who execution discontinuation also mix for good challenging Ruzurgi does when the haven't X,X-DAP to with going is then to follow-up. a good correctly you XX switching Pat, mention on question taking quarter. day but for those to is go went new been this questions it or to during of include on on morning. reason and I Firdapse on it or patients who discontinuation have have that and the who are had rates, rates, on have discontinued the regard just going have see or Ruzurgi Thank back Ruzurgi been primarily patients a Firdapse patients who then and switches whatever simply

Sure. Jeff do you want question? to that take

does XX-day Ruzurgi. include patients that XX% Ruzurgi. of to discontinuation The Sure. discontinue go rate to Any

Okay.

And you Leland your repeat could other that part question, second it? of

Yes.

the dosing of Thanks. on a going components the perhaps patients, or the for first to patients on as are handle new is are switches not more previously different going may therapy Ruzurgi want switches time, formulation. flexibility mix those who any as more from back and have and X,X-DAP or Ruzurgi have had so to on patients want reason the Firdapse we what that understand from So been provides who forward patients who we just of shifts sort for switching Firdapse. to if coming are it who on been understand on patients previously

Sure.

So the a in but that that getting we first mean previously X,X-DAP, right we are new any we we enrollments the first had, saw seen now couple all the what on those really that were naive on of haven't call quarter patients none that were of patients. a since enrollments so patients new I X,X-DAP quarter patients

we've the patients X,X-DAP are that seen to that are naive those enrolled. newly So

to could when you on Thank us you be Japanese just population questions be that's there. with the Thank approved on able Okay. you Firdapse Would then opportunity. treatable understand patient able be help with timelines you. potential be any what you. share the to And and might LEMSD would

to Sure. XX% U.S. population of With just the patients Europe population, regard and United advanced to there and is Japan so have care is like States. and the Western medical about industrial the the access economy patient

us early to U.S. to overall regard timelines publicly haven't to anticipate for would about we With this XX% it's disclosed I that of timelines numbers So too model about the say just Japan. at anything point. you can use

and that might review. a expect priority we do would add I come designation that orphan with

Japanese an is population. this by quickly as to as the drug for interest MHLW possible there approved So get

Well, much and Great. for taking the thanks very questions right. All congratulations.

Thank you, Leland.

you. Thank to back of further to the turn I'd comments. for out like session. any reached We've closing management over floor question-and-answer our

us very today. you you. We calls. much Thank day. future for Have great joining Thank forward a look to

That you. Thank webcast. teleconference include does chase

today. at your We this line time connect your participation for wonderful and thank a may you You have just day.