Catalyst Pharmaceuticals (CPRX) 17 Mar 202019 Q4 Earnings call transcript

Greetings and welcome to the Catalyst Pharmaceuticals Fourth Quarter 2019 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.

It’s now my pleasure to introduce your host, Ali Grande. ahead. go Please

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

Officer are the Chairman Dan Catalyst Joining Chief Chief me team, Brennan, of call today's Operating Miller, Officer. Steven McEnany, on our management Chief Officer; members including Officer; Dr. Chief Commercial and Scientific Executive and and Patrick

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

this At turn over call Pat. I'll the to time,

Thank for this XXXX for U.S. commercial joining truly you, thank company. morning. was year you, transition Catalyst a company us transformative from we into a and was as what Ali, R&D everyone an stage pharmaceutical

to XXX any Amifampridine. prior Firdapse and to are expectations X,X-DAP patients or exceeded FDA delighted had launch pleased helped are our XXXX, have more in of We than we that approval, form to access our never

expenses averaging I patients’ treat FDA-approved, available can $X.XX adult and per evidence-based patients, know, therapy currently an month. out-of-pocket that to As the you now monthly report now Firdapse LEMS is is

study announced from has positive areas to we've study completion announce results business antibody across this III for topline of important the executed XXXX we of the MuSK-MG. great with of this second year. team expect during And quarter and our momentum. in enrollment Phase very functional Recently, Catalyst our Our begun XXXX during all we

continued Since Firdapse physicians, strong the of support and launch in and have reactions patients, of encouraging payers. XXXX, to see we January from

phase both providers. over, we additional assist and launch Now look LEMS their that to support our forward introducing patients in programs XXXX healthcare the initial to is of

As from yesterday, of $XXX of fourth $XX XXXX our you seen revenues may reported net million have Firdapse release revenues full-year we press net XXXX quarter and of million.

announced will a million. to the continue previously in million further to Firdapse report full-year with ongoing product $XXX range call Firdapse. indicators we in January, commercial provide expect On you XXXX operations of performance on $XXX launch net to revenues to of Dan morning, and our As this our be related key

and we overlook business. is impact COVID-XX, today's on is can't how these coronavirus, minds that On topic everybody's our call, that the could days, the

coronavirus employees could continuous the about health that their continuous supply supply. well impact concerned for under that for drug we families, of foremost, concerns and patients welfare the of of possible maintaining physicians First the a patient's the as drug possibly as and are all and uninterrupted our We and and availability know patients. wellbeing have have shortages some today Firdapse and

Well has let to Firdapse variety wide of chain Catalyst me readiness for ensure contingencies. reassure constructed its you, supply a for

to We into manage safety have supply stock our also built chain disruptions. significant potential

around manufacturing finished Our United in regarded dosage highly manufactured in contract by API, manufactured one world the supplier. qualified the largest the of States is globe. API or multiple sites Active via is Pharmaceutical manufacturers United with the States Firdapse in the Ingredients of form our And

patients finished to has we end dosage supply in highly redundancies, through the We Additionally, uninterrupted confident and supply of our that us we Firdapse. in extent, to contract of that to to ability anticipated been with this Firdapse in have form and remain believe second the manufacturer a qualified year. all U.S. current

have with the within year. supply provide additional next we weeks two of campaign six-month underway that us a will this completed end Additionally, an of be will Firdapse that manufacturing beyond the

to could enroll is new that As growth have the the too ability of in Pathways, COVID-XX to and outbreak delays our assess of early on LEMS Catalyst impact the potential clinical now, data. it revenue patients

today, by of As we guidances. all of current our stand

In neuromuscular continue for in addition progress LEMS, portfolio. of clinical Firdapse we indications make our programs to the our potential for ongoing launch other to with

and Phase Firdapse FDA MuSK-MG. report in is our Agreement results announced trial myasthenia completed Protocol quarter for second of to enrollment positive expect topline As a the patients from have year. previously, of This MuSK-antibody of gravis Special with we the being III trial the or we for trial under this conducted this

for we As patients MuSK-MG, of treatments forward currently treating there no potential the disease. to with look this approved rare are

We in also Europe. Eastern our in trial have evaluating Type is SMA patients, which proof-of-concept Firdapse and Italy X ongoing

topline the expect in this results We of second year. quarter

conditions neuromuscular program Kennedy's will evaluated, provide our with to We proof-of-concept including palsies. in on begin neuropathy coming shortly. hereditary and with to months pressure Steve the will also clinical disease current you for liability information be additional more studies

to stated David all opportunities. a development structured will seasoned of our the licensing, President Business acquisitions, and opportunities. begun we Development month, Executive hiring and product rare a formal potential we additional responsible have is announce of at Ailinger, business become plan, previously, a Last disciplined has alliance company have for disease process or to be and David approach also strategic As of of for we partnering, to our Development sourcing, related providing are focal evaluation evaluating point management. our activities Business as Catalyst. pleased Vice He

for made footprint also great our global expanding have Firdapse. progress We in

and it sales for marketing an of second recently and year. drug our are review, having with NDS been discussions we accepted half several granted for in receive as the new Currently, NDS partner this priority of ongoing LEMS and pharmaceutical Firdapse has submission, we expect of have or in we to submitted Canadian Canada. this in Canada, in We approval companies look

what our completed have We approval. a Japan regulatory better understand likely requires will toward path to also feasibility in PMDA analysis

this months to coming Japanese input as of opinion we our to consulted with present key expect the with part numerous Japan seek As in authorities plan. have we in the neuromuscular analysis, leaders to their meet

As make you informed. appropriately progress, we will further keep we

As in see, funded our at cash and is balance you $XX year-end sheet about with no strong million debt. can

Our future to cash as build quarterly, continues well operations which we that to ensures fund potential able to as fund are able acquisitions.

in in for capital cash cash will ability to Our sources acquisitions. additional be traditional term anytime place loans require there to need soon. event we secure that banking anticipate markets the from We that for equity no remains to go the

we challenge it to normal Ruzurgi or of our on FDA's expected the Also some June with regarding grounds, filed FDA multiple lawsuit against approval occurring. progressing that more is in as delays the less

have scheduled of open briefs, filed now, though decision. motions a the to court a few and both are government there As reaching we address for all the before

third sometime expected year activities. second At for of or quarter decision remains on timetable in based our current the our this time, this

on of Of course, decisions. decisions on the delays pending the motions additional specifics may those depending introduce

turn the the to specifics on Officer, over Chief call on will additional Firdapse. Dan operations performance of Commercial our Brennan, our indicators to key and launch I now commercial provide you

morning, Thanks, everyone. Pat good and

our Firdapse first one productive The performance year. for successful a for of launch the was our exceeded as year all expectations commercial and

validated have all Their and support efforts the Firdapse pleased necessary we for all the execution, patients. medical both studies, launch utilization are our we efficacy widespread availability the safety to and to adult of medicine that addition regulatory that feedback patient has LEMS of providers. drove with and obtained from received approval patients In need the complete their create and the

XXXX, XX% January, in Firdapse prescription. adult about launch have our Since population the of LEMS patient received a diagnosed

grow, to no life-changing for prior there physicians care LEMS are approved continues percentage we as the with disease for a to see standard therapies. medication were viewing Firdapse this As and pleased of which a patients

Over this X,X-DAP to can disease the compared patients key are just naive the I calls. to medicine quarter. of had the patients the and of impact that our to we which Firdapse on have patients families. would for form these end performance quarter, highlighted adult over patients truly the like of run been and LEMS fourth any the prescribed the and patients XXX their of of of through At treatment patients we end these XXX third metrics to at pleased previous offer lives XXX were

end had Firdapse. prescribed quarter, the fourth writers of XXX the unique At

there addition, at insurer and therapy patients active on reimbursed XXX end In were the fourth quarter. the of

at to XXXX, of One X.X through satisfaction rating our commercial X and completed, has patient importance been key average satisfaction, had we and us remains of areas of the stars. out this customer end of launch of the surveys XXX as

Patients have the who patients being service In while the for down to gone consistently across Assistance with they prescribed co-pay Programs. monthly satisfied a patient of fourth month have average XX% the all take per patients quarter Firdapse advantage of zero. our per was just out-of-pocket Firdapse. has time those $X.XX the XXXX, been of received

efforts in initial more clinical this trial enrolled rates Discontinuation our rate with newly enrolled the where those under experience. is that our discontinuation we first consistent I level with with for are XX% address patients pleased we XX% a decreased now three to patients in over newly XX-day launch QX that in a and but early am quarters, was metric. saw has our higher-than-expected discontinuation where challenge to of for

the was and summer who see when September, number last early initially and did drug at down As November October the December. patients to to mentioned larger in quarter's XXXX, of commercially than Jacobus which switched available slowed a of relatively made end of the we it during trickle call, a in is November

were in because returned who patients patients trying we services drug Jacobus Jacobus former participants. presumably pharmaceutical patient not months, Most to Firdapse recent their Pathways switched to the a to Jacobus investigational and satisfaction. product were or study have seen few product, back Catalyst And the after Ruzurgi

on are focused adult patients diagnosed, them. Moving forward, physicians and we treating amifampridine helping untreated LEMS the who are and the finding but

and Firdapse the with We have are metric efforts expanded that believe success enrollments commercial month-to-month had the momentum in patients first through taking hold week XXXX and new greater increased continuing of March. our in in

generate training the institution last physicians, field non-personal increasing fully promotional number with recruited spend, These in commercial first providers. been efforts broadening include of our salesforce and and has agency which two our prescribers our target nearly targeting our hires to and utilization and disease new partnership expanded of among week now interest March, updated full and increased with sales rare inside tele-detailing an of starting doubling a

trained vet, of outreach work leads partner. new effort and and of group valued efforts and with new just this contract, this prescription speedy enrollments physician over partner. as have and begin a begun five and pleased train two to efforts already Firdapse and seen efficient with with this been telesales with We XX I've weeks the result

and seen, help and put programs that and social more digital started may of senior And have to integrated and effectively recently finally, patient work as doing loudly the we across and patients media all good the for manager communicate a have community. new place we work. you quickly She good in hired us is

more using across those channels. and the misunderstandings have product or variety the to patient on or and community share a update concerns, stories are We questions, about channels quickly videos additional services they our of social may social we'll

We matures. continue market commercialization strategy as LEMS the to evolves refine which opportunity our

of expand arduous be to continue diagnosis the While and year resources and obtaining I'm for an in journey and long important we successful help proud will a for on disease. what their towards diagnosed XXXX all still from XXXX, LEMS, to accomplished on patients suffering those our rare we've reach stressful Catalyst those as

as motivated diagnostic possible. help shorten soon as to find journey effective their are We and treatment

over Scientific on the to And our Miller, call with Chief clinic will development product report who Operating and in that, hand I'll Steve? Steve progress Chief Officer our Officer, activities. the and

update Catalyst the III indications. III multi-site SPA. an for international provide FDA commercial Dan, conducted for or now our we for this ongoing proof-of-concept indication. follows for and Special Firdapse an enrollment additional MuSK-MG, recently on pipeline I'll exceeded update develop the trial goals in is neuromuscular Phase to our announced clinical Protocol that which successful trial our Phase under Assessment, Thanks being trial This

acetylcholine XX sometimes enroll the the treatment XX amifampridine. at Catalyst would response type to were to by MuSK-MG MG at called determine patients of attempt In to to least and committed the that other requested FDA we with receptor SPA, enroll which would generalized if MG exhibits MG, this agency patients, least FDA-approved any

for Catalyst this include patients was the year. Catalyst enrollment treatment quarter to were gravis. this met participate and At for clinical of to our after Catalyst the which the close-out time, chose activities ethical trial they anticipates topline goals being were both enrollment are these myasthenia enrollment for that due of over of results goals completed and reasons. exceeded screened reporting to types, wished in last second and and eligible The primarily the

single III of cautiously a remains LEMS, the for junction of difficulty protein contrast Myasthenia Like population. treating Catalyst is outcome is the type a of symptomatic which a CMS disease antibodies MuSK-MG with Phase disease autoimmune the amifampridine. therefore single neuromuscular in homogeneous that caused damages in and to relatively optimistic the in Gravis is an by MuSK the treatment trial this about heterogeneous

the the currently believes a to the U.S. homogeneity in about no in autoimmune that in there LEMS to treatment States. United believe were reported trial similar previous all MuSK-MG disease way Catalyst we much should same treatment, and which for response result approved of is to similar. X,XXX MuSK-MG very X,XXX there is patients an proof-of-concept trial outcomes the are with

to provide patients. FDA-approved the is Assuming look option successful, these to forward able we being for an treatment potentially trial one-day

Type in This evaluating Atrophy ongoing in Europe and and second XXXX. and the quarter tolerability to ongoing XX safety, of the ambulatory Muscular We study is amifampridine topline Italy proof-of-concept for of of Spinal enroll from Type results a have study and to trial patients SMA we potential efficacy this treatment announcing is X. in or Eastern Next, symptomatic approximately SMA patients. the in X look forward study this in plan

SMN caused primarily homogeneous severity. not period changes relatively disease initial but result with neurons, to disease should to that time is in symptoms, treatment a SMA disease is related does by address study differs progression. a design the SMA measure genetic each defects and in in is protein in motor in crossover a designed one-month proof-of-concept which This

Type SMA resulting variations through X,XXX four severity, gene important of in estimated It as to same has X note are and patients. which is that various generic caused are types prevalence X between to of SMA X,XXX Types the in broadly all X. an SMA by characterized defects

both III successful, trial progression. treatment Catalyst of the trial to with If clinical in attend address this symptomatic the is Phase which and discuss we disease a FDA, design proof-of-concept intends multi-center to

Additionally, due a types SMA etiology of include will four to all of the Catalyst a SMA, of common group trial. to types also seek genetic clinical broader in future

wide very different like patients, X,XXX Phase trial in placebo-controlled to also to spectrum currently Phase be more CMS. of U.S. broad our a genetic clinical defects range or double-blind, was produces Myasthenic of variations confirmed last one about give possible symptomatic therapy, patient III difficult first the genetic CMS-XXX, to for representing of as would I Congenital CMS presentations trial of is This clinical treatment a on spectrum the XX than called, and Syndromes, to population that III X,XXX a estimated trial and CMS the defects. of is update genetically heterogeneous for This study. are ever disease response that to such,

score global achieve We the of given or muscle Phase measure. of phenotypic size treated primary two XX function did period in crossover and genetic patients to And and endpoint the impression not endpoint this trial treatment subject the patient study. the significance III overall for multi-center, sample statistical variation, secondary

set and even FDA with with showing that not most agree Catalyst and was the now some of showing in patients of few There has data their evidence all a adequate evidence condition. contain efficacy, does patients. the benefit these some a clinical for no discussed benefit worsening results

However, little-to-no level, observed individual the in benefit most. patient at was

endpoints and carry at difficult trial if genetically diverse Such in this population makes estimate successful trial would size very what to a out to needed an it outcome be all.

is not clinical this that to took years much four a patient recruit recently practical conclude for population. trial and larger simply Our trial completed is to almost it reasonable

to like again this agreed all in participate families thank trial. to would Catalyst who

will on XXXX. A Catalyst symptomatic treatment end the outcome poster has trial of the the discontinued development in International for on presented Diseases of Firdapse the at XXXX, Neuromuscular CMS. of Conference be of As

this Canada. reduce in Moving now and activities should started commenced, those Review of of on to symptomatic which of plans drug for will time commercial When a second yet for activities Priority the in expects treatment Canada. an update half submitted also six approval seeking has granted in has the market New XXXX Catalyst expansion has plans LEMS. provide NDS, the Drug NDS, this regarding Catalyst our the Firdapse, NDS months. review or Firdapse not commercialization approval in we cycle been Submission, of for to Canada, Catalyst

a the file expansion begun and recently Japanese have amifampridine of drug. the for MHLW, ago, companies Japan. we years Welfare, two also Recently, marketing Catalyst or of of the Approximately discussions develop for as to territories announced the to approval an include drug the of and MHLW, Japanese this actively regulatory an have approval Japan. in Ministry designated they Labor been NDA regarding priority and soliciting to Firdapse government with seek pathway Health,

filing file An long-acting what in update a provided eliminate doses NDA version required Patients Firdapse pathway will Firdapse. and that for order need to on to of MHLW be on once will be an their activities multiple NDA the come plan daily have to agreement Catalyst an regulatory in the of the in around Japan to Japan. requested

studied new and properties the of We are and LEMS. symptomatic product development been developing future At now have being product release drug actively developed updates characteristics finalized. long-acting treatment formulations candidate are the this stage order being program, the provide in to of optimize are this in when will the

to neuropathy with begin Finally, proof-of-concept near palsies. and pressure also studies the Catalyst such to disease conditions will be as future Kennedy's liability in additional for evaluated, neuromuscular hereditary

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

indications current better and well all additional opportunities a excited these for are Firdapse additional patients. the the as about in countries to product develop expand Overall, to we label as into

as paths they these any We clinical available. updates become regulatory will and provide on

to to trial prevent previously addressed the follow-up the The also robustness will clinical Expanded robustness on to Firdapse the health ensure ongoing trials, safety like public patients. for I ongoing supply of supply chain emergency studies would COVID-XX materials and to of our interruptions an Pat of our Program. to related ongoing virus. also same clinical supply Access comment

To and not encountered trials. any date, to monitor delays have in conduct we ability our

a of countries direct note our trials important public institutions However, being our at it variety clinical any are topline quarter. to will take virus report to control. private of institutions is conducted still multiple that in early actions to if the response It outbreak outside which are and healthcare is COVID-XX tell second to the ability affect in our too results in these

will I financial Officer to results. our our Ali review call the now turn Grande, Financial to Chief over

Thanks, Steve.

and from launch heard with our Report continued you've XXXX, revenue first share loss $X.XX achieved filed fiscal and As million disciplined $XX compared for team, on operating diluted $X.XX Yesterday, product our to $X.XX we we and XXXX, share Firdapse, from and full-year income of In net XXXX. GAAP reported profitability. or of year per the GAAP of our Form $XX basic a per million XX-K expense per net Annual basic or first for our management. diluted the XXXX of

excluding compensation net loss per $X.XX basic comparison, and For compensation share. $XX.X million expenses million XXXX was XXXX, per non-cash $X.XX $X.X non-GAAP net or In income of $X.XX $XX.X was per excluding share. million non-GAAP stock-based stock-based related and diluted to $X.X or expenses related of to diluted non-cash basic million

in XXXX January, for launch with Net $XXX.X the period $X.XX basic cost income the of $XX.X million and of share fiscal GAAP from For net XXXX. of was $X.XX in for $X.XX XXXX, we revenue of of loss or the per to $XX.X million. of fourth million XXXX net basic GAAP share diluted a year Firdapse quarter compared product and related per million per diluted $X.X sales same reported or the

at important of XXXX approval million. million margin the of fourth in prior that revenue was in is expense and the the this of $XX.X quarter will $X.X gross benefited remember product same cost XXXX, Firdapse rates with to related inventory the our from It of continue For quarter not net during XXXX. Firdapse, to from sales FDA the

$XXX,XXX year during December, that our collaboration with began the had XXXX, from revenues Catalyst of and and generic XXXX. Endo During quarter for fourth year-end Sabril

XXXX million Research million and as respectively and and and fiscal for programs quarter million ended our year Research expenses and compared affairs, for of quarter to the development quality million consisted development clinical respectively, quarter and ongoing and and Expanded Access expenses XX, the $X.X were fiscal for the December XXXX. Program. primarily and $XX.X trials Firdapse expenses fourth year for $XX.X studies $X.X year and expenses and for our assurance from regulatory costs medical XXXX fourth

compensation trials adults milestone we and expenses LEMS, for acceptance with from comparable and expenses medical development related and program in Firdapse of consisted costs our and personnel our consulting our expenses of our Research period as for clinical the and connection support submission, primarily NDA XXXX, in expenses expand for with treatment headcounts approval in NDA programs. the to of our affairs the

we clinical to our will Access substantial cost release Expanded We SMA research in and ongoing in X formulation for our expect that continue Firdapse. MuSK-MG XXXX continue studies and development complete Program trials Type sustained be as and program our and and

our increased supporting and fourth to fees and to selling $XX.X respectively, general The commercial professional including of million fourth year totaled administrative with personnel, fiscal access quarter XXXX. and in due cost of research $XX.X quarter and lawsuit for FDA. expenses compared increase expenses, year and system the and $XX.X implementation is market launch million million against our and expenses, expenses, the associated million product Selling, market salesforce and the $X.X fiscal primarily XXXX

support expects general sales We of FDA. against in to commercial build XXXX, patient to programs and the as Firdapse, and our in we increase and pursue activities selling, expenses continue infrastructure administrative and our lawsuit

investments $XX.X XXXX, full-year where funded of debt. for and $X.X income in don't of no we cash losses. available XXXX, million million Catalyst operating XX, net state have had On the For taxes provision December for a states and we taxes have

and the at yesterday, Form our and be maybe filed Securities website with company's on www.catalystpharma.com. March Commission on Annual was in and Exchange information the detailed XX, found the on Report which found analysis Relations More Investor XX-K, can page

to the turn now I'll Pat. over call

team am has I in across functional Catalyst delivered XXXX. areas the Thank very of proud all what Ali. Once you, again,

deal on look this momentum positioned and a XXXX to this year. well are great in is to to there forward build We

Firdapse, that to business a diversify other value Firdapse, standard deliver Most to global lifecycle rare for to indications, near-term beyond advance I importantly, by expanding further believe have and out for laid establishing Firdapse, Firdapse label expansion a for care patients, clear strategy adult LEMS include we for to of management longer-term shareholder the with our our opportunities continue people patients including diseases. a deliver Firdapse. we longer medications extremely look acting life-changing to formulation, focused remain mission on commitment our to to living and and

This like remarks that, questions. today. concludes the take call we prepared question, our first With can I'd Operator, to to open the please?

Certainly.

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

line is now Your live.

Pat morning, team. Good and

question. for and thanks First on transition something from the that's that congratulations successful not all companies this a commercial secondly, the all, execute doesn't that stage, development of to stage happen taking always our And well.

for patients of, or deployment COVID-XX? with say, some and you $XXX information ask you – business geographic drivers $XXX weeks the to the I'm on current I next new to million there guidance, additional tone months, challenges over the couple your me just guess, And patients? on continued expansion? about million. it what of Pat the that persistence or current I'd question Is field get key in range? teams' Let the Is it wondering or could a Dan, be touch course should if of current like are

address the employees, force, ban travel XX-day field all we you. a that for – question. ban our including Charles for for you, travel and for I'll issued a Yesterday, let Thank Yes. Dan

think thing the safety obviously So our as companies is it's daily. employees. something an most The most of will – tell important you, evolving that is this I again we're situation, monitoring

virtual teller patients, And of top are remote docs, the on working community so the really open communication payers. the meetings, of department meetings, of course, so and and with our a employing program, the doing is we IT keeping line

more as talk will you'll will evolves we So are we the that to I I and think, smarter little our evolves, extent, coranavirus about reaching to think be how with this see regard constituencies. the situation with of all have a that to we

of with So that to we $XXX the regard $XXX make to we guidance let are sure our not expansion. to and Dan global for any year, that metrics address that include I'll does things to this get those monitoring do the million But levels, million Charles.

Thanks.

thanks the on mean, new still out question and fantastic Yes, there diagnosed hit those LEMS patient are on The And for are drivers the much Charles. Pat. it. patients Thanks, medication. starts are really key and not that pretty with I this you've unfortunately

we some work. still So have

on that. remarks, about who have We – need we XX% have the that of mentioned patients I tried diagnosed to are in Firdapse, as my about continue with

back our inside what our are manageable, the studies and other early to it So know which salesforce new patient more in XX-day we line XX% is with programs as of especially starts us it to new key board, like and important is we so on expansion we then, and is the sales. able be starting and why on rise get and that with in a above starts maintaining the more have under to which metric, XX%, saw the from increase manage patients non-personal at and of promotion for was course, that's that initial discontinuation patients that

those the metrics driving. net’s are to So two be to such. really are seems stable remaining prescription and milligrams gross main that and with average Everything going per to else be

elements. those to down comes it really So

assuming discontinuations I'm – most that part for new really the current XX roughly No. patients? applies to only or XX%

level looking of what our of occurs. level What ultimately new days. enrollments persistence That's and in metric discontinuation Correct. within XX at

Got it me then I to guess, do in Canada, – need it. a would And well, seem partner. I'm you wondering why

can't and wondering regulatory reason I'm or you just the of – size is some the why doubled legal You've in salesforce, there market Canada?

to significant the a require of would and investment. our wheel in sales it marketing We team reinvent don't Canada. want Charles really

and disease find even drugs So partner to that's marketing rare community. like the neuromuscular we'd ideally ideally, Canadian already a to

imagine, can rule you in And proportionate. XX% that we they're patients to we Canada. U.S., of the on believe XXX XXX thumb based As is, probably that in think the prevalence

Canada. in prepared substantial not a to bring ourselves this really to market we're investment So make to

And Okay. the perhaps That's for MuSK-MG one helpful. trial. Steve, on last question

one You optimistic. being mentioned that cautiously

If cautiously part you the help useful. be us would that could understand

that's responded a of randomized enrolled number I'm patients withdrawal wondering study. that just I is versus provide any basis could think your that us the at What of clarity And a on you cautious? basis? blinded on the if least

my of when of everyone presumptuous pass me it's tell basis is that to cautious to still would it's be going The definitely blinded. simply

Okay.

is in see to because had patient reality expect outcome outcome. good that proof-of-concept a trial. the a trial the and homogeneous a But current similar the we're very population It's III optimistic Phase we

potential about of optimistic trial. remain the the outcome we so And

the in age enrolled the Phase Similar Phase – in I? versus two patients II in

Yes.

So a patient population. similar of homogeneous. that the same affects And the the for basically is the people It gender. main primarily reason because again same is and age disease very it's about

And patient populations trial look proof-of-concept so between this... similar the the in very

Thank hop Congrats. back I'll queue. Okay. the in you.

Thank you.

you. Thank

Joe Our next Catanzaro Piper from from question is coming Sandler.

line Your is now live.

questions. for the Hey Thanks taking guys.

ask their directly what there, follow-up those along the COVID So patients, impact you patient? the from COBIV by maybe opposite see prescriptions lines, of percent to if currently to from with any visit of delivered guess, opportunity to with prescriptions And and side refilled. on expect I'll or here follow-up mail get regards LEMS, I are the it spectrum have whether seen guys

take you please? Dan, that would

Sure.

them give even pharmacy So product all it letting out to by it can are our delivered variety that And patients know, directly we patients specialty through prescriptions that. fact, and shipped and different mail, comfort of and and a in we've can lot communications be had by on sent be to and of ease is out. there our all of just a system, available means, shipped them today focusing

work can insurers Earlier to we than do that. normal, with

worried – end are to want of about can't to seem patients So be we to It's and handle without for because know we therapy. very that and that really one-day patients don't doesn't disrupted that our that important COVID them.

can signatures have to We awareness, we ramped up enrollment We're to obtain and enrollment able take electronically And alerting forms. for still enrollments. So promotion that patient able appropriately. office the new TBD for physician and and a on visits, is patient new area on in starts still is to the prescription their new and our patients take forms on non-personal see for going being and physicians.

still very all coming else, to results So just everything kind happen weeks. eyes that But efforts way of in patient are our everyone but how our week. the we from we're assuring – starts. just new a on seen see good the to then the last go, we've it's And have expansion through is things like just

Got follow-up And then Okay. another just it. here.

So there's XQ. no if about of receiving some are at XX that compared the the to of benefits, sense about XQ whether the you've less us you look due any give an at how popping net-net, insurance-reimbursed events, breaks it just prescription between other maybe this? up discontinuation patients and there's switches reasons adverse to to other Can metrics that And provided, a numbers end drugs? out for you seems

our drug Ruzurgi I as of reimbursed as to Yes. Jacobus a that versus QX mean, patients as I've QX patients far November. the it patients mentioned September, end came from the drug. from in primarily different number discontinuations. had we October, or things and discontinuations of of at bolus And it's of occurred two to a the before, on that kind And

the of fighting that so under still – we’re new quarter, at has discontinuation did And have the patient beginning hand. that initial more gotten also we naive

have impacted at of moderated. QX, both time much elements And those new patient and we reduced in the a QX had number of So starts. same

other we actually starts. patient discontinuations for those ended than had we new So reasons more up having

started in QX. as these of a All trends patients much as Ruzurgi new discontinuations starts in better new from well have the direction discontinuations start patients to seeing the

the expansion same on enrollments discontinuations patient the are that that optimistic time, at side down. helping efforts we're commercial the going So our new are

one I of think but slightly entered able what of for I'll Would to different in ultimately way. treated ask be And randomized it what open-label asking, Charles say one was double-blind is during Steve. you maybe portion And and the the study? percent patients trial the a this quick run-in MuSK-MG Okay. maybe for were

I'll haven't say any rather is now dropped the that just in details completion. out through the right specific and continued and number We a enrolled, randomized. majority run-in All about participated given than large then were being randomization subsequently

taking for the it. Got questions. Okay. Thanks

Thanks, Joe.

Thank you.

question from Gershell Your coming Leland from is next Oppenheimer.

now live. is line You

questions. Hey, good for morning. Thanks taking my

Good morning, Leland.

of that leverage salesforce for get not Hey, are you for good current MuSK-MG, you types but versus patients LEMS, efforts, to In marketing your With redouble other you but all have approval words, presumably the physicians. indication? by could regard those same morning. to would your in MuSK-MG cared therefore

Dan?

and the fit salesforce Yes, and a our definitely relationships nice MuSK-MG with is targeting. existing in

physicians are our In now targeting Mestinon, patients both as of of most right which is with fact, in MuSK used well on the as use patients. that's a medication often LEMS

new that indication. this much very glove with the So we a is feel fit-handed good very

Okay.

And not any need so should any field that should get force you approval… expect for expansion, material we

now our materials any cost see the to not that of see information salesforce. in there, don't and and but benefits we expansion. create and right share key material differentiation We would as we salesforce in Obviously, need increase medication, there's

a second initiatives, for on Okay, your development question great. business just opportunities. as look then outside you And

pediatric? for there kind for you wanted the that? than it interesting squarely other potential Just there your Is live does is disease? Just to ask, as deals neuromuscular what Thanks. assets? focus rare Could curious of within be or look broader scope for licensing

Yes.

that to the disease X,XXX want the patient sort prevalence We definitely remain we'd to in be as is maybe we we want really ideally ultra-rare neuromuscular certainly are Leland, don't to that are for too stay So which ideally, or in really Also or beyond space, assets and early. in neuro companies rare looking are proof-of-concept. X,XXX difficult. defined in of that space, like

crosses for to initial our successfully interesting with salesforce market. fit would that that that's from our be said our but a we company's perspective plate like criteria, that be having might us anything So it looks able something or sort that of a

maybe and on that's little that But we're our busy very you a to change, criteria, front opportunities. may get that lot starting that say too certainly, initial at looking a of would to I So tight.

much Okay. the for Thanks Terrific. taking questions. very

you. Thank

Thank you.

is from question ROTH coming next Your Scott Henry with Capital.

Your live. line is now

good questions. Thank and you, couple morning. A of

get kind a to or climbing right might of of in of Jacobus market sense the state, be kind decreasing that share what or that dynamics? has should out now get sense share you a trying a reaching of steady if be there and First, the near-term? Just

would Dan, that? you take

It's we see as they perfect get very that And good Sure. people have be leave. Firdapse, trickle. to to discontinue don't from that, off. a to We even we would insight down we a discontinuing think why with low that handle on try will although – they has the leveled

continue And so perhaps, small but that month will – three one that may patients that, I a per at think stable. element to stay two to

have number we insight What don't is to patients. of any new the

the investigational that still were Jacobus program. so there's Most patients, somewhere drug XX initial of those total. XX, around we perhaps patients on And think

either been response And been supporting communicating to patients. into existing been their our shown them result diagnosed great that our have efforts or programs, which before strong – been satisfaction. even have among all treated so support awareness then all getting and about and haven't and physicians of and haven't had generating and strong And have quickly patients of great

then the Okay, great. insured it at would leading patients a And seem pretty indicator. be would looking metrics, like good

concern So fourth jump up? would a of little first you number XXXX, quarter the to months in quarter that that that X.X declined XXXX. through expect

up. and want definitely jump that to need We Yes.

due mentioned to earlier, those it in I As was QX and mainly discontinuations.

we Our do decreased. that is discontinuations see the new have discontinuations in QX What our outpaced enrollments.

increasing. are enrollments new Our

continue as what we these just underfoot we we new so our all for reiterating that programs is Pat in and have good good we salesforce and mentioned, think results, is look that partnership, and guidance sales a inside getting showing this which are And our balance. grow. to to new And already

you color. Thank for that Okay.

as to out. this just Just shifting couple model statement, income questions a we the of over

XXXX? First development of to nuances we runs some cost was Any sold. might see pick a up? of COGS expect in you believe that, we I out tick it in early up goods should had that sold when to and while goods cost that in

you would take that? Ali,

Yes.

call, to approval and had expensed mentioned continue XXXX, not prior used we our the same been the product been manufactured previous will years, during that FDA had that rate I XXXX. during the at as hence So in in

substantial. believe that will the but that cost that sold of we don't So we be believe increase, increase goods will

Scott, to and recall our that included we in goods, XX% to our Jazz. BioMarin cost royalty of payments

not Ali this going which so compared significant again costs is So that to minimal reflect the to we'll significant to was truer year, XX%, incremental and be last goods, later out, cost to of good. of the as pointed – to year's change compared really a start

we that increase And to that Yes. also have manufactured approval. manufactured, as but deplete only we that have API talent FDA previous the the not we will

purposes you the at Thank XXXX? statement, taxed Okay. to in you. would income be fully And staying expect on accounting entity for a least

at taxes use and NOLs we XXXX taxes during use for expect our that so also Does for we answer least in our our – our our question? XXXX, In XXXX. firms, expect firms NOLs for federal to NOL your to our

if but like profitable, when a they'll they'll gain for sustainably take for Well, then gets you're typically that just you fully company will those happen moving and not tax that. a NOLs seem basis. yet, report on it a would I'm sure into

Yes.

positive you decision a making the than of evidence allowances at XXX reverse we the requires specific know the before reversal to just very tax as for analysis is evaluation ASC So in that the waited asset for allowance. different operations. is accounting deferred and looking negative evidence,

such income tax. is said, evidence, to we years. as that more tax not forecast evidence. And three-year net such as income previous the income might negative pretax in meet always know, factual that for as you gives felt evidence guidance at losses positive positive evidence, the didn't while we the weight while The three-year XX/XX/XX and it And future and

a of continue XXX ASC will do purposes that and the monitor the we will. For on analysis, quarterly situation feel basis it to

with So of Ali, be if projections this year, most of year our sheltered. agree you Would meet we’re that? this would from to income that

Correct.

Okay.

think color. you question, of for fourth levels that expenses should for think quarter the we about about you do is representative year? Thank very we That's helpful. XXXX somewhat XXXX, think spending Okay. following – Final when in

an of lawsuit charitable us forward. us. had had hit versus significant we hit don't be have litigation that And that we the that in going FDA. fairly for don't taxes to number quarter. expenses hit actually Also contributions expect we expect on to had going also in were state took a our so, XXXX fourth utilized didn't We a things we Scott. accrual We that to the think I in are

expenses the QX for going representative that going see XXXX. what in to forward think don't I you're of So

for taking Okay, great. helpful strong you. on congratulations the Thank a again question. That's and Thanks year.

Thank you.

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

you us forward calls. Thank for joining to Thank much very We today. you. look future

conclude does That today's you. Thank teleconference.

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