ARCT - Earnings call transcript Q4 2019 | Arcturus Therapeutics Holdings Inc.

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Operator

Greetings. Welcome to the Arcturus Therapeutics Fourth Quarter and Fiscal Year 2019 Earnings Call. [Operator Instructions]. Please note, this conference is being recorded.

I will now turn the conference over to your host, Neda Safarzadeh, Director, Head of Investor Relations. ahead. go Please

Neda Safarzadeh

and Arcturus' Operator, everyone. you Thank Thank conference good call. joining earnings for you, afternoon,

We for and quarter and are company's year-end the excited XXXX this opportunity operating results certain developments. discuss recent to fourth

joined to Sassine, Dr. also address the Andy on questions are CEO; COO, and line session. CSO CFO. today President during Pad Payne, We and Joseph will and the and Chivukula, is by be Q&A available

with call a review the off kick will Joe of high-level Arcturus.

and recap the aimed discuss Andy results which company's quarter Next, recent fourth is certain financial at balance will developments sheet. the strengthening

Finally, and we will open the call for the answers. questions

this operations, and the the trials, the communication, programs, we forward-looking Before likelihood substantial planned facts the the of status strategy, the of the uncertainties initiation other except statements development like future statements this responses the the development programs, preclinical success remind the facts, vaccine clinical planned of for in clinical of and those involve regarding of the including likelihood of risks by management by provided of Reform everyone on historical coronavirus are of statements XXXX. and any Securities begin, success that would forward-looking COVID-XX I position made financial trials, initiation current Any harbor and of company's or the and products cash to other included historical of constitute statements company's purposes call of future Private questions statements statements. to for safe Litigation than the Act clinical and conference that

develop intention Form performance. XX, those year that under of on filings on limitation, on market in or on XXXX, statements Arcturus' achievements for file Friday, a Form submissions performance including filed SEC heading or involve otherwise and current report management's with the as risks forward-looking required the speech such results annual date to, ended the the Except to they candidates, subsequent We from to the projected by forward-looking future XXXX, or March events as as revise made, any statements an Risk report XXXX, obligation SEC. XXXX. and December discussed annual XX, December many and new XX-K may on XX-K a otherwise. in differ any fiscal any conditions uncertainties, unexpected based product of including and Factors statements, only expectations are result with, result general materially update information, prevent Such clinical factors, and results disclaims market Arcturus XX, and were without March which whether could circumstances or as in XX, or inability to Actual law, of the as those intend

I will over now call to Joe. turn the

Joseph Payne

It's to call you, be good Good our Thank Neda. afternoon, Thank joining today. with quarterly for you everyone. you.

we made progress partnership forward an on productive during with program, we've to pleased vaccine are other past coronavirus our year, will the provide This highlights. XXXX. a We ARCT-XXX, look update and with recent this our along afternoon, and flagship we

messenger enabling also and development technologies, We're innovative discovery, known RNA you the technology. our for and We of a and vaccines. commercialization we lipid-mediated or RNA manufacturing our processes STARR manufacturing product manufacturing. including Self-Transcribing drug And our Replicating LUNAR in our drug delivery RNA substance and focused are For are those Arcturus, therapeutics company who diseases leading to messenger medicines rare of for new on utilize

leads by are to OTC cells dysfunctionality enzyme. neurological disease deficiency common damage designed disorder. a urea messenger who our OTC potentially flagship to ornithine deficient also afflicted The to into cause effectively treat cycle Arcturus' removing gene, deliver LUNAR waste or and deficiency, OTC toxic liver difficulty are patient nonfunctional severe caused technology, the mutations or of the is most OTC enable disease, damage often a RNA OTC RNA which it's ARCT-XXX OTC the as with lipid-mediated can and products ARCT-XXX. a have therapeutics liver. delivery incorporates the to program OTC includes Patients this rare but deficient potential produce liver proteins a to transcarbamylase healthy, new functional pipeline to OTC ARCT-XXX digested. a as in in named intervening deficiency underlying directly for cells. messenger be patients. the has process, disease their in enzymes significant these own RNA By therapy and is

batches news since of the product. in our batches release have and been criteria, and successfully drug human multiple GMP IND-enabling our good call, studies completed. was we drug last we for These represents new as have for have drug company a be transition the planned investor Well, company. into This FDA. passed application We completed investigational stage with used clinical clinical substance milestone are to manufactured the trials. to U.S. share. ARCT-XXX pharmaceutical a The And today filed significant

the We that you for have orphan to worldwide designation rights has We FDA this program. remind ARCT-XXX. already drug granted

clinical IND we currently the the as the design including once and volunteers go provide recommended of been are plan, has to FDA we expect accepted. has the We well. a merits into of healthy that the study, utilizing details patients, stable on The evaluating can more directly study

XX and clinical we of the successful RNA in leadership Dr. his affairs, development approvals appointment rare career. His initial be throughout advances direction been experience, involved Chief seasoned announced will data into ARCT-XXX on biometrics Hughes Officer. Dr. human January, He's for Hughes and operations, focus sciences, as brings than will XX Steve more clinical our over Dr. In provide clinical clinical years drug deficiency and therapeutic it trials. to safety. including management in OTC as clinical trials Hughes Development diseases. of multiple

which also on partnership Now develop the is to and important with Medical announced Duke-NUS coronavirus a in Duke-NUS School as to on acronym technology. which world-class this partnership we Self-Transcribing Medicine X of vaccine STARR the vaccine recent development an March for named National a of Replicating utilizes for Duke between The is an institutions, now RNA COVID-XX. Arcturus moving is that X, the School LUNAR-COVXX Singapore. University and University And we've as technology, known

has RNA. observed multiple have technology Arcturus The linear preclinical our be XX-fold doses, large in effective in flagship conventional batches double-digit OTC program. efficient at models manufacturing low RNA to extraordinarily GMP of gram applied STARR in been messenger pure than highly greater process than more We

the vaccine. relationship. our know health is we become and As we effort by a resources strategic has to serious all are global coronavirus now, vaccine in RNA COVID-XX our coronavirus threat, collaborative a expertise announced develop vaccine and a collaboration This fifth recently providing

cancer As established sublicenses company biotechnology and a Arcturus a with collaboration large companies and a personalized animal has private also health relationships with through vaccines X for a prophylactic pharmaceutical reminder, vaccines. for

platform, case. to the technology resulting COVID-XX within as The trigger a the self-replicating with coronavirus with the other which the countries it's vaccine against Arcturus' development individual, an for be hailed such, could rapid STARR is dealing vaccine. And RNA, delivery program which if successful, the Duke-NUS utilizes as infectious vaccinate work nanoparticle The when of Organization team proprietary combines immunity and in our Health system. STARR is has delivered cells, is in to this LUNAR an pathogen, as World used Singapore to And develop COVID-XX host prolonged honored RNA exemplar we expression for in people. self-replicating protective will that with coronavirus. concept The underlying Arcturus antigen millions

messenger to sustained much immune technology due we other unmet conventional GMP important against production time the efforts We the with greatly COVID-XX. for there technology, This RNA There that competitive with contribute of potentially including that need. in has are vaccines. vaccine quantities is employing response an superior treat global many thereby, STARR lower by large multiple sufficient for efficiency immunity the is Arcturus develop will believe and to around our to and increasing advantage lead an vaccine to ongoing vaccines to the compared to more manufactured protein required Arcturus to able and vaccine be ability expression a develop produce is doses expect to technologies, possible vaccine a could at that which this produce a people protective populations. reducing to to efforts confer single COVID-XX pleased world batch, urgent

to who discuss financial over Andy, full fourth quarter call the will turn now our will results. I year

Andrew Sassine

Joe, XX, license year statements with quarter includes Arcturus' and everyone. is you, will development summarize. revenues briefly afternoon, today biotech issued fourth financial from earlier payments collaborative arrangements December I the pharmaceutical fees of and release and currently from partners. good which Thank source The and XXXX, research primary ended and press for received

the new fourth fourth our period associated program. was due the quarter quarter XXXX. development related of the $X.X quarter in reported tox preparation with for were XXXX and compared to of expenses Total the million during $X million our For XXXX, IND and OTC and primarily revenue million of the compared expenses expenses research to expenses to for driven operating launch of to million of $XX.X the increase of studies XXXX. company same fourth by The $X.X STARR

with from account requirements. program, our Fibrosis Although contra our mostly due Foundation we expense also Cystic increased saw grant the the these reporting offset to expenses CF expenses

more please filed XX-K, For which refer details, will to shortly. be our

to a quarter primarily offset lower the expenses. to year General fourth prior expenses by of in were compensation million $X.X administrative XXXX the and by higher compared settlement onetime due insurance

of XXXX, or approximately and the XX, a share per net year in net compared $X.XX loss or share of basic period. $X a basic reported and December per diluted ended million Arcturus $XX quarter fourth loss million the For with diluted $X.XX prior

XX, a XXXX. provide now results for summary financial December will year ended the I on

million For December prior XXXX, expenses period $XX.X XX, million year. XX, for ended compared operating the Total ended year with $XX.X in were December million same $XX.X year the of $XX.X the the of revenues revenues million reported in Arcturus XXXX XXXX. with compared for

$X.XX per diluted with XXXX, ended year per of or approximately or prior and million share $XX the million $XX.X For basic in loss net the $X.XX year. loss and a compared net December XX, share was diluted basic

Duke-NUS. happy cash $XX.X at cash announce the XX, quarter, end totaling March and equivalents projections, of receive XXXX, I our the operations of company's XXXX. through of we December current sufficient million million support the collaboration to Based Arcturus $X At cash XX, position XXXX. Singapore and current to first by equivalents December coronavirus from had first compared our cash will on with to be to expected for am to is quarter the $XX.X million cash of installment

back will over to Joe, the turn now call you. I

Joseph Payne

with provide the unmet where is and our advance is patients In as the who are at for all better for need Providing treatments. our Thanks, development there ultimate afflicted high goal us Andy. effective drives medicines. us and of novel to what illnesses day for summary, and of every partners hope mRNA was treatments we no health Arcturus innovative

XXXX, pipeline, and moving we'll enthusiastic our continue upon potential to forward additional programs and are execute We securing expanding about partnerships. our

the company, to of a is becoming to into advancement Arcturus of stage which the said, I something very As we clinical are ARCT-XXX this transition clinic the proud. marks and

time. your for Thank you

appropriate like an is now to time think I'd pause, call the open for I questions. and to

Operator

[Operator Instructions].

is question Madhu Kumar from R.W. of first The Baird.

Madhu Kumar

our I can of what you're guess that your lines, details learned you deficiency once first give through. I what of IND have kind trials the have one inform those the previous some gene OTC design you walk the happened, going the XXX? trials let's that or along to stroked with noting of clears, of OTC aspects Ultragenyx [indiscernible] broad guess, you are XXX said the the that would is, trial kind trial own about trials study And therapy from that more say,

Joseph Payne

IND We for to more from and we the do Yes, calling questions. just FDA. directly details we recommended thanks asking in on go all, of provide plan that accepted. first an foremost, some asked. we're for -- is has But understanding of and on into IND They've the right maybe the that have the to that first utilizing once the the do volunteers. clinical expect been details will reiterate, accepted, you the patients, comment time can there. merits us some what a aspects of is healthy study evaluating stable to currently more disclose once and other appropriate of provide Pad, Madhu,

Padmanabh Chivukula

patients. we Sure. other some Again, from what know avoided in in steroids the least at of typically OTC that we've typically, seen trials, are

-- their use. so we So will not encouraging that's be a

around treat to However, this clinicians is will trial a patient at the and these be going provided to plan space. the should details trials have this some symptoms we be looked may IND that be of accepted. develop. is some of And once any other And free course, again, the of clinical the in

Madhu Kumar

IND? program. I also for up study? kind And Is on type then the XXXX that remember, Cystic following program? updates And X the glycogen latest still there there's any Fibrosis disease Okay. that, to synthase What's the planned are a of

Joseph Payne

partner is guidance Well, with on X Pad active, that's that led GSD our look respect for Ultragenyx, this and programs, to definitely they're LUNAR our forward to as guidance And can well. I X, by giving was partner ultimately, that comment like ongoing because we providing but and this. both responsible that

Padmanabh Chivukula

I ongoing is again, reiterate Yes. our this the that by testing, And to is led just and GSD program preclinical want partner.

will So the by IND and Ultragenyx. be first-in-human conducted

So more I and I providing they'll to be this look well. guidance, as think forward

Madhu Kumar

Okay. And CF?

Joseph Payne

talk And With well. productive respect candidate But a meeting this on far. we track. aim We to select about call the CF, we that track year so in didn't in XXXX. remain on pre-IND to development as we the have to remain

So those for are Pad? our X XXXX. key objectives And

Padmanabh Chivukula

Yes. couple well. be of well And preclinical as sharing later more at we'll different as data conferences on

Operator

is of Guggenheim. question Whitney Ijem next The from

Whitney Ijem

what out the would as follow-up to move you minus said merit Or on study you understand pluses you're OTC. there. into a Just But study, Just about details the wouldn't healthy the that? of you'll the why know patients? more treating weighing think the on of at doing be guess, I and I you guess, provide time I IND. trying

Joseph Payne

things back study that not arm healthy the maximum quicker recruit, time. why intra-patient we're are of and volunteers recruitment and only dose, can quicker then the leave at appropriate with for the patient determining is to and negotiate these tolerated healthy into data, Well, example, dosing, we considering accelerate patients of to a with FDA always obtain a volunteers evaluate, the help the we can quicker obviously types to

accepted. are forward the with details it. those our advantages to is certain -- clinical respect So sharing doing there to when again, But we IND at plan, look to around

Whitney Ijem

for it. maybe partnership. And Singapore Got then COVID Okay.

product Just curious What second is of the what ongoing what what now? are -- work And guess, once is, question part steps next here? figure from you out then are the I you walk beginning guess, time manufacture test? from the make, of when to and ship need from ready have us sort you to to I you you starting maybe can through lines to

Joseph Payne

for vaccine? the is COVID-XX this So

Whitney Ijem

Yes.

Joseph Payne

Yes.

Okay.

helpful it I just think So might provide additional some to be background there.

for manufacturing. STARR There combining vetting the through as evaluation reproducibility that and and and disclosed in the it process by or showcase involved the We technology capability, discuss manufacturing Singapore scalability, the our processes drug and via ensued release. process So a the or press a that Duke-NUS product then and ultimately, selected a the go vetting to and ended government. innovative RNA And technology substance the partner we November, that was we with had of a up drug collaboration of delivery. LUNA opportunity STARR

we and pleased -- that. are So obviously with

going to and vaccine from respect as we possible, soon everyone we've is a of timing, With telling ask as this question date, consistent are ready. often they get the have this media, we course. example, To when to been for

the do very Pad, you are people as to a get want soon aware to We to of as possible. vaccine comment? urgency

Padmanabh Chivukula

say, happen. clock. Yes. key around internally, the and steps need to just No, I to we're that want Obviously, working there's

selected this the First is using making RNA. up be we're going to the for We've messenger already the target vaccine. RNA, scaling that

scaling evaluation. of with then there product, with some possible. can start clinic drug and and And and some soon this early so get they into the try the of as just providing in into we're having to conversations regulatory start process the the agencies now up get partner as we'll So our

Operator

The next question Li Ladenburg. from Wangzhi of is

Wangzhi Li

how Congratulation healthy long could to on after of cleared, once and give like volunteers is volunteer the on start And look for more But think enroll enrollment color will the you some would details what speed the do patient Look IND buyout. patients? later IND healthy forward IND in you -- terms the you to clears. first?

Joseph Payne

as for But way, the -- and the program joining the thanks the OTC question. -- Well, right. Wangzhi, Right, call. by and for

the do able process to recruitment. area. in to patient not intimately determine accelerate do the driving program Development matures, necessary of we We're rate share phase data we've of also our process. this we who's OTC the made that involved preparations experienced have We this we have can and is time. But what will have seriously very this Chief well, the very to at in taken -- that topic be Officer as the As this to

And people right patient going the we this. have to driving as this glad clearly, subsequent recruitment. an track question to is be we're calls in ongoing So

Padmanabh Chivukula

especially this to that is key going the this rare well recruitment, as process accelerate to to try with of about talking Wangzhi, challenges patient lot to contracting something our time, we're Yes. our IND but fast disease the take when a as academic Pad. talking that's once possible in we're of and the those again, onboard centers some going envision IND getting approved. is to And is as population, going centers one as this

Wangzhi Li

I to But understand or vaccine. [indiscernible] comment least advantages could just for versus I in think understand that Got the to companies? high then what's maybe other vaccine on it. the the RNA And also vaccine, DNA messenger at clear it's you level, terms we -- traditional easy by STARR COVID-XX your the think in disadvantage [indiscernible] vaccine of

Joseph Payne

Sure. Yes.

to micrograms And the so who to business are lower all antigen vaccine STARR are sort provide be RNA estimated thing. the produce inject even of like an vaccine DNA some vaccines to inject do the to than right? DNA you vaccines. often RNA technology, that companies for technology, or approaches. want one the DNA level, is doses. want vaccine are in we the much see are And self-replicating not that found in doses We milligrams the the lower to vaccine same of trying We of So response, it.

the manufacturability of And to service that amount of to a amount make that large is translates, a clear the So the doses suitable into differentiator. population. demand course, of and

differentiator a there. there's So significant

Operator

of Rahimi Yasmeen Roth from Partners. Our Capital next question is

Yasmeen Rahimi

topics. lot give OTC, I'm of aware size at that lines that. time of design the you Congrats this and IND. A and on not you color able across number Starting filling respect a moment, on on are to numerous with off questions and we the

endpoints to plasma love as neurogenesis well Pad's ammonia as would I hear levels rate However, on X key of proof-of-concept. thoughts are as

us So that should can of we little be kind around a What powering those looking bit endpoints we give of prove control? of you can how color for proof-of-concept?

question for the is the Second for funding? that what U.S. coronavirus government potential is, behind you $X.X for to scenes grant doing the billion are providing you qualify

able is modality? third which We would a give then batch is manufacturing manufacturing. could on very be over grams. up So question Are you scale that? related you you large are prophylactic to you vaccines modality intracellular And if know of helpful. color XX.X the Can that be specific, to that

Joseph Payne

Sure.

respect the about with to Biomarkers for address the So around first you Pad, how question biomarkers, OTC that? deficiency?

Padmanabh Chivukula

some course. Of sort of key will provide aspects at, other But in Yes, once we yes, all strokes, course, those Yasmeen. be this question, ammonia of of more for are levels that we'll the biomarkers endpoints thanks IND the color key on are understood well is space. broad well course -- accepted. as And that these the as our looking

So we patient our trial is population. stable initial the patient going when to trial -- be in

going and -- looking that's be to safety. where we're So for

lot to think aspect FDA accepting. or biomarkers second comment the And clinical is -- the question... the clarity the any can't other we on we So about of more I the the movement a trial have what then until

Joseph Payne

at Pad. I'll second give question billion. was I think the Well, $X.X that, first the about crack

the serious has think I And tracking the mentioned. now. how $X.X that everyone on you issued crisis update know government forward the is And has that it is. put the WHO billion we So U.S. COVID-XX -- closely their right

solicit the development. have that also to traded are vaccines $X aware -- all for if there's that companies efforts. the are vaccine that to of that right? cash We on pursuing can has that several table our see you of publicly government COVID-XX is So companies to the that are antigen, billion dedicated We some of U.S. we for to assume that these looking put

entity. right went the to government had the A process through deploy and just couple decision-makers educated Singapore with from comments. another capital Number one, through get the go right? people as and And vetting we we the this process government, to

different. well. gone will key through going U.S. the we think well vaccines, process through the size forward bode acknowledge benefit already. that with to So it we've to government And of and We look as countries as these The difference. We that process is address decision-makers relative tourists. dramatically this our the understand this issues

the vaccines issues do We've for the current is other your already the we've one size is But the this rather the will nicely I is, is of already And commented third into dovetail than of manufacturing, feasibility the on dose present But dose. that. one, challenges, micrograms. that number -- challenges milligrams of mean size what current numbers like and large or by the micrograms the of for question. COVID-XX commented

and -- the limiting by lower LUNAR of with dose So size our other doses are vaccines. technologies, much STARR by the addressing than

we RNA molecule, in the is manufacturing manufacturing to our RNA was completed large manufacturing With substance was of respect manufacturing, but showed messenger of and exemplified construct, of this campaign, very This where RNA the been has this smaller manufacturing the program. LUNAR is in to have feasibility something the is of in batches. RNA large. we've a in. significant date, size in with terms several the single large OTC manufacturing large And it drug gram, the our campaign, a RNA capabilities our that double-digit not experience We've

final point, time the the because to RNA, and dose addressed that my us double-digit product drug over Pad. size large then the address this the we've and the be -- large scales, challenge and or with and run of and of addressed a on used amounts to GMP of that manufacturing populations for both single LUNAR gram large can of STARR drug feasibility and and positions substance provide turn people. I'll manufacturing because well so of with And the we've But the

Padmanabh Chivukula

Joe. to very, we're well. be the utilizing it's platform messenger LUNAR to make going similar sure very our for just process as And clear we're that so manufacturing is for STARR OTC that for Thanks, employing again, what Yes. the RNA

a STARR just one very envision administrations, terms that envision be to we which for we the platform any So don't again, point sort single vaccination obviously manufacturing. having beneficial going this of situation. potentially And key of in in is is other challenges

Joseph Payne

as Yes. And that's well. a differentiator

Operator

is The Wainwright. Ed next from question Arce H.C. of

Ed Arce

today for on as XXX. the IND Congrats well filing

at few questions a FDA. do, on me. the But So for time waiting from course, of in point are, trial, how discuss screening decide you I filing patients? terms And perhaps that depending of as starting to or on realize this once how you either think the it you for acceptance proceed? then the you line see you healthies you about could patients,

did that, for all couple the to you that I patients related indicate suggesting stable to healthy agency And at as opposed And a what have you to was motivation person? perhaps their into directly then follow-ups. start

Padmanabh Chivukula

ready. Thanks Again, color just for as more we well we're accepted, we details IND provide been clinical around filed has we've calling. on and sort as of the of trial. with lined CRO. the more Once will in things But the IND, plan mentioned, and all so up discuss sort getting approval. with clinical get we're those again, anticipation timing of related to sites in clinical talk to that our discussions We're as trying to

Joe, So do you to say? anything else have

Joseph Payne

You also gain safe why agencies we agencies FDA of as -- want as reasons question patients for possible. as asked a these to have partners into go patients. to regulatory They access treatment view a this us in and other this, effective to but soon and process. regulatory asked asked us few the There's these us directly

the be accelerate way to and through would clinic. in that and positively time the view compress efficient the so they And that the most start the immediately to after we the -- patients, discussion, thought

Ed Arce

people specifically steps And the want but was that the year? as with development when has any time given but later been to Okay. reviewed not share, much Duke-NUS. there million perhaps, any $X what by now And that call Is the COVID-XX line in of in could also your partner, vaccine, steps, could there previous are next it, so -- other for? anticipate you is terms you just in vaccine the coronavirus milestone, of we perhaps this that terms now tied underway in questions, and that,

Joseph Payne

Sure.

So you questions first XX-K address XX-K, like to the there. agreement soon. included can an perhaps reference filing of to in the be be your all, that I'd of that and appendix you we'll And will as some sometime

Some comments, too.

we with First very also in Authority. Medical pandemic outbreaks. equivalent the experienced are the agency, regulatory and working with epidemic Sciences well medical school This We're the of Duke-NUS This this viral versed Singapore. all, working FDA and is School. country, Health this is unfortunately, with

got and type infectious we're a fortunate experience to diseases, of they've lot them. be with So these of very with working

And regulatory respect to a is product. we take drug very and how process we that's very conditions long time. we it and serious these what's that. the with manufacture though, through can something timing, well, is we to this do around point, is very go is good What the not it's and at it fortunately, will of spreading is coronavirus of to for messenger This what do about and well companies, know RNA the a very under quickly. long uncertain make it And takes how and all understand this the vaccine is approve long how timing

accordingly, hope with regulatory accelerate we'll that of So vaccine and to able we're be Duke-NUS as Singapore, where that compress time lines can to course, we in and it needs to so possible. this be get both working the agency as and we soon

Ed Arce

Okay. question on CRO financing with Well, Andy. may. you And program? you're OTC question a for if separately, actually, the then working I that your perhaps Fair last disclose for can me, enough. And

But wondering if was You higher comment than in had of R&D could as recent quarters. quarter also the significantly you previous the why on the well? the reasons some the in spend increase discussed share count

Padmanabh Chivukula

Yes.

disclosed working with. haven't who the that CRO, we're of terms In we

Andy? So

Joseph Payne

Andy?

Andrew Sassine

did of at because share we With -- to million. remains million the Yes. raised count increased X mean, respect sold recent $XX where and I $XX.XX, this we offering earlier our year about the we shares

that would in higher So shares to And partner at have that time. a sold $XX we Ultragenyx, also point our strategic at shares resulted in then share.

were question answers those share the accomplished Hopefully, that of we accounted this so had And the X most past efforts that for increase. for fundraising that earlier you. year, that

expenses that Obviously, R&D critical, increasing, because are is that ramping very the program flagship And the good I with think that obviously CF means and OTC initiating news program. up. we to respect that's the

a tell, So for for can time as was IND on able and the achievement certainly, you this major IND to ever company. being file the our first company,

we course, the Pad we're with about lot a we had to So going been of formulations on. solution that fourth of and Singapore. actually that related announced in top articulated of also that. ramp-up as we've expenses we're of STARR to working on earlier, the excited already a the And some program, be which working quarter, have studies the And designated

So I'm that. excited about pretty

Operator

The Nakae next question from from Chardan. is Keay

Keay Nakae

on you. IND. the to with to that for data in normal One, congrats moving some volunteers Joe, of respect study, Two wouldn't to upcoming want broaden database first-in-human safety? healthy why your you just questions on out the XXX filling have

Joseph Payne

think It's we fair that all question. a Right now, is data valuable.

road. with new our our study and discussions So OTC as is course, platform, detail but volunteer platform our plan, with and without the of program, potentially partners we're healthy present the in well. at look yes, to for for respect only continuous Because we much healthy clinical would our disclosing too valuable, down volunteers partners not being

support And would this so data having to of business. the be healthy volunteers valuable that in part

Keay Nakae

And Great. vaccine. a respect second, COVID-XX to Okay. your with then

this to your to geographically? Given in the this it constrain vaccine? in any if use of develop funding, more does able Does the ability are way way constrain source any broadly a you successfully

Joseph Payne

No.

to Singapore flexibility vaccine and other government complete the countries entities. outside commercialize of manufacture for have and We

data will Of people many we closely looking we looking other be generate will closely be and program. on this at the course,

Padmanabh Chivukula

paths looking I'll that of be possible of just reiterate course, we're doing going at trials. And and clinical to our

you. thank So

Operator

question Jen Company. of Laidlaw I-Eh next The from & is

I-Eh Jen

your congrats filing. IND my add also will for I Again,

a Just quick real few questions.

on know likely be can't fulfill that sort potential likely that XXX. more. that quickly into you some you reveal have patients volunteer the model multiple have of would Would But a -- hybrid terms objectives? be First, I healthy that of will that's and to in

Padmanabh Chivukula

lot options. more looking shortly. very all strategy a I comment And can't you'll our about But we is mean, be that. Again, to hear going about color at we're definitely yes, what

I-Eh Jen

expenses, the if as you as XXXX? the base that grow question will for follows you grow for number the My will or purpose third quarter lot is into 'XX into housekeeping base which look to modeling to 'XX second of one, previous operating is look the fourth a the the for if that quarter

Andrew Sassine

don't guidance Well, those that matters. unfortunately, we on provide --

to into with you to experience for we so assume with historical that and can of that we're assumptions think I not unfortunately, do guidance. good some hopefully specific But the you reasonable the and kinds just certainly going you up extrapolate to try the And provide can be have the with of best you And a forecast numbers, all do year. clinic. the just the come have,

I-Eh Jen

which So And last is maybe that's just the about here, fine. question COVID-XX vaccine.

that that earlier, a And You mentioned vaccine example, RNA is change for And already would identified potentially the, that the you would or going set this be virus target, any. if what if are multiple mutations strain in reveal is? you targets also that to could or you [indiscernible]. the possible given whatever, for do

Joseph Payne

community doing. for exactly not entire to we the be significant of vaccine target of concept, antigen if, what whatever the differences mainstream that evaluate plenty this we're But the as revealed and companies, and there's have vaccine we can is, We we reason, similar specific a approaches is going a what for working right? the And groups elucidate these people also just the background a is of with backup. other vaccinate revealed detail haven't very understand in in approaches to the all is. between approach And challenge to haven't terms that towards but of large antigen. what need we See, the

as well, that the So we're for there challenge. primary a are antigen working whatever on reason, people if, is positioned well

Padmanabh Chivukula

and And we're any arise, space that well. eye mutations an keeping if as the see on that there's and also to definitely in actively we're looking

Operator

question next Markets. Kumar from Capital The is Raja Brookline of

Kumar Raja

Congratulations on the IND filing.

So about mucus conducting mouse of some Cystic also of present? of in patients expectation uptake the in expectation what's program, in terms what models? is front? there a What's terms And talked And fibrosis the you the that guys translation versus cystic in in varied also, healthy studies. of what happening humans where had in the you lungs, see from the terms Fibrosis the in lot is earlier

Joseph Payne

We've animals, delivery done data our provided the clear sputum very to date some cells. the also of we've been data appreciate has of shown epithelial were to functional wild-type question, in shown and order foundation bronchial dictated The LUNAR in that RNA prerequisites that therapeutics Kumar. stability we've to the and in CF hit messenger engage as and formulated early CF patients' program RNA was milestones to in and CF funding. by I additional

functional as RNA evaluated have for epithelial it, wild-type technology So stability sputum. know, cells. you of evaluated animals our We've messenger for to in patient delivery we bronchial in

how allow the specifically believe in mucus biodistribution determined. be different question, of with that sputum. stability phlegm -- the human time of address LUNAR your we and formulated our perform because the of their to lungs, mentioned, and core amounts to in occur the that we stability that for -- to -- But types patients' this But is our RNA to being therapeutic of designed of will levels remains in that a what I are data and

Padmanabh Chivukula

of around And sharing data well plan we're ASGCT we'll May Boston that in the Pad. work Conference in as poster that preclinical that the out meeting. the -- the as doing and that lot of some again, for is And a we'll be and or translational collecting asking. aspects our And at we this of some more look key presentation And that in we're be just you're that doing data.

Kumar Raja

as have muscle. of you COVID, of the like ophthalmology. And data have in to to terms lung already like of target delivery, Looks terms as you well data in muscle? What need for guys the you also terms liver, do in delivering

Joseph Payne

protein and the to Pad? present And direct opportunity data higher conference, we that add, to showed a our controlled improvement recent showed technology XX-fold a some we had We experiment, or else at protein that this relative showed RNA. messenger production conventional Anything is where Yes. we a recently. STARR -- presented to

Padmanabh Chivukula

just intramuscular And to obviously, injection going directly be, site. therapeutic this a is the into local again, -- No.

as worked we've delivery so on. other as routes that the mean, high of not the some I challenges So are

Joseph Payne

differentiate we again Where much is Yes. allows and biodegradable at our Yes. dose. us be is more which technology, -- a our to technology also which lower LUNAR to tends -- STARR dose every

Kumar Raja

the share from meeting you can And expected there? in what's of some maybe thoughts, with FDA, the terms pre-IND like

Padmanabh Chivukula

the for mean you program or... CF meeting, Pre-IND

Kumar Raja

Yes.

For the program. CF

Padmanabh Chivukula

of we're we'll Yes, we for in we we'll collecting lot the CF a and some more Yes. probably half. of data anticipate Again, second have regulatory that year, program, share data, with provide mentioned, in then having the the that. -- that this the agencies And preclinical timing color. I discussions first data, the once we're as half of

Operator

session. turn will Joseph Payne to call remarks. now the question-and-answer the concludes This I over closing for

Joseph Payne

out for free everyone, address point, appreciate to just you, Thank listening call, to we're try reach but please the Thanks for feel We going Bye with questions. this them in. time. any follow-up shortly. We'll to again. At close now. the

Operator

This conference, your disconnect lines your and participation. time. you may at today's this you concludes for Thank

Arcturus Therapeutics (ARCT) 11 Mar 202019 Q4 Earnings call transcript