BCRX - Earnings call transcript Q4 2019 | BioCryst Pharmaceuticals, Inc.

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to the BioCryst Fourth Quarter 2019 Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speaker presentation, there will be a question-and-answer session. [Operator Instructions] Please be advised that today's conference is being recorded. [Operator Instructions]

to conference like speaker to now Mr. today, would hand over the your I BioCryst. at Bluth John Please ahead, go sir.

John Bluth

financial Today's much, welcome accompany quarter and to very our BioCryst conference Thanks Josh. on fourth XXXX Good call are corporate our slides releases and press morning, available and results to call. website. update

and today Charlie Gaier. Officer, are me Chief with Commercial Jon Stonehouse; Medical Chief Officer, Participating CEO, Dr. Bill Sheridan;

Following our questions. remarks, your we will answer

regarding and/or statements, future that note today's call including please financial those forward-looking as company's statements unaudited begin, as we Before information, results, will conference forward-looking achievements. and the contain well future performance

These or statements results, uncertainties, performance, known in implied results different unknown actual which to achievements risks future be materially this presentation. are and subject to or from our expressed may performance cause any and or

not these undue forward-looking should place on statements. reliance You

website. discussion which information, the to and documents please additional a For be including can our accessed Exchange risk factors, the refer of our filed Securities Commission, with company's detailed on

the Jon Now, to over Stonehouse. I'd turn like call to

Jon Stonehouse

disease told their a is shot us pill disease their a want. day-to-day role living. that Thanks for will tell diseases the life alone in John. who to diseases. address even let treatment much focus to rare on us we suffer this smaller at their but because targets from differentiates to These our oral opportunity or try drugs do is bringing because desperately capsule and tough very few to the play patients take biological them, and companies have their BioCryst seek ordinary They what a extraordinary patients succeed, they have an What is

our lead era. is for and factor something a D rare world, new we're Today, With for patients in a even pipeline and it disease inhibitor, this making an our moving HAE multiple with into the exciting reality approvals more company is shooting around for; our oral patients. are just BioCryst coming program not

only It's early are our very in Our executing this March clear. plan. progress making and priorities year we're

Japan would and review. is approval be far submitted So, this year, Berotralstat. has for we've under for this submission review which the drug application world Sakigake means designation, the first been in the in accepted our new The

we U.S. and the NDA. We Xrd hear hold from PDUFA not said NDA have we and has they expected committee submission, a discuss our now currently the we said to regarding are FDA Next, to meeting date planning did. the agency an advisory that to December

for Europe Lastly, the we're on MAA track end our submit by approval in the month. to of

U.S., in an to our XX approximately later extremely of to this all both Europe. Three then the company. and exciting year is months in in approval and up This next adds in and Japan approvals quarter approval year in transformation first

inhibitor, The other top BioCryst priority proof-of-concept effective is without oral achieving BCXXXXX. D for

study we've morning, quarter. first this second mediated announced we not this in As remain PNH dosed is PNH, This for on-track data proof-of-concept alternative complement patients for and to but have pathway. other the involving diseases proof-of-concept the the in only

the in and the we'll So, we quarter. proof-of-concept second the of to portion our see over expect study go of design what

having therapy. strong supports whether approval the berotralstat, and Charlie why once-a-day market acute switching will want that treatment or with market that so the in first injectable be our they're market switching the and go Torii we're from partnership from review this over oral therapy use in Japanese to very HAE injectable world. prophy for our about the And he'll our view our patients will many evidence excited

therapy. that we patients will likely therapy, such me a moment to Charlie, on over when our it have they're strong oral is believe data, use to clinical that many you turn of make let I evidence benefit before take clear a sure, why to have But a understanding keep and our them this

X, slide graphs X to of data pivotal APeX-X. turn you'll our you from study see two called Phase If

months the the approximately left, XX baseline the month by the data of result. month over there attack down graph attacks you on XX on attack rate mean per going the and approximately XXX-milligram Completers see This three one show per dose. is a to at months. is rate month for attack through On But those staying robust

in you What's them even patients in we per them response half about see XXX-milligram the per on when the to after a you second active a graph rate These months. is very month six then more for grey on interesting months drop are Again, attacks from on the reduction. month six and switch right. average the an dose placebo at attack boxes two nice to attack the look on drug,

works So see patients drug and benefit. meaningful this

slides of are it's in attack data six point out the look couple of free. patients results the the when similarity or whether where when more, median of this bolster you months the next The of APeX-S from even attack at half berotralstat more than adding rate, XX

conclusion. the works. same reach drug You This

events look see safety at tolerability you a berotralstat, transient if the adverse tolerated finally, profile generally you and mild most events. the GI and safe being is common that And with and well

So want the to physicians remaining it? questions left And will prescribe use are, it? will patients

the turn call to questions. and you I'll through the to market over the walk to Charlie research

Charlie Gayer

patients their data berotralstat about and are to very treat patients the compelling the seeing safety research Thanks they reacted to berotralstat of market from our HAE What regardless After physicians today. trial, APeX-X is the favorably how Jon. and is profile. willing efficacy six-month so how use

its drugs. also within drug current what use expect for injectable while of cost prophylaxis for prophylaxis they overall. physicians they to importantly, range prophylaxis their therapy, patients pay of Many our oral switch And the our say, to is reimburse payers expanding if will

for to grateful us is therapies they medication for treatments. lives. dream, HAE years advances to treating They an oral their told although, that add injectable are reduce have these with burden Patients in the the their want

Our many patients gives we that great drug. will survey of dream us realize when HAE patients XXX confidence our launch that

berotralstat the patients XX already are likely use willing modern are a And are very XX% on to too switch to they to our take prophylaxis to important that particularly drug. very What's and Nearly XXX of of therapy. willing action. patients is the

our say are patients are very or they interested who very the therapy Even are Cinryze, a Takhzyro very berotralstat. satisfied with to among in half switching -- Haegarda

So years, to advance. does perfect therapies patients many switch? is they to treatments continue HAE have have see oral switched them some Injectable and most big their recent want no in to as new next but attacks. have Many patients so breakthrough get control, patients why and therapy drug helped HAE under the

including in be and slower hear doctors prophylaxis and manageable. from for Takhzyro tend moving, average But of survey, we per reported that Berotralstat, an month. to patients milder over patients attacks on our treatments, attack example, X.X just breakthrough

burden What want a treatment. their about of their that smaller treatment lives, is now patients a helps want controlling and HAE reducing by attacks to think refrigerating don't They of make as once-daily their An that see medicines, scheduling and treatments way is their HAE more of discomfort to preparing patients oral, medicine dealing disease. treat injections. something normal and with a part the

Physicians prescribed expect selection treatment the drug shared patients to helping who their of recent their make treat be with most because HAE believe oral for they and an their recognize new clear experience value have should typically HAE patients, that patients most decision therapies. to physicians a treatment. Berotralstat It's switch

how XX% all their XX% of Berotralstat and to to the prophylaxis other expect their coming switching patients. who of physicians future patients treat We they HAE XX% the treat physicians treat our The over drug patients. use surveyed U.S. in from very current from of a with XXX would of with large they available. reviewing product After a from with sample their of reallocated the current half half patients about expanding XX% prophylaxis profile,

but research, in market of expectation and market. half is intentions capture likely even HAE their sizable overstate portion to cutting that can Berotralstat shows Physicians their a the

the and year. bringing HAE around the patients start drug in to eager U.S. this world oral are We to our later

HAE. attracted commercial talent They have and in believe We BioCryst previous disease recent tremendous medicine they potential. of because experience who Berotralstat bring with are is track transformative joining rare launches experienced a records commercial success in

We global and the has willingness the opportunity and of and over the Berotralstat This population in peak reactions factors Japan. product $XXX reimburse to million. to U.S., patient the HAE Europe the of payer size enthusiastic physician believe profile, in that market a

the Berotralstat Europe where in We are approval first in world. to the receive U.S. Torii our regulatory with the for our potential partnership launch prioritizing the Japan, preparations supporting has and and

our As our at we of JNDA the quarter. this John beginning submitted call, earlier to shared the PMDA

from that Berotralstat this year. Sakigake our potential has approved in designation, the comes be review half second accelerated the to With of the

potential As address to Sakigake its a unmet its medical high awarded patients. reminder, a Berotralstat XXXX in nature the PMDA need Japanese innovative and for designation, the back recognizing

and our the the from Our results done randomized clinical in trials. Japan. submission placebo-controlled, efficacy based from HAE was trial on first JNDA global ever It also included safety in data APeX-J

shared Berotralstat we and investigators The dose for by the the are would study the and the pivotal period. patient from of XX-week to endpoint associations results local for APeX-J. today. readout in met of reduction Japanese the It's Japan first. the primary excited note there Berotralstat XXX-milligram showed prophylaxis indicated Our consistent, are no XX-week HAE treatments be over attacks sustained in important

U.S., is, market a in saw therapy the decade So its in essence, which prophylaxis launched the XXXX. behind first

of there well With this patient HAE Japan physicians in the mind, their the partnership, in U.S. and patients identification Based excited care to to be to building level supporting Torii will prevalence Torii both HIV in focused patients estimates, raise on the efforts. education Japan. X,XXX a leveraging are market HAE for help in from the disease with on Gilead is success comparable our standard commercialization and as awareness patients as partner,

XXX registry. approximately local only the in today, are However,

when In will what education, focused rare and for and a commitment This there as diseases, seen happens increased we've is treatments available HAE, diagnose especially key priority Torii. become awareness patient innovative follows. in investment be and broadening

The of treatment of as Japan. well their complements potential the for first oral of dermatology the in positions allergy an Torii existing broad prophy and addition, Torii's In launch products. Berotralstat reach medical once-daily representatives portfolio drug

continue as the We success partnership to Berotralstat. this for of a great see

partnership. it contingent milestone threshold. clearing Bill, the wanted price our turning we I to over million recap $XX from a With receive a to stand economics approval PMDA this minimum payment to upon year, Before

range In commercialization of structure to net us allows status. to XX% addition, Sakigake This on sales tiered the based on Torii's royalty upside would participate XX% payments our in from success.

With our program. now that, D it oral on over update Bill Factor me to let turn to

Bill Sheridan

a promising I'd good complement. Factor a and start BCXXXXX by approach to involved alternative for is in with moment morning. such Charlie, why of to pathway share diseases targeting Thanks, the D taking like

means other nephritis as is XXXX alternative that D succeed independent will PNH pathway of pathway to target of a is setting. for Factor Factor the And pathway, alternative required inhibiting doses D in is D in disease Factor alternative and work. same it the as First the That particular diseases. block that the

just diseases PNH other the pathway, So alternative for of proof-of-concept provides proof-of-concept in not PNH.

to drug other Next, as lowest it complement pathway Factor any compared targets. of This less to means D that need inhibit enzyme. levels you complement

top not that, increase Factor do D of illnesses. with On inflammatory levels

So get not the dose does patients need illnesses like be when changed to influenza.

unique Factor likelihood of Lastly, effects in enzymes, specificity leads scientist a to other lower able therefore, to to XXXX better like proteases. been D contrast better that structure serine have bicruciates a safety margin. have and design intrinsically complement a other This inhibitors against off-target and, has

of treated to attractiveness in been very announce not the PNH XXXX. excellent we today target, ever that patients our D PNH, proof-of-concept excited were Factor or Considering with as have whether is the an inhibitor. our oral In PNH a to we develop CX monotherapy study started goal with with efficacy, have

of where dosing experts pharmacodynamics we I complement, proportional healthy linear recently, diseases large top safe in margins favorable with discussed PD meeting preclinical exposure XXXX, world and profile pharmacokinetics advisory returned and safety with and an high and subjects, outstanding to in and dose and Just of profile exposures. from the PNH

but expect is being see enthusiastic be and XXXX meeting, inhibitors. prospects the D these the inhibitor positive potent with that superior oral XXXX in experts like I to What strongly successful assessment about in I'm was confidence now. even the exposure an Factor for XXXX based PNH, excited great experts, before on their will more CX to

started shown now to Because rare achieving is We're proof-of-concept drug of study designed and a the PNH adequate XX. in step such look To study proof-of-concept seeing the study as Slide that second summarize for is through we dose as quarter. this it a on drug the to in matter the just their levels. scheme and feasible, proof-of-concept maintaining excited ranging evidence quickly of forward disease, has as advice,

for for days. for days. planned Twice milligrams in will who regimen receive or XX subjects milligrams milligrams twice will first for as is followed improvement LDH for total a daily twice a days, days a Subjects XX XX the the the treatment hemoglobin. XXX by day levels then continue daily day cohort such in showed XX for XXX day in twice benefits, XX a dose XXX Subjects days milligrams second twice XX cohort, of

of their not for patients two enrolling inhibitor, and are have but XXXX inhibitor, two We patients, only a still CX – types, are are these response who two, treatment; poor had CX a patients patients taking one, who to will be on treatment. a

patients, will be CX to For XXXX those continued inhibitor. added

goal first protocol treatment subjects The been continue allows naive priority. enrolled rash have to data XXXX benign sick very are XXXX a proof-of-concept patients patients for a first benefit with patient with of in the PNH any from Because the them, patients dosing. is could treatment study naïve our monotherapy provide and to

will I quickly discussed expert and and advisers the resolve expect and that rash We to benign a temporary occurs. before, events one-time amount any that

med the the subjects portion the in saw we dosed what in is this recall, may through healthy of we trial. you As

to We Hematologists in that with have and clinical patients are expect clinical just level of of baseline improvement in and for this hemoglobin. seen learned when laboratory from efficacy very a compelling. PNH, activity LDH even see of is looking handful evidence type in

for complement the is goal same, much no is program pathway The PNH. our disease. the alternative broader But of than matter this

no this disease, Factor and repeating and pathway an and space, So which D to and leaders approved other result set glomerulonephritis reinforced suppress kidney in have for also was Oral that worth many uremic can very opinion it's is membranous dense we including diseases, is treatments inhibitor such directly as acquired key us proof-of-concept by – PNH, and syndrome. nephropathy, complement, if rare a important, of in this demonstrate the applicable currently primary hemolytic alternative diseases, deposit CX the

in this data diseases. and other our PNH program So following proof-of-concept in complement-mediated we advance PNH, to intend both

We forward our and look excellent from proof-of-concept expect much have a study, in very data strong quarter. clinical reporting the and scientific very results basis to to second

Jon Stonehouse

Bill. Thanks,

we're stretch fantastic to trial will on be and excited and about having why see BCXXXXX. a XXXX results positive with the pipeline with so can attractive You in Berotralstat. home With possibilities with by both very a our proof-of-concept from the approval position launch

As at the end in you quarter year, million million ended the for that you press today, reflects with the we we RAPIVAB $XX does payment but last look last month guidance cash of not added the the million. fourth in reflect year balance our sheet of see $XXX last our shipment for received the year. the release at we year $XXX and This

funds We net in be our to range XXXX operating million XXXX to runway cash for cash in and the takes expect $XXX us used the for activities proof-of-concept of and $XXX million. Berotralstat. commercial prelaunch This the through

have we course additional that, the pursue the financial of flexibility that capital we of to over sources Beyond will year. provide

for We approval million Japan Torii. in expect up from $XX to

to would We at to bring options capital, add data launch partnership proof-of-concept are to have financing as with Berotralstat program. advance approval royalty that BCXXXXX, fund such evaluating in for the capital that we and a the with

starting by revenue drug has pipeline product million with with behind patients diseases $XXX that BCXXXXX a more in programs followed north of it. a next peak generating and year to be molecule going sales, for early right oral a in We're with rare

of of supports I are said call, beginning the All moving the a this company. new at era what as into a we

patients that the outbreak quickly the spectrum of you. can coronavirus outbreak. situations need the broad government treat we used be antiviral, of that galidesivir, call Q&A, information how spectrum up latest governments with a we to deployed wanted is could be world received inquiries the in and broad current be with the prepared to This we've Before on share like U.S. for our around and to lot reminder open a with the COVID-XX, stockpile antivirals

why the RNA our is galidesivir. government we've more including partnership past active years against the nature X families, and with research Prior several published in over coronavirus. XX in advance is This galidesivir has to that continued viruses different than shown

this galidesivir has trial specific well have The X volunteers, virus I under a we opened but safe activity galidesivir tolerated. healthy clinical causing Phase we do a fever in NIAT. was with and against with generally COVID-XX if is a galidesivir know Phase yet year, last corona we And contract not yellow but trials and in virus,

development in have we XXXX, that relationship with contracts them might and under Since galidesivir with emergency. the totaled we government, in working global health have the NIAID figure of we with been this Because $XX to partnership BARDA have million. and the out, we how been help

We are working to the against get galidesivir strain. tested

how supply. can We we in are and clinical evaluating drug participation trials increase

government with partners, are being is following done this their we of our lead. and All

As these we you. activities develop further, update will

open we'd This our now Operator, for questions. up it remarks. like to concludes prepared

Operator

Liisa with first Our Securities. from JMP comes Bayko Instructions] [Operator question

with your proceed may question. You

Liisa Bayko

thanks on products. Hi, for the question. Congratulations my taking all

Jon Stonehouse

you. Thank

Liisa Bayko

Just process. have than little bit and provide could Adcom’s any This you we different so so and where might other is some kind forth. if regulatory -- the processes curious update on on on of a

of we visibility year-end So don't have approval. we when lot a between and now expect

us you helpful? whatever kind XXXX? where So on are any of you. of you interaction, any terms be updates in Thank And would can review FDA give

Bill Sheridan

process. mean, straightforward I Sure. it's a typical

earlier, As FDA. the our been accepted we announced by fiber has

We typical that X and and December routine progresses. as questions answer they have the of date are a regulators review PDUFA we ask

in that not lot really there’s will a typical and go report there we way. expect a So to it

Liisa Bayko

you about and strategy that on how your thinking you Any pricing any that color you're equation? -- can have in share

Jon Stonehouse

Yes.

data his last I shared from degree the team did changed year. in we what around -- Day our think when to the you back research the and had that we Charlie with Memorial in view some summer, got

XXX lives. or piece an the what I this do PBMs scrapped over that insurance XX think it in to cover with million surveys we we lives went were XXX that into thinking, when million think needed Charlie cover I originally, significant of companies discounts important was

as our we're the they an a between range long get oral get and $XXX,XXX which reimbursement. is a competitors, that $XXX,XXX That said in for of year and basically somewhere as year. will need They

but payers good made haven't information we decisions. have us, the what was for any final that us told So receive. That's to

Liisa Bayko

think Okay. And do that how about in region? you sort the then European of

Jon Stonehouse

the Yes, competition is real discounts. in cases, think In see pricing in it's some all price it's therapy And market over there's significant now. Europe the in where acute rare rare cases, prophylactic opportunity you other drugs. therapy, you in diseases. no interesting for disease that a for an board right We parity, Europe see largely there's

making, and in us countries patients to they're the to of ahead we and to get -- to get remove competitors the the barriers much watching the is the access approval as moves that We're goal where that we access. and

So we'll see unfold. how and things wait

Liisa Bayko

Okay, great. hemoglobin. terms then XXXX, just changes about on you in your of And can talk LDH, in goals

concept, of your you as, those proof kind what your objectives For of up are actually? for what's on

Bill Sheridan

for couple of quickly, objective. question. through that's ranging an are dose Thanks a There the Get objectives.

it why way that's designed in so And described. I we've the

I Secondly, and see down that and here what how the LDH mean, the hemolysis extravascular quickly and that to hemolysis turning the goes that's the expectation D hemoglobin and up. very will block all to events goes is blocking Factor of attractive. intravascular extent downstream

our At range happen. the to expect fully the had the come right LDH the go they hemoglobin into the and that we've to feedback proof-of-concept, we'll The expert that in normal to expect advisers is from exposure, up. discover we back which

situation. So that's monotherapy a

very data and to naive first we're be much seeing it. Our looking forward will subjects, in

I a more bit difficult. quantitative think that being than is

Liisa Bayko

akin do it's, as sort of, where as if like feel you do these Polaris oral there HAE might of about you enough. molecules? other that? to some think an How you think option. good be some need you to you Okay, fair be and Do have flexibility Or

Bill Sheridan

see, from that get CX maintain upstream the alternative because an need adequate in of this as experts I pathway will mentioned, disease pathway superior inhibitors. PNH that. CX alternative can of expectation we if we and The if the position the Factor be exposure is to Because to D of and

Liisa Bayko

great. Okay,

and see Thank us? investments? forward that. maybe few molecule. be just to would of talked pipeline you And what the next prioritize you Looking for then And finally, making visibility where next you. points about you your Can

Jon Stonehouse

interactions So once out what the priorities proof-of-concept understand will what data. be we we is have need that regulatory really to to turn

explore to And so regulators. that we intend several with indications the with fully

have these disastrous of outcomes. them Some affect and therapies, diseases no some children of pretty have approved

So the that, like we'd I think course. invest across of our board, reserve to but judgment on

Liisa Bayko

much. Okay. very Thank you

Bill Sheridan

Thanks, Liisa.

Operator

you. Thank

Our next question Fye comes with from JPMorgan. Jessica

with proceed may your You question.

Jessica Fye

my on to on differences starting elaborate I questions. guys morning. Thanks close the hoping see And you taking I remdesivir? Hey, morning. coronavirus? any good comments contrast product work could the between how maybe compare was further guess, or current your you are and for and can galidesivir you you similarities this

Jon Stonehouse

Yes.

So That's they're make spectrum, the start I me much broad in as of we with can comparisons. about polymerase as both RNA latter let inhibitors. mean, first. terms

reminder rather of remarks, spectrum there's I reacting is As that such a said this stockpile, a got to of get important we've broad preparedness. into the because than in the a prepared antivirals lot lot

increased has a virus, NIAID And so conversations and that evaluating all and relationship in place, got were testing motion against out, clinical BARDA, we and in drug have laid I in because in working on. the a getting and we've that and currently, supplier, with been things things trials we're team looking contract at our three participating the

Jessica Fye

And data? to to releasing also street? fill Would before And you say study? many dosed before so many can any you dosing of Okay. far you complete us data the reporting initial want the see patients XXXX to how remind in cohorts want you've these you how can

Bill Sheridan

running. question. not And Thanks great on and It's give The of our announced doesn't trial we've for news. started. recruitment, patient That's usual a proof-of-concept a is blow-by-blow up practice lot here once the need we to it, patients.

designed second quarter. be the go. have assessed with And and just past, as few in all a we've the that terms as of we may subjects how outlined slide. is on seen many need. in be might or may some proof-of-concept patients can we'll programs just those will The in other need fine, patients to that So, of We us we the flexibility protocol give are not

Jessica Fye

you. Got it. Thank

Bill Sheridan

Thanks.

Operator

you. Thank

Tazeen from Bank America. Ahmad comes question next with Our of

may proceed your with question. You

Tazeen Ahmad

questions. guys. Thanks morning Hey for taking my good

big ones just on D. developed, next Since and think get factors, are on asset, from that inhibitors of follow-ups? the how from color the Alexion other might I you your Factor to namely that's some maybe wanted Just from your a you then couple molecule differentiate pipeline the view a being

Jon Stonehouse

So saw at in the out I fill to of early is programs other that fibrodysplasia it's rare future. seen impossible with disease. predict just in development ossificans palovarotene these for to we've think blow example. disease And patients a the what We progressiva that stages, that's

now stage. a So, there's of are competition, programs early all so, oral very the less that at

think we're the characteristics I of of information on competitor the very in about that rather excited to molecule, our so molecule, comparisons. not it's is about hard a the domain lot comment public

Bill Sheridan

see Yes. thing many the oral ability this last And players is, towards so a the for I you and oral. I'd space are this market in injectable multiple the say players. mean, is huge how

where big players a is this really play. multiple market can So,

Tazeen Ahmad

at your And already. going terms have proof- doses ones you looked like than that study, might of-concept great. Okay, then, looking the it of at in you're looks to be higher

your looked think trial. for XXX at mg you I previous

big So, are go you're thinking let's balancing if with say let's that based Is you physicians? how about observation on XXX, a up that of you any from what rash? hearing to deal say out

Bill Sheridan

dosed when the quarter. and now. We data day XXX MAD, milligrams milligrams We a the in have day a subject report successfully we forward twice XXX a cohorts, from twice second to the the proof-of-concept healthy data sharing those, look in

the And and seen in to is the been dose we've a the majority remarks. in has in that's rash PNH to as So, our the as cases. people outlined in through been before, PNH able we've in plan. use day our of mentioned MAD couple a of study XX in to XXX-milligram in Overall, twice those and plan I doses dose addition the proof-of-concept patients,

happy the about of we've that so -- and far. very, drug profile So seen feel very we the

Jon Stonehouse

And the Bill, the tolerability this, was? issue balance need KOLs the disease between the the about to versus treat to you when the talk disease --

Bill Sheridan

a the approach. I are sick. conversation. an are changes it nephritis mean, patients as the And there's PNH people totally of benefit, expectation very was disease It Sure. very with simple I when mentioned sick,

expert allows temporary protocol be people us will with the the that inconvenience. continue rashes expectation to from things and So, the immunology benign is And a these and dosing. our advisers hematologists from from

Tazeen Ahmad

lower in built allows dose? there Is to any you part protocol the that the of

Bill Sheridan

expectation by the what dose a The away will study, is with part limited through. that's rashes we of And saw dose the allowed healthy with continued people of in itself. subject where to that, rash go couple cases the the that protocol same the in

the plan. So that's

Tazeen Ahmad

I Berotralstat, And Or, it's on sorry, to Okay. name. could a squeeze to get to on take that in if me XXXX. going used while

in provided So, have the they what's drugs higher can case? that disease is that historically, Japan, U.S., still the been be for rare than in pricing

Jon Stonehouse

when think, as have well. some think Yes. a the Like is -- we I of Japan said I Liisa I disease rare before, on helps was U.S., prices it pretty country the drugs. there designation, about The that outside had question high where been that fact Sakigake have

also positive two math occur into the it You the effect occurs. Japan, I in premium pricing, that price or reductions a a in years and whenever that has for that every get goes think,

that and other that prophylactic and there's Biocryst think believe a I price Torii yes, in no market the especially benefit, reflects So, both the fair where innovation a therapies. really

Tazeen Ahmad

Thanks, guys. Okay.

Jon Stonehouse

you. Thank Sure.

Operator

Thank you.

question Tyler Sandler. with from next Piper Van comes Our Buren

You your proceed with may question.

Tyler Van Buren

a strategy you Hey, guys. bit you data? Good your little just see after experienced potentially proof-of-concept Could in naive morning. patients CX more about future and speak both terms going clinical of once

Bill Sheridan

of terms. development here way. with diseases in and advisers happy general usual of the activity the to pathway drug program complete for expert in array wonderful complement. consultation that by alternative will help other be patients there's describe opportunity agencies it in regulatory with to But be diverse defined of this exact The a driven I'd strategy are

how or will that's So goal superiority like it's the studies premature monotherapy CX program. this but what and drug need, of and the look to expected, are predict inhibitors exactly subjects many the we'll to with

Tyler Van Buren

to but just comparison on comment and could Okay. about recent the thoughts mentioned, your And what you there? Maybe on if CX, maybe data you the differentiation CX. published potential

Bill Sheridan

Yes.

pathway So think you way That's it. Factor blocking the to about is CX in alternative functionally abolishing should equivalent diseases, D.

volumes is large or treatments unattractive are think more a more far attractive. I of week for injection twice patients, oral a subcutaneous

Tyler Van Buren

Got it. Thank you.

Operator

you. Thank

with Our comes next from RBC. Brian Abrahams question

Your your may question. proceed with

Brian Abrahams

that XXXX, taking existing know so my little I perhaps any me. you Thanks the types love foresee few for on more viewed that to Would certain questions. XXXX, a ups. are their a you thanks for being therapies had really are managed patients off Hi. for types attacks be with terms as update. may A bit often? whether in of market the or few better have to research Then of interesting of switching switching much follow clarity even Charlie, equivalently guess, from I there more

Charlie Gayer

Yes. that. research, types consistent in oral No, Patients it's is our and therapy. our what interested for who in saw great question. really is, come conversation Thanks everybody. are from we And patients all with with this market

And in already remarks so market survey of on mentioned my in they over were have majority about high I actually therapy, injectable coming the well patients and our doing on the as research, oral. in interest a really and pretty to

think pull we So different we're types going of patients. from all to

Jon Stonehouse

in current therapy. patients add would are satisfied I that their Yes. really these

is and half you so to are oral. willing answer, patients to And is their our breakthrough current and switch is therapy asked just of want Brian, well are satisfied that inject There burden these have some And of attacks simple, Takhzyro on a they out have are want more. controlled, the therapy. And they very when to that them of having the therapy more real us, that their answer the it people is folks are no. very are And interested? with

Brian Abrahams

they looser maybe have associated stopping Factor rash? then to And dose study, volunteer D the Great. protocols the any do patients versus dose clarify through rash? the on drug are healthy the additional it. program, to to Got insights patients can guys you potentially And any enable you more differ potential into how the

Charles Gayer

Sure. Happy to answer that.

things are expectation. so insight the it's huge things So and you was by drug with look here these because the right, clinic driven drug with rashes benign the the of and develops limited physician for key the example. clinically, who self a runs is when benign and of a is a it's the amount And dosing. common, the Bactrim, benign these And continued down with microscope. allergy in field that's phenomenon, desensitization that pathologically experience that are it's skin local I tolerance expectation chatting biopsy and

this the a in learning that there's of nature benign. associated field So drug rashes are lot completely of of

benign X disappear, rash, is expectation for that dosing monitored. So that's and safety are protocol continued dosing obviously of develops our And are clinical With thing studies to The the putting days average patients despite the regard the allows will protocol getting patient carefully patient or the continued key there. the last rash an the first. X a done a drug. of proof-of-concept, drug. If with

So the that's plan.

Jon Stonehouse

Bill, have they use they cream. And can if or antihistamine itch,

Charles Gayer

Yes, topical Yes. treatment.

Brian Abrahams

you there? just then remind you IP would government the this on fund? Thanks. that control over something relative full how is coronavirus's studies you consider then Can or last And of for us economics that And the just of life BARDA the would to have fund remind the it. one just long Got Is your potency the and types drug's me might related versus are galidesivir. studies you on us these -- asset?

Jon Stonehouse

of lot a That's questions.

you to the other might take Bill, first? So activity want coronavirus's on

Bill Sheridan

Yes.

galidesivir they lines. viral their RNA cell the triphosphate inhibitors order do with work, So in like in up a disrupt The lot into lines And an to ability taken of of done be to to testing feature have cells. into in converted that. order vitro is to vary the RNA interesting in synthesis. is polymerase And nucleotide the cell

around with the in activity cells the to there's which vitro normal had to trial, So and government XX a Vero for go EX mind. clinical decides with the you has in in At whether efficiency has you day, assessments learn take cell have in to vitro end ECXX micromolars. the an that and compared if with a the in forward MERS, of We've line, activity drug trial, XX or X% conversion making human that about to example, And the activity. the not about the was clinical coronavirus.

Jon Stonehouse

there in Yes. around are Brian. the virus some And the U.S., in of China, There's your now. answer a to clinical question on is moving trials, them number

assessment high-quality of well. And gives so believe And way that gives study a that options good exploring testing also, give answer, done so all a is outcome the we as in we're the clear to different and it. best a it

method of out IP, use we regard With to to XXXX. have

with And governments, customer XXXX with to in we product. mean, a with regard arrangement is have pandemic. and This this ultimate space, here. the And global so the the one right? and there's then formula with and there well funding, of with runway governments working The plenty commercial not we I health customer HXNX right? government so RAPIVAB, the been been is has know it And we've emergency through

resources not the we're putting – this funding government So it. because is towards BioCryst

the to we're at people the that this. I following And lead We said have the day. as government's of end dedicated are on this before,

Brian Abrahams

much. so Thank helpful. really That’s

Jon Stonehouse

Welcome.

Operator

Thank you. question [Operator Wang Gena Instructions] from with Our next comes Barclays.

proceed may question. with your You

Gena Wang

on few taking follow for my Brian, a questions. will questions Thank also galidesivir. you I more asking

if color galidesivir the give you regarding Gilead any wondering from and activity compared Just to then… can

Jon Stonehouse

Go ahead. Gena. go ahead, Well,

Gena Wang

I can ask the rest later.

Jon Stonehouse

Okay.

reference comparisons, you don't we will that the SARS front vitro and send in me to – MERS and aren't that the set like to There answer. the that have John and I'd nature now. we you have so of I So activity you in but you because point can link, a get publication, things right can – show you it

Gena Wang

can moving very also, a preclinical you do have study? That's seems think to And you clinic? how study it helpful. to to soon do before – you And move clinical like Great.

Jon Stonehouse

I Yes. health No, a this global mean, emergency. is

things with that But working I are things rules three we're the So the the emergency in think in on. three an about government the very that COVID-XX. currently we're are When you're like before I different. talked actively with

be in understanding of humans testing second of would how clinical this our to participate helpful advancement virus it trials, testing to program. the the galidesivir, The in specific of very with is strain

is that. drug And evaluating we're then so the And supply. third increasing

Bill Sheridan

healthy your us the subjects, lot to dosing helps preclinical been pharmacokinetics comment quite about to successfully Phase information and in so administered the and just Sorry, lot. a volunteers a of safety the about we've Galidesivir healthy the which drug on has of testing. question in I studies, two got

we in opened study that recall a you And yellow fever in Brazil.

a So XX animal relevant model the – disease. for COVID nobody has

the of So or with any predictive value and to decision-making coronavirus anybody's efficacy is current human for relevance model animal guess. this

relying is on stuff. nobody So, that

Jon Stonehouse

around, in is another another more inhibitor is discussions the And with the virus. There's work we've question had government to like on which one virus they're issues may choose one government would if they'd of on antivirals, polymerase stockpile, because than spectrum resistance, broad the RNA have the one the antiviral broad spectrum right? Yeah. work may

more from one. So that need all they indications is the we've the gotten government than

Gena Wang

governments being you U.S. the any when to are Phase will in referring government, are discussion? trial? that then you how move other or only And X think And Okay. you you see quickly that, to

Jon Stonehouse

Okay.

so focus been Again, believe, a difficult years to So it's since, of NIAID year. X, the when and me a of a with both since health you're Phase relationship global with change sure our now. terms relationships, for rules we've emergency. and And in it's number in U.S. government had it's for the that's the I predict past right really BARDA

to vaccines on think help? take is news that I emergency the people the health global a infected With going how now getting while. heard important are thing can and this you've because we

only right the treatments So are defense really now.

we'll we do that. to so And if there, be help available can

Gena Wang

you Thank very Okay. much.

Operator

Thank you.

with Belanger from & Our Needham Serge Co. question comes next

wit may your proceed question. You

Serge Belanger

morning. Hi, good

of couple a me. questions Just for

MAA – remind looking European a your ready then the for filing strategy get on for you As is partner? what will be here, again can – you us And you the –

Jon Stonehouse

No.

Serge Belanger

No.

Okay. pretty That's clear.

Jon Stonehouse

question, Yeah, but I'll ahead. go expand finish your second

Serge Belanger

to is related one guidance. other The

So go ahead.

Jon Stonehouse

Sure.

experienced expert hiring, doing got his work in team team, already disease a and Charlie GM we've really leader are folks. is rare in and So that and Europe Europe an

pricing Europe is And Europe disease One and hands-on in it's rare beauty in in the big of experience and that process drugs for get to various negotiating the in countries have things concentrated. people pricing actually is the had through the Europe. super

of of a the U.S. of in necessary is resources. what a take doesn't small fraction a It's it ton So fraction

no, ready there, team partnering for on not So planning and we're the building that launch. we're getting and

Serge Belanger

Okay. on guidance? launch how for give in And of little going? maybe, Got the And new is is on the R&D spend then the it. XXXX? us how guidance, color search can CFO And more a the into spend then if much built you

Jon Stonehouse

Okay.

guidance. the So start me let with

that all all the activities, So all the of that guidance. to be the work sales force, to the – in buildup the is of ready prelaunch

So there's skipping no that for on sure.

building the your So was? And organization up that, question European all and of well. that includes as second

Serge Belanger

R&D?

Jon Stonehouse

in is So heavy on the you front year, is the of but manufacturing of because Clinical the over think closer the the to down of guidance increases year. the a the heavy course at as manufacturing way and closer commercial time, goes launch. and look front year we're getting the should I and – bit then it's

how at But is kind all it the going at point fairly of quarter-to-quarter. and spread up. a are to get or going lower out point So evenly components balances high various down you a

Serge Belanger

CFO then And Sure. the search?

Jon Stonehouse

Oh, I'm sorry. Yes.

going -- well. Now progressing it's

So we hope have to some soon. news

Serge Belanger

All Thank right. you.

Operator

question final with comes And Maury Raycroft Jefferies. your from

proceed may question. You with your

Maury Raycroft

taking Hi everyone. Good my morning for and question. thanks

I'm So for wondering Japan.

Japan. status, any provide you you does ramp Japan are you that getting inventory registry with the like? in into launch about what So warehouse? progress estimated the to in have they're then anything in perspective and radar could XXX made Sakigake revenue the reimbursement the say potentially And X,XXX in can patients if on new accelerate And on a mentioned in side? Japan, the patients Can but be look

Jon Stonehouse

Yes.

experience Torii in with we successful the have, just if have. only XXX guys of they Charlie They one also partnership, reach the that correct chose think, to but is, reasons Charlie, mentioned representatives I'm I that their that Gilead the me had wrong, close So Japan. not these

and actively they're So like. the work medical doing affairs

life. And that has is feedback tell once oral pill a gotten the we've once a a X -- right, it were day fact sick. from investigator, the had we've investigators Maury, how treatments, you're the an I market day, mean, that patient fine said actually because changed they a patient us We but their aren't they around exactly there forgot got

resources around disease world you And you and incredibly have the educated in you the around that change causes to able you're that and high. therapy, enthusiasm treating dramatically market the excitement for physicians that pharmaceutical have is, you and recipe quickly. kind the the get are the patient what markets association have is a when the this in So and seen -- other we've of

Maury Raycroft

of could Or revenue, happening thoughts? that contributing your by are Japan start the with what Got to it. And XXXX? end

Jon Stonehouse

Yes.

think period and then never since And one affects was negotiating is had price. So just the the clear, this approval. to of Sakigake And negotiation part, if we've there you're on give I through with add can't about you you Sakigake how process experience. that of the so XX price get another is the a process pricing. process been be XX-day other a you and the opportunity questions I lot to still days, really And

Charles Gayer

asked if just it XX speeds -- It time, over thing to the So mentioned, gives it doesn't speed the and pricing that in prestigious doesn't Sakigake some drug up it's the we but up initial then as for I speed you opportunities think up. days. X pricing. It it XX

Jon Stonehouse

drug got clearly, it's huge But highly if need. the a unmet Japanese Sakigake designation, innovative a it's for believes government that

So I think important. that's really

revenue certainly. in all In this well. you add next of -- I terms so stuff it's put wouldn't year, lot put year, as I that -- of a royalty if a any together, wouldn't remember, but

Maury Raycroft

Got it.

based guess in Congress expanded galidesivir, funding already could through now. from Okay. more does right become the more positioning that's U.S. galidesivir And And then on U.S. $X.X important, for arrangements billion one if coming have BioCryst trial Gilead the just But government. BioCryst successful? be you is on the funding to I with

Jon Stonehouse

Okay.

So one first. let me take the first

the because direct So that doing both it experience place, RAPIVAB. process contract galidesivir of get with the pretty we it's from in absolutely beneficial is laborious, and to all have have contract a years and a to

better government start the the to So starting has new ability from is the add to scratch money much that to than contract. contract a

worth. see, the existing there's so still mean, And million money $XX I we'll in

end activity drugs the money the idea just spectrum know of multiple absolute I and certainly antiviral the going next, stockpile. that it's away must. it's additional but then But said it up broad to a It's that in whole right? don't before, antibodies not progresses see And at what's the an take added pop we'll day, is and that you broad from like. of the spectrum are yet, to no vaccines And So how option. a have been but an must that has

antibodies. the at shot takes have that a make And to better vaccines. again, to so if you've activity, got it you takes time illness addressing time It then make quickly. And

we'll hopeful it's for important so in And have options. to space, progress rooting this that as we galidesivir. that really we're because I'm continue well, remdesivir

And so government work take we'll their lead. and with continue to the

Maury Raycroft

it. Got

you very much. thank Okay,

Jon Stonehouse

welcome. You're

Operator

like Thank for now turn concluding to Stonehouse And would call back remarks. over I any Mr. you. the to

Jon Stonehouse

can year So a for as be that with I molecule great proof-of-concept in beginning, will at start an with we're the said that be believe a asset. transformative one a we believe we and off entire company to approvals pipeline this with

So BioCryst. progress course as year. we for look a of in interest updating the day. Have our the continue thanks we over to And forward your good you as always,

Operator

Thank this for participating. today's you concludes and you. Ladies call. gentlemen, conference Thank

may You now disconnect.

BioCryst Pharmaceuticals (BCRX) 5 Mar 202019 Q4 Earnings call transcript