BCRX - Earnings call transcript Q1 2022 | BioCryst Pharmaceuticals, Inc.

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Operator

Good day, and thank you for standing by. Welcome to the BioCryst Pharmaceuticals First Quarter 2022 Earnings Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded. [Operator Instructions] I would now like to hand the conference over to your speaker today, John Bluth, Head of Investor Relations at BioCryst.

John Bluth

Thanks, Daniel. to conference quarter call. welcome and corporate and results financial morning, BioCryst's XXXX first update Good Today's available on release our press and website. accompanying slides are Anthony Charlie me Chief Dr. with today CEO, Stonehouse; Participating are Commercial Chief Gayer; CFO, Officer, Medical Officer, Jon Doyle; R&D and Dr. Chief Bill Officer, Sheridan; Thackray. Helen

we your Following questions. our remarks, will answer

These known be Before performance call to well and results and contain forward-looking which from statements information subject note as forward-looking and/or future statements, conference implied risks please are we and performance uncertainties, or future may unknown results, regarding unaudited in actual or today's any including this or expressed cause the results materially our future company's as performance achievements. achievements begin, that will presentation. to those statements financial different

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

I'd additional can a including refer Exchange the company's risk discussion Securities to Jon accessed filed our be Commission, the on like to Stonehouse. of and information, For the website. to with turn now documents call which over factors, please detailed our

Jon Stonehouse

With revenue the demand headwinds of global patient and peak nearly $XX this a confidence in revenue of that and quarter sales world impact strong payers some from and requiring team for expected donut get why they? as And on quarter, and still ORLADEYO creating reauthorization, that this successful There's be one the doing products, important the Charlie growth. role talk our opportunity in a wants on $Xbillion critical copay into plenty a it position. John. face And to stream evidence ORLADEYO can remain year, capture sales. for HAE no by had real What's of and into capsule $XXX this discover, hole particularly physician we patient tapped net to revenue track providing than commercialize who our higher they million the strategy. about quarter-over-quarter deductibles try in strengthening medicine this will we in is to would by important Medicare financial detail and things company every quarterly If with and on our first to once-a-day. year, world. plays the continue our more to is to quarter-after-quarter revenue develop evidence ORLADEYO to less this controlled provide we're value produced million us Thanks, chance go the too. around net growing

with far expect each and the the sales just there a So quarter launch. over for we $X of these million. will revenue on, year, of doubling trajectory of so That's quarter first no than the less remainder X previous we're quarter, expectation the what of this sales an we of culminating that the first-year on growth than see targets. year, the annual our billion steady revenue confident the be peak a more and remain we way achieve very to can in Based years $XXX we

part our important another is pipeline Our to strategy.

our in made patients the ago. first creatinine month this clinical since identified in BCXXXXX a has of team investigation increases progress significant the serum Our trials, in we

highly to Factor mediated our suffering from goal something safe know complement competitive many patients. D a rare paramount remains because to D XXXX, the While and elevations, on there's we'll details is this outcome, and more the believe will dose investing very not oral waiting. is share with or inhibitor Factor are regulators to Whether to it's a bring backups are Regardless likely to finding us path the to see diseases complete, cause of both, of patients committed patient work remains a most our in we or safety forward topic. effective Bill we inhibitor these of one clinical if trials. we restarting

this profile follow the will invest product it decisions. decisions important to in changes, and in also and us be thoughtful data on competitive based not our factors allocation our reach landscape how what in, Time how will capital helps to what to invest We goal. on and market, make the

look can when revenue So step peak. our coming grow will in a with at launch product we $X believe company drug it's a successfully billion team at from you'll you that's and a see company entirety, a back showing and you a growing that a its stream and to they execute

a Charlie to even sheet even we balance has ORLADEYO. to value solid meaningful innovative and a have the over have even further. products creation goal have see greater We the believe continue capital that, allocate finally, things diseases. fill bringing call to to of platform you And And molecules today to from of create to rare you more greater value about now turn with will market that and see patients suffering creation a to all more those discover our value. the discovery I'll we the we pipeline talk for When and to greater to value even future. create pipeline

Charlie Gayer

Thanks, John.

strong and million sales headwinds for The reauthorizations, the payer we of no another with to patient finished with even are on year. track in was for one less ORLADEYO than continued for and million the quarter ORLADEYO. first sales $XX.X base The $XXX grow in steadily

how these of ORLADEYO on far each that our injectable are we're is by prophylaxis and it on emerge. We're about trajectory and largest of who are ORLADEYO. and sales controlled before if confident growth. What well most is patient's still on stay therapy, increasingly stay well for and to size becoming are seeing because who -- the therapy doing cohorts likely treatment deciding doing on are is great three are is are is Those who the clear those this seeing best isn't. patients on those those doing of decide groups and group staying an growth driving the those the well well We the switching. it do but is who fluid, are patients them, The

switching described because making fluid at to access within setting remain months improved free product. effect in data median prescribers continued out because benefits ORLADEYO that to Further patients from patients the patients also The have overall evidence XX% members side after ORLADEYO of XX% the QX half continue per This access One-third We're believe ORLADEYO from about ORLADEYO. authorization The from year. TAKHZYRO. came with last shipment safety their cohort, prophy similar of the the they switching switching shipments. leading just patients in seeing XX% don't therapy are important of give support I pharmacy, have some on well, prescriptions TAKHZYRO showing in were at for data patients base. If patients early were quickly. on evenly expectation prophy long-term and our an months the processes example, for starting a Haegarda are probability switched receiving specialty and is TAKHZYRO two-thirds with reimbursement the who doing to on discontinuations switchers months physicians, are access of in split long-term consistent patients drug, originally no -- patient new months. seeing continue up now retention. because for the is added patients patients long-term too team that is for patients rate quarter, represent sure first of new they described quickly X evidence on patient showing XX% least are from depth real reach a changing. consistent has prescribers. patient XX the long-term the XX of the from two phase also on of on those convincing that last XX% clinical of might of QX free doing process. from patients evidence more high patients of a correlates in our Education they QX for trend of half new up critical It are equally are of paid We controlled patients patient the decision treatment. well well-controlled showing And that is acute-only retention all and of and That improved was nearly after another of but retention. second the well the miss also make to we on ORLADEYO. XX-week only real-world greater compared one therapy, at those from XX% sizeable of reimbursement growing of These all ORLADEYO TAKHZYRO switching size world in between study in to ORLADEYO complete, is about that who well-controlled ORLADEYO. of for and prophy patients the are first ORLADEYO baseline, and staying pivotal and are or and attack trial this I study and in XX% with therapy breadth how shows to prior attack example, cohorts have date occurred and are that too ORLADEYO because zero to and after breakthrough because and XX least from getting Patients In who repeat out want on switching Overall real-world on evidence We the over that on are XX% from attack ORLADEYO our we of early we not injectable sign paid trial. paid

coming treaters base broader in the from HAE We also top split and Tier prescriptions saw an of X new XXX treaters. even our

of quarterly prescribing next tells for of over opportunity and how surveyed to X TAKHZYRO patients and who average Our market more prescribing another neck with an us again, We trends treat latest predicted HAE were XX% allergists growth neck XX to future the already the ORLADEYO XX% allergists [technical survey and prescribers their much months, XX remains. They leadership. matches difficulty]. with

patient to that stable us million favorable to U.S sales outcomes, who treated generate of peak, confidence least a we base expect steady share year. and patients drive no way trends great our outcomes patient and and $XXX $XXX trends alone. have the see on of would million new diagnosed this reach the growth U.S to the That's than in we XX% at will We less from at X,XXX out improved quarterly growth The to the patients ORLADEYO. prescription annual reimbursement in up X,XXX just give XX% on

of -- account majority for international noted U.S momentum. gaining before, but will the noticed we've As great sales in XXXX, the are launches

countries the have own submissions. are we And Norway, busy launches with and ORLADEYO Japan We across and Germany, and strategy and reimbursement call to with world regions around several patients team on anticipate and multiple this and UAE. physicians turn the global preparing peak Bill partners Sweden year. payers, favorable regulators, and bodes launched clinical way on in from to for patients more in U.K., We France, to approvals our getting to is Denmark, our sales. reception bring the reimbursement A our that an ORLADEYO regulatory update well billion $X over program. I'll

Dr. Bill Sheridan

investigation we not serum clinical have we program. our Well, in creatinine Charlie. completed Thanks, BCXXXXX observed our into elevations the

We far, an thus learned have the you update to made are. we've steps for provide want today significant but next progress, and I what on what

indicated started which or ultimately trials, BCXXXXX starting As for extension both you in to creatinine randomized approximately the twice-daily. Slide patients the with up currently several doses, pleased were We during XXXX, Xx their who discontinued four from REDEEM patients the been serum XXX-milligram a period onset the in in Patients involved proactively summarized dose. for XXX-milligram were to trials serum we a three serum that normal. lower on three We're and noted REDEEM-X at weeks one-third long-term further. why [ph] of the moved of by to these thus the are starting their had These that other severe were after creatinine, April, and were to began function three to holding time we patients had patients injury, baseline. proof-of-concept at at evaluation, observation had and in represent of have studies. for rise moderate cases short RENEW may lab patients on XXXX level. milligrams we in Patients time. the BCXXXXX. initial findings fall remained And trials evaluation. is enrolled in patients of REDEEM-X has observation active XXX discharged and far. recall, These to study in hospitalized at have These and creatinine patients in Three similar REDEEM the who trial our elevations patient X. in their immediately all enrollment in in levels Two kidney from investigate dose paused of clinical all had our at announced Xx we the The beginning after in BCXXXXX serum limit with continues see patient serum increases has creatinine creatinine patients to upper the creatinine therapy. this improved with the rise about randomized Two smallest serum REDEEM we early patients the of renal

severe none onset of is during serum the learned study treatment serum onset, XX elevations. patients had X not about patients months the late Additionally, observed doses during than experienced evolving creatinine, mild and XXX XX% lower creatinine which that have dose. extension moderate after started XXX-milligram to investigation, they about early milligrams increases to the we of these with slowly long-term in switched in to This this

here is know the of what we summary far. so So,

emerging We serum an after treatment. are early during and signal creatinine observing increasing of also chronic treatment

in bid Although increases has have study, factors creatinine also proof-of-concept increases at there when other and seen patients bid we seen events, may serum and lower have a of start lower XXX both contributing doses. hemoglobin reduced the doses milligram is not dose. we we to BCXXXXX in noteworthy at milligram XXX be transfusions And signal creatinine that we than encouraging prudent seen as related XXX teenage is milligrams. with assess And not in probably to It the efficacy that with it BCXXXXX. this these elevations onset early have in to serum

Under may benefit and the other from hold. dialogue active perspective. investigators, independent be we been regulators. study the new a data program risk committee partial FDA program clinical and BCXXXXX need this are or experience for explore placed for and on of in BioCryst its a there clinical the our the clinical path clinical strong monitoring enroll is key the by events patients. observations. with we course, not trials. to question these patients treatments with considering No and disappointed efficacy whether forward better now wants for types in very Especially is Of Throughout have investigation, trials. BCXXXXX The FDA patients not And these safety us partial have a the for to to continue sites and BCXXXXX has unmet one we seen in our hold, regulators the with

can in However, other have patients guidance enrolled, already and same who to clinical action already investigators new the language study course BioCryst taken receiving have had be trials. voluntarily patients benefit with BCXXXXX hold. sites and prior we remain from are inform treatment and the no available to provided continue the consent the treatment dosed risk. of partial And to reflect safety for updated to clinical options

to and observations what approach then we complete they Since we with dose milligrams both preliminary elevations milligram of lower forward step XXX first, efficacy factors milligrams. serum a this on we're milligram at for with XXX dosing the in regulators the amended dose contributory date. another at have the program. of the on not These If serum the PD inhibition BCXXXXX with at shows restart data could the at efficacy showing Factor XXX supported also to at are regarding to and creatinine is So a period have seen as from what investigation, seen agree unless X. and without and dose milligram seen dosing likely BCXXXXX plan But both evidence consult we increases strong observed the efficacy this, XXX have the with we to This the similar BCXXXXX, next? we milligrams, not, dose, an protocols program. points D and pursue, plausible that would they clinical do a level investigation, path if comes we like side Based illustrated using possible terminate part twice-daily immediate complete We creatinine identify milligram milligram XXX levels. clinical XXX know Slide learning XXX as efficacy. to both clinical by XXX

We expect path the by is on updates progress from as clarity to quarter. BCXXXXX providing not We the whether these third interim or have forward for a not plan through do interactions. we regulators of there on end

our discussions. However, we next regulatory look We you the forward have to and plan we once our on investigation completing to update program. steps for clarity BCXXXXX

like over I'd Now call to hand to Anthony. the

Anthony Doyle

Thanks, Bill.

guidance that. the at million million premature we we If less global XXXX provide $XXX than our scenario But lower our of In stage would guidance give XXXX it's early for discontinued performance range can't regulators, the as be confidence previously. to and than the $X revised in well us of program, our of on. is peak guided in this ORLADEYO million more would sales other assets be our likely of to a OpEx. certain Given billion. where the then then $XXX strong the of revenue I low This to recommence and the discussions year, we to be enrollment, as end base pipeline no for tremendous of that spend $XXX with spend build

which likely earnings attention this was million XXXX less the few first press XX-month revenues as net to at You trailing with the $XXX of for draw $XX.X for we $XXX no items. million. gave sales of main end were previously that of like expansion progress trial in annual $XXX at in find agreed compensation was CMC Operating XXXX. puts Cash the to guidance we ORLADEYO. revenue higher towards in XXXX Revenue call from Orlando QX and our $XX your million the quarter XXXX OpEx drivers. including of a our detailed $XXX the from of release, expenses, and year. Having million, over the on $XX.X finished financials not million than can stock quarter Athyrium that to the with million $XX.X today's the will I'd million, with million, came XXXX given being quarter investment of we've quarter about be mid for for guidance non-cash

Our continues. can programs and this for We're company create in is towards value deploying Investments strong. that ORLADEYO balance very for capital shareholders. the sheet

We are the U.S. investing strengthen here launch in to our

also we bringing patients around While our world. expand in to reach ORLADEYO the

that invest The in targets to will we having paved there we berotralstat after and continue [indiscernible]. always with We that we with had in success challenges way are now the targets, our are are challenges. pipeline. for the The also hard HAE go that

are hopeful We BCXXXXX. that can to we move continue with forward

thus in be As shown think it. strong smart can we will with efficacy data If we can focus. complement If PNH we and programs that other But will add can patients beyond. in forward move have we far, will capital a that we speed with and help we about and we then forward, in diseases. value rare cannot, move to we reallocate that

pipeline the for With it now up discovery launch, sheet, like for balance we'd our strong the open are strength of well-positioned our team to and growth. Operator, future development ORLADEYO we the and Q&A.

Operator

[Operator Instructions] Our from first Cowen. question Cacciatore with Ken comes

now is line Your open.

Ken Cacciatore

update. Hey, guys. for Thanks the

a some Just get wondering on of XXX milligram. the little analysis into could we the if deeper bit

through patient I when that duration about safety, know shorter. you've or can was you little you as duration bit of were on into looked on moving seems Just if that, just the talk anything the as they efficacy, a wondering, you but group? from talked about kind learned treatment that

would commentary And give about just on we want understanding any to also the just talk that? some analysis you can the perspective, So R&D, wondering on just that's if on could us stop you percent know the reallocating, more do a however, provide allocated? then adjustments now little Unfortunately, being completely more we bit so lower? to forward much. think degree Maybe what right color going be on it, you would allocated have there Thanks to to it if be XXXX?

Jon Stonehouse

Bill, you one? first the want second. Anthony, to take the

Dr. Bill Sheridan

in as that for numbers in that intra a dose as well such this proof-of-concept sequential study efficient had Sure. escalation a done, was doses higher I to because be is patient combination cohorts at of starting The trial, small of can. question. way rare Thanks and disease, we people. the of

certainly milligrams the on quite So, duration but as that a the the was XXX trial result of substantial, XXX matured, was that duration is the bit milligrams longer. on

driving mentioned to stand obviously. So, a plausible can't that's other in this. as we But observations that's out where my factors, we It's confined But the hypothesis the rule are period. on XXX the remarks, I milligram data. that what's

Anthony Doyle

that program last And then even spend, so was total D of the and what going R&D to higher. that year, our XX% year be this in is Yes. for example, around to capital allocation, terms XX% we've was Factor of said

reallocated Now, It'll think that create with delay. I hopefully we really and we'll we in can can, those yes, team. been potentials think have move diseases space and sure in areas we that forward, pipeline we make another be good discovery we complement to the albeit we've a the potentially both and rare have additional If to value, strong clear. where a

Ken Cacciatore

Great. Thank you.

Operator

you. Thank

comes question Jon next JMP Our from with Securities. Wolleben

now line Your open. is

Jon Wolleben

the with the they between Hey, same the the across characteristics patients questions. make were the right. trials? Along this and any thanks patients all taking so far elevations? I And other just XXXX, the elevations, with or then responders? three enrolled inadequate want CX naive there I to Were REDEEM for heard treatment nine at update lines patients sure the common And were

Jon Stonehouse

question. John, for the Thanks,

the the elevations. the I remarks, the -- As had mentioned we who patients only in unblinded

REDEEM So REDEEM-X. those And estimate REDEEM-X from patients those approximately XX% the it's come and in that had has studies. an do enrolled elevations the patients three of both

Jon Wolleben

may. one I And maybe for Charlie, if

mentioned number how any you switching from TAKHZYRO. were patients on patients data those long on before TAKHZYRO the switching? Do of You have

Charlie Gayer

they stable to TAKHZYRO. most same patients showing control don't Hey, do switch to over is of switch ORLADEYO of And of Jon. level they were that when they on kind these No, what maintain But we once have we ORLADEYO. data that. have

what's that's really important. So

Jon Wolleben

it. Got

the taking questions. Okay. Thanks for

Operator

Thank you.

Our next question Piper from comes Chris Raymond Sandler. with

is line now open. Your

Chris Raymond

may if if a I Also XXXX you question. a couple may question the ask. taking of for them thanks on Hey, -- or

and XXX guess that have understand I clarify milligram creatinine -- try patients is FDA, have So, in elevation with with to -- what is just level any seen want and want something you I specify treated longer level see were the not -- dose, that can level? you to at term probably guess are to to I not bid what's I dose that dose but guess -- just going level? you -- seen I understand evidence guys that discussions then same guess of the for that issue I … how And this you your after at treatment

Jon Stonehouse

Yes.

So …

Chris Raymond

Oh, I'm sorry also to trigger? I in effect. the the extension patients, When you you're mentioned seeing XX% What that did was guess had up? actually that level start increases. that show

Jon Stonehouse

might went So, helpful -- describe the escalated, study to he then Bill, to be proof-of-concept XXX, it the XXX. that got how

helpful first for I be question. think that might this part of the

Dr. Bill Sheridan

Sure.

the put together XXX then milligrams then and we And we the the PD at had to subjects. those we XXX dose. increased milligram patients XXX, those XX with that beginning, milligrams. modeling, of pivotal, in higher did few experienced decided period was the at so So and all to that doses through PK milligrams. And at lower level right was At that XXX moved in the dose designed XXX started so forward patients stepped and a that and milligrams. study at healthy And doses patients were XXX, move

mentioned were earlier, the varies. were was milligram of seen XXX all increased in In only period. as of time that I on milligrams some And quite serum So, it substantial. dose people the the creatinine people, XXX period observations,

the clarify you? for that question does So

Chris Raymond

Thank you. Yes, yes.

Jon Stonehouse

the was question his and XX% second And rise? extension around and the then slower

Dr. Bill Sheridan

it's months milligrams -- after average. the XXX So on many

level level think [technical another talking difficulty] So, of that We're your question was, above are the normal. we I about about? what any part talking

Chris Raymond

extension first When you in the And that when you start? did see did effect patients?

Dr. Bill Sheridan

long it can at that milligrams. within average, XXX on several -- quite After drug on period months a

Chris Raymond

Thank you.

Dr. Bill Sheridan

those later, There the are pattern confined this two in those mentioned to we patterns There's dose of we are early subjects. here the my level. pattern studies REDEEM in that more XXX mild both extension, this that three the that saw moderate And in long-term in as to saw onset I milligram observations remarks. there's

Chris Raymond

Thank you.

Operator

Thank you. question Jessica Fye next JPMorgan. And comes with our from

is line Your now open.

Daniel Wolle

guys. is morning, Jessica. question. Thanks taking for This our good for Hi, Daniel

patients was us any after continues with that one patient is, question question. who continued similar between patient can First, And treatment? follow-up more the you BCXXXXX maybe duration worse the in REDEEM that dosing did -- the is the dosing? And two I of give still get who first discontinued the -- will second on

Charlie Gayer

Okay.

Daniel. hi, So

subjects better Now had and who had is rises XXXX, continued doing falling onset way in down. the now. patient one are dosing early come all three back with creatinine creatinine the who And it's

So, treatment. is was this all to similar the treatment early duration of of events, onset the these in in prior

Daniel Wolle

are I Okay, got on it. And who question could difference reimbursed Those guess difference? Can those persistency seen on color more free us versus maybe the free? one more that on ORLADEYO. you who are are give between What drug. driving be

Charlie Gayer

something into it. digging It's question. we good a we're It's

patients likely HAE meet clinical reimbursed the good to things that criteria for most of be the patients are is reimbursement. One who

TAKHZYRO analogous that's to Haegarda stable the I so was that describing. and And patients

comfortable have. to and help that seek a And is is it options. attacks, stress. thing through other we the if stressor, cause is paying, worry a not much it's that lead as payer try can them that that to make process, The patients people is physicians Stress reimbursement to it's them always, can and other cause as stress something can

we're base and that's is, started going So, this date, ORLADEYO what getting really patients. are really stable how is on ORLADEYO, on we're building launched reimbursement to to are patients X,XXX patients who and who great. two-thirds of patients about leading of about what That's the to what's doing still onto well pleased us to we're drive

Daniel Wolle

Okay.

Jon Stonehouse

-- of seeing Charlie we're the described. in patients and early this is Daniel bulk where piece is the other discontinuation the think I that's like process the

chances drug. you are going you're on to If longer, stay stay the on the

into so that's another this. factor that And plays

Daniel Wolle

it. free for Thank with I got what mean if are on you question on stay very doesn't a patients If currently you can potential could that more for don't molecule XXXX. develop? I one the the program? long? If path allow at? possibly Is FDA there the Does Sorry, that have the so, stage of drug amended forwards protocol, And that resumption backup much. what is trials

Jon Stonehouse

the investigation stage. We're still in

so, and you on And if we're path time, in to any continuing comment we're see point other working but can't backups. I look always And there can on imagine paths at pursue. other to that this are

berotralstat You Factor same complement in ORLADEYO the D with in area saw with HAE, we're and thing. the that doing and

talk So on earlier, backups. they and when working we're but always to ready are we will, and we're about

Daniel Wolle

Great. you. Thank

Jon Stonehouse

You're welcome.

Operator

our Tazeen America. Thank question of from comes Bank you. with next And Ahmad

Your line open. is now

Unidentified Analyst

This is morning. [indiscernible] Tazeen. Yes, hi. good for

Just one quick question on XXXX.

XXX you mg are profile? do dose, observed if the -- efficacy if XXX not stepped to move what implications what increase to mg So, you And like move the in the creatinine to serum for think like time up why XXX do taking not approach, stepped then or just experienced XXX below dose? plan mg you any have then on move not mg, versus to the And you approach?

Jon Stonehouse

the the is for why first as pivotal? choose in proof-of-concept? did the question dose did And So the Bill milligrams move we that beyond XXX why then we

Dr. Bill Sheridan

pivotal of an time, at try to miss On doses. the avoid to to opportunity breakthrough sequential first in patients we designed point, studies, wanted we the event the the give hemolysis

safety theoretical a profile the profile. concern. and distinguish milligrams, was time, efficacy of XXX couldn't And we the or So, XXX at that that

so With that, efficacy, that study regard on to the hemoglobin milligrams we that no XXX dose I very reasonable qualms about the the There's the aspect proof-of-concept this, stepping and on. very back increasing terms other that's choice. was have So efficacy. thought size [ph] perspective clone and a the evidence a in that to -- and of molasses controlling fine from of of from good we're

the that decision was that at point. So

in creatinine. lower up were a And market dosing dosing of information. none to of The step-up other are on step-up alternative strategy consisting Now limiting the proof-of-concept drugs dose of lower early a the proof-of-concept effects. the study in patients in started dose. And and avoids had all to sense the the in side the an there is we makes There out starting propose patients have because reason dose rises new study and for dosing dose. the had then it step early at onset at

way and move path we our a or we'll a to forward. whether see forward as not. So, investigation discuss complete that's with And a reasonable that plausible there's regulators hypothesis after

Unidentified Analyst

it. Got much. you Thank so

Operator

Thank you.

with question Belanger next comes from Serge Our Needham.

Your open. now line is

Serge Belanger

Thanks my for questions. morning. good taking Hi,

you when end a partial FDA guess just of the hold? to -- First long to XXXX. since wait. you're believe time mentioned you're seems going issued like And Can was interaction your And one I their quarter. It paused? study discussions describe FDA you on the the I at clinical the to third have planning have

FDA an to work So be or done meeting? is before that limitation additional needs this scheduling

Jon Stonehouse

which have upcoming we the we we that we that to knew already the few patients communicated had a pause the informed with alternative and all at enrollment so were thick were placed. have could we hold FDA that ago that consistent dosing we an put information the from had earnings was context. you and the were And better in call was that no had and continued and on drug instigation the thought we is in a a that once and it shared We weeks there benefit receiving partial

decision. So that was the

Dr. Bill Sheridan

the between question about Jon, like go I we asked when and he now to back FDA. timing think to the the

Jon Stonehouse

predict. a that's to Yes, one hard

And to able in that so there. a that you there's maybe a time, we If said, and of quickly imagine, to front we'll a the stuff. obviously, go so But work through get get we by we then and want that a we'd to as as clock we can all And go be them. get third goes, process. quarter, second first there may the the end on proposal that meeting of be can

we'll So, in quarter. happen it that the -- third of the think could that end unfolds. by But how we see

Serge Belanger

one then Charlie for ORLADEYO. on just Okay. And

some mentioned challenges were you quarter. [indiscernible] with first think the in there I

if usual were curious resolved? the Just the results of those if and Thanks. [indiscernible] was been there of else reset there deductibles, they've or impacting something

Charlie Gayer

No, expected was the Serge. stuff, it

as So, it's that, bolus a the there's in that of first in the always kind -- reset. happens quarter do payers annual

what So, it expected. we was

until product back stepping to Jon PA donut I quarter. the always exhausted and the was free Medicare his think then during first early in in it those commercial process. patients are deductibles mentioned comments, are higher, hole, It's

Serge Belanger

Thank you.

Operator

you. Thank

next Gena Our Barclays. with Wang question from comes

line Your now open. is

Gena Wang

XXXX. One also taking two regarding you I have questions. for is Thank my questions. HAE

protocol. the titration FDA resume if I will say you stepping under to allow Just wondering up other dose side those lower of the if,

assume need my patients assumption clinical reach And do the statistical So, number question the in design you a [ph] to is trial to reach order second to the your revisit of of -- significance? clinical and expand benefit ORLADEYO. regarding

For to the the of when is versus rate attack from they switch by what demand, patient, on patient that And naive or could doctors ORLADEYO? give switch community you academic breakdown a doctors? prescribed

Jon Stonehouse

about the that's to so amendment. first you do If the changing the Yes, dose, a definitely major you're do protocol? what question

similar I the those can't make But And things change is so exactly had XXX to think point, and like to don't this we and it largely need think so haven't you tell the We but study. that. the least the information same we they'll efficacy that, at other regulators. XXX we we'll At because have think more talk we'd that powering be I things would once but change, conversations. to of we

Charlie Gayer

shrinking. doctors products. portion acute And are your And with patients as perspective, those and ORLADEYO to has prophylaxis similarly launch on to prescribe also attack are But most I that's prophylaxis some to ORLADEYO pretty From well the the doing wouldn't been switching overall consistently describe it those prophylaxis. and more. There'll as shrink what to that to prescribing only half on ORLADEYO, Gena the ORLADEYO. patients as market who be less and that little would might rate And continues a from days they that's coming patients across on of these versus patients switch I described to therapy an staying and than who's board. here market as question say been patients only to where switching the patients academic other -- on consistent far when And I are to the acute switch the prophylaxis. are there community. overall, an since

Jon Stonehouse

shrink. Few markets

Charlie Gayer

market the is Thank you. Sorry, shrinking.

Gena Wang

attack when they in wondering rate switch [indiscernible]. the acute So the just

rate. you If what the patients? are giving attack a Usually rough those is range estimate regarding of annualized

Jon Stonehouse

down to per that's you. I the attack have to so refer where information mean, to don't month, we a attacks or they an data best got half probably average three I month. they what per can XX-week want and give of the

Charlie Gayer

-- And good most of patients control these point. which That's these right. gaining the we're is patients on are key very what is ORLADEYO, there seeing

Jon Stonehouse

in Yes, attack and out I made think the XX last other rate Charlie median point what was the zero. of XX months that the

And are really so a controlled. of lot people

Gena Wang

Okay. Thank you.

Jon Stonehouse

welcome. You're

Operator

you. Thank

Abrahams question Brian RBC. with from next Our comes

Your line open. is now

Brian Abrahams

detail my for and both ORLADEYO all taking appreciate Thanks and on questions morning. good XXXX. the Hey,

over Thanks. see more respect or did of XXXX, I'm curious incorporating was rest we XXX the titration, you either refractory some And to on the on the and to be study? would the discontinuation of more that or you perhaps the or renal then any drug with of investment gated with in in that to impact this or might on FDA sort evolving patients or So subpopulations investing if starts greenlights Charlie, way just there in there Or still disease designed diseases wave certain as titration mgs, including anything dynamics areas before, the patients? the first same new during a fully year? expect be would quarter, the of Omicron focus for switching course patients

Jon Stonehouse

Yes.

one, can't a dose. And but time all that do, be there's can safe things to it's dose you at discussions go PNH get be not timing that's and at it point we'll this would the I If go into the twice take that population indication need the safe after. because other We're can the on we a and effective if we with be only just into. effective determine XXX every drug regulators So going broad first that in Brian. we we the invest milligram that in I'll to give day, and to a intended proves and

Charlie Gayer

And QX patient in we quarter. we quarter, -- the slightly of What the say. honestly then, Hard to saw for But new rate earlier our say with I the last the question is second slower good Brian, that were same can in half at year. were hard on your Omicron. obviously quarter. the starts ended They

been So that it's something part COVID we've It's all of -- through. really way we've good momentum. before, a Was with said Omicron operating that? the as

Omicron. So, I'm our not specifically anything We're going right now. happy with to on pace pin

Brian Abrahams

Thanks Understood. so much.

Operator

you. Thank

Our Evercore. Bayko next from question with comes Liisa

Your line now open. is

Liisa Bayko

the Hi. and for Thanks question thanks my also color. for taking all

Just to XXXX. start with

expect you was into outcome the of follow-up creatinine. idea of what a sort just particular any different Just moving lower we dosing perhaps? saying, have maybe renal concerns diseases, Brian would some that do on the serum given about group And

Dr. Bill Sheridan

Thanks Bill. for Its question. Liisa. Hi, the

I is this think a complicated PNH disease.

not not of effects of with patient every treated, any tubules effects see from PNH because kidney PNH in you your tests. or hemosiderin clinical they're When chemistry or that can subclinical renal So, the an has analysis the your getting whether hemoglobinuria. on

pristine have to don't. begin They they with. like not it's So, kidneys

we've all patients the I then families to And your let's to you answer in can treat What the need and an I with have it. interactions think discussions and know tell with that you exactly have we inhibitors -- I diseases I've rest had your treatments complement need that's who advisors that the don't the with be the complete who regulators of ultimately investigation. going question move investigation, is with can and asked and experts nephrology to expert outcome complement forward. of when the people to through question, treatments. currently same the And we no responses, if diseases no see mediated our

idea of So of gives treat in it that's who an the diseases. story. end not But what's these the mind the experts the you of

Jon Stonehouse

to moving XXXX theme we support is I There really the think real that a important for community the in to understand. continue of got to feedback forward. that is everyone

to investigators, see But safely going have shown this and And it so obviously pleased thoughtfully. I'm molecule. key others the we're that and to opinion leaders enthusiasm do around

Liisa Bayko

how protocol take What doses get Okay. That's considering? then just it you're would feedback. and you to long that XXX up helpful XXX? And can the step start you describe to would the

Jon Stonehouse

study, the time. very In a took proof-of-concept

that discussions need about that we we I goes. exactly certain step be think regulatory the through first, to can how before

I think currently the -- might and data on the of there's there's be have, we basis what approach the flexibility there.

goes. that how see let's So,

Liisa Bayko

oral stages some just a lifecycle and other nice competition And for an from you've billion, wondering factor ORLADEYO kallikrein that effort really in guidance potential plans. various ongoing here there? management Thanks. like you have seems any, of inhibitors It created are Okay. finally And you do in development? of then you market have your if $X do just

Jon Stonehouse

market Yesh. and here's of now. there It's is out X,XXX years some That's we Charlie right? X,XXX. we and patients No, the share. out you definitely between get future which the XX% mathematically, how from that. But XX% consider competition told timing of

piece still switch? you is, lot the you What And would you market incremental we've We've to controlled on in a cause research, not you’re smart you and switch? what gotten done the others. there's if that So, can't a ton And I capsule see, would mean, be other I because convenient, controlled. find it's and efficacy, doctors. patients you can't goes think because they're once-a-day, about of of than benefit capsule by would had one can't one why market minds And get better really once-a-day. of a on reason we it

ORLADEYO, it on spot from find take now. that. people away weren't room and could there's people may just for other be for really a to from that And us so, that controlled share that we difficult injectables, get share They

it's number. think very we So, a realistic

Liisa Bayko

what then plans? about management your lifecycle And

Jon Stonehouse

HAE Yes, in pipeline with also beyond at looking like. to, obviously, meaningful are invested and we've But things the we're a I way a in always HAE. mean, we complement building

life so, a with patent ORLADEYO as and really long then we've got And well.

those then now right fast drug. and is pipeline. tolerate that peak to And and in can, patients as lock our as we to to well-controlled get continue advance focus our So, are the

Liisa Bayko

Okay.

so an of on, that -- mean, trial, we XXX% just haven't relief realize not without it oral the So, kallikrein that hearing? patients is even is like seen have a -- something Is [indiscernible] I'm really I in inhibitors? room front. difficult who that the that it lot wondering, on? it Or there's in to seems better seems And but molecule. is clinical it like still difficult? working is stay to on of clinical a you're trial progress just attacks just Because there develop what

Jon Stonehouse

Yes.

results, controlled. study it but that right? I really, world No you controlled, the well we're and the I real really want study work for seeing pivotal thing I'm really on, agree they're It for, But what way. pivotal people for the are one This convenience. is works everybody. well is efficacy that sacrificing Just doesn't results not, ones correct the to are in

incredibly your And to that's important of think I so, the forgot question. part I remember. other

Liisa Bayko

the in difficulty speakers]. yes guess, [multiple I --

Jon Stonehouse

we it's evidenced targets. I try, only by drugs a really tough come And won't others use up tough that as hard period an right. as with expletive, but know hard, we're the to with and hard. drugs fact this the it's yes, who's once-a-day one and And industry, for to up come come in with it's up

up with come extremely hard attrition has nobody it's point. so and a once-a-day inhibitor this And to is kallikrein And oral yes, the rate high.

Liisa Bayko

Thanks. program. the How your steps? next noticed So investments then, any last are got you I question about you designation for making for Are me. thinking there? FOP you just

Jon Stonehouse

is Yes, really get we And to get year. we're designation. are. patients excited our next And goal in work to that

continuing really move that any with a without we're so to treatments And right real it's horrible -- now. program disease

hope the be patient community, to going year. yes, patients And and so, the we're starting who to to we're full get that the on speed next and know treat in community, doctors

Liisa Bayko

Jon. Thanks,

Jon Stonehouse

Thank you.

Operator

Thank question Kim you. final from Justin Oppenheimer. comes And your with

line Your is now open.

Justin Kim

Hi. Good morning.

there get kind enrollment of you in there a just one just me. experience curious, evidence So will the this much really accrued of gain Or population? quick in there population? treating the will curious to substantial be Didn't experience? mention how confidence renewed maybe around from is sort a see clinical just -- And that

Jon Stonehouse

enrollment in trial. in got that just we Yes, started

go patients, and evaluating I was about very think in we got some process more So to diseases. of I complement then talked work dose, the We've the But nephritis use mediated that's few that if and into substantial number it wasn't Bill it diseases, indications, do. effective safe find you and and you drug again, can have think, different goal. a the you many

Justin Kim

Thanks. Okay, got it.

Operator

Stonehouse session. for back you. the Jon today's I And now to call closing remarks. like turn Q&A this to Thank over concludes would

Jon Stonehouse

you. Thank Yes.

a $X my then diseases. innovative more no inhibitor here restate it lastly, our peak. really balance this came all and challenges. year, right. the we've once-a-day got of along that balance company, What's a can And What revenue a yes, have then for What's challenges a less strong if and a future. to the again, platform but of we're and today. to sheet, we billion called earlier, engine you seen is potential face Discovery back So, So, I'm to that? market biotech and $XXX bring suffering said about bringing ORLADEYO. take still value is, is, million step than and look That's in over companies for back, value value more at patients we a of what's where of and have repeat time. able pipeline, highly don't from its see second being from, the in What's of that? And that? And really when the step million a I we strong you answer at the kallikrein sheet, we're company just some there's even you over going the same to over value excited which The faced this that value even you rare the have that to there's the lot the value way greater that? way, a with something on at oral -- and $XXX you products

drive confident tackling have ahead, one challenges the plowing ORLADEYO them build what next that keep and biotech to towards will $X face company. going great we these take remain sales we're to we so very we billion, it of a And in as

interest, your for a have and thanks So, day. great

Operator

call. conference Thank participating. today's you concludes for This

now disconnect. may You

BioCryst Pharmaceuticals (BCRX) 5 May 222022 Q1 Earnings call transcript