BCRX - Earnings call transcript Q4 2021 | BioCryst Pharmaceuticals, Inc.

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Operator

Good day, everyone, and thank you for standing by. Welcome to the BioCryst Fourth Quarter 2021 Earnings Call. [Operator Instructions] As a reminder, this conference call is being recorded. It is now my pleasure to hand the conference over to Mr. John Bluth with BioCryst. proceed. Please

John Bluth

on morning, Financial BioCryst's press Jon Results accompanying Dr. are Good Corporate Quarter release Thackray. Stonehouse; welcome with Helen and slides and Participating Chief Chief Update Conference available Charlie CFO, and Anthony Brian. are Commercial Fourth to XXXX and me Doyle; Thanks, today website. Officer, CEO, Gayer; our Call. Officer, Today's R&D

remarks, your we our Following questions. will answer

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

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

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

Jon Stonehouse

that in of beginning. year lead and ORLADEYO our the peak Thanks, will we John. in leader as forward took the you just to BioCryst to oral our success of XXXX great step base are of pipeline to for no on building to also we HAE. market the led company. big revenue But next than sales. advanced oral therapy, solid was additional significant creation. goal of biotech us treatment The million in an drug global HAE, for less suffering guide toward a our year, off. the billion to this and great $XXX value ORLADEYO, $X million a have launch we first from a our With becoming In rare a $XXX.X net patients way Patients want tremendous the XXXX, disease is got That off start. to we prophylactic confidently first build

capital moved in only note Phase in and I oral D is indications. we indication to important a BCXXXXX, of And pivotal studies inhibitor, come. different we're heels in a the on not approvals, Our diseases. the to and create greater entire major and in last additional but capital having additional finally, has program of proof-of-concept programs Factor of to ability retain value filings late-stage shareholders. to to from program late rights the that these ORLADEYO, we PNH with What's for to molecule have compound for do proof-of-concept That's here be programs this the the value pipeline. study launch XXXX, brought many allocating and gives into our us X and studies year, our rare approval for significant in a renal many we X this potential have pivotal Imagine what years an

our pipeline, grow and in advancing BioCryst. pivotal proof-of-concept to time exciting the So our enrolling year is increase focused at in continuing executing XXXX ORLADEYO world It on all the is compound plan, and studies, U.S. around value. to an and another

to ORLADEYO for on more turn launch. Charlie Now over I'll details call the the

Charlie Gayer

consistent over rather and base double the XXXX. are that more to patients future and market share. expect shows Takhzyro last recent now different base. predicting patient This quarter the launch the The and reflecting XX like therapies, with give presented great of quarter our staying from also be to consistent us mostly more of to ended with we We rate HAE Haegarda of XXXX become from prescriptions, We of matches patients by profile. year, source Jon. get research physicians on XX detailed in experience switching and in the consistently The ORLADEYO in also because doubling trends the results likely than each, entering a of use recent expectations among Thanks, sales with particularly strong matched seeing each high in new have XXXX specialty injectable we patient means pharmacy accurate their patients an research This already than experience XXXX, current prophy this the rates, each overall market injectable treating next months. market before partner. in representative starts The from to are in of deepening survey their having confidence is research We market, on physicians of their of patient was in switching regardless saw and prophy clinical of they retention. That demographics written patients prescriber are the and reacting about that half therapies from a prior sizable we current other retention year, are retention ORLADEYO leader. patients expanding guidance data XX%, more type on at survey patients than strong therapy. ORLADEYO a and physicians our are X real-world product real-world expect from

XX% APeX-S retention consistent have level college for remained most our well ORLADEYO history X least we of in we where from patients which therapy November switching patients shows the study of the with at Takhzyro that working at is to meeting. on nearly presented data enough of example, for patients, This months. For track, is

that outlier XX% confidence with the XXXX that to in for U.S. will sales coverage. has fundamentals ORLADEYO. The less strong entered the the access HAE $XXX which the give than us no least QX broad coverage be in also population with ORLADEYO quarter-to-quarter for payers being now underlying PBM major All of and means policies, million We have growth. at year,

were specialty into little X of reasons with QX. and built which are XXXX this, expect to revenue drug for guidance. QX growth We expect would over no There all our you a

specialty at patients some ORLADEYO, meaning year shift reauthorization will of require like to products plans temporarily product. many First, the free for the start

among other and the expect in net and Second, existing us to quarter. went U.S., from last of expect The the the we in assistance participating And for the year. both peak and several $X policy The realized trends, in up will in treated HAE patient Medicare selling will $XXX and patients share approvals annually. commercial more to again into benefits XX% to and PBM a and be diagnosed sales, to as the our but adjustment. million free for effect to in reactions, at XX% price contract simple the product. assuming and reach and XXXX favorable XX% we impact sales of implement in patients January, paid million there HAE achievable. to the most product the the Between this time shift gross in By we patients sharing world majority to account treater sales, rest D already quarters plans sales. is future billion on Based the has cost regulatory finally, math launches get reach global gets year, X countries, will revenue great average XX% co-pay X,XXX Part the launched results, of reduce strong expect U.S. ORLADEYO market we positive of least major but ORLADEYO this annual peak $XXX

more well of works we access and helping trial As we as most we evidence for ORLADEYO patients to clinical are patients, more and get treatment. how see see, real-world

pass patients see HAE goal how oral their and As is all why you over once-daily it for asking, is we world, the you. much to there try wouldn't get Helen, our need to I'll an therapy ORLADEYO? around doctors

Dr. Helen Thackray

of the of absolutely the effect their we're continuing and Charlie. better they staying long-term is baseline coming in really is the with improved. That significant in They It's and We of XX-week oral XX% rate once-daily patients. effect set see making in see difference at is durable. have exciting helps ORLADEYO What study. feel life an example real-world Quad That's seeing APeX-X, fewer study, more treatment attack that this sharing the in data lives weekend, patients reduction in That long-term We've evidence the how ORLADEYO both attacks, evidence this is durable of for APeX-S, the medicine. right, to from patients. ORLADEYO group abstracts quality observed this HAE the on from and got responds AI a well. pivotal the

like to to what's us. I'd turn for next Now

proceeding pipeline and Our is, our for set. evaluated expanding we to pipeline of is data inhibitor, busy and our very sites advancing and These are BCXXXXX, This enrolling to lead X which we're PNH next, as of rapidly, is this, preparing trials the comes being come. for for pivotal course, and more such program Factor What The exciting the with in is for for is registration molecule global treatment The full trials. D are to is twice-daily oral reach us. steps pivotal an swing. the countries to we pivotal filing well. starting prepare and are even time

that endpoint data in rate, as inhibitors. well patient endpoint registration We the through CX position in information regions. both program reads an broadly set change BCXXXXX, this highly data. Europe hemoglobin is the only Remember, a moving key process expect designed with those pivotal from ORLADEYO. such program reported and U.S. to secondary the the also such robust information And We baseline anticipate we a to outcomes, and to the in register of on broad to our give may in strength not that this to controlled our the be is as pivotal to primary went This supportive registration response are and designed controlled be the transfusion pursuing X set. this expect to prior in multiple score. to our in case U.S., regions the be move us of with provide outcomes, of data simultaneously but With therapy and with beyond FACIT-Fatigue We with of registration other countries. single inadequate the was one in as trial in data patients to naive

clear about achieving a about in registration Factor initiating towards of clear the our top we study countries. are also There of basket work line global when to for patient forward step parallel, pivotal indications, So glomerulopathy, making IgA better and by dysregulation D membrane therapies talk end these improve we talking both to in these big multiple diseases, for role in XXXX. as inhibitor program. announced for CX the our be that's them in also next in and the pathway trials, we a logical remains of patients is indications that enrolling readout the these need nephritis well. primary inhibitor the crucial nephropathy. we pursue goals, outcomes in X open enrollment Factor in PNH, a alternative It's yesterday, which screening and In the And where And indications our first to D study, is plan complement submitting nephropathy has

study XX proceeding into we pivotal this confirm will of on that to to to of reminder, confidence the each need not diseases a in in individuals these the up As have patients vaccine, proof-of-concept. many size enroll Depending a trial. may

may the disease the of each to what achieve they the at Factor each improve to forward with that and confidence where inhibition anticipate we enroll multiple patients of we'll proof-of-concept. well. at pivotal for one complement So XX that the pace. patients. other for not X and diseases different outcomes fewer because disease can D a are diseases in rolling These as by with pivotal advance and where the the of one trials gets inhibitor perhaps other than of these and be at this out we these XXXX end alternative as time. progress potent We clinical There a expected is And move for is pivotal trials crucial proceed specific to just dependent the pivotal start. the be pathway preparing are is And we patients point, to could pathophysiology pathway proximal X trials in a to each stage, are alternative on each some cohort, to

the we're in evaluating indications add the in to trial clinic preparing to so far. ones are We

a be wave expect studies in these on of there of as Factor to studies will of now. a already we behind you Think D coming number what oral other building it So treatment second diseases. see what inhibitor know assess of other you our can preparation, for

and it is and to pivotal completed We the the trial, about launch global way think for the like ORLADEYO. registration Another expansion this. then global

registration to PNH have for submitting we PNH. with more be Now of in and studies concept trial towards for indications. moving X By we having proof plan our pipeline next pivotal XXXX, in of We the molecule a end in renal X And proof-of-concept pivotal is getting advancing with our expect inhibitor BCXXXXX. trials This in Factor X have program that pipeline molecule the of indications. is to a additional indications. new the in set trials D underway whole renal we anticipate

We as are moving ALKX Factor including program really investing D in the the our I've and pipeline inhibitor, our be and trials inhibitor And of the here. in planning program our mentioned for to our clinic along, is the There BCXXXXX, rest is third FOP investing strong. this currently with and molecule more the Birmingham. in pipeline, center in our discovery also and we're unveiled from program, waiting

is Beyond to today much our the the and identify in discussed the team for targets. to develop molecules molecules There in more I progress clinic and new come. making we're the continuing is with so meantime, discovery additional

and ahead bringing and of and an ORLADEYO are whole to I to us. to patients to with multiple diseases. of to new we're what set hear pipeline increasing with in I'm we hope This bring can our ability excited and our expansion. level. develop patients is what's from drugs revenue deliver our my the voice, expanding capital, now investing, and doing you is This a programs building medications With

ways are in are the the still our our We advancing forward now Anthony. of proud I'm and the the clinic to teams come. very doing both programs pipeline drive patients. new with turn programs on And to to of the call of burden disease work I'll the for focused reducing

Anthony Doyle

Helen. Thanks,

invest to has a of robust Our investment early did capital research. value. been factor provide with And very our firmly off We key in always believe early areas investing those can that ORLADEYO, has long-term in significant strategy the allocation been early that we it this success. back market

into program, potential that there's this With a you pipeline even given we with Factor indications heard greater what go just molecule. the from the multiple D given in Helen, could

today's which like $XX.X nonrecourse quarter, $X.XX compensation You not release, came by for of attention negative can for to Revenue our find million per impacted positive of expenses, ORLADEYO. to detailed quarter related notes. the stock noncash earnings the for onetime This net I'd Operating $XX.X press financials items. few fourth earnings quarter noncash $XX.X of And pharma was share sales $XX a was a including the QX. your adjustment in was call million. and the quarter for was million, million the of extinguishment to from

a non-GAAP was our on EPS for basis, adjustment, negative $X.XX this quarter. Excluding operational the

our equity midyear additional in the Following royalty move also Athyrium this [P&L] and about have with agreed we on previously deals cash debt year ended that we tranche down secured the $XX million we hand. with we to described, draw the year. with in $XXX as QX, And million

a than last Additionally, year year we growing $XXX.X billion. have revenue million less this ORLADEYO no onward to $XXX and to from of million $X peak

of there. drug, between OpEx the 'XX run rate a our providing QX will about what majority ORLADEYO I investment the the why around XXXX, With a XXXX. year million, We're now expenses, we of ended to excited range Factor allocated additional being confident and investing progression Helen for end the a are in great of the get $X and D very program. investment clearly billion our articulated this with $XX so of get we're we'll to is operating of and belief asked to profitability. million us developing the this that often For path full $XXX Having million in program. of $XXX quarterly feel

maximizing team. our belief years. be to ORLADEYO value that, company revenues, gives XXXX by biotech next we'll drugs on value indications, and and that allocating enhance the the us D investing is the will creation, in ensure a great for continuing direct to given a the that multiple strategy over that in to a and pipeline advance for was though the With is the for the This to already we result now to Right robust questions. which profitable in coming will our expand continue launch, great across open capital all again. repeat achievement shareholders strategy the to is up new discover compounding over fact, we have it ORLADEYO basis, process In pipeline contribution Factor while and

Operator

from Our Company. [Operator and Cacciatore Cowen Instructions] with the come line will first Ken question of

Ken Cacciatore

Congratulations all on the progress.

that practices through On a the retention of could the retention hear to you've learnings best that. it's having like are rates, to there been even really And and described, anticipated. force sales than and nice we sharing clinicians as clinicians, rates? passing improve as experience, probably go about better positives. from originally other So further with would wondering along But the kind we more

we renal enrollment, it's I possible want hate year. you obviously, timing you given can then, any basket it this, see have like the kind qualitative might data to end maybe PNH the the to is could be by of this study, something seeing what On but it on look from does studies? those going? the just discussion how you're you lastly, push about of give able just talk when kind and timing of on of enrollment in of to I And

Jon Stonehouse

on want what other to Helen the Charlie, two. And working the retention, can do take first on? then we're you one take

Charlie Gayer

number how that prophy first they who therapies, just so. set therapy breakthrough first Yes, you GI to attack best expectations effects have GI thanks prepared what for expert be doing drug -- we to need -- that. breakthrough And few that to the work really so really about have are any a new within given to to common or patients chance on drug X-month-plus for tend practice month that seeing in this And effects That give every retention, that for months. see that, hear from that patients Ken, the -- one, the is question. you. what we we treaters anticipate do through see a the a so those need chance for to is is true if needs is a the Sure. all the and attacks that to we're know be and away go it's HAE

anything up we doctors process. set setting prepare can that making and keep the HCPs, expectations, sure this to So hands the that patients improve focused up rate that on we're we really through that retention to everything really patients' staff high And the that that for keep their those And hopefully hold we'll and even for year success happen. them might focusing throughout and on it. do

Jon Stonehouse

One really Yes. a Charlie the about good like, challenging answers Helen team other or thing before question. that more good gathering and of more our and part One and the job is your products. switches whether it's other product and doing data switches are information thing

learning And so by. numbers And improve with we're that that from getting smarter, month goes we're to able. applying we're Helen? Ken, what those if we're each

Dr.Helen Thackray

Yes.

questions. for So thanks, Ken, the

this On are has independently. different of the renal I enroll X in study, terms They and a timing, each cohorts. said, will, therefore, study they is that, independent, so as

pursuing this on science. based are We

outcomes proof-of-concept and sure to said, in to will we programs. independently, I We study. -- right of efficacy will can They the for see we're anticipate trial each get also the that we this we making go expect patients concept though, will so for that that they pivotal into that assess lead us proof as

in we to time. that, order at biopsies this say expect this when right with be advanced I kidney take active doing And confirm stage, able we are I patients beginning too assess. some to in disease for that is to So the mean the to not entry at

the these we target for we not looking these all over that advance separately. to go have to within see pivotal achieving the not we've time we we're for expect enroll So of but or XXXX. we arms which faster proof-of-concept will of And of by -- time, frame think to X slower, said programs end

pleased for across the as what PNH, heard. On what which sites enrollments we're in in we're the activation we're seeing of you've really both with globe very second enrollments seeing. question, seeing We're and was studies,

be more with course want this. we centers and we're and to program on to add right where be ramping this with So up

have investigators to happy centers seeing don't I So that, excitement around from that both trials. with on give more and very the that and really of the we're on color the except the globe we're progress

Operator

come will question America. from line next with Bank Our Tazeen Ahmad the of of

Tazeen Ahmad

I'm I your For this release in you these have ORLADEYO, you common color beyond expect a could said resets, in And growth comments to stage in XQ? for where sales, we to our asking a to should purposes. wanted gross this just of that, little net for a to your on to related the what help revenues statement and sequentially extra that clarify, just your to be to about expect experience, press commercial from number that with companies modeling no is sales This quarter? due then formulary phenomenon follow-up And lower PNH. QX I be quarter

Charlie Gayer

Sure, Tazeen. It's in phenomenon. Yes, you've got -- really this is QX. dynamic reimbursement a a just common

very -- growth. million the for will we then from XXX the excited and But confident expect QX QX, year. be the we're plus the So quarter about to this very lowest

So in quarters. higher future growth

us filling reimbursement our and all then of just it's And of all year takes tailwind we big effect. is be QX what So the there. described the a as I into just from the And of new of this in the a with coming frankly, opportunity driver patient in funnel a more QX headwind, that contract the continued will in. front see patients turn feedback that got will that, of we've really new expected field lot research in really

Tazeen Ahmad

Okay.

quarter? that to at the flat sales, for right, just is clarify, So expecting you're worst

Charlie Gayer

Yes.

little no growth. So to

the growing for be year. be -- So expect the it it lowest quarter-to-quarter at worst lowest no growth, is but will we the to

Anthony Doyle

about question net, be to will has Charlie reauthorization. mean, gross on a yes, the Tazeen, the there I right? about your talked spike,

from be in revenue spike. going the that's despite that sure patients And cause we'll no have there QX. what's so fundamentals to make fact things a that that will drive like have co-pay adjustment strong with combined -- to we'll give assistance, to in have And temporary break kind will that the free product of we the really, that treatment, effects

Tazeen Ahmad

Yes.

net. talked So for XX% range? that Would always than or still quarter range be higher to it slightly you've about gross that that this to XX% within

Anthony Doyle

Yes.

that about on those I think XX% talked we the is to XX% reimbursed.

than in the factor you yes, will when free that. higher it be product, So absolutely

Tazeen Ahmad

Okay.

Anthony Doyle

look be it when it the small co-pay but may at you only by well would a reimbursed, predominantly then And then blip, again, driven assistance. be

Jon Stonehouse

And again, the is products quarter. of this typical very during specialty first

Tazeen Ahmad

nuance And readout X oral Sure. then to their you've versus for just these finish provide have then enrolling will data occur? of you When an there prep data be events Astra years. XXXX, you and then will see weren't out studies drug, you'll market also that meaningful from X have to I additional a if year-end. SOLIRIS. to the those read-through patients And PNH, products? PNH this to Phase able on candidate the in will any at do of head-to-head with said on you'll as program? for wondering, studies for And different failures XXXX read also think Novartis do within when be its approval refractory first need each combo all could think you the landscape, And And to among you I'm year that CX believe would differentiation III competitive

Jon Stonehouse

And middle question. Charlie, if the take I'll of maybe first market. last the one then maybe could take and the in one Helen, you terms So

do end enrollment tell then middle of what we'll focus end a give goal think think the on we'll every take the of the you a at to so on week, want year be blow-by-blow right? we'll want then, to next as I next to And And not. it or update see you'll over you when you the we changes a as one? But is give our on to more make year, you decision hard do backstop because course learn really year. And of you the of it's the I where we Helen, you and priority. we So

Dr.Helen Thackray

sure. Yes,

what increasing very is at We're complement complement pace. programs, we're interesting, think advancing. in diseases read-through So in is mediated other from field I is of disease seeing implicated recognizing terms an rapidly the

And advancing. seeing that certainly field programs and so trials of in other that in see everything the we're other terms we

validation that contribute is diseases can that's concept think outcomes. of clinical I in to implicated alternative and the the these pathway

we'll what us, data be see the we generate regulators to learn those see to that. for can as and closely respond programs So watching we how as

will So and our be plans. always that into we always we incorporating do that,

we're we I so can think forward. confident up as in soaking as to our it as information be happy And much and program moves own

Jon Stonehouse

extravascular The to other viable that don't is we a I'd thing takes isn't us study only of we've combo is approach. got monotherapy combo therapy on that to read-through believe a both marketed Yes. we that helpful Having super care because is And a key. intravascular believe add and that, the hemolysis. the drug

talk is compete and to we So really Charlie about don't let I'll think that combo differentiate. a winning strategy, how but

Charlie Gayer

competing, as just first first of -- PNH is you've heard, of as indications. it's many as Tazeen, just the far all, So

for at drug. injectables getting massive to really So good there's opportunity this oral. patients switching we from are And

Factor patients come smartest switch if out space, being in even we how in think of oral we on So the really terms to PNH. to get that can we D, the second based Factor B compete there in in

in of where just PNH, be differentiated. then be even will opportunity indications. we and there can in opportunity of there'll are PNH, there each So in be plenty And plenty subsequent then areas

important with two the particularly be information many U.S. and only clinical for the example, the that program ability of in that outside readable more give will to markets, outcomes, patient-reported we on controlled have us So trials, have may

places a a lot team, of and I'm got So We've can it. compete. great we excited about

Jon Stonehouse

And against in decision-making, both better bigger here. competing is I'd patient behavior on market, HAE the starting see We're Yes. showing gets there we the and that. is really add, We're if in Takeda into to not where space, well, the and things ahead we're Tazeen, else we're that pride that about that in drugs doctor us And the and already. work that doesn't the is affects to what tremendous to going of the one and one understand take last trying switching. of thing and that the in We're the CSL. to learn new apply within going what we're works And entering are world gathering market. as somebody with data PNH space gets the

So done team that what space, and HAE in my bet the can on I've compete PNH. seen they because Charlie's is confident against anybody I'm in they've

Operator

come with question line next the Our Fye from Jessica will of JPMorgan.

Jessica Fye

little First-up talk SG&A line, within talked stock-based prescriber about expect that exclude ORLADEYO year-over-year that? see second, to at numbers And OpEx to you more we R&D? can you roughly It around maybe comp, XXXX? about to And in the OpEx. pointing you versus the relative some in elaborate XX% midpoint, significantly, guidance bit the looks continuing can on growth you like then the each the is driving what's growth if a base put growth on growth

Anthony Doyle

Thanks, Yes. Jess.

we D OpEx. for the grown, support going growth investment has and to the So Yes, the first, biggest to program. on XXXX is be if but based it's Factor -- at QX right? The look predominantly QX XXXX to on it, R&D, it's for to

opportunity, opportunity in about pipeline was in I a I about of And magnitude think think right? to about terms so saying when a the multibillion-dollar the earlier got in talking this molecule. market the growth, We're of what Charlie

cash we the growth. with I is out be growing mainly and support in a D, if opposed making think that this on utilization of that sure support structural Factor the the growth gives investment ORLADEYO hand on I net the as think I company's in going a a potential G&A that be that have But will we The from I will that about the can think to stand-alone it's this predominance that with the you're way about. be to we're global cash line think some rolling a we opportunity is said, growth revenue to number, we and perfectly OpEx that support So have perspective ORLADEYO, the strategy the the great I to have. be investment make then There talking basis, on us, allocating like capital. to there G&A

Jon Stonehouse

the around on world. Yes. you mean, in I And about studies many many, many, specifically year-over-year, X sites pivotal think going got it,

got getting other There's and prep X indications. a proof-of-concept in and the stuff those number and and when proof-of-concept programs of that pharmacology. got up there’s programs, other just a told study working toxicology, time. Helen increases And you've over of there's additional indications, work, we're you on supply, bunch studies there's You've pivotal there's just

that's big the in the costs difference R&D around driving specifics So year-over-year.

Anthony Doyle

Yes.

Jon Stonehouse

And then around numbers on for things on But seeing. we're some ORLADEYO give like you Charlie numbers starts specifics and the the to to with that. not regard color what growth, on going can we're patient

Charlie Gayer

Yes.

XXXX lot QX seeing keep is in getting we just and saw all about of all -- and this like our so broader, we talked all throughout we've a base for prescriber Just targeting.

is in maybe got We little So we've that growth research saw focused. we X then a very key Tier X they Tier segments thing are And going larger the -- bit as prescribing we doctor X more. other about, The they really than doctors is then and more? I like twice, QX. much big They're are QX yes, roughly are that particularly is, market a are the those both So are. just prescribing those where talked answer that's of had Tier within XXX that, we're they in in the deeper prescribers?

to go is to they our both research focus what and both, prescribe non-prescribers So And that next that's they prescribers to current I continue all and we XX deeper. research, go in in that over indicated the months. that saw market intend ORLADEYO that broad described our current

team indicators And are and the focus. there, so the that's

Jon Stonehouse

excited thing face-to-face another more us think that's easing pandemic it's with got looking really like is interaction. I seeing we're And Yes. up, the

So gatherings we're opportunity, to of just more more not the deeper of in and of only there's I, our very in first the week sales getting them looking regional prescribe, lot top a going prescribing. prescribers at also And terms team. Charlie last and the at but

be going there, we're also are impact each on this I has at and so we looking meeting enthusiasm and real coming Quad hear were And will And college And interaction, other really Charlie more about and to talking with excited face-to-face docs weekend. that that November. the for there Helen interacting at we're prescribing. physicians. AI a the to in

So real get and we a to that's over opportunity. think it's time, better going

Operator

come Our with Chris Piper the Sandler. line next Raymond of question from will

Nicole Gabreski

Maybe for just peak Gabreski on sales one around is guidance. This Nicole Chris.

make previously opportunity had I would the up will you said uptake. ultimate be indicated ex-U.S. estimate. So potential billion know biggest the benefactors sales of the on the But have that that revenue peak one the you U.S. majority guys $X of new peak

here? So I guess, I guess just frame updating help in global point? confidence given this, just you us what gives this you guidance your peak revenue can thinking at

Jon Stonehouse

Yes.

and Charlie. to I'll then it me pass Let start,

The of get go clear. But very me roughly, it math again. to very XX%, U.S. math to from majority and I'll you let get But share have more. reasonable the vast -- and achievable. Charlie, to global It's there. sales, be the peak XX% the through you can explain don't will there, really Let the come XX% have market Charlie

Charlie Gayer

Yes. a XX% million. U.S. that ex-U.S., net is to to gross I be you it's so it's lower, described of U.S. prophylaxis to X,XXX to that math And need gets equal prophylaxis and patients we've kind a the the again, and pricing towards The we already an has we'll to in said demand going start XX% of more more global And in patients the as be X transforming patient for there's go And the U.S. volume. real markets, markets oral in the about our pricing the XXX mean, to ex-U.S. X that Anthony as happened But thinking will therapy. to as to are X volume. previously

that adds the are sales. confidence peak that think I year it's X. ex-U.S. strong in global we strong indicators the U.S. up -- to indicators from from So of $X billion the What customers all future from hear

Jon Stonehouse

Yes.

of one X,XXX base quick patients that said what off treated. Just diagnosed to a and add X,XXX is Charlie

between XX% So is XX% that's important. get where really share, you somewhere market which and

Nicole Gabreski

-- from Okay. we've helpful. some sorry, editing off, just therapy competition gotten more maybe potential competition. gene then That's And approaches. around but around questions, recently a gene very this is for a and Obviously, ORLADEYO follow-up ways

like do peak I your these see ORLADEYO? I to about certain guys guess, sales about guidance, you you competing guess, subsets just, as patient with something for that? example, do restricted how maybe think I being, So guess, thinking or more other potentially them treatment modalities

Jon Stonehouse

Yes.

all away. is I'd The competition thing first the far say pretty is that

oral controlled getting controlled be that literally on therapy Charlie, a once-daily oral once-daily incremental to you and if cause years, capsule, you're then that those on going to the is efficacy from switch, is be patients is it. would years right? able that not other And to so share gives to ORLADEYO benefit capsule. that you're on get belief capture a So our And will be market what

you're right? If don't it's our need efficacy, so else. drug, on something controlled you got more any And be to

none drugs important a the so gene could in right? are And With really patients role patients treat, to hard that therapy editing think perfect. these are really drugs, that And there I is where and are still of gene play are struggling. all

think is over. I -- spot. got we there's But you've what that's in balance to cross continued there a so And we therapy, with And the switching. this any study great to a that's risk-benefit when new that learned

you're else. see controlled that going our incremental you're to on the to what's drug to benefit? if So And hard for switch someone us it's

Operator

with next our come Evercore And Bayko from question ISI. will Liisa

Liisa Bayko

into treatment or lots renal complement There's about. There hearing a with a nephropathy, little some XXXX. of elaborate on have -- CXG opportunity development. of the you And the treatment just lot are a go in Factor then of maybe in rest competitive can more there? treatment we've Factor in compounds the IgA of specifically answered, bit there do of you just pathway, the indications inhibitors the but been opportunity for see been the Where complement area. Most and paradigm fitting a diseases can lot pretty maybe D you of options? -- that through future B

Jon Stonehouse

inhibitor, D other and might you in at for then markets the of kind indications. And of Helen, products, we alternative talk sense is one the Liisa they're Because right, look inhibition makes So renal want not there disease. pathway working are of about opportunity. proximal maybe root cause of on start but with how why to these the terms Factor Charlie, just

Dr.Helen Thackray

Yes.

indications is in complement the pathology kidney. of nephropathy in glomeruli IgA So by one deposition prior these. driven is in The the

and pathway, then getting complement complement deposition, the deposition from or so And basis the to getting improving it preventing that root at disease. is the the the preventing that dysregulation of of damage addressing of kidneys

indication And reasonable be complement in to that this, and that multiple seems address getting seems we've proteinuria GFR. early the of understood to that field way the now a sort we're to the and and ways possibly role to now it at improvement natural, that the that. for the potential proof-of-concept is that, of there are in a diseases so What seeing through then be the these of improve benefit some is there an that is programs that seen of

across improving of including is and the complement IgAN to for you shown progression long be shown The hope And treatment has -- to reducing to go patients reasonable the treatment CXG, -- way longer-term improve so improving the as has targeting and goal these to reducing transplants in been the term a diseases, mentioned. outcomes, kidney for proteinuria each failure for outcomes. need of

getting ways is proximal So them. Factor a D back are in we one are development, -- X it inhibition that inhibitor, be -- could of we alternative pathway it with believe approach there currently of

for outcomes. it's think target to chosen we each be and D characteristics the similar molecule could think better to we're advantage a trials. clinical groups, but those it want outcomes has at clinical what will We've of that similar reasonably of see and think, each for down You X and structural that, with because the to those will have be, expected I looking come we of Factor a particular

Jon Stonehouse

don't the hit heart at the end that we're target the in if why We better pathway. at That's specifically biology to going know it will hope the of if our it clinical the doing the a day. the heart Yes. of the causes But that the And outcome case. trials. at be you research after of disease, this we're hit you the is that I think see

want Charlie, you So talk -- and market? the about to

Charlie Gayer

Yes.

X the differences. in Liisa, indications, some So there's

risk or So with much This a for ultra-orphan line in -- This mortality. no -- patients, treatments. HAE CXG like is are there more there's PNH. high is current of like is many

market. that So we're looking at entire

at are segments patients higher was PMN, higher -- market who end-stage that for and there risk at kidney disease. there Helen are advancing IgAN of to as describing, are For risk

with initial the where small teams orphan the within that And product we all all in, as and compete so set proximal those be our a space, skill can within segments a differentiated compete we will inhibitor.

Jon Stonehouse

about best mean after things math another market, in I with to and right? huge ORLADEYO So to some bigger up we're it's that -- is think these the way cases, all way value. the like you disease get or add Yes. real going just each

Operator

of the from Markets. And Abrahams question will come next line Brian Capital our with RBC

Brian Abrahams

launch on the on and pipeline. Congrats all progress the the with

I'm might how just How the wondering of changes a over transient pull-through trends the early revenues the should the start lot us dynamics we if wave you -- gross changes impact overall any changes on expect some can of give we recent we And at quarter secondarily, should patient? I'm of clarity first those WACC a would as Omicron of from reimbursement of year. on of how patients curious impact gave expect on effects for sense the reimbursed seasonal to those some You XXXX? the the to demand well? then pricing course net per expect much

Jon Stonehouse

that Charlie, one? got you

Charlie Gayer

Yes.

we seasonal as see as I the of out Omicron about you're we seems to here. it. that? glad the be the We're impact not going the asking and on -- until that of to have whether Omicron far I'm end way any quarter well, think on So -- reimburse full comment

earlier, As going Jon saying to we're AI. Quad was

lot a be to in-person going there's more So doctors. with visits

been if in really but team entire impact, So we've we'll time, see has done it COVID well. has the any this working and

internally So all do to a forward. as now opportunity that meetings in-person and tailwind It more just going becomes well able we're as externally. an

will of And to on gross week. I X% last price guidance referring we've have gross we And higher. talked impact part no, the for net, any increase changes, same think think our you're that to the X% this far the changes, the took just this I As as to to year. all is XX% XX% WACC don't net about,

Jon Stonehouse

I and Yes. we have this very with affect common. that. contracts mean stuff doesn't And payers the it like is

Operator

question the Jon our Securities. come next of will JMP Wolleben line from And with

JonWolleben

enrollment In in frequently on are the me Just coming how may But for there wondering the wondering be indications. pipeline. a between uPCR? the to measure you couple -- RENEW We patients know if different just study, want guys may uPCR? go you to that we want a FOP that to fast you see trigger call. early improvements to on X-month then mentioned study the And and Or pivotal could to on see hard very I'm earlier?

a while, XXXX heard a that but to give look us can on FOP? for you We in in about haven't update just much forward what to status

Jon Stonehouse

to want the take So I'll do you Helen, the first one? take second.

Dr.Helen Thackray

Yes.

is So terms with the of yes, we're the weeks study, looking uPCR. there for RENEW in XX endpoint that

we in We the earlier, the to up we'll will be assessing as XX cohort. -- And it course regularly trial. have said of patients over XX patients the

So we're sufficient fewer we'll the patients earlier XX we'll decision XX that. a to with proof-of-concept information We'll be and a than point. we to advance possibly looking point that see make consider think may possible we a I to as time in at possibly than weeks. we achieve that just observing, what And if and see data the at -- have it's

Jon Stonehouse

we program FOP, capital, honestly. And because first complement Jon, invested then we was when less program, it, indifferently XXXX had than on launched this back

And invest had so we how be cautious we about to it.

So an it did we serially parallel, you and in rather I'll than example. give

we which programs up I, tox the do building While supply. we're drug weren't we're work other weren't additional running Phase parallel, investing the We' we a doing speed. for that in in

bit year. a we're so this mode And catch-up of a in

it's the because to can their feel you like key in and is not lot planning and a this because to a lot aren't finish failure of patient the it’s disease. It's a in situation it's a But it's happen doing space. not stumble. And difficult a this need. where product really the drug got they for studies there's huge medical -- space this And to is is easy jobs, This across toxicology seen that get We've of a unmet what's bulk So year. you've we've companies seen going supply hard. of you get line.

and have the drug level and in data a excited far, about tolerability I lot Phase XXXX confidence So so of the both study we're and FOP. from in safety, really that we've a perspective, seen

more So year beginning hear and this end the and now investing, about this are of towards we you'll next. of

Operator

And Wang the question Barclays. our line next of come with Gena will from

with question of will next Our Oppenheimer. Justin from Kim come

Justin Kim

on retention me. less then these have the was like follow-up whether there the patients Just interested Cinryze insights of a few regimen to though. Are for retention on see modalities convenient strong dosing X and I And groups? patient group, BCXXXXX. difference Takhzyro among a between those I the on any

Charlie Gayer

Yes.

harder patients What overall to just it's the we dose about every with of in these I are see patients' dosing, travel place it's don't the on U.S. the lives. think is is that the drugs. But about required. It's most Takhzyro. burden injectables refrigeration even X-week really And taking Justin.

#X is oral switching something think an transformative. switching. it's truly -- Takhzyro And to I And the So patients has consistently see that's prophy why that's we products been ORLADEYO.

Justin Kim

disease? the Any of Okay. specifically. commentary tees just transfusion PNH side for on have And discuss can pathway and renal AMD protocol looking for up sights Great. the you the their naive XXXX, hemoglobin to company study? things, alternative for the setpoints And then on GA how on a of set number suitable begun peers on dry monotherapy that beyond and the targeting indication

Jon Stonehouse

Yes.

challenges eye the getting challenge and and one. get explore for from companies one. a complement disease. spot that diseases keeping historically, on I'll so indications. company, real disease the there. could someday the eye rare been we've the a as those has and really and exclude take we're in drug But that then seen oral to So We're suffering is the to patients far you I second the focused rare we won't the with the take diseases, may Helen, thus delivery first and affect it

point, opposed priority not And to right Helen? it the some exploring so but now. I'm at not

Dr.Helen Thackray

naive if in transfusions and was clarify the what on the And the study patients Yes. I could PNH around question transfusions? just about

Justin Kim

and of the criteria the for transfusions points what's sort for hemoglobinsort that? So of set

Jon Stonehouse

low. if their and one Helen, variation they is So who maybe, it's qualifies as the of hemoglobin if get don't a transfusion, what around even gives transfusions

Dr.Helen Thackray

point have on and in is transfusion for to But decision rate guidelines an as give endpoint. when to transfuse. is, transfuse. protocol to physician’s We design [dose] an this the in where Well, it the when course, interesting of choose protocol you

be then the when it consider analysis what an actually consider So set patient we assess patient and look point a at how the to to we physician not there's you you level transfused you transfused, at whether gave hemoglobin be the at data, planning, the or which in which that patients. a a will

a So certain patients transfused well. perspective, and hemoglobin that standardizes or another and a for have data endpoint data standardized. they We not, whether but way. We standardize in received if on the would transmission collect then the analysis we also the consider their which it's been to reaches

Jon Stonehouse

the that different so understand the -- of from differences Helen, the very of And Is they're of correct? supportive regulators world, in approach. physicians and different parts parts this different

Dr.Helen Thackray

ways treatment that treat get individuals typical different used has been patients Absolutely. analyzed It's This Yes. and by the that may way of across is sort data their same a the regulators. to across fairly manner to in world. recognized

Operator

from line our with question And will final Wang the of Gena come Barclays.

Unidentified Analyst

Gena. is for on This [Harshita] me hear now? Can you

Jon Stonehouse

Yes.

Unidentified Analyst

from our have Most but of Great. questions ones quick us. been Okay. X answered,

under assumption you would we color Or is should color there company? on a And thinking you there, quarter. that there stand-alone So would For be thinking to for in about linear growth? second more more could ORLADEYO a of term, But provide any any the the or is umbrella? growth, of how question quarter-over-quarter helpful. there you've color be to helpful. are BioCryst? you Is plan first the a longer guidance big given XXXX? enough the think growth be how be the like for it If Do any

Jon Stonehouse

the shouldn't you me attempt first and I'm on the answer unusual one, going forward. to linear both. Anthony first be the situation to Charlie expect I'll path. you've an it's from if getting got down Yes. that that wrong point help if quarter, But in

second higher, and could some The could there second then and to fourth. quarter be quarter variability in be -- third the first

see along no little for to you we're what what confident what will to trying both first than quarter-to-quarter less growth. for the guide million we think confident and very to from in the the see do first that that growth we're on continue then for we be clear very, we the the is no will here year year, quarter And the and to reasons Charlie And we'll the $XXX we is way. So year. all that in very are listed ORLADEYO in

Anthony Doyle

revenue. patients events to important of in Jon focus, continue X. grow. it's And think terms in QX will we'll said, I centered normalize to drug The highlight revenue Qs that's onwards, QX on The see there I it right? around X in more from that like getting think and will be X, start fundamentals to

Jon Stonehouse

lot could biotech our they platform. pick build to very look from revenue. going And say next and right Yes. ORLADEYO. we bigger a one, exactly we is by to significantly allowed that building in pipeline hard, full their came think, pretty stated. we a I have great or And that -- XXXX confidence And favorite peak second of in valuable while investing our pipeline we're new question, this then bringing product the but to engine than that's got got next that, billion you us is all And get the we simply And market. your $X approach it. in company. we've your company. sounds how and behind value, is exciting the great be the if And tremendous discovery at because the that's drug will stuff really reason It's and on a companies, great you is it And got mean, to biotech of that, to a they're we it's think allows

Operator

That for is all we time the questions today. for have

the hand I So Stonehouse closing and for now remarks. to back conference to Mr. over like would comments any

Jon Stonehouse

Thank you.

what, be company years, been the in mirror. I've best had. the XXXX, ever year it's rearview we've But it So the XX might guess in

our of and ORLADEYO, We that, another great laser-focused other great pipeline. to growth it of getting that the at a end 'XX, are on performance advancing focused countries, to approved share we with And have continuing off so intently in on U.S. year build remain in driving you. will we the

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

Operator

Thank today. conference this you. for Everyone, call our concludes

day. now may have wonderful You a disconnect. Everybody,

BioCryst Pharmaceuticals (BCRX) 23 Feb 222021 Q4 Earnings call transcript