BioCryst Pharmaceuticals (BCRX) 3 Nov 212021 Q3 Earnings call transcript

Company Profile

Participants

John Bluth	SVP, IR and Corporate Communications
Jon Stonehouse	Chief Executive Officer
Anthony Doyle	Chief Financial Officer
Charlie Gayer	Chief Commercial Officer
Bill Sheridan	Chief Medical Officer
Helen Thackray	Chief R&D Officer
Tazeen Ahmad	Bank of America
Brian Cheng	Cantor Fitizgerald
Liisa Bayko	Evercore ISI
Stacy Ku	Cowen & Company
Chris Raymond	Piper Sandler
Jon Wolleben	JMP Securities
Gena Wang	Barclays

Call transcript

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to the BioCryst Third Quarter 2021 Earnings Call. And at this time all participants are in a listen-only mode. After the speakers’ presentation, there will be a question and answer session. [Operator Instructions] I would now like to hand the conference over to your speaker today, Mr. John Bluth at BioCryst. ahead. go please Sir,

John Bluth

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

will Following your answer our we remarks, questions.

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

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

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

Jon Stonehouse

drug XXXX, of they guided the Three are the of amazing we evidence. sight this quarters achieve everyone’s million and a once-daily John. opportunities line exciting started. color for company. get now Charlie the advancement launch even are will net our what and pipelines their prior way now the is therapy are of The What’s They patients Why? of between the We rapid these want and the by on to prevent is of year, a control ability oral they into ahead. attacks. trajectory to their to burden of have of it in an ORLADEYO more. pandemic, bottom-line with we all with medicine including expectations full reduce lie sales the ORLADEYO significantly been that more exceeded therapy. million did and launch to The $XXX Thanks, controlled strongest Because option more want measures, will our treatment to share that an in have is, the execution successful we has we oral good that switching $XXX and market for a when and year The continued evidence to see our even revenues. options. growth from launch. and for and first the is they disease just are getting satisfied to of injectable HAE

will research future. attitudes two-and-a-half we market told in us those playing same recent are out in surveys the share seeing that exactly ago, show marketplace years and Our the will Charlie this it the way increase

the and capability a rare over us and world-class market make oral BCXXXXX to Factor that’s compounds targets. ORLADEYO different was continue the it will growth launch strategy As around from the gain consistent available countries we to over exceed we and launch that to this next. many difficult the the very we approvals develop on the and of in and first million our is platform D our global drugs confidence in steady impact can to disease world, continue potent, with once U.S. our our discovery substantially of we have and ORLADEYO showing engine lives unique bio orally its stronger was studies, quarter. target of that a oral But $XXX peak sales in through our repeat inhibitor against other patients can again disease. differentiates grows biologic rare have that huge every discovery specific,

suffering from disease, to only Going diseases D complement allows to demonstrating key but After patients the are target to as in treat in study a there bring an nephritis PNH Factor XXXX, oral as and a inhibitor with three plays after a not pivotal oral role pathway rare us with D drug Factor multiple many are proof-of-concept studies mediated a late in alternative in we proof-of-concept now diseases. last indications. in another PNH year patients

extraordinary is BioCryst quickly their we let full are pursue the Our alone working just pipeline to good and treat oral many be news behind started. and many ORLADEYO. any disease, diseases are continues There to rare drug and patients and drugs to year following an for more getting is, XXXX for

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

Charlie Gayer

months to months off got drug launch after expectations. high as were in of grows oral really eleven an going to prevent months. exactly wanted knew taking better ORLADEYO This than that and a start patients even we that what seeing steadily great John. ago, is attacks Thanks, patients number the We our own HAE

prophy More well. other third our from the and continue staying volume starting than are therapy line saw ORLADEYO. in For are straight switch same quarter, patients with to we they because really on doing an the of patients treatments half expectations, new strong

physicians within but patients program, also XX% now half patient now the nearly and base treaters. Quick ORLADEYO. We to also includes continues XXX XX average Access top more prescriber Nearly all product the benefit QX paid see embracing The days. in Start new to continue from improve. HAE new receives another grew our by

treat very Comprehensive been, to we to we clinical of ORLADEYO the we each. this favorably reported with large HAE months. ORLADEYO XX has as of expectations and next over another leader launch. shaped what They XX patients has prophylaxes. accurate see to physicians HAE surveyed samples August, market an experience strong are a As our strategy in on prescribed seven physicians prophy has the U.S. average predicting use that see most their patients become In of HAE their our treatment in trajectory and the seeing market doubling ahead make and guided research long proven in launch be will and

launch. XXX substantial and number is Our prescribers the fuel back has internal prescription up. top the since physicians also multiple doubled are will continue this quarter. There this The data among first HAE that to of catalysts repeat

showing the of data sustained from the even XX-week APeX-X attack attacks to therapy to them in is persuasive from new That a data reduction expect give long-term they For confidence prophy more example, what prescribe control and level is physicians. XX% ORLADEYO.

conference We our New be with also attend recently in-person patients began week patient programs the in-person critical mouth spreading activating which excited College major were of to Meeting very And education Orleans. and word will we to and medical in this first experience.

switch do We will present patients HAE to from products. other well on they data prophy when ORLADEYO how

of of treatment will two access group Takhzyro products month. received XXXX, for HAE of QX the future add per majority experiencing report of ORLADEYO attacks recommendation covered is will for or launches in account much key having will points will the patients what reimburse for example patients in to covered first-line week. more prophy program for market. UK more So the In a This of ORLADEYO more we our two are but stay UK. in the and be and the coverage global tuned. attacks limited U.S. for all end this make to ORLADEYO sales will inflection the contrast, to Cinryze patients nice By A nearly history or month, favorable allow ORLADEYO growth. great per team’s expanded convert in for a patients

world we approval years seeing. world and a only is treatment. HAE ahead. with are controlling Germany, UAE reimbursement to turn Japan, clinical composition Norway, We minimal and also lives are available. have burden bring underway of where currently the of to the Cinryze a and I’ll with to in to France, We on that all experience we intend Launches over it XXXX, secured It boils patent a protection many align patients with the the over global of to down October more to with are matter can this. androgens data of recently growth in drug Helen expand attacks of through change how patients market by come. our real HAE ORLADEYO ORLADEYO

Helen Thackray

are impressive APeX-X was potential based HAE the consistently Charlie. experience rates. their is just ORLADEYO The has understanding our HAE attack hearing impact Thanks, beginning ability of of the the physicians truly from XX prevent for of ORLADEYO’s prescribing been of on this in on experience trial The something weeks the to and pivotal trial we first attacks ORLADEYO. own

baseline Now, in have in it a patients The reduction in XX% placebo XX-week drop XX% XX the time attack was rate. then, the pivotal weeks, from long-term attacks reduction rate to XX% chose attack early the average reductions when registration of an the or the was compared follow-up trial trial greater in and reduction We greater the This their in were demonstration point was we patients, original Even having a with then at we’ve into the seen in attack observed baseline. of baseline endpoint. own from in patients at saw which XX% shows a is that attacks rate. there group for clinical durable, comparison substantially data, attacks. substantial

are U.S. we from us observed is and is of XX% we providers. well in to high so. launch the Now more patients continue that that there what and world tell real that doing with rate evidence from reduction They the are do consistent hearing

little durable. have attack trial, go here. rates extent Now, is the Conference a find July they why. we’re large a the attack the patients can generally We so at understanding, on in outcome ORLADEYO. opportunity as what see greater in compelling To in was EAACI we more reductions follow-up know we long-term newer rates that our this from is and future are detail presented that into well to reduced about excited the information continue long-term With sustained do this the and we treatment,

of can months reduction in the during attacks and and Patients in experience almost on treatment of prophylactic only on What’s are rate completed here month patients ORLADEYO experiencing see And was decreased two ORLADEYO, per is month attack enduring treatment the that, durable. three. treatment needs of notable XX% over on-demand X.X of large decreased who in attacks treatment patients month median Specifically, average staying rate rate attack regardless the attack have reduction results. prior and that baseline that we XX weeks XX on per two time attack during observed and the parts X, for Slide from you have is and reduction Not with rates in to three. treatment. per have mean XX zero at from three years and baseline parts see response,

also continue long-term is follow-up world Finally, through the through generally launch. XX real the treatment, be of to ORLADEYO which consistent with months our well-tolerated observed experience

ORLADEYO. most for excellent the ORLADEYO, on therapy tolerability ORLADEYO. are switch on strength patients and More seeing seeing happy staying more the efficacy just and patients profile, are excellent when themselves their they with physicians to of and We having are experience durability how

be to Allergy which as we It each excited are American be experiences Asthma a Immunology the we and through heading to College together share mentioned, to of opportunity we meeting launched. be New first Charlie live As other. with an come for Orleans doctors came Meeting, will their since and will

for prophylaxis to injectable We data will new who ORLADEYO. presenting also on from patients be our switched outcomes

from earnings right robust clinical for the total, how program we last the an across multiple including evidence exciting pivotal will impact towards on effective to proceeded provide indications. observations call focus the and pivotal behind XXXX patients. Center BCXXXXX. and study update of Of be for into the our seen diseases trials have of PNH Discovery we’ve course, treated Birmingham, treatment full our Based coming In we from changes advancing with in to rare subsequent of Bill for clinical feel of that of build. is Excellence that we’ve the on continues oral reported Today, concluded an and our PNH medication ORLADEYO, effects momentum expanding development at in BCXXXXX, have to rapidly Bill PNH also next data, our these there in and we the give in turned therapy patients potential and indications. this study

durable with Bill? both drug burden potential in the and of for deliver do treatment has the to We are disease improvements that confident and benefits to burden of patients. so this

Bill Sheridan

Helen. Thanks,

and Slide X. trials, both endpoint is of change regulators. moved proof-of-concept a ranging the from selection secondary trials important pivotal dose two baseline As pivotal to quality of measures hemoglobin endpoints that that blood fatigue primary program secondary also to trial The endpoints Helen key noted, The the BCXXXXX the primary with directly the has measure life trials reminder is red Both trial PNH and in on FACIT include transfusions adds measure. cell an designs treatable and and from score key dose trial. as shown In endpoints. agreed of

reflect in and these true REDEEM-X Importantly, same PNH with CX symptoms. the In inhibitors endpoint. patients patients transfusion placebo-controlled trial baseline treated from also not endpoints with is to of or related LDH PNH with goals in the complement reduce the treatment of change secondary inhibitors, a eliminate

over benefits hemoglobin analyzed turning the outcomes As who BCXXXXX measures bodes patient the is of continued valuation to in mg is mg REDEEM-X shown each we the on monotherapy Slide Slide or on table XX. with XX. and the panels time with from to months patients XXX the proof-of-concept in the the as trial, X, the In at slide. patients the BID explained We The who the that XXX proof-of-concept REDEEM-X trial, September. according long-term in data up relevant had same figure CX X Rollover naïve received in to the our on XX have week end safety of to inhibitors the

not from or in CX nine proof-of-concept long CX on Six for rose FACIT-fatigue Slide trial. naïve inadequate per scale for as As same outcomes through inhibitor are responses to who points. perform X, inhibitors, treatment. very come in confidence week-XX free, by X.X with inhibitors dosing XX% continued cohorts. see, so will will in X.X placebo lowered you that added REDEEM-X, BCXXXXX BCXXXXX also against scores hemoglobin CX initially to be that’s minimum data are the well REDEEM-X. give inhibitor clinically can the way into transfusion taking by deciliter rose grams term With not is The difference see had total trial in In display from week-XX. And These results patients can oral efficacy they the patients LDH you measured from impressive. proof-of-concept patients the up set baseline XX their us familiar and important these score with those are

baseline data and weeks We one from XX main have is presented analysis, through XX per excluding would who both in be that overall deciliter. the from for sanctioned In the ineligible results patients REDEEM-X. grams X.X hemoglobin

the unrelated three by discovered As to you and who regulatory hand for warm Anthony anemia FACIT-fatigue score and trials antibody X.X very us can PNH excited conclude can after the see that vaccination as and immune patients over was disease. transfusion-free in the anemia reaction have but COVID-XX patients BCXXXXX. benefits safety to many against in world. was us well developed evidence the described from recently patient give approval or signals we to seen remained This things all and hemolytic quickly patients that dosing transitioned but confidence into update. normalcy. unrelated REDEEM-X at BCXXXXX call perform inaccurate long-term The BCXXXXX. BCXXXXX dosing results pivotal to These follow-up to without transitioned like benefits to to was The were diagnosis other and to U.S. points. file have favorable One withdrawn monotherapy developments will continued monotherapy, patient In with at rose through or make this and profile, vaccination our other BCXXXXX CX to as response get to now safety with the long-term around long-term for accumulating applications I No I’d in of the an inhibitors same. patients.

Anthony Doyle

for deliver Thanks, presents multiple bigger both from across XXXX ORLADEYO fantastic value for a and opportunity with drive path patients significant follower revenue indications to shareholders. Bill. even an Having and

of including $XX.X third of attention $XX quarter million came non-cash in to Operating million. expenses which the a financials I’d from was Revenue quarter items. release net You like the can for sales quarter find few ORLADEYO. not compensation detailed were our stock for $XX press call earnings to today’s and your million,

gross to net including towards drug Our access improve is free to to target our and of continues XX% track adjustments sales. XX% on peak to of

available R&D continued well for As strong million their runway cash, meaningful, strong are and to markets us access strong revenue Jon full forward launching we and enhanced puts of a see, continue ORLADEYO and that We positioned advance very and XXXX us execution with very program. gives ORLADEYO ended are beyond. Athyrium revenue Q&A. in path international of of be positioned additional QX position fully revenue like different be position expectation trends for our will and the for $XX that sources the confident more XXXX $XXX Based here as international and capital continued open and creation in with invest is engine, become as our $XXX that we’d we our million, from outstanding significant with set ORLADEYO on revenue and well as to sustained and more a in financial come for an the growth Operator, million and U.S. the said, to It net to very year regions we from BioCryst. time globally $XXX to now results at Factor ORLADEYO online. D generate XXXX access exciting on it in to the shareholder. is launch million multiple With growth are BioCryst to This from will cash few biotech hand, come a XXXX. we enhance to cash into cash from to up

Operator

[Operator Ahmad from from of America. of first comes line Instructions] the Your Bank Tazeen question

is open. line Your

Tazeen Ahmad

questions. Thanks A on my couple, one for taking ORLADEYO. maybe

give a reason your dynamics in of criteria, way the your PNH, get be sales? where So, might seen wouldn’t Jon, starting in you. dynamics how maybe you’ve us do And for think Europe. in any U.S., think Presumably, here sense be Europe a in uptake how can you countries. are be there give than question of the in data? see the those Thank us you And any Based enrollments you’ve now position is have would your in to that seen a be then, when different Germany mind a for a that you one about inclusion a sense plate likely to to can in fast Bill. proceed? And on uptake you’d you to here. fast secondly, top-line Would meaning you

Jon Stonehouse

Charlie, question, just Tazeen. this the for to Thanks I Sure. sure make get right.

were So, cohort have are and in in ATU actually Europe France. in drug Germany. are a the we launching selling We contributors

the is, So I the can had be as and had UK than in is UK. France. will patients the in has the bigger have patient The going And we selling drug difference by impact. we month, and will less Charlie access end Europe the in of mentioned are tell on there telemedicine you in There that in COVID less. has been interaction a doctor so, U.S. selling been

why the at think I a little to ramp Charlie, you getting hard is expect we XXXX teams pretty But a EAMS been about really just for the And NICE bit up. so good working a contribution you are might UK. about the and talk want bit slower. and set excited has more So, and little

Charlie Gayer

UK mean thanks, limited fact things. very attacks more. very going And two One positive going that ORLADEYO two for it’s to from NICE to I recommendation per and deal. much what’s The any Jon. been per that’s the mix with - Yes. wider exciting month that more. rare anytime a big has or modern a two with prophy week you to is of open disease But get or patients with it’s attacks –

expect convert be and we that ORLADEYO of majority that have eligible over do the we we are EAMS those to few can the will that and market the frankly So there next for lot, months. patients

a can probably where the the faster the out of we little to gates extent. UK the get U.S. is place one so, the like And

Jon Stonehouse

that to an the before the and country, course last is to generate over and opportunity we continue XXXX that and and it patients as beyond. to revenue Norway I’ll hand more a Bill then, is, get we’ll it’s more example an Tazim, make And point of do add I over

Tazim’s So, criteria to and Bill, impact question on do take entry you want enrollment? around

Bill Sheridan

the Thanks Tazim. Yes, Sure. for questions.

is and of the eligibility opportunity these characteristics plenty attractive complementary to mitigate other studies is XXXX, and actually. I And studies of characteristics very inhibitor, the think a the there in both see the criteria. to up. in eligibility on community particular the hematology barriers from around regards track I are don’t excitement the with world, studies these start studies Both participating proximal to but

predict will field really busy We that now. are finish so competitive. It’s doing we to right enrollment is hard when because the very

the predict what see think will have on first when until time and it build few we to I is be will So, we the months the trajectory exactly finish. the like,

Tazeen Ahmad

developed you at there world? do And think kind COVID least impacts of Okay. would that the any be still in

Bill Sheridan

So, how COVID. figure to I continue did trial. mean, the think to that have trials clinical proof-of-concept started I people in out during we

course, Telemedicine one. adjustments but medical the You have an got make to as practice to example of in go U.S.

we to I is big clinical there supply out continue as impact. did But think important in are I all know chain the that, also general community, trials. disruptions how think figuring

Tazeen Ahmad

Okay. Great. Thank you.

Jon Stonehouse

I’d great different high and every done has are Bill of that point for demonstrated a and of he that connection many enroll. monitors program. but absolute Large numbers on, investigators to of him to sites break and site, with with pride patients quality other that I’ll execution Bill’s on quality job add really team make won’t like then HAE the is medical has to probably about risk One the the can that planning team touch but his talking around ensuring single countries

big that of trial. oversight the in a makes high the not so quality, touch but and And in difference quality high speed, outcome only

important to So, piece study. success that’s in the an pivotal

Tazeen Ahmad

Okay. Thank Jon. you,

Jon Stonehouse

You welcome. are

Operator

comes the Brian Your Cheng of Fitizgerald. next from line question Cantor from

open. Your line is

Brian Cheng

team. morning, question. for my taking you Thank Good

to that then, the versus base? have adoption pace second you follow-up. you. on mentioned the any summer you Tier-X do on the more the I more previously base. the last. a difference your provide compared significant the It if a growth Thank to any of quarter in How can the the prescriber seen wonder, headwinds holiday And, prescriber fluctuated I And call. Charlie, you rate a Tier-X has this have for variant Have two? maybe growth of much one fluctuation on the quarter, So, think is that color of you bit do delta bit due seems and you color between

Jon Stonehouse

Great. Thanks, Brian.

growing strongly. it’s the overall base, on prescriber So, really

And base so, grew XX%. in you that I that are said by think we the to QX, referring

And This about Tier-X, on much quarter are I excited of as as far I the penetration particularly in it thing And to. attention in Tier-X that’s what key is some base. and top said a the XXX think grew Tier-X by to versus larger pay growth we my XX% the comments, but as physicians.

we of about up prescribed. almost are XX% So them to have

We us market amongst are double a our docs with and tells that prescribing seeing the doubling repeat research of just those like docs then a twelve again forward-looking months. that over next they expect

tier everywhere. So still going We are still fantastically patients going well. next really we But market, get to going well. business Tier-X every it’s corner is the because from of there and are explore this

Brian Cheng

Great. think And commercial access. on about how insured us then, access the now more on And access Can for how ORLADEYO you patient? your remind nice Year? in looking we Thanks. should then, one New

Jon Stonehouse

Yes. Payer go access continues well. to

product really first the paid are gets average – but patients quickly. the off, patient getting So,

We through more great the expect what it QX, continued XX into of we we So policies. throughout expect of expect in had coverage the had days, have progress we and couple terms improve. payers but a go to and year. we rest continue within next still year wins this that’s in some early progress and to to We year

be full a plans switch that are into that. turn for around as to time year, will ready the second us we key and for patients As we

where about are with good really access. we we So feel

Brian Cheng

Great. Thank you.

Operator

comes the Evercore. Liisa question Bayko line from next of from Your

open. Your is line

Liisa Bayko

for question. my Thanks taking Hi.

in you guidance that for revenue clarify include you can Just, what ORLADEYO?

Anthony Doyle

Yes.

we in included of revenue vast So, the U.S. it is like the net said the it’s from is, going come to majority

and U.S. mind, to based the that’s have from no began gets and in the And so stockings it QX, to in we – be stock we when bear just I will in will here it countries – XX.X the the European followed, that it in included recognize when U.S., the plus patient is just it’s added then year-to-date revenue. revenue a direct no perspective, on shipments, think

Liisa Bayko

fee – royalty? doesn’t not of it royalty our and that’s not include And to Japanese it’s your the net Okay.

Anthony Doyle

Yes.

The So, they’ll borne What the Japanese, see line. Torii, those include will Torii. revenue royalty will of is, full the things – the not the line. include amount we below it from that that’s with take our it royalty, because Japanese by hit you that’s portion does

Liisa Bayko

Yes.

gross give Okay. of us a flow the And can to what sense quarter? net you for

Anthony Doyle

better. said What gross we continue is to get that, net Yes. to

So, vast majority improve. net free Charlie products. the access continued The continues has gross adjustment of the for to to about talked the how to be

adjustments We about when when defense. to least we talked better XX% continue quarter-over-quarter still XX% that we and payors get get at to are seeing that we rise and will to get track get on added. to It are our we

Liisa Bayko

Okay. Great. And seeing kind thus far? distance then, what of what compliance, are you

Jon Stonehouse

Charles?

Charlie Gayer

Compliance – people our about has high. taking day compliance, pill one talking are if a you really medicine, been the

with our north then, strong, XX% was in line like been expectations. it’s So of just as well, saw it very we clinical been in has trials. And persistent retention patient

Liisa Bayko

mean? are expectations? your that What does What

Charlie Gayer

this see on one on settling at we are still started – now, year period, who we that’s to out patients ORLADEYO. QX, And period right So, a of have see ORLADEYO XX% settling XX%. around that overall,

of across staying about XX% on So ORLADEYO a year. patients

Liisa Bayko

Thanks. Great.

you can is then, quarter, you there maybe due? in include, there And assume. when just is about third talk just net revenue, talk ex is describe, for that I is revenue U.S. Could about the mostly you there and it Okay. or some

Jon Stonehouse

Yes, all nearly revenue. U.S. Lisa, it is

Liisa Bayko

Okay.

Jon Stonehouse

see you will U.S. in outside XXXX. more The – contribution will the

Liisa Bayko

so then, final And me. Okay. question from

over kind more to of talked engine. about developments from You research your looking

Thanks. any can on the you a And program? lines, that? update FOP about little those bit talk along then, So,

Jon Stonehouse

available and this orally and we proteases like serine bio the we The as molecules really Sure. I prepared platform have is potent can hard strategy specific where of build beauty and discovery said biologic targets remarks in the kinases.

that, have been so, unsuccessful, have stumbled. And others and right and tried seen and we’ve it

And to bring patients. deal all with do so, an medicine three you oral to these got

Factor And Helen and inhibitor and the others – we so, four study. this are hard what with as you starting working are we as D already are we and oral chosen beyond do are we’ve our working know indications go

bit confidence we a thus space. said But then, inhibitor of in the have else to really to as working been And we a is But shot, catch-up for come. going be on It’s are ready playing see will are hard real a sharing other lot a are work. they is far investing talks of rare in for this that’s these a fast disease. for so, lot that we XXXX supply for and front with time, tackle there team when of more after Babu our a and for nobody in data disease with targets unmet and you And therapy disease trial need what more drugs still drug us we there getting gives prime and X Birmingham going that patients rare LX the are and believe exciting. then, will on what we in be patients that has Phase and nothing. FOP, we

Liisa Bayko

next So, when precluding will what’s moving it what we you FOP timing? like about for hear and the from steps now are more or

Jon Stonehouse

Yes. supply and been The year and bulk talks. of this it’s the about all drug

no regulators any Then So is step of the about what talking kind path the opportunity there development. get clinical important was really another into to to is.

more the disease – that treat, impacts you is the making And earlier this a you have. so,

impacts. children of kind to to And so, have to what’s age required those get that

design got of we to work and moving trials through forward. continue then So, the out figure also, that to the

up you to feed more we plan to are to are So lot right year. hear a – start we But a on about next we when clinical trying give go that it and trying trials. I really can’t now, timeframe

Liisa Bayko

repeat to He doubling to catch clarify, wondering sorry, didn’t everything, kind And kind if Charlie, over you color Great. talking just something adds. I I could of Okay. as then, catch didn’t about patient gave next everything. like twelve I about. you said six were months. of the on some was just

Jon Stonehouse

market The Yes research.

Liisa Bayko

yes. Yes,

Charlie Gayer

large We seven next We seen months do HAE at Yes. so what Lisa. really doctors. are become another said physician prescribed those and they with what Sure, prescribing prophy XX see they twelve far and is surveys of doubling the survey with their most they happy their docs each patients to therapy. did they’ve average for HAE over

Liisa Bayko

Thank you.

Charlie Gayer

Lisa. Thanks,

Operator

Company. & line next from Stacy Your question comes the from Ku of Cowen

Your line is open.

Stacy Ku

have I questions. thanks for and progress a our few. the on Congratulations taking

QX to continued First, about commentary the growth. forward given your months, tailwinds encouraging see in very last the few

current – follow-up. And much can any also of on percent comment point? So, of expect this face-to-face the COVID? Could you can to on a back on provide have expectations provide do you at sales been early that year? level impact growth without I and a you this thoughts calls going some magnitude you the How some have then,

Jon Stonehouse

don’t why we about think in in to about kick talk handle value you I’ll expect. question to And going that add the what things are Charlie, the the XXXX? you

Charlie Gayer

absolutely. Yes,

first that data reduction, XX-week starting call, the mentioned to been Stacy, So, about roll talked we talked I are And out. sustained the off, persuasive about, the XX% showing that on of some to that’s Helen just things I really physicians.

So, that’s one. big a

like HAE to do are don’t We word face-to-face because the to the thing, things do just then, and key prescribers contact on And patients This there. meetings meetings, College in starting with live a going is important. really Zoom. be weekend to big Orleans, patient is doctors. in lot that another which of New with And mouth live states are the of Meeting

with be them. We going are to meeting

data more new to many coming in We kind those expect of have of meetings we and live the have year.

All that in and to patients the growth seeing. going steady to contribute strong we of this already are is

Jon Stonehouse

for a we just not guided And with expect regard Stacy, to guide. year, today. year, we to this then, next what are position We in

we will. but So,

what don’t and we from Charlie, we – caught, prophylactic of in see line be physicians, the of a but patients. HAE very of this have the market good sight that you trajectory treatment now what expect a if we the will We hear know leader that he this launch. from on has I said

And discontinuations the our as line of filling and base with slower discontinuations Right. new as much he a you rate a you amount we on. consistent is told and team are steady the expectations, than funnel there working adding we as And so, that Charlie’s is single really of asked patients in the solid month. question said, about that Lisa rate And is funnel. the are the into every which

that’s So hopefully, very am picture a a that curve I just of you steady growth. painting shows curve, the

more on give your the a virtual have So, to definitely global a we than of the about because And the for general, we that we just little answered QX, of on top But at In calls in we’ll expect our give into question we accurate. Year, but don’t also it’s top calls. – peak, and got XXX% is point, longer XXX really think XXXX In forward. would peak the side. sometime a we I going number in expect our continue calls QX, sales I bit, in-person some calls. New that adjusted reached in-person physicians. the prescribers, are an And I sales Tier-X no that lot more dropped and update will plus making guidance I Stacy, think

Stacy Ku

helpful. really for on It’s And XXXX. one last pipeline Thanks. question

data? basket we seeing renal initial interim start when the study, For could or

Jon Stonehouse

tackle that Bill, one? to do you want

Bill Sheridan

renal alternative up nephropathy. glomerulopathy, and that in a membranous is basket pathway as specific set complement And nephropathy three CX are by nephritis primary real learning the Sure. experiment conditions IgA study driven

So, possible. is do as what as to going we are learn much

that that sure that also and is that allow the now. all forward We months. to periods to enter to do screening started the are we up study the to a and at and make want both recent of getting the of results patients up to days getting seeing pathology The illness look next right active are an set have of this time we biopsy. require we course sites XX for the they over intracentral have to Broadly review

really when what and data hard results to are enough at rare, take going because a predict disease it’s these think, like. mostly to look we I are have to the look

As a patients to design reminder, cohorts. that’s the

it expectation in in to that a field exploit an of lot to be to you benefits nephrology a extension. research there and the going know of experiment is this complement and is as exciting So

there approved. So nothing is

Stacy Ku

Thanks so much.

Jon Stonehouse

Welcome.

Operator

question next Chris Your Sandler. from the of Piper from Raymond comes line

open. is line Your

Chris Raymond

Hey. Thanks.

couple a side. Just questions on the commercial

XX%. of from quarter, about So, I us a year that I from of I then, previous new that I now, question expect give XXXX, And it how ORLADEYO two to of give question, drug, due one to maybe that second you last sense you year last think now? switchers driver? drug sort most the a than a was patients more Thanks. therapy? patient is of your from the think sorry, half can to to evolve of now but patients the steady you sense versus discontinuations think two know kind you today it Can efficacy/tolerability you to What’s I years us I of those that a or you is to it are access you common Do pools, Is commentary thinking around or XX%. and maybe just year or new guiding heard are a say guys, of read have state injectables I sense noted the that not more discontinue? the switching new so, those around related? you think

Charlie Gayer

the Hey, Chris, thanks questions. for

from next and see coming continuing all and the that we referenced, market First of was saw, trends all Yes, the evolution was than on it I’d over the than the see patients months. XX%, to acute in greater doctors research XX%. see They we closer prophy of XX% a I products continuing It different actually little switching. to QX, only. twelve that the back that but

towards trend treated prophylaxis growing that So, see the the best been for over maybe being over patients. XX% to the more is more see we general, prophy patients and in plus, XX% number with And long-term. eventually, we has the

the the therapy prophy switches start probably of some from longer they are of being as then less year, only XX% ORLADEYO. growing the on usually rest newly acute treated newly prophy market, next diagnosed from trends come or will patients than for term patients kind same over the So, and we growth their and see but

drivers some far has have to are said the events before, do be and – the we expectations. set as adverse adverse as We As patients so some of going particular. XX% GI retention some that drivers the biggest

know the to that away then patient. and drug is be And go going No drug likely patients early for in So relatively to that just therapy perceived those is is perfect no every helps. and are other efficacy.

job And access give they be helping really will really if that them it, we and other that’s patients So therapy. doing not cover thing. because the has been big done anyway access doesn’t payer we’ve if access of well It good are ORLADEYO. on a

are and well And doctors that. so responding really the patients to

Jon Stonehouse

other, to be to to I’d there are Chris, new what opportunity continuous to prophy to talk that’s that still haven’t on on of huge talk the going get made drug. think And we we patients time. well the of add to yet that switch each more said patients over get their when when patients when beginning we patients, believe and therapy a to is doctors And controlled our at flow that access steady them, a will Charlie decision

Chris Raymond

Thank you Great. very much.

Operator

question from comes of Wolleben the from Jon next Securities. Your line JMP

open. Your line is

Jon Wolleben

the Thanks good progress. taking Hey, morning. for question from for on couple ORLADEYO me. the congrats A and all

world? the you assumptions. you to expect are You leader a And the mentioned HAE wondering it ORLADEYO to are is supported am a you demand couple Or the be market peak also, seeing trend your in you this out discussed? sales looking real if from for now that’s with times also survey primarily then, prophylaxis. you I change mentioned Jon,

land to am dollar I I be wondering is still think a Takhzyro expected So where about billion could product. that the

that be That’d how time? think discuss out dynamic So of if you could you over kind plays helpful.

Jon Stonehouse

Charlie? Yes.

Charlie Gayer

leader growth spec So, on seeing steady and thanks, strong what patients. month-over- as in market described, Jon. is, month the are we for And is I really

And data these the repeat happening the our see that and fact well. we growth internal groups. the in so the that’s physician the just physicians. physicians primary prescribing see in thing. see steady We that the What then among And supports research it that we know market opportunity as market own

our the what all with on that. for patients’ once-daily that that’s orals put – outlook you If desire our that drives and together

Jon Stonehouse

drug the to Charlie degree what referring tone Yes. that docs And and and leader say then, that’s market then market marketplace then believe have the to we prophylactic and most leader and will is we where we think on the now up. back it are out, of is peak prescribed the seeing net high allow we to evidence – the in the really and have confidence switching the

a at are not significantly is, of point to of that it’s XXX, tell yet north In we terms but what you. number

which to think is, evolve part biggest contribute does be ex does number. will that the I how we and So, soon, how U.S. it

Jon Wolleben

us It’s on adds don’t And per me. it’s out days what before. percentage for mentioned I and But on. given patient You are month of you just free last you’ve one patients can one this XX consistent about helpful. the tell think drug?

rollover expecting So, terms, in kind just flow paid we it have is but steady drugs? us to out when a XX% so tell sense those just of that today, patients are you we XX%, quarter-over-quarter, a can XX%, of nets

Jon Stonehouse

than So, those happen are free Jon, more who happen as to at patients we of year of free paid patients third as It’s QX, get on wins. to with to payers. it’s new time move all starts over they drug and It’s to It’s drug of not expect government over going a to half about payer we as once. going product. a additional going happen the

over patients get are be QX, than So, It’s won’t but it that’s to we happen it’s to for opportunity excited in growth. more time - and half not something about we expect of that. going over another those it

Charlie Gayer

adds And new every big free drug that the source patients of or month. patients of Yes. his a the those team chunk

Jon Wolleben

Thanks and congrats again. the very helpful. color for That’s

Charlie Gayer

Jon. Thanks,

Operator

next of Your Jessica JPMorgan. comes from the question Fye line from

line is open. Your

your ask may question. You

Unidentified Analyst

Hi this for Daniel taking questions. our for Thanks Jessica Fye. is

of of a the about from paid With the XX forward? sales paid new average representing should think we patient less receiving of to tailwind unpaid for drug within patients going now, conversion days

Jon Stonehouse

fact the from to two is And that Daniel, It’s That’s growth to with progress we something to – over patients gives to confidence. and but forward. lot two tailwind. going worried going product, the parts the it happen steady One a it’s part – something so contributing there. paid time tailwind, patients an revenue paid over that and a expect quickly coming is instant product what and that’s about really there. think, converting the and mentioned in not it we about patients months just to getting patients make is, doctors pleased I that then, progress I are free of that are they are so

Anthony Doyle

phenomenal Yes. of add many to record, job like also with want month I every don’t and function and a sound it’s how a done new we team Daniel, book And again, Charlie’s but that. do has patients

Unidentified Analyst

of can about about cash, that attempted you you given earlier talk think and have then, XQ you Okay. company cash through following in offering XXXX, funding forward? how into going And go anything Great. terms runway the for the equity in

Anthony Doyle

Yes, I it. can take

we it’s are two think strong a driven think position. by I really primarily So, in cash parts. And I

and on in burn. sources through the So we we that numerous terms have access the the have, to a access cash net that the of a future coming get hand just And if to that deal, revenue ORLADEYO then capital. capital in from to cash how Athyrium the calculate of we in perspective

on So, when I gone other we the have multiple deals for that equity debt us think days reliant be are the are to proved and that we avenues and solely we raise. last royalty that the with available December

raise, a strong really are and cash feel in So, we like, we strong position sure capital. cost of we’ll I a make do make when if

the equity mean, to we attractive probably a surprisingly at QX. raise perspective. us source referenced You use for capital in of least this withdrew that in honestly, the point time I from

Unidentified Analyst

R&D comparison this Bill. frame for, hemoglobin to who and you deciliter? X.X a deciliter? then, are grams And per maybe XXXX, update six X.X With grams for update one patients, patients of that where Great. that new inhibitor plus in change per Maybe those question of can saw last CX if the elaborate in on change responders, we that you the

Bill Sheridan

question. the for Thanks Sure.

way they going update approached calculated. be to today's think the trials how is and are the the pivotal about endpoints to we So in

XX baseline are study, a has anemic the XX. for randomizing upon XX, inadequate and through CX take changed with despite CX both inhibitor the or responder from week continue to where to still endpoint patients stable visits FACIT-Fatigue, the will inhibitor at BCXXXXX, primary either specifically a measured the be who we week the So from dose looking

So XX, that's XX, XX. XX,

the the last average which so data whatever baseline. about difference the the primary to That's a was compared before, treatment visit that's the difference. way of just that, talked to was we've So

the illness, subjects that development of anemia the autoimmune obviously and we which on is like bottom here panel of affected with had happened we've you the that row. hemolytic hemoglobin, be difference events to – the that the some second in unrelated could in one of the described The middle the one in had a I an see figures couple

the they So, key are differences.

here Obviously, right just happen we active I arm. of the likely is are is there as reports this the is they arm complications. think around control of those what's on type the are the what on context in now. to see – important that There literature of cohort world

and changed to to inhibitors nothing show ability clear terms meaningful positivity So in that difference has of the compared our clinically CX enthusiasm and continuing to study. around in

Jon Stonehouse

that calculations you this is data that the thing Bill intent our study, right? – One showing And is pivotal pivotal to that's how would Yes. measured you in add, the we I'd based the if so succeed, did we if tell are whole the the on behind study. way did he was calculated why Daniel, we endpoints the

So, with high. incredibly in encouraging very pivotals patients, this is the to data probability of it's to six nine success see that and

Unidentified Analyst

Great. That makes sense. Congrats. you again. Thank

Jon Stonehouse

Thanks, Daniel.

Operator

line the question from Capital Abrahams RBC of next Your comes from Markets. Brian

Your open. is line

Unidentified Analyst

for is Brian. the for This question. progress thanks our good Steve Congrats and Hi, morning. on taking on

more the a about for galidesivir You program forward? in pipeline moving FOP earlier bit about in am but your I particular, the spoke Thanks. bit you if share curious plans a can

Jon Stonehouse

Yes.

with the We work to continue government.

it's dealing of be COVID. COVID important to look part You anytime with are away like know antivirals soon. an that going doesn't going

important. so, at moves the speed These programs an go treatment antiviral and And them. still the government that is

to funding at get program. where that to are are But continuing do that's so, and continuing And right advance to we now. we work

Unidentified Analyst

Thanks.

Jon Stonehouse

You're welcome.

Operator

comes question the of final Barclays. line from Your Wang from Gena

open. is line Your

You may your question. ask

Gena Wang

launch. for taking few I a ORLADEYO Thank you regarding have my questions.

that one newly the in less of the Wondering is, mentioned than flat. seems pricing your next patient your overall the is you first at then diagnosed how versus And number would that When are ex-U.S. we U.S.? guidance. newly in is question be, guidance, look The patients. And largely Is conservatism? my the regarding due would the XQ lastly, what be pool. to the that patients percentage XX% diagnosed patients it context

Jon Stonehouse

take don't I'll first you and third, then last. take and But the you I'll can the them take both why do second. the Charlie, the

Charlie Gayer

Got you. you. Got

Okay. Hi, Gena, thanks.

they newly as newly the therapy. if they don't that So, XX% that. newly they to appear diagnosed. be treated should know We far about specific as are less We than – us, newly are know with to be just all HAE I diagnosed,

patients that's therapy. some at those a it's coming expect least, relatively it's diagnosed, So market, -- treating but we that who the percentage. the are out, sidelines, U.S., of an are it's we small who think with every oral a the on their a In newly year, sitting there patients most or patients mature now and come are but forward relative, and actually

EUR GBP As is WACC. to price – UK the U.S., But is to launch far to price XXX,XXX. not final sense, give that it's our then That's price be our list year our a in, clearly. ex-U.S. you price launch at as price the set price Germany launch, the lower, in versus we going the XXX,XXX. get in to price, per compared US$XXX,XXX And our just

makes a And less so price U.S. to going but price, that give XX% opportunity sense. the to In or we general, it is the European will attractive the us. volume really you be see of

Jon Stonehouse

and can it's and holidays. fourth patient we affect holidays guidance, we with now. in accurately and visits until at really way. nice regard don't wouldn't There steady the shipments the we by guidance where give quarter, base. feel It's And exactly And feel guide Yes. know our to first that you is the you point told is – are growth of and number We we a growth a could we off this how we you, alike. prescribing and we

that as factoring And we're so, into this, well.

Gena Wang

just main terms will does driver growth am ex-U.S.? you versus see And in in be USs the you do XXXX, anticipate do I that of ex-U.S. like, growth? wondering, the mean how Okay. contribution

Jon Stonehouse

just from come that interactions, direct me to heat Face-to-face we've face-to-face Charlie up will patient-patient the going remind U.S. The of It's sales ex-U.S. those a will growth meetings just like additional what points from difference. that start is largely us. the coming are implement to going to talking to they aware you that. not about, help meeting to to other, Yes. talking be are to is we're to, No. let that SWITCH be at because we're doctors are those And docs And Helen as be meetings sharing will is inflection what the make and the that each to meetings, getting data than the well. there to each Charlie able talked big college conference go they to are launch The that other other they how about doctor doing we're from said hearing started is that. understand going going then really going even use not getting the that there XX-week on data about we'll

study. prescribing So double thus has another their last are prescribing of is that big forward. have the accurate in to the far. sales physicians going it Charlie's And future. all says research that on an the piece, going pretty going impact And he the of trajectory then just U.S. the And been to did market that are

as we will So, that well. contribute expect that

definitely is doubt there no continued So the that. growth in U.S., about

Gena Wang

Thank you.

Jon Stonehouse

welcome. are You

Operator

more concluding no Mr. are questions. phone Stonehouse, remarks? any There

Jon Stonehouse

Yes. Thank you.

nearly meeting. and just tomorrow. is the said, the New exciting and date PDUFA we our excited. this commercial incredibly heading us Orleans it's Charlie as So, And of months are to three true because approval XX first and so us, for team are medical our our face-to-face since Helen and is This

packed. are schedules Our

to session. with We are and we've oral posters. – an got meetings. really other sessions physicians got We've got looking forward We've

the first real we couldn't in excited booth And the major to time our a to be down And to first conference. so, face-to-face will heading have we it's be New hall. medical more exhibit Orleans actually

meeting, to So you you're and if would I encourage hello. stop by the at say

So great always, for and thank you a have in your interest company day. as our

Operator

call. conference today's concludes This

disconnect. now may You

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