BioCryst Pharmaceuticals (BCRX) 3 May 232023 Q1 Earnings call transcript

Company Profile

Participants

John Bluth	Investor Relations
Jon Stonehouse	Chief Executive Officer
Anthony Doyle	Chief Financial Officer
Charlie Gayer	Chief Commercial Officer
Helen Thackray	Chief R&D Officer
Allison Bratzel	Piper Sandler
Jon Wolleben	JMP Securities
Tazeen Ahmad	Bank of America
Stacy Ku	Cowen & Co.
Justin Kim	Oppenheimer & Co. Inc.
Serge Belanger	Needham & Company
Liisa Bayko	Evercore ISI
Brian Abrahams	RBC Capital Markets
Gena Wang	Barclays
Maury Raycroft	Jefferies, LLC

Call transcript

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Operator

Good morning and welcome to the BioCryst Q1 2023 Earnings Conference Call. [Operator Instructions] Please note this event is being recorded. I would now like to turn the conference to John Bluth at BioCryst. Please go ahead.

John Bluth

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

we your questions. remarks, answer will our Following

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

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

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

like to the now to over call I’d Stonehouse. turn Jon

Jon Stonehouse

very less the patients on launch the confident and the of of number Thanks, share of Charlie total growth when our you should understand now very details quarter-over-quarter, new guidance third strong patients ORLADEYO therapy you $X rates we this remain of peak. both and why year million adding John. year-over-year the will than year. no hear The remains and at entering and $XXX in billion

starts each the a ORLADEYO. is patients of just base patients many there yet reflect on therapy new and quarter large so to patient number upward The this as consistent trajectory. the steady beginning is And try more of

injectable an in therapy day a once a have We marketplace.

great their and it. more patients lives. tell And that us more many wanting are makes changed and to sense its try benefit see It

more in will ORLADEYO value. continue patients to reach Beyond this, create even we greater and to R&D invest

more program. expanding with the adding we access ORLADEYO gain to label around where will the doing the and countries by pediatric by world patients this like do We are

through ORLADEYO discovery find continue wisely efforts. also invest to our We pipeline and the will and

is to to another disease find inherent target, best-in-class able rare while medicine where the patients, development. goal risk we're a drug Our balancing to bring in

have and in we refinancing. position Anthony us sales. repayment Lastly, to This current team better but our closer improved strong time the debt much do by out got peak recent in and environment, to push where terms was should financial a our the to we the be hard already

This capital the to plan value That success. dramatically will markets where our allows value ORLADEYO today patients real dependence on in the reduces even on and another future to we ORLADEYO adding shareholders. the lead and execution growing us and and repeat focus finding molecule of our by for can the more

the call to Anthony. I'll over Now turn

Anthony Doyle

Thanks, great million, quarter Jon. $XX.X last QX compared year. a of of ORLADEYO QX with to XX% for was revenue

no growth the and underlying less of kind quarter, us mentioned overcome year be to headwinds to onwards than patient that slightly what and a Additionally, with of confidence million. full goal the a of reauthorization for we believe will to our us we move the revenue gives $XXX as lot forward prior just strong to see QX Jon below

at putting today's revenue Total quarter million, over few a in million at for You the ORLADEYO find attention came your earnings million. trailing financials $XX.X our which our detailed call XX-month and to $XXX in of first to like items. from release came can quarter I'd press $XX.X revenue

our be to should and strong quarterly QX similar see prior year. play XXXX we expect revenue out similarly to to We -- rhythm

to approach segmentation. revenue making change to our a We are

of coming of ex-U.S. sales from revenue $XX.X remaining the U.S To into million that the will non-ORLADEYO we came segment U.S million Moving global ORLADEYO for And our ORLADEYO be revenue. segmenting into $X.X forward, and revenue $XX.X will with end, ORLADEYO revenue we million from ex-U.S. and

global because of XX% expect for the the for at XX% ex-U.S year revenue while sales do the expect of gross for than is for QX quarter. of or ex-U.S total. we quarters percentage in represents to And revenues of in XXXX ex-U.S higher remaining the come the U.S. We approximately for to in this This headwinds global the QX net above

to $XX not to including down to XXXX expenses the ongoing the from to our quarter continue clinical were year noncash XXXX the is close of investment compensation the globally for year R&D. We in program. QX in as invest Operating reduced to stock and in million decrease OpEx which million prior QX full Most out of This launch of at maximizing ORLADEYO area compared 'XX. million, flat of following is a the reiterate R&D of continuing approximately and million. $X invest due guidance we termination $XXX and $XX

evidence we as three opportunities in continuing R&D, pediatric mainly key are identifying expand For label such the develop to generation. investing while the world by areas, in to also continuing invest ORLADEYO real to trial,

to D with to to if we discovery treatments differentiated and developing a and forward potential of disease. safe with the Our our effective And profile. goal invest development continues to we determine XXXXX lastly, get oral early continue program move best-in-class and selected a Factor dose rare can once-daily capabilities

the Cash at the at $XXX quarter million. end of was first

of closed pro we $XXX million. with Following bring month cash refinance last Pharmakon, to deal forma proceeds the that debt deal up that net

around parties some I competitive think very It that to deal extra done before. color any a have it's than we process. and that was more deal add with process helpful interested

driver a lender interest. belief such strong secure the launch to deal the to Pharmakon. for peak plans and of ORLADEYO of at of execution and our it strength The a that to the a to And great billion of the caliber $X date get in was our throughout was main

goals helped that we the number of deal had achieve of a us out advance to in set This process.

importantly, we'll XXXX, for bullet the to much point me, Firstly, which from XXXX payment and closer to enabled ORLADEYO delay most to at us be it sales.

in the our for prior Athyrium. loan margin of XX% that much to competitive expired to we're for interest preserves six on in the environment very which quarters, rates. a spread the pay agreement to tough significantly able in with cash as secure Then Next, option we were more first kind interest had reduced able

We also have $XXX to ours. funds decision of additional them the XXXX. to not September draw an draw and ability the is are million committed or These to up

we value flexibility funds only We so, it optionality. by the will additional can but continued doing if us and generate draw access to gives

it reliance in sales, billion current company equity. centered and Lastly, not additional capital the markets. do its capable on value discovery true balance a be prices, is a way in product equity development engine event third bringing At significantly the a to to financing and market third strong equity refinancing a the reduces around we peak with row behind be our or for not on drugs believe of our a markets to what reflect a to $X sheet. This it

for And best so use being is instruments able option us. to the non equity

revenues value to moving to this and allow we growing approach With the for financial for allocation, and continued strength closer disciplined ORLADEYO our us unlock to profitability, to our capital refinancing shareholders. us company greater all have the

And I will pass it over to Helen.

Helen Thackray

ORLADEYO have that once-daily The launch medicine succeeding oral in to a marketplace. differentiated patients best-in-class delivered Anthony. Thanks, is because the we is

the next more are products best-in-class disease. making commercialize standard set ORLADEYO patients develop and with with our both investments and of We deliver we the to treatment reach goal more is to highly market. for have is ORLADEYO molecule differentiated our rare This R&D to the to and

are HAE use studying of ORLADEYO before, under shared age As we've the the in we XX. of children with

be to the are under start building our communities. and excellent the to initial set initiated ORLADEYO And HAE. program patient with this in for for dosing dose in the to and trial. had pleased have medical pediatric we've data in for registration on support the in We pediatric an off children groups the Enrollment tremendous fact, in evaluation way we XX and excitement is

mediated differentiated opportunity be oral BCXXXXXX In Factor D our disease, has inhibitor best-in-class complement next molecule. once-daily our to an

therefore This and target inhibitor While both a address would patients. this target program succeed, includes substantial mediated investigating and Also, complement combined therapy diseases of currently terminal the we pathways, are the it CX complement. oral if unique system, bring is we our classical, to has optimization. single to lectin program lead by risk, still approaches pathways benefit alternative any in and with in of early the

so for best-in-class future system the in As complement well medicines for within system. the investing other we additional select as rare we can complement deliver we're diseases as targets look, the outside

candidates bring through target modes programs and risk discovery, early pipeline diseases. forward Our multiple an to variety a approach spreading and across of is of with therapy

an investments disciplined where offer We molecules structural are advantage early so biology discovery focusing just believe potent, the to R&D in these targets. best, Day believe on have we ORLADEYO. we meantime, do In to and bioavailable sharing we provides compounds we programs develop more capability next can do forward we year. best-in-class look detail later or We against done what efforts continue our difficult selective make additional based we an year the with differentiation, as at can this our

Now to the call I'll Charlie. turn over

Charlie Gayer

today continued updates, year. reached Trajectory the more this X recently on on Helen. I'll in patient U.S the provide $XXX no therapy significant Thanks, keeping will a QX active We years in I after surpassed X,XXX of track most just milestone. million revenue has us patients on in than launch less market. than the specific because for in metrics over ORLADEYO

QX. in up over those patients the by of over since up ORLADEYO. ORLADEYO sales. yet majority and confidence U.S strong taking end of XX% time seeing at ORLADEYO growth opportunity The gives and the of the X% of remaining of that front have we U.S steady even tried There's more X,XXX in most us of will reach patients number end try the us The QX peak the point was we're not patients same the XXXX because that

supports customer our confidence. Continued also demand

softer to expect off so first in reauthorizations. than were four up three the quarter year, starts team payer a We XX% of But focus U.S new QX new start prescriptions there's QX to year-over-year. greater because generally XXXX. in Our great be on starts this in much quarters got with the

pattern few coming XX% physicians, consistent. of What year and on come. top this than balanced again first all and from deciles, after more XXX that of consistent to patients through retained patients remained was many years starts each patient growth lose lose retention means we we about that. for with continuing their the Prescribing is And very see XX% treatment patients across quarter. new this we add We

temporarily hole down reauthorizations. was in moved larger slightly quarter QX obligations co-payment assistance QX product because of and Medicare patients are And revenue commercial many from patient free the expected, first year. to As during the always donut

therapy. reverse that I've second We're the assistance starting half pay XXXX. focus on commercially product, been crept up before goal to of QX who trend commented on on percentage our patient prior to we particularly exceeded free moving our increased And appeal had for program free long-term submissions. with patients patients high-quality product through this long-term authorization in of and the insured over

As great are access of you've steady revenues initial seeing modern are where we our prophylaxis pattern standard continue QX patients as seen, move ex-U.S the were consistent well The we to physicians in progressing majority growth of reporting similar the on Once Europe, care with in toward therapy the U.S. to launches are as international markets, sales time. the HAE. first in for patients ORLADEYO for from gains these in

peak we for expect revenue. are markets in strong will remain this of billion. the reach consistent ex-U.S globally, this XX% we for demand $X and that peak of And U.S and drive the We gives on Based slightly international will us about growth confident seeing confidence account early ORLADEYO will over that year. growth revenue sales markets XX%

Now, open up to for will Q&A. be happy we operator,

Operator

[Operator from Raymond Chris Our with Sandler. will come first Instructions] question Piper

go may ahead. You now

Allison Bratzel

Chris. is for on guys. Good Hi. for morning, Thanks Bratzel our This questions. taking Ally

in is should BCXXXXXX and was timelines the if this towards pipeline. missed curious [ph] the if what But you complete or year. clinic? visibility on work other So next tox just program? is on I the first, status chronic latest remarks, any this just Apologies the the think just had but the I prepared current on update steps underway

Jon Stonehouse

to you take that? want do Helen,

Helen Thackray

not There's at update Thanks the sure. question. much moment. for the Yes, to

as just preparing are trial with once-daily. that's a patients in dose We ability PNH to the initiate to and

we'll this on in more So regulatory we year. the through are process, working have and currently the later

Allison Bratzel

Great. us accurate line pace filing? And just time opportunity discussed sNDA then ORLADEYO date But development the think or there? I of the trial the And in about in I had remind pediatric we clinic that's 'XX. curious for path are should think you how just what's completion patients. primary on the you potential your just and expectations that mid there? for trials, if think enrollment what to Could

Helen Thackray

It's interest enrollment good the We've So said, there for and trial -- too as pace amongst that's of investigators. pediatric patients had our soon to we and the that. is timing and is, excitement enrolling. predict

as this have update as we we'll trial proceeds. So more

Jon Stonehouse

about and important injecting this unmet I a therapy. them it, think need. mean with But is really kids

a formulation apple we. parents patients sauce and on particular, excited so are or -- granules that this program have sprinkle We yogurt their you are so can about that's and in really and are

Allison Bratzel

thanks for Well, taking Great. my questions.

Jon Stonehouse

Welcome.

Operator

Our Fye next Jessica with question from JPMorgan. come will

You may ahead. now go

Unidentified Analyst

This good [indiscernible] Hey, morning. for Jay is Jess.

a So couple questions. we have of

percentage share update you XQ forms First, start guidance, the the beyond? reauthorization you you the drug is step And your regarding on it patients of Thank lastly, same think require [ph]? anything secondly, up year? there? provide in lab of expand can you is the us, the conviction percentage there can does there to similar And in of how of drug example, any elaborate regarding substantial on revenues that a required then can for year full And XQ on terms can And process then you. for patient us? the test, process reauthorization for imply right, should and or we about free the does that over remainder your of with free the kind for or

Jon Stonehouse

Charlie, you can ...

Charlie Gayer

third with [indiscernible]. start Anthony have first Yes, the questions and Jay will the two I cover Hi, one.

teens. So into on last down And XXs in we towards were patients XX% update, free up of free this we down drug, we can the I eventually the of long-term, mentioned, bring drug. into think trending percentage lower and the on our

remarks, prepared of game ahead the I So patients product quarter, free of are we from who as my patients the first paid. in insured in commercially terms on we're actually to converting long-term mentioned -- in over

So we're doing well on that.

little that afford drug. like external patients over that for drugs that a ORLADEYO for reason on of had their funding. are Medicare unanticipated short an total the the the was charities And in is free which bit quarter, We XX% of headwind ran help patients copayments we

who those therapy, patients And control. can been to to have And would so convert we something afford otherwise made they assistance paid that decision had -- insurance. copayment so because the their able some not on we've patients thus, to over that's we're patient

year ORLADEYO, means what to therapy. this on back over expect the these rest those product to of is patients but be for XXXX, patients on be doing we able convert free So that paid that and they're of we'll will in great many

that free we're So getting because of making right year little but team down percentage over XX% with drug, paid come a to overall. now, is the the over really on patients progress therapy we expect good people to

Jon Stonehouse

real quarter Yes. Charlie, a you just crisis to a patients these a don't this add, in imagine have of the medicine and they all first sudden, for I access anymore. senior mean, because was can

an Charlie result then or help something We in good program, like think, free some in are patients. Act, our investment step by and next because and to the very is quickly as our number of know, of $X,XXX, with again, company so care me drug And were XXXX. year this I Reduction we of a capped, that take that. many even But able got a co-pays switches inflation with as the you X,XXX of

Charlie Gayer

right. That’s

Jon Stonehouse

believe get we so patients. these population patients Medicare to best of going paid in we're back patients some that paid. to paid fact, And in our are of the And

long-term. So our one thought good it a smart we was that a for business and patients one was for a move

Anthony Doyle

patients point. Medicare would or there, co-pay issue this XX% had at this we [ph] free Jay be on So not under product of been a at this

for the re-auth It's Your just just yes new on second there's process, standard nothing reauthorization question patients. the there.

It but tests, new this stuff, history, sometimes standard there. consuming just it's a process. reviewing is always Sometimes time nothing lab it's confirming means clinical

of through base, to we a helping back temporarily lot product while why patient step across process. that's our them free that patient So were overall

Jon Stonehouse

that had Charlie, people quarter. we Quick a And the because switched we Start bigger to of as base, had more first result a in

Charlie Gayer

That's right. quarter a overall. busy And it was

of depressed the free impact So revenue. definitely product QX

maybe, Anthony, so pace? And over the to turning

Anthony Doyle

Yes.

[ph] for XXXX a play used curve as we'd Jay the expect [ph] out. think, surrogate I to how

will in to I a up to revenue in then step continue revenue would QX So be QX and in expect increase. there QX, and

year just three. by take remaining divided wouldn't the So I

step QX, from then I up steady think QX the see there I'll out. and up growth will a step and QX be healthy on in

Jon Stonehouse

-- And these very we're will be positive switched reauthorization patients is why the and is That's million. in paid these going that conviction that process quarter. our the $XXX fine around the to second

Operator

Our next Wolleben with question will come John Securities. from JMP

You may now ahead. go

Jon Wolleben

me. patients with the Hey. ORLADEYO Very congrats helpful number patients us A Thanks and looking the progress. could for go ORLADEYO think growth have the additional number have then in step couple all for of are the on do started Wondering today. throughout tell today? seeing what investment where if year of you color SG&A as to the that on you in to we to the total? And also, needs you're up spending, continue

Charlie Gayer

last Can Jon, I last. the Sorry, missed part question? the you repeat of your

Jon Wolleben

Yes.

additional are guys you investing where growth, that to spending are maintain you this If the capital? SG&A year

Charlie Gayer

Okay.

in the -- So the we that At we allergy that total, haven't over latest disclosed and of the prescriptions. November. promotion, number, it's Allergy in the the our but quarter had actually, in we use have Meeting we year, disclosed point, College last -- something fourth X,XXX on of --

a X,XXX with drop of talked year off this it's about We with and a retained patients after on therapy, consistent with patients. patients roughly very XX% what year And so we've at that. then at and point, few then

we quarter-after-quarter. growing why that's consistently so so And are

as add and Anthony I on as color. can SG&A, that And far can start

territories patient new side services. made to U.S. couple that regions, to into year, market just ORLADEYO in more on made team we've this a then particular, we supporting some So access before, help expansions and just and our of the with add team about commercially field investments the we've talked

and and our do and Italy investments. we countries that's like investments And reach X to and And something international are international Spain kind efficiency like out may expansion, make our that we to future. we'll of our continue ability customers Europe, make so in the just those incremental we of to course, then, improve Wave building and

Jon Stonehouse

and why exceeded that an what for about just go to we from free quarter. said you Anthony, patients you're to you in comments that goal biggest Can the we talk Charlie, -- confident? talk one bit the and you seeing paid we long-term our that's made. progress challenges little your drug about a Can Before switching investment we and is the that you of said you're have of

Charlie Gayer

Yes.

main I've this the health want. making about So really the provide that practices focusing insurance thing the in the the just sure talked information that patients is and on all care that companies

services companies, them sure And patient writing allowing of that we've helping got that put is making so expanded our team we've prior with the authorization then information ready and access on letters to to us insurance customers greater our focus making collected the and and that team, process. right the proper as appeals market the that's case the educating in a and to help there case or all

investments in the seeing is getting made the to as so people moving is year that the we we're on. that the improvement goes expect product in keep And to correlated people we've product, to paid directly there from free and better

Jon Stonehouse

Okay.

Anthony Doyle

in predominantly by the to I kind be to in last think is The in made -- like investment areas international to it's driven step the Charlie's I SG&A as at that. only mostly will just talked of we Charlie's by driven thing incremental Charlie increase moment, add investment Charlie about going that It the we'll that of the of anything and QX think be U.S. that larger here will But increase side have right? the high year, growth. significance and QX between see incremental, the Yes, be team major I'd comment made

Jon Wolleben

for if that Got pay one you I the on guys it. XX% Very more helpful. of the And interest Pharmakon may. Are facility? taking Anthony, to option the

Anthony Doyle

does it select to on with what terms It's the and of of would to kind the for as we we basis. something safe yes, take at Athyrium. But cash moment, it between do us start in I I'd cash short-term, assume X quarters I net the for likely. think of while opportunity of it to We quarter that say see preservation it's least at that have have likely did given it. think in a that advantage we take had revenue utilization it's per advantage we OpEx, converge the to highly

Jon Wolleben

Great. guys. Thanks again

Operator

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

may You ahead. now go

Tazeen Ahmad

Thanks my morning. Good for Hi. question. taking

ORLADEYO Just on stand wanted sales. dropout what like in Is great to you compliance need to in today? to billion target? a that more to achieve it drug maybe, about here have talked from You Jon, about of target? I initial about $X it also achieving your and through? in Or in, really a color have your order get of a leading What's guess, that rate of amount certain had just do matter a to is you order a peak. bit assessment, of where target little that going long-term view launch having from on a of certain achieve Thanks. lot patients We've to

Jon Stonehouse

Yes.

on think I rate comfortable pattern discontinuation getting think we're the the that stabilized. the really have piece, and I

And mean after to the remind from that the and first XX. to it you the pattern, months the got people make months few is and discontinuation X we really, so that just year, if discontinuation discontinue you. months I rate to drops a XX% X in X rate you And discontinue, low out of really months. the to

more now. So that almost has year, not changed what, even a maybe Charlie, for now,

feel think with I we so And comfortable that. very

So more then patients. adding it's about

almost And patient I a X,XXX and important of year, patients years we from last threshold that point we grew of is really think of a on couple XX%, that is therapy crossed remarkable which in launch. this

quarter-after-quarter adds just patient new of consistency is the and -- so And unbelievable.

There's very oral but everything competitive here. is a no there. marketplace. hanging an out low is have This down where injectable. we [indiscernible] fruit is drug the slowing else market, in there no So It's

a it's steady And so trajectory. upward

Charlie we that and team. The changes to one problem made that seeing drug with we had are the the paid free in change was

we the is fantastic confident a patients. our and to this therapy ability billion. for And to in patients molecule very for get $X think And so

you is So when of chances that, doing have your high. well

Operator

Ku will come next Stacy with Our Cowen. question from

go now may You ahead.

Stacy Ku

Hi. did a Thanks for have taking our few. questions. We

of kind can Is to ORLADEYO to first, So it of We these see retention a the XX just you you willing patients be how long-term where can XX%? the sense exactly beyond hoping some Are stabilization, program. very of clarify discussed off are to sticky very you've you close but for provide months? that might free there? for details that get be kind they on ORLADEYO, more toggled drug and since

So that's the first question.

The you second the growth for consensus is up. question number see guidance similar closer be million. be a terms is your in QX, it would around QX that Will from which even step expect or percentage exceed year, to that $XX quarter-over-quarter imply of could last

if curious going to what having around discussions like are treatment? there. Thank on or the planning Is nonclinical to pipeline FDA thoughts And the would you you having have be that color could duration like any major then get you end so, XXXXX? the Any you'd -- additional with And is covered higher it you. for the Helen, doses, if to question. any tox correct? timing are are So And around year for the signings sounds your be appreciated. factors update last XXXX. early that

Jon Stonehouse

Yes.

fallout is year. a post X% this our because me It's let been you've like for point X% year. drug people important to post if on that really start this get a a So XX% that want a because or sure year, there's I not make

bigger. say you, we why got that we've base if to you to so And a I and getting if that's point for -- people a keeps And bigger got that just bigger and we is so really remember. important year,

Charlie Gayer

of same the do today, math patients, at to you I just gave you kind going we we the can we that grew and make about XX%. the with numbers Jon, was point Yes. then add X,XXX are

are we So the XX-month what demand adding are that really is stable adding patients a XX because basis XXX plus months. on and retention every we consistently of

market are that in we in we see the demand signs so market. in see, forward-looking on all the that of And of market early we trends research, the that's the where are based stages we this still growing

confidence to retention And so going keep have the and demand pace on lot the we same the we $X of the a of towards are growth based billion. that

Anthony Doyle

Yes.

In were of give about we there still were of contracting We talked the PBMs last are in the in. out year. the figuring different the in Bear specific mind, We've on dynamics, QX, all throws getting not last going we some guidance. to how insurers of last that looked terms and at right? year, and

I last talked significant terms have of that Charlie think in about seen. growth the year we also,

within the of remarks, So wouldn't is the that we with in as the I of point QX expect at that in higher said and year. the or What a top confines to will grow on what was for metering was now than exceeding guidance I the it we'll percentage see significantly base it QX be I up, step QX for $XXX we assume last year. do as time. say expect million the in be and QX high what will to

Jon Stonehouse

And then Helen on timing.

Helen Thackray

question. Stacy, thanks for the

the and certainly something year discovery pipeline, on Day We'll talking or part year. our what's have in next we going in an R&D on on So think late about of color we'll on be pipeline early then. the more update the

XXXXX terms coming program In nonclinical in that discussions, are and of with and with we discussions this the program the program always that's any study from around so ongoing. regulators in it's

to so it. the complete for would that, we we conclude from study to And what understand need

discussions going to the study. regulatory PNH through process the regulatory The focus process the and our moment start at in is

we patients this interactions with that's of drug where the once-daily of can are the have regulatory be this the for we And with if in so assess goal we looking focus to of and effective data at what terms started, for but need set get safe dosing. to moving our into

Stacy Ku

much. very you Thank

Jon Stonehouse

welcome. You’re

Operator

& Our Company. from next Justin Oppenheimer with question will Kim come

ahead. go now may You

Justin Kim

$XXX Thanks for the just taking Good Maybe million guidance. on Hi. morning. questions. the

QX In terms seasonality that rely much the QX? of sort at who on free patients of how number, assumptions beyond least reaching of to the drug of conversion are of on

Charlie Gayer

Yes.

major part the that organic of growth Justin, our some into in patient So but of this assumptions, there's is just built base. our

it's to re-auths, in in the just constant the make through then then continued always We but we'll improvements demand first expect improvement. quarter paid to the the the improvement years. the gradual So therapy biggest people and make incremental over getting

to expect we into again, XXs But and percentage described. Jon the number that paid the difficulty]. constantly that year of the in part, as [technical new patients the base patients is then the biggest So drop growing coming --

Justin Kim

are epidemic, could Okay. patient of community coming any from they new volume provide the these low on versus you high whether prescriber, switch versus color low volume And adds, patients, background naive or prescribers? prophylaxis

Charlie Gayer

So really, the it's to very consistency. but sound great I'll here, repetitive see

still it's getting the So of that the we only. XXX are Those XX%, are prescribers we top half half XX% of the mostly switches, bigger about mix acute cover about prescriptions -- patients getting then were the the And marketplace. XX% in the I prophy mentioned from other are from patients treaters docs. -- the

is other much doctors, the from the And HAE so coming treaters. smaller so half but the not the community

have X in practice. their So the who patients ones X, X,

of to Our and reach segments those this some effectively both even our team increases more is able team with year.

expect we continue continue last this and And same those pattern couple so going of the we've over years, patterns forward. to seen

Jon Stonehouse

XXXX spread. Charlie be the every before, we great really that's due that tapped with summit the piece place other actually another said first think talking in into we market. times patient are U.S. into takes a and and the and COVID word of of opportunity mouth place I to a product booth haven't fully for hasn't mouth we that the patients on years excited. will important have occurs each word to time that's just bunch place X is And Yes. a the And it's spread, patient the this taken really where of but an in This are July, to and that

So forward are we that. looking to

Justin Kim

just Okay, great. Maybe a final one.

As rate might ex-U.S. just think you I'm the percentage a And peak basis numbers, for of expect sort we breaking reach potential curious, out about we that I'm the sort ex-U.S. sort to item of U.S.? on the wondering see should outpace so the U.S.? of line growth whether

Charlie Gayer

Yes. No.

going little still our the is come It's to of time, above. of the think a So market. ex-U.S. I But indefinite kind of an from is more period for U.S. growth bigger

now. As Anthony XX% about said, it's

pace to access in new little to it to getting those but slowly more launching get the launched At about doing Once -- in But for we what market places we and is growth. in going and are all are we've year. the just few XX% U.S. and But the we just in growing peak, as building kind steady dominate for expect markets. in sales particular, are the launched, more patients will be U.S. of We both same because adding a in in it's markets, in it's are we years. next XX%, Europe, over to evolve going steadily it the seeing is

Jon Stonehouse

it other just rolls Europe to peak, value dynamic even up it business how are performed expect it patient the in are has with a the Italy, if year the for haven't isn't is U.S. a to really to patients It's haven't of to X countries. of pleased X of those we takes to really still And XX%. inside we make Europe, we'll we But important. the Spain, that U.S. to And Portugal out But next start we Yes. -- slower. the big launch outside and the we gotten confident chunk example, get And Benelux, at in yet.

Justin Kim

questions. for Okay, great. Thanks taking the so much

Jon Stonehouse

welcome. You’re

Operator

Our Company. from & Belanger Needham will next with Serge come question

go You may now ahead.

Serge Belanger

morning. A couple I questions, Hi, guess, for good Charlie. of

drug to was trying in the of year-over-year in on larger one, the was than XX% difference XXXX? Just First increase impact first between the reconcile it quarter in patients free sales. the

from in a starting X% level you had at of the like in new a quarter Is starts had 'XX -- the of and in looks XX%. January. that uptick secondly, price increase I patients significant And quarters a you think first then XXXX? increase think And on the drug rest stronger prior that's a of sustainable for you patient in it XX% year? do

Charlie Gayer

Sure. Thanks, Serge.

was first greater question basis. free relative patient the XXXX, the in this quarter overall than on So the of first your quarter, size impact the considering free in product yes, in even product of the

consuming was for as is a two complicated reasons. our time it's just was process and the that And base -- One process. it's so always reauthorization bigger,

average free we were And little or just bit on as doing longer free so a drug, patients were the reauthorization. the on

So that's the factor. first

not around Medicare outside earlier access to The is as second able help the and much doing patients I being to what factor having their mentioned copayments not so. afford

did that new that was year. this last And so year something happened that not happen

together, Medicare expect year. And X% start growth We the XX%. at net for of the two those patient the took increase, growth would put have increase. price price our the you then if we price things So revenue had not the at increase happened, a comparable about the been the to

out expect improvement in We over to the doesn't of just That up QX. show X% a all about ASP. year course in to, at net the

QX as pleased providers And XX% as re-auth particularly were last yes, -- the it as mentioned, year really the to then the new busy. growth because, this we have care I in keeps health is over everyone, process far starts,

be our do is But team prescribe best to the I with a went -- going of necessarily as how many that. the started I'm And QX. are they much our we through will great we really the rest well and in so the I'm in the just but sign. it doing think is XX% fact that don't try means to members of pleased team how that QX year, expansions. they we with up new for

Serge Belanger

the just And trial. on -- pediatric one ongoing pediatric one clinical last

opportunity really ORLADEYO? the goal market label So is this IP an expansion with additional here for it's expand ORLADEYO's pediatric just to or

Jon Stonehouse

incredibly Yes. I mean IP. we have long

is in way an can injections instead, high So and sauce. having the therapy, on going parents their summits, sprinkles mean for on need. attend when piece back or that just old, unmet sprinkle X when something [indiscernible], to like I mean, apple opinion. is we years just It's kids a would our these not X, yogurt When I oral pediatric is get he think had you critical you patient to coming. I formulation pediatric about this the

there. important think an there's we life, it's demand for right? It's it's not a to huge but serve. patient And so market, a a And pent-up segment of market the

-- and all So need. unmet that unmet need really serving about this but was high high

Serge Belanger

you. Okay. Thank

Jon Stonehouse

welcome. You’re

Operator

Instructions] next Evercore will Bayko from with ISI. [Operator question Liisa come The

You may now go ahead.

Liisa Bayko

squeezing for me Thanks there. Hi, in.

-- net drug? from gross to was ex-free what of free on the the you drug, can Aside the rest

Anthony Doyle

Hi. Yes.

part always QX perspective is So, it of to be relates going the highest growth in it. to as from -- the reimbursed a

that for to is even and then assistance, year. XX% by we go you to the So by time we've the to in say get through right they'll driven of effect QX QX as XX% move to again. co-pay the XX%. as that continue year the less previously towards at to the lot I end that and of QX, the have the a XX% minimal better guided would top again, that reauthorizations, we and to of hit year things starting through But you're normalize get a range, you end then

Liisa Bayko

you're be helpful. trying -- next That's rest Okay. why And of on longer what the will [technical difficulty] facing no patients for Medicare free the year, changes? they issue understand I drug free guess be year on [indiscernible] these drug? to I'm

Jon Stonehouse

the future. sense her the co-pay some of versus in co-pay Give now

Anthony Doyle

for in Part be D depending the of way product is $XX,XXX to rare copayment like the ORLADEYO, right $XX,XXX. their on going now for Medicare a north off north So on of responsibility person's is based year plan, the disease

pay senior pocket. out a can that citizen, for few that very of to actually afford So

Act recognizes for So part these of by this not law, patients. outside to afford charities we, allowed because that. the manufacturers in, challenge do as are When this help it rolls them Inflation Reduction

a drugs then patient pay $X,XXX. $X,XXX in Part across is drugs. just And number that capped is So around individual XXXX, take, next year, the that the D they will maximum not all that at

down, And will that patients the that to it able mean patients be the more will And so they copayment may much help afford which it these be charities because of is assistance means able more provide lower. be to out means burden average what goes pocket. also, will overall outside

that's and why are IRA. the we optimistic so much supporters And very part of we of that are

Liisa Bayko

Okay.

in would then they on rest Because they why will the free be why so And drug year? that kick of piece, this won't Medicare for above?

Charlie Gayer

once That's on to keep on that [ph] the a rest them we just patient law we long-term of by assistance, for have calendar the patient year, put it.

all it's of so complicated of government part insurance. the And rules kind around

Liisa Bayko

percentage are of research like broadly, just what now [ph] then And the on patients is X,XXX tell more your helpful. market Like prophylaxis? That's what you?

Anthony Doyle

in U.S. … of and North XX% we the see

Liisa Bayko

Okay.

Liisa Bayko

… see we time. maybe XX%, to growing that over even XX% still

Jon Stonehouse

as comes or talking go And you're promoting that about one three why when more the going is XX% you've new it of is We out, will to you of prophy therapy. each for entrant got companies would somebody even to Yes. now reasons everybody that injectable prophylactic on put have companies. higher think

Liisa Bayko

the for changes inventory Any Okay. quarter?

Charlie Gayer

for is sale is patients. us every -- to issue No. direct because really And patient sale pharmacy. sole shipment the flash again, a source we an -- not of inventory Every is

inventory So we don't an have factor.

Liisa Bayko

Okay.

Anthony Doyle

with from of coverage dual the an plenty make up we Yes. Strong inventory down sure to are perspective, supply and channel supply. --

Liisa Bayko

compliance [ph] And these seeing are general rate? kind Okay. what you the of days? What's

Charlie Gayer

Yes.

the to too who job -- very high, once team So overall, make But compliance some smaller side take of best patients with But some day. they they’re to struggle of see compliance them services be on expansions that be percentage plus. educate their It's a XX% and described, that It's strong. of more. bit having always we are compliance pill do it's overall patient and part little one a help identify remains that going challenges sure patients will the plus. to help XX% their I

Liisa Bayko

Okay, one clarification great. the Phase you of in of the clear FDA kind PNH? stand? What's having you're then just a start conversation to study. on just Are Or now? And II point with PNH exactly

Jon Stonehouse

don't countries access again, So, looking the risk, where to where patients are have other therapies. we at benefit

therapies plenty of U.S. other for so to. -- to patients And access have the

give through the update outside the working we can't more looking are So and than that. U.S. process. we you are of an We

update. point, but this a goal Our start will announce we if an give do, we I we study, and when is and at to you do that can't

Liisa Bayko

Thank you.

Charlie Gayer

to want we PNH again why -- might clarify why there you first? Helen, And are going

Helen Thackray

the first diseases. renal fastest effective PNH the to a to are move path that we to able opportunity pursuing assess dosing. safe into then before and as understand we if have And the drug be to we that's once-daily drug can an achieve do So that

Liisa Bayko

Right.

you. Thank Okay.

Jon Stonehouse

You’re welcome.

Operator

Markets. Capital with Abrahams will question Brian RBC from next come Our

You may ahead. go now

Brian Abrahams

Good questions. me. taking morning. so for Thanks for Two Hi. my much

can as it the apply year are really potentially off, and here, through you year? able from more to high learnings retention that after First you've patients dropout gather initial this to around a reducing data and longitudinal take rate been more within the rate anything

more added there who are like likely and patients insurance challenged it them just prescribers. about launch, it where versus in earlier drug ORLADEYO going starting that for existing the regards guess therapy or at on And are is switchers consistent through with longer those this time those anything process Thanks. quite on being stay terms sounds in the coverage, region, free on paid point drug But or less them I make who versus naive then to I'm more coming to less of different from the in the or started curious, might of newer terms secondly, to therapy they're term?

Charlie Gayer

questions, Good Sure. Brian.

the surprising decade the first than but a a learning is -- biggest and in so patients more or isn't we patient collect ton many have learning more So therapies today And they data, on the long-term fortunately, I did and yes, mean really They've physician ago. think it's data. I of confidence biggest HAE, confidence. this about

So to is these to that they now working therapies access that know drug really get want for them. their they

be can And switching and so a trigger something switching attacks stressful. is that is stress for HAE

So thing the how to do is drug expectations biggest this what learned we've set of is is the works.

this really could that for that's you. months X happen, working drug to period breakthrough out to need give you a normal, to this attacks, but or side time, effects figure if things is are Here of X

that is just a improving stabilizing retention big just and that, and rate. our we of over and that's and educational message are we to that's keep repeating, time promotion So have that part our -- seeing

Jon Stonehouse

Charlie. add, would and just Yes, I

once the tolerating It's controlling capsule their a year? You asked retention disease, they're Because is day. Brian, so they're well. they're right. rate I -- question, why high taking after the one a they great, really and doing are it why mean

ideal almost so And the right. it's therapy,

why. that's So

Charlie Gayer

And with of breakthrough we doing When same the to they're kind attack other who injectable patients on rates rarely over are well, on they switch outcomes absolutely when learning low ORLADEYO, from data that attacks our therapies. are having ORLADEYO.

the just of And the I physicians understand of part so the has question Then is that types and your patients mix similar. education. of patients, making sure been that mentioned switching on

difference difference really getting the to been insurance. consistent. no think from patients -- there's I That's

into -- as been is on biggest are as more are other the market, describing. patients therapies we difference further They've approach. there these of we have [indiscernible] just wait-and-see taking a were are getting the who -- Probably been

mouth to before. need word we So of mouth, of educating and just keep And sharing mentioned data. Jon pushing driving and word

challenge is biggest product chance The getting happening, who's someone well And that doing but to on everyone. of we switching. we another see the haven't take gotten to

time, them convinced We will to product. we growth but and convince haven't what's steady over the that's everyone, of leading this

Brian Abrahams

you. Thank Great.

Jon Stonehouse

welcome. You’re

Operator

Our next question will Gena come from Barclays. Wang with

You may now go ahead.

Gena Wang

Thank you. quick questions. three I have

regarding new prescribers. first one is The the

from X XX% difficulty] from for from And said the and switch a patient. or acute XX% other [technical you and that also switcher, mentioned other parts. naive the XX% XX% Tier You from

apply this the That the guidance. was the question. on -- first to is currently patients My second that ORLADEYO? does X,XXX question So

million. Regarding $XXX guidance of

plan when any prior Now to beginning you initially that then this your is soon? the XX%, And lastly, profitability year? anytime guidance assumption expect with XX% achieve ex-U.S. of gave was consistent from you

Charlie Gayer

Sure. one over I first and turn I'll then can do the it to Anthony.

So two the the XX-XX -- XX-XX.

only that and big half lower to prophy half have So acute tier -- being patients. relates X,XXX switches the then half and of roughly XXX, prescribers top how half patients the be from the then

all it's X,XXX So of all very the consistent around. patients,

were whether a our or prescribed from came they or from So treater. similar, is HAE prescribed patients very prophy came KOL whether a were retention acute top by whether another they smaller they amongst by whether therapy only, they

consistent -- segments the it's year matter you we've the really with across how all consistent I has ex-U.S. far these -- really, it. it's no ex-U.S. board, -- -- million in plans. think with as the And our growing, $XXX So slice been this different of it's guidance, split. as consistent and

this we're point. splitting we see at XX%, that's it consistently above And why out so and it being

Anthony Doyle

same out of million it's now we line Yes. kind $XXX when guidance, And initially put in the with assumptions.

better the combination million to For then way than a billion. much are on your previously, revenue question $X been we and $XXX to So on our right? profitability, I in growth mean have we spot up of

I gave great I put that the guidance us last control signed OpEx in that flat we to to puts then month, for be think, with think, and we achieve that a the there. able we've deal spot place in Pharmakon And it.

of continues I not think level get our the that term on longer both profitability. speed to just there focus the be we

as invest the in we get we it's in to real-world when continue can in And in the and so then making make evidence there, significant we it ORLADEYO, sure just be. generation as that pipeline label,

are the on when to think be But I financial this achieve variables. best the great in depend to come profitability company financially a spot is it in. able we those will has been will position

Jon Stonehouse

the like And from? can about Where have in from on past. make And what and smart mean do to when capital compared dropped were. where markets execute we investments to now a And mean allows are we that next is before. our spot money worry different so we night oh, day it's the the growing compared to dramatically. and reliance we I I where it's strong getting we such us were revenue. has God, our not plan Yes, and is the rate? are

Gena Wang

you. Thank

Jon Stonehouse

welcome. You’re

Operator

Our question Maury come Jefferies. last Raycroft will with from

You may ahead. now go

Maury Raycroft

and about the what would can create some tree of you the million. potential going additional more draw down Hi. And about perspective provide pulling was to ways taking Thanks Can loan optionality your for you decision capital? $XXX my the another question. value to on new talk ask with I loan? trigger

Anthony Doyle

Sure.

September we So XXXX. until up have of

goods years, or of X.X give the take. So

obviously that period, have, to like pipeline. that We've time other the about we'll we during then XXXXX. we've talked that continue in. think there's we'll are think, invest and I to We'll talked investing areas also to as expect where I [technical we continue, previously difficulty] in revenue for clarity CX about to additional are

see if to in dynamics when and out we and play that it creating gives think constantly I we see us value, we those create value as we ORLADEYO. where are time get there, value would

will international, it, if could we clinical there's the to ourselves accelerate here if something there's it one that could growth, to was if value, or then accelerate another we whether that do trials generate in think use that or could area bridge to it's of to something the we profitability. U.S. So continue do to that's we that

value company. for another would opportunity I think be creation the that

as our option it's It's we So to fully at it. do committed funds. whether

I -- we and kind targets, And us. Maury, So if if those the gives will draw only is only flexibility the of for we some optionality us, huge think can that meet we funds. of it those

Jon Stonehouse

with that we are we trial that too Yes. There's getting you. early value. other at on one expanding there. and and And have And remember, too, of investing for we as share and can there's patients I said ways to those pipeline we label, the are ideas is that the things then But R&D that investing are in the are on real real just more and create therapy. a looking investment return ORLADEYO pediatric said, thing Anthony

at look So that's it. how we

Maury Raycroft

question. clarification quick one just Maybe sense. makes That it. Got

free on this quarter whether to around bit more X, Are cycles? first the re-auth same Charlie, first patients 'XX. specifics free the X product we versus the And change stay back going longer for the mentioned temporarily the they drug? should were of future X was a in it months little metrics expect quarter in on or calendar to For process. providing 'XX quarter on you you drug or

Charlie Gayer

in Yes. are segment use market, actually patients. plan through as commercial and patients the not just now there but just average, free -- of is product, ahead mentioned, which re-auth over was our on the the the those vast are our specifics, more patients we XX% providing No, of process majority I the of I'm of of

paid of patients the therapy on so the will And those be year. for rest

making to we'll keep So as we in therapy. then the through as just improvements people incremental getting and go paid year, --

now. demand and the the and see So better people based we really what -- doing in retention paid long-term with the we with just we patient better the have, are on year paid continued particularly getting and at base we on and that the therapy, then confident

Jon Stonehouse

have process, a have go of patients can to through that you longer. it take re-auth bigger the when Yes, base

year, it a year. longer take might base well, with next next right, So as bigger

… So

Maury Raycroft

it. Got

Okay. questions. taking Thanks for my

Jon Stonehouse

You’re welcome.

Charlie Gayer

Maury. Thanks,

Operator

the Thank as our well concludes for session This today's presentation. conference. you as attending question-and-answer

You may now disconnect.

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