BCRX - Earnings call transcript Q3 2019 | BioCryst Pharmaceuticals, Inc.

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Ladies and gentlemen, thank you for standing by and welcome to the BioCryst Third Quarter Corporate Update. 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] Please be advised that today's conference is being recorded. [Operator Instructions]

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

John Bluth

slides commercial our much, Sniecinski; Officer, welcome are Bill Today's Jinky accompanying Sheridan; to and very CEO, and our available leadership Thanks Skyler. Medical from Staab; Rosselli call. Charlie press Good Jon Stonehouse; CFO, releases Gayer Officer, me Tom Participating Megan with website. morning team. Chief and are our today on Dr. and Business Chief

questions. your answer we'll remarks, our Following

we to use slide forward-looking factors Before want two which BioCryst. statements risk to I your attention in begin, direct potential investment discusses of regarding our an and

As detailed the performance and/or future call regarding results contain on unaudited achievements. and company's forward-looking future information, conference as will statements forward-looking including well statements slide, the those financial today's as

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

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

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

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

Jon Stonehouse

joining John, us Thank all morning. you and you, thank this for

The willingness from are We a supports from clinical today; APeX-X patients the research. the XX-week and results strong share trials physician benefit have APeX-S support and use meaningful getting and with new new patients to it. clinical market important market very payer and information the you results from XXXX extensive to research patient

financial XXXX the States. we in provide financial Q&A. in and this NDA next I will any quarter, happy year the questions and in on JNDA United remain We our launch submit update be first take of quarter to to to MAA also will a and track our

release. can earnings third details our quarter in You press find financial today's

market clear. a opportunity. have in patients and with updated clinical the view attacks. Bill, share our of once-a-day XXXX having breakthrough treatment results few we data year you taking of will oral new more I our Over As are of very full a well that's therapy, today, very the review capsule mentioned, lot detail and on information one doing to are the

you We also market from the comprehensive with XX-week sharing ApeX-X. conducted today readout research the are profile we of the with

and in Our previously population the team standard estimated. has used HAE industry and a data U.S. quantified than larger methods

We also to conducted the of current research future landscape. the and understand view treatment

can having is ground challenging research sample a This insights. in our any to diseases. Fundamental be to robust rare

a in and large However, to physicians, understanding. our in patients detail. review Charlie adds the able very we sample gather results of to which confidence were the market will

data with physician current are to their prescribe. summary, in The satisfied therapy willing No want to consistent is more. and very their the with their use very while But patient data XXXX. matter is, therapy, they patients are many supports what current expectation

payer a willingness to the value supports from significant this research together, reimburse. adds Lastly, to All up XXXX. of payers

over I'd over the clinical data. that go With to new the call Bill to intro, like turn to

Bill Sheridan

very Thanks Jon. much

and safety attacks in discontinuations. sustained efficacy XXXX. of delivers and trials a XX continues a clinical of safe describe We weeks of and treatment be new of the tolerated that rate time, well important analysis gained to into prevention and rapid The low HAE with have insights through drug over

progress elements let each weeks. of how through and XX me you First, design to patients trial walk describe the key

or three, placebo. HAE XXX doses trial on in the of XXXX one with we to slide As or noted two randomized ApeX-X patients

Part X. And the the randomized milligrams or had XXXX X Part XXX in to May, on one two all who reported milligrams completed were doses. we patients part into continued part in trial. XXX Placebo patients continued patients one two received the in of who As of XXX same dose then

of that We XX% milligrams have the the XX or randomized the pleased patients weeks or to study are start completed XX% of XXX see at treatment. of to XX%

the trial dose, The shown on XXX-milligram APeX-S dose. XXX X on open-label XXX-milligram Slide on enrolled XXX XXX and patients, the

trial, their that was if physician's HAE assessment oral prophylactic benefit could from patients were this For eligible treatment. they

XX analysis XX is reached NDA of through at XXX-milligram subjects the XXX-milligram estimate The The not dosing. on XX and had XX had weeks dose the weeks XX%. XX attention completed yet weeks dose for

Clearly, Slide to X.X month rates to rate turn to XXX are had immediately treated through with per The X.X respond through the month attack continuing attack are efficacy displayed let's Now, entire month completers XX. patients for to and weeks. months. baseline the milligrams results one a by on XXXX of XX which declined in to X.X populations XX X. ApeX-X the Patients

analysis placebo the XXX-milligram addition, X. dose. These showed Part patients after X.XX XXXX with results who XX In dropping to part two the to for randomized at rates support effect a from approximately month attack were strong treatment results month primary per weeks XX strongly in crossover

month our show efficacy reduced less efficacy sustained rates outcome for clinical medicine. outstanding entire XX-week approximately an one Overall, Patients This the or significant results a is to month three months. that ApeX-X experiencing drug. through of oral per baseline per with us attack attack these were patients' are benefits a from from once-daily XX

similar attack on analysis In is X, rates XXX-milligram pattern of The the Slide APeX-X. APeX-S story. a tells for mean APeX-S trial shown patients strikingly to in the same completers the

attacks When six had and for we experts results on month in showed they X the APeX-S, shown about HAE including more Slide our the these XX In half median in rates studies. are also months to than both of patients XX. attack asked X leading of

in picture safety, daily patient integrated studies received HAE representing XX of on X. an all follow-up. a analysis with summarized dosing Slide patients and years included up weeks provide In XXXX, total, exposure from data XXX was of safety oral to of both To XXX full

X.X% and safety XXXX sequelae. adverse resolved or safe patients generally events after and interrupting was no no were Drug-related stopping in were new occurred or There and findings. three well-tolerated. uncommon BCXXXXX with serious

and events similar. more in the XXXX. adverse and The of Nausea rate for placebo was patients. also with gastrointestinal was interested diarrhea fairly GI and of placebo slide. common other types on pain overall We tabulated more of incidence with these fairly are of were abdominal common was XXXX events pattern same and AEs the The similar GI were

Generally, month. GI these with in events the did decline medication, in mild and only brief, treatment discontinuation were occurred not to of in need Both XXXX early after rapid of incidents first led patients. and X% events

In transient enzyme affect to several previous we without are which sequelae of treatment androgens liver. patients noted known with all with elevations the prior HAE whom well had in trials, the

in detail in that analyzed we So, APeX-S.

in starting were Slide prophylactic of treatment X. X, times labs Slide overall had normal. on of three current shown continued. These remain was passed than drug. androgens symptoms whether analysis up interrupted is antigen to and XX associated treatment XXX could subjects this on androgen trial And dosing seven and or ALT the two-thirds The of before our on noted stopped, days up X.X% subjects limit XXXX with or As resolved greater with a had lab not or result not patients

one month both of ALT compared nine and of a androgens limit never prior androgens. within patients who discontinued than XXX only than patients entry more prior to one month who study discontinued in who seen XX of XX to of used entry XX study to normal one patients of androgens was zero groups, had had Over ALT The recent stopped interesting seven increased across days very there to to prior subjects androgen two level Summing high clear-cut with times ALT three more those had study. is with finding the use. androgen association that is up

patients Even to though beginning washout period the and studying findings elevations test were our the limited order have experience patients, best XXXX. lab in for androgen will drug, a we in to temporary for current advise asymptomatic results, before one-month

that relevant U.S. will market androgen is Indeed rigorous androgens. States, United treatment XXX less U.S. the the patients outside currently three be much than of the more in Charlie will as in only other were advice describe, taking This common research, countries. in

So compelling benefit safe of evidence, patients. generally with have that the is well-tolerated and drug we a this demonstrating of profile significant for body a

on As XX milligrams NDA earlier, XXX I weeks. we our through daily patients XXX at threshold of crossed noted least

We're of the on the it NDA FDA to to the compiling in now and this submit final stages track quarter.

moving the for exciting. delivering and are an therapy in patients very a major advance in on HAE close With we NDA these is hand to that results and way, very

like with you. the evidence market to call turn share our I'd over research Now new to Charlie to

Charlie Gayer

Bill. Thanks,

HAE We BCXXXXX, a for willing in-depth qualitative significant and it have since receiving to completed will clear. results prescribe and XXXX, want in opportunity of are use XX-week patients market amount quantitative message reimburse. research payers resoundingly to the is understand Apex-X May. The physicians the to

addressable the critical study I population the size in XX, start I'll step market recently an our was completed. the of Confirming a the review key but States findings by the Slide in on describing assessment. prevalence of research market we that HAE will United first HAE

U.S. relied estimates on conducted prevalence Historical epidemiological outside the studies

administrative used medications. specific Symphony Our methodology and from U.S. tests codes, comprehensive proprietary claims medical applied specific laboratory and unique claims recurring patients to HAE HAE study for with identify a Health data diagnosis Solutions

being HAE-specific population demographic U.S. factors. diagnosed XXXXX with informs extrapolated XXXX treated medications. U.S. for then a HAE database The from and with the normalizing We total the to study patients prevalence population approximately of

HAE a us Slide evidence the studies XX XXX and patients ApeX-X. as base HAE. the well evaluate physicians very market treat XX-week and to of quantitative from current that describes data physician provided to market we XXX reactions conducted as large primary studies patient dynamics who from These with

more The which patients U.S. doctors patients HAE XXX over or have average treating physicians patients X.X XX% XXXX perspectives per the HAE -- means year, who manage an we treating of of from HAE in all reported than

on to of We their XXX patients XX with was XX-point physicians and study. ask Slide XXXX an willingness profile Likert in shared shows about treating use patients blinded those the that HAE scale. XXXX the

Slide are predictor scores, future strong willing their product physician behavior. it. top very very a to XX% to recommends measured XX% patients use of as that use which XXXX, if willing three by XX are the of is Furthermore, shows box

those infused We current on shows further with box therapies. of XX analyzed survey, or or top by our are XX HAEGARDA looking injectable again willing at XXX reported BCXXXXX. XX% using the as XX% XX% that measured Slide three of reported very scale. XX-point who scores and injectable patients patients CINRYZE, reported they very an use patients infused their satisfied responses of prophylaxis to of being even therapy HAE TAKHZYRO in

columns use who current the patients their of XXXX. are in also being light very the willing green see injectable You reported with therapies that very can satisfied half to

Turning XX. slide to

reported attacks; to HAEGARDA You and and was the can TAKHZYRO and attacks; is X.X attacks perfect among have The therapies. no drug on injectable number of continue past patients over breakthrough X.X CINRYZE attacks. attacks mean that prophylaxis patients months see X.X three infused

quantitative repeated their our experience that that products, highlight data you lives. will and we of for the and HAE demand an HAE patients over sticks. to that of advances are as storing are directly repeatedly effective coping having patients discomfort shared add eager And are past in lives, APeX-S as XXXX. show of the of refrigeration, During The But research, correlates heard ease and help planning therapies APeX-X grateful with Bill to patients injectable with a tired This the their the very infused are transporting oral make prophylaxis our data use with part strong earlier. require clinical therapy as that and and smaller needle the insights and the injections also qualitative burdens therapies qualitative patients such preparing decade. in these outstanding

patients. current physicians Today, distribution patient patients. with slide HAE-treating aligned XXX our indicated the data they the XX% used prophylaxis reported are of of within using treatments The on report therapies shows base. on their Each their XX in of that study physicians

reallocate current physicians them to over of treatments the a as in to patients XX% current XXXX their where to prophylaxis use their blinded with therapy patients as patients their increasing we available. anticipate overall ask They expect for profile, product of treat XXXX After data prophylactic the show is switching future XXXX. to patients. showing state of well The their physicians XX%

medical priced the expressed oral prophylactic of XXX lives. broad data a options. current directors willingness interviews from over is demand an blinded on million the and understood physician-prescribing BCXXXXX infused U.S. line They with it injectable HAE conducted value prophylactic therapy with to a payers pharmacy treatment intent. aligned that market cover of pay our basket for We and and for and PBMs patient if with profile in and to XXXX, reactions of also Payer

meeting U.S. with will MSL ahead, competitively HAE our Looking out are now deployed sized is using the marketing we sales the treaters. marketing, of a build and understanding the successful HAE team that detailed – lead access a launch. market to initiatives top-tier market to recently We critical that of

records head months, of as diseases, well in hired a whom of U.S. of sales Over including the team success we commercial have as past outstanding HAE. General Manager U.S. the ultra-rare both our several of

progress services best-in-class. We will patient developing for also believe a be we strategy in made that substantial have and BCXXXXX hub

I'd now, call like And to the over Jon. back to turn

Jon Stonehouse

Thank you, Charlie.

With of presented and patients population market HAE understanding an $XXX understand the oral size the the sales. global once-a-day physicians the preferences Bill peak research XXXX least see U.S. we evidence-based from additional in future million treatment clinical data and current to generating of at robust and

after are of $XXX have goal for investing I've To mentioned by previously to fully launch ensure, XXXX it patients add We accessible we million launch, our approval. the approximately in balance the year-end. soon the additional make an a sheet to to resources to successful

of pursuing for different a this capital. and evaluating We are sources number

an with access from additional million option to debt have We midcap. current $XX agreement the the

exploring role. Yesterday payment, a play $XX of equity announced Japanese a which financing year. Torii to add are is we deal also we and a a $XXX this with We will million plan million opportunities component we licensing royalty expect with upfront the

say partner and about Now, the excited to to I'd opportunity like in why a few about a call as so the we're Torii Japan. words Megan over to turn

Megan Sniecinski

Thanks Jon.

treatments tremendous Our today. partnership step patients unmet of high need for lack represents given a approved the with Torii prophy in Japan, market and the in HAE forward

After for several a we running process, selected Torii competitive reasons.

an medical increasing of engagement, HAE broad to bring well U.S. their with biotech. in in disease existing patient Japan launching positions their with in franchises. awareness education representatives successfully have oral products to that grew established to fits investing treatment and a partnering KOL patient prophy XXX Torii track complement combined patients first the proven experience license of BCXXXXX This and HIV from that in with Japan. groups. their strategy record First, They reach business They their products in-license by growth successfully once-daily

in As pricing negotiation. milestone a Jon $XX of $XX and there's million, mentioned addition approval a there's on timing payment. our to depending million And up approval PMDA of future upfront the of outcome

We to sales of royalty the level will XX% and payments, our Sakigake also receive from tiered mid-teens net depending status. the on potentially ranging

quarter in Japanese motivated of next with With remain approval submit on to track next potential our half second experienced We partner highly as combination accelerate a treatment an we of to important our partnership Japan, XXXX see to launch year. an NDA in with treatment the the for today options Torii in access Japan. this of to lack patient support PMDA exciting HAE's and successful and

turn now back to me let that, it With Jon.

Jon Stonehouse

Thanks Megan.

Let me this. with wrap-up

consistent APeX-S. long-term availability who Patients, see We data attacks. sustained from as the weeks HAE therapy are and quickly and significant start APeX-X evidenced this XX% XXXX effect have through both of and sustained by XX stay of despite the their reductions other in powerful completing on on clinical Patients on and having They weeks. XX treatment therapy, options. of patients are

understand accurately data We've XXXX. more to value market tremendous and amount a work of also the the important of gathered done and

the HAE population. a patient methodology have size utilized We to U.S. robust

and gathered current We our comprehensive insights through also critical treatment future have research. choices market into

than and capsule want the us willing to their therapy treatments prescribe patient patients one Because physicians very when of XXXX and regardless even use thing. and Patients very injectables. data more therapy. with expecting have Both satisfied injectable once-a-day a they're Why? are physician same tell are current oral we to current

disease for. many what life And with every or believe a They time reminded they've medicine. want in they their refrigerator they're we want their a been They see XXXX look of needle themselves where is not and for waiting their stick more.

significant XXXX. We exceeding sales With hand, $XXX peak, see in potential with see million. global this value information we

of to you now through very to plan; first U.S. in about year-end all we at starting with we successful the it's available year, times executing execute continue preparation look quarter and continuing plan. are next by and our the have These progress our this of plan. So regulatory capital year, sharing us to for submissions forward resource options more different properly and next exciting a the now we launch for BioCryst as in the accessing our

open for up we'll operator, that With questions. it

Operator

first Our Instructions] Fye JPMorgan. Jessica comes [Operator question from with

line now Your open. is

Jessica Fye

Good my data the A Thanks for questions. couple guys. taking Hey, morning. morning. about this updates

patients elaborate discontinued didn't discontinue four for who the you like studies, for? what who events can side I -- First, these discontinuing on were think, adverse they

Bill Sheridan

Thanks There of for reasons Jessica. question. treatment. other the choosing other for Bill. Hi, have example, variety a are some including of whole discontinuation, type and inter-current for illnesses, people to

as I on was due So to the call rate events adverse very the discontinuation large. mentioned overall

Jessica Fye

under are captured other. talk of Okay. I about that were other of reasons some events, Can bucket were? few of for And the kind a on adverse what there a the are that think you discontinuations

Bill Sheridan

example formed instance. a pattern case. events None in having rose one of for of one Depression to than more adverse the that discontinuations

pattern there. a just So

Jessica Fye

four-week you deal Okay. basis But with do versus for something clear. that prospectively? washout see And to other the study respect want would then picking for the any to And FDA androgens, the looks some that like pretty possibility four timeframe? you what's weeks the

Bill Sheridan

The answer to is, the no. last question

the nobody's reasonable finding that out a timeframe one but very stop that seems clear. the function length androgens. time of going test. is we stop XX it the like think Four think it's weeks when of to with I a interesting in I And all, people the based that information measure patient data a because had on Typically androgens. crystal

washout patients labels the time of X% above rate normal limit it days or out So actually. in a than XX And bit was randomized the the XX. a this placebo-controlled trial that our look was quite it's of been ALT coming course so. actually. the upper in one all randomized about that longer In had product study, of And environment is is when trial in you first detected the of other placebo-controlled in times X

Jon Stonehouse

when you Bill to you're other referring drugs HAE. in other And mean drugs

Bill Sheridan

Correct. Yes.

past. that's landscape So HAE of on to androgens been have part the the of in treatment of

And still our decided taking coming them and some of studies were stop. into the patients to

how we've So identified manage I to it easy. think, basically it's now quite and

then You little and bit just and a androgens wait drug. our take the start

Jessica Fye

a And couple have for Okay. I on Got side it. financial the maybe Jon.

monetization extended kind You look what outline sort something cash. that to on mentioned be would of or Any in could, priorities the like think strokes end that? broad of related transaction in you Should more chance a into this think And of, the to potential would your like to as like what the a within from means materializing it's on bring bring frame? you I the terms of the cash royalty month. additional I we MidCap time think tranche to that the that is Thank you. deadline the about company plans

Jon Stonehouse

Yes.

not the go I'm get So going potential into we until any to of done. financing details it

So any the can't sorry, give royalty. detail more you I on

we I an was think -- think have the for we of us million. main as $XXX with And to part MidCap. have flexibility and options one the that have I to get option important to MidCap from a have choose we point is the renegotiation to different

Jessica Fye

Okay. Thank you.

Jon Stonehouse

Yes.

Operator

Liisa next from JMP Our with question comes Bayko Securities.

line open. now Your is

Liisa Bayko

data in on questions. question the people taking to was really are differences interesting previously on for the you androgens Thanks lot and response But is comes rates see PX inhibitors? I with the any just versus that who Hi. nibble did curious and to into. dig a mind

Bill Sheridan

significant we and May Liisa. APeX-X things of randomized disease Hi, were factors We model. analyzed that demographic covariance the in final analysis the reported patient as trial none Yeah. controlled in in really baseline and those

is one at. the of androgens we things So looked

Liisa Bayko

the am going back I Thanks. hop in Okay. to queue.

Operator

question with Jefferies. next Raycroft comes from Maury Our

now is Your line open.

Maury Raycroft

month one month to less the attacks I my progress. wondering per have the the And baseline that patients attacks these attack mg that XXX having severe? would updated Hi, per patients residual on are month questions. Congrats decreased XX term? the and And therapy had do stay patients longer a for on sense at you then at X.X these the the for – everyone. taking and was for XX thanks data

Bill Sheridan

attack. which think From So they question. daily report their great have a that's on – if analyze box I we is all an they the yeah, data check form did can

with doesn't tell full the patients sites that really that investigators on we I they're the study still mean, who us story. talk have transformed, has and telling attack can physician the occur do more at things So uniformly manage when breakthrough life the have manage I drug the that my life I like that my the changed this attacks can patients easily. – said my

there other I that the because is with time, of story will stress. less follow manage interesting all that helps disease. their like the be think treatment that stay the prophylactic fear on to over longer drugs And people and less

Jon Stonehouse

last Maury attack, And they study. and a was some big one treating were some they occasional even couple of I that attacks. have with was they're not they the – said our Yeah. the severity breakthrough week talking KOLs of on have they of about these I patients in cases but

– got So that's are these of Bill where life-changing. quote the and

only that don't we patients the things real the So from study certain it's our data feedback hard manageable. there's the have because getting we're But from check.

Maury Raycroft

And Got for for And responder some your the data feed of to can into from that info helpful. then if analysis? switching it. use and discontinuations with strategy That's discontinuations you're you XXXX? your seeing wondering just align

Bill Sheridan

That's an interesting question.

physicians be to know Make you the and it. drug that drugs. data they're drug. we've think done XX-week to is got good. main feedback last that all and and people here insurance. Yes. that, from That's months the they're Side out that few hub the boards get to had we've feedback. They you've another. an the the us appear cover why put how across I'm that the they our sure benefiting. know in oral medical The advisory after United getting the thing right how this of the put to don't on staying use switching efficacy that people It's new And one issue manage from know to we on because is I on are benefiting tell people an the of people effects looks not got States the drug. data oral how And drugs we

Jon Stonehouse

is at can over strategy I What Jinky the regard patients first jump really X,XXX right, said switching and of size patients sample guys use XXX these willing population. very they're caught where a large satisfied, injectable a drug, us the doesn't HAE data but you the very XX% HAE therapy physicians to And are telling that to And drug. if in U.S. our different it. you they Charlie cover the and the try patient If shot but I'll breakdown and very drugs know the you with patients What he us being right? willing and data in, on from. it are it saw don't Yeah. matter tell different the take of come in to and Charlie? That's they're still our

patients data to therapy. are willing very the So use our says

piece doing other that's And the well by therapy. you they're XX the we're see on rate of patients that's patients on right? for you go is when on information, sharing and that XX% therapy, they're from staying why with really they attack therapy, on stay therapy evidenced reductions today So And of staying the weeks. can

two, of on don't know combination very I their you people add. And our and believe shaking guys on. anything I strategy. the is switching So if head have getting that's no. people They're the keeping to strong for

Maury Raycroft

where were guys question or data weakness last and just different payer based if a on focused on can could strategies your strengths And outline that few is out. points? play pricing the payers for you And Okay. scenarios

Jon Stonehouse

Yeah. Charlie and in. I'll start jump then can

not of to we're get going obviously, pricing. into for detail competitive the reasons, So

you I research we payers And this some patients oral with more and if market Charlie, a any the think color market the profile add physicians the with isn't and through once-a-day know that fantastic payer with research to new this therapy. data have both that the we is I research. don't necessary this got idea discount of with to steep want information

Charlie Gayer

Yes.

X,XXX Maury, research they to price the they with the We into of that did of show therapies. the prices acceptable think the of would first asked the I we just said payer that payers infused range ask the the and scenarios. before thing is current be and find them to pricing is I them found getting blinded prophylaxis acceptable and injectable they is other even thing around add be range what profile

they to therapy. for oral think would very product understand prophylactic that they is reimburse willing They and going to patients be of for the useful an value it. a be So this

Jon Stonehouse

And the charged me a tipping and got right premium. we cross if kind I point of this where we had is if tell Charlie

Charlie Gayer

for That's for right. pay but patients. extra important going why not it's convenience, they the understand to They're

Maury Raycroft

Okay. Thank you taking my for question.

Jon Stonehouse

You're welcome.

Operator

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

Your line now open. is

Serge Belanger

Hi, of questions for A good morning. couple me.

be recommendation? be filing? And do First, can in ALT and androgen association us to doses reflected think seeking the dosing the a NDA then expect label? upcoming warning remind You if FDA just could be of of approval elevations both in you XXXX the how you black the you'll a of it box discontinuation in or kind of

Bill Sheridan

your Serge, question. Hi, thanks for

expect that last We at absolutely question all. not. don't the on So

to is drug. prior androgen use days said least which the XX We will to on what at our I advise cease propose call, starting

Jon Stonehouse

Let point one me more to that add Bill.

our space think and other similar liver drug the we this a it's drugs rates I to other We is you see elevations think that have the in androgens. And is think don't piece the phenomenon from distinct enzyme can percentages. right?

don't this is So believe is XXXX. androgens. -- it's not unique this to We we think

Bill Sheridan

And It's Right. XX. that And the controlled in than space random was the of this trial about the that one another had it's from a their now you in the statistics this it look recently how phenomenon XX. out of XX,XXX. chance that our And we've of drug and taken the Like androgens. less in controlled identify do is find table that earlier -- contingency you've effect at prophylactic it slide, if if to another opportunity in trial exposure HAE three you all was with androgens. to patients one on you is will a mentioned randomized out I to randomized got

a compelling very evidence. So it's

that's that what With the any regard tells of evidence relationship should better we're to effects XXX-milligram market at dose to proposing us doses, the with of do. any adverse the efficacy XXX-milligram and we the the whatsoever and the dose dose lack of

submit what that's we'll but course the all day. of label XXX a We proposed So milligrams data of the submitting. is once be

Serge Belanger

the Can for just formulation one key ZENITH-X that then on And acute before III undertake to done Phase mentioned trial. the you milestones Okay. to the this comment tell acute program, be of you what HAE oral you additional upcoming program? work needs us

Jon Stonehouse

So are That's the and have understand current research A of sample around, we breakdown I interesting is people one data had size. saying not how prophylaxis. like very going from tell. -- really what And our robust the of findings just have things said acute of we to lot future a thrown market we numbers acute. been to and really move the

You the say physicians heard future XX% Charlie that prophylaxis. see

it's tiny when when acute you disease current small through that we people ask very percentage currently was patients their And there a segment. managing are on patients then of So the a at look treatment. on therapies

faster So data we robust data supports that. prophylaxis set set think thinks. this is Everything's a everybody probably that than going and great to

to the we regulators So in still work prophylaxis program, the out talking secondary things with We're formulation working with acute program to the regard the process across finish to on and through the the it's with path Phase of need III. forward getting for to the regard obviously still our line. we're

get that through we as let that work you we'll and know. through soon As

Serge Belanger

you. Thank

Jon Stonehouse

You're welcome.

Operator

Brian from question RBC Markets. next comes Our Capital Abrahams with

Your line is now open.

Brian Abrahams

on and for of safety. data sides. the for couple all and very guys. on detail clinical helpful Thanks the efficacy thanks And questions. for research much questions then A both Hey, one market taking my

those going in APeX-X, looks a Just per portion. rate six efficacy, in substantially from for on month more six versus the the placebo lot like placebo-controlled attack declines in second mgs to XXX of first it months the months the patients first

potential explanations for for just you have make differences completers? product just this. Is Are these it any the wondering different? a a I then, lack follow-ups. potentially Is potentially arm? a So responsive? if any couple of the any would this have selecting these I'm of more Are of And patients that patients there placebo

Bill Sheridan

question. Yes, Brian. Hi, Okay. good

what May. what we that see I in with here think I reported is consistency

dose. So had XXX-milligram statistically compared the dose was than Both in XXX-milligram that the to placebo. May, significant we reported improvements better

that seeing XXX to XXX placebo better milligrams is or Now milligrams XXX milligrams. we're milligrams than we're switching XXX and the

So it's consistent.

I in trial evidence of exactly based this market. think per control further XXX-milligram analysis. is see the on what to selection we expected dose XX-week that view, is randomized the our supporting And this

Jon Stonehouse

I number there's big the see Brian, important placebo striking. one. what's is you And effect, is Yes. data a think, really

these on so, per aren't month at starting more an they it, six down around if you go two month attacks and out And at eyeball three at patients a attack. it's half to They're placebo. --

starting. striking, see So it very here. be benefit we where But from may they're that the it's

Brian Abrahams

That Got sense. makes it.

point. I about completers guess, And product So attack it at can APeX-X, -- of guys the just reduction could also for data basis rolling be through weeks a versus intent-to-treat talk you on on XX for starting from over XX then, an placebo different the XXX on are weeks also who milligrams those analysis? XXX a and a rate of patients to on

Bill Sheridan

Of course, analysis. those have we

dropouts shown for we've about by completers time rate. intent-to-treat a data affect But a over over analysis every dramatic people analysis, with got time. we've analysis be decline in we've more think the every for That's month month the have why interpreting person the the studies, month decline the how got attack you bunch I careful data. ITT show to because show by And month. longer and a of

Brian Abrahams

on That's very last safety it. maybe And helpful. Got one side. then, question the

in to did the to Or ALT did androgens be For the patients Was dosing occur? tend who of recently, when course spike it was tend XXXX later? on? early were -- it that on

patients rates period? washout research, of your recommend the prophylaxis would just androgens who to treatment? And through actually sort plan Would XX-day are, the you then, Thanks. patients -- coming low. some market for those or are how But I alternative, pretty on-demand in on bridge those you realize be that, based who are would on of

Bill Sheridan

Sure.

really So interesting questions.

So week in detected drug in normal, case of actually at next it the three one week three XX our normal more upper two, started. almost before times visit two first who of the more had ALT limit or at the of than times has was at at the every the at baseline, limit study even And abnormality upper four. the than very was

extremely there United -- opening waiting our and androgens. it's a started protocol had So stopping this up people we initial looking earlier sites, androgens strongly, in And year when discontinuation temporarily States APeX-S the associated, before and XXXX. month recent prospectively with of at in set starting

the prospectively started. few absence elevation elevation today, other the enrolled, is who we've two mature. any of week drug we study because But within We a the in part we've have two not a in or in of least ALT androgens that that a first androgens of or in subjects haven't in getting presented week couple, at data – of who week a had seen stopping within

what's It's to clear language And And things, when You happens? simple what ask steroids. hypertrophy. in a here? And can are anabolic catabolism. stop things liver on So cells anabolic right? story. are get very going get going steroids you're grow you topped. They to like Androgens get going well you

before of would never have It's none at because just you? should this surprise So, be that in reality. looked a why people it

Brian Abrahams

Makes Bill much so sense. thanks Thanks and Jon.

Bill Sheridan

You’re welcome.

Operator

comes question Barclays. Gena from next Our Wang,

open. now is line Your

Gena Wang

taking Thank you for my questions.

how these? you The are first question just remind the wondering milligram, many can us pills if XXX

Bill Sheridan

How once many capsules? day. capsule One a

Gena Wang

of Okay. mentioned Good. think use I you to androgen the mechanisms, just androgen of liver will our we how causing And then enzyme users, know the lead much underlying also use for -- wondering will treatment safety? why that that the the

Bill Sheridan

I'll didn't to I And both. answering other quite the Sure. question, do but them finish try

liver even with alone extremely let you first and thing well-known the body androgens. been point people about chronic get also you carcinomas hepatitis have doses can steroids course in are of And the the can over and So not HAE abused those you use what out adenomas, building familiar for story therapeutic hepatocellular been features, at androgens the androgens, reported. type have to with hepatocellular of taking get liver who whole are or reported And anabolic they're doses, toxins. they've in you, toxins

And whole with hormones. there bunch mass effects of course of generalizing are associated a side other

extremely that acute So off androgen withdrawal, there's effects people well liver new androgens androgen withdrawal but The effect. get to are known. is here an with them, observation want

great. really that's so And

small. stop think I high a than more a is predictive value the the most data that month you more you important likelihood actually androgens takeaway of extremely The than month XX%. whole message is then will is out negative the wait and ALT get if

solve to way the problem. the that's So

Gena Wang

I see.

expect And on based price or Takhzyro? then versus Great. last comments, and Okay. just largely in the question we Haegarda should line regarding price your

Jon Stonehouse

pricing Again, of do is Yeah. tell profile a the prices. profile. going this attractive on lot don't to past specific not into that flexibility. get remains Gena, changed steep true we pricing discount have What with I'm the is What's we we've need That's payers the said us. to that based in in a what

Gena Wang

you. Okay. Great. Thank

Operator

Your comes Jaffray. with Buren Piper final question from Tyler Van

Your open. line is now

Tyler Van Buren

see a at it's quarter responders. about response natural of guess it placebo. a respond -- some thinking XX had out guys. I additional in the Hey, the It’s Brian XX% responders taking related of XX that you nicely XX% would the not relates XX% weeks to completers the great guess durability question surprised But to I of enrichment that response. patients to kind have the is them you'd half see to survey about But mentioned for have XX% to line and And about is see data my which of as weeks. the and after clearly

weeks, XX% can patients completing For reduction are in HAE XX the attacks? those you XX% proportion update a a of with or who at

Bill Sheridan

We do but could we analysis, certainly don't that certainly could. I it. have

consistency consistency compared main think start, is to we after response weeks I study randomization were drug -- the the were studies things to rate in attack between of over -- placebo the compared looking that think the in they XX for both right the here. after I time and at placebo

a from both look idea So very APeX-X. the completers in happy And with completing getting to weeks. the ITT of XX% XX% we're good responders in and coming XX population than the the at weeks. study the with XX patients very I'm XX% of response happy baseline more patients over into it's But

Jon Stonehouse

And right Yeah. your thing for rates studies. attack studies attack I the for median draw to eye other just both to weeks the and the XX mean both rates are think for then

well. it you get to I'm really trying -- You can get tell Really doing that a what people sense are can well. I you guess is

Tyler Van Buren

have proportion zero, attacks idea had Do of or... period what Yeah. the any in you patients second

Bill Sheridan

slide seven. done, shown that on we've analysis we've the Well

by that it's And month. so at median pretty just that attack studies. rate? similar two the looks you can What's the see across And

And six subjects we've month. half individual of in or in where XX months the APeX-S had attacks got of the that in zero XX more months,

in them the the look one all single of So fake, the that Charlie every are of data is attack notion breakthrough attacks. showed, free at and therapies sort of prophylactic there of approved

that's if a breakthrough of -- the going time, for to long the you just look because it's attacks, enough So nature you're get disease.

Tyler Van Buren

questions. taking thanks for my Great,

Bill Sheridan

welcome. You're

Operator

this remarks. at Stonehouse closing for the call like back And to any time, Mr. turn I'd to, over

Jon Stonehouse

Well.

thank So joining interest in you your us for today. again

the in XXXX said really I significant once-a-day these As with us HAE. are We see exciting times BioCryst. our with in prepared value attacks oral remarks, for for at preventing drug

presented go therapy no therapy. data There's The works. Patients doubt today the stay who on just on the drug works. drug

to ability keep right? And more, want high. our people going says to is research patients the So be market

what high. is to they're attacks their drug seeing oral a extremely with while prevent their they're -- satisfied disease They're their with of the once-a-day, injectable therapy ideas manage

peak significant adds to think per of And million so, $XXX year. that we that global up all value and of north sales

launch. where maximize we're the all for It's that, getting getting filings you us very fantastic about the put a And So, hard working about this value we now. ourselves execution count ready to can on it's position of in, asset. in

thanks great day. for So And a interest. your have again

Operator

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

disconnect. may You now

BioCryst Pharmaceuticals (BCRX) 6 Nov 192019 Q3 Earnings call transcript