BioCryst Pharmaceuticals (BCRX) 7 Nov 172017 Q3 Earnings call transcript

Company Profile

Participants

Tom Staab	Chief Financial Officer
Jon Stonehouse	Chief Executive Officer
Dr. Bill Sheridan	Chief Medical Officer
Lynne Powell	Chief Commercial Officer
Jessica Fye	JPMorgan
Charles Duncan	Piper Jaffray
Liisa Bayko	JMP Securities
Maury Raycroft	Jefferies
Brian Abrahams	RBC Capital Markets
Serge Belanger	Needham & Company
Tazeen Ahmad	Bank of America

Call transcript

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Operator

Good day, ladies and gentlemen, and welcome to the BioCryst Third Quarter 2017 Earnings Conference Call. At this time, all participant lines are in listen only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to Tom Staab, Chief Financial Officer. begin. may you Sir,

Tom Staab

Participating morning Bill and to Today's financial slides Lynne Chief Commercial our and Good today Chief with XXXX Jon Powell, Chief are press third Medical corporate accompanying Stonehouse, website. on available update results Officer; are the and you. call BioCryst's me Dr. welcome Executive quarter release on Sheridan, Thank Officer. conference and call. Officer

agenda agreed we for we financial remarks, answer questions. the have will discuss will provided oral market of two, some results. BCXXXXX will an pathway third the call, Following remarks, our formal discuss opportunity will details our bring HAE make the On your market, Jon and whereby Bill slide and Lynne therapy to quarter will significant to introductory discuss regulatory our I

Following our your formal answer will remarks, questions. we

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

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

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

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

Jon Stonehouse

regulatory results is full both approval our pivotal over of our front to opportunity started. us long-term joining have European determined this the and trial the approval. design and opportunity, our status, of of study ahead. competing are to for the last larger to to is FDA gives safety is Wrapping get These of up take and the thanks X The advantage and morning, is of in APeX-X regulatory we’re important Replicating Good just from APeX-X a us high. protocols design US plan the robust The XXXX and so in APeX-X, Orphan successfully single APeX-X finalize the completed this very trials Bill interactions BioCryst result being at full a and feedback major path Tom. minute. will longer. EMA FDA critical in now both similar in Drug update, efforts this significant We and design us recently it’s speed to probability confidence and is success executing also by market week place. as two of the Phase the the on granted Thanks, our today. and

and completion. execution HAE sales annual running up these on mentioned significant. the focused is opportunity trials I product driving totaled billion. getting to them and earlier We’re and Currently, US $X.X

on really research, inhibitor, in these at my normal one you when surveys our introduction. prophylactic convenient However, effective want they to To they switch something patients at couple will college an normal is and That therapy, is to concludes get to prophylactic number presented say medical it Once to turn like a CX therapy. the oral day fits look a that want. and conferences at the life profile of a ago, of patients in population. have patients total Bill. consistently I now prophylactic in A weeks over the meeting beautifully. them treated have only BCXXXXX prophylactic it’s about with What allergy life. they get public more they to want market of they forums, half the

Dr. Bill Sheridan

the move the to And the and controlled the with long-term optimize our can changes evaluate very regulatory our levels. terminology. Phase of and and we opportunity These Thanks, I’m very on the applications. study dosing were doses Phase finalized the useful, to efficacy is comparing program with change and to size. the of done to and placebo. those to into authorities. program with more safety FDA five. blinded After analysis X guidance X precedent subjects of mapping both note month, than one is APeX-X X interim subjects both on forward month, once normalized The a Subjects Phase salt all the proposal primary described record shown each regulatory the base results. and To milligrams that event. XX XX% milligram completed is details doses attacks. placebo to blinded rather inclusion XXX a a will [inaudible] European to the there is XX milligrams interaction milligrams. milligram of arms to reduction of the today. A screening weeks, current trial. same, period review to we be a with per daily at this as in and to the randomized safety as needed dose from labeled to recent have trial convention, to The in changes compound, on XXX Expanding Subjects once of the efficacy means to Angioedema with power completed and this attach attack with final will you very form a [inaudible] with can will milligrams was went detect tax the of of placebo XX% with either The two that slide The sample dose. content assumptions with per at the the no BCXXXXX duration size one in share the regulatory competitive BCXXXXX XXX per more ratio total group have XX XXX check now BCXXXXX productive Recently quick rate pleased or supports both Jon. confidence be broadens confirm with X the The weeks XX on for population analysis They is and Phase one-to-one-one time. weeks, will XXX for efficacy safety XX% a increase to [inaudible] key then endpoint standardized is sample way have least dose investigator-assist efficacy from The that of weeks study dosing of duration choose are XX XXX than for than [inaudible] we day. day salt for one to the

a with open in milligrams will in once the to when oral or receive can Conservatively, XXX to weeks. participated APeX-X The in with roll XX at each XXX trial XX both APeX-X subjects over the APeX-X life our a opportunity continue milligrams have XX subject in for dose study. endpoints trial day that per safety Those per needed so trial include Hereditary ratio. through The during period XXX subjects dose a level milligrams Secondary this trial a least APeX-X of a XX who over needed subjects will the the Angioedema safety long-term a weeks. long-term milligrams will Dosing XXX with at placebo the through in and to or safety roll safety And continue either study. and XX XX total in separate over directly is X in label into dose weeks be weeks. Phase per will continuing long-term quality that conducted expect the APeX-X randomized safety plan therapy those of Approximately level. study The XX randomized XXX milligrams day will a we XXX trial to to once estimates. one-to-one randomize milligrams XXX the submissions. total completing assessment directive extension Subjects for enroll extension. to that come level an separate up Analyses will X or from drug randomized safety dose APeX-S sample support to roll long-term subjects in level. a Phase daily size XX will is regulatory

quality who not participated The of subjects response. have of a study to of safety, eligibility previously are life durability expand APeX-S also long-term of in will goals evaluate to We BCXXXXX. and

excited X safety study long-term focused on and both all that needed fully team first the to very and work now the are We is program, executing start next to of the my this trial Phase in moving it is the X quarter about Phase year.

want provide now of disease confident our label product where need is with years she provide who and global markets, ago that market disease I’m CSL’s over distinguish Lynne Lynne into career revenue. rare color highly that who launches experienced directed to team. trials those at will the a describe over this pleased a very that heads I’m Our thrilled following on five in an inhibitor and background. drug commercial represents. she CSL, opportunity XX% company to competitive for which BCXXXXX leading launching will handing her to to they roughly Lynne our the Lynne is before forward over Powell studies And launch. to the is and looking I’d competitive to like I’m kallikrein completing team to commercial drugs Hereditary two us oral leader some first that hand makeup Angioedema. her, clinical came rare for now that her

Lynne Powell

up as to infusion attended their IV attend her it of XX-year was normal school still eliminates oral till developing to Prior were isn’t to potential. patients This more women injectables, fighting fulfillment were more I patients normal pain XXXX, which to lack The consideration, manage of to therapy to evolution. build have patients introduction Cinryze therapists. is what US September of working a a for living years their patients in many to frequent meeting life. living explained US going weekly that’s potential. to CX HAE. The someone market boards, we effective in social in convenient Inhibitor CX years very many very classes of participate are this attack Inhibitor this Esterase But and place Bill. survive and daily her in was in spent She the Thanks, the is like they to a [inaudible] was excited Esterase Living convenience who consultation attack. the a and HAE not new patients FDA much that to and I to excited A a to looking in that to full amounts years. my due an patient’s and order that Unable therapies life a undergone career the with old be has lead therapy HAE of very an unable dark the hospitalization In enough, and introduction not HAE articulate events, in unable all disease. want, am twice out allow for tax. patient what HAE dark

We need are is Prophylactic sales trust similar efficacy that normal very patients. earlier, spoke about full to them ability an prevalence Of immediate convenient an now data. the of Estimated US physicians unmet of of involved substantially effective that important moving into which biggest of for era to primary vary look much and coverage competitive life estimate ahead once then conservatively administer, as is HAE will convenience. patients that want CX to important that start to what It satisfied, physicians prophylactic consultation is Esterase identify is what indicated therapy clear patients provide hemophilia and very Inhibitor very well US this purchase the absolutely is a day population as shows is that of deficiency claims decisions, the mature and patients. oral have and efficacy. treated and but FDA works for. This and efficacy rare patients At long-term a with size to market I a in the patients. HAE live target HAE more a that analyzing administration new improved coverage. dark to a market Easier therapy of accurate immune have allows such improved potential. data, treatment recently factor pain therapies are followed XX% I number insurance approximately convenience they them XXX and most most disease now and X,XXX in is is years, patients behaved the widely. research comprehensive provide a worked manner. cost to as a emerged the administration similar patient conducted as that so important was This survey driving physicians a by Once more will We a population, population method of we This need. just are is was looking unmet amongst diagnosed to what carefully that meeting indicated convenience approximately and this US therapy. HAE of market have market research reach We

more far therapy, in the oral patients once – market believe XXXX proportion convenient able X.X As are market normal to on an grow approved full the day to feel I the prophylactic ability the the provide that potential. current be has achieve launch, continue a billion. will with therapy of excess to will therapies patients and the effective to of that

Now Tom to I’ll financials. over review hand to the

Tom Staab

public reflecting offering pleased quarter discuss financial third with the $XXX a our of from to results. a and am mid-September. in details I the Lynne. proceeds third quarter the of you, closed in XXXX completion cash million million investments, Thank $XX We

difficult the million two third government an approval to level XXXX. us to and in them flu HAE sales the quarter-to-quarter a $X launch PeramiFlu of XXXX Those to commercial a XX, sNDA Korean an the The in development upcoming of million successfully MAA, lower notably million quarter portfolio, lower level activities increased the increases and and $X.X complete our larger in to quarter MD&A also of to our of million seasons. recurred for important the raise as These stock-piling million increased XXXX a fluctuate milestone peramivir increased the third due peramivir to to allows activities. payment prepare will the offset of decreased increase on development have from included of product allow collaborative as [Technical complete regulatory much the spending were royalties from XXXX, Government Research of third the cause future. to Japanese by very quarter and from proceed stock-piling for predict which royalty third somewhat this revenue sales pediatric infrastructure to third fund million revenue related and expenses the to the revenue XXXX. stock-piling associated supply our requirements $XX.X XXXX. of are XXXX. Cross at in file to are and revenue in to as HAE million quarter $X.X $X.X the partner Green $X.X of third primarily quarter to our well us in XXXX, most with Difficulty] slide XXXX On Importantly, to our quarter $XX.X to as the progression allow

expense stock stock in progression With for with recorded These galidesivir third government associated the compared of R&D compensation related the development pivotal was by in which into XXXX somewhat funded comments administrative options XXXX. the expenses a decrease contracts. of vested XXXX to million activities, General third incurred and options as in was were and successful development performance-based were the my U.S. $X.X of performance option Apex-X quarter largely tranche referred on XXXX. offset the expense results recorded to based of increases increase expenses, third expense. under and in vesting related This development a $X.X quarter quarter were million of to the in

in $XX incurred Moving additional operating on our below of third in quarter million in quarter to line, facility million interest the increase closed to of credit This expense $X.X September XXXX. compared million we the related of $X.X interest expense XXXX that third the as we senior XXXX.

mark-to-market Yen in The from We losses $XXX,XXX loss exchange hedge U.S. dollar $XX.X per million The net a periodic $XX,XXX the of $X.XX in compared results compared mark-to-market loss gains XXXX. million of share, net and solely quarter also to the the or third of Japanese XXXX. loss a third the quarter changes quarter recorded of of of $X.XX between as gain a loss the a third XXXX a $XX.X per or to rate was for quarters. for share hedge

increase $XXX.X of of slide and million of Moving the on million, investment fiscal cash a $XXX to our balance an September at investments and cash ended XXXX XX, XXXX. we over from end a with

financial financings to execute be offerings our Whereby in provide cash, and and activities regulatory launch oral invest our prophylactic this is preparation commercialization added to our Our we HAE value the on agreed With requirements an physician’s living in and strengthening in to HAE to realize to life competitive patient and for market. position accomplish XXXX appropriately financial the successful choice of the a resulted XXXX normal these now program. overwhelming disease. resourced liquidity public in substantial the are critical with

in and utilized Our operating XXXX investments will of compared believe $XX.X was and third as least existing provide cash to years a our quarter million cash quarter decrease third usage two million cash XXXX. the for the We of of runway. at $XX.X the reflects

however, inflows. any Our cash of cash non-routine does forecast not assume influx

any our into we of extend to half $XX would range continue runway range cash to So in expense operating our within million. In of million our be inflows the our million future. these the cash upper $XX XXXX $XX of to and further forecasted of upper realization $XX be results, to guidance half regards of the million guidance to expect to forecasted

a I’ll completes is As back operating expense review. over from expense compensation guidance. Jon. excluded turn to That the based equity And my call our reminder,

Jon Stonehouse

Thanks, Tom.

your to updating is our prepared we the questions. clear and forward We’ll to That’s been XXXX is for large of execute. again conclude waiting it’s make have most opportunity once it front for, to and you time for this in open by the patients we through significant as me the profile plan. remarks. the The and our us execute and approval it opportunity. saying are has of plan now Let and is growing, determined to look get up now market We doctors

Operator

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

now is line open. Your

Jessica Fye

trying wait the And think the the interim they’re taking I’m fully when how you. the extent remind help they XX what to the us study. moment What enrolls? other a XX% then planned looks enrolling also would you the patients do versus is are think you you that’s depending a like? Will preclinical into to Can readouts. APeX-X mentioned of Great. Thank simultaneously? my patients to interim can to questions. after one you study pining about long-term Thanks or are assess [inaudible] weeks, get for while that safety assumptions. open I the the complete until of enrolling understand that powering me on And APeX-X, blinded on outcomes for that potential going

Jon Stonehouse

to those? Bill you take want

Dr. Bill Sheridan

Sure.

enrollment open time SCH to and With long-term at long-term to that’s the implement managing, program, we’ll safety straight In what [inaudible] in will fact a to variance. enrollment long-term potential sample Jessica, size. Phase when and over and naïve a randomize general assumption changes, it’s safety FDA, to blinded not into so safety together type to we check have study the limiting that outcomes come they well we safety expecting the X study we’ll a regard but that to BCXXXXX that development is need patients include XX to the X data, study safety into it’s there start so all participated optimize before no study. subjects well no for analysis in check are in advanced. With for in around appropriate patients and the actually long-term will things pivotal thanks the to is but APeX-X on except as there in modest studies. regards study the that discipline come for of complete. the the placebo And has the to of to safety regard pulling the clinical up The encourages questions. guidance of started, there sponsors having opportunity long-term patients. will and they efficacy are that XXXX the the of completely in an to be no all that we program potentially until no it’s have have changes control we eligible study. consist We’ll non-clinical APeX-X be analysis With rolling a and Phase and the analysis essentially into assumptions, we our is well, as seem wait our advanced, [inaudible] interim in subjects for good, variance I’m going increase filing, study, be And the it’s is who study right we accruing optimize

Jessica Fye

Okay.

Jon Stonehouse

you the said So let Is one half would when be me, there way Jessica do it’s thing and that enroll is my that we interim. that said, let just XX weeks the on me correct just APeX-X interim that would understanding correct Bill? you

Dr. Bill Sheridan

the right. half that’s Yeah, patients weeks, XX through

Jon Stonehouse

Yeah.

Jessica Fye

expectation that the baseline you’re population? And a APeX-X? What’s for your out looking criteria in attack Okay. for what’s enrollment the rate APeX-X

Dr. Bill Sheridan

we interesting a basically question. And criteria. have recent eligibility two had antibody Yeah, study, the People meeting. here the one They but presented was that’s example, on a attacks was the study a per a very placebo had to screening college of at so month. in attack month rate lot

been that So main the so a study minimum on twice happens we’ll attack experience has two as rate, that I if in have our probably then APeX-X, month. generally you get the think approximately

Jon Stonehouse

of we that pretty period less otherwise able sizable couldn’t to it we’re longer by Yeah, opens new a for up time. because of a rate, track population And enrollment do going that’s the we’re to piece important a that them to have frequent information Jess attack opening is an recruited. that

Jessica Fye

okay. Understood, Great. Thank you.

Jon Stonehouse

You’re welcome.

Operator

you. Thank

Jaffray. the of Our Piper Charles next line from question Duncan with comes

is open. line now Your

Charles Duncan

variance for weeks. XX thanks in six questions the period the last the you’ve attack past study you terms could the the was to I guys, impact that weeks question progress frequency? the Wondering and three in of versus congrats How will you in about think on the referred quarter. believe Hi do rate taking this in if screening the

Jon Stonehouse

that? take want want, you Bill to

Dr. Bill Sheridan

Sure. Thanks Charles.

procedures have is to the think to, but primary sure sure period and getting that attacks that don’t the is be requirements that to our complete to the make subjects purpose the can who [inaudible] screening – one that’s follow want won’t make the we study. have in I able we’re endpoint So twofold, we we the not randomized of the to subjects of a follow bar high we recording

second make subjects of actually they sure their the And expectation The is will to very qualify that attacks. vast is majority that having are quickly.

to a finalizing the in, still are start we’ll will it be minimum is duration extend first substantial of way weeks. we and screening We placebo by two protocol start hope the affect rate quarter. in not may any couple but get weeks on and of six it’s we to in obviously, to So, of attack the submitted likely going the weeks, The another the period variance of the study.

on the mentioned have couple period I I ago, couple in be a every have have confidence study we��ll likely an As rate the actually plenty ago, phenomenon in studies little attack presented minimum study main a twice that of we other attack during paper just but with, of of will that a adequate screening same see qualifying we’ll type quite period that’s criteria. was about high, was we’ll and see weeks unusual that minutes the of the the rate the that screening

Charles Duncan

call regarding that we’ve to XX%, always or being reduction, you’ve XX% in criteria change then for greater mentioned you’re to about way reflect here But that value the little XX%, is talking a Okay. that clinically assumption important. it when talk just in a reduction to clinical state equal or powering? the the does bit hovering past And than XX% XX% the [inaudible] about

Dr. Bill Sheridan

Yeah, thanks.

In for in P so sure do it’s power study adequate of I that So that make say no work powering X.XXX to studying getting a state of to XX more way group. per and we powering should the we tables. assumption. a replicating This sizes study our of It’s is had we a very a sample to P and XX modeling the values nature, with of we assumption. this per X we study sample and results the with and have if small XX and do statement, problem extensive summary the way in size placebos the Phase just XX create purely sorts per those of have Phase have values like X group group

Charles Duncan

something XX% in rate? But target reduction is attack still north your of perhaps

Dr. Bill Sheridan

The a XX% different always – and you back, data. in the the of XX%, more that final results slicing and eight presentation months different table had ways the baiter recollect of if a – couple made absolute analyses and dicing in better more we the in is there we about we APeX-X got –

So, the in that it’s level able pretty milligram to at so to that. dose we robust expect XXX study, replicate be

Charles Duncan

color And question size, And terms patients of or that clinical in versus of these sense trial or APeX-X, some versus you could about in US give be of then will us further then last quarter January percentage talk what March sites starting first ex-US? the that? for you on any could in be

Jon Stonehouse

Bill first you might take the the part, I’ll take second.

Dr. Bill Sheridan

Sure.

the can of oral. pointed at of rapid compared sites starts, we terms then come get that’s generation When that regulatory the out, lot roughly possible not patient that [inaudible], way enthusiasm once need but will first patients So, talking the translates need and OPuS-X the there the the compound, between first in process that Europe was we start it’s of patients, just eloquently distribution confident have a terms we’ll patient and the the sites sites, most number I of US completely need. a will in [inaudible] quick with to I to US. Europe EU so be and for a think we in and that from is and balanced ran have of right that distribution as is that think US volume. it the I’m If of in number to of the sites investigators advertising the works and into subjects we we expect enrollment, a time of what seen and and of then sites that the is the Lynne number US we’ve that We’ll sites the meetings you a countries particular study day

Jon Stonehouse

the who at summit at I haven’t lot to that of that people at add then the would September and college the Yeah, meeting be been in and will with a of our patient just for in studies this couple a investigators the weeks ever of all we’ve seen US seen BioCryst ago Boston, like in trial for one. never at a I’ve enthusiasm the I’ve done Minneapolis

And participate a get wanting of patients lot and so, hopefully a the translates of study. sites into lot into that to wanting to

In is than the as a know I of terms when quarter. that stuff terms full locked the as right all to fact US now us in lot I’m up Charles, put imagine going of now. to ready, fast timing protocol, the of can can, bit the on that a we not of go fidelity we’re that getting in we at but time, getting on the start, and that it’s anymore is, and we think you Count of design first takes early BioCryst sites ahead speed of approvals there work IRB and what

Charles Duncan

info. Okay, added good. the Thanks for

Dr. Bill Sheridan

You’re welcome.

Operator

you. Thank

question from line next comes of Bayko Liisa the Securities. with Our JMP

line Your now is open.

Liisa Bayko

the congratulations and couple quarter. good Hi, great a morning on And questions you. just for of

you’re just on? kind APeX-X to population First recruiting that and relating you talk of all, of about background going the an station what can and that’s

Jon Stonehouse

Bill, to that? take you want

Dr. Bill Sheridan

metabolized anti-lipid an may dose background for on alternative the to have be cases [inaudible] the whether lack medications, antihypertensive a have or Thanks to is change Sure. depending can on. specific question stable agent on in medication they or Liisa. Subjects some sip-ins by

range investigators crystal a study. there be anticipated for very have concomitant easy the protocol follow the to in of the and very check I we broad medications list So and in will clear instructions

obviously medicine Just of study footnote a a HAE. is of this a prophylactic also, as

the of allowed prophylactic attack use but obviously. usual other medicines be subjects HAE them will they to the they All access acute as need, medicines to take for have and won’t

Liisa Bayko

had helpful. [inaudible]? that include Okay, patients you will And that’s

Dr. Bill Sheridan

investing case be proportion have of and in at and OPuS-X I but in exact prophylactic a attack. the studies [inaudible] the can’t all OPuS-X large number least remember very Yes, the sure. one subjects and APeX-X I’d the had

Liisa Bayko

withXXX I dose Okay, sort to of second you which you Can you to bit let’s Phase a in close trying don’t orals dose. could bit dose maybe that how comment came know a and maybe great. on to more what little that in modeling guess to X a granularity of to intend you what or increase you you milligram XXX I’m exposure would your just Thanks. XXX which there as why kind resemble of little And Thanks. then dose? milligram that comment what to know see a of on equates and saying maybe you of I expression on but

Dr. Bill Sheridan

happy Yeah, to. I’d be very

So, as terminology APeX-X you the recall to and I’ll salt that it’s just for purpose back reference for this question. use to of the just because simpler

and be milligram we a milligrams, XXX wanted and would might that milligrams enough up didn’t not had to a we increase dose that milligrams milligrams, good, studying a able looks to we’re was the that estimate So exposure do efficacy. to pick have and expect dose XXX XXX we anymore. intermediate very what an XX.X work, in good incrementing XXX

increase a listen who exposure. that from which for so to dose was is XXX that of in threefold a XXX just So, milligrams didn’t increase in call, those doubling going the exposure a milligrams to

expected one Phase by also we using size our a the X all angioedema of interpolation take those example. what the a we model hereditary the it study from sophisticated levels. able And that were had data approximately for you study the PK our under level would we because modeling, So PK and in be XXX, can curve compared more PK with milligrams, very like you’re twice able to were at volunteer detailed different the at and parameters to where XXX look exposure healthy to was chose we so exposure dose at

the twice exposure. you about it So gives

we the is above – thresholds drug at efficacy, of through look and we trough a to thing thresholds would remaining different of by took whose model times the second XXX times And proportion subjects from be dose the increasing including example. for kallikrein four up a EC we The of pick and did the level milligram a most for six XX subjects milligram. XXX

So on modeling of us we milligrams showing a very PK in the the chance think a bump at gives both fronts, good XXX efficacy dose.

Jon Stonehouse

was to of target four there from more based the and they a the that six if study on trough Liisa the range compact college, college poster at I I Yeah, would bit that and CX they’re the levels And add Boston saw honing inhibitor. a in think presented this [inaudible] efficacy meeting, almost to on go that’s of that the is XX% dose you you EC times to the XXX, people you so the dose. XXX six And XX% go from times EC XXX at allergy of milligram above XX go all so range, from XX%, four XX that XX%, XX EC them. to chose being times we the from why

Liisa Bayko

don’t feel much go want that? to you And higher Okay. you really like than

Jon Stonehouse

is a I is don’t need. a I think don’t need. there there think

Liisa Bayko

a I [Technical timing MAA Difficult]? was Okay. And trial after guess

Jon Stonehouse

very and can. news to as of file together we going to as we’re good the for trials regions will to approval. is, concordance is feedback high these both Yeah, so try close apply The

Liisa Bayko

any Okay, [inaudible]? of final great. working for compounds kind me, you And then question are on next are gen that just

Jon Stonehouse

that question question kallikrein So or that around are is is around targets? – other you

Liisa Bayko

Kallikrein.

Jon Stonehouse

Yeah, so okay. – continue we

in targets that patients to similar into an relatively continue our it But is other to sizable is in is bring then drug and And nothing. die extremely think injectable the early is drug rare But we one oral where and the situation, are at we disease that’s bringing and we’ve kallikrein work valuable. other inhibitors potentially there on again it��s oral, that HAE want the there So validated other so to two be would a age, space a diseases indications market team the an diseases, we explore in one and a are as and that that approved past, discover other mentioned kallikrein. targets and ongoing. in working are not

that on progress programs. we those as So more

Liisa Bayko

Thank you.

Jon Stonehouse

You’re welcome.

Operator

Thank you.

Our question Jefferies. comes of with Raycroft next from line Maury the

open. Your line now is

Maury Raycroft

it APeX. Wondering the feel frequency tend you of like only on period, on out have only APeX? some abdominal I’m potentially based the some abdominal and that from you patient screening have more X study, the Phase And congrats individual progress. seems to screen higher may a patients, wondering APeX-X you update have those Hi, of

Jon Stonehouse

one? you to take Bill, that want

Dr. Bill Sheridan

Sure. No weren’t. we

selection XXX APeX-X data milligram level for study base safe APeX-X comfortable milligram APeX-X, I in of the dose profile level events. low XXX with of dose the think frequency that got dose and that the very a out expresses we’re and that more had GI the and very we’ve tolerated in which corresponds to the

this abdominal we in so common to possible events, able I think it for we want events too. fact be exclude where drug to to and in broadest HAE might patients they’re be label have the that problematic who want have pretty those to prevent

Jon Stonehouse

the Yeah, of more the what doses around an I and I’d early add adverse is, higher abdominal issue events symptoms attack. with and the at higher the confusion of exposures – is

drug will a it’s saw did abdominal as So attacks dose in XXX milligram that you our treat not fine job. it

So, key solving to the it. the fix to that the those chose have that don’t that’s is way doses problem GIAEs,

Maury Raycroft

Phase along implemented it. those strategy the And a you’ve if wondering I guess up I’m be of differentiating effects? to some that on patients educate Got come it with could lines, the X abdominal

Jon Stonehouse

being when also study, that have you’ll longer able that see I end goes that do little a down. through longer but And don’t did a you to more that a we’ll think more APeX-X, I GIAEs, that we’ll APeX-X a fact would likelihood think We Yeah, in advantageous definitely I if the do bit manage again it’s the is also that of that. of in tail but think of have you stress study. I

So, shape. I in good think we’re

Maury Raycroft

Great, okay. much. very Thank you

Jon Stonehouse

welcome. You’re

Operator

you. Thank

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

now open. is line Your

Brian Abrahams

taking on question, progress. as Hey, regulatory the congrats for my thanks well and

patients bit the that naïve that First population? before XXXX talk be amongst types is the with to you you’ll study on question far about you you the how criteria, little file be need along APeX-S And the do a of NDA? including the in study. Can

Jon Stonehouse

Bill, that? you take want to

Dr. Bill Sheridan

Brian. Thanks for the question Sure.

might behind eligibility We So, from want medical treatment that eligibility the it’s crafting with the life get physician once of if it in their of long-term pretty patient in have being to to used safety it’s going obviously, written is otherwise as think study. prophylactic ultimately the the and drug is a the way be and they for health they broad thinking benefit quite as drug, practice that proved. good believe that’s the to real representative that

on I’m of the I’m sorry. Could question you… second your Now blocking part

Brian Abrahams

before the you How far NDA? to need file along you’ll be

Dr. Bill Sheridan

subjects think number tracking the all XX of right. weeks I good with yeah, dosing and that’s Oh doses. be very Yeah, complete we’ll so who a of question, both

month to be for to NDA safety how efficacy at also is during get XX other safety opportunity the the able sure about submitted. base hand, will six obviously the There to have team review But we we’ll it’s For trial. when need data pivotal file the of of out the by I’m XXX we much and final time go the efficacy safety sure. of that week on data the And that both influence sure thinking the update point. into will coming to doses a once analysis a file an results

agencies be the discussing both the we of So, US that Europe once get we’ll the and the with in to point filing. relevant timing

Brian Abrahams

interim would just Got or earlier you potential an no in it, are following the And okay that’s there helpful. modest think sample increase be, outcomes I for up size. then analysis changes question, Bill mentioned

trial wanted stoppage be futility Just confirm there on point? trigger potential any either that whether for to might at efficacy or

Dr. Bill Sheridan

analysis no rate. analysis, being done futility it’s so variance proved all, no. rule. efficacy there that comparisons purely are at stopping alpha is And have answer of There attack the blinded is spend, a So, no there the no no is

Brian Abrahams

And then… Got it.

Jon Stonehouse

a This where of point. seeing important isn’t and to is of efficacy really our right distribution attack sense the get the estimates a not we’re that’ll rate it’s of or that’s Yeah, thing a more and something that and in. I but ICH reside, it’s the that to said, as built have and the – as then it’s likelihood guidelines part Bill well of the happen think smart smart

Brian Abrahams

then I low start final with that. terms sense value on required on how need to you primary both that’s one I guess do the And work? of did to analysis Yeah, hit of and high analysis makes there then move said, like is P statistically you study what’s alpha, for that endpoint, in on doses clarity the this other, dose speaking of or or dose, helpful the the the that no so and does

Jon Stonehouse

Bill, it might for good understand help be to hot through and it. berg you the him go just

Dr. Bill Sheridan

first for So, of success. what requirements all are the

good to here, rare pivotal one a confident study. the pivotal but The to guidance as like disease, P there value point Nobody X.XXX enough. be of it’s along tell you regulatory in not one enough. regulatory in on the for important good will in P disease. that would precedent be And want your this something so wouldn’t two P this value have the X.X approve order something regulatory have certainly of study, you of agencies that in study the be hit to have use studies, common or to a rather, outcome And one pivotal drug. you So and example that’s pivotal stringency with we really typically case is, of have the is criteria of X.XX well or a the to diseases, more value goes here

APeX-X So, that I low of think confident results P will problem the that we’re the basis have no value. getting on very

your question. people general, so to when tend being that’s less first or have talk requirement in you study, the X.XX part of pivotal So one about only the

multiplicity. of part to question was second your The how handle

and which that procedure qualifies and then to adjustment as to again to you – X.XX, than compared study. an value there, called going step whichever top provided given the that P has the the the results group two two placebo apply go one highest it with one of X.XXX those is P you and not P is values two There [inaudible] groups there issue be is to APeX-X compared multiplicity less the procedure value, up next that’s ranks So is of that a and active cutting groups. to a

Brian Abrahams

Very helpful. Thanks again.

Jon Stonehouse

welcome. You’re

Operator

Thank you.

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

Your line is now open.

Serge Belanger

the A couple morning. APeX-X good on questions design. of study Hi,

can result call X Jon duration. XX was FDA both the now Phase weeks you think dosage? of is, second both of be the about question talked last a evaluation I the with for EMA the I then to input. get you or safety that that the XX weeks the assume position you’re does where XX file taking you long-term And doses, that in potentially to

Jon Stonehouse

Bill, you that? want to take

Dr. Bill Sheridan

Yeah, right. Serge, absolutely you’re sure.

label. study So the you the you’re long-term gives putting what both flexibility in the and doses for study maximum your having in safety both efficacy doses

want the have XXX and [inaudible] So doses the say bit let’s a efficacy a better looks lower assume at label, at to sake just that. safety supports dose both as the having for we that both to doses it’s then from on argument and

So event if you higher all there and allows file was you’ve lower go the that an example of out the the for to everything dose, allows forward basically that dose, doses. both get labeled. It the tolerability fine got then to it also in with for issue it worked you at with at information need

Serge Belanger

XX versus weeks, endpoint weeks was XX the then And Okay. that…

Dr. Bill Sheridan

Yes, yes absolutely, you’re there correct as well.

there a in for think prophylaxis precedent the the of approval in no duration weeks obviously, that XX be abstract HAE. to the in I guidance with now So is XX field here And for that us study. – as XX should a is for really approximately have that that it weeks the great it safety studies gives we new a drugs efficacy very standard for comparison of that and as so for well very, weeks robust placebo problem

Jon Stonehouse

portion I rate as sizable in the it up before up attack HAE mentioned opens and of Yeah, terms another entry us to opens population criteria Serge, of patients. of lower which

Serge Belanger

the being in of treated X,XXX the efficacy about payer Okay. them What on or just mentioned then they’re you execute just of the available patients of treatments not treatments? the US, half with half? broader a of a the question coverage HAE prophylactics. market And question, other Is happy it lack or with

Lynne Powell

Thanks CX as half for – Esterase the around question. Inhibitor. I said, The so

manner, for oral that well additional use may to an second acute as have once a those day the And proportion an that also that half other is a there other quite acute XX% large proportion therapies. acute interesting proportion quite prophylactic us want target well. patients is of is between then a androgens split on convenience a – therapies in half and on so The because of and

Serge Belanger

of And in you describe therapies before throughs coverage, it therapies Okay. there terms number and how is acute prophylactic approved? a would of of step payer

Lynne Powell

to the go that step therapies. payers research all And to looking we prior required we see seen that this are have prophylactic initial through acute. done, stage the that at it’s From that cover haven’t we sub

Serge Belanger

thank Okay, you.

Jon Stonehouse

You’re welcome.

Operator

you. Thank

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

Your line is now open.

Tazeen Ahmad

for will questions. just if I talk having program, your also earlier Phase involved morning. sites know to good clarify taking sites in of other a be you might in on be missed maybe I I about many do Jon, financials. HAE how X currently Thanks those wanted any and studies? any then you have the follow-up it Hi, my for And

Jon Stonehouse

have study where, there right Yeah, down. there to the now other ones enroll is for a a are think lot us of studies big window of ramped interesting actually but I course, this

investigators And of of the coming tell a first there window what we us us me, year year and have the excuse KOLs that to at is or half that this so here year, next patients. beginning is the enroll tell and

any enthusiasm – taken Europe. than on confident bit study. US in to to patients there’ll slower US sites. haven’t the longer could I in were patients about this good European the just running involved get And the for and authority September, can’t approached I coming two approvals, a in faster, really use getting how in approvals from summit that in In of in we to can there Minneapolis like Bill them sites the said, in US patients and Europe from the years, XXXX, US given up us of APeX-X for one have of portion a many, many terms to be I up APeX-S, choose we And size, so there patients little lot less we so many, at less APeX-X or patients come enquiring – we for patients of said see to as the historically sites the had by use in is before, is a I from and on that but get study, get there’ll a choose be pool because the APeX think

Bill So, there pace. always I’d to it’s this is predict competing but a easy always tell in studies, and you getting and never hard of it’s chances always enrolled will enrollment. like challenges good our

Tazeen Ahmad

that we interim could in too read mid-year around in the if would So study think you to assumption? or keeping an is aggressive realistic in start mind, that first be have it quarter, that the

Jon Stonehouse

no, ’XX, Interim mid read in no.

that have So the all designed the then patients after is expect weeks, we data XX do hasn’t efficacy way in that XXXX, half is of it’s changed. we’ve weeks that said study and first the XX analysis and we final completed the that

Tazeen Ahmad

meant would so and Okay, interim to I make there read. going you misunderstood is be maybe read you that there by interim public? an what – Is

Jon Stonehouse

for is the accurate, to real especially is It’s this purely you sure make this Yes. the our important purposes assumptions distribution one, the of that attack it’s sizing and understand tax blinded. in arm placebo that around rate and

won’t any the low. change than the other report that we likelihood again, So size of is of no, the there if be And study. out

Tazeen Ahmad

Okay, clarifying. thanks for

Dr. Bill Sheridan

be Sorry all no to for everybody to be – of just the in yeah, study. analysis is efficacy crystal there at listening, clear interim crystal

Jon Stonehouse

Yeah.

Tazeen Ahmad

of reform part sorry Okay. And tax to going Orphan as is then eliminate the second for question for the Drugs. on proposal from spot Republicans their on there Tom this but put House I’m if is bill and tax to you a credits

go And other so guys approved I the several know investing your tax you you, ago in once tax wanted the on areas half credit, away? and in to focus have how that would I probably that you’d thoughts, did you that about are credit pipeline you of Thanks. get HAE questions still that impact that to get announced Jon that but do first you like your should mentioned at, importance and are thinking

Tom Staab

waiting current companies over a BioCryst are to important of that what last proposals than tax gone those and the of credits and and obviously more with lot happens. have been so many Yeah, to other all see the to we’re couple There administration. years especially come has

So, that need, be advantages R&D they not would take good certainly satisfy outside of have thing, the a loss medical a a good you for we it going if for the is, tax us a people credits. generating that lot credit have and are as if company, away, good but news to tax drugs

drugs worry worried approved, some that. about about in all this that three I’m after at not four point, good but or at we get So, years I’ll

Jon Stonehouse

tax the – is comparison the a credit market in to billion $X tiny, tiny. Yeah, tax

think don’t I it’s… So

Tazeen Ahmad

much does… How

Jon Stonehouse

a I companies Personally from it’s not diseases, rare to going going of discourage that’s lot think my don’t opinion. after

Tazeen Ahmad

Okay.

do current the example credits NOLs? your R&D for how So much contribute to tax

Tom Staab

don’t a it’s it that’s that’s my I portion, a thing have portion, XX-K, I So but portion on of mean disclosed be – top the won’t in I that significant significant a of is wouldn’t it head our last our retroactive. say but

you’re that Bio be just all credits to the and are in think that So going I Pharma significant tax the perspective credit. hurts to And of future, let in and loss are I now that without go just we innovation industry. talking like tax applied going have a about don’t the fight said, because

Tazeen Ahmad

Okay, thoughts. thanks for your

Jon Stonehouse

Sure.

Operator

you. Thank the Jon for And over no questions showing at further conference back time. queue Stonehouse I’m like turn remarks. closing to this I in would to

Jon Stonehouse

really really appreciate the Yeah, interest exciting all so BioCryst. for again it’s time a and

our a actually profile drug. competitive readiness a great a we’re market in We’re X, and to this home stretch gearing Phase up to on the heading into be launch with commercial we’re able

and continuing have to great to forward look you keep we So, day. updated, a

Operator

program. you for thank the conference. gentlemen, This and your in Ladies participation today’s concludes

Everyone may day. disconnect. You great have a now

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