BCRX - Earnings call transcript Q1 2020 | BioCryst Pharmaceuticals, Inc.

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Operator

Good morning, ladies and gentlemen. And welcome to the BioCryst First Quarter 2020 Earnings Call. At this time, all the participant lines are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. [Operator Instructions] As a reminder, this conference call is being recorded.

to conference like host, to now Mr. would turn over the your I BioCryst. Bluth John at

John Bluth

and Good Whitney. XXXX results press our and quarter welcome corporate financial available release website. Today’s call. Thanks, conference and update morning to are on slides BioCryst first

me Chief Stonehouse; Anthony Participating Gayer. Commercial Medical Officer, today Doyle; Officer, Business CEO, Sheridan; Bill Charlie Dr. Chief are Megan Chief Jon with CFO, Officer, Sniecinski; and

questions. Following our answer remarks, we your will

regarding forward-looking please the begin, conference that results, statements, financial including forward-looking note statements and/or company’s achievements. as contain we Before call information, those today’s future performance well and unaudited will as future

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

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

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

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

Jon Stonehouse

Thank and you’re doing morning. and this all safe for I well. all you, joining thank hope John, us you

extraordinary an in for within company months BioCryst. next at receive X are We approvals is said the berotralstat. XX The to position

second our approval Japan expect half in We of in global year. the the first

next from a December in time X approval FDA. We validated have Europe, PDUFA the year. MAA expect in that about was date our And March we and

oral Factor BCXXXXX approvals, have a inhibitor, we with a D these alongside our complement-mediated in Right PNH. diseases pipeline for including molecule

XXXX for first the data We you will time share in PNH for with patients exciting today.

the Both in research. benefit for peak demand, stating berotralstat. we north berotralstat and in sales generate we our patients are demand and of Patients significant consistently customer expect $XXX experiencing clinical Based create physicians oral HAE to HAE value of with with start medicine response the million. market the and Let’s our will on our clinical trials. strong are expect

in our extend we billion of market new significant yesterday to opportunity will patent a years value for even to XXXX. more an X greater market we Add and creation. matter have than complement-mediated that patent of announced that for protection diseases, see a established we that XXXX composition of by And for treatment potential $X

the on have commercializing oral rare We focused developing for discovering, company drugs and built diseases.

advance rare discovery pipeline, BCXXXXX to oral continue diseases. disease additional rare also for includes We for our which and other programs FOP,

always our an believed Beyond important antiviral health. we’ve play public that, role programs legacy in

is support the a add across we trial Galidesivir and our spectrum patients. to relationships in developed broad The past programs funded have decade, antiviral continue NIAID over government U.S. in experience antiviral to a COVID-XX the value.

We have quickly been galidesivir to million $XX totaling move we’ve for with of funding and patients. able COVID-XX contracts into government galidesivir

Brazil Patient could this if begun in in dosing we to look in trial help has and data galidesivir generating determine our to emergency. health global forward clinical

pandemic the of every impacted disruption coronavirus The company. has

the While progress is to the situation BioCryst for its maintain and fluid, most continues part, timelines.

berotralstat the fortunate that and pandemic. data for trials time the We’re the of support regulatory already were submissions completed our to at clinical

on remain are regulatory and activities As well a approval underway, our launch track. our timelines reviews and preparation result,

progress wrap will drug Charlie review will the Then support new Megan sharing teams affairs building will upcoming galidesivir. with and our our I commercial by been data And up update. Bill provide with and Review supply have XXXX. medical an and a they’ll global launches. Anthony Financial and our to provide

to turn Charlie. With over call that, the I’ll

Charlie Gayer

start XXXX. We’ve since of been the John. busy Thanks,

in execute markets. for commercial to hire team complete U.S. leaders representatives key are access Our individual are regional quarter. marketing action. European in and teams market in the our third launch our experienced territories sales added efficient preparing And to an We’ve and sales also and

slowed clinical us our research our I’d are completed launch like last and in focused of plus will has year, review first, our Megan we growing efforts work Our the market down. the not describe to data. preparations But because of smoothly, more team. moving readiness forward detail. COVID-XX

prophylaxis. be launched States recent can injectable unpredictable to United attacks products new patients which and HAE Several in devastating, in years. explains why have moved had most the

their treatment experience have switching them. So, patients to physicians that and best is the find for

What many is is that our prophylaxis switch big to to disease their patients oral switch. want focus and A part burden of of on to reduce the now strategy control treatment. to

treating this physicians Our based willing to top-line with APeX-X. to We the market growing recommendation. of XX% a us confidence profile use XXX and give patients and clinical strategy. XXX on data and were said in berotralstat, research from data surveyed physician XX% very presented patients they HAE

these Haegarda berotralstat. with Even Takhzyro, are willing satisfied use very Notably, most among XXX also to use and Cinryze, them their using XX our of very half or very injectable, drug. willing current patients of those to were oral were already

that? is why So

want an experience One patients most preparation oral reason reduce to and even patients of attack our reason treat is of patients Physicians and to The understand current benefits may such the month. average oral be burdens, attacks, still storage, reported injection. they current XX% that with breakthrough X.X in Takhzyro, injectables, for drug with per treatment expect future. about on as broader prophylaxis example, the patients

to switch. also that injectable data clinical are many Our patients show prophylaxis on likely

were newly U.S. APeX-X prophylaxis. patients XX% come see our anticipate last previously These in As Since the of on about Takhzyro, patients XX% prophylaxis. treating numbers Slide APeX-S you can opened use in they the physician Cinryze. who enrolled share previously with switches research. from half expectations in enrolled of berotralstat, year, Of inhibitor align current XX, Haegarda for from CX reported with XX% or

are trials from weeks a our but baseline only placebo attacks month, for about APeX-X treatment. in in average XX half of X a X month. milligrams just Those on per patients milligrams per year XXX experience attack attack on had to switching staying benefit. really Patients average a they after averaged an month because on XXX berotralstat, Most per

gastrointestinal free. half in self-limited the out events And shows of The the from the symptoms, months, trials generally treatment. The long-term have are drug is experience within tolerated. similar of well these but results. both of of attack months Patients are first milligrams most X and mild, these XX XXX safe main resolved and X taking are more in APeX-S adverse or

interviewed enrolled APeX-X XX in berotralstat how United helping patients recently on been for a them. represent U.S. have in own The year. quotes over their words We who the States XX in Slide is

close lives. what to our we on see berotralstat can And HAE bringing be You drug patients. to having once-daily a impact excited is dramatic oral are so to their

to it Megan Now, readiness. I’ll describe global turn launch other areas over our to of important

Megan Sniecinski

good morning. Charlie, and Thanks,

the berotralstat. be to excited of launch preparing We’re for

ongoing agencies major track on As as approval with John X shared by earlier, planned. we have timelines reviews the regulatory

what of highlighted, a our Building Charlie readiness. few touch like on on to I’d additional launch aspects

across pre-launch opportunities exchange our continue education to First, turning on our are is for engagement, supporting EU, we and the a the medical fundamental berotralstat. the part interact and U.S. which scientific clinical to These with evidence important the KOL of activities, community. HAE

major posters series Recently, affairs community great shares virtual poster which data hall our and to with for cancelled team response accepted scientific as HAE the to were opportunity of have seen, conducted sessions, due virtual was a in you community. and present our COVID. physician of a the to from The a March in medical may annual AAAAI a hosting congresses one shifted Meeting, the the Annual congress

calls of the as we’re continue clinicians In continued organizations. light will more virtual patient region well that to about clinical Overall, to as Europe, HAE what meet in accessible various see trials a we unmet treatment program. prophylactic of pleased and strong by patients. also KOLs still treatment with via COVID. learning we work, HAE our continue how once-daily be need the interest significant better the continue really oral, across for in to interact area its From And in this is

seen to the committed past, not berotralstat. to HAE supply having readiness, for BioCryst ensuring shortages that supply for other treatments our happen Next, early would on moving in

chain supply We stage. redundancy have for manufacturing sites each source dual with X across the

with partners, I’m we We today, and for ample manufacturer at are for have positioned PMO time, a more well final of in packaging, foresee because than don’t early this impact We product happy COVID terms supply supply well-established that to share our are already any launch. and investment. commercial to of working

fortunate trial think this we We Site month as screening pandemic. even while once-daily to our and mentioned, And virtual continues clinical companies onsite the to visits stopped despite prophy continues Charlie consultations. U.S. the APeX-S operations, studies many been enrolled for unmet clinical alone. have APeX-S from to last several need we’ve transition treatment the patients current continue and monitoring for speak in oral, Lastly, option. APeX-X demand and

remain focused through our partner on U.S., launches in Our preparing successful teams in the the Torii and Japan. EU for

we approval contingent and following With to to the a including launch Torii clearing approval as building minimum milestone a with PMDA reminder, payment global potential pricing receive Japan, million our preparations a underway, receive actively are threshold awareness discussions. first in price And education. disease the $XX MHLW upon our stand

a as It’s BioCryst, transformative clearly months the year launches. what’s to us our ahead of we with next in XX for look

we’re now. In that Bill? you focused XXXX program. addition, will on walk through advancing And Bill also our

William Sheridan

much, very Megan. Thanks

the dose BCXXXXX. with cohort very are D early of study data patients PNH our inhibitor lowest Factor in oral share We our excited to from treatment-naïve ongoing proof-of-concept

milligrams You and goal our of on XX the well monotherapy. data a to XXX from we way twice are will achieving day a see that

was effect the signals. subjects on benefit. have control and healthy the to compared dose We no study, of pathway seen lower dose We’ve activity milligrams day related of effects milligrams superior XXX doses. at on and XXX twice the hemolysis alternative clinical XXXX in seen And ascending multiple a safety

XXX we hemolysis. should cohort twice you tell the data move next PNH treatment-naïve As day control a and These see patients. milligram us to complete XXX in of

the As X will the a XX twice shown Cohort and milligrams twice day, is XXX testing XXX of and a X Slide milligrams cohort on XXX study a PNH day. test reminder, is XX. design

XXX as benefit had continued of in drugs. X that is the far, X patients long-term on drug, days So days XX have visit, treatment-naïve from the milligrams XX we enrolled day we a inhibitor day completed of XXX are that today? a XX where have XX milligrams orally twice assessed twice the not clinical a PNH PNH CX administered by So all day. means all have dosing at milligrams and monotherapy. by followed day, investigators day they a At X twice twice so dosing patients XXXX had extension.

One, PNH. seriously third second the cerebral aplastic required with vein see cell had that and On thrombosis Slide the and – had you PNH. XX, were patients disease, transfusions, from a these red anemia had previously ill the can

degrees among liter, elevated of had or the patients units bone reticulocytes counts, variable marker of was X.X XXX X.X to grams PNH the dehydrogenase sensitive a with patients, of And X,XXX LDH per to over degree deciliter. the try over times of lactate anemia, level X quite limit from normal. X.X anemia of before treatment to severe hemolysis XX to overtime In the range hemolysis get show patients and X the to or per working reflecting hemoglobin upper marrow All hemoglobin our up.

encouraged the responses with a twice very doses of laboratory hemolysis the day, and day We and key of are milligrams by milligrams seeing that clinical all twice the a biomarkers improved. lowest XXXX, XXX but XX we’re

individual PNH effect magnitude another of in dose baseline Total can clinically given patients in on All and hemolysis Slide Bilirubin, sources had in these the dependent You XXXX. patients. see fill normalized data low of and X on elevated The X optimized. Reticulocytes that meaningful marker is at and drops not in counts impressive, XX. are was X all LDH.

X XX.X eight day red see week takes it day. study already from This hemoglobin stabilization doses, XXXX Previous dependent at and at Hemoglobin of studies the and shown transfusion a our of cell while study of example, have for about transfusion in to on X window to week X post hemoglobin free XX. X now has in hemoglobin doses. unit transfusions X the is X.X on on has milligrams study, X.X patient subject for at with XXX lowest following day our complement X, increasing weeks been a weeks, transfusion a optimized with twice risen inhibitors at

observation on effect shown The profile no XX safety is serious which headache drug complement patients inhibitor well-recognized healthy earlier volunteers, early evaluation the drug tolerability PNH. transient in treatment in our dosing, XXXX during a XX. in no a common day Phase Unlike There related Slide were of experience X Most rash. and of period adverse is class was developed events.

patient on and taking and who other dangerous event event clinical exposed occurred to corticosteroids this disseminated subsequently contracted lymphocytes. varicella treated determined to to the was Varicella led period, patients serious the known adverse drugs azathioprine, a steroids has a worker, suppress XXXX. a be unrelated unrelated extension Based varicella. that that investigator with history, is death. especially One was in we This is infection to was in frontline PNH patient healthcare chronic

twice X and CX following XXX we cohort patient a strong treatment-naïve continue patient interest And patients fourth are we patients, despite testing begin X, enroll to was Our to to inhibitor of receive efficacy in PNH recently Africa, expect day. South responders in naïve to XXX from the milligrams cohort inhibitors. COVID investigators challenges, who in cohort CX completion enrolled X, enrollment and of poor

We expect responding the patients end these to report beginning patients rolling the quarter and from for third year. in of by the data

about excited XXX patients. and a milligrams XX early at day very in this data We’re twice PNH

MAD milligrams profile were the the Note for subjects. state in and a pharmacodynamic MAD have for consistent there period cohorts profile results were individual safety hours for XXX shown for XX especially the The both XX The twice treatment, is and day Hemolysis superior dose. PD doses provide the are XX XXX for to PNH doses twice signals. last Weislab the we coverage, the no Slide continued the higher completed the after that the AP and milligrams these in Also, XXX in XX healthy of subjects beyond and assays on of healthy dose, importantly day a more hours clearly AP after steady assays.

super the The values level may healthy the characterization see milligrams assays XXX dosing interval of at more subject milligrams, as pharmacology XX, on at twice When complement you cohorts blocked XX% of this additional clinical be. daily and well up once-a-day activity plan might than profile by day, study, steady you the a AP of do as explore in study of Slide was XXX and have suppression, ask MAD state. of main be if doses. XXX we with the could in throughout we by therapeutic both shown drug, testing XXX both wrapping it XXXX once as And the of to dosing

XX we have twice what a treatment-naïve and patients XXX So clear learned with dose? is benefits. at response clinical there First, about milligrams day in dose PNH a

the to results day level to Second, CX subjects XXX were XXX XXX in superior twice lower and healthy plan milligram. cohort doses that Therefore, the i.e., dose PD milligram poor a begin at milligram, doses. of responder we XXX

MAD monotherapy PNH The support is develop other as strongly BCXXXXX we and mediated healthy share for complement data subject to all that goal. today goal PNH Our disease. an patient and

of complete about PNH excited with proof-of-concept and We’re to study diseases a speak other by to and regulators in dis-regulation our next caused complement. steps

Jon Stonehouse

Thanks, Bill.

monotherapy. XXXX into for us have are of get through you our study QX, early the investing we capital funds As oral capital of this imagine, and of $XXX goal reported an efficacy having with launch part proof-of-concept about year sufficient year. excited that next This as preparation the the berotralstat. D and getting to has in completing can these fully at inhibitor we results and end our we Factor With million great to very closer

the the that We also our bring with out ranks Anthony gives rising candidates And CFO new capital as past and in flexibility to among a spent He and conducted nationwide some CFO, to you. of exceptional plan that at stood majority prior a into search pleased the additional through for very I’m introduce company. us X career Anthony to that Global a his to spent of years Doyle them. GE. We describe comprehensive CRO, the have the

intro, the over to turn I’ll With that Anthony. call now

Anthony Doyle

to It’s a Jon. certainly me be great an and BioCryst. for joining exciting time Thanks, opportunity

launch the help has coronavirus company and it, commercial future. opportunities to D success tremendous for pandemic berotralstat, near Factor them an in the galidesivir, behind of the a upcoming the with runway including With inhibitor, pipeline strong oral

with the from financial to did where can upcoming the and in You I the detailed press find first results in are to balance approach highlight our quarter the capital months. sheet want But the release. we

the $XXX noted and on QX into million early with through based outlook Jon provided. on ended we’ve As runway This XXXX that gives cash we side, us XXXX. the

financial through additional potential progress capital the several to have We year. we flexibility sources provide as

with $XX And the to add launch. partnership berotralstat that we’re for from bring evaluating advance Torii. as milestone fund to BCXXXXX financing additionally, options royalty and/or debt capital such to our Also data to – a million from program. in to at expect would trigger capital provides We up that approval

this extended behind our with into through Stepping role, than of next Jon? very greater dynamic looking to I’m forward starting now year patent believe new much product peak sales generating with early a a revenue have very we pipeline $XXX us. will million, and XXXX that

Jon Stonehouse

polymerase placebo-controlled galidesivir I into had are also trial. Brazil. announced COVID-XX. galidesivir randomized testing with The Thanks, opened funded for are patients that dose progress potential In is clinical update patients which on trial NIAID. you Part treatment has study the a ranging RNA nucleoside by we want the our This started in a inhibitor, we COVID-XX double-blind as April, trial Anthony. with of of part in to enrolling currently X, we

look in on selection Part informs is in galidesivir that’s COVID-XX that activity. forward you dose in X. antiviral how X progress demonstrated broad RNA our it’s adenosine to and we spectrum analog Part data polymerase rationale The We inhibitor for updating nucleoside what see an that and studying

MERS different and against than working also that the is additional data causes potential our partners SARS-CoV-X, test against collaborators In galidesivir RNA the vitro identify and to are testing corona X could provide of SARS. in experimental more XX SARS-CoV-X. And COVID-XX, viruses models that in with that families, conducted We’ve vitro underway. virus including against cause government we viruses animal

COVID the on that benefit data forward as end provide At And best the data quickly placebo-controlled drug from clinical of the as randomized day, getting looking for possible. information we’re has the patients. the will to trial

extraordinary is I where me wrap an So up in BioCryst position. started. let

within approvals berotralstat. data and million peak and clinical next coming led patients in demand sales to from forecast physicians of a north market months XX product. the X have The $XXX for have HAE this We strong us for

have early this our help In adds add additional pipeline we in a the today multiple And health to the in diseases. success our of emergency programs a a global value. address positioned to addition, program molecule data shared we antiviral are complement-mediated And XXXX. with confidence across and

disruptions Despite We I progress BioCryst, so investigators, are by collaborators thanking and at we without you. made daily world, in wouldn’t want significant of challenges the our current where patients our their team have own possible. lives. close be and around today the to who all thank this and you,

With turn that, the over to it for we’ll operator questions.

Operator

[Operator Instructions] is first Fye line with from the question Jessica of Your JPMorgan.

Jessica Fye

Hey guys, data. XXXX good Thanks had morning. couple questions. I a the taking for on my

First, for appear were not Was volunteer healthy after observed on X no suggested would you a even sounds the if think do hypothesis to in there you like second, why patients. And why the there have initially rebound that when data transient And first curious any fall you reticulocytes they treatment? might. rashes seen here I’m have rash? was

William Sheridan

Yeah, going anemic. and are for the It’s are while reticulocytes hi, question. active to Thanks subjects The elevated Bill. the Jessica. remain

see subsequent no the it mild we to come data and first and weeks you’d at with the X mature rash in normal the and we And as into stay no, the in So any expect see all didn’t rash the subjects. range. see rash, up, the come regard to hemoglobin

getting saw we’ll rash of similar see healthy compared interesting HAE. had a how to evolves. And Why? higher we That’s In a we program, it berotralstat where in question. phenomenon incidence the people an subjects

Jessica Fye

in The galidesivir Okay, there’s some that anticipate And are ask shown of open still How galidesivir think couple you’re reports indications any many vitro and sites evaluating data? also great. I when we on it coronavirus. release on sound in you well? saying you has just the the press And makes against I a should activity can activity? been like Have that. Brazil seen web current as early

Jon Stonehouse

Yeah.

get on to think have sites, confirm head, But back off So the I’ll I that of X. you. the top to are and my there

is regard are report it fully completed. there comment And that the Brazil. that And to isn’t testing, want is, we work I vitro With data. sites a in until don’t X SARS-CoV-X on will getting when we’re it fourth. and think working in to

Jessica Fye

And is for there clinical data? any the timeline

Jon Stonehouse

to Brazil is right a That’s pandemic now. hard one in widespread predict. The pretty active pretty and

patients. And so, we’re dosing

really, predict. enroll as as faster as one. sites and to part The to have really can we we hard quickly in But we’ll in we’re that it’s the to can. it be much we open We’re move Brazil, doing able more as

Jessica Fye

Great, thank you.

Jon Stonehouse

You’re welcome.

Operator

is from Gena next Barclays. Wang question Your with

Gena Wang

want Thank you to follow you cohort for this my for the patients cohort, PNH do I questions. just want And not to data, penicillin. the use taking rash question. confirm

William Sheridan

whole like against outstanding. thrilled infections of a the And, in with did of a this, the The subjects right. study, treat rash, we’d That’s through here, just first if it. get the in X evidence the we’re at we body not with this benefit here [nicirial] vaccination. Prophylaxis data that looking in serious [ph] even have is rash, patients disease absence

was [nicirial] penicillin, there [ph] no So prophylaxis with vaccination.

Gena Wang

And for trials Okay. the the not penicillin, new cohorts? you proposed the use all right, all for would

Jon Stonehouse

She’s future. asking the

William Sheridan

Yes, yeah.

other antibiotic I we’re about or of relaxed think co-administration completely drug the by way. any this that with penicillin the

use really If issue that rash people there us. So they a it’s can requirement benign. penicillin of reason, was healthy pathologically, them. We an need through was antibiotics and get the no And, figured drug to there’s and subjects, couple at we which clinically protocol But in whatever all. out treated for cases. a not for

Jon Stonehouse

use we’ll But vaccine… the

William Sheridan

is the vaccine approach, the Yeah, yeah.

Gena Wang

see. I

eliminate I likely would unnecessary – is think how to the be rash [ph]. [tuck-on I’m asking, based wanted any the there just And any penicillin. see data] reason to due

William Sheridan

Huh-uh.

Gena Wang

Yeah.

think asking. the reason – I So that was I’m

Jon Stonehouse

this incidence and a in higher a as see at And Bill We’ll we can’t fact as penicillin the patients. lower in with comments, healthy way we difference patients. the the of no X we phenomenon is berotralstat saw And volunteers we in mentioned HAE we’ve had PNH. that we’ve well saw as volunteers the matter seen healthy that patients incidence no, is Yeah, PNH the with and it’s The rash. in say HAE go. between that rash his

Gena Wang

patient do left only another side, X question. milligrams. that wondering, the you Slide XXX regarding have have patient X XX, data hemolysis, using then, AP milligrams just you that’s XXX on to Okay. And

patient inhibition had there. There’s of one increase LDH outlier rebound. have basically You X – hemolysis

And the right later. up then, when also patient the additional you we’re on on Weislab have using assay, side, showed outlier

if little that or color on bit contribute baseline additional on this anything give outliers, of you wondering color can hemolysis a the inhibition? Just could these more basically to more X rebound any

William Sheridan

Sure, a thanks this for the this comparing through XX because XX milligrams It consistency, with question. and the chart. milligrams. a hours we’ve is shows love dose milligrams I spectacular milligrams And assay XXX reason that dosing better really and the XXX XXX versus the data done way, shown after last we’ve regimen. the twice-daily

So it there’s all measuring the AP not assay with suppression assay. a whether the almost activity, way through or hemolysis you’re Weislab of XX complete in hours,

see Of period, from over levels great odd a the to where that individual results we’re here enough, will and it’s eventually course, next eventually system This chart. the get the not starting and in in the And as the drug will suppressing, complement disappears assay. get it there. to you positive

Jon Stonehouse

But twice-a-day so hours, it drug, it’s a up everybody is to XX which fantastic. covers

William Sheridan

Yeah, this chart. a is great

Gena Wang

Yeah.

I’m asking what terms that there? are QD how in like could who – to or of colors actually any are if that the other likely I So see think these patients the is be drug any – – baseline, a differences reason

William Sheridan

to to in be do we’ll data, we to might this MAD. – it’s And what to the out once-daily we’re think going able what with doses it’s achieve is know that. but yet, study dose, figure obviously us don’t exactly encouraging have which I

Gena Wang

Okay, thank you.

Operator

with Your question Tyler next Buren from Piper is Sandler. Van

Tyler Van Buren

XXXX next] potentially the ask guys. the It’s Phase taking days, and for I favorably. questions. other early to Thanks some But small Factor patients. PNH morning. It’s seem oral I the to initial make of see comparisons compare you data Hey, they guys Good data. of the to wanted exciting guess, [the to D. X number for [ph] to just course,

any And that the there question, that the clinical have in you noticeable order point do can guys confidence you incorporate in into what dosing, to study and in MAD then, second that program? Are you would on patients once-daily the see use differences out?

William Sheridan

bilirubin, we’re the right of The the data the reticulocytes, have X Okay, LDH, so, drops in first direction And say really, hemoglobin with I’d a first short all, and the happy in we subjects from rise. in going pre-treatment. period, where here. the didn’t incredibly really up strong to first milligrams PNH mention on call. twice-a-day. It’s starting in for it the I dramatically, clone size XX come the X has even subjects was weeks pretty this in fairly And is later. the on But pending all X low

good anybody in other studies, at And of agents. at especially is I people studies with comparisons very terms people But data to you’re are anemic at our least severely the direct at look with other earliest would who as as else’s, when baseline. looking

a So tremendous this is result.

Jon Stonehouse

would the where the Hey, one comparisons Bill, add of on start. comparisons. challenges See, I in is do patients

As Bill said in his remarks, these were really sick people.

compare. absolute percentage did fantastic change we think that number, is And I And of front. on I to important the at looking so, that think instead

William Sheridan

every dosing, hours of gives we to absolutely the reason that to want we’ll of what XX and XX milligrams test cohort. PNH every to a is position persistence great and milligrams And majority go and see or who do and in people And once-a-day – That through we’ll clinic quality understand dosing. to striking, get, include in be some XXX pharmacodynamic once-daily us good, had whether XX hours. that we then with a the in effect regard it’s hours XXX

Tyler Van Buren

Great. Thank you.

Jon Stonehouse

You’re welcome.

Operator

JMP Your from Liisa with Securities. next question Bayko is

Liisa Bayko

in of of Hi, to you versus quite attributes? maybe there. remdesivir similar, of through contrasting some sort talk they’re where and taking the differentiate; maybe you are other galidesivir comparing see about question. themselves terms opportunities go you Can similar and molecules kind where of Thanks for exposure just know the I

Jon Stonehouse

can the like the experiencing. a to Bill in then one combat that and normal seen where taking share needs start multiple like this and market the and we’re I’ll other another, areas we’ve from – let currently the viral specifics. Yeah, outbreaks This not weapons into to of the one product is market, government you’re get government in smallpox some arsenal

the around both galidesivir the so ability globe. to And have see in remdesivir stockpiles and national we strategic

to need – even heard room the officials be of drugs galidesivir. to polymerase inhibitor done. that Cocktails like another you’ve for needs and government need And plenty some to of from be done, studies more of RNA be there’s so

William Sheridan

it’s Yeah, world. to we In remdesivir to good the started And that good information And to really aspects, good very comparison I analogues It’s both we’re make coming it’s obviously, just remdesivir adenosine structurally, happy And that analogs. for analogs. not other in any terms nucleoside they’re data see of it’s think for emerging They on the galidesivir out positive. enough field. nucleoside comparisons. versus That’s good different. the both study. detailed for

Liisa Bayko

Okay.

Can terms line of pre-launch heavy your you maybe further terms are, for ahead plan activities in do down salesforce of HAE. talk of and how plans out what do much approval? the talking to about kind building hiring you of the about infrastructure, In lifting

Jon Stonehouse

Charlie?

Charlie Gayer

Yeah.

I So our as complete our in-office hiring team at this we’re are for is leaders regional team the sales mentioned in the thanks ready the question. sales for complete, point, And comments leadership salesforce. my were and – getting

that doing be we’ll QX. So in

Jon Stonehouse

the want And hit medical affairs to Megan, piece? you

Megan Sniecinski

KOLs. And deployed, in the my talents rare experience well actively we’ve from as perspective and we’ve encouraged a the specifically think pre-launch into a teams remarks, got we launch diseases to been I really of in that Jon, similar are as phase, their and HAE. and brought affairs sort medical our Sure, shared engaging Charlie, full the with team by

forward are just work and where later months a and do to about coming this he launch know today are excited really feeling in continuing I to important ready for the the and year. looking So I we

Jon Stonehouse

people and into tell came because Megan’s you can’t for have are in tell an they and affairs, Yeah, said I number can’t you really – of the drug come that we the organization oral one. important points have medical commercial HAE. I

tells you that something. So

Liisa Bayko

talk about with can manufacture kind supply track travel and there, thinking to and kind just you that curious just where are you of that’s just about drug they FDA, about thing? due all restrictions. color be how I’m inspections some And then on I’d and of scheduled tracking?

Jon Stonehouse

question? take the Megan, you to want manufacturing

Megan Sniecinski

Sure. Sure.

I things highlight. So couple to Liisa, – of so

again to, we us lot investment and throughout the think dual the a BioCryst work able chain early that do that before made I source has positioned fact the in COVID. of well redundancy were

what feel are terms we from So we of in track in on for great in PDUFA to and launch for a successful supply a like what the supply ample December be everything need got really perspective. and need we of date shape we’ve

Jon Stonehouse

started one virtual thing we other the to saw starting Japanese agencies. is place by with we’ve to even FDA, PMDA both that with see And are inspections take

So to. that’s encouraging

Liisa Bayko

does work? that That’s How interesting.

Jon Stonehouse

technology. Through

Liisa Bayko

the for real something. Okay, inspection or

Jon Stonehouse

Yeah. questions its and… you answering Yeah. of and lot a document Yeah. remember sharing Well,

Liisa Bayko

Okay. Understood.

just real then possibility. on to QD And a looks question, like Okay. follow-up Gena’s

when with – on You can have this maybe kind that? to Slide don’t then, explore might my really XX as And of you outline QD, last and speak you it is question.

on a XXXX, you working and other makes start might you as that? third a about when indication think indications, sense maybe For second so explore what as you. to Thank and

William Sheridan

Sure.

multiple ascending is the So dose study still healthy open. subject

one in So we way the And study unfolds. we’ll year whether looking the the or justifies and the study, to then at look PNH plan QD, it added of another. data decide as rest

Jon Stonehouse

And… The on indications. other was

William Sheridan

landscape indication is the So here rich.

incredible number and make can a the of diseases is this, the of specific things that’s lives like Factor inhibitor one D treat huge impact potent So is patients’ on the a that about fantastic.

for will meet the and the regulators as iga no CX and deposit nephropathy field of we with those program. a So, various in develop dense glomerulonephritis bunch disease, property. the of nephritis. things are they make are There example, decisions It

Jon Stonehouse

this not very PNH of all multiple diseases. data, you excited And have it’s clinical data we the talk development well see And that indications. just we about Yeah. complement-mediated do that offer program we’re with about excitement a we could the around when broad the

William Sheridan

particular the is other others the nephritis, easily the all right. I are. of scheduling data And in hemolysis, PNH currently None that can by is marked that Is There’s PNH and see we once between with the diseases. have. day difference one a the

Liisa Bayko

answer Very for lot exciting. question. a Thanks my

Jon Stonehouse

You’re welcome.

Operator

question Your with from Brian RBC next Capital. is Abrahams

Unidentified Analyst

had AE on for died, hello. transfusion. question I can the D the patient post also the trial the And impacts might that that required how out can clinical us viral pandemic? if And terms Factor middle to remind in it if patient also still they This days, Hi, the Brian. is of had curious, on another just drug. just drug I’m were XX increase the where recruits of play they so Leo and profile on the same you of does, in infections? inhibition the susceptibility

William Sheridan

Sure.

had healthcare and had had patient, a this chickenpox who was unfortunately never vaccination varicella a never and worker. had So contracted

So this that Chronic number corticosteroids the circumstances getting disseminated is corticosteroids. for factor and of combination is taking an unfortunate was person one risk varicella.

congenital Of disseminated any at with sort course, deficiencies. including varicella of literature complement of extensive we’ve There’s deficiency. not searches, Shiga congenital case and done around Extensive very boy a complement extensive this, was not diligence looking a single uremic a but reported death, eculizumab. case searches There had eculizumab. recovered, is and hemolytic who from syndrome varicella toxin getting literature got around young of who one

whole looking FDA corticosteroids of mofetil. adverse extensively who system, come but of that we’ve in then a was specifically, event the there and And variety is And getting at eculizumab. also one course, reporting individual drugs, mycophenolate through

corticosteroids, want So all of very of play to course, here this evidence we the towards points safe.

make we’re are immune So changing to people sure protocol that the against chickenpox.

Jon Stonehouse

as his question in week. Yeah. patient, a recruitment, last had coming we And Bill to then prepared your remarks about said study the X

So there’s enthusiastic build it of about investigators really don’t lot impact expect recruitment the the drug all. to still trial, at and still are enthusiasm and we the a

William Sheridan

the damage summary that says if in inhibiting you doesn’t up seriously that’s here. of system that. that the lymphocytes So sets system And all do the risk complement complement what – diligence

Unidentified Analyst

Okay. Thank you.

Operator

& from Belanger Instructions] the Needham of Serge [Operator question next Your is Company. line with

Jon Stonehouse

one. to want They the next are. Whitney, may hold to go on, Serge? we Hold on.

Serge Belanger

A button mute was questions on of couple my friend. The not XXXX.

on the and think dose in that milligrams I part compare doses XXX? healthy the does XXX XX, sustained volunteers. multiple the reported, lower the of and ascending pathway over at that How the trial you Slide you of of dose XX suppression XXX Bill to First, and achieve alternative XX%

William Sheridan

at look consistency. the chart, matters we is if the what So

So if pathway activity XX assays. at a in X% you are and than of look alternative at the see have the more individuals XX-hour at individuals couple who XXX, as there residual those that you time point, many can

here milligrams. XXX% is XXX exactly of and want is what having you which what subjects the at get achieve to we So more XXX than XX% suppression,

Jon Stonehouse

the And on Bill? that, slide number

William Sheridan

XX. Slide XX, yeah.

So the it’s of all effect. consistency about

the the second of make call is on persistence clock in important because mentioned here especially you beyond already, the a as that PNH, effect times the is, So of coverage. sure hours want couple we to you around XX have thing

Serge Belanger

an talked Where contract? And million here? are in Okay. And any BARDA Brazil are NIAID. to you that the and there then gets you on vis-à-vis contract of $XX about efforts galidesivir, with that underway expand span trial as ongoing that the

Jon Stonehouse

money. we’re total halfway through So about that

consumed contract. And the contract it’s NIAID look to add proposals or the of largely to additional to a new think, Yeah. than course, more opportunities side. other I government we’ll continue money, BARDA to side get on to at the this either

Serge Belanger

for as you And of then, the key I one on label, negotiations aspects as aspects well, label? of thinking APeX be are what Charlie, guess, label berotralstat berotralstat data start for see the that just to Okay. the key looking you’ll the a want of you’ll for

Charlie Gayer

Yeah.

So thanks question. for the

obviously, we a in the marketplace. that’s submitted label we’ve have consistent other that’s, a to products we So kind draft and the – label of expect with

so we’ll on far, based get. we’re in seen what we’ve and we’re that’s what so, And confident

Serge Belanger

Okay. Thank you.

Jon Stonehouse

You’re welcome.

Operator

of Your Maury next Raycroft from with Jefferies. line is question the

Maury Raycroft

transfusions just that, questions. on patient morning, may expect good for a transfusions And fewer treatment. one everyone. patient Do taking one that while XXXX patient patient at the looks and on I thanks quick Hi, need as the baseline getting like It X. stays my on had was for needed time XXXX. over

William Sheridan

at day on like patients that the XX X.X clinic or we’ve X, site. the have relayed to made now transfusion risen had from hemoglobin Maury, a comments the has week Anecdotally, when visits, investigators post-transfusion XX.X. something by had principal individual and the of That at

they’ve is ever felt. this best So the

the not got you approved. in that to are So remember South inhibitors Africa, CX

drug, to really, but they of on anecdotal, fact that disease – at low the symptoms doses hear. not that are And fantastic the feel encouraging. the seeing the is really yet great we’re optimized it’s And is So which have been it’s severe. this really

Maury Raycroft

it. Got

then it response not other X, Okay. as patients. efficacy robust was for And the as seems patient like the

type related the dose anemia, baseline, – aplastic that’s if wondering of to Just and this patient patient and could patient’s get overcome respond the higher better? get to you the think

William Sheridan

aplastic a really that’s think patient PNH. a question. and anemia This combination, I does interesting have

be are to where to that But the extension in patient respond to to – these it’s marrow hemoglobin bone the escalation of those who the who under predict ability is subjects we to more continued going can’t the but limited go for dose also going the phase cohort X. amending allow And I has So are completing ultimately. going protocol regarding rise. circumstances, are

happens opportunity what will get the we see to increase to when So the we dose. be able

Maury Raycroft

any have salt new new Got Will patent question, commercial the be bioequivalence the berotralstat. it. the And with you of forms quick And just testing done crystalline the regarding last new for in forms? the setting? forms used

Jon Stonehouse

API. It’s the

automatic. the it’s formulation So in we commercial so as and speak, it’s

Maury Raycroft

it. Got

Okay.

thanks my question. Okay, for taking

Operator

Barclays. question final is with the from Your of line Gena Wang

Gena Wang

Thank you follow-up. for taking my

about I headache think to I forgot question. ask

of the class, so had X saw moderate the weeks] had this is for headache. So X, [aX like since likely drug subjects we the [ph] We X headache. all understand

can a the onset, color the days, resolved. Just a it was bit, and regarding color wondering you more X you a regarding like than X if on less lasts long give little give little onset, bit headache can how if to little it more and bit you then mention did how

William Sheridan

day X that this to onset the when two. in days. control reason get hours, you scavenged. in or first is few couple people out than X, because immediately means The every complement is nitric single that starting a to less the individual, oxide is these of release because we’re Sure. what’s headache inhibitors, duration quick from start is And really they being hemolysis, about a very The striking

this the out. into the details, investigators in have going And the all field without worked of

top the class effect it of quickly. disappears of that So on with intravascular drug getting a and know you hemolysis it’s very

Gena Wang

So, drug to other alleviate the any headache?

William Sheridan

that So complement investigational. this been and and with are other inhibitors eculizumab has Ultomiris, reported previously

Jon Stonehouse

a it’s going So sign to work. that’s

William Sheridan

is in found is we yes. that is Exactly. vivo And too evidence working, that it what drug then, the

Gena Wang

Yeah.

any do mitigate part, say, drug need So this to but anything like, that understand any I like or you headache? prescription

William Sheridan

Tylenol. the is this No,

Gena Wang

Okay.

you. thank Okay,

Operator

remarks. I at am now questions Mr. back time. further for showing no the Stonehouse I this turn to any will call closing

Jon Stonehouse

Thank you, Whitney.

PNH. – revenue generating this X to are gives the year, that the that that that beginning, drug in we diseases have X more generate next can excited a As that beyond proof across be couldn’t this with about. of we launches, at company have with XXXX coming board be data And year, starting starting I complement-mediated we them the said transforming we real used

that pipeline, with add a a our be So the the to couldn’t opportunity to a tremendous and better spot. global pandemic, we we in just in play role then got antiviral,

we’re so, And sit. we about super-excited where

we approvals. hard working get to along And really continue be launches and keep the the programs to ready our going for posted will We’re move way. to for you

your safe, great day. and interest, stay a and for have you Thank

Operator

a Thank and you Ladies today’s conference. participation for you have and day. wonderful this gentlemen, concludes

may all disconnect. You

BioCryst Pharmaceuticals (BCRX) 6 May 202020 Q1 Earnings call transcript