ALXN - Earnings call transcript Q3 2017 | Alexion Pharmaceuticals, Inc.

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Operator

Good morning, everyone, and welcome to the Alexion Pharmaceuticals, Incorporated Third Quarter 2017 Conference Call. Today's conference is being recorded.

For opening remarks and introductions, I would like to turn the call over to Elena Ridloff, Vice President, Investor Relations. Please go ahead, ma'am.

Elena H. Ridloff

Thank you, Rebecca. you performance us XXXX. for thank to today's and Alexion's discuss the morning for on joining call Good third quarter call by led will our Today's CEO. Ludwig Hantson, be joined Officer; and Orloff, Financial be Ludwig our of John Paul our Commercial Goff, our Chief will Chief Officer; by R&D. Brian Head Clancy,

on slides will the our of Events presented access our this on page website. can You that call the section Investor webcast be by to Relations going

a GAAP be you point non-GAAP provide after for we that performance. to I'd will statements, press making our Thank guidance ongoing begin, take results no update financial any will forward-looking the to remind These statements I Please that in discussed like apply Before Reconciliations not of our certain be our included substitute could uncertainties in only believe we considered non-GAAP we which materially. business actual forward-looking but to the These duty our these results and to out and statements today, involve factors undertake like SEC for our look as statements of to measures using and the for also useful financial we detail. in differ at filings financial understanding Ludwig? are measures, addition you. release. of that a should additional results. we would be of the risks risk cause call. information and financial

Ludwig N. Hantson

their myasthenia more as accomplishment FDA This for saying Soliris is reality, tremendous teams our review waiting a to for for very a want start been by to trials. for this significant to make and and milestone application for this the you, I'm with for thank cross-functional approval a Alexion. treatments. of Thank and the patients is I like long investigators' Elena, families have time the patients their families in with today's thanks the joining But Alexion, a generalized treatment new participated at who I patients us morning. importantly, that everyone would gravis. a this FDA, to and pleased for our call worked who approval

discuss will the later call. more in on gMG detail We approval the

to performance, X. now on our shown as Turning QX slide

advancing quarter, we and bring diseases. mission made to the to During positioning significant deliver families progress, on growth to long-term the our by patients its hope company And rare objectives. affected

Recent serving momentum execution X% commercial volume growth revenue financial driven R&D despite Continued generated strong of organization volume Soliris, ALXNXXXX includes again highlights delivered once the on and in year-over-year continued rapid XX%. enrollment EPS commercial in and of and studies the of of including by X%, performance our non-GAAP of growth continued quarter, growth Strensiq. patients, growth more

In Soliris MG in Germany. launched our addition, team

for we achieved with more which the patients EU, in in team FDA We're earlier R&D week. this the received label refractory detail. John discuss approval and gMG regulatory approval Our with very pleased will

subcu. the our delivered and with addition, pleased highly a accomplished refocused ALXNXXXX progressed plan we Phase Soliris respected the with aligns And and performance and third our greater with other strengthened and Phase experience ALXNXXXX to in initiate with streamlining work that our X, and we Naïve leaner with protect global By executive Friedman, creating extensive added a completed issued to year-to-date. studies Switch R&D our PK-based We with biotech and I'm portfolio our in board study We X in new IV operations, of These PNH our a third Paul all exclusivities single directors. Soliris, In accomplishments patents indications. patent the U.S. regulatory restructuring three quarter XXXX. patents financial strategy. concert we enrolment extend our the in for organization organization flexibility. patents are announced plans to the into that protection in patent new quarter of

Before it our guidance to next for slide growth our as discuss phase Paul of reiterate XXXX, results drive updated the I objectives including shown financial over the to four on will I that X. turn want to

continue see meaningful to to disease rare our business. we First, opportunities grow

we our expertise core Second, and our therapeutic development on on will areas. focus research complement

will pipeline flow. our expansion, fourth, we Third, build and increase cash growth deliver through are disciplined indications. margin development business complement we EPS optimizing to and our And additional infrastructure

and Our well beyond. results remainder quarter third us the for XXXX position of

shareholders. to turn review strategy our are Paul? generate by build We strengths to increased quarter on third executing will value the of position long-term delivering to on our over with now it clear goal in great our to financial I growth for a a Paul results.

Paul J. Clancy

revenues Starting with grew reported million Total in slide and Ludwig. third $XXX were X. X% the year-over-year. quarter Thanks,

As we due had quarter previously and lower growth a quarters factors. the communicated, this two few forecasted than was to previous

growth benefited $XX approximately of quarter-over-quarter year-over-year second to First, quarter orders, for the quarter. impacted this from million and due directly which timing

this trial than as X% was first pricing of and impact in welcome although year, approximately versus a increase had And, clinical that a headwind increased. the impact headwind enrollments This million first from half we continue we of program. to is QX, was lower a versus half the the other this the advance for year. third, million slight anticipated. estimate QX $XX Second, ALXNXXXX approximately the in $XX We

year-over-year. per earnings strong quarter $X.XX third non-GAAP share XX% a of grew The

X, in to timing by of of impacted Asia Moving Rest whereas our in was growth slide were region, the orders. driven Pacific sales Europe, U.S., the World the the product growth and by

hedging period last X% of can compared You we FX volume, X% to inclusive year. the partially less delivered increase of in than pricing same see X% a headwind headwind of a and by offset

XX. to Turning slide

trial growth year-over-year growth the other was Asia ALXNXXXX to Pac and from volume was X%. region. in and Europe geographic year-over-year Soliris revenue enrollment million the approximately largest was headwind clinical in an with X% quarter $XXX and the enrollment impact volume

specifically, continue ALXNXXXX in to few see the underlying which when number what with we More last Importantly, over quarters, five quarters. QX away a volume maintaining when Soliris for in the patterns, for XX% around you strip the volume adjusting pieces we've moving growth consistent ordering seen was growth its momentum. is of last

on slide shown therapy, continued work Growth changes on of $XX we dose value momentum by our payers the well Strensiq patients. strategy continuing patient in with reported revenue was from million impacted had pricing as As U.S. additions. to we to we as existing new align for higher as better patients as the XX,

$XX revenues Looking at million. of Kanuma, we achieved

Turning to margin on XX. operating slide

to non-GAAP and due total expenses. from Non-GAAP revenue. cost a investments was XX% quarter to XXXX of terminated During or of margin the of in increased $XXX programs on from compared decreased R&D announced was savings offset deprioritized delivered quarter, revenue operating focus million ALXNXXXX. The we XX%, operating benefiting growth the partnerships, R&D and third spend by

this from in XXXX. quarter. of earnings audit quarter non-GAAP and and QX build includes to field tax year expectations. XX% dedicated was This preparation for gMG expense the the is routine $X spend in conclusion expected by of benefited spend year-over-year due This in the for drug the the of was revenue. share Non-GAAP R&D per Germany teams associated XXXX million the QX. $XXX timing benefited in IRS approximately of to XX% batches million, of now total Additionally, launches with the to our the of in and effective Japan. in years approximately SG&A the rate The compared increase contributed clinic The a $X.XX U.S.,

$XXX and revenue favorable by XX% related of majority be the in a EPS million we closure the and in of full facility year-over-year, the and cash planned XX% now in $X.XX tax on XX% Rhode XXX,XXX in and marketable the expect were and of Of million in cash which GAAP generated with $XX associated of XX. XX% the year, expenses $X.X We ended be repurchased cash free outlays. to including an over QX reported quarter. growth approximately in XXXX. quarter, in employee XX% is to million the we States. outlined shares securities, and $XXX driven expenses these recognized costs. In For rate United Non-GAAP this quarter September Island largely $X.XX with the and as $XXX will XX% billion manufacturing expect the million, grew separation our slide rate. approximately between XX and flow We restructuring approximately tax increase benefit, The to EPS of

and to mind as billion slide in walk to through headwinds move $X.XXX midpoint, of I'll Total of low Let's $XX year-over-year. billion; billion and revenue we up expectations million updated discuss $X.XXX the of range. guidance increasing to impact XX represents of the other now includes of and full-year a turn the $X.XX are ALXNXXXX foreign keep range items few now trials to guidance $XX moment. million total billion, this Soliris, the for At we're currency to revenues from in estimate an a growth be prior on forward. end the This between our and which our revenue effectively XX% $X.XXX to expected

Excluding these, growth would approximately revenue XX%. be

extension aHUS that ramp billion studies to impact continued from XXXX, assumes growth billion. well XXXX which revenue do Soliris $XX underlying of low PNH within For volume the is Soliris, XXXX. believe these ALXNXXXX $XX ALXNXXXX in our we're to This of on incremental also briefly targeting now. million ongoing $X.XXX we as Similarly, It from revenue as other until a of we've the million trials will through the regulatory range. remain which increased revenue end To studies, to half for assumes given patients globally. first the expect the guidance the fall with headwinds and PNH in touch we of $X.XX studies, will and Soliris decision enrollment

in limited to due our Phase In the launch, timing initiate addition, With in subcu QX XXXX. the to in X given regards MG year, contribution some MG from the we to we study XXXX. a expect incremental limited plan expect headwinds

to Metabolics. Turning

expected assumes increase fourth the be Our due million sequential operating to guidance anticipate expectations GAAP a earnings to congresses, the timing XX% operating rate end will quarter mentioned. expenses. $XXX low at the from restructuring to of partially medical related unchanged. revenue guidance margin Non-GAAP XX% tax range. is prior per be which and well effective XX% savings to now in We increase also and GAAP margin reduction of with as seasonal $X.XX and the drug of due is restructuring. XX% expenses. expenses XX%. batches operating is the an non-GAAP a $X.XX guidance related million between our to is restructuring includes to offset in of to $XXX the This XX% previously the of share clinical versus as The I by

share guidance mid-point of the is earnings the guidance $X.XX of our $X.XX, Our represents range This increase $X.XX. growth $X.XX to range. to non-GAAP at guidance EPS revised over prior XX% per an approximately

financial to will over XX note, to experience Dr. to XXXX Senior slide as global we provided institutions, revenue ambitions the the this call XX, Brian. include science, Turning EPS Strategy. turn month Aradhana a to and excellent XX% the transactional experience. investing over On Vice greater simultaneously margin of in brings These professional Corporate us XXXX we're of and and for to has slide development. working joining an Aradhana plan restructuring growth. leverage objectives through years operating these sector business anticipate at I'm the in I'll Development double-digit understanding on bottom growth, line, on the growing We strong non-GAAP line executing disciplined while as financial be pleased top our announce now deep delivering of Business orientation shown including the Alexion on last towards XX. Sarin that pipeline, President in to to from leading

Brian Goff

I'm Paul. starting performance commercial slide our in quarter third on to XX. in you, Thank with Soliris report pleased the

with and yet patients on patients patients a and growing we're believe enrolment to education despite initiate aHUS, starting In continue operations aHUS focus. continue have number to majority to still us Soliris of trials. PNH, treatment. that new more with patient ALXNXXXX longstanding patients rapid In in of to commercial new serve Soliris we on disease seeing PNH global a our identify the PNH the Our remains focus the enables and with retention

and of patients for for with understanding optimal programs our we Our amplifying with further treatment conditions with target disease complement aHUS. specialties potential continue education patients to high

also pleased the patients We and growth aHUS trials opportunity both to and even aHUS. Soliris with we in continue into the continued QX greater ahead simultaneously the very than in even is performance as believe PNH PNH. year-to-date We're ALXNXXXX and us that with enroll expect of that of

XX. slide for Now, launching moving to to this gratified patients generalized on gravis be with myasthenia chronic We're debilitating and neuromuscular extremely Soliris disorder.

the this these Starting the U.S., This earlier with treatment the week, years. than Germany first patients in Soliris of for gMG more is focus be approved initial gMG FDA Our are FDA the the approved will treatment launch adult in for XX who for antibody and U.S., with the Japan. positive. anti-acetylcholine patients

gMG. In addition, first Soliris complement available only and inhibitor treat is to the

the continue total to unresolved focus on disease trial, gMG AchR be MG patient REGAIN the who studied represent appropriate to Our antibody-positive response with therapy XX% patients and patient inadequate Phase X% had will suffer the population which approximately in symptoms, of from significant population. immunosuppressive to X

care total XX% complement-mediated respond community so do will XX,XXX slide through As in to treatment. field suffer from by XX,XXX the a just to shown weakness and inhibition with intensive the patients study, crises existing neurologists, talking, and the now of of XX the They're Having profound neuromuscular over estimate severe be the role swallowing, that the team returned They and may AchR and gMG staffed, launch in be many also in are at on we difficulties who cycling To estimate life-threatening. of the deployed and fully despite neuromuscular observed risk breathing. Soliris. from XX, I the will hospitalization population on the including often exacerbations of MG specialists, the and, muscle in patients Based may benefits that of specialized complement opportunity require patients approximately U.S., increased therapies, serve most as that Soliris dedicated junction need our X disease to in continue field. respond can symptoms urgent and is we including we reminder, say years. now, critical is and Phase REGAIN disease gMG with that patients what treatment, U.S. to team that to treatment also the and about of excited on meeting, weeks trained, our antibody-positive educate limit walking, These destruction of function,

Our expectations another for treatment are that are been waiting patients option. initial there who have

be verification, can to weeks. vaccinated which prior patients as However, well through scheduling infusion need as several treatment, benefit and to initiating go take

the XXXX. in year Soliris timing largely expect with on or to the the gMG later would year, Given this U.S. we patients in start early

Moving for Germany, supplemental accepted by Labor, the a in refractory And initial early for August review following the gMG Commission launch refractory approval decision the patients Japanese Ministry Soliris underway Welfare, Japan, antibody-positive to new our expect gMG. and in XXXX. was by for is European and application AchR now Health, with in we in drug the of

QX, Turning HPP also Germany, Strensiq with starting now to to expansion. geographic we our patients metabolic and therapy and to continued with on new the U.S., on slide focus XX. Japan in In identify continued

funding will in HPP with have starting most national onset to with NICE England, access patients the treatment NHS November. in Following in pediatric and England need of Strensiq agreement

similarly third agreement an patients reached France quarter in also cover that the all We of ages in will England. to

that other transformative patients countries also go into ensure to of also access agreement we're the expect diligently We XXXX, HPP benefits by the with France Strensiq. to first have will funding effect and the of working quarter that in

on having target patient are continue higher elevated additional hypercholesterolemia partnerships in population levels. expand drive Kanuma also at NASH/NAFLD to likelihood such an ALT with establishing We're LDL lab as at that an increase to LAL-D XX, who testing. and familial those we and enriched testing for Looking to have lab slide

John team's reached we the Italy an into in of commercial go the which year. outside beyond. with for quarter and XXXX to the overview very report from over with in QX, an this treatment remainder infantile-onset to highlights patients third LAL-D effect pleased the addition, agreement and the continue we now call the of we're are I'll And growth well-positioned to to performance later I'm turn quarter. R&D of that of John? U.S., will in believe pleased In on the deliver for the in

John J. Orloff

antibody with I'm proud complement of approval and Brian. of Soliris to recent deep treatment for you, anti-acetylcholine Thank biology FDA patients the the receptor understanding Alexion's EC that gMG. of positive, led

therapy our As response will receptor evaluated to positive the continue suffer population study, REGAIN and unresolved in which patients focus acetylcholine appropriate the inadequate on who disease immunosuppressive from Brian patient mentioned, symptoms. to were had be significant antibody

higher want Turning highlights responders the the XX REGAIN which in Soliris MG-ADL prescribing difference QMG significantly and to robust and the XX. with clinical few change therapy MG-ADL. QMG first trial the was a scores, The total statistically favoring of comments to showed of proportion to FDA-approved significant from data rescue baseline Soliris information clinical includes for placebo compared a Also the to study, compared moments, note, label. slide make I to for the placebo. no experience both section on, from from including week of

and endpoints clinical placebo. for of proportion both For greater was higher consistently thresholds, Soliris the responders response at also to compared

the recognized of anti-acetylcholine neurology patients XX, with in the disorder, PREVENT trial clinical report We gMG. a in pleased expect Soliris supporting to Also, enrollment benefits of with spectrum the data or are now X the slide and that optica multinational in antibody in eculizumab FDA NMOSD, neuromyelitis Phase patients relapsing positive receptor for completed comprehensive single have data on Study, placebo-controlled mid-XXXX. we

this attacks on showed small XX severe that reduced reminder, is patients results relapsing NMO. trial based in with study Phase from patients a As a X of in frequency a investigator-initiated

Beyond potential innovative, development CX the are of our to rapidly patient innovation, and needs. has progressing next to drive ALXNXXXX, Soliris the we address antibody generation continued important that

in have to exposure. now now XXX and the patients development Turning ALXNXXXX of XXX years we about enrolled program over slide XX, patient

Before this congress. will that registration additional to Phase at ASH trials, be discussing mention X/X ALXNXXXX wanted year's data our I presented

experience modeling in discussions and and the XX reminder, regulators. determined data complement dosing studies, Phase the empiric development, on with study of X from designs data a PKPD were robust based Phase years As our X/X

and – we both Switch X intravenously both administered the study trials, ALXNXXXX every PNH enrollment X study to PNH the have completed Phase weeks. which Turning Naïve in the

We the treatment-naïve in from aHUS of atypical Ludwig continue study in enrollment in adolescent half studies expect XXXX. as inhibitor patients XXXX. complete we quarter approval to in in mentioned, in first these the of are we X the and an and to Phase complement On data early second also and XX, XXXX PNH enroll targeting patients slide adult expect

a we children study aHUS. with inhibitor enrollment in X complement in addition, atypical In Phase initiated treatment-naïve

data X we Turning encouraging for which developing with To concentration provide higher of optionality proceed registration process in Based and ALXNXXXX with are to and are discussions PK even tolerability to a also the more subcutaneously. FDA Phase EMA, XX. planning patients, formulation the a of slide delivered we we're designing. on and trial,

We expect X bridging evaluating PK-based registration of support available Phase a ALXNXXXX, to initiate to a aHUS, single formulation both weekly study PNH subcutaneous commercially and utilizing a in device. atypical

a Phase volunteers. a our As single-dose reminder, was X in healthy study study

are and to drug in late We completing additional study a work initiate Phase X combination XXXX. device

R&D we'll build drive greater strategic R&D stage efficiencies With that and ambition build briefly we and to patients future pipeline, place, update and calls. business on to disease Our on to that new diversified establish care to a strategy I'm with returns, our is disease rare PNH rare in complement, a confident by Touching that first will, progress core beyond and on ALXNXXXX grow leadership our leading on and the forward for parameters the be ultra-rare and opportunities expertise execute able on in an our look space, clear our I evaluate to is of R&D and all, a of disorders aHUS. as development standard providing disciplined portfolio focus strategy, risk. have expand

Now, back Ludwig? to I'll Ludwig. turn the call

Ludwig N. Hantson

Thank you, John.

path the innovation leader today's I'm Alexion on rare to confident me saying by for be diseases. we're deliver and Let growth that sustainable the conclude global in to right call

updating on a you and have calls. deal, our I look We accomplished to future great progress on forward

performance. dedication to our like thank for their would I always, employees As and

questions. We call will now to open the Operator?

Operator

and Ladies conducted the be session gentlemen, you. electronically. will Thank question-and-answer

and Our Cowen first comes from question Eric Company. Schmidt at

Eric Schmidt

therapy? you queue for Germany is Thanks is Can for on Do on in it where in drug Thank a patients you. gMG approval growing Maybe drug you the are seeking Congrats Europe. already the of available? there question. the who just the a patients question on myasthenia the have gravis. launch Is characterize reception? some Brian or

Brian Goff

for Hi, congrats. And Sure. the Eric. thanks

imagine, as case approval. have who FDA-approved XX to in important is the really for not really you not – we're had they've in As in mentioned years. can the have of the very new comments, patients had over the excited And I U.S., treatment a prepared option this

it's statement those a So patients. quite for

in German launches look we've late end one now pretty early to launch specialists. Europe, will in mention, neuromuscular our particular course, launch the As trained. terms of They're early our then, this sense we'll year so field Germany, And far, you and are for U.S., didn't the U.S. trial in And unlike deployed so They're into year we uptake should probably Germany, we in and the so in too comment Japan is year. give engaged. Germany about as like. highly awaiting They're team. next of to have a I early be ideally clinical talking this make that focused the what experience. far, approval It's to that any then, in have of

of we're terms the have thought of U.S., in a example. to that'll we limited who the first-hand might And number with than likely for expect lead uptake slower product. So, in experience leaders the

Eric Schmidt

Thank you.

Brian Goff

Sure.

Operator

Securities the Thanks it back all as maybe which all the Credit for helpful, Alethia was talk think bit volume around seeing kind a a XX%. America? basis, taking trends, there of up. Credit you the I Young particular when What his Latin my on was and stuff, from in can more year Paul you're how And I Thanks. guys. trends little Alethia you geographic throughout holding are the Suisse. (USA) out But Suisse know hear Young - the execution Hey, well. next, script, Congrats had in LLC on we'll with question. around

Paul J. Clancy

Alethia, what spot around with impacting probably across think the probably kind kind – that respect as the is I actually well. affiliates of in America and dynamics our And affiliate macro terms performance, the the is down, question. soft I are for world. we think trends the us thanks to noted common the whole of Yeah. it's Latin we of When think in ordering dialed sector, around you

of there, probably what which we've over So, future. seeing global we're I overall those be – what countries won't trends growth the seen the on last think a probably light is, global in countries volume – five, that But countries, will maintaining some that, had kind seven in of basis, six, growth been even of quarters.

Operator

And Meacham hear with from next, Barclays. we'll Geoff

Geoffrey Meacham

Thanks front. with guys. the the I'm ALXNXXXX would lot morning, But progress for be subcu impressed guys IV huge have Good a for the obviously, dev you quite and question. it bit some on a the been to And talking portfolio. diversify about biz time.

So the Thanks. point timing? expectations? the the question there XQ a a of In data is, approval tipping decision? MG upcoming for is in it to influence Or other ALXNXXXX trying seller your matter does manage in is words,

Paul J. Clancy

try this the Paul, Geoff. Thanks to take, me question. Let is for

to we're the respect disease the an ALXNXXXX we as it sense outgrowth ultra-rare of feel towards need of we tied rebuild a have to moving No internally we've in period commercial capabilities it. what we itself want of over before this It's to it, is to rare despite beyond on we'd out and the great the still understand need, do good se don't business to pipeline. X have I'm programs characterize capabilities progression prioritizing Phase the are for build into our talking pipeline of time. within focus, that, I certainly it that we've get areas mean characterize of we would efforts in we flow leveraging We number that building little do I launch in lot be a of terminated X, And from and that of believe the strategy, alone. But about pipeline development of I the I pointing certainly still of about executing a as it, a out, development talked we kind that per staying our ALXNXXXX. think think bit days. think I'd actually a a in a to our just XX that Phase a I disease trying head doubt about terms and begin capabilities – comes weave kind in We'll group. to I we even long with set and kind last new we in. more think to rare, hired transactions. team assets, to of while of able been

Geoffrey Meacham

Paul. Thanks,

Operator

From Terence Goldman Flynn. Sachs,

Terence Flynn

Hi. Will Was and details experienced maybe bioequivalence. wondering of lot. you naïve wondering a the just on taking remind to Thanks mix of Phase more if subcu for of us Thanks any just going ALXNXXXX requirements fixed? could the Is give us questions. the a kind trial, dose weight-based it for the non-inferior enroll subcu X patients be or

John J. Orloff

Thanks is the John. This question. for

both dosing that, will EMA regimen. later at design, is as XXX design, So, for the for we study and on later the The we the initiate we recently the which FDA which got will clear back trial single a feedback from XXXX. required bridging disclose PK-based study details and patients in registration, roughly to to of a XXX IV comparison a in point be trial

Ludwig N. Hantson

be bioequivalence... it But, will a not

John J. Orloff

That correct. is

Ludwig N. Hantson

profiles ...approach different. subcu the PK and because are IV of

Elena H. Ridloff

please. question, Next

Operator

Geoffrey come from Leerink. will Porges And that with

Geoffrey C. Porges

Thank again, approval. on and the much, congratulations you very

a questions Just follow MG. few around wanted up to

though – indication Given to you right an as other it you importantly, this when the a more and numbers are be ALXNXXXX you might then, it that MG indeed we way. are have at give than U.S.? new other the pivotal as concessions introduce make or Presumably, outside price And about But just could center the that you markets indication it might U.S. front the in in thinking in look payers start you should as in trial? in study you some you have any expect sounds MG? and potentially we're populations now large sure patient that's of to lastly, as those going studied that that be to and suggesting, PNH the

Brian Goff

very Yeah. It's a it's important enthusiasm few for important for Hi, for start We say as Brian. again, Geoffrey. I'll different approval well your that I'll one gMG. with again, very thanks maybe and and, as well. And in think obviously patients a parts,

As a the assessment. be factors kinetics the shape There everyone difficult putting that launch or number the is we'll anchor always to in into that knows, on going trajectory of different of curve. early it's a

First inadequate antibody-positive and were therapy. is as know, our response mention be which, I the population the and had gMG patient who REGAIN an focus to appropriate on will immunosuppressive AchR you patients study

So, you XX% call about that I estimate forward, X,XXX to we total population, you in to of the math, an talking carry my population. patients the patients, that and U.S. on forward we're the comments, in And to anchor carry X% when the XX,XXX mention MG prepared that, U.S. X,XXX opportunity then that's XX,XXX as to of

that'll our opportunity. do focus. it's So, a significant We be think

on the are as Some the in curve the the the factors cohort go insurance will been we takes of as need about there calendaring itself. consider logistical verification, look like of vaccination, to the and launch when the on of do other including of waiting infusion for patients steps benefit one initial the And course, of through, believe talk treatment. what an scheduling that hand, uptake, is there have timing you hand, the is, patients the to of well the terms of that puts will who

price, and many around some Soliris of question we mention, take of time. of indications I recognized. as across what where is the them obviously, customers as to benefits will possible, those as in conversions And markets so, Soliris of ambition, about then that is your moving as the expected quickly of to value patients. the course, we're to facilitate to And has the get The focused into of those educate help portfolio on that the terms

then, the we'll I've continue countries different looks support as And initial going forward, what the well Germany are U.S., assess and the So, of mentioned economics as of Japan. willing cohort that for the uptake like is as value to countries to Soliris.

Ludwig N. Hantson

So, reason will pricing rest have we The pricing focusing we're this ALXNXXXX, on strategy that's the basis. countries very one-over-one MG... going strong a undermine on and then, and strategy we're for – And at that Germany of discuss to Europe point. Soliris not a the the

John J. Orloff

and PNH execution we're course, on aHUS management regard life ALXNXXXX, the With Yeah. to the trials. now, cycle right for focused of in

that of year. the MG We up disclosed considering of with clinical And with of proof-of-concept number assessment two be in a be the position ALXNXXXX for gravis this the share will in have we'll and different more of also scales, a evaluating LCM to success program trials coming across our Soliris we're clinical course, in the XXXX. opportunity we plans significant and to impact myasthenia

Ludwig N. Hantson

But be will – option. certainly, be great MG treatment would for version a subcu patient an a

Geoffrey C. Porges

Thanks very much Great. guys.

Operator

from Harrison Morgan hear we'll Next, Stanley. Matthew with

Matthew K. Harrison

question. just morning, topics for the on two that I already. asked Thanks Good about people everybody. Great. have taking push If could

on near term, first of revenue second stream in both question just to urgency is BD, Thanks can just that around those many subcu needs on you following to current your work planning I'm I Geoff's plans. be you to see think, to device is very to just what expectations then, and can related So, up And there? done be would done? investors trying or work through question like initiate the and understand to sort Is there ALXNXXXX, the the more think, the much. formulation relative what's the of has walk diversify I I you just study before think to it sense that study?

Brian Goff

done device work do to use the patients and program, respect some that in is performance, be to Phase bioavailability trial initiate that therapeutic need compatibilities have the with formulation factor stability X testing do physicians ensure that subcu the testing, healthy we before we we've well human we study. additional as now as can concentration the testing, to to What and device. studied and already the volunteer higher So, needs then

a go into once-weekly in we we subcu some as XXXX. later of forward said, using commercially de-risking the provides program we're the device, which available As

Paul J. Clancy

question, Paul. here, With second of formed. development business sense to a the here. we the respect part, absolutely second this Matt, is I being new team have team it's a mean, a urgency new

competition believe with focusing that. than will assets. quality, XX be ago, more probably X was undoubtedly the We fundamentally on balance in quantity there's or of strategically, we're not years looking there but offsets set is, likely that opportunity that I to where

in absolutely I sense get quality I there's assets. but So, making think balanced a sure – – by of that we think urgency

Ludwig N. Hantson

of Sense disciplined. well. as urgency being discipline But and

Elena H. Ridloff

question, please? Next

Operator

Jaffray. Raymond from with come Piper That'll Chris

Christopher Raymond

taking two-part a Hey. the on MG. question Thanks Maybe questions. for

benefit investors – by endpoint. was struck were that the I lot think stat a sig primary So, you the and I label, in of the don't fact a got

with of was stat with now you you maybe not then have forward? what how The benefit? And a maybe label, aHUS that you if in you it, also impact great. follow-on about it warehousing I seen you going the may talk the team wonder that as – Just put here dynamic. – PNH. without on potential thinking or scenarios were effect, used if having And or can sig some view not But will, maybe that. management mean MG. with with there the downside around eyes on effect benefit does but question a to maybe, – prior set stat without potential about sig That would've that Is mean your about guys you the Thanks. having is or payers? – talk quantify, sort maybe I of a there with for existed of not? commercially having could obviously, brackets fresh the that that maybe

Brian Goff

Yeah. It's Hi, Brian. Chris.

the think terms So, payer I'll into I since prior downsides, of as found question And just that's obviously label on the to I REGAIN, be focus, well. in Without forward gMG, patients population. AchR as in That's necessarily that the FDA label on for moving are as launch. Those the total the us as will MG point anchor is antibody-positive as aHUS core well with looks there we the what you will benefits of a course to known well we called system, and a in on point label. there are Again, to in atypical system than have already the right patients, PNH are as we've adjustments well the at with patients with that as population. early who what the are so at I our from very John anchor the who positions reactions, well on. who do like diagnosed, And particularly of reflects with I going we're one think on. specifics payers. Soliris, make be hand, that had going space, react and We that a make make hard approval. the identify respect want the from we the least pleased with we'll in new and to of have, as get start the and into really few identified, appropriately we possible. think then it's upsides, quickly look gMG the that we're mentioned it is of as the that right working the well, to And then, comments mean, probably course, and to targeting on if that us, as entrant will in in neurology comments, positioning, what asked REGAIN about a uptake and to different we the the dynamic the you sure as XX% X% clinicians warehousing With our real effect, prepared them patient

that's So, side. on the tailwind

benefit level, the to itself. logistics have process had calendaring the I need work the in the we vaccination down verification through that On the REMS mentioned the as which as some of well we'll at as infusions side, patient headwind to comes

terms puts in So, determine takes, do and early with really of will that the how we uptake. those

Ludwig N. Hantson

it's maybe what that, bit prescriber that's a see little broad base PNH going And and to a be compared addition as in to we but very with to aHUS, different.

Brian Goff

(XX:XX)

John J. Orloff

of out is the reported can reported that. add came versus in Lancet what label. earlier to clarify Neurology in week I primary If the manuscript, results Yeah. this some in the To which

data, was is MG-ADL a approach we think gMG We differences maybe approved around out of reported accounts in I the what's robustness underscored in that the manuscript That the using worst-rank two for a endpoint I statistically meaningful there of clinical were indication. to was of the assigned the limitations end the important meaningful. clinically regulatory XX major and the were with bodies data label. in primary it's language fact analyzed and response. XX that REGAIN clinically was to a that statistical And Soliris secondary The even number that now for primary and worst-rank of analysis And that significant. statistically primary have had additional the was various the recognize totality clinical patients have of robust the by the – both points discontinued had analysis of test a though reported and they and think in is the the worst-rank some had in study the analysis methodologies. in also who sensitivity primary that pre-specified therapy different and

reported statistically repeated (XX:XX) provided in of sensitivity p-value what's And is the significant so least other more the and with the squared along table the analysis which a the that's means and label. in label is traditional measures

Christopher Raymond

Thank Great. you.

Operator

to go From Citi, we'll Robyn Karnauskas.

Robyn Karnauskas

Thank you. guys. Hi,

Elena H. Ridloff

you are there? Robyn,

Robyn Karnauskas

everyone me? (XX:XX). Can you phone hear Sorry. dropping My keeps

So, MG. the questions I about around is follow-up real question have

lot a lot do prescribe have around out doctors to find who of and So, and of there who work hard MG people it's to try market a the patients.

situation data that comfortable the international you market? the the after your second question's you comfortable base in how and the you tell market on this about identified and are go that identified given how have doctors and MG on doctor how in steps its that And doctors your broader the more to research can you all IP. (XX:XX) So us are actually you've

of You far are be think have If Thanks. you IP when would issued. issued, patents World issued EU that patents out U.S. take the and are how would Rest or issued? you do those

Brian Goff

again. Yeah. Hi, it's Robyn, Brian

rare U.S., great what our from you back it, could come so, in start we just I meeting in best I the as and part launch say, would having obviously hand-selected about that. have, And call feel neurology finding I question about of and new the good from really to the I'll industry But we on the is your diseases. that felt us. across first mention, on call targets who is the about of for clinician how

We the in have that we've now a lot team of deployed neurology U.S. the experience for

have And we a group so, the that very really well. space understands on that front,

the physicians. learning the call therapeutic However, we are about area in process, our working would I potential we good where through high a understanding new feel it, of

get this and centers hundred side a wider as Soliris few admittedly our is the as pencils and treated. about where patients you in we Ludwig continue neuromuscular more the Here, learning experienced But as well could prescribing a that predominantly deploy of and we'll aHUS. in MG accordingly. the, mode. alluded that I'm other experience, is we it, talking in have as we're And call other are we with neurologists, sharpen targeting base to, our dynamic, than to PNH And MG specialists, certainly

Ludwig N. Hantson

very might approach with but in the we're to Japan. happy might the before Soliris take to XXXX. a months difficult – we what XX timelines Europe, With respect ex-U.S., similar the take patents following until find X as another it are, to this U.S. new it's We're out. as IP, the say well And

Robyn Karnauskas

you. Great. Okay. Thank

Operator

from Merrill Huang hear Ying we'll with Next, Lynch. Bank America of

Ying Huang

Thanks morning. Good question. on my Hi. for have AXLNXXXX. taking I one

before more study, it to formulation reduction? we X Phase or subcu fair Phase such Phase going as X Thanks. seeking a is data the X to approval the are assume from PK you're see LDH biomarkers AXLNXXXX maybe secondly, based with For on result? when And registration then, the

John J. Orloff

So, first I'll take question the second – first. the

patients, data We a We've mention, program ASH December. dosing results to SBC-XXX are shared Phase X a be dose. we'll sharing the minimum – congresses. patients for Phase on both on be data the for number of first arrive aggregate time, and with SBC-XXX, that based maximum SBC-XXX And at ALXNXXXX maximal we've sharing data. we'll Based couple previously benefit at from the a modeled in X/X achieving SBC-XXX data in of the that's some we as out at

do C and intervals, that we'll all for dosing as first we'll is be to at a concentrations. of question LDH The program looking had and the biomarkers had registration that at you end basis the how subcu be looking normalization study. designed. the with At trough Essentially,

Ying Huang

for clarity. the Thanks

Operator

Rama. Anupam from hear we'll JPMorgan, From

Anupam Rama

of how And was much. so these price should term about patients? I be here how some here provide we higher on the thinking Thanks on the so of guys. in Thanks I wondering strategy taking a the net much little and if bit Hey, dose long for payers U.S.? quick you a one maybe Strensiq, with for could color ask actually, your question. could

Brian Goff

Brian. It's morning. Good Anupam. Hi, Yeah.

and course, by to that affected I'll to so, maybe adjustments you the Strensiq, for With our prepared color. comments as been in has highest Paul the U.S. growth that start with patients in And and to focus, of pricing heard serve the added And continue remains the HPP. with over turn it identify level

point, NICE the with I continue maybe, in, Paul, which patients. payers the we and to this of that, funding is into particularly think else? characterize of And way access in go effect of month, add anything expansion. I I dose to is to next to the a mentioned, much geographic now aligned as continue the good will worked November make U.S. agreement had to this if work is sure on example that year. want don't And U.S., in of you know higher work at for we that that value pivoting Strensiq that the We've from through the

Paul J. Clancy

No.

mean, adjust point pediatrics slightly talked different starting between was and that mix I adults for just to thought it think, through. that originally trying I a is the we before,

of in it it not our what think changing really peak of achieve could sales. for I So, outlook as all at terms essentially

States. the So, smart actually United just strategy it's payers in I working a with think

Anupam Rama

for question. Thanks so our taking Great. much

Operator

Bank, Deutsche Peters. Andrew From

Andrew Peters

Hi, my on guys. taking and execution. congrats all questions Thanks for the

to that hoping you guess, pulls the So, think bit kind I you've guess about question, coming was outlined. the on kind through run more. can of of ambitions guidance of you the I a a puts the some that back top picture if to bigger and financial of line, when contribution, see

marketed indications Thanks. underlying have? Specifically, relative you how think the that about how three each? of about now across you you And patient think demand contributions growth the in

Paul J. Clancy

Yeah. This Paul. is

is MG think change to cause aHUS that question focused lot year-after-year I they're by driven up The relatively now but of still is if the ends franchise where I timing will, over And hope XX crisscrossed PNH. basis. can markets, as continues continue PNH. that a big, Soliris, to, doubt, we well. but seeing we're, out really the Strensiq, growth And a on that doubt accelerator as I think and you PNH is then as aHUS, on, contributions I'll well. on outpacing obviously, have best contribution out hope of penetration, a it What indications, of in headroom. Metabolics out X your meaningful few quite, quite PNH broaden no being whatever actually of kind timing our kind for of us, to think No a be growth months without of of, in across a is aHUS sacrificing, is that We actually kick-in growth months both it unclear, or is is. or the what That estimate where continued revenue kind almost is of consistent.

Andrew Peters

Great. Thank you.

Operator

From Suneja. SunTrust, Yatin

Unknown Speaker

Thanks (XX:XX) on my is guys. David taking This Hey, Yatin. question. for for

enrolment. us the elaborate Just your on on market primary the you endpoint? Could question on for you this congrats need How a remind quick of see on expectations to much improvement NMO, event-driven the also completing success? do And opportunity in indication?

John J. Orloff

our a to time in eculizumab of in significant rate. X:X and showed reduction this we study a is, the event study in The relapses, So, Phase X is design randomizes Phase X actually placebo. versus relapse study

two in results is So, we'll arms. the share being the a at looking middle those to trial an difference enrollment. of able between with It XXXX. completed We event-driven anticipate the be

Brian Goff

the And just on then, Maybe this is Brian. I'll jump market. in

number. is U.S., cited the X% the to NMOSD the on X% population. MS cited So, generally generally That's about anchoring that it's of

Soliris in of nice which, think think the again, out. could it well as be you if U.S. opportunity So, the as thousand a plays patients few a for we data could

Unknown Speaker

Thank you. Great.

Operator

Somaiya. we'll From Ian BMO, from hear

M. Ian Somaiya

bit maybe IV subcu maybe Thanks to concentration duration patient's little related infusion, hear it question. different love profile of formulation develop scheduling. even in perspective about concerns a a the allow more from a to to higher to ALXNXXXX, and of the Would for taking my just some provide the does address you

John J. Orloff

time are with Yes, as the start I'll concentration us with infusion So, IV to it the that. for has opportunity formulations higher substantially. because we pursuing the well reduce

something well that's this So, will we be pursuing year. as

Ludwig N. Hantson

don't think else I I should Yeah. add there's that. anything to

as well for it's opportunity a think us subcu a great to concentration. IV, as with higher the I address

M. Ian Somaiya

to what similar be just subcu, advocated bridging for of sort will a path regulatory the assume you've we study? Should

John J. Orloff

small much we IV a just program. have to fairly because subcu be the do the study quicker, formulation the to DLA actually, bridge existing with to It'll with

safety to to also the I addition PK earlier. alluded have show that primary analysis will to We in

bit said, subcu I fairly we're more But little for to limited, estimating So, program. program. patients extensive XXX, a as XXX the again, that's

M. Ian Somaiya

you. Thank

Operator

we'll And Kennen Mackay with question from Markets. take RBC Capital our last

Kennen Mackay

just and MG. could have strong what on for guess I here. your congrats the amount wanted to turnaround of clarity Soliris coming on we quick Hey, and the unmet a for additional fitting thanks MG better in the really need Refractory seeing really say me looking to what of sense forward that a the I'm question, wanted versus Generalized really I in accomplished guidance Maybe just just front I you've short time here. in is the a on to on down get perspective John. line. the M&A such

the unmet really understand us the drove applied thought MG. Generalized thinking I'm is Refractory, have to being grant that what for where that's you're a can I you but in how REGAIN focusing just which must Refractory but decision label label, broader of know So, was is of FDA's help it a really, versus you need population? MG broader the outside

John J. Orloff

population current in gravis that and and responses Well, a immunosuppressive therapy address two inadequate who know or neuromuscular receptors, generalized underlying IVIg however, for therapies the study immunosuppressive at use activation part to that junction, of complement complement of population overall, exchange. patients that that destruction patients positive the do plasma one with were enrolled had some that antibodies their myasthenia chronic and disease that related that's not the or REGAIN, acetylcholine we we have to who immunosuppressive therapies is activation, to of

The of the we can't in at outside label healthcare use patients enrolled eculizumab study myasthenia on the I I So, a gravis comment therapy. we'll the really discretion obviously, complement. it prescribers generalized appropriate and for to providers of are the that. product and of determine beyond for FDA, those can't granted whether that us leave

Kennen Mackay

complement opportunity mechanism any there the that the to of where we given And just crisis of you. don't is something Or, fast data have say? action here? treating is in Got

John J. Orloff

crisis, have again, don't there's with data IVIg that have or role we but in you And speculate, a typically plasma on for treated in setting. could, data complement, they're We I the the guess exchange. don't

Kennen Mackay

for again my Thanks taking Okay. questions.

Operator

call. conference this participation was your concludes and you now today's that for and And you our disconnect. last question may Thank

Alexion Pharmaceuticals (ALXN) 26 Oct 172017 Q3 Earnings call transcript