Alexion Pharmaceuticals (ALXN) 8 Feb 182017 Q4 Earnings call transcript

Good morning, and welcome to the Alexion Pharmaceuticals Incorporated Fourth Quarter and Full Year 2017 Conference Call. Today's call is being recorded.

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

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

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

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

to for Alexion the for success advanced position a diseases XX year our made outlook I their to and today XXXX. was Thank want us months important rapid I morning. employees rare joined saw what value significant proud for their performance patients on and and passion and future. dedication, build transformation you, accomplished. Alexion this for report pleased about and I'm our We're mission. has for and continued of XXXX contributions Alexion in for our company increase our Elena, to thank to and the their position and joining opportunity the to we to grateful and I'm team the families ago thanks XXXX changes position leadership as creation. for for

want Looking slide moment to we've a I on X, growth the to our to take at taken review long-term deliver objectives. actions

experienced we leadership First, seven the strengthened adding highly executives. and talented new qualified, team,

We with directors also deep experience new appointed independent our to biopharma board. four

shareholder based of we Second, we strategy established on refocused business, value. review long-term our disciplined announced a comprehensive financial and and corporate deliver to a ambitions

strategy that the also $XXX through our program with savings approximately a will annualized We restructuring aligned XXXX. organization generate in million by

Soliris long-term. business believe protection and growth for drove from into in building rare double-digit strong the gMG patents leveraging we we regulatory which Japan, are in meaningful approvals rare, our business ensure the and achieved we patent leadership to focus XXXX. our Soliris ultra-rare and Japan received volume execution. and the a new and we Third, revenue that Fourth, extend for prioritized We in Europe the obtained complement and XXXX, through U.S., that expanded U.S. growth our diseases, will on to in impact in have in pipeline

trial, ALXNXXXX R&D Soliris program. the development as completing the advanced in subcu by as PNH ALXNXXXX trials also Phase X the and programs We involvement advanced NMOSD our well

XXXX, we performance financial revenue our non-GAAP continued XX% growth deliver XX%. to growth EPS year-over-year growth of briefly growth of X, mentioned, on including slide and XX%, of in Turning double-digit as volume to I

initiatives as highlight five the to shown X, I growth. on key would like for sustainable drive slide to Now, XXXX, long-term

agreements and PNH of more LAL-D; Soliris; increasing and the benefit access with growth funding volume aHUS, patients Kanuma have from markets. for and diagnosis serving momentum. in-line with patients our additional This can business to to Strensiq continues and to HPP First, treatment revenue who and expand new includes reaching and

and are focused a about and and, half very in with patients U.S., milestones studies for of IV we the launch gMG positive, to file excited patients a Switch several our our and executing Second, and care and in U.S., opportunity plan for the successful on new the in Naïve first Japan. from the and serve PNH quarter in XXXX. will standard priority second highly for ALXNXXXX, leader Germany is will are complement, expertise. year XXXX with in are we of if crown PNH XXXX us the XX be jewel the X is of in global our Phase goal including is important year, we of leadership to raise extending where second than pivotal years EU ALXNXXXX to our Japan third with with data aHUS. A scientific more the

in ALXNXXXX, terms risk. rebuilding business In disciplined of and to on pipeline, will stage addition be to we diversified development is on development advancing that the focused executing and including leading rare pipeline our disease build

XXXX and XXXX. a to to good growth. on in achieve turn the Paul revenue XX% are including growth, last to we the laid path XXXX of margin double-digit I will now EPS position the and our we to than targets operating greater ambition revenue financial guidance Finally, July, progress discuss financial Paul? for it out results over growth

Ludwig. Thanks,

year in XX%, across our X, share volume of to our led This patients, focus as and non-GAAP earnings growth delivered growth saw revenue more We per serving double-digit basis expense on all the coupled volume point control expansion operating products. non-GAAP in approximate growth XXX slide full for XX% with an shown by margin. of continued Turning of XXXX. to we growth XX% momentum

X.X% to outside for quarter tender fourth approximately from million $XXX forecast. were sales turning $XX grew quarter the year-over-year. Soliris slide favorable to did and our million The Now, in Kanuma compared orders by the in certain benefit U.S. prior net and product XX, markets

headwind on ALXNXXXX was revenue in In other and trial enrollment million approximately the from clinical addition, Soliris $XX QX.

the QX of and million $XX ramp headwind as the enrollment. million the welcome As $XX we continue of a program. half first This in is to expected, was year increase an approximately to advance due from this for the the

Pacific product of America. in by continued growth Our and the driven World Latin EU whereas by in Asia was U.S., in softness impacted were Rest sales growth the

the see increase of we side slide, XX% QX. can delivered a volume in On right-hand you the in

the and in was markets and low-double headwind million patterns, $XX $XX to Soliris It's volume to contribution growth we've was exciting quarter digits favorable the six ALXNXXXX last in was with five for and and was slide adjusting volume year-over-year ordering launch. consistent million QX will enrollment our orders context. In the approximately QX, to gMG XX, X%. for from launch When what other the in very timing quarter. or $XXX in revenue by Brian tender provide approximate million in growth Turning quarters. the seen and forecast. to in growth volume a The given us prior a was in fourth the certain greater benefited year-over-year study X% minimal PNH aHUS of revenue ALXNXXXX compared

of As XX, million, growth reported XX% growth Strensiq and revenue XX% revenue shown slide representing $XX in we volume year-over-year.

which recognized markets. achieved in funding tender orders reached Looking we we in at favorable previously forecast to European million, in $X Kanuma, prior in compared agreements of that deferred from million by $X million was markets revenue certain benefited quarter and our the revenues as $XX

grew $XX share, of to non-GAAP operating $XXX GILTI $XXX of which of sheet $XXX per offset from Turning earnings million operating and upfront the million and $XXX expense $X.XX XX, was tax rate. XX% minimum was EPS XX%. tax for SG&A licensing revenue million charge $X.XX includes the for XX% XX% in to agreement, slide diluted non-GAAP transition as expense related Halozyme as in or share was and rate the $XX the revenue. growth million, Non-GAAP reform reform. margin charge of we reported the included million of to related per of quarter from was revaluation non-GAAP $XXX by This during R&D million million driven restructuring well P&L tax GAAP by per related taxes was new a balance leverage. partially in and the We tax XX% $XX quarter, the benefit tax share. The new revenue to XX% effective expense the QX tax fee This a million. diluted deferred non-GAAP year-over-year. The of delivered expenses,

and of revenue with we $XXX XXXX, XX, lower marketable rate. driven was increased for across the in growth from a products the to in we year operating full partially slide IRS by the had the continued net routine on Non-GAAP by benefit to [increased million tax growth was by for approximately XXXX, conclusion and X% of share to clinical Kanuma full expansion offset for of $XXX of In a grew in margin shares of XX, generated effective XXXX, and of XXXX, actions growth a patient approximately operating headwind Soliris The driven X%. year benefiting regions. $X.XX headwind margin both the per XX%, volume the audit EPS was increased XX% restructuring free Strensiq year Full from flow (sic) billion third the securities. We billion XXXX. ended to revenue of to as XXX% driven noted utilized the sales increased year-over-year, the Revenue GAAP $X.X XX% headwind driven by in benefiting portion Phase ALXNXXXX cash product X% Turning million in XX% geographic and $X.X volume X FX $X.XX which XX% in XXXX, rate repurchase billion] $X.XX, from earnings growth from expenses. of and slide by share XX% per pricing year and cash flow revenue increased growth other compared On despite strong additions. benefited from and XXXX, XXXX studies. the enrollment quarter. in non-GAAP the cash X million. includes a $X.X and tax

$X.XX now range. as our guiding the of assumptions total currency. Soliris, for the year-over-year of XXXX let's expectations billion at price and midpoint for This well turn includes trials $X.XX on impact slide So, guidance and for to as to between our XX represents the the other We're billion, which foreign growth XX% ALXNXXXX revenue guidance. of to

and would Excluding growth be these mid-teens. pulls, revenue the pushes in

Phase trials For assumes as million estimated the Germany is this in from other $XX between assumes ongoing and to initiate U.S., Soliris, the in a on $X.X our ALXNXXXX. This revenue top launching to our Included XXXX. late million Soliris from $XX plan to ALXNXXXX study on impact studies It of billion. Japan. for is in a the as impact well year-over-year gMG million of revenue guidance headwind billion also subcu $X.XXX in XXXX X estimate $XXX

six to adjusting and compared high-single volume the We digit PNH this in for assumption a in growth planning volume the for last and for planning five adjusted prudent digits is assumption. aHUS when low-double trials quarters. ALXNXXXX Our as other to believe simply is growth

to guidance Turning our million reflecting $XXX continued metabolics, revenue is $XXX patient additions. million, to

million currency revenue Our guidance $XX or assumes benefit. XXXX foreign X% tailwind for million approximately to $XX a of

approximately addition, the we attributable price to to representing expected inclusive expansion In X% operating reform. and be of earnings the IgAN. XX%, and restructuring be growth. estimate earmarking is tax $X.XX expenses. in to operating tax be XX% GAAP point and reform. rate and this basis in the This earnings be growth range guidance provisional is attributable expected profit studies XX%. year-over-year. is business EPS to basis represents at inclusive gMG increase related impact XX% effective of this non-GAAP of a referenced is to XX% includes XXXX with the $X.XX, to expenses. in to impact which is will $X.XX to XXX two-thirds approximately to guidance, of XXXX prior Non-GAAP represents $X.XX Non-GAAP expected the share to October U.S. XX% during our midpoint for to to of the U.S. per to to of related This Soliris which margin XX% XX% this be metabolics. and XX%, in an midpoint restructuring range margin XXX million non-GAAP one-third is of XX% new At share had we over headwind approximately includes to per operating tax from points ALXNXXXX of development a expected XXX in GAAP guiding range, the We're $XXX assessment of QX

a in P&L of XXXX mindful studies, half markets in year-over-year our basis in XXXX. in enrollment revenue of forecast, XXXX. the pace of ex-U.S. to first the in prior Moving to dynamics orders the from to some change highlighted be are recognition, challenging. we've and due particularly quarterly dynamics of a These on comparisons favorable in XX, to compared This slide comparison of XXXX lumpiness through ALXNXXXX makes the

second our low-single-digit high-teens More in more on financial us Brian. and greater whilst well as the the longer-term of financial leverage is through double-digit and line the year. we call pipeline to of from close, a in margin position as these this gMG half specifically, first normalized ramp However, per XXXX the our bottom revenue growing business line, non-GAAP year comparisons including earnings in growth, ambitions turn To in revenue to expectation over leading slide top revenue continued will objectives these that growth. growth in expect I'll revenue anticipate second growth in half result our comparisons We XX% to half. XX, to achieve the on in including the to development. from the share objectives delivering simultaneously investing operating

to with pleased I'm XX. slide Paul. on commercial our XXXX, on performance Soliris in starting report Thanks,

more commercial the well new greater XXXX aHUS, driven in of pleased to operations even yet of and despite on enables have global PNH enrollment still continue IN continued the that starting our a that Soliris is new our PNH, and excellence ALXNXXXX in initiate as and disease aHUS that seeing rapid number very patients in expertise, aHUS Soliris patients education that continue treatment. with We're majority the PNH. continue with Our on with PNH commercial by growing to aHUS in to trials. with and believe we long-standing believe patients growth to underlying of the than disease PNH as identify patients, us In serve we're to execution. focus was opportunity

both While we of from trial and ahead other in expect PNH recruitment and headwinds continued continued in aHUS. we for expect ALXNXXXX growth XXXX, Soliris us

time disorder generalized slide with on for to patients XX, neuromuscular this new grateful been waiting myasthenia a who and now Moving treatments. debilitating chronic to have for for long we're extremely serve opportunity gravis the

are first-in-class in the and approved patients with Soliris complement labels approved already these for is a Germany the U.S., therapy and based pleased We're patients. serving Japan.

to These Our continue the trial. focus unresolved total study AchR response the estimate XX% MG population approximately we X% population the are significant More in which to represent is symptoms, gMG had the of filling therapy immunosuppressive patients U.S. X,XXX gap to Germany target of knowledge PNH U.S. population. Japan. which a in REGAIN appropriate who and disease the be and patients and our and approximately aHUS gMG about X,XXX with launches, physicians of patients to little to treating seeing in to limited patients on X and from the specifically, X,XXX a suffer inadequate Phase in antibody-positive time have each, we're we're actively expertise. launch with some talking breadth like with of depth complement that Similar I'd is dynamics our complement, about now spend the to our

complement Our of benefits is of specialized including team role actively the junction, with inhibition and neurologists, as and dedicated well critical field the educating specialists complement-mediated trained of the neuromuscular on destruction as neuromuscular Soliris.

good in with with U.S. approximately coverage. lives the payers and very made we've now XX% of addition, have commercial In policy progress

now to have which in the XXX that approval late includes XX, I'm pleased report we in slide FDA than the to enrolled patients Moving XX October, now over OneSource, in U.S., general more overall on only Of represent the adoption the by neurologists. Soliris. OneSource, the community a of cases and an patients number from suggest small which early open study REGAIN prescribers, investigators in following

Managers once treatment. they're assist through treating a patients, our and initiating verification, as vaccination options, enrolled As selection, in finally, site benefits Nurse physicians as reminder, OneSource, treatment Case well Soliris

best PNH our the ambition and to or experience serve aHUS significant for above is our this to launch launches, aHUS. revenues. gMG and our about excited PNH launch and of with very be Soliris at with patients in We We're have the gMG, opportunity process

metabolic to Strensiq, and Turning growth with our therapies starting continued for us. a driver

agreements new you France a to secured made see patients UK, Israel in XXXX, in funding and in with in just XX, we HPP and on Canada. were in slide the have Japan recently agreement expansion. XXXX, identify and pleased funding the U.S., As to Germany with geographic progress reached continued and We

can to in see geographic transformative growth and Strensiq. in access We We're patients trajectory, in additional benefits Strensiq's significant countries we the expect early so still other launch have XXXX, expansion of ahead.

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

to performance the U.S., with in in expansion highlights XXXX. R&D turn John the positioned very the agreements I'll funding XXXX XXXX for believe and outside to addition, call in Israel of reached the in now of I'm over we expect in quarter. growth the Italy, and the team's overview commercial pleased well In XXXX. we continued and John? are Netherlands deliver an geographic from additional Spain,

happy I'm to made to rare our XXXX progress significant disease the advance the Brian. you, pipeline. Thank R&D report in on organization today

regulatory wanted refractory we Before regulators our they in forward gMG our with look review working approval R&D under review. for programs, to and with I to share that filed priority Canada I as which Soliris application, for patients discuss is in

address Naïve has the XX, on X ALXNXXXX Phase enrolled inhibitor now potential ALXNXXXX eight patient we important to intravenously to every both administer next-generation innovative and In Turning PNH needs. studies that Switch both slide our weeks. XXXX, rapidly CX the which

patients. if in also of Japan two-thirds We EU slide half This in studies data in second treatment-naïve the study complement X study patients HUS for positive, quarter. will Phase expect the adult we second atypical U.S., approval the enrolled. enroll non-inferiority regulatory we about and And adolescent On continue to file XXXX. the from is these in and in XX, inhibitor

complete ongoing results is year. in complement with the children treatment-naïve X study quarter atypical in second the in the We a expect Enrollment in announce enrollment also fourth Phase quarter of HUS. and this inhibitor to

in Turning who ALXNXXXX of enrolled anticipate XXX half half of ALXNXXXX and exposure. XXXX. second moved in pleased registration. of program to two-and-a-half to teams have I'm the on is an the slide the development targeting early execution ALXNXXXX initiation the X patient have filings XX, X first the We filing over the for with IV Phase development in XXX and Phase PNH program patients timeline approval very XXXX. initiation over of now on This from just years We our XXXX progress in to years from

Our as and for We've PNH program atypical HUS. standard of new to with the ALXNXXXX made a also ambition of ALXNXXXX develop our progress subcutaneously. concentration delivered care to patients is formulation establish significant in higher

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

In our to collaboration agreement subcutaneous or next-generation a to we in development products. Halozyme every their for once delivery interval into drug entered of the portfolio subcutaneous technology once a of is addition, formulation dosing month. potentially two with the agreement extend this in Included further of formulations of weeks December, per in ENHANZE ALXNXXXX

trial Phase PK initiate single-ascending this year to a expect our later X We decision. dose to dosing support

now X NMOSD of spectrum mid-XXXX. neuromyelitis PREVENT multinational or with XX, optica single in study, placebo-controlled to from trial Phase slide in Soliris data Turning the disorder patients report to expect we a relapsing

a our to our hematology, objective a trial and As our neurology a is NMO. leverages of of Phase attacks on a pipeline and metabolic showed is that in relapsing fundamental focuses small patients an patients therapeutic based that on biology is with study areas in reduced nephrology, frequency severe close, complement disorders. reminder, core strength X this investigator-initiated build To in results that event-driven XX from

additional are devastating slide on late-stage two gravis disease XXXX: ALXNXXXX our second initiate preparing study in studies we called and myasthenia kidney a with in to XX, advancing in indications IgA in proof-of-concept shown to a study also registration As a addition pipeline, in nephropathy.

turn our business by development focused updates inhibitors also Ludwig. leading progress deals I in rare executing year. the preclinical look call the diversified on back to throughout a I'll now to Ludwig? innovative with forward development build and to We complement mechanisms that development risk. disciplined have additional novel providing stage on disease is pipeline and are

Well, thank you, John.

saying conclude by me accomplishments XXXX pleased today's very Let call with I'm performance. our and

financial in-line We have launching in the in with in a we successfully gMG, achieve extending XXXX, pipeline, Soliris rare growing can complement long-term and advancing on of sustainable leadership including many milestone significant ALXNXXXX, standard create and and leading shareholder delivering By ambitions. confident rebuilding value. our focusing opportunities care our areas, disease I'm business, our and and these raising on growth that

call open We to will questions. Operator? now the

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

on on coming for at Congrats top Maybe lots on quarters. last you. then seen if you're a at that this also the line digits what that conservative? guidance XXXX I see over volumes Is more on pricing PNH your it declines, of or execution. a X% all, low-double-digit Paul, we've range bit we're to that really Morning, historically? questions the Is just just the Thank is versus aHUS the for maybe pick morning based and being assumption And XXXX. pricing. to few guys. anything assumption little can question seeing high-single from going second down prudent a in

Eric. is Paul. This Thanks,

growth what seen remarks is in ALXNXXXX the with for other great so in for the identified of last or volume essentially So, as nub low-double saw in we and the fourth adjusted What the we prepared issue. questions, talked to quarter what PNH six and respect for I competitive right when is usual five of to the about getting kind aHUS quarters, digits. we've trials

it's go XXXX. seeing into as not So, anything we're we

assumption measure I prudent XXXX businesses bigger, just it's indications, assumption. against a think high-single-digit I is bigger a it And planning of it. mean, line the volume lot of moving numbers, of growth. to part top function just to and simply small a that's the it's a I into it was little going way – bit – same those to same getting think in

we past. pointed seeing. headwind With been the the it's company. effectively is a this of think company, the question, second of So, bit we've in in pricing. a which thinking about respect volume-driven we're than seen to nothing we more the little your in kind X% is out, Alexion overall the is terms – of meaningful States, has as CPI part terms a of We what for United Recall underneath always have pricing, in

So, what talked is the the of about some we second Soliris, then more as And largely annualization little is little the as happened half the dynamic price in of headwind in and that a than XXXX. are a quarter metabolics, registration which one-third is, – points into by bit around third in with where Brazil in lower. is on Strensiq, getting countries the what good it's a bit driven Rest this of of well markets World of that price example late

the color of other of and to Brian, Rest impact, markets. give? as that some World Europe kind So, then of in countries well as other any other annualization

team is Well, would through a thing to it's XXXX, obviously the mean, about quarter. – this add you've only our fourth results navigated underlying They've to now a a I I I lot front the seen change the growth, is your question, the in Eric, and mean, for lines. back right first of

quarter. You saw the results for the third

it's talked now has well, you when takes, just and opportunity So, out is navigated as continued of. and that's fact group we're than in as encouraged opportunity, the of ahead. times net we exciting change many on we're the comments, aHUS, that top opportunity pile gMG, proud Paul we a more even by We've the prepared and the said, a very bigger look PNH They've growth as than behind at momentum have. that about and us. the PNH, net puts see a I even ahead you that's noted that And the we especially when

color. Thanks for the

you growth volume underlying The then when for you and include remains MG ALXNXXXX. adjust double-digit Yeah. Soliris of

Welcome.

please. question, Next

question We'll with next Stanley. take our from Morgan Matthew Harrison

that could a heading Great. things context to question, was Good the for a I ask into morning. you I investors are hoping us into just on couple read-out. put and PNH focused ALXNXXXX wanted of

you're put could to risk, discussed for now the look is encourage meningitis just that the you we much. maybe So, for you Soliris. one And you Thanks you've endpoint which of just into when previously at and talk PNH? the ALXNXXXX that things study, see co-primary that about would the how a for historical context Naïve us how Soliris if using view the right then of versus data very some data separately, in

is a LDH question, think, that the as This Sure. regard non-inferiority With the I question. co-primary Thanks is normalization the trial for designed avoidance. with transfusion for to and John study endpoints Orloff. second

For LDH that leads ratio levels, we'll weeks margin measures then the eculizumab model based through every the a repeated for be day an XX. analyzing of XX on ALXNXXXX calculating end week two a be study that. non-inferiority from we'll core And mixed those odds comparing to to and

what the clinical know CX Burrow out related we in on the risk infection to that based Soliris. meningococcal about our seen development of so patient we've with seeing cases at for program based on X/X differences consistent regard have and far, knockdown to With calculated the what work have And rate from of For and Also, previously of Ray transfusion that's extensive between margin the of our avoidance, and years we're on exposure. XXX done against Phase UK defend is to three data has we experts he's groups. historically. we'll of be program, meningococcal about looking assessment We the two you non-inferiority the and we risk, over to this XX% Soliris one probably believe with actually that percentage infection. activity what meningitis based CX traditional The approaches based world's in our on XX% need area.

there CX inhibition. we in don't risk think between targets Soliris differences So, material be or any the other for will and any ALXNXXXX

Robyn next from our question We'll with Karnauskas Citigroup. take

guys. Hi, my Thanks taking for question.

the grew much guidance, impact. a is it the the more color given. and still hundred But – clinical grew just dollars confused million by a also I a last that growth few more don't little I'm you all – had feels think you trial you've I understand why I and – like year, when still maybe appreciate you

to I just the but and a I'm to, seeing MG So, you're just better, being like, to And sorry ask that you're – to confirm seeing think did wanted what this, it include missing still out MG, slowing in I again. guidance? of I mean, there if the mean, patients I'm question help to in indications? for either I just And on me number of still aHUS, super include said identified, and you then seems isn't conservative, how something. And same trying understand it figure PNH those I'm just decide just in – your you

Brian. Hey, Robyn. It's

underlying trials where, in case That mean, think first a continued with communication trials, then the of obviously don't for more our for the mean who doctors growth estimate. have anything. and growth. first dynamic. on net PNH, lot it's talked to we the aHUS, product. own, PNH, we really that their get same see track along. times, and of, takes there is is Paul go we And we've you're are I to I'll continue of to that demand – So, themes continues both From to same missing I in the experience growth, many just it's I if field, us, that prescribing in the to see do that out – the generation to anything, wants we and and competitive the that's an less it's add the or about, have see puts Admittedly, the

you continue. and been – where that MG at that now our XX and And gave ahead We is and now exciting with Paul So, encouraged. with MG, for will said, about Soliris that's view to big will look and an patients ago, that's best we were therapy it's growth they with numbers our the days month base update a It's over an are can early be business, are and on that a over XXX us. those have there, where opportunity. focus cases, that converted the as of

set dedicated neurology believe that a a have that We as broader to you we Soliris be will know, teams, of and looking ahead. business now contributor

a just I quick ask If it. Got follow-up. can

the your numerical think even difference thoughts by a patient numerical you or is will is, So, trial? even primary difference are though the for lot patients it's ALXNXXXX in be asking whether endpoint or whether of on not non-inferiority What's a influenced influenced doctors though by a investors in non-inferiority that data what be uptake would data. a

is first a ALXNXXXX a step back look for an of mean, I course, high Alexion It's already course. foremost important ALXNXXXX are really Thanks sound continue that Soliris that's to to think for into moment, looking of the for standard emphasize surround patients and advantage. well, opportunity when competition is we it's Sure. from significant for and bar PNH, as the important for as space, for sets of to a at that for and to a the to us and business the quite enter we patients, have important question.

important And standard we're care care. we what's and what from from So, our of patients. for have Robyn. And to with we're is what eight for shift every PNH, account counting moving weeks significant is that the every his for that's, essence, a that to win. ALXNXXXX, in standard cycle call the treatment two And treatment, the really objective achieve the ALXNXXXX see on and make will excited of once into of the week be readout from data, mission and it, fact team non-inferiority a if significant design that's the new non-inferiority in John once the interval takes we to

back and LDH Francisco regulatory means, that success. endpoints and is And the are month those for Soliris definition ago a the as LDH was as all us, authorities. I clinically discussion has first of high respect Brian about versus with clinical as LDH It San to us Robyn, that on know success, want normalization. one with agreed in success all, to regulatory endpoints is of normalization And had And achieving set the it that what's bar reduction as to the with we're because ALXNXXXX. saying, success know, to of come from Soliris you was here, a relevant. also transfusion co-primary avoidance example you moving non-inferiority

which before that, efficacy looks on like. those is a month, instead and response So, success, the then spending XX they the or of a subcu it's Brian journeys, almost we're big hospital, being what the like And going deal. on hospital to go of patient the strong. about off talked into week ALXNXXXX raising six, once in and on day bar through, days days hospital, in what that. understanding patient the On will a insights the allow top even so patient longer are have The formulation to a – than hopefully us

So, raise we facilitated and believe bar. the have we to aHUS that be with have, will a and Soliris ALXNXXXX for from we non-inferiority in great to a conversion PNH position

discussed, for are we quarter second looking really the timelines on And readout. as still track forward. a we're So,

you. Thank Great.

Porges next go question our to Geoffrey We'll with from Leerink.

for understand, patient is a very or I for XXXX. the question. it see non-inferiority or with $XXX if where Thanks. populations in quick a want million much Could a opportunity? give free of the opportunity flow actually. consistent see of profile going us is you an PNH penetration the in have Thanks the design, then, aHUS to the addressable just do XXXX? to And of you about switch increase sense primarily Appreciate ALXNXXXX it. in You cash you you one Paul,

Yeah.

for Let me – Paul. Geoff, thanks question. the

range. shareholders, wanted deployment for would deployment I'd and is Let to if and me $X.X million, of $X.X $X.X be perspective, part the an as is have should you about for or which I base so place said return don't that say business U.S. From spot plan move development characterize talk it. above cash news. Organically, our the it million great deployment that the the to it and me I no organic flow, things saying on free first let we longer question. of in with and ended we numbers year to underscore billion. ex-U.S., the beyond excluding start We have for cash but with some

from to but per that implies flow. earnings actually bottom cash deploy think leverage I margin Now, line a free profit even we hope the that the to operating it share the and

into one manufacturing for high-water a lower it a way, growth we investment to we mark, think, should of of XXXX expenditures. towards been that years is of And XXXX and financial ALXNXXXX into moves this So, a and plant. continue coming the of ideally have high-water in off past and expand achieve Dublin it more. of cash if is to expenditures, potentially much flow capital fact, respect and XXXX biggest We're mark last in numbers, couple kind we've headwind expand the cash if right that XXXX go also The out completely to... we that that. large-scale coming with just XXXX point are on reasons laid to to we we free as And going the free Turn that of biggest said tailwind. kind expand objectives actually the XXXX. flow ideally up a drivers and like part can of in capital And a XXXX, without margins, into a another the respect big get is I with in we more over making the giving for and open over exact we XXXX even

a is ALXNXXXX opportunity conversion the growth Yeah, us. and a for

it's add but to it. color will Brian some both, So,

speed and way patient look the the respond as how could might want opportunity for well, question because in speak, you who John's potential enrollment PNH. course, well there the the the of Naïve data are what have fact the to on at opportunity. And enrollment Yeah. from is I'll that as is a penetration But speed saw we for and sidelines, And so that that the like. looks we that all the further be of population, have wait product the just that be. the that in said, potentially a look to remained significant see like of the as patients suggests what the we it trial that's as trial we group Switch best of to at add. both I maybe mean, proxy a I ALXNXXXX Naïve

Thanks Great. much. very

bet. You

next Flynn question take Sachs. from Goldman with our We'll Terence

some of Thanks reform, your to just just Maybe intellectual are hiring, think how you BD, question. taking Hi. then the and a that an you follow-up property of the of changes? guys footprint maybe thinking timing differently for the should manufacturing And where or update deploying about Thank given here, capital. you. about we Paul some you stand, just us could tax on post give for respect structure with team guys

for thanks the Terence, Yeah. question.

Second really have the is part a and I great of actually question don't yet. for you complex, answer pretty

of want that flow predict. hard It said, rebuild pipeline, though, if in I coming of result different team. better We transactions. build all to hard program with beginning also, fine the but about when get over we business talked new to sort internal even look think use that of manufacturing definitely that plans a we're kind into it's We're the to that it's to as be out. to to beginning, just With to or we – respect to our part year if is think you this the we hired footprint. team. last of and month in we've regime, just so, to It will the potential to will. pipeline. really is the disciplined, extremely continue exactly of, a respect truly, course, you like going predict No with still out the development, the the of leader will, will We're hard

diversified word on the financials strategy, discipline is Discipline as (XX:XX); on opportunities. as well

the So question. take we'll next

Great. Thanks.

Raymond from Piper our We'll Jaffray. Chris with next go question to

Great. Thanks.

any? asked converting patient? OneSource guess, patients I expect the of many and just I maybe at patient to like in when any last the paying think, actually XX could looking as month dynamics, the you on you're XX seeing on another sort way, success if terms getting OneSource reasons, on a – ago, I are rate, program of looks wonder So, color patients just and updated these dropouts update guess, added on I It versus becoming that patients, MG your the are you of how paying if you Thanks. we maybe program actually to the provide sort should biggest to the the drug. what and us incrementally OneSource I a think, drug.

Yeah. Hi, Brian. Chris. It's

launch relative around and parts, of starting So, we're the just the that really the at now, them well patients. come call and But are, REGAIN back the two point to I'll far, the dropouts, patients, that to to most ourselves made the a conversion patients, study one to believe little are with seeing give from of which characterized the maybe well is update out-of-option then in it, had. first findings where we with that we treatment I'll the to – come OneSource we have mean as to numbers I the of remind through progress patients the take I last types top-of-mind, the so as you system characterization we've

studied. In crossed the that's more of once also steady, as the probably somewhat new we that expected severe system, year. the a continuous we we fact, over been reported into the population into we they're patient of accelerating, And on would've movement REGAIN side kind

that's continued we've encouraged to that been progress. So,

that The the majority I those from of other the not is would point study. note patients are REGAIN

the dropouts, the most small. you are a So, on have the launch XX% very where in window are from and, still so really that to get the XX% REGAIN Soliris. been days dropouts the clinical of at early of And non-responder the you patients of And that about first population. That's the rate new these inside essence we're into terms it can response XX-week major reflect still point. in design, of then mark. and patients early in was study, enough most of then We're far, trial when

take question. We'll the next

Levi, the next question, please.

our And we'll next Huang. question take from Ying

me? hear you Can Hi.

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Yes.

Great. Okay.

ahead. Go

questions. the taking for Thanks

So, have on couple trial. ALXNXXXX Phase X I a the

actually you the First in you to XX baseline can normalization week to original Soliris? of to then the you. from Phase rate XX, of for the secondly, not LDH, maybe week in X this that what trial, through higher at hemolysis Thank And confirm you expect do similar the of end all, or the it's saw be of period.

the place begins X is at weeks Orloff. because few it Naïve of core month take for which take goes actually, patients, or study does and XX. end The with evaluation day Thank the is so you, week a of to Ying. John LDH it to normalization This the XX

a be two We weeks ratio the odds measures, measurements end won't will We'll an every ALXNXXXX incorporating the versus model six. month be to it looking repeated into all the calculate for so Soliris. at just be of

the it. So, And of looking it's way at then robust a second very question was...

Hemolysis...

The rate of...

Yeah. Hemolysis, yeah.

to Anecdotally, we've for be than reported that based of nobody's have in clinicians on trial at substantially clinical criteria in lower carefully ever that the requires – using which this historically, a way looked prospective XX%. times to defined an X.X We rates of we're a So, up breakthrough that LDH elevation criteria. rate upper they XX% anticipate the with it normal the than upper back down less limit they've hemolysis. of times symptom normal; or would criteria sign to and once brought satisfy X limit of if to they a go concomitant have

reported. clinical more we lower rigorous been reports So, and anecdotal the than than anticipate the that's XX% rate is it the to be

Thank you.

Okay.

question. Next

we'll And question our Barclays. take next Meacham Geoff with from

of what guys' like been as to volatility see I they it you biggest I durable any patients seems occur? it, all it or complete the from to And Good when to have NMO ALXNXXXX Phase morning. return can is? in is the say guys. study, reflects Hey, just prior for Soliris, question. then the to your stay Paul, patients Phase Thanks the had NMO study To updates wanted on the a Soliris effect want X guidance I'm just approval? on commercial I risk view. few. obviously events Is would me, in and really the assuming you how this X? the get for your that's obviously the PNH there that to headwind ALXNXXXX but

but for question staying said, a clarification, registration. effectively much it's Geoff, – out extending the trial for in I'll exact is very until patients start, the point Paul. good calls of trial there. extension it's as Thanks It's this you

be it's half that's successful, they it the that driver that stay in we our a on XXXX, So, year continues so clinical plans, of why of would second big XXXX. in and a file and drug current is to would assuming the headwind into

and X X And John on programs. extension then, second. both studies those And a the X, from are Phase Phase

Yeah.

how some we variability mean, the and out there NMO, to I define that. point you're is relapses On in that right

defines we'll with Phase time. It a were X There an X per XX event-driven at that to study, trial. not it's study. mechanism prior as now study drug course year see, do and again, months committee and from relapses looks as to action, three study. it's had them But and size. frequent of you there a the extrapolate. anticipate to Phase all a down the trial. after a When the patients a dramatic reduction adjudication relapses Based look Our attacks durable incorporating XX of as sample the carefully is we zero at back central wears during going in the of but prior hard not really effect It's of a events a it's those follow-up. was going There really XX-month a patients on median off was over entering some to relapse, of

looking we're results forward the mid-XXXX. to So, in

the As into MG is double this discussed the done the is any is And conduct. blind higher a about study. have we as talked since insights pathophysiology NMO of fact, what we don't understood. We this not also with we've than much risk

those get forward and hope option make on looking patients. stay this we're sure on we profile to the focused we risk data really will to that that to a program treatment here. I our So, want bring

Thanks. Okay.

And with we'll JPMorgan. Anupam to Rama go next

slide and the about for shift of for in you XX, so ambition, so PREVENT? Soliris NMO, But taking here. XXXX success failure the much could could up Thank Thanks NMOS much. question. dependent how on financial just assumptions assumed these Hey, I on I'm Maybe guys. on there or follow what's question the thinking

will think you of of the the us the this half second of year. data XXXX how submit dates fast the in conduct about mid and time NMO, in the sometime PDUFA John the go. When midst take still having of regulators of fact with the we're go the for the about the talked how the that timing on to fast we It depending some study regulatory,

on limited. impact part we financial the And our you. ambition, very gave that's double-digit of revenue XXXX that is So, revenue guidance

Thanks. Great.

next Bank. go We'll Andrew to Peters with Deutsche

question. Thanks for on IP the taking actually. Hi. A question

update and Japan. granted the the status if us you've can to in granted you see the progress, wanted plan been how potential to see do to you U.S.? just Thank new companies as the on patents front, Soliris, kind defend and that new Europe. I you biosimilars portfolio of you patent related, in biosimilar you. in when So, then highlighted And the the last on Soliris year U.S. Soliris

in Yeah. With respect is Europe, to our the IP ongoing. process

we in as Japan. know, successful you were As as well U.S. the

similar to before, patent biosimilars, we should and So, there defense in believe is option. We're back focusing And dialog as XXXX was With best said and here program. think over to is very position. that's team that is and is. are treatment I us, success from that raise our PNH office outcome treatment of best objective offense with to better go the we're we're with on, approach. HUS, – a what sometime in taking to the you have a always our Christopher options talking about, in strong respect hear we We and what that the in patients define and bar becomes is hopefully, to the a preferred defense. ALXNXXXX a this more And we what the for it the product

ensure So, flanks ALXNXXXX through strategy sustainability IP, of franchise. our complement our our protecting successful that and to is making

Next question?

Marai Nomura next We'll with our question Christopher take from Instinet.

with wondering Soliris. Thanks are for Hi. if perhaps bit about little could how many I inadequately question. help taking a patients think was treated the you being currently me

Just treatment. around frequency otherwise, compliance that or rates of really thinking about

survey price? from think sense pipeline benefit protect price? And know, at then maybe walk franchise presumably may in there candidates of how just You subcutaneous formulation, MG, patients look to through that. a how of or sort about ALXNXXXX in your your should thoughts interest any there get And given development. or way How out candidates many secondarily, creative you. benefit, give high to we Is perhaps us highlighted ability us years to several on physician profile. the on a on in Thank recent much using

The versus and first we ALXNXXXX the the Soliris. differentiate respect maybe – with question how of is clinical can to profile

based on the have to extending Yeah. would dosing X/X is dosing coverage the I regimen eight to even it weeks. our make Phase adequate optimized interval, we throughout that that sure data say What we've

all analysis So, achieve really that inhibition defined trough allow would was maximal an with of to dosing model at the the for patients interval Soliris. for the ALXNXXXX. conducted even Such CX concentration never end

trends support normalization a potentially of we quarter, as reduction we least, second So, for And and since LDH said and patients. data, comes the out hope breakthrough it coverage will that hemolysis. LDH optimal more in the numerical at in

patients we the to is optimal fair clinical – it's is LDH development normalization, last troughs, us very But say we've couple on. define this plan for because through repeated design, talking talking PK/PD John, deal right for have have of raising that we big you you a been the or helped which The their us also trial our dosing bar we're for the about model the enriched, is that to in strong measures as and the the the months dosing. to and about, think listen we so do patient journeys. is be which every a could can I They those weeks fact endpoint two that when go were

we're So is I dosing are assessing those the feel that we achieve. to the for patients optimized how design and trying what and

Soliris, add to will with the that PK-based program. patients that of just there are anecdotally, in in heard that the ALXNXXXX end breakthroughs address extent interval which dosing the we've the would we I of at

And yeah, Brian. Chris, this is

then I and guess... Right

Oh, sorry.

Yeah. There were still...

have... We

was... I

ahead. go Go ahead,

actually perhaps following patients to Soliris I patient. the you meant could on treated I aren't that? number every week maybe inadequately refer that two of if address to to And with sorry, up – was visit, color currently regular hoping that's provide how on that that

Yeah. This mean... I is Brian,

Thank might I how mean, that you. on compliance improve? there, yeah, and

in is mean, cycles about so I Ludwig the with going of that. had the of that differences, on touch two I'll much not every of ALXNXXXX every key be eight has the an once hear to I weeks get life referred non-compliance. from ALXNXXXX It's for to from to we that we Yeah. part a meaningful of think standard point when the benefit potential out. we the see of living that why that's the weeks, play opportunity we PNH. that's that such data if And will care believe there patients frequently raise before,

question. We'll take next the

Okay, MG? then on

No? the That Yeah. was question. last Yeah.

rare diseases, will about a it Chris, differentiation the about think many MG, in way of your is, competitive and experience new approved XX term, years. than gMG make for standard we aspects. MG approach for us the first itself the One of It in the to profile who was longer is, it on Sorry, treatment be FDA combination clinical is our certainly it's course, and treatment. is ambition way of Soliris, the with question have the more to

meaningful So, our the Case of substantial, staffed is are that dedicated Managers, the there team, well. as elements The expertise are other expertise OneSource of up all all the alone together to for as that so patients make journey for benefits, and substantial too, competitive which team, is but will well it becomes the by as those that to be goal our neurology well. of clinicians package nurses, as

participation. question, and call. your concludes last today's for That this you our Thank conference was

now may You disconnect.