Provention Bio (PRVB) 9 May 202020 Q1 Earnings call transcript

Good afternoon my name is Kate and I will be your conference operator today. At this time, I would like to welcome everyone to the Provention Bio Call. There will be a question-and-answer session to follow. Please be advised that this call is being recorded at the company's request.

I would now like to turn the call over to Mr. Partners. Argot from Martin Sam

Thank you, on call. for us you and Bio's first thank Provention conference all quarter joining operator financial results

Joining Officer Co-Founder; Provention Chief today's Financial Officer; Officer; the are Leon, Chief call Palmer, Chief Francisco Ashleigh Scientific Andrew Bio Drechsler, Executive Co-Founder. Hoitt, Jason team and and Chief and Commercial Officer from

will Jason today's review for commercial PRV-XXX or on call, a with focus the will we Ashleigh On and questions. on and financials, will will PRV-XXXX. then update landscape, call us provide Andy a corporate the update teplizumab open

the First, success include remarks plans the let me projections, statements of expectations, clinical about Securities potential prospects. Litigation matters remind you, statements financial and Private our purposes the will that forward-looking today our time of future for Reform under the lines, regulatory the various constitute our developments, Act These make XXXX. and and candidates, provisions Harbor product Safe we of

Form Actual result by results in Factors may differ most statements, section those on of various with recent Any which the SEC factors, may other we of as filings discussed Risk the is make of and with our on that SEC XX-K the future. views our represent indicated including forward-looking forward-looking in these report important only. statements annual in materially as the from file today those a

of these subsequent at you obligation our point today. on some Therefore, any as update statements forward-looking rely in we forward-looking views to so do to as to any these elect even specifically not date representing future, may disclaim we if views While should statements change. the our

you Provention the under documents SEC. on These website are with section, encourage statements, There to www.proventionbio.com, risks is forward-looking the review information these more carefully. and files Investors documents we at uncertainties Provention's and available in the complete reports regarding

achievements. Chief clinical Officer an now will development turn Provention's and update who corporate, on Palmer, and call Ashleigh provide to Co-Founder, business the Executive I over will

Ashleigh?

thank during you your and today. Sam, healthy hope you, you joining ones unprecedented us these We all Thank and for loved times. safe and are

across living mission. of at report daily While has continued to our execute that been I tremendous disruption pleased there COVID-XX, all to to our team Provention am areas has due

have pivot new As and to PRV-XXX. to achieve Company, BLA our rolling to Application remain able key XXXX, submission or known also been the on for seamlessly we operating completing of Provention a our environment, goal License for Biologics virtual Teplizumab, the track as

dedicated compelling made and now well TXD review our progress as the me been commercial type X path has organization, disorder. as that Let plans our think the autoimmune with recent forward. on going threatening or Provention or becoming life a impacting you, diabetes way to is people life about transforming other to

been since mission which disease has therapy, new for was Our treatment a there TXD, significant create first in for in introduced insulin advancement is paradigm to a no XXXX.

Our of an lead the possibly therapeutic monoclonal even stage to the antibody, clinical re-dosing, anti-CDX with TXD. potential or onset prevent candidate, timely has teplizumab, delay

reactive eliminate Much course reboots cells T producing immune precious neutralize insulin pancreas. Cells mechanistically the to single we computer auto teplizumab would of a the system to pathogenic the reboot like destroy that otherwise malware, in a beta

the the We of which based Journal to results the market, June. teplizumab New are last TNXX published the US Medicine bring on were preparing trial of in to England

individuals the significant as is single was TXD years two a and of at by trial delayed highly teplizumab stage clinical median onset course with that placebo. a XX-day at-risk demonstrated clinically remarkable This relevant. result of highly statistically of This the administered compared to study least

The corresponding to denied. potential be cannot benefit families their patients and

by identified having indefinitely or diabetes opportunity type timely delay to insulin individuals disease an have least the the re-dosing, or being teplizumab prevent For dependent X at end-stage at progression two in intercept with with more or TXD-related first risk autoantibodies. time, to we of as

you might rapidly within market and tremendous excitement TXD modifying disease enthusiasm As --. as we is community, generating ever and the responsibly for and to first bringing the as teplizumab therapy this remained potential expect, laser-focused on

progress and We in recent for to cost pleased teplizumab charting our in last individuals. are months prevention delay designation potential obtained Having or regulatory commercialization. over for clinical therapy the our of with at-risk extremely breakthrough year TXD approval

modules of of the BLA FDA be and the U.S. to Administration. announced submission we and month, for Drug of Food submission Last rolling company's our reviewed to submitted allows potentially the manner. filing non-clinical by in the sequential module completed Rolling BLA the a

three in this quarter submit clinical chemistry or in four. the finally, manufacturing our And modules and of controls Next, to year. we module quarter expect CMC

BLA Our completed Lilly's module contract completion successfully for submission. manufacturing the To of historic we plant process of Eli the CMC manufacturing filing partner, rolling our end, this transfer path in from teplizumab's represents of Ireland Seattle, Bio commercial-scale Provention's Washington. critical in to manufacturing AGC

of first we current report year, the delighted with last in teplizumab. Following milestone to successful of a Bio's another run CMC of completion four quarter manufacturing completion cGMP practice this or of quarter engineering I'm run that important an AGC of good year, reached in production

and to the the the comparability data made IND comment material with at that previously analytical intend from teplizumab submit our and engineering to allow Lilly. FDA both by to the runs to between respect made now and We cGMP AGC review to

or User or proceed final summer. enable and by to performance, BLA process, the our batches completion Fee set submission the determine module, will PDUFA expect rolling support submit PPQ if the CMC filing the date. with Prescription Bio and we will three FDA is These acceptable we Act qualification, Once year-end. a Drug module commercial of AGC scale; at the forward, this Going

the mid-XXXX. in could we breakthrough review priority potential a stage teplizumab's eligible for approval connection to with Assuming designation, therapy a are file set be for which in

designation the review with quarter to scheduling that the in received also from In Medicines we from Recall breakthrough designation for discuss year, last held the a meeting and teplizumab kick-off year addition pathway of Europe. Agency. FDA, received the EMA the the in therapy PRIME to European we first this regulatory

support newly believe Authorization FDA, the TXD diagnosed with the from with Application TNXX As teplizumab or studies at-risk data patients along from study MAA. EMA we Marketing an in of our will database historical that

be this of We able expect XXXX. to second submit the half MAA to in

the the company for FDA indication at-risk experienced in United EMA any or our related not the scheduled interactions the BLA COVID-XX has in interruption submission the in our with the or To-date, rolling of here States.

brought research newly teplizumab diagnosed elected subjects in Phase However, and temporarily we into public to TXD conditions rapidly pandemic, of March global patients. by COVID due the health the in study deteriorating pause randomization to of our clinical PROTECT X about

of and the caution and and and patients this their for clinical care We employees, of operations, took for an measure caregivers, proactive and our site out staff, clinical abundance our contractors. protective for

complete include allowed diseases--. were recommended Patients who were time expanded an drug by the study's data PROTECT we as their study study of undergoing infectious which course at to monitoring the therapy to committee,

specific conditions we study manner have country any guidance when yet within permit do and will that not in regarding anticipate a at randomization as While recommence, sequential we do each will each local PROTECT so it site.

to the with being Let turn made me progress commercialization Jason now Jason? teplizumab over the an update on our provide planning. call to

our Provention's excited you, mentioned commercial Ashleigh and We've strong advancements about Market Access the leadership in afternoon good with quarterly Thank made built year. have last We're Vice Distribution the everyone. who beginning on a President foundation operations I a call. of and since the of we

President of us who of of more experience, the a Architect Chief recently, Marketing, more And joins than Vice blockbuster product with having industry launches. years been XX Marketing multiple

space. and as markets as therapeutic novel we have a product launches and in disease nascent of X launches Collectively, diabetes extensive well rare deep understanding experience type in indications, the

the are that for foundation commercialization most path of effective the teplizumab. position We laying toward us will

covered us the most our payer in month effective vision will representing board a standard treatment. more care lives assist This to for messaging we to building engage advisory of new XX than TXD of a million communicate

burden feedback of how also well will illness TXD burden view disease, as gain they insights as We currently. of and market the the on

last we the of patients screening the teplizumab. our commercialization of quarterly it On to call, discussed relates as importance

engaging offer a family screening patients, their third intended advisory physicians plan In payer of also to campaign, encourage healthcare members a which committee, to the addition in launch we're to quarter. proactively provider to to awareness we building TXD

scientific Our of published mass the Bavaria. among the impact a in about highlighted JAMA efforts positive the recent A of in screening. effort coincide with study screening broader community importance recognition

The efforts way in under for of course are a Colorado. Germany the that shift autoantibodies impact on Similar or with potential Provention. also therapy attitudes Northwestern could in as two is and patients U.S. helping to states more disease such in the

study, Kordonouri, screening. in Germany article Dr. that recent pediatric the diabetes a Science the from thinking" Olga in a "changes teplizumab Hanover, children's specializes who the hospital at magazine, data In stated around TNXX in

direct a of familiar at launch X stated, previously at-risk commercial As type strategy patients are were subset on of our who known diabetics. focused initial relatives

TXD, have stage subsequently be X of total, these XX,XXX relative it the that for individuals. possible roughly in XXX,XXX patients from as literature published patients. XX% patients Family for with TXD apply we of familial would with an having In know screened a TXD. relative XX% members We to as that and addressable diagnosed and identify amount teplizumab autoantibodies many are of believe market with XX,XXX treatment initial will States of a dysglycemia there newly if United you the two to patients

available years. blood and people X.X are have million screened recent more in readily Standard than tests been

TXD. risk in In the every addition, factors diagnosis given there are tested hundreds differential of of thousands, year or

clear, families clinical and screen this feedback highly from managing are to TXD ones. motivated initial caretakers be generally disease the experience To loved and understand of burden with their

into advocacy Type groups this and Helmsley population goal. we closely who further working screening Ultimately, look Foundation, we expand to other JDRF, and a to such forward share this patient with X, will the as broader groups end, Beyond

of TXD known Over direct X identify market greater the who to familial stage relatives who size have dramatically. time, allow type us diabetics and and thus stage a this of not one progressed increasing to clinical will proportion individuals, two are believe we not

by through and subcutaneous and teplizumab management, broaden will therapeutics potential term, targeted we seek life at to looking market beta-cell eventually exploring and with cycle age the dosing, repeat islet with expanding other formulations group, combining potentially the Longer transplantation.

ahead the opportunities the to and the many remainder progress, teplizumab commercial on insights for forward the throughout of community about our year. and you excited planning, we are look updating TXD We and

Ashleigh. you, to Back

more progress Thank We Provention, you, building, making. elite be at about your pleased not commercial the you're team and leadership you're could the Jason. arrival

a progress financial of let our quarter first of you such candidates advancing as results, lupus we and one disease. on targeting celiac continue Provention's to discusses the our diseases pipeline with me update programs Andy other provide autoimmune therapeutic brief Before development life-impacting another as make

to potential in humanized proteins PREVAIL autoimmune of targeting the scaffold trial. have B-cell surface has believe last We the PRV-XXXX we March, intercept early and bispecific B-cell Phase lead we prioritized positive the a discussed healthy of Xb the lupus volunteer as CDXXB. PRV-XXXX our the the call stage topline and On mediated PRV-XXXX diseases, results indication. our is CDXXB

which been with is disorder CDXXB in with and inflammation for mechanism chronic of major lupus, has affect all failure. a organ At are some least with autoimmune system clear can organ X.X patients every established. results a need million afflicted dysfunction and of which therapeutic Lupus proof Americans in forms agonism for nearly that already unmet tissue-damaging severe

our to and the address purpura, look We autoimmune conditions has more Phase well thrombocytopenic potential play in you of diseases during the and orphan as neuromyelitis along as keep way. gravis. of in role updated PREVAIL study lupus rare optica, XXXX, lupus to B-cells forward the indications a half such as first we as commencing will rheumatoid including patients such arthritis, pemphigus, PRV-XXXX Xa the pathophysiological portion idiopathic other which myasthenia in prevalent

the has this this opportunity animal also products, in potential gene from therapy including we PRV-XXXX the or studies with half the associated second reduce immunogenicity of and to anticipate supporting prevent presenting results biotherapeutics year.

now to the will turn first results the Andy? Andy financial will over call discuss Provention's who quarter. I for

you, Thank Good afternoon. Ashleigh.

our I of and begin, would press discussion that read our The release statements, Before to and condition. well as risk factors, XX-Q today. our I was earnings financial analysis our encourage filed financial XX-Q includes management's you as

our cash with cash and current start me projection. position Let

XXst, As $XX.X XXXX, was of cash balance million. March our

three the was months cash March $XX.X XXst, based XXXX Our ended for expenses operating million.

the cash, $XX to of to of operating and million continue cash to be to million $XX projected to equivalents, and fund XXXX, use will for We continue approximately first sufficient requirements current six to securities we operations the our XXXX. marketable expect expect months middle in that

our The net for From a same $XX.X $X.XX general corporate of compared quarter a diluted of generated the loss of share. basic perspective, the P&L or net is the period loss million infrastructure. pre-commercial we in per a in XXXX first result to administrative and including build-out and and costs increase greater,

received New was and New certain Jersey The allows and administered $XXX,XXX by New NOLs program Authority operating Provention biotechnology April, Economic Jersey cash. qualified from the net for the Company's Lastly, sale in in of Development the portion Jersey sell of losses. a businesses state to technology

the With closing that, to let remarks. me Ashleigh call his turn for over

Andy. Thank to-date potential to continue this Throughout and viable in will anticipation will the has parallel, we commercially teplizumab Provention submission. teplizumab demonstrated you, progress transform manufacturing organization XXXX, of company year. our BLA the XXXX. rolling outstanding focus of remainder a and the in In transition into of on launch we

We treatment by for the the creating are first-ever driven therapy possibility of diabetes. type X disease-modifying

therapeutics teplizumab, help pipeline autoimmune opportunities to other we diseases these and associated with potential address passionate to patients with our rich the to their and unmet advancing addition is In caregivers. about are fundamentally need

take your to we now, And call open will questions. the

session. the begin now will [Operator Instructions] We question-and-answer

Young go with Please from The Cantor. first Alethia is ahead. question

for Hey I [Indiscernible]. guys, Thanks the study. just guess is first call. PROTECT taking this on

milestones what restart drive the study to will and again. sort for Just to that you're you COVID-XX related looking wondering that are the of

I some time their immune-based that you still we just more they then, comfortable you're patients. things can Thanks. And an from that are treat working using physicians know on average launch, to educate so about with to have therapy commercial talk but

Thank the very you question. much for

each lift we that they crisis, of criteria will the be level at the other measures and local in to the patients country lockdown So, PROTECT and will that as have reestablish use to a begins the through place. procedures comes randomization into

opinion the York expert appreciate, leader be will our or of be to great country's example, each be can you Francisco, in it maybe respect will status and middle us is committee, begin key who some San to infectious an there country, as again. data diversity We in disease each on each heavily helping randomization before then, may for New ready time sites with access site monitoring including because relying

patients other on more local safely that they those caregivers personnel sites Whereas have will switched allow and can or a and that states back sites the on confidence different manage in site local switched situation of be to quickly. their

therapy is to please endocrinologists with to done ask, that will education going related previously of I'm be comfortable therapeutic intervention. that seen Jason, immunomodulatory he in clinicians and comment respect type particular could an to that with not if get the indication

and Yes, the thanks thanks Ashleigh, for question.

endocrinologists is their members endocrinologists that be primarily around So, care taking on I to of the commercial think X and are going the get screening, that encouraging we're -- family diabetes the type approach awareness, to that focused ensuring side screened. they the and under pediatric patients currently have

affairs X to Affairs, the be together market side, will regard While unmet paradigm education medical that plan exist meet simultaneously medical Adamoski and both putting that education live Medical that on our virtual the treatment. driving Head is to in with continuing Robert include will events the in robust evolving the type needs of a diabetes

approval. gives won't we able promote commercial us until As on FDA teplizumab the the actively the you like product side, a know be to

Got it. Thanks.

is Leerink. go SVB question ahead. from next The Please Thomas of Smith

progress about CMO on kicking BLA Can and off teplizumab rolling Thanks questions pandemic? taking guys. partner, congrats is manufacturing. your COVID-XX questions, Hey navigating the and of how couple the the submission. the the A AGC just you on talk Biologics for

run have they aren't How to I disruptions notable any they guess going you, related their any And are COVID seen issues? any across operations? into of comfortable that

Tom. for you the question, Thank

put remain site an has scale They just at many open together particular contractor their a are So not commercial Bio manufacturing manufacture teplizumab in the manufacturing products, facility in essential and at and AGC Bothell. facilities program. their throughout they great and

no the area peak the COVID-XX as issues. seen the Seattle confidence crisis. right that our gets the of any indication on they have we side To To-date, grows encountered have contrary,

in I far remain in very as environment. be confident so anyone confident can think this we

we're rest supply long pandemic of coming in this can and can the Obviously, place in our making that we evolves and eye doing avoid in on we how delay and watching quite and chain weeks be differentiated contingencies global possibilities everything keep manufacture, and we really proactively and the months. biologics putting is sure that

guess, this these teplizumab Thanks. these quarter, on work payer us of pharmacoeconomic you about Okay, you've you're sort pricing? helpful. Jason through how thoughts off just meetings, engage kicking any updated guess, conversations? then And I talked That's got engagement you're to what in can I it. how approaching thinking then, And undertaken walk about

can that here Jason answer you Tom. Thanks, us for please?

Yes, question, sure. the Thanks for Tom.

So, as actively and of the also additional lives, are XX how million shape I engaging be participants board mentioned advisory way but payers about represent into board view currently is board intent the story both in the build, and get and X ad that this will they to payer month that over needs we a the help advisory that covered to to we're market, that insights teplizumab. pharmacoeconomic working type the thinking

be product the showing how be us approval. time about they them we'll teplizumab of to of core profile coverage able have and give on a a target potential the So, at to them feedback will think data

but evolve the to the and way advisory from decision-making in paradigm actively So, the going helping on setting get they're them thinking is health the how -- prepare gain on how standpoint stage treatment really that coverage exists around the with of are understand and then that as intents to potential context messaging, of about the insight economic into of a teplizumab, insight and market how factors coverage. the and a their board the today, approval an thinking we're on read initial working to those the data

that analog appropriate? given any the not indication in guidance And to have seen you past on is Jason, the why we've date pricing

No.

work previously want that that you about believe the know, think valid. close today seen the it are analogs doing, reason No, this. Tom, reminder that for and we've possible. thanks actually. haven't pricing to do it longer as to been have the to no be that -- relevant formal we'll we I discussed approval, any so So, But as But we talked as is the

to So, have until our a well-informed wouldn't pricing we've the would evolved engaged we the final most date pricing which then potential think group formal leading and as more the research to possible Board, to messaging, us in give knowledge at our point into up we work about evolved thinking, price. of be engaged this a doing announce Advisory participants payers relevant, and approval, the able

in And June. a we sneak guess sense. a if for of should I ADA, one give in format you're meeting, here the virtual switching there, what engaging us at and about question, with could sense that wondering meeting? KOLs just makes That I you Sure. was I just approaching watching how to last how be if you're the could thinking

Ashleigh, do to that one? you take me want

on I sorry Jason, that? I mute. what said, was you would taking exactly you've I'm heard My mind apologies,

Yes, medical Tom, there, that ADA, given so so have so many major of meetings for circumstances it's the been the out unfortunate canceled.

found meeting has a move environment. ADA their virtual to to Fortunately, innovative a really way

and don't will teplizumab on So, be specifically the we we'll and an hope know while participating out obviously, data be presentations, would the be there coming what update we actively watching doing group. trial that would presented, and of

they we're to but fact publication in it limit data, the able how As KOLs. engage you for responsibility know, of have full does

Obviously, in things if that our at meeting up, we the way we of and a to about this and up can really by open ways fact the thinking KOLs a that following to So, part engage latter we'll can at year, we're ADA, national things advisory rapidly of thinking open event advance that start that. so in together level. engagement boards the virtual the KOLs about putting local them engage both don't augment live

it. Got Great.

taking Appreciate for questions, Thanks it. the guys. Okay.

Thanks Tom.

ahead. Selvaraju question of next The Ram from Wainwright. go Please H.C. is

front. and my questions. on Hi, Queries commercial, regulatory, taking thanks for very the much clinical

was if in onset So, the see commercial. on to you versus and strategy the let's the start I brought with sales the population? targeting differences the early marketing would could elaborate at-risk be principal wondering bear population you that that

from population may - the of the work onset label that be teplizumab population the learning data potentially since you for teplizumab have timeline be you're this to to But position apply premature you launch the what indication? the in mean, study know in at-risk is a can juncture. bit PROTECT company I at this target we doing could for and the when population, And and that effectively PROTECT a the early the applied don't as that and what for

Thanks Ram.

individuals how XX% symptoms diabetes, different because at presentation at Jason, this are within very other nascent type of the of the markets. diagnosed often which six One ketoacidosis. with weeks which very is again, clinical-stage of So, explain you they market, you screening requires could diabetic if a identify looking is newly X a risk, are to

a an they to are So, looking referred endocrinologist, versus exactly know where we Jason? they're a where market where for and market we going how patients to are be

for the Ram. thanks Yes, question,

hit So, lot the I right. points think key Ashleigh there, actually a of

and have I be being caregivers launch ultimate the direct of that familial diabetes should diagnosis what patients group families discussed and that knowledge for and our really that screening HCPs we think those at-risk loved specifically talked they while on to indication ones the on during as the for for educating be and types and endocrinologists able X the relatives. advocate get to that about type screening pediatric to offering that implications well-educated is simultaneously using about initial remarks to diabetic the are leverage we've going ensuring and focused protocols progression going both other prepared their X having of really relatives specific screened, and

about care we're that that these lot networks for learn diagnosed the caring that work we starts have as of newly broader team to the the will to direct familial tremendous community the pediatric there, that starting I right about starts and population, endocrinology When pathways about will community, with think a gets commercial the think are our with out now. and referral relatives, insights to engage we engage we

of is system. that, scale-up legwork the to won't with And necessarily But don't perspective I a the things one a to to to that patients fact do the have is to newly infrastructure of this way think adequately commercial are envision we the because screening, market. needing all from healthcare of the in have commercial I regard presenting us diagnosed the about cover attractive the our really just

likely by we're well-established target approved, Provention is endocrinology the the will community to in populations engaging deep have and about exact to time groups will administering teplizumab. and of be we with to the both newly significantly relationships those how providers Since indication Bio, have going and understanding physician the same pediatric and diagnosed acquiring they specifically go a

helpful. Thank very you. That's

On was questions the European wondering if as as I front, you both of could well are answer a the regulatory that here. to the commercial aspects couple relevant

Ashleigh, thinking above. how maybe regarding the optimize teplizumab's curious or to are it combination know Europe, Firstly, go distribution Jason the the strategy value and I'd you're be strategy views or alone in the partnership way best to some strategy, your the about whether of evolving

juncture And also a then countries rapporteur on this whether begin to who date, even was juncture interested the start have with well of with actually the front, know of the at And MAA. might regulatory sense the I seeing MAA formal to submission. the interactions at discussions be regulators as in to you might CHMP ahead what your on European perhaps you

the question. again great Thanks for

will be doing categorically in not this have we stated ourselves So, Europe. that we

So, distributorships. a regional combination partnership it will of be a or

the discussions. could the the the you we in of United in essence, the in Jason, value those distributorship and States time the in difference that to so be XX-month we the here share lag States? have Europe therapy partnering we're Do want And time therapeutic approved progress what and compared to United on this as is more at how the value while and United looking of learning are do comment, sort States, in have of we Europe, to time here from a and

Yes.

on So, seen in as what partnership distributors, own depending whether the the ex-U.S. other mentioned, and I partner, building on working discussions intention we'll geographies of it's through then partner to be remains regional we a how infrastructure to of European the looks and think our be nature Ashleigh based have like, no partnering and Europe, looking evolve.

what get be the said, the pricing we that States. be know value that in able and being in we will likelihood of With will to able all that than get to that United in Europe lower likely we're

the But talk may what to start we of that engage work we're just now help what will I that doing value value the through to they starting that some of it know to we because on of the we're think the European payers to see through work European independently some working with side some want we're that's that And add potential discussions pricing look will partners. like. having as

to we respect PRIME EMAs review meeting kickoff of provided have while. to-date, the any or but we with indicated, we had the but regarding regulatory the the the And interactions scientific rapporteur, following with details will have designation, in guidance we meetings not yet short the

taking us risk to were And at if PROTECT at Okay, into was or this you randomization into steps follow-up to loss perfect. are had integrity you far against the that how on of effectively trial of who pause in the a kind also, issues been give And then paused. comment juncture you you particular randomized mitigate on what just study three quick you items the time already patients the relating clinical the to issues Can data before randomization. PROTECT of sense can front.

And then lupus Thank anticipate profile being efficacy extent also, would what that? to a of I quick the on contemplating are readouts To the elucidate what had that allow you. just program. explore drug the you to and specifically nephritis lupus specific able you question you in

for again question. very much great you Thank the

person question our Francisco you could data the XXXX also details program not us? do integrity regarding lupus that asked. please Eleanor Chief but you to Ramos, obviously Francisco, for take with the those we of And both best the to the Medical have is us questions Officer So, today, leading protocol. he the is closest discuss perhaps the

Ram. Yes. Hi,

the the to not that the affect the ensure that place both data has will have there and have integrity are on regulators. guidance what's appropriately and all been this from down going of of into data, statistical are with the terms happening discussed topic in is the situation. provisions to FDA taking ensure that be all methods of in So, that appropriate account we out come And to the the EMA analysis interpretation specific

we that did conclude We guidance was this provided of have had on we end were time and year. with -- And not target we the by enrollment on the the to not enrollment randomization where at we indicated were paused.

point results year, enrollment next it's much indication that of into paused, are XX-month after So, completed. And the to then to Francisco, we long to be an we of you expect be with enrollment however topline a lupus will at actual announcement the would this are how -- know completion. not unreasonable protocol? follow-up, XX assume speak want as the you further to the do months

you topline is it to design digest And want which the know, data recently trial some appropriately. to to as the with lupus, Phase share very and take Xa For these we exciting. and time reviewed data, our we KOL,

At this haven't and identifiable are likely part we as scenario of And evaluate of time, if be been such going Phase objectively are dermatitis, readily which we determined the etc. is to that the arthritis, nephritis most substance the patients, lupus Xa. will

Chardan. Gbola from of is ahead. question next Please Your go Amusa

a have of then behalf on call. my Lee two Amusa. Thanks Hi, and follow-up. it's I for Sam taking Gbola calling questions

of First question, the approval? seen us mid-COVID-XX on finish you've any BLA pandemic update to rolling in as breakthrough your therapy teplizumab U.S. designation the hoping then advantages you would and on having you're

products both there XXXX, approved. approved run that, in obviously are trials then so given from to combination be parties difficult in when it's fund external were teplizumab do will the more combo seeking yet think not And But to interest be increased you trial? combination to second,

you. Thank

remarks that comments time, the at complete we our CMC and and no are we do on intend our QX, schedule beginning commercial-scale in the delay on summer disruption. at we to there the in seen the in three any back presented PPQ indication the the remaining no So, batches based again schedule, file quarter back regarding of four that to manufacturer schedule this of as see would pandemic, be in our that COVID-XX a on call. the runs, fixed we've in to module module, indication my assuming point

at-risk point an is the side this additional at strategy approval for that indication. and of development other philosophy, all best the our question, combination is the done So, on

enhance the about So, disease we're the of and/or talking combinations delay in progression to re-dosing.

approval onset breakthrough X evaluating to therapy a gotten based this amazing from results of have over sponsored received therapy then having course have also re-dosing. patients then on Our the into combination, then of is KOL the those studies and medium we designation in delay dose, population diabetes but the a approved the single not registries, only get two-year type into will studies, can goal and patients into divert who one

are combination, on a you're to based therapy thinking to work a So, right approaches more that trying of follow-up people now. cell as on

gene in or So, such being protect how through as system out out therapy invasion be see strategies transplanted protection regards from you immune stem the to by strategy? cells figure to as companies there editing. Thanks. alternative you synergistic to to Do would trying to an cell attacked those the teplizumab those immune

Thanks very much.

definitely cell X present as companies. see we those diabetic best and answer have therapy their this, is production cell transplantation are capabilities, their to essentially beta Francisco to the by opportunities developed So, therapies type market other of insulin and but where established lost again,

is relevant that stage, of body. perhaps to We lost it of immune cells, so maintaining believe the that still very therapy recruiting in to play cells be a the beta very necessary role in in protect individuals cell new establishment who even autoimmunity, due at more system significant them the the teplizumab will that Francisco? can protect original have a to lost their

you, that Gbola look And despite therapies gene cells, like are for jobs out. immune and attack will system. the better the cell-based more antigens Obviously, the thank companies by they cells to those more there editing the advances, Yes, beta certain will edit you of subject those do, the question. developing cannot

So, insulin for example, T patients against health Cells against and TXD to insulin, insulin. have have it's very common antibodies immunity

that's So, the edit make of out those so cells, you cannot making insulin. purpose can they insulin because

we this So, as adjuvant absolutely important in see therapy. kind teplizumab an of

transplant cell-derived an to teplizumab or do used We but with good islet been new the going be had synergistic. in experience it's entirely don't published results, with cells. we combination if stem alternative and been know with has that cell haven't it's We know

forward We in that the some future. experience getting field in to will look

so Thank you was That helpful. much.

Hoang of Please from SMBC. is question next Your go ahead. David

recap great to the I all Hey guys, a And thanks really questions. taking had the for progress. couple.

regions U.S. in guess, broader the the then screening So, been, had campaign, mentioned mentioned Denver in format I geographic Bavaria there you Germany have you in certain and area

then need guess way broader you the that of guess pediatricians should campaigns screening as in a what to like. do I'm look campaign, many those looking is there how among to How of just we times curious general be the driven color maybe would a at? And screening? screening and type best will doc of endo what little child, any, I kind terms that age ones kind of are be agreement forward, were I bit on a to they more do

It's diverse Finland. X in ranges for country the autoantibodies diabetes from a type and population [Indiscernible] and question, it a full-blown like and great screening is general and

as then, Davis part screening maybe the in is our pockets And the But teplizumab not behind give Francisco of screening as Barbara expert drivers and is except diversity role those driving States currently or indication centers-of-excellence United general where familial for so could in less population as like that to it in Francisco and around some a areas [Indiscernible]. screening is like make what consortia you research really and are catalyst established. in going what

Yes. Hi, David.

autoantibody genetic consortia So, for typing either with subjects in risk autoantibodies we as Ashleigh most And in identifying number with determining common in a oftentimes is most the of part, Europe said. the have determination. disease method HLA at U.S. of celiac and the or the risk, the TXD, conjunction

consortia. subjects million up which X.X this about have for academic are there total, signed In

substantial the amount So, patients subjects population. a of in general and very

general Some population. members, are are family some

despite participate? to which costs. that diabetic subjects substantial of have of those and We reduce risk life-threatening you has drives you of incidents what complication TXD is know mortality undiagnosed morbidity, the a a can So, that healthcare and serious TXD, knowing ketoacidosis, very DKA,

reduction main academic purely So, research. been drivers the DKA a one and in of screening has of

key is can us grow what screening that some market, and of these based at consortia, the addition doctor's all is of screening these you intervention that teplizumab because on that the going change exponentially, which once we now happening know on disease in screening, offices have will is Now to to expect in opinion telling clinically leaders are the organizers patients we of worldwide. there and this trajectory the be groundswell academic a indicated are

of And covering hopefully, with one day, we'll are country. all they planning be for entire half like to they Finland cover the and screening the TXD population now are currently where

was one had follow-up. That just I Thanks. helpful. And other then great. very Okay,

our time. looking you T detecting whether that indicator phenotype Cell the over sort an on really mentioned at past So I are be think of conversations of persisting an exhausted and phenotype effects could teplizumab

that T is So, lab of the something is in in to that's at a Cell looking something as only terms or or to of be able in teplizumab. would the something clinic, when academic test similar that maybe available that really through arena, re-dose exhaustion, take determining physicians in advantage

Yes.

for the So, ratio the cytometry. importance, the it's because locally. tertiary technique. phenotype clinic of or leukemia, one CDX hand do in have or very can cytometer example It's is hospital a to hospital common has accessible Every on used secondary, determine very it lymphoma. Flow a a the hospital flow test flow CDX, any HIV that cytometry

sample who the And peripheral shipped temperature. they room have sample send don't central that the blood and for cytometer, just is at to flow a the can just is lab those

it lab. it's central So, a a simple and mail goes to envelope

believe help very something re-dosing easy decision. is going it's be that we guide implement to to So to

Thanks. I that. great. appreciate Okay,

back like This conference would remarks. Palmer to to Ashleigh question-and-answer concludes our for I turn closing the over session.

So, and today. we and time. Thank again thank your our hope loved joining this stay on at look ones us for forward you We that soon all to and continued sincerely you progress well you safe updating you.

you today's Thank presentation. The attending for conference is now concluded.

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