Intellia Therapeutics (NTLA) 23 Feb 232022 Q4 Earnings call transcript

Company Profile

Participants

Ian Karp	Senior Vice President, Investor Relations and Corporate Communications
John Leonard	Chief Executive Officer
David Lebwohl	Chief Medical Officer
Laura Sepp-Lorenzino	Chief Scientific Officer
Glenn Goddard	Chief Financial Officer
Maury Raycroft	Jeffries
Greg Harrison	Bank of America
Swapnil Malekar	Piper Sandler
Terence Flynn	Morgan Stanley
Yanan Zhu	Wells Fargo
Rick Bienkowski	Cantor Fitzgerald
Raju Prasad	William Blair
Dae Gon Ha	Stifel
Debjit Chattopadhyay	Guggenheim
Rich Law	Credit Suisse
Jay Olson	Oppenheimer
Brian Cheng	JP Morgan
Joseph Thome	Cowen & Company

Call transcript

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Operator

Good morning and welcome to the Intellia Therapeutics' Fourth Quarter and Full Year 2022 Financial Results Conference Call. My name is Drew and I will be your conference operator today.

Following formal remarks, we will open the call up for a question-and-answer session. This conference is being recorded at the Company's request and will be available at the Company's website following the end of the call.

As a reminder, all participants are currently in a listen-only mode. Instructions] [Operator

turn Investor to at Relations proceed. now Vice Karp, Intellia. conference of President and the will Ian over Senior Please I Corporate Communications

Ian Karp

you, Company's Earlier this to Intellia issued This topics everyone. and call. the Thank Intellia's the release on year quarter morning, good today's outlining as press be fourth as This the full is call. intelliatx.com. Therapeutics Investors website Media this discussion be quarter Welcome earnings will XXXX a on and operator, progress release Intellia morning, live for found Company's broadcast of & section at replay and call being can website. a on archived well

potential a Intellia this today, time, on take current forward-looking like call to certain for is I discussion sec.gov you no remind at statements to a and undertakes and this of presented ask information listeners by uncertainties. law. to this unless required Intellia would available may duty this filings of management our SEC that All as make risks to and At call, refer information update minute that, during

Officer; Officer; Joining Laura Officer; Dr. Medical Chief me Sepp-Lorenzino, Dr. Executive Intellia Lebwohl, Chief Chief Chief from Leonard, David Financial Goddard, are John and Scientific Officer. Glenn Dr.

call then some Company's the open with progress will then will John the programs. And Intellia's begin across before XXXX. and for concluding John will update results highlights. will our remarks we full financial fourth David Laura quarter provide will R&D recent an Glenn recap business review an offer for up overview year of clinical on Q&A. the pipeline. our

John. turn now the With that, over call to I'll

John Leonard

are At leadership broadest delivers and for thank morning. this solutions. and advancing industry's genome as deepest editing you, all the the you Intellia, and joining of toolbox editing with innovative most Thank novel our company, us position Ian, we

for our two. we our another NTLA-XXXX patients were clinical our clinical vivo Having reflect strategy durability and first in significant more proof-of-concept candidates, be accomplishments two dosed of against two studies vivo milestones safety our steady than These years applications. clinical for for two data harness out Intellia, vivo and proved list execution CRISPR-based ongoing pipeline. Notably, near the patients. immense XXXX in for demonstrated core have outstanding and across CRISPR-based and XX achieved now both to technologies, human in vivo to year NTLA-XXXX with power several with ex with now therapies

Building this have momentum, ground year's on the hit we already strong last running year.

U.S. of we patient initiated U.S. NTLA-XXXX this We are study the pleased FDA the portion outside trial. an in enrollment X the application of also share today submitted enable that Phase to to IND the Recently, to we

rapid we the hear expect are in coming have from progress encouraged weeks. months We the highly back by just few to made and past the the in FDA

for to by to ahead subject cardiomyopathy are amyloidosis trial IND poised ATTR anticipated end pivotal year, NTLA-XXXX, initiation Looking midyear regulatory for the submit of manifestation a we trial an with the feedback. a course application of for the of

NTLA-XXXX ongoing forward to interim in new Importantly, clinical both NTLA-XXXX from studies and year. this we look presenting first updates human

T-cell we allogeneic insertion gene its are capabilities technology of This designed our both and in first advancing to NK-cell pipeline, platform includes and vivo engineering mediated our Turning the research kind CRISPR-based targeted to avoid to solution, a clinic. new cell rejection.

further to editing with us our and gene and new pipeline will allow reach modalities, of of development the leading drive genetic a patients greater number growth delivery medicine. which we're Additionally,

across to David? Medical greater over I'll introduction, Lebwohl, pipeline. our and that will NTLA-XXXX hand Chief our Officer, call review who detail David in updates and NTLA-XXXX the With progress outline clinical

David Lebwohl

of we living with begin shared with halt additional disease dose XXXX, and In interim presentation serum reverse American than potential of that may a of Phase XXXX. treatment and These everyone. which were clinical following a demonstrated review presented people the breaking late consistent from TTR data November, the a welcome the reduction dose in greater trial cardiomyopathy Heart at XX% of XXXX. arm I'll Association positive John, for single Thanks, a ATTR the X oral ongoing results Scientific single amyloidosis. Sessions,

well at level. The the and with patients. events. from No were either observed transient dose reported all reactions, clinically cutoff treatment sustained only reductions which ranges observed XXXX related to patient laboratory six was XX as data XX date. the patients of of resolved Two which quickly deep was follow-up months, four the were in adverse abnormalities significant infusion generally tolerated

deep, these protein believe of best-in-class a regardless to agent lowering XXXX's continue potential consistent and manifestation. support of reduction levels durable We TTR to be disease

last for of subsequent pivotal enrollment studies. the completed portion dose to from and few these dosing will our the the be for polyneuropathy selections planned the arm. used expansion months, cohorts In decision inform Data cardiomyopathy we

study For feedback. regulatory ATTR end application a by CM, pivotal we and to plan an to initiate subject plan global submit midyear year to IND

present later and to interim clinical this plan we endpoints. as as year, new durability emerging data and safety longer-term clinical Additionally, including well important data,

amyloidosis continuing ATTR authorities regulatory including ongoing to the for For clinical prepare with Phase with a we and forward look data are discussions from hereditary study, X study. to presenting polyneuropathy, Phase X additional

for these Meeting in ongoing Asthma XX led to patients milligram demonstrated in sustained from XX. week data the hereditary American Scientific in at X/X range second additional shared November. plasma the data XXXX our I'll off, Annual cut were deep presented of of or Immunology turn Phase XXXX, all and to dose week this from a attack now and We kallikrein treatment past study in clinical of program, angioedema and a through robust reductions therapy kallikrein which The HAE that XXXX to plasma the vivo XX With HAE. College results positive rates. reductions our milligram single cohort, XX investigational Allergy,

the date. Importantly, week Patients XX observation pre-specified cutoff XX-week two period all XXXX have levels, completed before data data of we in attack mild presentation the this milligram who was date, primary tolerated as events observation all patients and XX cutoff of maintained cohorts, period generally an in this severity. the so cohort these well to dose the dosed majority completed on year. later not presenting yet three adverse look status cohort risk and attack to were the forward the rate At have

single We address preventing burden XXXX's faced the observed. by significant HAE permanently these laboratory data adverse In living significant related were a swelling attacks people events X events were Grade higher were clinically adverse potential dose disease the those reactions, with or limiting debilitating serious no events, abnormalities no X which one speak believe frequent No to and infusion within to of by day. mostly resolved dose. no adverse Grade toxicities, and with

durability, We attack Phase cohorts. look including rate three of the across all X additional study data data forward presenting to XXXX, term from safety, in longer the and portion

XXXX, receive which the which As January, to John XXXX We're awarded a and Intellia the was ILF pleased to aims for accelerate the licensing designation, pathway. for to program. to market provides entry to productive passport been and UK's United mentioned, facilitate time the innovative medicines. first it's innovative months very access In Kingdom access patient especially in year, two the innovation of

in an patients expanding United in in evaluating application expansion country support FDA, inclusion on that to XX States, To submitted New application announced forward placebo-controlled to is Phase participation portion of look with and coming the site the IND status of recently providing an and Intellia that months. you update the we the the portion review. study randomized Zealand. initiated X two X We Intellia we and doses, milligrams in Today, study patient screening of anticipate a XX has Phase milligrams.

standard XXXX of As we with to we and closer XXXX these for a care people living moving continue serious are diseases. setting progress, new believe to

I'll over now our platform efforts will call hand CSO, on advancements. R&D Laura, provide to the and who our updates

Laura Sepp-Lorenzino

base the new Thank technology Intellia platform vivo our growth of as to allogenic stage insertions, next We're capabilities clinic, advanced entering such editing. at gene as David. you, the and in we

the of progressing For lung programs half to both targeted insertion, we're for in application platform. second This year. we wholly-owned disease, or which and IND IND our vivo partner of candidate in treatment the modular insertion equivalent AATD-associated this submit wholly-owned includes our for NTLA-XXXX, plan leveraging an gene

we're we're important collaboration AATD. activities for programs advancing in NTLA-XXXX, hemophilia as our for making a local Factor with treatment for progress parallel, Regeneron, insertion advancing our In In people third the candidate of B. manifestation IX IND with also enabling vivo liver

partners, ex to we're do. our advancing our the demonstrate vivo gene proprietary with the Turning capabilities platform. untapped pushing what but further therapeutic platform and pipeline, research our can utilizing so is allergenic potential we're These opportunity share boundaries robust there and of ours still more multiple editing engine, broad of those programs, already

technology. and have the We proprietary of DNA development with headway writing our made rapid significant

versatility. and explored genome our in pipeline. capability, being Intellia's Since of We're the and potential Rewrite the expertise, target the and we expanded by beyond us leveraging acquisition diseases robust and newest have potential demonstrated currently performance those the implemented editing toolbox to excited platform offering our of Therapeutics, platform

And now full who call of results. hand provide quarter to XXXX the Glenn, our and over an year will fourth our overview financial CFO,

Glenn Goddard

strong morning a pipeline Thank that to you, maintain allows our Laura. on continues and balance sheet Intellia to us Good everyone. execute platform.

XXXX. billion cash December of securities XX, Our and X.X marketable equivalents compared of to December XXXX cash, as XX, X.X were as billion

increase stock in employee driven proceeds the million from $XXX.X million $XX.X offering, program at in was net December the million based following The of $XXX.X market by proceeds our companies from from plans.

in used in acquisition fund to operations offset Rewrite the $XX approximately Therapeutics cash The million of million. increase was the part for $XXX.X by of

collaboration increased fourth compared XXXX. million million Our by quarter the revenue during of fourth XXXX to quarter to of $XX.X $XX.X $X.X during the million

million to This growth the by during our support mainly million million during compared R&D increased of driven Our $XX.X the fourth of programs. programs $XXX expenses personnel these $XX.X of lead and fourth quarter to increase XXXX was quarter to by the advancement XXXX.

was to expenses to million G&A the of the compared quarter Our $XX.X quarter related during $X.X of by XXXX. related increased This million primarily fourth to increase expenses. million XXXX, employee during fourth $XX.X

our expect we plans next beyond balance months. operating fund to XX the cash our Finally,

the turn that, remarks. will back now for With closing over to call I John

John Leonard

you, Glenn. Thank

executing steady As and add to we're to to are the and capabilities. clinical against while you forth on sites. advancing towards forward X strategic This expect look new year, we which U.S. bring late platform NTLA-XXXX our making study focused innovate our stage CRISPR-based of therapies, of priorities, can see, the continuing progress portion development Phase

cardiomyopathy manifestation We're for also ATTR trial initiation the focused of amyloidosis. with pivotal patients a of NTLA-XXXX on global for a preparing

provide programs, we further in coming the updates to both plan months. For

continue on vision to our reach the the impact we rapidly editing, we the platform accelerate to by our for expanding of deliver Intellia gene promise maintain broader patients. of As on

With be now you the Operator, to happy Q&A. may questions. call open that, answer any we'd for

Operator

now from [Operator with Please Jeffries. first We the The ahead. begin Raycroft Instructions] question-and-answer question Maury comes session. will go

Maury Raycroft

FDA requested ask my and do the taking I IND that in you Wondering on timeline going progress was what the program? IND. And be for anymore X? expect question. for exactly the to thanks if HAE a the on commenting IND included Congrats the to typical for you in what you're and acceptance IND about Phase

John Leonard

for good comments your for. Maury, what with question. It's FDA thanks we've communications application pretty the looking morning, prior made in of and standard is of some aligns

this where ongoing were other may are that, pretty States. from IND and from point view think the able from a the to outside along clinical supply I we differ is study far done of United INDs we information clinical

standard were, to update of very, it's information. complete pretty of very careful course, stuff. we But So and a provide careful otherwise, that

Maury Raycroft

And follow-up. Okay. maybe one

streamlined bit could potential patients pivotal how and design types a you in little more can HAE? if of Phase you and talk about study leverage data the the X for Just

John Leonard

you David, address that? to So want

David Lebwohl

Yes.

of So, it and arm There pivotal arm, and idea arm X X is And will in a study. its shown select dose the the placebo but here and be we've patients. XX arms. the XX at basically XX, three Phase an an slide, for at publicly. the size milligrams milligrams, It's to XX the our --

at seeing results pharmacokinetics. terms some XX much We already X, have of and XX the the good very obviously all three Phase same in from was doses. very information really excellent

good, milligram choose little as a terms look So, safer but in possibly dose, a is well XX the And quite of we dose lower different did dose. did pharmacokinetics. being a

Phase use our It for. already main informing will trial. additional the be getting will ready which information patients, thinking about eventual BLA we by It are the to design and trial. the X, the really the of that's contribute but So, about that's

Operator

with The comes Greg from ahead. next of America. go question Harrison Please Bank

Greg Harrison

future the ask wanted sickle thanks cell and about taking how landscape. morning, I good the you curative question. thinking Hey, to are of the for

do win investment beyond competitors you next be Now that some vertex discontinued the what approach? have space, the CRISPR will the there? how And in do think step you

John Leonard

Company. even thought from we Yes. the question. of Thanks and an certainly one the about stage for one the It's early important that have

what however, been think that's is challenge The and seen I that curable. patients. that, question news great have we our answered, I has I is for where think the make patients, of think we for progress. mean, that in sickle it's cure. judgment that cell of we in those nature I application is can believe hand, The editing is the significant editing that

the we focused can transplant as disease that doing an setting, since case really where the outset, the been bone Western So, mortality that comes in patients That's solution for in think fact, morbidity we very, marrow with with solution most broad-based have thought a avoid it where reside. outside have one vivo in about even general that. patients cell markets, more it's in patients. the a a for standard And I sickle very from

first. wide there open from perspective And we based that's we we seen to is that have intend what on our So get space and focused our where efforts.

Operator

Lee Please question ahead. comes Joon Securities. go Truist next from The with

Unidentified Analyst

morning. Good Mehdi This on is for Joon. Hi.

on asking strategy sickle what what quick of And is type following cell? will now question your And your previous is I be that So, the and have follow-up. edits a follow delivery you're up mechanism? I same would the for

John Leonard

hasn't in the setting. on vivo years changed always for strategy now. Our been focused We've some

by Just news, space. only vivo the followed to ex to that an them be application that has and the was clear in respect one and Novartis others with been

that but we've on For we've any of in an is think be one disease setting. editing that is our particular I learned view, always potential point we focused is there's the approach think terrible. us, the edits And successful. number we've not From can of disclosed, that what vivo

a well So, it's in works and matter that that's of robust making and really sure done a way patients. delivery, is that for

Unidentified Analyst

it homologous would color techniques out domain color that like to And is your appreciated. please Any similar you could to provide recombination? on some similar system? underwriting there is existing it idea [indiscernible]or Any be in more

John Leonard

in we're most in enzyme the specifics. we've change is, one terms disclosing the there, the proprietary how and which develop. get interested DNA the of is not particular capabilities, edits. in, think what we're of the it We gene to Yes, about resulting worked is our and case, In specifics the that resulting you system

ability great great do introduce fidelity excited and with changes the great we're So, our about frankly. intended that and and activity, to specificity,

we progress a excited in her to are, as and about very, made So, we're making. on in mirror where But we're needless to results it, we very as will we about more say, go we're the headed. position the clear Laura be where share remarks,

Operator

The Please Piper next comes from question Swapnil Malekar with Sandler. go ahead.

Swapnil Malekar

of in So trial you an had readout having pivotal previously interim the mentioned cardiomyopathy. the ATTR likelihood for

new So how analysis? decision the an just and data in release that press you endpoints informing the like mentioned interim clinical of this wondering the emerging new are emerging

John Leonard

disclosures what trial. results to you you data just about want do that? with pivotal not ongoing just for the David, be clear, I interim sharing to we're talking of a Maybe address David, fee? plans clarify are could their

David Lebwohl

analysis, interim will I an be terms design, of in trial clinical mean, yes. there if just

yes. Okay,

you So But have the endpoints events that some that questions. we're we're important we to we other now, be large about It know, trials you've exact have these cardiovascular for of think mortality. there. And seen study a the designing the to out details. Phase trial not X, doesn't talking the and said are as

because analysis, because best could that trial which that looking But and have best at in big that we we advantage be that whether analysis we're the will of do interim positive the and from expect we efficacy a in TTR, be trials, this we carefully possibility. the interim how see on trial. valuable here, design other to reduction may early And also have to would our have the trial considering as results will start We'll an inform we trial. help are

Swapnil Malekar

with I one mentioned patients is, And than patients have programs dosed some mark. across you follow-up had so of XX there like more the two year reaching that been two

dosing? that Thanks wondering any questions. if just require So my or in three TTR the were patients of any there kallikrein, for taking relapses

John Leonard

I across drugs. seems seen see these permanent. this the our effect time reduction is think to We be studies what with you've same in

expect clinically what we effects seeing So and to based do now we're the permanent. be on mechanism based on the

Operator

Gena The next comes go with Please ahead. question Barclays. from Wang

Unidentified Analyst

on for taking morning. hear Hi, [Rashida] Can good you for questions. me, is This Thank our you okay? Gina.

John Leonard

We can.

Unidentified Analyst

Thank Awesome. you.

you investor to submitted how are a you've NTLA-XXXX, the recently IND? communicate community will the how disclose able Can And For to we expect the release? you outcome? press

John Leonard

We're we submitted giving today. when not IND. sharing a the We're broad update

review will course of results have information. so, the we of And that FDA when of that's we but that the that process, part share

Just as outset here. we from you promised, know the

stay have report. So to and we'll results hopefully some favorable tuned,

Unidentified Analyst

think new the we And that great. endpoints David That in I endpoints expect mentioned other on much. prepared I it you in Thank a expect elaborate XXXX, be like XXXX. other follow-up. quick you on? would remarks, later Can On had data so what very emerging can to

John Leonard

David, what in store? lies

David Lebwohl

patients what the trial have with information the would from so probably the the most Yes, we're who longer neuropathy. patients where looking at, would possibly we have been

score, patients the things the symptoms. the is gives the on we've information from plus which similar NIS, most slightly but looked Some NIS different at neuropathy include a X, NIS

thing we're the both patients is amyloid. in is polyneuropathy at CM and looking Another This cardiac in patients. present

information that are of the year. have we this those So, may enough on things later types

Operator

with ahead. The go Terence Stanley. Morgan next comes question from Please Flynn

Terence Flynn

primates. preclinical And work any one if if second chain impact not of might CRO Therapeutics some I at broadly Thank I'm just I timelines? or that was think your you. sure think business any announced wondering, a or or yesterday, was additional the but for issues just you tools need you non-human need you the know again, there's this more just which then you the a you And question, wondering, of think I it's development all deal, thinking not, have applicable about John? supply you Rewrite broadly, pipeline. as do point, with kind but did do

John Leonard

have impact that thanks the BD, it the that doing. respect to situation. With the Yes, hope two any I that news work We're Yes, current aware we about for question. on that same the think we're we read today inquired. and change, guess. there's we that's but doesn't not And ways,

You asked DNA. that complete, able introduce that that capabilities is changes different into part terms in strategy to and the an terms of toolbox about think we important have of is it in be in to terms those about want tools, of of we a to our

toolbox. We think great We outside. what's on got a the we have happening watch

and think would own new are We toolbox. our the we look we to augment efforts this with eyes of capabilities open. our satisfied have we scientific things But that pretty want will for do. keep internally, course, we At point, that the

collaborators. approach are BD about is all think we are seeing we we of broader thinking some some for kinds of opportunities, bringing deploying which toolbox, to it. advancing into not wonderful I with What the just of advancing us things that are which ourselves, is,

last work own have we in And that we four collaborations to augment that, XX have the different that different pipeline. months, do had we in our struck, four collaborators the

are with Kyverna, cells in and work So ophthalmology, autoimmune O&K. These we hand AvenCell to in spin vision, more. NK we sparing exciting or with with whether do our it's the applications of out already disease are capabilities expect really

that, the for ours, with that's commercial different pipeline back if replete rights of from those a that company opportunities. a at wonderful think especially each you look that pipeline clinical rights we collaborations this this I we have in And and you have, see is like and our mind, step bear size, of

So, good going it unsurpassed. work that a sure to with toolbox, of remains lot make BDS internal

Operator

RBC. Luca go next comes from Issi question ahead. with The Please

Unidentified Analyst

on questions is all Luca. for our on taking the for congrats and progress. Lisa Thanks This

trial. on the pivotal Just one cardiomyopathy

as maybe why here, based Just clinical program? expand further cardiovascular six-minute one disease, decided share the and trials can primary be will Novartis data to the another to sickle any you. available, wondering, Thank walk if outcomes there maybe two you a And you position test discontinue if on it? endpoint, is in And the on with study? we cell with should expect

John Leonard

through that, to been can't that their pipeline we speak just for been in, ago. Novartis's going to of I appears would they only what reagents approaches one it. Yes, provided to has some broad program, review other not have I a us, have them different than to a and have be we that you remind directly decision Novartis their participated years

I'm vivo that of some and the We and at we vivo approaches sure have some that may with have we ex had lies space looked in that's they on future always the had believed the the work same focused been ago. the realizations that do. years

space you that where But trials. apparent will the to it's pivotal actually How answer do can think about many want David, I question you do? go. ultimately So maybe

David Lebwohl

seen is the to not the But are events how great have idea you the but are that understanding that's we in. those and of are dividing test don't are a exact But program, interested patients in valuable amyloidosis. or telling trials important in forward. into the we it going interested in And walk mortality think again, and interested think two more Again, six-minute questions the we cardiovascular things some is how trials is in that doctors about are endpoints patients recent doing. in, are the what payers

So, course, for most regulators, very are it's interested what those in really we're interested factors. of

Operator

question Yanan Fargo. comes next go ahead. Please with Wells The Zhu from

Yanan Zhu

do this of X study? treatment rationale the arm the in a appropriate terms approach? Phase type or this I also this in placebo cure complete treatment for Are you, wondering, for On could program, is the this what's and for Or And think was still of for this kind different the of the outcome HAE reason of having outcome onetime kind goal approach? expected be approach? of you there

John Leonard

role placebo what's does the what play? David, aspiration? And

David Lebwohl

Sure.

placebo controlled standard the trials are randomized the gold placebos and trials. are So having

a seen can Phase that and arm a trials it's not placebo but also Of them of to arm, it's many trial of strong effect course, in -- comparative there be in having that's inform the don't little XX course, benefit to we placebo risk expect have on attack in have a the the placebo to effect arm and but X, to we've no that reference arm. not the rate trial a both of XX and very will help choose that, expect the dose. And or the us what we again, doses is strong and effect see the help design pivotal and and to

It completed. contribution should study once part cross active to the course, And because drug of making the that and their the going our their get arm of that. part as be and of offered they're patients mention evaluation desire of their potential we this of over this placebo therapy will the with to get is cure second to type into do

idea of the this we're cure we can with early whether idea what is a zero that So, it's functional treatment. results. that and say, attacks to And encouraged achieve you single is could to early patients get by too very the this

that deeper getting the to are achieve are with out in agents and reductions We best there. kallikrein

So, have it have possible lead no to a aspiration attacks drug. or also will to cure And that after receiving better functional efficacy. to again, this our that patients this is

Yanan Zhu

If I be program. XXXX cardiac might have a a quick for follow-up amyloid readout on mentioned the the that you

is stabilizing resolving expectation the be the amyloid no is the that amyloid more just or addition? is cardiac it that Just wondering and expectation will that additional

John Leonard

system well The established there. been

rate course, that the levels, the get the will this there area to from amyloid investigators do that levels TTR, and very at that, to due small key you we of into if we we're that the therapies that deposition will other TTR the the of amount other the heart be low so able reduced amyloid be the Of to greater equilibrium hear would to TTR than from is enough of get be point. words, achieve. the removal have amyloid addition in will that of low way. of in idea aren't you blood But the organs the In

heart is rid to amyloid. possible aspiration disease for idea our we're So, reversal a is that of that that's the And getting that what of then by trial. the hoping that

Operator

The next Please comes Goldman ahead. from with go question Salveen Sachs. Richter

Unidentified Analyst

when you for on having are study this how regulators this And of do discussions both also mind BM? CM and pivotal Thank on for Could And in nature global timelines regarding have much information? you're that [indiscernible] update. NTLA-XXXX, confident IND the any ATTR, get provide some with you Hi, NTLA-XXXX, is the about regarding the you regarding for so you in the Salveen. acceptance? color we'll

John Leonard

including how the it you we regulators can with up the run and address variety about Maybe I we've FDA just in this. to IND, think I a this share had when to the information. way, the to of we'll be David, position interactions respect in with would pivotal a say various

we're questions boxes, that wanted We extent of the list to address. questions, that it's goes we're data It's just helping the we've believe they not checking us product. that that we're to addressing answering but evaluate and to the of actually not them just to that sure making the provide those tried the but really questions

space. over that's is the the their to FDA information bit some supplied consider years Additionally, clinical think respect as that think played And a outset, evaluation. with the I information, they of other last few I said for do at out which in unique the we we've to of the helpful as INDs have that

with we've review, their job respect we subject So, terms everybody we the Obviously, outcome. to of done said, of the in to think information, we the information. that as look supplying of robustly review, outcome when it's to forward have same but will timelines, time. we've a we share And and good will the not

in want approach the we'll position just a to that share you about how when to few a David, words be and say pivotal we information.

David Lebwohl

designing with and in of have both investigators have John in leading IND setting this Europe. the discussions the that So, in in have And clinical our a coming gone all we us with pre-IND that that this design, aspects regulators we said another submission, the supporting trial around world, are what on of area piece trial. with about forward

for have IND which that CTA do well And middle we'll applications the of the with that the the And have And year. for will the time around start the of year said of the that go point in for IND X as by thing Phase the well. the other end on Europe, the trial an said is we've we've XXXX. to trial the that's as will at agreed

we So have we that's you. share sort those have details, of the more with as you timeframes will

John Leonard

I the XXXX distinction to from differ add respect keep in one to mind the for might application. which listeners IND with important XXXX,

pivotal about We that IND would are an trial. a pursue talking

So the end it's not would It's respects, supplying FDA. in also work. some just typify information of meeting all X IND with an the with an that Phase preclinical

So, do it's have the application which very different XXXX in that from very, a we the when certainly substantial for be most we filing expect and fact the from different INDs XXXX in applications. of space other is to first-in-human it, been

goes this. we year sure, be talking more So will as I'm on, the about

Operator

next from Please Bienkowski Rick ahead. question comes Cantor Fitzgerald. The with go

Rick Bienkowski

taking Hi, all Good progress questions. and thanks on for Congrats the morning. the everyone.

For program, the initiation U.S. Phase the submitted be looks FDA. X application was with a staggered the while to IND opening bit to the the XXXX first hypothesize trial worldwide

So for the review, in the does have question after FDA is, any outside mechanisms my study trial trial started? endpoints or are sites IND either on there trial any design feedback if U.S. already has protocol the amend the after the the

John Leonard

you trial, a consequential. want I'd everybody this to to want you are some X so, is that? But David, of mean, details study. It's do do not to and Phase to pivotal remind that? less David, I speak speak a the

David Lebwohl

of picture. the when around trial the world, will that, big usually, not Yes. submitting I regulators say have details should about often different you ideas different trial the a

and either So, the the think happen, just ideas, the Or outside it to various direction we can that yes, to for it's trial. mechanism amendments doing We feedback amendment but to trial which for But there reasons. follow that occur that, related have the good for is a sometimes certainly could U.S. process there's is there is the be very good U.S. very possible in helpful. from the that, sometimes

Rick Bienkowski

got Great, Thank you a follow-up. and quick it.

pipeline editing and technologies base difficult So, is a clinic there gene far the be from LMPs. the writing, to guess in away of early it's to are getting few a now, these novel into or address. technologies might get able proprietary indications technologies of these sort what I sense all how

may So, are there get the stage of any earlier substantial technologies? some timelines progress for for more we these updates when

John Leonard

haven't particular any We or to guided products pipelines.

the advanced knockouts. makes of we have a simultaneous bit that generally hoping platform about have tends setting, primarily about. spoken allogeneic very, space about that where our sense. is in ex we little we excited many prior very, base presentations where we to and to most vivo are It editing introduce very one And work applications fall very have talked We it in in the think useful

And fit respect so, into that stay you in to sit we encourage would updates tuned that with may I they or to and where see. future will they space

introduce it's intended in days With are changes progress we really the of to earlier approach, able getting gene to we of that are high making looking very we levels but terms are do. specificity the and writing excellent

progressing find that the So the on continue and as that we'll and focused And what we more demonstrate we to current think appropriate with XXXX for where technologies we a these along, that. any we're mattered meanwhile, about XXXX But way. of in think making moves progress. are patients meaningful that we the applications talk that's sure really and are in

appropriate. as we'll and share come to updates those more So,

Operator

Please question next William Blair. comes The go Raju ahead. Prasad from with

Raju Prasad

XXXX? Just of Why kind the applications just XXXX IND is clinical of much data because higher? XXXX Did come of XXXX it wondering, and the for the referred larger? and the as cadence wondering because come amount Was to the durability on going I'm in you ahead -- side to the of is earlier, submissions.

John Leonard

acute is but following getting work which of have at point its X. Phase timelines the add I likened substantial period have some which XXXX the editing actually necessary filing application. just to that, Phase anything these a It's in patients really the at the short-term clinical essence to unrelated more I typical the results, David, proper for It's exposure information, X information complement time IND, than if And the again, just work. begin an that's IND, human to first of out. it. support will to to begin XXXX point to in far to XXXX the a full be not you meeting X way Phase to of part is what is the of important the you're that mean, an saying that

David Lebwohl

the important timing. features are the Those of

Raju Prasad

follow-up. a Maybe quick

rationale follow-on, the wondering why? QX on just of Just in behind the top wondering ATM the

John Leonard

want Glenn, fundraising? do speak to you for

Glenn Goddard

and use an opportune QX just, the thought end year early of time We Sure, the QX was yes. last we to program. at

then that. opened advantage Just based on volume. late was of how on December opportunity up And do trading we the and November, took follow the stock in to

it just out. So wasn't to things preplanned do way. It's how that worked

Operator

Dae comes go The Please question from next Stifel. ahead. with Gon Ha

Dae Gon Ha

question with stick again. IND the just I'll

programs submit, that publicly So what the the extensive review So with regards many of from to do submission? and or your the extent the to limited that you XX-day obviously of think competitor is disclosed of the to factor it limitation extent XXXX, how FDA we're case you And pages a will pertains And your be very I in nature guess, within address companies? to period? as one depth of peer what to you fully concerns? what's some the

John Leonard

I to that's that the the use. But read not FDA pages at the and number metric and can't fast we various speak the how of reviewers them.

to the large, information posed because we try tend but Although, regulators. of know, there's shared, as the you be questions these very applications a by address as very, lot to

important FDA kind is, not And drug think it's, space, principles robustness that tried thing to molecules, but system. the we antibodies, stuff. in not I data, it's an to whether And testing our how whatever to these really to experience, thinking, from emphasize apply it's are just general, the development of of about apply prior small that doing the knowing thinks this you're but just

smaller we robustly as work, we'll questions address quickly the that in IND know, complement of what clarification the can't sure as other them there's to possibly best major to nature they if us. respect a to we as it, answer etc., answer With can. them. available. we I expect is The we But our and post think see. review I full do I'm FDA don't have they don't will on have we as But it, that. questions to We'll the people, will deficiencies information want of

Dae Gon Ha

with more or they need yes, where just binary a just to John PDUFA to hold? extension be there clinical have regards does IND it is question, analogous the something review, naive to a time to a Or review?

John Leonard

it's My XX understanding is days. that

You in if follow-up, a has when provided to in a formal by in you're ask a go answer FDA a very of but looking and clearance that's provided of information little a and full position for then if terms is it to they're then takes or stuff. there's again, for, way, them longer get through see, best hold, we been additional the a this them. information We'll hold, their believe what to there that the complement

Operator

Please Chattopadhyay comes question Guggenheim. go from ahead. next Debjit The with

Debjit Chattopadhyay

an provide how treated many you the been patients including stage update dose clinical Can have of across each cohorts? program, extension

John Leonard

of and A keep we'll it at think collectively etc. already terms different in information high-level over is been lot I have different groups there the XX the of patients studies, out treated.

current. complement of is information of in will full second to later most patients fairly the So, on year continue this those give you that we information. and better phase the Obviously, of the study,

Operator

comes Please The David go next from Lebovitz with question Citi. ahead.

Unidentified Analyst

this the for David. on Debanjana Hi, is

reduction and kallikrein you So, we in are you collecting of wanted the provide data timing And addition the to respect data also, with in to to any ask, activity further XXXX? levels? can if us color protein in

John Leonard

flow crane, data to the the to speak with respect what Cal is of year? David, actually we're collecting to what XXXX program clinical anticipated this do you and across want with

David Lebwohl

protein We the those Yes, presenting more kallikrein year. activity. be so are and of towards collecting we data the we end said will

XX able haven't milligram XX be zero, and in to step a We and tax terms call but later of dose of reduce that's to year. XX, The that XX because activity we expect, attacks both then dose, I sort fill they said, good XX is response. and found reporting our back XX to in is we and that as to a course, of get will a this of on to XX reported similar important XX to as piece as data to were dose we went that we should we

Operator

The next from ahead. Rich with comes question Credit go Suisse. Law Please

Rich Law

the X the hold dependent example, a is HAE timeline for on not you. Phase Thank U.S. receive study on if such you clinical that sites, IND.

in You completion trial can the the run ex-U.S.?

John Leonard

can. We

Ian Karp

Mr. repeat Law, could question. you your please

Rich Law

sorry. Yes,

timeline Side. you So dependent not that is is the Phase the X that, study would HAE define on question U.S. such the

X run study you if can hold Phase trial the clinical So affect wouldn't development U.S. of the entire a overall the completion? and outside get IND, it for you the

John Leonard

Yes. to mean I that yes. simplest We question the can. answer is,

Of is and very, course, to the Sites we hard U. S. that's priority participating are and going to very to expectation work that's something have the do.

Rich Law

group ATTR for about pivotal? you or external And the use control you also control thought have will active group for PN what an an

John Leonard

designs? a you position polyneuropathy are about talk David, study in to your

David Lebwohl

Yes.

exact giving not are designs. We the

[patisiran] not been the arm. used to so very what's seen or have in, of as all comparative for with the We has the The historical have active that have you disease of about data reliability pivotal far [indiscernible] that submissions next been an And trials happening in some example, patients. do used the these submission. the and

have details. we We forward we talk coming are will we that looking at all that about design when be and the with the will

Operator

from Jay ahead. Oppenheimer. with Olson The question go Please next comes

Jay Olson

update you thank the progress. on and Hey, congrats the for

and reason particular XX any milligram For IND? XXXX to And apply XXXX, the the plan to there or dose? XX pursue Phase XX using about not for Thank And lessons the then XXXX from highest any you the read doses learned study? you. that talk rationale can you milligram X IND the in to the are across

John Leonard

think to speak will that. we the how doses about David

I in the applied do are programs. doesn't through, the But can the think the going programs, would will see. differ across which if to XXXX. with of we prior looking same respect those for has there before been we to they regulatory things information basic read that philosophy agencies, certainly with engagement of sorts both to with or respect And that Obviously apply, we is XXXX is that. feedback

be be XXXX part be in nature. we pivotal clinical material, selection substantial Maybe program etcetera expect will distinction, again of to of be the -- the X? of But in programs that in pre-clinical more all information, them Just terms similar. quite and would dose speak moving XXXX of want Phase to you which the think we trial to basic review, dose draw the pivotal to to a far progression program is IND will more see that the selection, and advanced the nature where

David Lebwohl

the rather were study in out the dose This to doses method zero attacks. it planned preferred dose the in levels, X. really always a agencies case, picking three this safe, two than right they was addition to coming is X Phase regulatory of And lead the understand all So from placebo. to Phase all best to

see similar in what pharmacokinetics more we XX reduction. little calorie of terms XX pharmacodynamics, we're saturating we think a very very reduction was challenging. it's So in milligrams, that so reach did milligrams you about is similar, the crime looked time But the by

why XXX potential higher think additional chose that do not optimal as as we a is is risks a in you to to things. We demonstrate we to that is bit doses you in for of of a those of might to activity. that But concerned like thought, regulatory crime. higher pharmacodynamics terms the reduction it. aspect little XX%. you as sort with this was too be a of based We're chose one good. need XX in you reaching lower less dose a better saw, second calorie as All of XX% get we And But on the to higher the important high go again, of, the doses instead towards than really going about agencies, are the dose safety all, dose because so dose. going that's It

Operator

JP with Brian The from next Morgan. Cheng go comes Please question ahead.

Brian Cheng

orphan clearance markets. especially TAM? you the if gene benefit, orphan for thoughts the the disease specifically in to regulatory feedback, bar in the the with any larger how provide handling curious, some vivo indications regulated can have editing larger for non of on are IND in compared for Any they in risk U.S. the around indications We're smaller

John Leonard

to how speak assessing is can't benefit I risk. FDA the

the it's approach example, factors, should about application tried careful to clear In agent. for very, choosing risk which patient we've Treating has that single exposed own be editing pretty benefit an disease of an of its very that to the actual approach, editing in states challenges. terms is every

I be do think case made to good a that. can

think you're apply dealing But therapies, that setting right to is to risk illnesses of or the particular backdrop where risks when a mortal post great those point take in the my And patients we can benefit patients regulators, different with evolution tried of that, guidances approaches. in about the at do with for this that of illnesses these I released, the think, I in sense but some a one they've considered experience FDA has modalities. what shared to view think

they the I'm assess by as of will it one indications, that by various one those conversations other so that. sure as some do these And sponsors play to out they're are brought up

So that would be our experience thus far.

Operator

today question last & Company. Thome with Please The go will Cowen from ahead. Joseph come

Joseph Thome

did one Maybe that timing cohorts? initiation those just is IND HAE of one the of How see time? terms just you the from clearance Or extension appropriate trial on can is to at main want the wasn't certain this why factor there have of U.S. And you then the in IND? first. pre-IND of gating that CM a amount maybe follow-up a formerly the just meeting or before follow-up a

John Leonard

the to speak nature of our XXXX. can FDA David conversations Maybe with in

with intend without trial, global means which the now a about it respect for least to wouldn't pivotal sites. we at begin be program, Just sights. way think pivotal those trial, U.S. to it We a 'XX U.S. we

extensive information, in to includes X Phase As that for looking a very tantamount what the clinical is we've said, filing. previously, robust filing we're

in trial. you data a work material what certainly certainly When to that collection want pivotal be available is. the this of That's a well. commercial used application program, of that as underway, to you have is And will be do part a clinical part your final

advanced the hoped. moving we program quite as So is had and

tuned. stay would I So

more share we we'll David, want XXXX? it. information, anything about you to And As have say

David Lebwohl

for we questions said in here, that we've we pre-IND we've this. that the because addressed have So before And in we raised they've have that have, meeting formal a did process.

Operator

I Ian like turn the to concludes This over session. conference back our question-and-answer closing any remarks. for would Karp, to

Ian Karp

the we your Great. for throughout so for as certainly us to today we years. joining updates And everyone and providing additional thanks And much, continued forward interest. look progress

have and a great thanks So day.

Operator

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

disconnect. now may You

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