NTLA - Earnings call transcript Q2 2022 | Intellia Therapeutics, Inc.

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Operator

Good morning. And welcome to the Intellia Therapeutics' Second Quarter 2022 Financial Results Conference Call. My name is Andrew 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 on the company's website following the end of the call.

participants in all mode. reminder, a are listen-only currently As [Operator Instructions]. Intellia. turn I Relations to at the Ian now and Karp, Communications Investor over of conference will President Senior Corporate Vice

Ian Karp

Welcome of This Thank as to earnings you, the call topics Earlier update discussion Intellia call. like the for company's this that website. and and a to release morning, our duty a This being call make for unless current this management archived website good release minute take available quarter All may company's a listeners on operator. morning, you Intellia SEC Investors progress call. Intellia on second forward-looking as is this filings and call, quarter sec.gov At And potential today's Therapeutics found I of to today, XXXX Intellia's be Media press and statements on and required outlining issued refer Intellia everyone. discussion by as at this this information live, be no intelliatx.com. is broadcast certain presented will would a of section law. can to ask risks to replay that, time, the at on information well remind during undertakes this uncertainties.

focus for Officer. Laura provide me Intellia programs Chief an Financial Officer; begin Sepp-Lorenzino, then will second our Dr. Lebwohl, Scientific clinical John a will Chief are Intellia's vivo Medical Leonard, Dr. Chief Chief to And provide Glenn with remarks Joining with business update Officer; from of highlights. I'll over John recent David Executive John with then Officer; provide an quarter. Dr. and turn David financial overview on on Goddard, we some call strategy. an now that, results Laura Glenn NTLA-XXXX. our and up call the the NTLA-XXXX ex open update before the will John. Q&A. final And from will review will to

John Leonard

Thank morning. editing all building for joining therapeutic each delivery novel and you this in leading based you, genome We're with and Ian. of to ex company. the revolutionize including us harness power we're broadest Intellia, medicine. vivo and editing for industry's vivo applications, technologies to toolbox, solutions At deploying And the the immense potential thank CRISPR the deepest

all During second advance with continued more Starting pipeline will evidence TTR full NTLA-XXXX, the NTLA-XXXX led platform. and provide across relevant progress doses. a we and and the clinical additional the we've presented in June, excellent made study. elaborate administration data deep quarter, to a Phase David recently, the findings ongoing durable to from we've that and recap one-time therapeutically the EASL latest of on study. our reductions In presented X following an spectrum

cardiomyopathy In portion the addition, completed escalation arm. now of dose the is in dosing

of finalizing With dose evaluate corresponding expensive the dose selection arm. dose data in cardiomyopathy more the per kg we're to a fixed expansion to set, of X.X the portion the mg

first-in-human engineering vivo which potential leverages angioedema, and hereditary reproducibility platform. LNP-based with platform. the remain first safety vivo for of study activity in the a an data opportunity NTLA-XXXX unique also our With later allogeneic only NTLA-XXXX knockout to technology, strong a on we our people second as the share year. proprietary of track but momentum, at treatment represent and Our from look has ex will modular and demonstrate we're This this to living our efforts, not cell candidate advancing

focus therapies. strategic limitation durability investigational allogeneic NK exclusively overcome Our novel to making shift by on a we're approach is preclinical data, developing on allogeneic key current designed cells, therapies. rejection Based cell of T the our host a to to and compelling

preclinical development. allogeneic patients our ex an our allogeneic autologous decision pivoting an we share now program, for of on will is version, to recall For may shortly. Laura are in more platform lead advantages which you NTLA-XXXX, and which vivo our program the is

position. a We quarter now prosecute in the this we capacity set of strong $XXX it the with remains the turn Intellia continue ended strategic Finally, forth to which million the priorities to at to financial David. gives year. beginning in us I'll over cash,

David Lebwohl

deep sustained EASL's tested levels follow the in International dose are respectively. gene John. of with protein a arm Beginning led six all thrilled with Liver treatment X provided welcome, genome up. mg resulted period mean the XX, of months At than XX% June. by achieved XX%, levels study a protein the data X.X of a reductions NTLA-XXXX year. per Phase XXXX over TTR at report over dose to escalation causing amyloidosis, At ongoing reductions continued greater in in disease our Thanks, XX% positive X.X we months the with in to And at portion, that amyloidosis, up These the data early time. six durable four for to kg a editing ranging in everyone. ATTR in remain Congress polyneuropathy These ongoing of interim confirmation or one-time treatment results doses, and XXXX the from through durable the and fact TTR maximum reductions transthyretin were editing follow reduction day from

consistent durable halt potential disease. that possibly of to as and reduction deep, continue and NTLA-XXXX's could levels the protein reverse We treatment a believe one-time the support remarkably

in pivotal continue now the in to administered cardiomyopathy the studies. are forward systemically the candidate. with and been dose we potential There XX are to ever first make of Recall CRISPR across safety optimal great both and vivo individuals over objectives NTLA-XXXX who arms, primary have addition, in dosed establish polyneuropathy study. with progress in the move an to both arm the arms In cardiomyopathy

the the cardiomyopathy completion announced of we today dose escalation arm, For the portion.

for this have on that readout data, with been the per the kg we we're X.X per findings X.X ported the similar expansion polyneuropathy forward reductions, initial While fixed pleased evaluation a to arm the of at Based finalizing the well dose EASL. kg We're tolerated. data later dose previously equivalent now consistent have a presenting to share in year, fixed a recent near look at first TTR or and interim X.X mg cardiomyopathy selection the been very arm the from generally had from portion. mg dose

We fixed second arm. the to decision to three factors. was dose in also today dose evaluate plan The polyneuropathy announced in a same dose portion second a main the that the cohort based on we extension of study

the data emerging dose of cardiomyopathy First, the the arm. portion escalation from

of initial number small a tested. is after in data still two dosed, TTR reduction indistinguishable patients doses While

per liver was our day routine dose X.X the The decision. the tolerated, significant in asymptomatic enzymes non-serious been Second, normal of the in events a medical bilirubin. laboratory has in comparability returned in And X.X considered the event while mg informed the kg related per no and A was levels dose possibly elevation NTLA-XXXX assessment. arm. investigator recent by was in has patient well The in drug. mg increase XX arm and was per observed to patient study patient intervention. without this cohort a a third, to kg XX portion and liver of expansion there at generally enzymes polyneuropathy doses performance that deemed in The in milligram polyneuropathy at adverse dosed escalation the

days. X to line with delivers dose the common to amount study evaluate design, disease. patients the Overall, our we expected goal a reflects that arms NTLA-XXXX We As of a halt strong that reverse achieve the strategy amendment the potentially of a and plan levels dose plan of both our in to Phase the reduction lowest submit in to the dose protocol at fixed possible. to benefit inform conviction better future to a result, identify coming this the deep maximum can study pivotal in TTR

dose the cohort at data from released the the the polyneuropathy initial NTLA-XXXX later addition, milligram this In to extension expect XX we upcoming present year. data safety from arms

X are our rapid incredibly protocol HAE. continue And or of of our enrollment regulatory And all in by now to to to the proud subject investigational the angioedema, XXXX. Phase of feedback turning the hereditary end to for progress treatment from we NTLA-XXXX. complete study NTLA-XXXX, amendments, We the therapy of expect ongoing

in have non-human sustained single KLKBX greater its after activity in targeting in be activity permanently to If shown we've plasma compelling primates XX% a shown option and NTLA-XXXX, a with protein the are achieved attack. have kallikrein we HAE and results we where reduction HAE. liver a both data translate relevant transformative treatment in than therapeutically reminder, a and for kallikrein these gene NTLA-XXXX kallikrein reduction Other living dose. As could modalities With leads of that reduction the XX% its activity. reduce to people new a humans, to

later this kallikrein from is Phase and to HAE both of This continue X/X progress initial data profile ex our make reduction R&D XX interim portion CRISPR-based safety escalation in provide pipeline to and view the rate the include data. modularity and potentially on Laura concept initial and to dose LNT These safety, of results study of offer the will vivo dose expected I'll the activity the to call milligram of cohorts platform. XX sharing attack to proof We turn results our and look forward readout year. steady progress. proprietary demonstrate for of over now updates NTLA-XXXX our an and

Laura Sepp-Lorenzino

NTLA-XXXX decision impact investigational Thanks, allogeneic highlighting clinical have on exclusively this vivo David. the I'll to start will therapies recent focus autologous and current our with developing ex on program. our

cancer of the treatments with autologous well key known. you therapies of some As are many cell for are challenges aware,

patients engineering. cells exposed First, an for with for who starting already collecting been provide ideal point to cytotoxic healthy not do have cancer treatments subsequent

response, the and on field present for solutions. In required complex In off-the-shelf addition, manufacturing have therapies treatment. the these in associated costs many process focused further to developing hurdles high

to development criteria. solution needs following of optimum an the the allogeneic However, meet

from administration. readily available First, donors cells be be should sourced for and healthy

with efficient clinical must gene of cost. produce we importance, for last platform developed sequential allowing remains lasting with time, an allogenic anti-tumor attributes achieved long recent manufacturing these to unsolved At for engineered continued finally, clearly able there responses area, persist that accompanying approaches. at activity. novel persistence, hurdle. And And cell a to are cells not Intellia, data Second, a product an desired of lower periods edits. an needs leverages This proprietary be of process key the has have which current demonstrated combination critical

Our both and activity for strongly strategy persistence highly should our preclinical high patients. anti-tumor editing that achieve in responses sustained data the differentiated required supports

with collaborations owned already wholly allogeneic recent AvenCell that candidate. the and Our underpins is a NTLA-XXXX our and Kyverna platform technology

Now for NTLA-XXXX.

the based to it have program data on pivot allogeneic interest the using safety the to advantages product at quality we this We in from high fighting In aggressive consistently to is any discontinue the version of due to will study or efficacy on decision to significant our The an support cancer. the version. the switching TCR. preclinical possible trial. Instead, Phase allogenic an conference an not NTLA-XXXX extremely to as X later is a allogeneic present concluded this by of same additional importantly, of believe as platform an to is to patients emerging scientific plan allogeneic best further in of decision, cell potential it patients quickly type deliver current version of provide XXXX. data Most we

our the CFO, call to host Our continue innovations and CRISPR-based financial platform an second our investigation partnering wholly now provide and on support Glenn, robust of results. our a will research over therapies. owned who I'll overview engine of quarter to hand

Glenn Goddard

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

$X.X net the offset from June proceeds cash part and $XX XX, was fund acquisition approximately $XXX.X decrease the securities agreement $XXX equity as from XXXX. The used of as of Our $XX.X of was to of and Rewrite company's million as billion cash, equivalents December driven decrease plans. cash in to by million in by were in at marketable proceeds XXXX employee-based market XX, operations stock as the compared approximately The raised well for million. million million $XX.X

by collaboration by The to to XXXX mainly million and revenue during quarter with of XXXX. Our second increase $XX the driven Kyverna. collaborations our $X.X second increased compared was of $X.X AvenCell million quarter million the during

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

including of XXXX quarter stock-based Our increase $XX.X G&A to million. compared employee the by $X.X during expenses, $X.X expenses related $XX.X to quarter to million second was the during of increased second This mainly of million XXXX. million compensation related

closing Finally, to With plans cash remarks. fund over operating will our we turn balance XX John the expect for the that, beyond our to months. call I back now next

John Leonard

Intellia. at incredibly you, Thank been active It's an Glenn. quarter

to adapt available. in as continue the bring how experience industry's our I important of Having our progress previously has it of forth of and our decisions CRISPR science for I'm groundbreaking development most significant medicines, patients make made to to medicines need. We team patients a in and some led pipeline information pipeline that best based is from the shareholders. interests confident mission the our to know follow across quickly the the it are becomes in to

and Looking continue updates to the be we and NTLA-XXXX performance year, will note, the advance our happy answer NTLA-XXXX priorities. execute against to ahead clinical at to the look Operator? NTLA-XXXX of our from questions first to that, we as share platform. we'd safety a of well of interim With and remainder about Of development any as plan pipeline the and data NTLA-XXXX. strategic particular

Operator

Instructions]. [Operator

from comes question first Truist. with Lee Joon The

Joon Lee

questions or you attend I'm plan heading into for What are top virtually. presentation August your assuming at APOLLO-B to ISA one data X. two least on

John Leonard

set the validates reduce amyloidosis, is to offending have think all, we're would and to is getting of all see the But particular in any reductions, deep is how some full best test because of really on. of First to able it I We, way a cardiomyopathy of very will these six profound the interest deal we TTR to excited that the been like if very, performed. and you in patient's for as interested benefit Obviously, harder with long studying what take terms endpoints patients expect expand understand, form might levels. inform you of a outcome But important and time, that patients. the don't to we'll know, we're I data think a anything in we believed amyloidosis, a demonstrate are minute about walk the targeting clinical low to about in already really protein we it of think apply what course, which particular result. we've think readout. David, want we how I to to there's be think particular which observers,

David Lebwohl

think who deeper in piece, tafamidis I the patients to did, we of be couple do reductions do we And or that better than interested did think didn't receive of be things seem what biggest other we'll can received how tafamidis TTR. of part we a in, get the they story. because the

in we important trial, might be we as than efficacy So, trial. our will design that that point show better to show seen we've how

Operator

next Maury The comes Jefferies. question with Raycroft from

Unidentified Participant

Hi. Maury. This is [indiscernible] on for

does the harder pivotal? Just on in a data follow up APOLLO-B steps six as like well. or next rely inform for the would as prefer walk test endpoints? endpoint Like, you an how the the you like minute designs like, would your on

John Leonard

think regulators, endpoint not minute Well, of ultimately drugs. six but as a first payers definitive some effect I about the pass, walk test the really it's I has these utility, the most in think terms of and think even how physicians,

there's what minute And walk cardiomyopathy. a doubt the our that's for no think at validation so improvement. we've is core that So, again, program. degree far, readout we to is some of useful any knockdown But David, of entire hypothesis other that of us, thoughts? for seen the six

David Lebwohl

term. some As It's been that trial of lowering than mortality. for we are by don't think important trial. the It's you a and we've show short in talking others. yet while say, It that most cardiovascular be that fairly do this see TTR. events smaller to important also And a you endpoints benefit the will

Operator

next from Gon Stifel. comes question with Dae The Ha

Dae Gon Ha

pivot APOLLO-B number get the because all other of set I questions little questions. a different a a progress. to sure I'm to Congrats bit on you'll of wanted

would the berotralstat the you it rates. follow reduction a specific data see in relevant outline or IND in I as KLKBX safety, the I to And the attack to release heading comps? we lanadelumab? equivalent Is of press up, guidance. didn't or and XXXX, guess, On when terms second the filing what as opposed the half, should But else? it, any XXXX in expected something as into it think guidance HAE be the for about Is

attributes guess, broader the SERPINAX for XXXX, IND that XXXX or I shared there window unique or equivalent filing? gene since now and XXXX, gene between the program is a about So, generally, AXAT anything has XXXX warrants

John Leonard

first. along, the can issue get question we do moves are point, related XXXX that which it. is guidance the to they're When how we'll We programs other, program do we each to appropriate as typically distinct. but The

at is that SERPINA. XXXX a mind. insertion gene, offending And in so, it's keep gene important a different an at targets think whereas that different I itself, to XXXX locus, the Remember

programs. a is independent XXXX are ahead XXXX. of little They So bit

other that sort that, similar of can that departure, each a is exactly benchmark the or taking. way same think you view. the approach as relevant the this What patient. That's reasoning, of from we note tied clinical we've other. TAKHZYRO same seen of that they're that designed But levels. together and at standard separate a here. them them of that to inhibiting more have the effect, pickup point, the in point the care there outcomes. endpoint a point we we of of agents in biological we is bigger to, HAE, respect kallikrein we're With if So, look choose bring all is we've And are sorts met of at the We get from that which is reducing a

that extrapolating and the of to perhaps our out with that be way they've So, TTR objective. course, from the meeting should reported surpassing believe we've our us approach, is, seen on we with which data our minimally which we well them, already, that levels

Operator

with comes question Wang Next from Gena Barclays.

Gena Wang

you long dosed HAE expect liver that more of there? HAE you many enzyme since update, XX do color elevation did safety [indiscernible] milligrams And how patients, rate say and similar a second question last? case you regarding attack how did data. regarding question one XX. one at Can bit HAE regarding reached have also little And then I give for the it you profile day

you baseline if follow a patient? give attack rate? little individual And can comparison and, one wondering color give each the two to what regarding bit If bit more little the can that color, would from Just a up? you or months be more was say, the

John Leonard

is as starts the time. With baseline we'll give that we'll have, up we have, the have give patients the we'll data you HAE, and at at we expectation which are as we the follow show that with much information and

presentation, seen did programs finding backing gratified a thinking, essentially of work our respect when patient as you've laboratory the I that, indistinguishable would some transient as emphasize to between is information on on of we X database and reasonable selections, as work, room believe know, expect similar get X.X actual we've very, point But approach thing It's we because TTR our our was So, one reduction as the our the on Remember we'll possible. we the later that profile influence to be the of – asymptomatic. the do you program, along, general I I will seen the essentially have to a X.X. because that we XXXX that dose don't try entirely want as do, And our through well. has related. safety if doing all actually go what are milligram. do format that data that side excellent very people. since so it's we performance when the outstanding abnormality drug. you in we With to first we TTR the But grown, with been the to that shared it efficacy just as anything we've our with so we've extremely the continuing TTR was principle else script, transparent accumulate Phase It's and been it's to be it. nature. And will dose in did I'd of levels patient very know, and will particularly there's dose But David, much year. this the have we off disclosure a to that information we'll based said in three as provide. the the think as

David Lebwohl

few I questions in think there. other are a

no there patients, elevations outstanding and been there. HAE the safety have liver enzyme been For has the

to events usually how will way there many events on is months, there's this get we and in And In a terms the the And by more treated depending of running follow-up. four are main give two of just then patients a rate, at done they there, be analysis. little looking are is that. after that doing detail the attack a analysis, looking before many there period over at then to how individual

John Leonard

you it's other one thought asked that, remember, XX. is the on just about, patient guess day Gena, I at

reported very idea and out have LNPs around seen the be time important to tend although it class, is finding One day it's low that of they about LFT when we've may transient of to considered the like one, or a which as lot on. nature. which elevations, look three. the no things in the possibly that a elevations, This drug, be think to even related was we so, we class. weeks it's a again, We've with later, right occur, is two we've certainly which if dosing, it is transient

dose the bigger to the think go we're achieve, about got thinking just balances of room lots again, picture to right side. the here efficacy trying we we've choosing and on that So, what

Operator

question with from The Cowen Thome comes Joseph next Company. and

Joseph Thome

when flip a XXXX Just will maybe move clinic? over in how relate into be going that milestone? be for to development? would the nearest term And terms that that a to to Is maybe when allo, could the place to clinical enter XXXX of in

John Leonard

said which the the be cell receptor. incarnation we've this to T date Remember We is same haven't with guided of yet. particular will a next what

their the cell cell WTX well, of all excited the is this that, remain logic we opportunity we T absolutely we most target, promising remember receptor with performed based TCR certainly So, and Car And that is other cancer have our well etc. for place formats. own – one with Ts about at is CAR behind looking biology, programs, and T therapy the those. the think have And larger believe are we

many respect place, about excited to of that. you innards to platform, forward, got with which moving we've the incredibly really allogeneic adapting it's will. So in And we're if our

forward finalize steps, telling So those that that about upcoming time. as you we'll to look that at with and more share we updates

Joseph Thome

Maybe quick XXXX. just auto going the present a follow-up patients on there? to you the if program from dosed data Are of you've any any

John Leonard

We’ll just we what faster, future provide. appropriate. allo that, certainly we to following we as on an are collect you patients the we look going at able safety get not as anything and get. we context autologous that updates, particularly we've what merits in of program, to or are efficacy But performance just superior Remember it allo about think the making based dosed. we readout. format We're really the cells, switch and information based think see better just as any out, to on would is that is think Laura of This we'll laid an share that about be think in be the approach

Operator

Piper next comes The from Malekar Swapnil with question Sandler.

Swapnil Malekar

couple program. One a HAE on them. guess of I

the has regulatory So, safety given the a like to XXXX for requirements chronic follow-up. feedback And program? kallikrein additional And TAKHZYRO of have concerns approval are then any that I there been there on pathway, related during monitoring this program? the were regulatory suppression some what

John Leonard

moving is very HAE briskly.

if don't you know I All been to of straightforward. interactions share? want any David, there's color our But, additional regulatory have pretty

David Lebwohl

doing. happy nationalities, we're multiple in good with the with the seeing. just we're what safety we've this you'll also in put the year, later say program They're And feedback safety are had on place. data but we've see pleased I'd as we've to, that alluded No,

Swapnil Malekar

like X.X enzyme one pursue of safety equivalent, regimen the dosing mg elevation fixed was liver per looks to the For of that. reasons and key kg

elevations, that any like, like enzyme question the had the date, patients So, minor that ALT I of dosed patients other there even if have to any elevations? any is significant, or incremental who XX not or are is, total liver

John Leonard

Certainly, EASL of level the In outside you'll we've think presentation, that around normal infusion, excursion presented. at one . the At some Well, information. time who get of an an you've was seen low of I just we've presented day just of patient very XX. the that all had example there data limits the last increases cases,

So, examples. that there's in other yes, sense,

Operator

with from next Richter The question Goldman comes Sachs. Salveen

Salveen Richter

And could With you regards second rationale you that's dose your to a switch get timelines? understanding here? the better delay side. sense and separately, here on for the AML do TTR trying the behind just expand in to to the the I'm just on decision to this study, a study work program, of allogeneic allogeneic anticipate much the will development product pursuing of your on construct foundational a how

John Leonard

the Maybe first. you can so And David timeline. and on the then say if how through the allo it. go any, Laura about you to why about a thinking impact, any have word I'll about take we're David, excited But,

David Lebwohl

we looked showed per milligram mg kg. per milligram saw program. the mg reductions polyneuropathy up per and you was EASL XX see really with at At and starting And forward it a what we X durable was X.X to better went that. mg is kg per back we like patients kg really it, from through where there where take the me mg deep Let point, than X slightly kg that X.X

said now, have As we than we've more patients. XX

We of kg. have per of at X.X really mg patients multiples the number

per that mg it's indistinguishable. efficacy data, quite mg kg really at are clear X.X kg that and with So X increased the this per

doing well. about with this excited We're – the really enrollment the investigators So and program. of very that at point, are see rate that you

kg both is very to of this What order And per cardiomyopathy. the the year do say. curve. how that in study everything we end with question. In much of per really on gives cardiomyopathy a we'll flat the we've as in So to dose still did enroll this a what program has mg that, product mg down to patient lot – the that the we're going and development, want we've you we patients patients, we efficacy. dose have recall XX And pin seeing we forward, you with X.X kg the go with information lowest response by And do be guided learned. from all able to that

John Leonard

you the little and think Laura, to a about we're about how it bit pivoting. why talk want and allo platform we

Laura Sepp-Lorenzino

learned but study, is what goods for allo for compared importantly cells efficient high as to allow manufacturing, an engineered periods that also resulting of is continued in an the solid these to with But in cost from is quality, must therapy. of importance patients not long we've lower anti-tumor critical importance a time What of only to our platform that for autologous product others, allogeneic consistent of have the persist activity. is

So achieve, avoid T been having we want host allogeneic only seen is we we provide we not have able approaches for the two don't cell that programs have that we what by to rejection the graft disease, versus very that is that cells. And current bring persistence. cells, which rejection or long-term killer important And host importantly, natural believe for by like to others, ensure avoid are from for that about we persistence. as the but And sponsors, excited do benefit we really or therapies not clinical responses several months patients. complete month with last other really

the sharing more be data the actual and cells. detail to the We performance data editing on year, continue into going going in share to strategy of later are this

having to at time. forward looking So, that this conversation

John Leonard

manufacturability Salveen, the it's important to also about think this. of

use time literally same at you're for brings patient time, a people XXX this And in achieve gets lags, even opportunities of autologous tremendous the outcomes manufacturing time, One of perhaps to XX think with can particular, of sometimes in efficiency, one And cost, that expand at we can approach lost terms brings format. the we with tremendous the that with products. of better ultimately with the etc. each them is manufacture just able improvements one autologous goods an things the these persistence to think which current cost that you're if

that's So move And excited. quickly. we're think very very, that can along why a we program

the well, timelines, down So, a when I our the and although that, guiding start not look plans little at yet, we road, look later. allo the we're from standpoint of think may

quickly, it performs the very, we think it levels finish very expect. can I assuming to that

Operator

Issi Luca The with next RBC. question comes from

Luca Issi

you think X in that of per cardiomyopathy. kg X.X indistinguishable I kg to TTR back go times couple mentioned But to mg the a Sorry mg or this per were one.

polyneuropathy, obviously drove TTR However, to beat a better the a in to X does look kg which is that knockdown, me per important it mg silencers.

polyneuropathy you but understand, kind think the to how between Maybe wondering IONIS' serum love your hear thoughts the small difference mind us in data? in PKK with PD for would cardiomyopathy? if the your see keep XX% and to antisense in numbers, on right to as knockdown difference. how rationalize oligonucleotide. I HAE, show Again, Wondering that's So of on bogey TTR we that you rationalize their XX%

John Leonard

we Well, to on get the with data more see to remember, respect you XX, a than do. lot

And say know so, you about… I certainly think how David, two if don't this word informs about to want that stuff. we a or

David Lebwohl

see about more And data, a you it. give actually. me Let detail it you little our could in

this their you by we per an but with X.X driven outlier who's patients when kg. actually the per X.X polyneuropathy, from standard mean at mg kg – a mean And not If and at per actually mg mg a reduction. recall, were is kg average only an kg, for patients had wide looking X from X.X per also mg patient, single outlier, the

this different. So quite is patient

looking of in these happening be really like the patients. it terms for looks a probably outlier what's – looked Now with something in sample, cardiomyopathy that results isn't would that have that good not not is at to at

we reduction collect continue kg. mg the on – at polyneuropathy react data, to we're biologically will per pretty does it we TTR X.X identical modeling cardiomyopathy what do and the the on patient and X patients So will mg kg both look based seeing also same be to per with but think our that with much

John Leonard

use was, for benchmark we just what the other question as guess I HAE. again,

David Lebwohl

benchmark a is we're about TAKHZYRO at reduction. what looking Yeah, – the XX% gets

You able recall TTR. in we're our And with preclinical seen models, a XX% you've what get to reduction.

able think kallikrein. good that we a will to So, target we to with get be reduction

John Leonard

We anything to the we surpass that way don't think is haven't we ultimately it. beat or seen think I can about guess

Operator

Rich with Suisse. Law from comes Credit The next question

Richard Law

as you to and studies, the demonstrate its changes benefits regimen that, XXXX, you to anti-histamine to elevated the looking enzyme, in follow up liver is And are then test for safety as make steroid a you well? walk be question the you what about think regards outcomes? label to the tafamidis For think in for to six showed need competitive registrational minute do

John Leonard

study of a space, we so. obviously, others the David, tafamidis, first thoughts? competitor the going think regimen, any think that's different But point with there, improving the design that. approved about think a knockdown you standpoint are respect to ultimately be in that? to on We with the appropriate upon position our any out substantially tafamidis, With how relevant surpass that a approach for the to agent. have that? what's for in to studying seen of drugs what's about we're thoughts do and for entry that outcomes patients. that to other our an who good respect believe from you it's appropriate pretreatment It's any because as should David, of And think we've we do

David Lebwohl

do there. a people that think think managed you've time. repeat the over disease are it tafamidis. I to better cardiovascular of Just And the we the than the way minute doing and mortality BridgeBio were was changing endpoints The six events the most data, in really days is walk again, in important is seen

patients second hospital, to looking really we're are terms less cardiologist, healthier, The walk can general keep in we we're what in keep of who are useful we become to a patients about the longer? more, from of is alive measure maybe hearing part… can is that The benefit thinking able out benefit. and them real the going So,

John Leonard

change? regimen for Pretreatment XXXX, any

David Lebwohl

of think any are With changes needed. don't patient, they quickly down treatment. any without we No, that type came very

need we the So, don't pretreatment. to think change we

Operator

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

Greg Harrison

toolkit to You've going overall? technology How you new mentioned capabilities about it add could advancing And forward? year. your where platform do feel you this

John Leonard

you that. think My the Laura, about guess into ask like was would is you question Laura a insights to toolkit? that hoping any give want how you

Laura Sepp-Lorenzino

Yeah.

as really by unlimited editing the want as modality modality. gene to we the delivery be well So,

for applications. So, vivo well as we're as investing both ex vivo, in in fronts

we tech are data to sharing our experienced in But with we team, more we will research, that robust when they ops be ensure yeah, available. tools So, working develop are really colleagues deployable. deep, and leveraging very collaboratively and very a

[indiscernible]. can we So,

John Leonard

reemphasize the going view a the available to modality more to than modality or notion point idea just things that There's toolkit, biology, want is from And of homing with that pursue. you. of have everything of begins one anyone you on for that address working that's would no either the all proteins. engineer we to I single CRISPR/Cas basic

always access whether start approaches we a is skill accumulated. be to cases derivative goal so forms think area, of all And that we have those that in to or But of here go certainly with want it's tools. that have to which that that and deep can you our use other particular on combined,

about the we're in want particular and unlimited capabilities to think that to problem claims. we have those to the we engineering to of in to that think terms be way So we genetic our solve And able on we space. make we well being want want

Operator

with The next question Securities. comes from Mani SVB Foroohar

Mani Foroohar

potentially into little that way? that? you've baked to manufacturing As really pivot flowing you a the R&D that meaningful reason Or we are infrastructure, ongoing specific on the bit to could sufficiently a through investments, is comment if infrastructure of on in think about kind underlying about built technologies out more the think CapEx tools allogeneic, focus the there of spend, some the that is and

John Leonard

an is in patients Just cell the our and equivalent I them that from effect element But the to be. approaches produced to any the budget thus be need think themselves, cells, do to of more as when to that healthier spent, see terms be that. put an it Glenn, And benefit, receive, cell What a big I actual color Mani, run. engineered the other interesting in at a high of be, level, they approach. Laura is per when want ongoing think in, persist autologous But going near should R&D downstream you for the don't the emphasized source to I where In an think thought. efficient there don't reagent essentially much and term the a there's material going add other with would you manufacturing to you a more that? what words, that it's is we're going far effect. missing ultimate critical

Glenn Goddard

you've it. John, No, hit no.

think making thing, future. one establish a this really mind in listening I year relationship Cellex remind we'd the to that is, too people pivot ago we in this had just with a that to did the

from established we're think the perspective upside. relationship I well externalized an So on

Mani Foroohar

something approach, scale missing of didn't up. I is, the the of don't performed scale ATTRACT. something likely your the I have if TTR. of clinical an overall benefit one survival I party was editing unlikely follow ask on trial like a a that's to about change. I genome approach whatever showing for the really Obviously, feel it's look But statistics like

six past study? cardiomyopathy paths for pursue So, clinical going path we do the cardiomyopathy outcome be minute sort you editing compelled pivotal is should whatever of a TTR genome even in that some of feel the of endpoint, in do you pursuing in to kind to Is walk, plausible an expect that Or approach? etc.?

John Leonard

in, through additional lot of different from done. models and we're and to more information out around Obviously, recognized look as there cholesterol for Well, expect has factor conditions. to formally as with a But approach that that rates event which Administration would already us not would the gives I'll other that confidence just closer be, first we working But a surrogate about load Food design statistics been disease is that's and take that. the the emulate to David that's a comes say, the will or respect like. the will And that that coming and in from work inputs And that here. that regulatory at viral Drug world. trial markers, yet, and pass the I cardiomyopathy that are the agencies are

so certainly outcomes, that far, thus measured, would doubt for look effect. to that David, regulators has TTR no of readily is stance there's been of So the process irrespective but anything? clinical actual the speed the those TTR because the

David Lebwohl

a understand this what going them achieved TTR are again, result agents – type see And really those study. of that points. to with of better the advantage We're we getting back as the need than deeper study that can we this none the an think what results to reduction reductions, achieve. they'll I that any do outcome of the us wind and We're of we help by a our sort to because anticipate out. to of see main the with see seeing figure we to have that Alnylam all can

Operator

The Jay Oppenheimer. next with comes question from Olson

Jay Olson

what genetic diseases. from gene human about publications? novel disease was indications Are related I follow Intellia comments or provide program a pursuing that which been could interest there broader effects they editing. be revealed in germline as studies And cardiovascular at metabolic those a have as certain such for publications And recent or up, disease mutations? protected against on seem you to level protective your is showing curious any targets mutations observations interesting of share you that targets targets, to liver related

John Leonard

can that you But benefit? to me the sure you you asking for physiological question question? a asking understand me question, make some about are are germline I somatic Or your mutations have

Jay Olson

specific was I can publication perspective, Well, germline to suppose. But either I referring you the from question was answer the a mutation.

John Leonard

something and it and in we was for you addressed some it on. technology work as the fact manifest a we patients, mutations could for and that way somatic that thinking to appropriate carries the going. themselves we the really what were on point was biggest we change. of view, that with years certainly genetic from all, the us And about where readout germline today where respect where do to But of First someone germline work a benefit as any is ago not make just mutations, see that's get from

ours, the where opposed as level somatic as space addressable what that stands reduction after. extent look with all targets risk the reasoning that done the relevant particular would is ask the that's have go been to that so, level? some at certainly draw behind going to genetic germline benefit, mutations, these for which after clinical that's informative some etc., we of kindreds to to line. So, And we we of there's the But that

Operator

The next question Debjit with comes Guggenheim. Chattopadhyay from

Ry Forseth

Forseth Debjit. Ry for is This on

do And enrollment? or in Assuming challenges authorities ATTR of in around the an approval you CM, foresee need siRNA requirement you how comparator? do think about

John Leonard

David, do you address? to want

John Leonard

in an that approved, are course, study certainly. studies What we as if as don't currently, And comes done of approval before will Certainly well likely be a have Europe, running any active our is the silencer being comparator. see, all US. the

we our feel point. study would an exactly So that yet – this do haven't about need comparator is. design But we at what active talked we know we obviously don't

In this what we're terms of enrollment, as doing. mentioned, there's interest enormous in

we're that trial. I do interest those And of that to has depth patients in with think reduction things one-time treatment going with think well. as I a our of the that the will for fact drive it's a lot

Ry Forseth

The not typical follow-up. XX of PN showed subject And bodyweight one day note elevation, or the the anything that that enzyme to for individual?

David Lebwohl

No.

John Leonard

don't difference again, starts substantial of we mg X patients, the again, kg, we And that we Remember, We direct see and almost which, at really think you related. as that just said infusion, it. them extremely of to the just infusion. LNP open patients don't respect point an did it's the information well efficacy all as between look the as we're with XX has But at being was patient the efficacy that additional the thoughtful to very have, million, of per And sort very, from at with as this when drug a we accumulate finding related an day view. the study question X.X it, that have we It's done, accumulated who even transient. with David point. just is it's know out, this don't a pointed we've

easy. the makes it decision So pretty

Operator

comes BMO The question from with next Biliouris Capital. Kostas

Kostas Biliouris

a to I myself but couple of will one. had limit them, I

So, on at of this occurred reached. day that the And when mentioned is the liver these XX. is TTR enzyme elevation, maximum you inhibition exactly

probably with do unrelated? view, month wondering has very I'm your this So two one in inhibition if, anything the to are or at TTR high

John Leonard

David, thoughts? any

David Lebwohl

– these believe liver going the changing the any can't changing. of connection between unrelated. think we are enzymes TTR We function down and Yeah,

John Leonard

equally which have all who patients we other And these there's for of no… course, inhibited are

David Lebwohl

Yeah.

Ry Forseth

higher wasn't really of than the So, this inhibition patient the others?

David Lebwohl

Yeah. No, not distinguishable.

Operator

then with last from the Zhu believe will come Wells I question Yanan And Fargo.

Yanan Zhu

question. two-part have a very quick very, I

by that taken TTR per I us outlier kg reduction You mentioned percent know is outlier. they reduction you Could if now, XX. out? X.X Right skewed think is the mg let average at the

just If a you great. around have elevation i.e. but unlikely response know transient ALT CasX? be that you then on if that ruled a percentage XX, have response, that. you at outlier, can specifically day us is immune would cell out I without expression, that out to know the ruled because wanted for you to inform And this T

David Lebwohl

be the XX%. than percentage Yeah. greater The would outlier without

Yeah, will it's we possibly we cell. a There's it of detailed don't related. causes. that. be out Again, rule lot just So, a mentioned T more than

routine found are causing We discovered this. that any looking, we of the haven't what's testing

have don't an answer. we So

John Leonard

asymptomatic. we we just have, It if this And they an outlier lot a question much report seen. vigilant quickly. laboratory on out progresses. we work. we've any, just patients that insights we was study get we go. is or How as proceed that bear had, carry have any we'll Again, that don't the not? a this disappeared our we're as and finding mind of that as And in the that But, at is, Remember obviously, point, patient, we learn the And as know. more, we

Operator

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

Ian Karp

to for to your updating Have everyone forward Intellia. of interest your and questions all today, us support day. for and much joining continue Look we so a thanks of progress. great And great Great. and continued as

Operator

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

now disconnect. may You

Intellia Therapeutics (NTLA) 4 Aug 222022 Q2 Earnings call transcript