NTLA - Earnings call transcript Q2 2021 | Intellia Therapeutics, Inc.

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Operator

Good morning and welcome to the Intellia Therapeutics Second Quarter 2021 Financial Results Conference Call. My name is Jason and I will be your conference operator today.

As a reminder, all participants are currently in listen-only mode. [Operator Instructions] Following formal remarks, we will open the call up for questions. 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. the Investor to at Relations turn Vice Karp Intellia. now of President and will conference Ian over Senior I Corporate Communications Please, proceed.

Ian Karp

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

me review John then the the John? remarks we'll call followed to R&D CEO. then will for make some let recap Q&A. will Glenn an the and Joining who will with the by Goddard, Chief Officer; the Dr. over turn up Chief by quarter. Medical on business David, John Officer. today David company's will lead call highlights; me clinical Officer, efforts and Laura Leonard, results recent Financial XXXX; on open today's update Lebwohl, for Chief company's Sepp-Lorenzino, Glenn, that will who financial surrounding Officer; With Dr. are our Chief progress; closing call provide and John call, Scientific followed Executive Intellia's NTLA second Dr quarter On Laura the

John Leonard

Thank past company patients. Intellia, Ian, may CRISPR-based us been in systemically first data therapies. became and ex report revolutionary out today. genome The our patients for you, for applications to well our addressable in This to significant treatments. be milestone represents both advance battling platform fully-realize for technology. editing vivo that all genome mission and of as therapeutic our in to for therapy CRISPR-based in have joining modular Nobel we months June exciting develop a few across that notably, Most broadest To the deepest clinical the as thank we've a medicines. with Intellia, you and end, solutions promise incredibly diseases delivered our to deploys vivo toolbox of set build the curative At editing differentiated prize-winning

causing genes selectively disease For genetic disease, a we're genes or system to lipid LNP-based delivery proteins. nanoparticle desired or inactivate precisely produce insert to leveraging

advancing Our these homogeneous quarter's system that cell significant product strategy made epitomizes ex progress a we've Across designed cells. efforts spectrum immune is vivo approach call. natural eliminate robust cancer to to our last produce since patient's full a

serious in portion interim In analysis dose in safety We one-time this results XX. in polyneuropathy. for reach. patients therapeutic targets at XX% encouraging gives pursue new for with not indicate NTLA-XXXX day as important patients other by the non-viral ongoing the treat first liver and support in and delivery from data reproducible current for only steps clinical ATTR maximum levels, results the elaborate level, the standard which to the June reduction for no landmark with of these the Intellia, detail we of in XX% will a data a production. These a the exceed dose two medicine. protein generally this profile a XX, disease NTLA-XXXX's amyloidosis adverse serum program. data both positive led landmark potential with day medicine More to for added to Our It Notably, may David ATTR highlighted genetic in broadly, NTLA-XXXX treatment knockdown via dose-dependent path. six as of open us cohorts by the offer these results cohort was diseases At platform. be profoundly that confidence and originate events mean care data our with believe for second an a three outcome amyloidosis, the for patients, of trial presented X on of view TTR also escalation included more development interim era curing These next concept NTLA-XXXX. well-tolerated in response. treatment dose within the for Phase our the Shortly, proof but patients

or In hereditary our the readout, and Beyond regulatory regulatory CTA acute leukemia CTA knockout the first this HAE NTLA-XXXX submitted morning or angioedema submitted first NTLA-XXXX, of to vivo trials are human begin AML in our towards clinic. for authorization, we first candidate track for both NTLA-XXXX have marching for end that shared as June, to that continue lead-engineered announce in we NTLA-XXXX on we're pleased to year. TCR authorities the by our myeloid this T for cell therapy and well. our we Pending NTLA-XXXX,

new professionals company a construct with cell T Life we in universal clinically-validated CAR Universal allogeneic together engineering This Intellia's Sciences, Cellex combines a platform from GEMoaB. launched with CAR company. addition, T cell In new Blackstone

our a co-commercialize Collaborations company options U.S. Universal the In with technology, Intellia are which from company equity therapies reach is two technology rights of allogeneic of Cellex, candidates of exchange can patients. instrument and expand will financing landscape. potential retaining the the diverse aims key T our manufacturing enable full broader on core benefit countries. us develop ex beyond autoimmune They a later. for to vivo a one collaboration immuno-oncology along resourced in areas our brings two of new differentiated a for receive bit to CAR our corporate will while additional to this key extend the significant scope this Additionally, Laura technology this our strategy realize focus, co-develop agreement to and to across and of European which up commitment this a a concurrent with million a In stake license strategic to Blackstone. with $XXX of and like

will no General recap warm team: to mature. great a Relations our as passing our hand like David? more Ian to prove President be to will the David over now President before the Medical Intellia and them Finally, and and Executive doubt [ph] ew call Senior company We're Vice members to value Chief call have NTLA-XXXX Counsel; Vice and continues Jim of data clinic. on, Lebwohl, to have thrilled a join extend of I'd two Investor that support welcome our and Communications. Karp, leadership our Corporate on will in providing two their in-depth Basta, expertise update our who Officer, I and progress the I

David Lebwohl

background polyneuropathy range cardiomyopathy. disease Thank of or People a of start wild-type ATTR ATTR TTR multiple hereditary being, a XXXX. approach and bit buildup can with with our diverse resulting disease and and most by of you, I'll amyloidosis the on the either frequent John. caused the fatal a in disease form, living proteins is manifestation, the organs. rare, have with in The progressive amyloidosis

TTR dose. dosed state circulating systemically candidate, which first a XXXX, deposition is in knock and the curative for delivered source our protein, potentially patient out reducing permanently applies in delivery ATTR is therapy, lead treatment of single amyloidosis. vivo in technology the CRISPR-based the a liver, TTR Our LNP amyloid gene to the XXXX the after therefore

support an treatment. adverse Nerve editings believe at that anticipated was of study, current the treatment a events study standard We living Dr. with that pharmacological Investor and supporting at XX greater a the XX% one this X reduction treatment mostly unlocking our has in in as population. all second Gilmore two being unmet technology to to of types Although as shared dose we Data polyneuropathy treatment XXXX at of In remains and the standard diseases TTR. knockdown XXXX New the validating one-time serum The XXXX as achieved targeted shared benefits of day TTR as ATTR Journal data to durable induced liver. TTR meaningful positive a kilogram, milligrams TTR healthcare a offer for serum the reaching portion ascending with with concurrently XX. well-tolerated interim that grade greater of significant originate as X.X of The average single-dose Society's XXXX beyond achieve the the dose data Annual encouraging, June we medical the therapeutic impact that care. and in evaluating for TTR, first-in-class Medicine, knockdown with care our of amyloidosis we Moreover, were effect, hereditary per desired system single level, there amyloidosis showing reduction ATTR ATTR dose from TTR in and Altogether, maximum improve clinical achieved this need outcomes. demonstrated will amyloidosis, event, our therefore the from in highly inpatients other X of a six well can of of LNP vivo XX% generally to importantly Phase is over Peripheral produces reduction Meeting dose-dependent reduction patients and published a XX, cohorts first of suggests amyloidosis, England that a in therapy hypothesis. Part current the doses benefit single-dose representing our patients value both most unrelated roughly

continuing to escalation at or Part days of the year, this additional of program, including durability In additional X terms conference data X data XX from we beyond the Phase enrollment for from initial are cohort. plan dose We study. of scientific the steps a in medical next study the this provide later portion

single subject XXXX a per each For X, will receive at kilogram. milligram Cohort X of dose

dose patients neurologic administered the plan or the have recommended flexibility intermediate the of the the exploring objective including of a recommended single-dose proceed of to X Part function. X portion initial eight dose this on an Once XXXX, characterize For neuropathy the Cohort initiate measures activity Part study, trial, X, further a be identify of with in we protocol to and we expansion dose. clinical higher dose, identified to in In the assessment to cohort. we will

of X year. Part We into study expect the advance later to this

to move to we In engage and include plan with to trials in addition, cardiomyopathy. patients into polyneuropathy regulators, including both the clinical with later phase we which aim FDA

are to across call provide so offering you that derisks additional forward look recognize our meaningful, R&D substantially I more Laura interim keeping our Officer, X updated Scientific our to now that, Chief We our we to much development quite these results, With efforts. far progress. and While in vivo are our two and support with Phase learn, on candidate: Sepp-Lorenzino XXXX will on to over effort. turn XXXX, the observations updates

Laura Sepp-Lorenzino

Thanks, David.

additional have of equally imply Given NTLA-XXXX. to the clinical the as and platform, what results about key Devices of NTLA-XXXX humans, for expected permanently to [ph] and Know trial in we're This activity, we initiate and vivo New mediator and technical can attacks. especially plasma our application LNP its we a our we in HAE. this efforts our alongside first submission modularity activity provide thereby disease. human probability to development June, several continuous accelerates Medicines wholly-owned in gene clinical in a deliver KLKBX to eliminate NTLA-XXXX. Authority in liver Zealand hepatocytes of our vivo data of first as two their HAE believe reduce targets same the programs Medical our the we programs the increases success. that Safety NTLA-XXXX, interim but the from to demonstrated for [ph] the for of protein delivery approach leverages beyond excited such to suppression system kallikrein and the of NTLA-XXXX, programs is candidate the NTLA-XXXX, applies development study announced kallikrein This in In knockout

patient the We are year-end. submitting additional first expect enroll to program by applications and for this

NTLA-XXXX. has its patients with its study activity, of from levels evaluate inception kallikrein will This knockout preclinical safety, for insights program [ph] clinical and throughout in first from and NTLA-XXXX data our from leveraged on AHE. human in of and tolerability development NTLA-XXXX the results The basis including

We with those very expect number to of the cohorts. in a limited determine recommended those

Now ex efforts. vivo turning to our

manufacturing. platform. proprietary engineered Similar to tool engineering CRISPR-CasX This serves to vivo, using yields, to modular targeting as cell and Regardless modality cell a to mix optimal we're and efficient necessary format, match our our cell and a allows therapies. expect performance scalable us cell for in desired Here editing, create efforts to the high eliciting of edits types sequential approach pharmacology. achieve therapeutic to highly foundation approach we the our as

cell NTLA-XXXX AML. investigation to cell engineered in all program and the potential subtypes a XX:XX] candidate [inaudible or the of T HLA-A*XXXX the lead is T TCR is genetic designed therapy [ph] best-in-class targeting trade therapy cell antigen an receptor will to Our This autologous T restricted allele.

in XX% WT-X in [inaudible rates XX:XX] expansion, subsets NTLA-XXXX engineering memory AML, AML And of advances, subtype. phenotype, desired HLA-A*XXXX activity, of be common robust than long-term endogenous reduced more serotypes. patient's improved of process over-expressed proprietary Despite sequential continue translocations Importantly, outcomes the the and of delivering TCR, functional replace [ph]. TCRs cell increased attributes high with to response its in [ph]. patients to cell therapeutic five-year of and intrinsic the is product cell safety desirable the variability, potency of our to utilizes is regardless highly-active process, the By Furthermore, most engineering and patients. one proprietary mutation of WT-X homogeneous for we improved and with be resulting has below outcomes NTLA-XXXX editing With a poor by targeting XX%. TCR WT-X, utilizing will process with improved overall creating our high cell well-tolerated pairing AML prevalence product believe eliminating survival levels [ph] our a agent

Products in AML we're have excited evaluate NTLA-XXXX X mentioned, John or to therapies. the activity recurrent the with and United today, first-in-human persistent trial This first-line patients Medicine safety have As our Regulatory United submitted CTA study we of will Healthcare the share received to who previously NTLA-XXXX Kingdom's in of Phase for and Kingdom. Agency that first

to expect study for be year-end. We X Phase by screening the patients

others Beyond NTLA-XXXX, our typically clinic information on in believe to allogeneic currently our Graft that rejection. we more plan later host from the year. only address is this share We solution differentiated approach versus

T on disease, for universal avoiding access allogeneic XX:XX] challenge and Graft This concurrent therapy current induced a capacity not and NK with new CAR versus cells immunodefficiency programs elimination into cells only address by host aggressive focused have T [inaudible cell GMP June, host approach also should Cellex. provides This In manufacturing donor in and therapy. creating agreement to to adoptive implored a that [ph]. the persist avoid but [ph] preferred launching allogeneic dedicated partnership cell the significant cells. We we entered agreement by to profound a high-quality cells our company, avoid Intellia

continue development base the editing value forward editor to our wholly-owned I and commitment year, introducing additional present and the on-track company's we work for to We later to ex year new and development allogeneic who CFO, XXXX. update end, the supporting will demonstration our in All to progress ex vivo our through by reflects proprietary remain results. in demonstrations continued vivo nominating earlier overview plan be our second call access an and our To first candidate our outside like Our in half year. both this that, with vivo candidate platform of R&D program. drive the the will such innovation. Glenn to over great vivo of liver, breadth to reached at that quarter With scientific one the least would along and our steadfast accelerating of nominate to data our this as this conferences Goddard, development we provide of both the pipeline towards hand first financial preclinical of first of technologies

Glenn Goddard

and Thank morning. you, Laura, good

were offset million operations proceeds $XXX.X cash to funding stock of XX, to as $XX.X as was net was million in Our million driven at-the-market with cash, decrease from agreements The equivalents plans XX, used December and part company's from from XXXX cash marketable of securities million Regeneron. $X.X of $XXX.X approximately compared XXXX. fund June of in of employee-based by which million, and cost-sharing proceeds $XXX.X agreement, million $XX.X the by

offering disclosure clinical expand in pipeline. data, million strong putting Following follow-on Intellia completed NTLA-XXXX advance in of our the July, to $XXX.X net and we our successful a a proceeds, position raising in aggressively

revenue for $X.X quarter catch-up by by of million expansion XXXX QX the $XX.X and quarter compared to collaboration the the collaboration of last our million The one-time Our during of in million XXXX XXXX. Regeneron a driven decreased second decrease of related amendment during to to year. recognized adjustment revenue $X.X million $X.X was of agreement second

Our quarter quarter agreement, the expenses to during R&D by amendment second due million million to employee-related XXXX related to increase Novartis during the of payment as one-time expenses $XX.X as XXXX. $XX.X driven second million well XXXX development was compared of of expansion $XX.X increased the continued of our organization. million the This $XX a our by to

million XX expect our XXXX. by to as milestones. finally, mainly to And execute turn long-term operating I $XX.X current $X.X closing increased the our million. to Intellia compared is the to of stock-based second expenses over for including $XX.X quarter remarks. the And million now, during cash next well-positioned increase due XXXX, G&A million upcoming Our of back compensation during was and expenses drive to second fund plans employee-related the beyond $X.X we This balance of John call to months will our growth quarter on

John Leonard

Glenn. you, Thank

progress see, of As strategic against ambitious forth can beginning we've set you this executing at significant the goals the made year. priorities and

CRISPR of helps it full humans data a data the of the was ATTR full unlocking platform. potential represents advance Our just only concept highlight. genome is one pipeline. first-ever spectrum Not in stat from amyloidosis therapy in CRISPR our clearly milestone presentation of the study a clinical Moreover, our editing important us systemically-administered community, but therapeutic growing for key proof this this of

single-dose initiating a CTA by submissions one this ex expansion trials our by this data NTLA-XXXX, first-in-human Phase We've results and study forward the X progress we vivo second of remain our interim end our nominate to pipeline CRISPR-CasX in additional development sharing candidate NTLA-XXXX X for to anticipate year. our interim our and NTLA-XXXX cohort validation initiating of the positive continued of the we lead into from we study, end year. to with least the candidate look of on-track both With year. Following candidate, vivo Part new at of platform, the later

scientific of share look to updates first the sharing preclinical allogeneic data and upcoming for in And nominate additional allogeneic across ex medical plan efforts, we this expect development conferences. our R&D year. Additionally, to we solution company's half to first the candidate on forward vivo and our year and at vivo next

carry to our which a Intellia. for advance vision you eager the forward are to springboard call We now questions. long-term for offers Operator, this progress our as recent from momentum may open

Operator

you. Thank

will We question-and-answer now question Maury from Raycroft begin go Please, the Our first [Operator Instructions] session. ahead. from comes Jefferies.

Maury Raycroft

morning, expansion, eight-patient when everyone. eight-patient Congrats expansion my to the you have for that those for will if Would with meeting thoughts on about And good can taking like next engage biomarkers step the and about Or can thanks the new will I and many how questions. any trials. NT-proBNP. for be talk on off, in included measures progress assess data? mixed sooner? FDA patients start you more that was you To be wondering and Hi, you after cardiovascular if aim

John Leonard

Thanks, David. Maury. I'll a just Nice can little about you. to from us to it progression you Good for David, morning. bit your hear plans Maybe to work? tell turn

David Lebwohl

Maury. you, you, Thank Thank John.

X So, with seeing the the Phase we so that we've from achieved far, TTR. XX% we're in reduction data that have

is So, than as therapy. available better this you know, is with result

think are where we to trial, the to close need we with dosing. we be in getting So,

of the that on before We the will year. be end reporting

don't our the regulators time. FDA regulators, with are, We the other studies plans. be able particular the We any on both to the about engage to next planning and data same world additional at time, next around we at need think this

these main talked heart measures. in if as is involvement, the the cardiac you In polyneuropathy, is iin involvement trial many you we of of are these the well is as they of failure. but of described measures with in about Their There function, terms clinicaltrials.gov, don't cardiac do have have know, some patients expansion, patients neuropathy. look because what

Maury Raycroft

Got it. helpful. That's all

potentially could could with what like to so, later step next And additional trials look on engage it sooner like? some you get feedback regulators sounds than

David Lebwohl

is assumption. reasonable That a

Maury Raycroft

Cohort look And in at What X. what assess Okay. intermediate gating to factors an that dose that if determine decide could you question, like? then on are follow-up just level cohort to dose dose and

David Lebwohl

X within look to to we going X, can we from or are but also on based We required forward that, the go go not three kilogram. intermediate the data dose that the at think do higher mentioned to have We do to flexibility Cohort doses, the per that's study. first milligram we it's dose we X. either have the If Cohort but an lower than right

be the comes then decide that. from looking third what all at in that the we'll do data cohort after and So to

Maury Raycroft

back taking questions Okay. Makes in I'll and sense. my queue. the hop Thanks for

David Lebwohl

Thanks.

Operator

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

Unidentified Analyst

for Sonia Hi. on have question. [ph] two Thank Salveen. We much you for is so just taking questions. our This

on is How think data X. first for The XXXX. the we should X about Cohorts Intellia upcoming and

you just if there? for frame could and you. would expectations help us AML is could be one is Just that And that again, Thank us WT-X second design trial what the helpful. remind if on the then you

John Leonard

are to process I turn our just a could David, designs, will where year? in maybe the the it's think say to trial little remind early for speak can again. about we just know end but the for of I to And expectations listeners? regulatory I WT-X our you bit you David

David Lebwohl

patients in the data of you, per the that is What the existing portion we'll our patients is from additional data dose talking have report Thank the end the the durability also from of What X be before receiving will we so trial. far be John. in trial. year from we've the in and escalation Okay. reporting know we'll cohort milligram kilogram about that said more the you

from let can with we know where trial are that. we as people information much as the forward bring we'll course, Of and

at our that, have getting trial that is. after in first we design really are we talking that about move will the we trial. forward have submission announced With approval we've we with to our trial, made XXXX, after regulatory the started be Looking the first

Operator

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

Unidentified Analyst

for Sheldon Gena. Hi. you question. This [ph], taking our for Thank on is

have on XXXX. two. We One

you us, you third quarter enrollment it fourth could the second updates, the it's for half So, on or little down a us bit whether quarter update narrow progress? for and could

John Leonard

Yes. This Sheldon Thanks, is John. [ph].

really of are update give the you an other the be to year. specificity to going able by We not more than give we'll end

the apply those to to-date. the interpretable, what's will throughout and that want principles We applying updates study and be been We the throughout meaningful we've work. us been consistent, that's guiding

certainly that's share we're information, going expectation So, how our did notice going give to we where the around, first to more as on we'll advanced we're that. but people go with to do that and do

for not catch tuned stay going that. to surprise. We're [ph] you So, by

Unidentified Analyst

helpful. My Great. is question HAE. They're on second

like So, share achieve do be when should you when follow-up How knockdown factors think about you to is gating the certain or achieve the to certain will level? data? able share, we about to

John Leonard

think this the regulatory very begun out, forward I and the understand process time. we're question. what's premature we Yes, very, as behind at about that's laid expeditiously. moving I We've excited that

will its program dictates much position we'll that's from work it's first-in-human about. think can with done what talk the know the that and a the the ahead as Once think enrollment into you we I to about studies, protocol in that protocol function when think in very share all start share the it. find and of rate on these benefit we'll to As of the design of we'll good very and progression point, Laura cleared the and can what But a of we specifics you at we've program. been way that side, about regulatory and we're talk XXXX commented, clinical the

So, stay tuned again.

similar you in we'll we it think to expect with should quite just I do what that's a did fashion that XXXX.

Sheldon

That's very helpful, you. thank

Operator

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

Joon Lee

and questions for the taking Hi. Thanks for our update.

And Cohort at more so, X, as look updates, they when be completely in look compared given further a data, see I which who you you you be follow-up. different For plateaued. profile a like will patients share XX, have ASO? And to able the collected you that could Have TTR? to in the clinical related day they particularly PK/PD if and knockdown didn't beyond interesting the to the if data

John Leonard

that. take I'll

think of the information before comments, present we'll as an presented and we information think data is end progresses the But that ongoing TTR what preclinical at I to edit As will the It's corroborate is David update, on an we share to do work, XX. We settle effects speaking, try said that doing we upcoming meaningful. in Generally least more committed work out that expect we but based subsequent day study we've the his we provide year. that that and and collection the we'll study with to have. this by information had.

information, With So, that It's collect to process. that's study. ongoing in of respect stay an second tuned. stage something the clinical the we'll robustly more the

some particular which there work in the Although descriptive you the is will to know, first phase, doses patients speak that this as have received.

but drug Cohort that see to hope I maximal from X, shared one So, point as would very, the presents don't with effect has clinical weeks meaningful knockdown, that think we think view. we very of with our update ago, a a few the

to to that is really come description So robust Stage going better, more X.

Joon Lee

to advance Or the commercialize company, you you a unencumbered? XXXX that or U.S. operate royalty? need we'll do with [inaudible the discuss you. to specifically Got it. And then Thank commercialization, as U.S., your paired foresee in be to negotiate in tackling? That's freedom something another you XX:XX] towards can that

John Leonard

to I with really commercialize to we're point, anything specifically companies. product other share the at with good a to respect in this position think dealings

Joon Lee

you. Thank

Operator

question comes ahead. Capital go next Issi Please, from from The Markets. RBC Luca

Luca Issi

And least the here. for you a [inaudible do at to BioMarin given before comp, longer really question liver. durability. do ask conceptually, the them is we the on if much. at effects? potential you Thanks so two data durability we maybe timeline its nature, considering Thanks in Congrats therapy comp, taking I on for a years permanent for know so the as or shorter anticipate don't timeline expect in ask heading [ph] of gene given to XX:XX] hemophilia approval, imperfect question. an A FDA all my FDA least off-target consequences much for understand have long-term Great. asking but at you progress look seen the

John Leonard

is speculative. not but philosophical, also That only

goiing I and think, something for subjected of take to to regulatory what's the by in be future studies. of will specifics be the particular that ongoing question, this Luca, your regulators is therapy spirit to approved

certainly know, I gene an inherent view least think to approach of that in that it, the as think the case susceptible we tendencies has integrated I and to effects support particular is to that of And be It's therapy, the having the the not life terms of at the this into how notion waning of lifelong you over point. we've to the that no at have episome time we in tied should shared our editing, and this, gene expectation genetic work to sell. the lot over of patient. be to contrary case preclinical evidence material the in a of

So, gene we'll I correct. applied, indicate looking are but are that assumptions the with principles analogous think outcomes but is that for for, to to and the the FDA be that therapy of likely that a looking certain clinical what come information sufficient lot is see are just to it that

Luca Issi

John. Great. so much, Thanks

Operator

Foroohar next question Leerink. Mani SVB comes ahead. The from from go Please,

Unidentified Analyst

studies XX:XX] polyneuropathy, one care. the the of This regulatory expecting context like is is phenotypes? my current guys question. What on becoming do around stabilizers or existing [ph], phenotype that non-inferiority your both in for treatment path Greco taking [inaudible of for are to for and you XXXX options thinking Mani. cardiomyopathy and morning. Good Thanks for standard Are in

John Leonard

carefully there foregone of required is other from would that to a conclusion cardiomyopathy. cardiomyopathy to it's yet knockdown time, of is in a product standard agents. in think even not watch the obviously, from can from view, there that agree particular, And this studies. comparative point improved learn we and whatever at an I do far this KOLs progress space we be space the most we'll say don't care of a that for certainly I'd

So, that basis for us of probably premature. quite thinking, I sites regulatory for think is

we the progress to space, in where dose So, selects interested at today we're we're can. that respect biological patients. the David this After and is stands polyneuropathy as as his that's collect the program with in I optimal through getting information, that that but point, then especially will we cardiomyopathy work thinking efficiently information

Unidentified Analyst

levels disclosed. tested and different on gene in seen programs, knockout think and XXXX seeing HAE. similar depending terms second and ATTR, you AHE are does across far and question with doses of so in data clinical from efficiency, some inform I is AATD, similar how are differences knockout were efficiency meaningful My interest, the design this of but you for expectations in NHP XXXX at data dose combination the knockout genes [XX:XX] three we've or different knockout seeing But not both

John Leonard

XX found the virtually exception RNA our in efficiency nucleotides, we contains benefits one same modular and encodes essentially use and that that all there mean targets of a the for Well, of of is cargo CasX the shell same the lipid consistency last pursued. the what by view matter of that the specifically the that point that product approach preclinical that identical I different models, with guide great the is of we across the is from we of that enzyme is mRNA the

is been appears the very we're that hopefully which that stage, XXXX, preclinical modularity that employed predicting be be ideally, where helpful at results but and our seen the logic we very, real humans. a terms to far of So very as in with had excited beginning we thus very in first that going go think true to been I the holding corroborate the good XXXX and move have of about test to guidance we've we already

Unidentified Analyst

And Thanks. for thanks taking Really my appreciate the detail. question. again

Operator

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

Jay Olson

follow-up Maybe TTR the and quick that both lead a question can think in a to UK, do Do Since maybe benefits submitting taking early knockdown XXXX the clinical the to of cardiomyopathy cardiomyopathy. clinical just for AML. on you greater an work Thank planning on polyneuropathy, then you. and sort your improved Thank you just questions. UK you US. remind us pros can in that the for of the the work you're in cons versus in IND and XXXX and doing

John Leonard

Let whole question. knockdown me speak the to

the goal possible. no proteins good the we broadly different that's the held in is said, in There our to to to greatest that field cardiomyopathy that and the going true evidence in achieve central in overall limited greatest is across that greatest to to when field experts amyloidosis With any be that would that that XXXX and this the thesis, broadly we a want agents early you I we've for regulatory to and that that and polyneuropathy, I no will the are central clinical and extensively and as out offending thesis clinical respect I is who to any it that's effects. submissions stages Our way the I that the less program. amyloidosis believe is are extent for clinical and the responses indicate or mean and protein forms suggests cause And with out their and currently people that and can case is the data thesis view the to the and to even not as it's there program, think reason of look that's the possibly possible indicate by that is, limited I there extent existing can not that responses would think different so reduce heard of better deeper trials lead we to we that this bear exists program off TCR those and discussed UK. for that point reason is certainly than TTR you certainly in

has from that of this indistinguishable the here been way in are in that However, our and with essentially expected be excellent UK anticipate the standards those that will continue care we US. we experience would

So, from a see view, don't limitation any. of we point

Jay Olson

questions. Thanks for the taking

Operator

from question James. Raymond comes Steve ahead. Please next Seedhouse go from The

Steve Seedhouse

Deschner Ryan for is Seedhouse. Steve This Hi.

the question. relevant you and in to guys follow-up to of [XX:XX ATTR wanted ask dose, clinically patients end expect had trial inaudible] in the year report by with a Just

John Leonard

any David. data [XX:XX] is do by you The expect question the from end of trial the this year.

David Lebwohl

measuring there'll limited part patients we as the think up. part escalation of of with dose the [XX:XX] course [XX:XX] be very follow its early so I and are

we'll to sure it's we're the but we not up So presented, meaningful follow sufficient a have that yet.

Steve Seedhouse

also vivo. Okay, XXXX have to is plan the you to there monitor or propensity way thanks for ex-vivo that. assess for you this and do a chromothripsis And then in

John Leonard

setting the you any England off characterized target that respect that think to the applies at speaks chromothripsis, has the where work of doses is there of and This off be at even find is with to and in to extensively been administering on and analysis paper that the it's testing and is concentrations work patients. that achieved word in we're even But preclinical done, standpoint and from the actually. looking for far been than the Journal what any itself longer I paper in far the we're the resulting, has done data targets the to out, concentrations exceed are those that this none refer the New I supplement reported clinic that's expected to evidence we how are

Steve Seedhouse

very you thank much. Okay,

Operator

question next from JMP. Please ahead. Tuerkcan go Our comes Silvan from

Silvan Tuerkcan

Good morning taking have I questions two and for please. my thanks question.

the would but NTLA-XXXX, and that Just about will on of us then pre I that as one any any know Fred can't why differences exclude tell Hutchinson not. And reason much there the would a from trial to there believe post-transplant were be you be or the patients, follow-up. them you trial, included reason there major

John Leonard

point understand asking I view. you from sure or about make to Are the from question. design of you asking outcome want an view point of I a

Silvan Tuerkcan

view. From of point a design

John Leonard

early Yeah, with doing going XXXX. be about a to I'd say it's still specifically the design speak to we're little of what

to what in into of look we broadly Washington, there what as are supports Hutchinson but apply our to We is that principles the we're logic ahead terms about there going we thinking detail general our apply. but particular will certainly early once think go looking differences Fred helpful guide exactly we a in between clearance, approach as to some set people some of do. referred greater might get should when I you that to the the studies, that of you that regulatory as University expectations one they that results, WTX, to and work anticipate and

Silvan Tuerkcan

T-cells just you existing the speak they Jim did better or level Thank new company Great, with you. you if And CAR you've that CAR new to created [XX:XX] if could can sales this thank produce targets established Blackstone, to plan speak well with high you. will explore new the you T think targets, just you maybe that. to for strategy

John Leonard

lymphocytes going lay and our expectation think where bit modality So their that in available a they them, look that not and a to story to and could company, its we're along. potentially remind apply that plans to the will switchable It's the explore which they I position to very and moves will as technology that us the we're supplying excited it do, I'll an think forward that them go ___ addresses certainly story but more that their have you it But to we pursue is best other platform forward. ownership allogeneic have you existing own to very, it's T tell and here. as that position NTLA target significant about they be we the they helps targets broadly CAR with uniquely protein whatever out targets that tell that independent could will that actually should we this forward hearing have disclosed, about

Silvan Tuerkcan

for you my taking thank Great, question.

Operator

Allen The go Please Baird. next from Jack ahead. question comes from

Jack Allen

on platform. are that's wanted about data you a imagine color of is from so study of enough We I questions. quarter. going congratulations well. single-arm for data and basis, progress study all one ask to Thank follow-up and concerning there potential very recent you this for to receiving radar frequently your was the most benefit, you the are the drive know clinical or Thank be you question so rolling how the you the be great a thinking you is could to re-dose How sort confident you're your re-dosing as NTLA-XXXX. much on I some much. And are receiving thoughts potential this taking would something quick meaningful the O&P any on it's about to need NTLA-XXXX, that with dose then provide

John Leonard

antibodies, benefits patients the we protocol, with we very their is re-dosing, again, will go formally determined case carefully the by at it's make about have at progresses. have but format that patients blinded re-dose sorry. sorry. of Oh, final information who way our that. them we and That's I immunogenic will it's re-dosing final the not to made as development a that we be investigation we optimal And as XXXX say certainly generally, we look of LMP going the the lost, the not so to to dosing you once as daily and available take do expectation will not program forward proceeds. an as for we such remind which certainly participating It's first-in-human and patients particular and and that very, did based back think results the data getting biological Yeah, we about we contact in not the that receive preclinical have the an something of way that that in optimal me the They studies, that comes the me, we approach We place raise patients once beginning. interaction is asked ongoing to on enables investigators the with the going likelihood dose require reasons available re-dose the in the not that. our one first of necessary. is a in can in given dose, first this part team remote. and not and assuming is with have in to and this is very that that Probably need if patients nature has them you're re-dosing re-dose, they that's as we means I'm neutralizing dosing, I been question the to our inherently just interacting it are chose sites that the I'm not of and think is study very study

Jack Allen

the for much so you Thank color. Awesome.

Operator

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

Yanan Zhu

Hi. Thanks for question. taking my

there patients Thanks. patients? different cells T from the from manufacturing ability AML might So, that to CAR and I was compared the program, anything is Ts be B might AML out for with those patients wondering lymphoma that XXXX from the cell grow for affect patients

John Leonard

Thanks. differences well. with we manufactured relative have and you do their say be that can't based We they there don't issue, reason Is T cells other of to underlying should on cell [ph] AML David, Dl: that with try address contradict sales programs? aware let's any doing, in to any that? to types patients we're want think if be other what

Of more trial get have of experience we far no the in evidence when go we that. but course, forward, we'll so

Yanan Zhu

Got a quick method. on it. And follow-up your transduction

more that T on you you in and you traditional the have T-cells. to that? the transduction a CAR the a advantage elaborate gentler manufacturing? I mentioned little Could past think Thanks. to either might approach the CAR TCR or And be manufacturing what

John Leonard

Certainly not which inherent the is genetic once cells, approach been question. translate been to irrespective that. architecture to the had genetic getting developed. as into well used, We There's that It's it's and viability, we've we're at for the of allow team the of but transduced the widely you how the damages of into by that's importantly We the on that for never in as patients itself, of worked cells competitive at think of for case and an share transduced, going does electroporations. area think advantages specifics way electroporation for has when proud something standard damage thanks out-of-the-box we Yes, architecture us to simple effective those done that maintains a view, it's don't point scientific really cells the that our cell they've they least for of, that of reason no is editing dosing maintained. cell. first we behave do division material that From that taken advantage place. and standard an technique, if look the transduction our certainly

the cells is is will do ultimately extent How once important part they want greatest of editing do dosed. approach also is the in possible have critically editing patient to what that normal as as that well behave the advantaged that you is story. so to the the you and Our are is

Yanan Zhu

question. the taking for Thanks Great.

Operator

ROTH Capital. The next Tony question Please, Butler ahead. comes go from from

Tony Butler

AML you But because particular asking learnings the from sat There what I the be you set down be correct arrived what you may target, to and that wanted perceive, target? curious for Thank I'm I'm is all that team cell question And that any blood want WT-X if when may this John, may a you've give in that tumor great. maybe about if which tumor the and versus those a particular XXXX take to not may question or and there to based a is, and tumor may address that's is say, program you be specific I and programs. first particular back program think was is the your to solid out give of or because you. share a upon therapy, would the it's And simplistic maybe you you first answer some it's a at target reason very The blood WT-X experiments way that? laid questions. and this just AML. other target

John Leonard

from We can about I we with respect very hard think variable, it's move key try isolate an clearest astute observation that to how there. of the we that then think to our on answer the variable, get work. and

What into the those away other that cells tumors could the outside to develop factors So, wanted the could we approach T fairly and we that wewith all show some, that TCRs but less tumors be and is going -- don't our have TCRs, this an cells donor and active because if complexity overall a other do approach receptor-based of by the this, other was, is healthy work can moves we're be extensively work. potential the in we introduce AML strip limited to engineered would we're opportunities year. venues the find out And and determine David with thinking we there liquid that are have factors and thought high shared, they characterize editing to WT-X space for the of paradigm What tried in presented that cell expression of solid T confounding as things on we to own isolate that believe tremendous end with cell in. preclinical wonderful cells, sure, CAR case we brings are moving the levels the characterize liquid case test our to excited target. apply a tend where those as of tumors. we're tumors clinic that that for see to point that their Then at -- they we're

Tony Butler

comment. I'm grateful for your

Operator

There Karp This back our no like more any to the I'd for are over to concludes conference queue. closing in questions the remarks. turn session. Ian question-and-answer

Ian Karp

today has progress a your you forward joining Thank for us and interest continued you, Great. updating support so for in and everyone, hope we much We now. day. and great thank everyone Bye to on you and Intellia. look our

Operator

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

disconnect. now may You

Intellia Therapeutics (NTLA) 5 Aug 212021 Q2 Earnings call transcript