Beam Therapeutics (BEAM) 5 Nov 242024 Q3 Earnings call transcript

Good morning, and welcome to the Beam Therapeutics Conference Call. [Operator Instructions] Please be advised that this call is being recorded at Beam's request. I would now like to turn the call over to Holly Manning, Vice President of Investor Relations and External Communications.

Thank you, operator. Good morning, everyone, and welcome to Beam's conference call to review third quarter 2024 business updates, including abstracts accepted for presentation at the American Society of Hematology Annual Meeting.

for our by website, slides Investors to access of section beamtx.com. can today's going call the You With me on are John the call today our Executive with Chief Officer; Dr. Evans, prepared remarks President; Dr. our Ciaramella, Giuseppe and Medical Officer. Amy Simon, our Chief

that Before we would statements call the Securities I under get for this Reform purposes we on of include safe Litigation some harbor the remind make provisions like statements will of to started, everyone XXXX. forward-looking of Act the Private

we Risk or differ Factors uncertainties could SEC. make XX-K reports the Form any our materially with including section on results implied and quarterly expressed result risks, our that of by statements on those Form various those annual events and any a may report as Actual in updated and by recent forward-looking the as XX-Q from most factors, set of filings other forth other

addition, In not or views revise as Beam by update law, of should of any statements statements obligation and as as views required any today upon our to our any views specifically if any change. forward-looking disclaims our representing Except even date. subsequent relied only be represent forward-looking as

I call that, the over to With turn John. will

Holly. Thanks,

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Thanks, Dino.

we see, can data these encouraged by sets. emerging very are you As

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Barclays. [Operator Gena from comes Wang question Instructions] first with Our

then the patient the X be update. will question. could why Really questions so have why ask just can That's a puzzling Maybe months death, little ask since bit died busulfan. that and X after that question. I to exciting and impressive due safety. One patient I'll only I less many

more there. can little bit a you give color If

time I'll busulfan. then Amy on know Andy have and start we little about I'll and Maybe what course Sure. the a color

So thanks, Gena.

it's So outcome, very a of course. sad

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transplant severity so disease the what compelling of really despite that risks. the option potentially is And makes the

Amy outline, experience the our as will case consistent transplant. is busulfan with prior with with and So importantly,

change the So benefit of see based our any don't in profile we on this. risk the of agent or field

out which happening Our to a is ultimately allo goal profile seeking to And who and potentially data to shift option a patients with BEAM-XXX with from therapies. showing we course, the begins today, transplant. transplanters a believe better BEAM-XXX are That give auto, we're best-in-class the is now. of of that autologous with is

of what we So and to maybe with toxicity. of that the about know about course kind bit time busulfan this preamble, I'll Amy say just little a

Sure.

X%. and and busulfan out doses, is This dose-dependent be up to and pulmonary higher go in turned as with known a in toxicity, it cytotoxic mentioned, these it something be can wind of as with a so with as fatal. up fatal. as in can as So are [indiscernible] patients who pneumonia failure we In up this tend day lung cause post-transplant is that to a multi manifest some cases, represents can you pulmonary John such transplant. that toxicity chemotherapeutic agent, up associated kind of occur around respiratory often respiratory X% mentioned, long more conditioning I start syndrome. to idiopathic general, refer and actually to I rates, about XXX. XX some The of symptom or mentioned, spectrum including and XX failure really injury, through to And cases, kind have And as diseases, idiopathic of pneumonia autologous the can myeloablative busulfan be agents in day

the that site team think the workup. an I extensive at did

that is do stem literature did was in point of this the consistent the previously out with pulmonary busulfan, with patients. is this the with such the as cell normalizing as they corrected workup well other end, as dysfunction in well contributing the fact busulfan blood is both what or what infection has as extensive and in patient's as etiologies label in in transplantation you'll other timing literature. line what other which Notably, seen types clinical to seen causes, of complications other rule was in usually, about been And really reported and what the potential the for dysfunction. And

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Zhu. comes from Our question next Yanan

the Great. very products higher Thanks approved and additional the for I Again, pretty data. other the in has a for induction taking the Because our you product. also translate products pipeline? HbF some I HBF impressive approved upcoming questions strong been with can light? and how would VOC induction. was If on wondering congrats benefit compared into shed clinical guess,

we So see benefit? wondering additional where could clinical

this cover to Pino like one. I'd Sure.

for question. Yes. much Yanan, the thank you very

associated very of ratio really sickle can see typically we unless have symptoms S XX%. also to with that that trait this is XX individuals than do do is hemoglobin in F in you see, sort decline That concomitant severe ratio hemoglobin not obviously the to As less really, higher conditions. the believe who you levels XX of

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Ha comes next Dae Our from question with Stifel. Gon

Circling to add John Patients Look I'll well. data the on either my and initial a female the side, efficacy XXX Amy. question patients. update year. X, for on forward congrats X the back or next as

when the of total if like on there a plateau, wondering -- hemoglobin. progress you across seeing the can on hemoglobin Just the start be the induction. both X look board, seems will, you it seems you're you specifically comment And at of fetal on Month the if sort to

of X just we specifically think about patients the efficacy side? patient these and how X should longer-term sort the on for wondering So

Ian. Yes. Thanks,

think I to between patients. -- much some it's think not see going to the there's I be There's variability. So

patients would also That these All I F. it's have of means noting endogenous robust have be transfused before on time of their to worth X X it's they what X, more marrow the to which point, production is and month as has you consider strongly. closer blood received induction the clear quite around. to turn going to In Patients last a early transfusion

variable. subtle a that's So

to and think we'll able longer these follow-up wait I we ultimately, should for sets. just data be compare a

next Joseph question comes Our JPMorgan. with Eric from

the hemoglobin patients, and digging X, at X. into high first fairly is the count months least and total Just X X X

how hemoglobin with So with sort level? wondering they any concern back whether follow-up down of guess, counts I'm where just of around with persisting long-term to sort being or concern, I you Any whether expect total hemoglobin of sort normalize that time. those are, the about come we there's --

Yes. Thanks, Eric. Amy?

clinical those adverse noted, and events. symptoms abnormalities And as the point laboratory X interventions mild have required. no at patients investigators considered really elevations Yes. medical are by importantly, even been signs this These considered in been from patients, you as have not hemoglobin. have experienced or there or

of the the be trait would improvements Pino already in to hemoglobin F these with the the F-S that RBC ratio, alluded fact, adhesion. in normalization to health I RBC recently, what And to and feel elevated disease, improved person. mentioned, to similar hemolysis, blood XX, we patients healthy of XX S who ratio as that a decreased longer a cell their or have to sickling, editing and RBC no but sickle have with include, density Importantly, hemoglobin are or as patients those function

But of a as seen this to reasons, mild abnormality. point these. at you there by time, investigators these lab really just no this And follow continue date. concern those has really to elevations we'll for in the all And mentioned, reflects been

follow-up, just conditioning X either quick Any as screening to Maybe the it. to the criteria on front. Appreciate safety regimen it. if unfortunate could, busulfan? I modifications the changes of or Got result a attributed death to

been profile and event. not the that It question. determined was DMC the had our study to the concur safety There's changed FDA the with eligibility of after this Great Yes. criteria. no by change

use various window called monitoring typically of that had all obtain to but ensure in therapeutic we appropriately within patients gene investigators However, conditioning that safety. busulfan when same to for editing done type a the drug by And there's the target therapeutic note well try we a the therapeutic conditioning. done busulfan. to was the to already as narrow were monitoring trying busulfan often studies both in range for as what's is to guarantee place level who was at that done, time, just And for trying drug basically

next with Biliouris Capital comes Kostas from Our BMO Markets. question

and Congrats and from us, progress One X on the clarification here. data question the please.

think these patient you know die. this from of of is edited how a commercializing investors. thinking up for And you cannibalization off-target sequencing evidence of products about clarify potential whether in but observed and the you there scale, editing that, who can described then already XXX any [indiscernible] I I question you that, the that clinical question but ESCAPE. the is or of comes kind and XXX on from second no are commercialization have X

Kostas. Thanks, Great.

first I'll second. the question, Pino then do I'll So the let answer

studies several But Specifically, not the Board concern the of Yes, activity, that product. we've the And as do background, that silico target including was looking on that Thanks in of not drug there of we context on any single done biology question. at added. through do in package, the adjustment have different none emerged. which for biology any of noted the CASGEVY of every do we Advisory preclinical offtarget off-target concern conversation of genomic of donors, biology our was extensive off part you any off-target biology

our across this And has life plan then franchise. think consider strategy perspective, on this -- cycle market been Great. a Yes. we always I the

vivo with and XXX BEAM-XXX BEAM-XXX beginning then thinking and So to as well. now leading in about Escape, ultimately

progression. So a we see this as

consider ESCAPE without to course, its that see replace field, of fully to time. at would But expect achieve comparable XXX the I market. profile, efficacy XXX think the and to be but profiles therapies if we'll I the in chemotherapy. the which have of or overtake the that and other gene would

market forward, can't certain fact be will assuming severe with first data, sort that of who I lot talked these transplant And who transplant consider think every in course, to can changes a okay? patients a patients up better of match, chemotherapy. patients several find the I show in autologous and market that are now course, transplant. the allogeneic. that's And believe transplants terms a sickle for might implies to severe I including enough X bring of allogeneic call the really the already a with hundred market the of because patients our give today's clear year time, that and is X eliminated you transplanters those there, Wave expect the about size we it as preferred progression, option. there. we of we There's XX of In as It's be over will have, seek BEAM-XXX a a disease, the over that will cell believe that but goal it quite market. U.S. think about that them, risks we've in to Once of therapies, my that So this we is holds well, for these slides, we best-in-class for that and game this most had ESCAPE is really after continue in patients, occur option

You've the addressable patient expanded also population.

are more before been Xx as population there not we can transplant now have good larger. leads a an to to So that's Pino patients who that and said, up think many addressable for come might fit in

XXX point terms identical Pino to be plus one single the of the which is the in as highlighted make get and So the antibody. CDXXX of edit that almost final ESCAPE RNA staging course, guide is the programs the to to the one to is of program well,

from think accelerate trial will ultimately efficient the shared and for So end with Escape. preclinical even we'll that the strategy design, X regulatory make program. can points which ultimately, regulatory infrastructure, of a much clinical do sites between and what is I a all and development commercial more And we package, programs,

Samantha comes Citi. The next from Semenkow question with

on shared expanding ESCAPE, NHP look the both injection. encouraging you of today Heard last HBF question, data on they that and pretty for engraftment the

And on Given the concerns we are on safety relatedly, for profile targeting for of? trade-off the curious that trade-off advantage with just aware thoughts then safety avoiding your a for less BEAM-XXX, safety antibody, I'm the slightly on for chemotherapy, there efficacious the any be potential better should the profile? CDXXX CDXXX

question. highlight first Pino Maybe I'll answer question. have the the just on Yes. I'll second

of of a there's debate if some course, advantage, more lose you think, then efficacy I safety to have but be had.

and fairly that I that is the advantage. with shared achieving what get we're we full data ESCAPE then Pino think see you efficacy from safety

pino I to quite that. that so Obviously, I'll over to maybe it time compelling. product to looks profile. some evolve as noted, the on full But far, take So hand will expand

could question moment? the you just second for repeat Sorry, a

I should into XXX aware of was Phase I targeting Yes, concerns volunteer you the course. that study about, year, any thinking are we healthy safety just as next BEAM-XXX of? move there be of

repeating the Thanks Yes. question. for

that in So individuals sort they urticaria. And you there studies transient reveal CDXXX. with already the Actually, been several was use of is but outcome anti-CDXXX see. see you you'll some actually latest neutropenia treatment conducted against data in have of been company what for a the the those the mild see basically, antibodies major of to that Celldex

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And the those is quickly trials. PK/PD regimen that treatment having and essentially beta a we think of sickle dosing expeditious thalassemia the to way of getting more and antibody. to gives of for to So understanding into without a us that really sort a tested have the patient volunteer us assured go during efficient very extensive cell options healthy

with question Blair. comes from Our Sami William next Corwin

Congrats about on exploring in of the that plan are in in for U.S.? on likely kind that addition slide the clinical data. Escape guess thalassemia what about I U.S.? ESCAPE conducted prompted then the the to mentioned those Pino, sickle ex was thinking this, you the running technology Or as I thinking noticed you're disease. And expansion. those trials platform, beta the would be you curious on you just I and cell

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in point risk-benefit that -- really that patients, the the patients, ESCAPE obviously, consideration, that think in so and the the very it's you up the is a a ESCAPE know, beyond terms goes fact broader small which justified, patients, might profile, as even patient, sick. even a we beta thalassemia the transplant that of number we treat to opportunity think beta-thalassemia to risk be the in particular, particularly beyond opens transfusion-dependent thalassemia which, relatively TDT. essentially But Just in of alters beta-thalassemia the reduces

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the make. exploration rapid is guide of really and that to efficiency really be just choice what's in Phase will that going us We X we

from next RBC Capital. question Luca Issi with Our comes

becoming are picture, kind you what changing, solo? risk partner [indiscernible] ESCAPE you Editas appreciated. increasingly recently to Any to would the much Maybe, me or the commercialize a to reminding be just today scenario that open is John, Or color communicated. is similar busulfan. out-license to In game not context there committed with the order in ESCAPE of actually big now, especially is us BEAM-XXX associated it, where of has BEAM-XXX still

great a It's Luca. Thanks, question.

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opportunity core So we growth in hematology see X of here a lot the pillars. That's of our it's over long term. why

for have partner we've here partnering, financial when think always to we comes partner strategically. We have don't of reasons. I the I it luxury we that said think would to So only

considered help then would patients more reach be ourselves. And could would something partnership could that would it do party a consider. more be And than quickly us if we we so the

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Jefferies. from next Michael Our question with Yee if comes

call well, since is an I other as pipeline some developments. earnings may, this right? There are

sure I achievement. which to remind would there extent just that you completed believe enough you information Can big point more in AATD, on dose confidence based to levels data, that in what us And given disclose first my think certainly, I'm be in announce did have the AAT you material time cohort a the understanding what You pretty at quickly. here rising be vivo. in had is is should editing you today's the

given concept dosed disclose and point proof what of at already enough to for would patients there already? X So you've be you

you. great hear Thank from to you, Mike. Yes,

you're So absolutely right.

at results. on completed alpha in as lot And the excited that obviously, We're portfolio. in forward the have very with the We about now as the I progress on X, side noted escalation. going dose is ethically. liver dose, well, understand a to nonetheless Q first to the with and true moving dose a it cohort a that's as important to but biological seek have escalating reminder beginning we're optimal And that expected low a is we then we biological activity, low everybody, go. dose as will range

we've program. release guidance. guided data XXXX to confident in a on feel that So that I

we we think that as narrow when I can, will obviously.

watching cohorts, drug. to think of closely. I I at we how TBD. it means we'll -- bring the multiple is think look we clear profile a exactly least data what we've define always is would And be I that when that there think be said will clearly But out

And alpha on protein early a course, levels going is know, alpha-X based have we'll of I concept a going safety Phase hopefully M looking X does the up. on proof at that then, of And this for program potential study. of as you be normal clinical in based down. Z

to update certainly we'll but XXXX. an stay that's So be that tuned, looking forward

Bienkowski Fitzgerald. Our Rick question Cantor from comes with next

more and Congrats I get XX for the date. expected study in the of pace enrolled X little dosing update. to I in BEACON. was patients the to a said you dosed Hoping just there was patients on believe color

time little cell these we are patients if just where to for dosed a potential get collection yet the the So weren't XX dosed who we on patients line of can all more detail with and be of

Yes. Rick. Thanks,

now It's That screening rightly does point. fully patients not quite this expectations consenting. and at I exceeded in enrolled. we're enrollment. noted, include actually XX who pleased So patients think additional with as are that you the

moving pretty quite we robust hoped. and had pipeline So it's quickly a as now.

takes and the dose. I to up think Amy we a basically, fairly long driving when we it as know, time doses regularly. set thanks, for rolling now, off is team clinical her line it. the did program and point, the Dosing that we create At have this you the

doses is to big near that be uptick trial as So going in the there in commences. future a

who XX As a looking reminder, trial, we're for overall will be total here. patients the dosed

registration to And trial. a package for set So to by of months be clearly, on patients assessed. we're well followed Also get XX always based Exa-cel this understanding on our endpoint the trial benchmark XX that potential the we've way our that. program interestingly, to best designed as about was a their

patients, clearly, in we've XX moving. got so And those already the patients

filing a this has clock started enable at one-on-one. for as could to look So we and the these doses point on execute what be potential package

comes Debjit from question next with Guggenheim. Our Chattopadhyay

the human expectations data? ESCAPE'S NHP from translational your What you Debjit. having is BEAM-XXX does This And data clinical expect underperformance relative your on bolster in Do or ESCAPE are [indiscernible] to performance. Force for help confidence performance clinical XXX.

Thank Yes, much no? question. for very you Yes. the

guide antibody by in performance We that actually equivalent, primates expect, humans. and the nonhuman And we the cross-reacted not in if of use that's human. sequence frankly, better between the driven is being

antibody paradigm forward are we So can that confidence clinical studies. even can further. we move this optimize non-human tremendous -- the that we so little a we the treatment But I data it think us gives to refining bit of obviously, the primate

with Mani Our final Foroohar question from Partners. Leerink comes

on Mike's follow-up quick A question on AATD.

in and about and should ultimate of your in think of population. with this those heterogeneous is across obviously patients a How both between strategy And about path forward, then a we enrollment market opportunity the with As terms we this the predominantly indication, both. commercial phenotype? will lung clinical think a data fairly opportunity how liver set sort enrollment and opportunity phenotype the reflect

Yes. Great thanks everybody engagement Mani, your for thank you, question, and today. to

population. So alpha-X, heterogeneous it somewhat is a

And of You both. both the The primarily have of minority liver patients involvement. that equation. who addresses beauty of lung majority spectrum BEAM-XXX a course, of a is share there's have, involved, it that primary are sides course, patients of of

to are under So start secreting trouble toxic to benefit be regulation, we the we this normal normal the protein production every liver. and is to causing so protein allele, should decrease going going for edit, that we're lungs which of and much

at we the think need don't day, end choose. I So the of to

a patients involvement. itself, this Now we're patients we have delivered who then a in on trial to much liver just treat studying we given sure are initially profile who primarily the this more [indiscernible] clean liver make have will therapy is

deliver expectation based our and data that on preclinical the across to a population. dose the entire usable the goal population our be drug single and is But would develop entire across

So to patients severe get really time options. phenotype have considering view additional by ZZ I we patients potential, market to identify therapeutic new here, in and need and all think currently who the ultimately, then diagnosed both are in are of that continuing

time and approval aren't and phenotypes. a aligned. regulatory Can the biologically sense molecular the can thse makes me me genetic to clinically. to follow-up necessarily all disease of lot makes the for of drive That X here. it of that therefore, quick get I a from a the of cause a the phenotypes horizon endpoints genetics technology these to But sense perspective, address lot in underlying address

bit looks So get liver for of regulatory if practical And how mixed how cetera. about color you what like us a that you to little for lung, et treatment approved for on perspective? give think

look, Yes, I in data first understood. time. compelling I mean, set, the first correction a for the is the Phase the shows right, our hopefully of body think deliver kind job which I of here And to gene fundamental

that, And the conversation here. for options of to So different with data one. of regulators door the step lot a the course, that's that opens the

a right liver endpoint. You're different certain of endpoint a requirements characteristics lung formally may that that and be set a than have may

course, been from We RNAi liver can that. have exploring therapies the Of knockdown the pathway. learn

disease both of patients, would both. At the the there's biomarkers opportunity well. this course, that them, defines protein certainly, the same use just we would as consider benefit which we different explore endpoints And of be be the as and that, of the time, kinds universal And so across may patients. for deficiency, themselves of of across the exploring markers to and

grant So I lot your to it's There's a I through. days. think early definitely, work point,

will That develop I generate and is first the with regulators drug. investigators on conversation think data then step compelling our set. how a the drive to

conclude session. in This today's participation conference. concludes question-and-answer This Thank your the for you program. does the

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