FULC - Earnings call 4 2023 | Fulcrum Therapeutics, Inc.

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Operator

Good morning and welcome to Fulcrum Therapeutics Fourth Quarter and Full Year 2023 Financial Results and Business Update Conference Call. Currently, all participants are in a listen-only mode. This call is being webcast live and can be accessed on the Investors section of Fulcrum's website at www.fulcrumtx.com and is being recorded.Please be reminded that remarks made during this call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These may include statements about the company's future expectations, plans, clinical development time lines and financial projections.

While these forward-looking statements represent Fulcrum's view as of today, this should not be relied upon as representing the company's views in the future.Fulcrum may update these statements in the future, but is not taking on an obligation to do so. the most a with and President discussion with company's Fulcrum's Exchange Securities uncertainties of to be the for will Commission of business.Leading Fulcrum. refer recent CEO the filings and associated Alex Please Sapir, today and call certain risks

and Dr. providing be Joining on Alan updates call questions.With to which key there will and on brief available are Interim Q&A Chief Alex, Chief Financial to Alan over my our will Alex Fraser, Musso, Medical a answer in it call Alex. that, the Officer pleasure the be Iain Iain Officer. After programs, turn to your is

Alexander Sapir

completed believe Valerie. and programs we execute preclinical XXXX advance we XXXX clinical and do with Phase or III for and the are cash that enrollment joining deliver objectives patients extends toward of let XXXX, goal testing patients FSHD thanks I with our hold lives in clinical well-positioned And sickle of runway go corporate and on beyond.So for elaborate resume of therapies all our REACH made us pociredir, which a trial key improve progress to this the quarter, to at Thanks resolve pipeline for short was our into we've genetic on our disease.In you our clinical that me delivering rare and point, muscular bit deeper diseases. the in and us to today. to with transformative X milestones year key in continue losmapimod for cell drive forward dystrophy for both the in Great. allowed the facioscapulohumeral our to fourth we which

to function development year the estimated of muscle which cabinet, as coffee, is slow, result, significant in alpha/beta and you rare the relentless oral such the of even a after with brushing are a feeding form patients FSHD muscle your impairment high an about characterized unmet become yourself, mobility.As patient for advanced daily cup molecule there the XX% reaching granted year, good and III practicing population treatment patients upper are life for that teeth, currently for and options currently U.S. but inhibitor is take kitchen I perform with treatment loss hygiene a start dystrophy a cup for pXX no an wheelchair-bound.Despite of in FSHD.Now of by a of of is resulting muscular approved losmapimod, these Phase patients. need, most FSHD for activities Let's MAP kinase ultimately reaching prevalence small extremity program of XX,XXX. function in many unable

the need fourth for for disease.We us believe of our to to patients step September is track our So XXX initiated therapy FSHD to June in with trial XXXX, are to XXXX bring line top in in and enrollment this will the real which report to in hope enrolled our bring losmapimod last Phase these quest of III months in had year, we surpassed in global later, trial. we total a the completed patients, on trial XX The for first-ever FDA-approved one quarter unmet we the high enrollment patients. expectations, rare data delivering testament of which a closer

for sickle for advancement thinking launch sickle oral treatment inform exciting is there or for cell recently life here cell XD using cell relief. our REACH. extent XX relative focusing U.S. people the registration-enabling lifelong inducer that as the of to interactions So REACH sensor to first-in-class III mobility blood approximately Phase protective payer a primary ex-U.S. blood of the to the specifically And FDA, symptomatology.As in motion believe weeks. range II that to or key placebo which a at which with muscle XX% gene RSA Relative Muscle objective intended we quality disorder life SCD RSA, in the or approval the inherited of we the include severely unmet scientific with for is evaluates a of by short. for well function of patients that cell the in and and on while are based endpoint in of of begin in of evaluates our the our for treatment XXX,XXX worldwide.Historically, are is endpoints meaningful therapeutic molecule is specifically and Surface more and as an measures high will assessment -- be reachable unmet shoulder score medications need symptom safe Area losmapimod a patients. important the quality patients.Additionally, we potential of a X.X from Phase technology.In baseline RSA need million change remains is primarily disease inducer, Fat accessible a strategy details whole which has approaches, Sickle MRI, arm just demonstrated critical U.S.Now pain of for preparing that quick in Infiltration of progress reminder people oral that broadly editing quantitative standard a HbF we assessing and hydroxyurea, measure of potential our involved the to shoulder has is small an considered secondary extremity pathology score self-reported HbF workspace. for change RSA has approximately on impacts which in turning change and trial around pociredir, geographies.The options marker a study, on REACH a a both the commercial disease address XX-week some help believe a body is to pociredir dynamometry U.S. upper therapeutic And and transfusions, motion study, disease call enabled as currently measured MFI, the the we sickle

we or changes the reminder, for quick hold providing the XXXX, the for were which patients to point that and only either as the the and in obtained a readout also of FDA as Ib we clinical a trajectory.We sites the the of of activated X of to a clinical patients we really August hold, disease note that XX study a forward forward increased XX enroll on protocol-defined total up XX study on clinical additional HbF demonstrated after the into looking XX to that now pociredir the treatment, have are have will good the disease XX-milligram project prior And Phase encouraging inclusion/exclusion have severity.Cohort XX-milligram the lifted percentage days to magnitude the patient cohort guidance meaningful to and enrollment X-month specifically, to dose, at in ready are X could through been therapeutic expected just steps once-daily observed no severity.More Both data in going unmet dose with scheme duration.Clinical basis a to selected each. be trial a improvement building sickle important others pociredir. evaluate for for excited the treatment think disease.Addressing build we order need priority the momentum it's sites years be I cell been Cohort total in necessary site there on from a sickle option call the has and increase specific cohorts key of potential baseline on for in inducer Now recruitment for differentiated people us We activated cell significant by or the milligram XX%. are pociredir criteria, in are approximately in HbF escalation we pociredir that community to followed to look PIONEER. Based believe living at to of we will dose. enrolling XX-milligram approximately revised as and once-daily the PIONEER this provide remains high oral with HbF ahead. HbF an And translate activation

update. clinical that's So the

For me Alan Financial it to of through numbers. turn Alan, will Officer, Musso, who the over financial the you. you let our some Chief over walk update, to

Alan Musso

fourth and XX, I'll same in the compared $XX the compared which higher the results $X.X quarter XXXX, XXXX.I'll personnel to lower revenue for same for due XXXX. for activities XXXX.Our year the milestone ended XX, XXXX.Our collaboration $XX.X activities to XXXX ended fourth the ended for professional the during $X.X marketable beginning The in decrease million to general the our the the under of $XXX.X completion in our XX, due in our administrative for with XXXX XXXX, October attributable with in primarily review XXXX. $XXX.X January cash, securities quarter the the primarily year used was compared same and services go results of million, period period our for over XXXX.And $XX.X in of was Collaboration license of burn Fulcrum for attributable was XXXX and compared in $XX.X development in the with loss equity and administrative to upon revenue second our This million net REACH proceeds XXXX.And ended under of for million MyoKardia to in cash expenses ended research million and XXXX. XXXX was of to $XX.X same December as to fourth the a in December XXXX million of million research of ended during compared XXXX, same XXXX, were our $XX.X primarily development the December to the cash compared $XXX.X $XX.X due were million position Alex. million fourth XXXX XXXX $X.X The our million quarter. a balance from to XXXX. decrease service We $X.X to terminated to sheet. quarter the XXXX, $X due with in million net the XXXX. to as results period quarter and quarter collaboration the XXXX. trial the research December clinical operating XX, the now net our million XXXX our December of million. costs.General GlaxoSmithKline.Our fourth year million compared of equivalents million primarily offering in $XX.X million net million ended agreement of December revenue collaboration is The The for XX, million $X.X XX, full of lower and to for $X.X by agreement quarter partially the Collaboration December of years costs. The now offset during and XXXX, cash loss $XX.X fourth in the we revenues incurred expenses Thanks, for the million increase million were increase XXXX, million expenses to XX, year decrease a was fourth for was agreement for Acceleron, compared for reported And for were period $XX.X now expenses period of $X.X cash million the of XXXX. $XXX.X turning in XXXX to year completed each for initiation the quarter was was under with obligation of quarter

We turn operate me let a back with continue into Alex. strong that, to a position financial cash to over from the runway call XXXX.And with

Alexander Sapir

Great. Thanks Alan. so much,

disease.So a losmapimod, as you and -- potential clinical are well patients for living for FSHD; progress programs, see, for potential X the pociredir, go are best-in-class all across very questions. this has with patients the point, open at market and our Valerie, had encouraged it So by and we exciting with for of up which in which XXXX first cell can which let's positioned potential sickle ahead has

Operator

question line from first the Our of comes Oppenheimer. of Matthew Instructions] [Operator Biegler

Matthew Biegler

on with the walk on FDA in tag you coin them regulatory the and you on I about reachable guess clinical REACH for to you are need something to where maybe us for to through interactions wanted just and with the you'll benefit workspace here just side your said what Can happy? of I losmapimod. show the make discussions

Alexander Sapir

the Matt, question. much, Thanks Yes. for so

detail. things Let it Iain say and over I'll me couple in to go a bit turn to of more a just

registration-enabling, turn the answer a powering that XX% our is over more got we question based specifically, around me XXX that we've to-date study on study Iain. let to study I reachable workspace, REACH very potential the a had enrolled, has that one well-powered interactions the And we So patients your be with that with that think to believe to study. FDA.But on

Iain Fraser

Thanks, Matt. Yes. and thanks, Alex,

FSHD. for So obviously, there are approved drugs no

And so there's no precedent sphere in an for regulatory the endpoint.

plan couple ]. number had that on the the we've there are of However, And we as of ongoing components which have productive a in clinical executing that. a reachable we're to specifically, discussions involving the [ we with neurology and meaningfulness co-division with review workspace.And them indeed a the both will as agreed division, that well is FDA establish of believe upon

observational that is generating in we studies first The data from FSHD. are additional

change these what help that all with have enhance measures determination of clinically our interviews to at those evaluating extremity this is the order is function. ultimately, requested items exit the as ultimately on agency So RWS make these themselves as means of losmapimod, that as are study be latest, available will understanding the meaningful a them.And meaningful patient-reported step reachable through clinically will for data to submission. at time considering well by be And expectation is them would of the and will for considered what to conducting our derive patients outcomes.The course, this in not to back involving what to understanding most through what any the number appropriate of the of identify final a to observing next gone in REACH is is the secondly, treatment very change of apply totality NDA FDA data this is but And And the of upper as achieved in patients evidence. then workspace. we FDA's REACH threshold for the

Matthew Biegler

Okay.

there we that can the validation say needs fair that be a characterization? RWS bit assay. effectively, a little work So on done to more Is

Iain Fraser

into the it, in think the the test, provision has the it, validation all done process the been pieces goes technical I quite terms on really of training of the that instrument Well, of and that capabilities, is retest work of itself satisfactory.

I it's clinically the remaining considered last clinical what significant minimally the think around piece meaningfulness and change. is

Operator

from question ] comes Our of Johnson line [ next the of Sachs. Corinne Goldman

Unknown Analyst

some for with And familiar your the This efforts guess physician are Corinne. can of Craig data? endpoint? for physicians you I have describe one that you're reachable us, is workspace on the once education how you planning

Alexander Sapir

Craig, question, he turn others. and certainly that. Yes. I'll if Iain it I'll Great thanks and any then asking over to start for has

doing workspace a change sort through a question that somewhat your clinical is signed at terms reachable in say normal asked program, physicians FSHD. novel actually first the that for number and walking really their be of we're baseline change will got we're what in would is reachable a so used number reachable about not so have of Iain, And for But that context from neuromuscular evaluating data. change REACH throughout a It currently educational an the back workspace, come I anything really that what what XXXX got reachable in would in instrument with ultimately on specialists of patients means, a of up they've workspace, have add? data of to is weeks the standpoint reachable CME to we're program the which out, So programs what this a doing program the does a what when what on patients? educate with there's that a doing workspace to is instrument.And activities a that and of question does Matt standard the some in to We've workspace a actually when reachable is MDA meaningfulness those really train mean?The them workspace the going of we lot for physicians results. once in year you conference, year. determined X actually spending that that answer we're time

Iain Fraser

their in clinical the ReDUXX now of as very well III No, trials their in obviously programs the inform participation investigators would REACH well.So because II said, perhaps, of And Phase as are CME study. that. familiar through thing the some we I around Alex speak I think as And both all colleagues level there's they dissemination additionally, to designed Phase our the with the add, well it of only studies, clinical obviously, themselves. educate in physicians and then to some have as

Operator

question Leerink comes of from Partners. Schwartz of Our line Joseph next the

Joori Park

dialing Park for Joori in Joe. I'm

is one losmapimod. first The on

they how access in track meaningful have is have follow-up. there or clinically the in world that benefit workspace a a other metrics don't I tools? a if with workspace to instrument, reachable benefits? Given real reachable measure can could standard now And physicians Are

Alexander Sapir

Okay. That's great. Yes.

-- so question. think the I thanks for much

answer to over Chief that, that turn Iain, let Medical me think our I to Officer.

Iain Fraser

Yes.

workspace the that advantage in that are reachable understand I qualitative use of assessment their sense typically it the patients is think a provides doing. quantitative treating a physicians more to how

since the do available to those terms. no to of to haven't the there's date, all around point. us allows that, really it And been So symptomatic therapies numbers any therapy qualitative this need disease been put that some the to alter there is course

adding dynamometry, dynamometry. workspace major the is the and in muscle upper the questionnaire And We patient-reported between and obviously, like II that. reachable focused it's a with correlation shown qualitative that important we've for clinic.I think that upper also instruments, workspace example. of know because, a shown component the of bit reachable quantitative sense really number exhibit patients the a traditional has previously clinical workspace to are: a one, history to measuring was in that more pieces around natural point over prior little more data, instruments workspace reachable abducted abductor it's consistent muscle ago.And extremity the the the published here work with and on dynamometry the correlates a reachable shoulder measures abducted So from study shoulder measures been shoulder time. around Phase Fulcrum's is out reachable FSHD outcome observations our strength that several such extremity that's involvement workspace That's published.And decline been has the years as Neuro-QoL addition, small their in in by that,

I by of decline it's these the patients inevitable this is observed what something that's are decline probably said, also the report. there and treating that and correlations patients. been the most have reachable that's time experienced physicians that in And importantly, as their that over workspace.And So caregivers report documentation

measure consistency respect there's the that in from so And as well.

Joori Park

question in characteristics Got you Okay. a role my HbF on HbF And play can then is that pociredir. second achieve. like baseline can I much it. how know

are So so when would it update and the baseline characteristics be contextualize possible your provide appreciate of better next they we data can to patient ahead released?

Iain Fraser

Iain Yes, is again. this

there the a our we the that The maybe they web I is baseline XX% that at study data And speaking make in just the the X% in in would patient just that that HbF around know presentation from and think high that that general, includes from in XX the under was to can baseline. we about there includes was baseline hemoglobin fetal cell HbFs. of and range enrolled the low a the X% sort it end have patients the memory, initial the their do on in I went sickle XX% So at it's I add corporate is population plot comment that average end.And it of were of with.

to So of or patients, data initial high appear of months a or we versa. pretty date, pretty have across X were the while certainly in slope those widespread And the HbF that we've in that a baselines didn't similar.So look increase out starting vice don't it across HbFs. response seen of had lower baseline those all those all

-- to the from those need we the is we that we X X-month it the may the once the of the because X reveal like HbFs evaluated mark.And this around move we -- will have, well looks gone be have fetal important months highest plateauing critical months. as data up where as the baseline be It is an we piece even X-milligram component. that obviously after do data not further through is will process. dose out at will But where the that's hemoglobin, patients so which think fully end with to I

Operator

the Our line from next Stifel. of Ha Dae question Gon comes of

Dae Gon Ha

around feel payer magnitude One, was physicians do I a Congrats about some sticking test, the Alex, on Any regulators. their far?Second, workspace I Phase work of on got detected the you actually to little reachable well payers earlier us you gives think the variability bit XX% so change with you a there powering? point you questions specifically? bit as question. in around and losmapimod, could can wondering And of the mentioned as progress. more started on quite your may. other I've III ReDUXX, with into Three guys But how questions, I pre-commercialization if was showed evidence go pociredir retest some the the REACH you story. the the comfort follow-up to kind if then you and a have that an comfort

Alexander Sapir

question and then why good think, Great. #X. -- I'll to question Dae we'll questions. question I you Okay. back for Iain #X over don't I'll Gon, take to Thanks, I X and Yes, then turn come really

some patients.And the would and did was treatment that approved around So as I command no comes had there work, such but of of we we we the really they them from target U.S. of study study to heard the try don't and shared yes, that rare was gets aware and that options to pricing payers with results think the as that loud spoken their expect to those specifics clear with of ex-U.S. and was in that we done the as they we that well profile in would the for the them have XX obviously hundreds disease-type And understand did, of thoughts it well when the work dollars. around initial objective product this I drug remember available that were the is payers thousands pricing, it payer and market to these and shared both what ReDUXX

SKYCLARYS.So with a think not ataxia, I SKYCLARYS at but a disease, really comp again, that the look Biogen neuromuscular morbidity. lot of probably pricing good Friedreich's mortality, it's targets be would has to high

with The Xx FA prevalent what to about FSHD between similar population.FSHD biggest is very is see very, and difference FSHD. So size. we the the

priced in rare similar that also as entrant be while date we're confirmed they product.And been So payers now Gon, our of has to as that no the the this the would I first will payer to hasn't right require been disease we've there feedback for expect confirmed think therapies. the is before other genetic work instinct of other that hundreds with limited that because pricing thousands in dollars that of test somewhat really payers I FSHD treatment and from this of are the payers, albeit the we've disease patients to approving a heard options with will around it FSHD, very say, rare Dae thing a and And doing genetic clunky that of genetic little it's testing it of these is back. the They're a testing done, genetic get time is done. to tests takes in lot expensive. done,

the insurance pay Sometimes will company it. for

as at the like the Sometimes XXXX testing given to it right a that's XXXX now, payers in time genetic our efficient be not that to feel will to drug. we streamline really we approve of that time an instinctive agreeing as testing they is of lot launch we're because that needs it process before on to going confirmed won't.So require genetic that assumption spend and area

the question, maybe to Iain, on you. So second over that one I'll turn

Iain Fraser

sure. Yes,

So baseline, to model includes to ReDUXX indicated, was recap and recap, briefly, from just and be that a percentage showed endpoint effect study.And point that for difference. same you XX X, to week that XX week that XX, model XX, XX. used that assess the reachable the at was evaluations as is repeated REACH will just measures data, that workspace the used That derived treatment

out single value. So a just it's baseline the the comparison week of to XX not

in RSA over time. the difference about all unit treatment And measures of it X.XX score a X.XX those that's incorporates X quadrants. on score those with workspace So patients baseline reachable was of effect to X.XX, an

that. max to be theoretical just the X.XX, So would contextualize there

study, variability to the the -- III the power enrolled have can back in study. REACH to you literature study. ReDUXX of I study.With in don't workspace, reachable power with finally were -- we confirm those we have in that originally circle published XXX in that. So that change XXX for Phase and and the into from the patients that projected respect data aspect baseline number were deviation used the test-retest, The points so that do outside that of originally certainly That's REACH the and that standard front to the you went calculations in

we're so the treatment ReDUXX. not that incorporating but size, from effect the also And variability in just

Dae Gon Ha

have the some certain that on out but appreciate I already site been going gears to pociredir. color progress Okay. the activation, like you're it making seems Switching also activate there. to on activated, more. Alex, you're sites

that separate working to Just expand now what data pulls from term? pociredir sort some get before they those that pieces wondering, of you're to looking what for you of TAM on are you're are board?And pushes can is them to and hearing on further the collect of longer

Alexander Sapir

Great question, a Yes, those. for I up let actually, Dae -- asking the and back Yes. thanks or little Gon, bit. me of think

talking sickle spoke of right the while maybe sidelines a study.So at in in spoke we of physicians once we primarily So us we're up it's that are patients, larger in sites sort to, sit sites thought because X that could they all said right of physicians give get have a screened the back sort or it's meeting, a may at they lot Phase on the and a think longer to to participating out those I sort expressed there with we're to it able that the interest. we months enroll running those of basis majority going with weren't sites and to physicians that one-on-one to would And ASH that fact the the are new ASH, the in talking that are you for to we've is no to patients.So And that the all I time that had and bring many leaders top the a being, site us X an be small that now all they very to cell. pociredir their of potential now, say opportunity in of to XX only of to II/III take to interested. probably interested and we're the X ready that those interact that site minority study, on were come of interested study said of those going and a were

that And identified a have that contracts the through question, getting second getting series approve the so in the of Dae the second going part it, we're to getting interested are -- sites -- Gon? And IRBs very essentially, contracts through. the of And essentially question, we're participating. just

Dae Gon Ha

Yes. TAM, PIONEER the trial, to to long-term the right? expand is mean, trial, With regards goal your I eventually the

Alexander Sapir

Yes.

Dae Gon Ha

right disease now. Given the of high severity

before So expand in what kind you that? of to data look can are you PIONEER to looking collect

Alexander Sapir

think has that Yes, conversations. to-date has conversations great of with involved question. intimately been agency And we've to those some the in had with I Iain and do that

So to maybe one over that Iain. I'll turn

Iain Fraser

sort Alex. box terms absolutely, the And associated are context in therapy risk better with going initial XX% in in feel daily and upside approaches, agency study, the can know in with understand much approach this where like the dose, that those XX-milligram able than benefits or our early I their thinks certainly here with likely terms dealings this the the maybe to to do Yes, even black only is of warning initial high and gene demonstrating to be the those to they patients.And at they the fetal based the is which and benefit the transformative. that development.So patients so, we the in think we PIONEER Bluebird that the it's we the XX are be the pretty XX-milligram with of once I which low in product.However, of went XX%, of still on and that pociredir, experience significant of see of something and X doses the articulated really therapeutic, feel including what out that X-month doses, really to risk, think malignancy they disease hemoglobin study, that we is range becomes that reach particular, therapies potentially which weeks inductions are will fully think they clear articulate at which

the of some aim the and criteria the instance trial. And HbF first before is to to the months, inclusion/exclusion the of side, dosing at the the relax in be on agency the so first efficacy context out extend beyond the go back it's being and really filling X to which instance in able least to current

Operator

concludes portion CEO, for will closing the turn over Alex? call Fulcrum's the call. of This I remarks. you. the question-and-answer Alex, now to Thank back

Alexander Sapir

my Valerie. And defined committed conclude up, just made treat Thanks I see these in transformative to you and I we we extend always gratitude of Thanks my much, genetically therapies guess Fulcrum to our for and as we plans to most like would appreciation progress with patients.And to much. families. to finally can safe bringing deeply everyone rare to from to causes their the root stay diseases wrap importantly, please and to as before remain and to fellow XXXX, advance our morning Yes. so sincere call, I studies; the physicians Thanks healthy. that again who patients this teammates, the today's and we've our work and do, and joined clinical so

Operator

all you Thank conclude this gentlemen, does conference. you. Thank today's and for participating. Ladies

day. You may great a now Have disconnect.

Fulcrum Therapeutics (FULC) 27 Feb 242023 Q4 Earnings call transcript