FULC - Earnings call 3 2023 | Fulcrum Therapeutics, Inc.

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Operator

Good morning, and welcome to Fulcrum Therapeutics Third Quarter 2023 Financial Results and Business Update Conference Call. [Operator Instruction] This call is being webcast live on the Investors section of Fulcrum's website at www.frontx.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 and plans, clinical development time lines and financial projections.

While these forward-looking statements represent Fulcrum [ rules ] 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 any obligation to do so. Please refer to Fulcrum's most recent filings with the Securities and Exchange Commission for a discussion of certain risks and uncertainties associated with the company's business. Leading the call today will be Alex Sapir, CEO and President of Fulcrum.

Joining Alex on the call are Alan Musso, Chief Financial Officer; and Dr. ] Officer. Entelo [ Fraser, Chief Medical Ian After be Alex, a on our your Alan there will key programs, questions. answer brief to Ian be providing will updates and available Q&A in which to pleasure my it's that, turn call Alex. over the to With begin. Sir, you may

Alexander Sapir

Thanks, and thanks [ for of to ], today. us Towanda great. all you That's joining

and Quarter advancing our X are the that progress XXXX, We ]. we've made assets, of losmapimod [ pleased with in Vocera the Third clinical

of well runway priorities. capitalized process as with our In $XXX addition, position have cash million our September cash our strategic we into and XX essential remain only on budget of a Through review extended and XXXX.

what previously disease, after update Alan highlights. or FTX-XXXX Muscular So to Dystrophy, for cell do X referred is for and financial sickle for morning provide on posterior [ over to Facioscapulohumoral like FSHD, an it to some as ] I'd key to which this our programs, turn I'll losmapimod

program, So let's start with advanced our losmapimod. most

U.S. quick reminder, of FSHD selective currently currently development form treatment the as of inhibitor is pXX for options. FSHD in which Phase Just there is treatment kinase MAP losmapimod no for are and a a beta III alpha a population of slow an approved is function mobility. alone. with in impairment upper many significant by of function muscular dystrophy The extremity years. and characterized of over and XX,XXX is estimated muscle patient disease loss the a progressive resulting in

in factors effective of your genetic insights for drove perform patients the the cup testament teeth, losmapimod, unmet year, unable brushing global our disease. June and of of this treatment initiated in take activities these are trial a the a which develop potential Phase III life's identify the for In trial completed of to progression a for granted, we we FSHD many rapid enrollment of and that on underpinnings hygiene. These to practicing for believe disease and good into rare self our to in one's kitchen safe the us is of result, this the a patients. patients many to critical high even we reaching As with all pace and slow need that XXX of feeding the the coffee enrollment cabinet XXXX. We option September REACH, like of real daily

top will We step which bring are to closer FDA-approved the on delivering first-ever XXXX, track the in Quarter report us Fourth X of for to FSHD. therapy line

function and in daily motion upper extremity is is workspace about of and or of of I a arm endpoint range technology. just RSA change the ability a a critical RWS. This about study reachable trial the XD specifically me area to in using Now earlier. give these extremity limiting patients REACH is quantitative evaluates in shoulder perform some their their enabling this intended which score, both everybody U.S. to independence REACH. motion upper ex let and function to quick maintain geographies. baseline XX-week that is and for U.S. relative assessment sensor activities of or mobility of The surface be spoke measure that registration Preserving from reminder the primary the is to for

as measured or U.S. in be questionnaires at marker other thinking help begin MFI, secondary commercial this our like also preparing looking of quality fat whole health muscle is by care study, body pathology disease key part utilization of endpoints inform on reported As we'll as important will infiltration here an measures life the which that our launch for we payer strategy as well a MRI of and

we this results look top milestone, sharing in Fourth year. of is and pun There there, critical no We Quarter line the with next are pleased to to have forward reached. intended everyone

HPF cell for XXXX, sickle disease. the investigational treatment the clinical or to on hemoglobin on drug with potential cell move In of new now patients treatment me the let short for hold potential our lifted sickle application August of the So for FDA inducer, for fetal our pociredir, disease. oral pociredir

with productive revised and this interactions throughout targets the and collaborative patient aligned on population more criteria to pleased have exclusion hold, the inclusion, FDA study. Our severe a were a Ib Phase that for we're

I in X-month the that dose there also think changes it's scheme protocol-defined important no to duration. or were note treatment the escalation

by are the Each resume these are the to Phase scheduled of at pociredir. dose XX-milligram Ib enrollment to in hard of XX-milligram dose XX We working cohorts study the dose enroll patients. followed

and We U.S. ex-U.S. identifying in new that are as to sites current as excited well are the participate. reactivating sites

activating of Now given approval know, process. IRB and contracting as the many time, you new takes sites

I is and the minute think we'll a be with fact months position in our XX-milligram expect this to estimate of few just to so share about once to results XX belt, only of excited we when study. better far we Phase a and everyone. I the enrollment under the have in a talk take Ib would have to that about we're pociredir given want why a

pociredir translate So demonstrated improvement hemoglobin the a clinical increased into data that obtained to of meaningful fetal in severity. disease a that prior magnitude total hold we that could

remains believe oral pociredir Specifically, XX%. We of that potential after patients the baseline therapeutic priority for we we to only observed days HBF XX our XX differentiated with as and need up has total percentage inducer, point approximately progress. believe forward look cell option from in we hemoglobin or an increase to sickle disease. us, of fetal unmet that updates for significant living sickle cell a a to further treatment, provide hemoglobin fetal on a key in providing Addressing the

transformative Officer, these delivering therapies. I'll move in of a will since on financials. Alan Financial over that to the our mission Chief to it now update, invaluable great has will with advance next provide turn be who as growth. the our and to he our been update August, we team an into Alan, and phase clinical addition continue So joining of

over So to Alan, you.

Alan Musso

Alex. Thank you,

cash, position. during XXXX, of securities And Let September million. $XX.X marketable and our cash was me cash start cash net our with Third We Quarter, equivalents million. with the $XXX.X XX, ended burn

on And recent to collaboration Quarter and expect guidance into our a based Third XXXX, of from We prior Third projections, was financial $X.X Quarter our to $X.X That of of continue extend runway results for to update now our operate cash the mid-XXXX. In XXXX. we million our revenue An compares from position. operating strong million. current XXXX. the

including of Third advancement with of the September $X.X Our due during for were completion of enrollment Quarter million for research our Quarter million million of and trial, primarily development of III Phase $XX.X compared to $XX.X XXXX. The the costs expenses Third increase of associated XXXX. was the increased to XXXX the REACH

expenses increase million administrative Third to $X.X million and due general $X.X was facilities, primarily Quarter for Third services Our XXXX. The to as Quarter compared were of professional and costs. million of the $XX increased the of software XXXX for

for XXXX, it $XX.X loss and to that the Third was for million back with Alex. Third compares net over and $XX Quarter Quarter the me million XXXX, let to turn of of Our that,

Alexander Sapir

Alan, to have and the team. great again, Great. you on Thanks,

], up ahead Towanda [ So let's it go and for open questions.

Operator

Sachs. question Jenkins comes with from Goldman Instructions] Our [Operator line of the Corrine first

Corinne Jenkins

is This for Corinne. on Craig

in the And to pociredir. Ib study, see X And had Phase agent do forward current program? the to on show I the maybe get a you to do hope with based view what patient consider need maybe think reconsider the moving you're order I broader guys that, point what in to FDA from evaluating you to to the you specifically order population to a guess risk-benefit one? So of related

Unknown Executive

the Chief turn our over Medical Officer. Yes. let really And to Iain FDA. has Thanks for Fraser, conversations in me great. with It's that the been instrumental question, those Craig. Ian driving

I think best So to equipped probably answer that question. he's

Iain Fraser

Yes. for the Thanks, Alex, and thanks question.

As patients earlier, and XX cohort, a to studied days. the we've only X mentioned which at that was longest dose duration Alex only essentially the XX-milligram partial date, of

sickle to extend the we of do most think out And will how at I So the hemoglobin. Obviously, X percentage I at weeks the we're which to much think convincing of seeing to treatment reach. dose think be HBF the entire halfway was range those fetal we period, high depends HBF it and the end patient where high literature XX%, significantly relatively cell dosing baseline how reach. we and your of of just the you hemoglobin demonstrate total range XX% the into with X starts you points field out individuals the each trying this that hemoglobin, symptoms reached where by to XX least of once as months about the XX-milligram full higher were fetal impact and across at there started X that out duration we is enter rise what to in high disease. That's different rises the as baseline you up get around believe XX%, I backed do that cohort with and there the

earlier expect we even That's believe that's the dose. shooting. milligrams, to next volunteer we've XX previously, dose are that outperform And we on even we which So as intent. milligrams our is around that, go as data, articulated be that up healthy efficacy there further incremental our XX from gained to we the the towards target based will and XX-milligram fully

of will So in that for cohort. that's we as be the data dose be that we the region the HBF we that's this transformative, total we'll think start what expect that to significantly directionality looking and

Alexander Sapir

Craig, Yes. this And then, is Alex.

to like specifically do to your we just we feel to what show FDA population today. Let sort expand the answer patient of me question where beyond about are the need you

number I a everything very albeit benefit small shown looks right perspective. some in of risk think And the from -- obviously, patients. benefit, the we've at FDA a now,

spoke very if expand to this able armed the that population. more And information there, able much take we from of we Iain with believe can back over discussion thoughtful have that what what the be long we to and to will a is So believe about, how run, to show drug we're we'll patient that data achieve patients. sort benefit FDA

and X that level dosing And XX months what a potentially talk for the community the what's that think of specifically. blue very the up fetal they're able couple hemoglobin birds. Vertex the to to there's to I think CRISPR we compound in with well we've to if -- be with the of as get sickle lot with important -- based look past levels. XX-milligram may show prior that And with believe at as been able on up of therapy the milligram, whole, recently [ of that do cell is the weeks of very, the I you the for cell that's to X to dosing to we patients similar been and exciting months with able believe and ] get over that I gene we think remember XXs

Operator

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

Dae Gon Ha

Beazer on This ] Ha. Go [ Dae is for

not that company? in you're drug it going been the X some prior trial a maybe but refine about we feel in conference, has at And challenging on they Just exactly what then presentation the on color be questions it follow-up How have or just at couple to some clinical and that point. like about a To sure that participate somewhere some not the is on Can data to pociredir, about Poferator. for this that? safety? trial more comfortable can are going the is they're or more sites? having of between, know if to share you the how of a hold to midyear a And or year-end you're

Alexander Sapir

Thanks. great. that's Yes,

I then miss please to in if Let me and start feel Iain, anything. free jump

quick a in So a hole. Phase prior we initiation study as as study. Ib of yes, the reminder, it's so the had was all-comer just an And just reminder, to the sites that

is of need to as were the defined number criteria. our U.S. a double as our U.S. they double sites, So of the the we of well. all to -- the simply plans, to sites the specifically inclusion/exclusion we we because outside in And specifically as feel have look number a specific, and greater of is plan number sites, well go to that narrowly U.S. more to U.S. in

will in difficult trial it more have enroll So we the past. a to be than

this until an we've what of very to that that's XX-milligram I I as what I'd a inducer get to think XX. really study. And exciting hearing oral patients we exciting well. I at physicians in I the the think sites think provide HBF And of to as total fetal like up also Iain physicians, that's to for that this well. But and think levels hemoglobin exciting patients I that's benefits it's think really right? We've dosing is the -- this the show can physicians I dose, that can weeks able comment been hearing going for participate been I think to on are from as been X and with what for drug patients, the now from we've think

in terms that, up, been of now, we've have I has able engaging centers. centers stood show it. focus when we we that have think be I to already really think with approved got right IRBs we'll Once of those couple a on the think our

We drug. that around we're the is the And lot have very also a in. very, to in those about X cell the that participating sickle with start is now sites of educate sort center community study interesting and really community site to receiving ready community doing this

really that's been So our focus.

guidance is in that ], be I indication. point, Benaron of The if I think would know thing great that to would certainty I any this only I indication think that at sort [ or you was incorrect. give with

couple So under us our us I a months, of couple get patient give enrollment belt. a let months of think of

in said position I've at specific that past. think that be the I about. much point, I guidance provide comfortable to a As in more And feel we'll more better

So anything [ in around centers you ]? the else think

Iain Fraser

of it was color Thanks, little around And the types all-comer study. alluded can earlier, bit Alex. Yes. as sites an provide Alex I to themselves. a of

patients sites to And care cell to lot hold the number great pediatric because were patients so of that those disease as older as transfer go of the because have don't is A they we partners way sites. administered. to. at sickle of adulthood pediatric essentially into their a started sites that out on they And

the patients severe we a had less those originally. Those number less previous go somewhat have is so finding experienced sites might And the they and that given sites experienced. the disease, younger a number or study the out we that need don't treatment they've of those are severity level within quite of experience we're we protocol. overall, of obviously treatment for what feel that And impact as just of duration that

are that but some sites them of are those patients. clearly have so And do still not continuing, those

net these at then that's that exclusively been that and impacted are obviously broadened many do a and have more experience, X our patients. sites severely treat So we've also number of adults looking of

them the into as so bringing And are we fold well.

We've So we're investigators. of noticed we're the casting at net. those patient sites. subtleties different a with wide And the of meeting populations lots

Operator

SVB from comes Inc. of line Schwartz the Our Joseph next with question

Joseph Schwartz

baseline at those in you starting of more for more if for XX% from It's severity? with will generate with who levels hemoglobin patients I referenced clinical influence Joe the treated increase negotiated be reach Leerink could a compared I'm FDA sufficient generating new about they're pociredir inclusion Partners. in the disease earlier think severe which you lower be think Phase were And in to I the disease? do range opportunity if [ how an is in to Schwartz could will the patients still future wondering if previously? that criteria benefit ] the much wondering was out of as Ib for cohorts reflected you able future talk pociredir? the some guess have you profile Which will cohorts an the to

Alexander Sapir

Great Yes. question.

answer I over let that, think me Iain. that to turn to X

Iain Fraser

Yes, absolutely. given interesting will I a as it's to, X And lot thought out. see think rolls that of the it to it data we've and be

all patients in HBF that consistent pretty about we've hemoglobin. a those. in of the in increase fetal think of all comments is full months of Ib make rate I initial far across study as trajectory them, the a have And X don't I'd thus XX One of while in is it. baseline the of we pretty couple treated data high

steeper seem to low it the rate though HBF the baseline appear higher responding was ones and higher at a well. it be as So just as didn't

first So point. that's the

HU advantages The to mechanism being but Obviously, which response, second point to erythropoiesis-type demonstrate susceptible as it's from the you description patients, the impacted that be or stress distinct the this whether append action some a severely cells to about is, marrow it. depletion that fatigue I of discussion into stem want think we to or through operating some of of mechanism whatever not might has we'll is. There's respect. more that in HEU need in move

through patients transcription the patient that gene we'll that's So and severely mechanism in differentiated we'll have in but more be acting are think get alterations in successful likely demonstrate and that I in impacted of population, clinic, to action the our expectation. to the

Joseph Schwartz

develop pociredir are inclusion And failure that now? X,XXX to the just us wondering estimate criteria United you then walk the you in sites going calculus screen in expect confident Interesting. your employed And engage And could to do for be for the you covers XX,XXX to I'm what the States. sickle saltation as just that with estimate patients you forward? you how rate through the

Alexander Sapir

ask got bit maybe me to to I'll in rate, But let it of number. yes, that answer we terms that. Joe, to failure screen the Yes, relates as a how of Iain give color,

I are co-pays think to inclusion/exclusion the for too tried advanced in failed reason, or fact their don't crizanlizumab looking these have are they people access have in the looking of what's don't or because criteria. advanced and simple the high. insurance Boxelador These or proper that therapies, the whatever some at have at remember important either of that therapies to on

didn't both have some cover Box is know to We have for And XX,XXX during always year, the that we know a about products with actually there numbers. to XX% you patient let rate it's VOX, didn't maybe about VOX, it and give to you don't could each through XXXX, patients numbers from didn't of sort our the of XXXX, issues. What is because we that little infusions those discontinuation first rates bit start. an access does XX% high-level you prescriptions. simply So center. about the to insurance have never at access infusion a or those prescriptions assumption converted proper assume me CRIZ could -- But We you in that that year. when generated probably sold that those both have require payer-related in specific higher discontinuation first crizolizumab due drug similar know also of launched

to the that math those but So that in of severity sort that XXXX. where you to prescriptions of We of patient is may all include fact overestimated XX,XXX around have through underestimate be of the that be Where not the all is prescriptions doesn't meet XX patients. it any number doing fact XXXX. patients number that may sort criteria, only gets an

taking of how population. all the sort arrived that at patient sort the So to U.S. that patients total inclusion/exclusion XX,XXX want numbers of of about we second X% you consideration that's meet those address criteria, represents which into XX% the question? XXX,XXX in to your XX of to Did

Iain Fraser

Yes.

is this So Ian.

We of recruited projected you this fail point. a screen that study, haven't for patients, phase fail was rate XX%. the the screen can rate that I where little a initial tell at we XX over

So be high population of a is moment carrying at more rate likely that And point. it was to that we're patient though severe. sort screen relatively at forward even the fail

We patient sites in have to that expect more experience with the help and fashion. with selection with the the to sites able so of education protocol the and around be

Operator

with from Piper comes Edward the question next Our Tenthoff of Sandler. line

Edward Tenthoff

a poor little So question ever no one losmapimod. on asked

you're project kind Just that. next coming So your head us it, the are the and some yourself, what's sort of doing? X in the half I launching -- on overseas? update back is of of here, where be that And as the to between With on partnering commercial I'll keeping partnering year, it calculus guess prep what data out it. you maybe it with of all ask the the of it

Alexander Sapir

a you. so I on asking Thanks much And question. the losmapimod. absolutely. question Thank you appreciate for Yes,

So Yes.

neuromuscular ourselves XXXX as launch that's -- ] plan We would approval would to line a to results NDA So quick have for in just we ability advocacy we in launch world. reminder, fact help be infrastructure, we the probably have probably while Commercial that well Myostatin year, it sort behind be clear at excited with that the X the this excellent FSHD. to early this be a options successful beginning They're from behind been number commercial that in we U.S. first think ex tremendous help file that treat have these end very, to of we this organization able commercial have the same believe us. XXXX. We've and would hire, as standpoint, the the time, in Society. And drug ex in inhibitor. of a Roche's the Officer. And FSHD key X the top number fairly of doing we market. of process with of [ an of the with It's patient to closest in partners would drug Chief no about modest starting partner a the we of quite opportunity then sometime X are ourselves believe the Quarter. [ Fourth looking working that to patients X small really at concentrated U.S. U.S., U.S. standing is relationship with well specialists that which up as kind so the patients of successfully organized very that to will for a sort and and sort of an our and with begin an can fairly do of It's product, fairly reasons the which be sometime U.S. a a we're around ability that years patients X ] currently collaboratively next that's that I us to with there we the treatment this the year, years, our do is a very

Edward Tenthoff

helpful. as That's attention more the garner will we And into it get Year. do New think I super

for thanks So the update.

Operator

Matthew with from question Company. Our Oppenheimer line of the & Biegler comes next

Matthew Biegler

specifically, the bandwagon maybe workspace out the reachable the hit what about what X. endpoint also page ask forward or FSHD wanted thoughts [ narrowly now end biomarker couple if Like jump path last misses you here. saw taking sufficient, maybe of seen kind on ] your I the will you might guess, the I think a Sarepta's a study over What And to if REACH some be weeks. point? are different there? of doesn't I we've for playbook scenarios do and of

Iain Fraser

Yes. Thanks, Matt.

REACH, than to the for reachable are really with guess which muscle endpoints are MRI endpoints, II feasible just in in biomarker, dispersed the in however, evaluates we measure that evaluating workspace of secondary make. what clear, I clinical not contractile What biomarkers that DUCs fat. we different evaluated transcripto the in study So like be endpoint. not a trial was which still terms are And those which a accumulation considered normal that's of include fat be tissue in muscle number the a of to the Phase could looks is has do,

And study as showed an to that who of relative you've so II the also that Phase increase. reachable we showed for got placebos that workspace, the in the accumulation fat stabilization a

that's endpoint. important that, an secondary think we clearly So

of evaluation muscle so an conventional which have evaluating. also testing, We we're dynamometry, strength

that patient-reported but some patient reflect specific related FSHD also on questions have both itself. We of to change, outcomes some global impression

context. you in direction are those primary think the having of endpoint. But that to secondaries narrowly, what be secondaries I happens there be that And will of favorable articulate think over a extraordinarily I if placebo misses irrespective consider to trending losmapimod number the so important primary persuasive towards in helpful of a and will those all as

Alexander Sapir

looks The I pociredir benefit back add is regarding something thing would from Alex. at Ian only to said earlier is this to everything Matthew, and going what how perspective. I risk a said other the And Yes. FDA

is safety think we over also this very, been X,XXX to a I important our note the drug. that we safe our has will database. have patients when very shown FDA, in be NDA what's to patient file with And

And considers whether that that for will efficacy as decides or that, FDA which approve to think for really not piece in patients drug the had [indiscernible]. of of safety a the look is so data this they I sort totality and it at important and information

Operator

with Gon the from a follow-up Stifel. have Dae of Ha We line

Dae Gon Ha

more X from Just us. question

it regional about can test, For powering and think mobility and workspace? again went assumptions the that the in of REACH? powering what IMbark assumptions a read-through should the context for the the FSHD, for ] you the trial we of for into how it's us remind that Given failure REACH for recent [ it

Iain Fraser

Yes.

the So REACH that may an the arm. treatment minus placebo took II Phase observed was was REACH the variability ReDUXX from applied also in losmapimod type even study powered reflection prevalent, the we for study. each of relative to study [ type assess them XXX study, reachable you we was X the that it effect measurement And or type powered and data be XX the the a X. the then and Xs the of in is and on [ workspace, total XXX originally essentially, REACH the most an some from ] XX-patient and are ] based XX% The study the that. on baseline with enrollment would calculations enrolled though And power what patients epidemiology on from type we of type overwhelmingly change Type that only study. The REACH cohort expectation of on only Xs and particular XX ReDUXX the of the in that's type was recall powered to X. X a them study, so X would the FSHD versus in type so be of X enrolling

the of XX% expectation ]. of was XXX using deviation and that or gave study standard us from and of a powered [ XX% ReDUX REACH an magnitude AR the patients type change the study, on power the in So

to on thought rate it patients the screen ended the down, going REACH order and dropped enrolling As study fail up turns out, when a be more to of what number in based we screening off we rate for the those fail XXX patients. cut was screen we reach

the And that enrollment XXX there of so FSHD Xs. of type XXX XX were at was total and XXX of day, end the the instead

So we now have XXX FSHD X.

the versus [ from the X XX% assumptions that using up XXX bumps So original powering same into XXX, Reduc the around ]. power over so

reflection screening a patients fail gotten inadvertent That an the was that the But screen and dropped fact that better have sites enrollment. was of down. at the wasn't sites the until over So the intention. enthusiasm the at our rate

where actual enrollment in based REACH. now And we that's on so stand our

Operator

concludes CEO, and to answer the over this portion the call call. now gentlemen, and Ladies to remarks. like Alex closing question turn I of Fulcrum's the back for would

Alexander Sapir

Thanks, [ ]. Towanda

of quickly transformative to remain, deeply up, to bringing and treating to we cause rare therapies committed patients. wrap just defined the have, these diseases So always as we root genetically

fellow the do, teammates to my the I conclude finally today's we just advance importantly, and physicians to and sincere who participate their we Without trials the most to stay our we extend trials in I'd and to our call, meeting Before like the with as as able appreciation to Fulcrum joined work to like clinical would gratitude call my always our everyone, safe them, Please achieve a as Thanks be morning. joined not this again, who goals families. company. patients healthy. well to clinical who and as

Operator

call. today's and you this conference Ladies Thank participation. concludes gentlemen, for your

now may You disconnect.

Fulcrum Therapeutics (FULC) 7 Nov 232023 Q3 Earnings call transcript